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gfp, KZIA is about as speculative as it gets. All the caveats about nano biotechs apply PLUS the caution that this is an Australian biotech.
My two largest positions in terms of number of shares are PRVB and MGTA. I'm underwater on both.
August through the first half of October are traditionally the worst months of the year for biotechs and the Market in general. KZIA is presenting at SNO in November in Austin. Then comes ASH and SABC in December. Those conferences tend to buoy the biotech sector.
KZIA market cap is around $75 millionUS.
There are 94 million shares out. 64 million are owned by Aussies. The other 30 million are in ADR's through Bank Mellon (3 million ADR's). The Aussie shares can be converted to ADR's (at the ratio of 10-to-1), if the demand in the U.S. is great enough.
Good luck.
Bladerunner
CBD Progress... Cardiol interesting move.
Some updates and other mixed info; https://www.barchart.com/stocks/quotes/CRDL.TO/news
Did u see this one; https://simplywall.st/stocks/ca/pharmaceuticals-biotech/tsx-crdl/cardiol-therapeutics-shares ?
Interesting piece I cut from above link;
Is Cardiol Therapeutics undervalued compared to its fair value and its price relative to the market?
Undervalued compared to fair value
Share Price vs. Fair Value
88.1%
Undervalued
Current Price
CA$2.94
Fair Value
CA$24.72......!!! Any thougths on the calculation?
Blade, Thanks, that looks like an extremely comprehensive report on KZIA.
I started a position yesterday, though under my new disciplined approach there are strict position limits on individual stocks, especially biotechs. I'm trying to prevent a repeat of earlier misadventures :o)
I can make up for the position limits somewhat by owning more stocks, though there is also a sector allocation limit. So many 'rules', but so far it's working great, and I no longer worry (much) or lose sleep.
Everything you ever wanted to know about KZIA (by Edison Research, paid for by KZIA). Will be presenting at SNO in November in Austin, TX. NOT a virtual conference.
https://kza.irmau.com/irm/PDF/4b39cb93-1de2-4542-92b9-07098509c6fa/EdisonResearchKaziaTherapeuticsBringingavetteddrugclasstothebrain
Bladerunner
The bull case for MGTA (from "Seeking Alpha") I agree with the article. One of my two largest positions. Well underwater. Got in too early, before discontinuation of its lead molecule. Comments welcome.
The Low Down On Magenta Therapeutics
Aug. 31, 2020 7:01 AM ET
|
About: Magenta Therapeutics, Inc. (MGTA)
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Summary
Busted IPO Magenta Therapeutics, Inc. executed a late-June secondary at $8, shortly after announcing it was abandoning a Phase 2 trial due to enrollment challenges.
The company will reposition resources to its stem cell mobilization and conditioning programs.
A high-risk investment with multi-bagger potential and recent insider buying, Magenta deserved further investigation.
Today, we take an in-depth look at a Busted IPO that recently retrenched and raised additional capital after abandoning a mid-stage trial. A full investment analysis follows below.
Company Overview:
Magenta Therapeutics, Inc. (MGTA) is a Cambridge, Massachusetts, based early clinical-stage biotechnology concern focused on the development of compounds that upgrade the current immune reset process in patients with blood cancers, as well as genetic and autoimmune diseases. It has recently reprioritized its assets, leaving it with only one ongoing trial, a Phase 2 study involving a de-prioritized candidate with upcoming multiple Phase 2 trials for its mobilization agent. Magenta was founded in 2015 and went public in 2018, raising net proceeds of $89.9 million at $15 a share. The stock has an approximate market cap of $325 million and trades for just under $7.00 a share currently.
Approach:
Resetting the immune system involves the removal of disease-causing cells and the transplantation of healthy ones to rebuild the immune system. Even though stem cell transplants – the largest immune reset market – have demonstrated curative power, only a fraction of eligible patients for these procedures undergo them, owing to the risk of adverse events, such as infection, infertility, secondary cancers and death. For example, nearly two-thirds of eligible acute myeloid leukemia patients do not receive a transplant despite it being the standard of care.
The immune reset process involves three steps. The first step involves the collection of stem cells from the patient or from healthy donors (known as mobilization); the second step is the removal of disease-causing cells (conditioning); followed by the infusion of the healthy stem cells into the patient (transplantation or cell therapy).
Source: Company Presentation
Each stage of immune reset is riddled with shortcomings. Mobilization usually requires multiple invasive bone marrow harvests requiring several days of drug injections that chase the stem cells from the bone barrow into the bloodstream, where they are then collected through a process called apheresis. The current standard of care therapy is granulocyte colony-stimulating factor (G-CSF), which requires at least five days of injections and is associated with significant side effects, including bone pain that often requires painkillers. Furthermore, patients with autoimmune diseases or sickle cell disease can have severe side effects with G-CSF, while mobilization failure rates for autologous transplants (harvested from the patient) can run as high as 40%.
The conditioning phase involves the use of systemic, toxic chemotherapy and/or radiation, which is akin to carpet bombing the patient, killing not only targeted stem cells, immune cells, and diseased cells but also healthy cells while damaging DNA. Since much chemotherapy is a derivative of mustard gas, nearly all transplant patients experience complications, including severe infections, organ failure, and death. Conditioning toxicity is responsible for ~35% of mortality following allogenic (donor) transplants.
An ideal transplantation procedure involves healthy stem cells infused into the patient through the bloodstream that travel to the bone marrow where they start producing healthy blood and immune cells. However, there are many instances where the stem cells are either rejected by the patients’ remaining immune system or fail to engraft in the bone marrow, resulting in the need for additional transplants, the outcomes of which are extremely poor.
Magenta’s development platform is designed to produce more precise remedies that reduce the threat inherent in current immune reset measures, making them more accessible to eligible patient populations. The company is developing three classes of medicine: a conditioner that removes the disease-causing cells before transplant, a mobilizing medicine that collects stem cells, and a cell therapy with a high stem-cell dose to rebuild the patient’s immune system. Magenta’s assets are being developed for use either individually or in combination with each other, allowing for bespoke patient programs.
Pipeline:
Source: Company Presentation
MGTA-145. The company’s most advanced stem cell mobilization candidate is MGTA-145, a CXCR2 agonist being accessed in combination with a CXCR4 antagonist (plerixafor) as a first-line agent. In a Phase 1 study involving healthy subjects, MGTA-145 was safe and well-tolerated individually and in combination with plerixafor. Additionally, the combination demonstrated rapid, single-day mobilization, collecting (what was deemed) a sufficient number of stem cells. Magenta intends to initiate multiple Phase 2 trials in both allogenic and autologous transplant settings with the hope of producing data in 2020. One of those studies is being conducted in concert with the National Marrow Donor Program/Be The Match to evaluate MGTA-145 for single-day mobilization of healthy donor stem cells for patients undergoing allogeneic transplant. MGTA-145 received orphan drug designation from the FDA in May 2020.
Source: Company Presentation
If successful, the market for MGTA-145 is significant with ~55,000 transplants performed in the U.S. and EU annually using mobilized peripheral blood from either the allogenic or autologous setting. The compound will enter Phase 2 development by year-end.
MGTA-117. Magenta’s most advanced conditioning agent is MGTA-117, an antibody-drug conjugate which is designed to selectively remove disease-causing cells and, as such, is expected to be far less toxic than chemotherapy and/or radiation. MGTA-117 is still in the pre-clinic, undergoing IND-enabling studies. A single dose of a CD117-ADC selectively depleted hematopoietic stem cells in nonhuman primates with a therapeutic index of 30 – two to six is typical for approved ADCs at that stage of development. Magenta expects MGTA-117 to produce clinical data in 2021.
Source: Company Presentation
On May 6, 2020, Magenta announced a research and clinical collaboration with AVROBIO, Inc. (AVRO) to evaluate MGTA-117 for conditioning patients before they receive AVROBIO’s lentiviral gene therapy candidates. That news was followed by an alliance with Beam Therapeutics Inc. (BEAM) to study MGTA-117’s utility conditioning patients with sickle cell disease and beta-thalassemia receiving Beam’s base editing therapies on June 15th.
Source: Company Presentation
CD45-ADC. Magenta’s other program in its conditioning portfolio is CD45-ADC, which has demonstrated the ability in the preclinic to remove disease-causing reactive T cells in three models of autoimmune disease: multiple sclerosis, systemic sclerosis and inflammatory arthritis. Magenta has identified a lead antibody for this program, and IND-enabling work continues.
MGTA-456. The company’s most advanced candidate in any class was MGTA-456, an allogenic cell therapy designed to provide a high dose of stem cells that are well matched to the patient to safely rebuild the immune and blood system. However, the program received a setback when Magenta announced it was discontinuing enrollment in a Phase 2 trial for patients with inherited metabolic diseases due to enrollment challenges amplified by the COVID-19 pandemic as well as “a growing understanding in the transplant field of the current challenges of allogenic stem cell transplant in patients with… inherited metabolic diseases; and feedback from the FDA…” This negative development came after the FDA had granted MGTA-456 Regenerative Medicine Advanced Therapy designation in September 2019 and is part of a reprioritization of resources away from cell therapy and toward Magenta’s stem cell mobilization and conditioning programs. The company has completed enrollment in a Phase 2 trial evaluating MGTA-456 in patients with blood cancers. No timeline for data has been put forth.
Balance Sheet & Analyst Commentary:
With expenses expected to increase substantially in connection with its preclinical and clinical activities, Magenta executed a secondary offering in late June 2020, raising net proceeds of $64.9 million at $8 per share. The company ended the second quarter with just over $175 million in cash and marketable securities on its balance sheet, providing a cash runway through 2022.
The potential of its therapies has developed a small yet supportive following on the Street, with all four analysts rating shares of MGTA a buy. Their median twelve-month price target is north of $17.00 a share. Wedbush was the last analyst firm to 'chime in' on Magenta. It reiterated a buy rating and $22 price target on MGTA on August 7th.
Also loyal to the cause is board member Bruce Booth, who represents the interests of Atlas Venture Funds. He purchased 1.25 million shares on the June secondary, upping Atlas’ position to north of 4.1 million shares.
Verdict:
Magenta, at this early stage, is basically a seven-dollar option. With the curative promise of stem cell transplantation, it is a very sexy line of therapy and significant upgrades to current mobilization and conditioning approaches could result in blockbuster therapies. However, with the discontinuation of the MGTA-456 Phase 2 study for inherited metabolic diseases, the only Magenta ongoing clinical study is the deprioritized Phase 2 MGTA-456 trial for blood cancers with no scheduled data release. The key data will come from the yet-to-be-started multiple Phase 2 studies for MGTA-145 – data from which is expected this year. MGTA-117, although auspicious to date, has yet to enter the clinic.
However, with half its market cap in cash, and the potential to be multi-bagger; this names set up well for a covered call strategy which I have used to establish a watch item holding in this name.
Bret Jensen is the Founder of and authors articles for the Biotech Forum, Busted IPO Forum, and Insiders Forum
Author's note: I present and update my best small-cap Busted IPO stock ideas only to subscribers of my exclusive marketplace, The Busted IPO Forum. Join our community today by clicking on our logo below!
Disclosure: I am/we are long MGTA. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
Bladerunner
$HALB 0.007
Here we go!!!!!
https://www.accesswire.com/604004/Halberd-Selects-Arizona-State-University-ASU-to-Develop-Its-Patented-COVID-19-TreatmentsHalberd Selects Arizona State University (ASU) to Develop Its Patented COVID-19 Treatment(s)
Monday, August 31, 2020 8:00 AM
JACKSON CENTER, PA / ACCESSWIRE / August 31, 2020 / Halberd Corporation (OTC PINK:HALB) today announced the signing of a development agreement with Arizona State University (ASU) to conduct sponsored research to develop Halberd's Covid-19 treatments. The work will focus on developing antibodies in support of Halberd's two issued patents and three Covid-19 related provisional patent applications for extracorporeal removal of the virus' antigens from a patient's blood. It will also focus on neutralizing the effects of antigens and development of a potential therapeutic medication.
William A. Hartman, Chairman, President and CEO of Halberd Corporation stated, "ASU has demonstrated its expertise in biomedical research, including in the coronavirus arena through its recent development of the first Covid-19 saliva-based diagnostic test."
>> My cancer docs like it <<
You don't have cancer, do you? Yikes I hope not. It seems like everyone is getting cancer these days. Apparently the average life expectancy in the US is now falling for the first time in history, mainly from cancer.
I read that statistically, 1 in 20 people died from cancer in the 1950s, by the 1970s it was 1 in 10, and today it's 1 in 3 and heading for 1 in 2. I spoke with some cancer researchers at Jefferson Hospital in Phila and they said they see so many new types of cancer that they don't even have names for them all. One group was studying childhood cancers and another breast cancer. These researchers were beyond alarmed, they were completely at a loss to explain what is going on.
gfp,
I'm in KZIA in a big way. Got in around $4, so doing fine. Potential to be a 20X-bagger. Virtually unknown on The Street. A miniscule 7.5 million shares outstanding. A possible presentation at SNO (Society of Neuro-Oncology) in Novemeber. All the caveats apply including it being an Australian biotech.(ugh) My cancer docs like it. The trials are being run at Dana-Farber and MSK and St. Judes. Drug is a small molecule developed by Genentech. Recently got orphan drug designation. Also, fast track. If Phase II data is good, KZIA will look to get accelerated approval with priority review.
Bladerunner
Blade, Just curious if you are in KZIA? Even after the recent move, the market cap is still only $50 mil. Looks interesting.
Here's an overview from the CEO -
KZIA get ODD (Orphan Drug Designation) for brain cancer drug
US FDA Awards Orphan Drug Designation (ODD) To Paxalisib For Malignant Glioma, Including DIPG
PR Newswire PR Newswire•August 23, 2020
SYDNEY, Aug. 24, 2020 /PRNewswire/ -- Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of malignant glioma, which includes Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly aggressive childhood brain cancer.
Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare ('orphan') diseases, generally defined as those which affect less than 200,000 cases per annum in the United States
ODD can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), extending the effective life of a commercial product. It also provides opportunities for grant funding, protocol assistance, and financial benefits, such as a waiver of New Drug Application fees, and tax credits
Receipt of ODD follows award of Rare Pediatric Disease Designation (RPDD) for DIPG on 7 August 2020
Kazia CEO, Dr James Garner, commented, "Taken together, RPDD and ODD provide a powerful suite of incentives, opportunities, and protections for the development of paxalisib in DIPG. We look forward to seeing initial data from the ongoing phase I study in DIPG at St Jude Children's Research Hospital during the second half of calendar 2020. In parallel, we are working closely with collaborators, advisors, and researchers to determine the best path forward for paxalisib in this devastating disease."
He added, "This award of ODD concludes a program of regulatory optimisation that Kazia has initiated for paxalisib over the past six months. As we orient paxalisib towards commercialization, these special designations from FDA will allow us to move forward in the swiftest and most effective way possible."
Orphan Drug Designation
ODD exists to recognise the development of a drug for a rare disease, which may affect adults or children. ODD provides an additional period of 7.5 years data exclusivity (for a paediatric disease), which allows companies to better defend their products against competition. It also results in a waiver by FDA of fees for a marketing application, under the Prescription Drug User Fees Act (PDUFA fees), which are just under US$ 3 million in FY2020. In addition, drugs with ODD may be eligible for orphan grants by FDA.
Kazia previously received ODD for paxalisib in glioblastoma in February 2018.
Summary of Paxalisib Regulatory Status
Glioblastoma
Most common and most aggressive adult brain cancer
DIPG
Highly aggressive childhood brain cancer
Orphan Designation
February 2018
August 2020
Fast Track Designation
August 2020
Rare Pediatric Disease Designation
n/a
August 2020
Next Steps
Kazia expects to present further data from its ongoing phase II study of paxalisib in glioblastoma at the Society for Neuro-Oncology (SNO) Annual Meeting in November 2020.
Initial efficacy data from the ongoing phase I study of paxalisib in DIPG at St Jude Children's Research Hospital is expected during 2H CY2020. Precise timing remains uncertain due to pandemic-related disruption in conference schedules, but Kazia expects to provide an update to investors at the earliest opportunity.
Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.
SOURCE Kazia Therapeutics Ltd
Bladerunner
>>> Antibody Drugs Aim to Fill Covid-19 Treatment Gap
8-23-20
Wall Street Journal
by Joseph Walker
https://www.msn.com/en-us/health/medical/antibody-drugs-aim-to-fill-covid-19-treatment-gap/ar-BB18h9Rz?li=BBnb7Kz&ocid=mailsignout
A kind of drug that takes a page from a body’s natural antibody defenses may be able to give many people early protection against the coronavirus, ahead of vaccines.
Few treatments so far have been shown to be effective against Covid-19, and even then, only in hospitalized patients. Potential vaccines, meanwhile, are probably months away from completing testing and becoming available to the general public.
Researchers and health officials express hope certain drugs, known as monoclonal antibodies and in testing by companies including Regeneron Pharmaceuticals Inc. and Eli Lilly & Co., will fill the breach.
Get news and analysis on politics, policy, national security and more, delivered right to your inbox
“There is a need for safe therapies to be given early in disease and monoclonal antibodies are paramount among them,” said National Institute of Allergy and Infectious Diseases Director Anthony Fauci. “I’m very anxious to see what the results are.”
The drugs, which are injected intravenously or with a short needle, have the potential to work soon after someone is infected and still feeling only slightly sick, stopping the virus in its tracks before the seriously afflicted would need to be hospitalized.
The monoclonal antibodies might also prevent infection in healthy people, though likely only for about a month. If the promise bears out during testing, the drugs could stand in as a type of temporary vaccine for people at high risk of infection, such as nursing-home residents and possibly health-care workers.
“Today if you get Covid, you will have a wave of fear of biblical proportions,” said Myron Cohen, an infectious-disease specialist at the University of North Carolina who is overseeing antibody studies for the NIAID.
“If I can give you a shot and guarantee you I stop the progression of disease, the world changes,” he added.
Among the companies planning to launch antibody studies in people this year are Vir Biotechnology Inc. and partner GlaxoSmithKline PLC, and privately held Adagio Therapeutics Inc. AstraZeneca PLC this month launched a Phase 1 study of an antibody drug it licensed from Vanderbilt University.
Covid-19 clinical trials are now under way for 10 new monoclonal antibodies, known as mAbs in industry jargon, according to the Antibody Society, a professional association of researchers.
The most advanced are already in mid- and late-stage studies in newly diagnosed as well as hospitalized patients, and in people who haven’t yet been infected. If they clear testing, the drugs might be available as soon as early in the fourth quarter, according to Geoffrey Porges, an SVB Leerink LLC analyst.
Early signs are preliminary but positive. Regeneron’s antibody drug both cleared and prevented coronavirus infections in a study in monkeys and hamsters.
Another reason for optimism, infectious disease experts say, is that the antibody drugs developed by Regeneron and the National Institutes of Health were shown to be effective in the Ebola virus.
Yet some researchers who expect antibody drugs to work in clinical trials are skeptical how much real-world impact they would then have.
Doctors may be reluctant to prescribe a brand-new, expensive medication to patients early on in their disease when the drug is most likely to be effective, said Florian Krammer, a microbiologist at the Icahn School of Medicine at Mount Sinai in New York.
“The problem is putting it into practice and when are you going to use them?” said Dr. Krammer. “Are you going to give it to someone who comes in who’s only a little symptomatic? Probably not.”
The drugs would likely be used preventively by those at high risk of infection or severe complications from the disease, such as nursing-home residents and workers, or people exposed to a household member who tested positive for the virus.
Regeneron and Eli Lilly say that even if the drugs work, there won’t be enough to treat everyone because there aren’t enough manufacturing plants able to do the high-tech manufacturing that mAbs require.
Finite supplies will mean that government officials, nonprofit organizations and drugmakers will likely have to come together to set guidelines for who gets the drugs, said Eli Lilly Chief Scientific Officer Dan Skovronsky.
“It’s not a question of cost or working harder or a desire to make it. It’s just the simple math, the number of manufacturing facilities in the world that are available,” he said. “And we can’t stop making cancer drugs or insulin or anything like that.”
Regeneron joined with rival Roche Holding AG this past week to more than triple the manufacturing capacity for its drug, but said the increase probably still won’t be enough to meet demand.
Monoclonal antibodies are human-made cells that latch onto proteins that drive disease.
Their discovery in the 1970s helped give birth to the modern biotechnology industry, and they have been turned into blockbuster treatments for cancer, vision loss and chronic inflammatory diseases.
Doctors and health officials are looking to the drugs to fill a big gap in Covid-19 treatment. The only drugs shown to be effective against the disease are for sick patients well along the course of the disease, not for patients in the early stages of infection.
Vaccines promise to protect people against infection, or at least reduce the severity of a case. The most advanced are just starting late-stage testing. And if they pass muster, there probably won’t be enough initial supplies for many people.
Regeneron and Lilly executives say the complexities of conducting studies during the pandemic make it difficult to confidently predict when they will have firm answers on which patients respond best to their drugs, if at all.
To quickly enroll patients, the companies have had to predict virus hot spots ahead of time and then work with overworked doctors who may be too overwhelmed to always focus on clinical trials.
“We’re a little bit like storm chasers, but we’re pandemic chasers,” said Regeneron Chief Executive Leonard Schleifer.
Delays getting coronavirus test results for potential study subjects are also complicating enrollment. The delays, of as much as 10 days, wind up disqualifying some patients from studies that require them to be enrolled within three or four days of testing positive.
“If you can’t test people rapidly, that precludes being able to do a study in recently diagnosed people,” said Lilly’s Dr. Skovronsky. “Improved diagnostics is part of the answer to improved therapeutics.”
In August, late-stage studies were launched evaluating Lilly’s lead antibody in hospitalized and nonhospitalized patients, and these could be completed before the end of the year, depending on how quickly patients are enrolled.
Regeneron expects to have the first data from its study of a monoclonal antibody in hospitalized and nonhospitalized patients by the end of September. The company could seek an emergency authorization based on the data if there is a strong indication the drug is blocking the virus.
<<<
KZIA up 43% in AH after receiving FTD from FDA for brain cancer drug.
Stock was $2.65 at end of April. Now at $8.75. (What % gain is that? 230%?)
US FDA Awards Fast Track Designation (FTD) to Paxalisib for Glioblastoma
PR Newswire PR Newswire•August 19, 2020
SYDNEY, Aug. 20, 2020 /PRNewswire/ -- Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of glioblastoma, the most common and most aggressive form of primary brain cancer.
Fast Track Designation (FTD) is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions
FTD provides Kazia with substantially enhanced access to FDA, including opportunities for face-to-face meetings and written consultation throughout the remaining development of paxalisib
Drugs with FTD are eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) submission, which may result in faster product approval
FTD also allows for 'rolling review', whereby Kazia may submit completed sections of the paxalisib NDA as they become available, rather than at the end of development
Kazia consequently plans to begin initial preparatory activities for NDA filing for paxalisib in CY2021
Kazia CEO, Dr James Garner, commented, "in awarding Fast Track Designation to paxalisib, FDA has recognised the drug's potential to meaningfully improve outcomes for patients with glioblastoma. This is a very powerful acknowledgement. The opportunities that Fast Track Designation creates, as we move towards an NDA filing, are of great value and have the potential to substantially accelerate the commercialisation of paxalisib. In particular, the 'rolling review' process enables Kazia to complete and submit substantial sections of our NDA filing in advance, saving time and reducing risk for the product. We look forward to working closely with FDA as we move into the final stage of development for paxalisib."
The specific indication for which FTD has been approved is "for the treatment of patients with newly diagnosed glioblastoma with unmethylated O6-Methylguaninemethyltransferase (MGMT) promotor status who have completed initial radiation with concomitant temozolomide." This language precisely reflects the patient population studied in the ongoing phase II study, and is the primary proposed population for the GBM AGILE pivotal study, and is the intended indication at commercial launch.
Kazia announced on 7 August 2020 that FDA had granted paxalisib Rare Pediatric Disease Designation (RPDD) for DIPG, an aggressive childhood brain cancer. For clarity, this granting of FTD for glioblastoma is not specifically connected to the prior granting of RPDD in DIPG.
Fast Track Designation
Introduced under the FDA Modernization Act (1997), Fast Track Designation (FTD) may be awarded by FDA to investigational drugs which treat a serious or life-threatening condition, and which fill an unmet medical need. FDA notes that 'the purpose [of the Fast Track program] is to get important new drugs to the patient earlier.'[1] FTD must be requested by the sponsor company and must be accompanied by a detailed review of both preclinical and clinical data. To be awarded FTD, drugs must generally be able to show some potential advantage over existing therapies, either in terms of safety or efficacy.
The key benefits of FTD comprise enhanced access to FDA, with regular and more frequent opportunities for consultation and discussion. In addition, drugs with FTD may be eligible for Accelerated Approval, in which a new medicine is approved prior to the availability of definitive data, and Priority Review, in which the standard 12-month review process is reduced to six months. Drugs with FTD may also enter a 'rolling review' of their NDA submission, in which sections are submitted and reviewed as they become available, substantially expediting the approval process.
Next Steps
Kazia completed recruitment to its phase II clinical trial of paxalisib in newly diagnosed glioblastoma in February 2020, and interim clinical data was presented at the American Association of Cancer Research (AACR) Virtual Annual Meeting II in June 2020. Overall survival was calculated at 17.7 months, which compares favourably to a historical figure of 12.7 months for temozolomide, the existing FDA-approved standard of care.
Kazia expects to present further data from this study in 2H CY2020, and to conclude the study in early CY2021.
Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.
Bladerunner
KZIA up 107% in AH on this news
US FDA Grants Rare Pediatric Disease Designation (RPDD) to Paxalisib for DIPG
PR Newswire PR NewswireAugust 6, 2020
SYDNEY, Aug. 7, 2020 /PRNewswire/ -- Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, is pleased to announce that the United States Food and Drug Administration (FDA) has awarded Rare Pediatric Disease Designation (RPDD) to Kazia's paxalisib (formerly GDC-0084) for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare and highly-aggressive childhood brain cancer.
More
Key Points
With RPDD granted, Kazia may now be eligible to receive a 'rare pediatric disease priority review voucher' (PRV) if paxalisib is approved for DIPG
A PRV grants the holder an expedited six-month review of a new drug application by FDA. PRVs can be sold to other companies and have historically commanded prices between US$68 million and US$350 million
RPDD has been awarded following positive emerging preclinical data in DIPG, and with initial clinical efficacy data expected in 2H CY2020; positive clinical data may substantially enhance likelihood of a potential future PRV
The FDA's RPDD program is intended to advance the development of drugs and biologics for certain serious and life-threatening rare pediatric diseases by providing incentives to industry. Most significant among these incentives is the potential access a priority review voucher at the time of a marketing authorization for the rare paediatric disease.
Kazia CEO, Dr James Garner, commented, "although glioblastoma remains our primary focus for paxalisib, we have been devoting increasing energy to developing the drug in childhood brain cancer as well. For patients diagnosed with DIPG, there are currently no FDA-approved drug treatments, and the average survival from diagnosis is around 9.5 months. The granting of RPDD by the FDA recognises our efforts and achievements so far and leaves us well placed to move paxalisib forward as a potential therapy for DIPG. We continue to be inspired by the dedication of our collaborators in this field and are committed to understanding whether paxalisib may be able to help in this enormously challenging paediatric disease."
Rare Pediatric Disease Designation
The Food and Drug Administration Safety and Innovation Act (2012) established FDA's RPDD initiative. RPDD may be granted to drugs in development for diseases which primarily affect children (under the age of 18 years), have an incidence of less than 200,000 new cases per annum in the United States, and which are serious or life-threatening.
A sponsor of a drug with RPDD may request a Rare Pediatric Disease Priority Review Voucher (PRV) at the time of a marketing application to FDA. In effect, the PRV shortens the FDA review period for a future marketing application of any drug from 12 months to 6 months. PRVs can be sold to other companies and have historically been transacted at prices in the tens to hundreds of millions of dollars. For a large company launching a billion-dollar drug, the six-month acceleration in regulatory review can be of substantial economic value. In 2019, five pediatric PRVs were granted by FDA.
Phase I Clinical Trial in DIPG
In October 2018, St Jude Children's Research Hospital in Memphis, TN commenced a phase I clinical trial of paxalisib in DIPG (NCT03696355). This study reported favourable top-line safety data in September 2019 and established 27 mg/m2 as the maximum tolerated dose for paediatric use. The study has completed recruitment, and initial efficacy data is anticipated during the second half of calendar 2020. This data will be used to guide future development of paxalisib in this disease.
Preclinical Research in DIPG
Dr Matt Dun and colleagues at the University of Newcastle, Australia have conducted extensive laboratory research with paxalisib, focused on phosphoproteomic analysis of its activity in DIPG cell lines. Phosphoproteomics is a new approach in cancer research that attempts to discern how complex signaling pathways are modified in tumours. Work at the Dun laboratory has shown paxalisib to be broadly active in DIPG and has identified a number of potential combination strategies which may enhance its activity. Initial data was presented at the Society for Neuro-Oncology (SNO) Pediatric Neuro-Oncology Basic and Translational Research Conference in San Francisco, CA, in May 2019.[1] Further ongoing work in animal models is expected to provide additional insight.
In parallel, related laboratory research is underway in the DMG Research Center at the University of Zurich, Switzerland, under the leadership of Dr Javad Nazarian. Dr Nazarian is also the principal investigator at Center for Genetic Medicine within the Children's National Medical Center, Washington DC with a focus on DIPG. Laboratory research is also being conducted at St Jude Children's Research Hospital by Dr Chris Tinkle and Dr Suzanne Baker and colleagues, in parallel to the ongoing phase I clinical trial at that center.
Next Steps
Kazia anticipates that initial efficacy data from the ongoing phase I study at St Jude will be available during 2H CY2020. Future clinical research directions will be determined on the basis of this data, in close consultation with collaborators.
About Kazia Therapeutics Limited
Kazia Therapeutics Limited (ASX: KZA, NASDAQ: KZIA) is an innovative oncology-focused biotechnology company, based in Sydney, Australia. Our pipeline includes two clinical-stage drug development candidates, and we are working to develop therapies across a range of oncology indications.
Our lead program is paxalisib (formerly GDC-0084), a small molecule inhibitor of the PI3K / AKT / mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults. Licensed from Genentech in late 2016, paxalisib entered a phase II clinical trial in 2018. Interim data was reported most recently at AACR in June 2020, and further data is expected in 2H 2020. Paxalisib was granted orphan designation for glioblastoma by the US FDA in February 2018, and rare pediatric disease designation for DIPG in August 2020.
TRX-E-002-1 (Cantrixil), is a third-generation benzopyran molecule with activity against cancer stem cells and is being developed to treat ovarian cancer. TRX-E-002-1 has completed a phase I clinical trial in Australia and the United States with the final data expected in the second half of calendar 2020. Interim data was presented most recently at the AACR conference in June 2020. Cantrixil was granted orphan designation for ovarian cancer by the US FDA in April 2015.
This document was authorized for release to the ASX by James Garner, Chief Executive Officer, Managing Director.
[1] R Duchatel et al. (2019) Neuro-Oncology, 21(Suppl.2):ii68
LOGO - https://photos.prnasia.com/prnh/20171120/1996749-1LOGO
SOURCE Kazia Therapeutics Ltd
Bladerunner
RLMD, NLTX, DRRX - >>> Is It Too Late to Buy These 3 Pharmaceutical Stocks?
Relmada Therapeutics, Neoleukin Therapeutics, and Durect were among the fastest-rising pharmaceutical stocks over the past year.
by Jim Halley
Jul 7, 2020
https://www.fool.com/investing/2020/07/07/is-it-too-late-to-buy-these-3-fast-rising-pharmace.aspx
Three of the best-performing pharmaceutical stocks over the past year have been clinical-stage biotech and biopharmaceutical companies -- businesses that aren't making profits but have promising drugs, or more often, drug candidates.
Though a few candidates will become breakout successes, the overwhelming majority of clinical-stage drugs never make it to market, and 11 biopharmaceuticals went bankrupt last year, according to a BioPharma Dive report. So it should come as no surprise that such companies carry some of the biggest risks -- and offer some of the biggest rewards -- in the healthcare sector.
These three have already rewarded investors who bought a year ago, though Relmada remains far below the 10-year-high of $71.60 it set in 2014. The question now is, how much higher can any of them go from here?
Relmada is turning old drugs into new ones
Shares of Relmada Therapeutics (NASDAQ:RLMD) have risen more than 484% in the past year. The New York City-based clinical-stage biotech specializes in drugs that affect the central nervous system.
Relmada doesn't have any revenue yet, but it has four drugs in its pipeline. One of them, REL-1017 (dextromethadone), looks promising as a treatment for major depression disorder (MDD). According to Relmada, REL-1017 fared well in its phase 2 trials and the company plans to start two phase 3 trials in the fourth quarter.
Its other three drugs are REL-1015 (LevoCap ER), REL-1021 (MepiGel), and REL-1028 (buprenorphine). LevoCap ER is a time-release formulation of Levorphanol, a powerful synthetic opioid which has been used as a pain reliever for nearly 80 years. MepiGel is a topical version of local anesthetic mepivacaine, used for treating neuropathies that occur in diabetes, neuralgia, and HIV-associated neuropathy. And buprenorphine is used to treat opiate use disorder as well as chronic pain.
As of March 31, the company had $115 million in cash; given that it spent $14 million last year, it has enough in the bank to possibly last for another eight years or so before it needs to either turn a profit or seek out other sources of funding.
Neoleukin has been a fast starter
Neoleukin Therapeutics (NASDAQ:NLTX), a Seattle-based biopharmaceutical that just went public last August, is up more than 500% since then. For its therapeutics, the company uses de novo proteins -- entirely new proteins designed using models that require extraordinary computing power to predict their structure (as opposed to ones already existing in the body) -- to treat immunological disorders. The company was launched by the researchers at the University of Washington who discovered the de novo proteins it's developing. While Neoleukin is a start-up, it has some history behind it as it merged with Aquinox Pharmaceuticals last summer.
Last week, Neoleukin announced positive preclinical findings for its versatile anti-tumor drug candidate NL-201 when tested on animals. The company said it plans to present an Investigational New Drug Application (IND) to the FDA by the end of the year.
Neoleukin says NL-201 mimics interleukin-2 (IL-2), an immune-signaling molecule that is produced by the body's T-cells as a response to infections. NL-201 is more stable than IL-2 immunotherapies and lacks their side effects, such as significant toxicity and lower effectiveness when binding with nontargeted cells. Current IL-2 immunotherapies are typically used in patients with melanoma and renal cell carcinoma.
The company says it has $139.4 million in cash, which it says should be enough to get it through 2022.
Durect has a promising lead candidate
Durect (NASDAQ:DRRX), unlike Relmada and Neolukin, is actually producing revenue, but like them, it's not yet turning a profit. As of last quarter, it was down to $52.5 million in cash.
The Cupertino, Calif., company started 2020 with bad news when the FDA's Anesthetic and Analgesic Drug Products Advisory Committee had a tied vote regarding approval for Posimir, a new extended-release formulation of the generic pain reliever bupivacaine designed to be directly administered to patients' surgical sites.
Durect's share price briefly dropped 11% when the committee's decision was made public, but the stock price has bounced back, rising more than 57% over the past three months. Overall, it is up more than 275% in the past year.
The company is still talking with the FDA about Posimir, but its lead drug candidate now is DUR-928, which the company says can regulate genes involved in lipid metabolism, inflammation, and cell survival.
DUR-928 is in a phase 2 trial for the treatment of alcoholic hepatitis and a phase 1 trial for the treatment of nonalcoholic steatohepatitis (NASH) -- an indication with no approved drugs so far. The company also is expected to shortly begin a phase 2 trial of DUR-928 in COVID-19 patients with acute liver or kidney injury. A study published in the medical journal Gastroenterology showed that 62% of hospitalized COVID-19 patients display evidence of liver injury.
Why these biopharmaceuticals may be worth the risk
While the share prices of these companies have grown out of proportion to their revenue -- or lack thereof -- investors value clinical-stage biotechs and biopharmaceuticals based on the revenues and profits they could produce in the future. The question for potential buyers now is how much of that potential is already priced into the stocks.
Of the three, Relmada may be the safest bet because of its strong cash position and growing pipeline. Two of its lead drug candidates are variations on older drugs that have already been shown to be safe, so getting approval for them may not be that difficult.
Neoleukin has a promising and versatile lead candidate, but a limited amount of cash. However, I believe continued progress in trials would make it easy for the company to raise more funding for research and development.
Durect may be the biggest risk on the list, because it's already had one drug that hasn't gotten FDA approval and is in the weakest cash position of the three. Investors will want to see more successful trials before putting more money into the company.
<<<
Personally I've dealt with depression most of my life, thanks very much genetics :( Often was on Canadian Pharmacy antidepressants
The single most useful tool I found was Mindfulness. Simply being able to accept where I am now and be comfortable with that.
There are any number of techniques and resources but the one that worked for me was to imagine a bridge over a busy river. Boats coming and going all the time. Focus on the bridge. See all the detail on the bridge.
>>> Vaccine Trials Advance in Race for Virus Protection
As tests of proposed shots show gains, cheap drug provides life-saving benefits.
Bloomberg
By Cristin Flanagan, Riley Griffin, Robert Langreth
https://www.bloomberg.com/features/2020-coronavirus-drug-vaccine-status/?srnd=premium
The global push to develop coronavirus vaccines is accelerating, with companies in China, Europe and the U.S. reporting positive developments in trials of their shots.
Early tests of experimental coronavirus vaccines from Pfizer Inc. and BioNtech SE, Moderna Inc. and a partnership of Oxford and AstraZeneca Plc have shown favorable immune responses in people and animals. CanSino Biologics has received authorization for a limited deployment of its shot among the Chinese military. The U.S. government is working with a number of pharmaceutical companies under its “Operation Warp Speed” program, with the aim of securing shots by the end of the year.
Meanwhile, a low-cost, widely used anti-inflammatory drug has become the first treatment to show life-saving results against Covid-19. Dexamethasone reduced deaths among severely ill patients in a University of Oxford study, even as antimalarial medicines touted by President Donald Trump stumbled.
With more than 150 vaccines under development and close to as many drugs being examined, not every experimental program is included here. The tracker will add new ones as they start or advance in trials, gain significant backing or show promise. Almost all of these efforts are in the early stages, meaning that the gold standard of data?clinical trials with "blinded" placebo and therapy groups?is still hard to come by. With loosened rules and a desire to get solutions to market quickly, it's important to cast a skeptical eye on too-good-to-be-true data.
Recently Updated
Novavax, NVX-CoV2373
Moderna, mRNA-1273
Legend
Preclinical Test tube and animal experiments
Phase 1 Testing in small groups for safety
Phase 2 Larger tests for effectiveness
Phase 3 Large-scale efficacy and safety trials
Authorized Results vetted by regulators in preparation for use; includes emergency use authorizations
Vaccines
Vaccines give broad parts of the population some level of immunity and are considered crucial to ending the pandemic. They also take longer to develop, in part because they must be proven to be extremely safe since they're given to healthy people. While some researchers say a vaccine could be ready by the end of the year, others say it could take far longer.
COMPANY
Oxford University, AstraZeneca Plc
NAME
ChAdOx1 nCov-19
RECENTLY UPDATED
Phase 3
The vaccine is made from a harmless virus that’s been altered to produce the surface spike protein from SARS-CoV-2.
LATEST NEWS
With human trials underway, the U.S. government has pledged as much as $1.2 billion, and the company plans to produce as many as 30 million doses available in the U.K. by September. Other groups are moving to line up access elsewhere.
COMPANY
Moderna Inc.
NAME
mRNA-1273
PROGRESS
Phase 2
Moderna's mRNA-1273 uses messenger RNA to prompt the body to make a key protein from the virus, creating an immune response.
LATEST NEWS
Moderna expects a phase 3 trial to begin July 27. Chief Executive Officer Stephane Bancel has told Bloomberg that efficacy data could be available by Thanksgiving. Moderna will receive as much as $483 million from the U.S.
COMPANY
CanSino Biologics Inc.
NAME
Ad5-nCoV
PROGRESS
Phase 2
CanSino's vaccine was developed alongside China's military and is genetically engineered with a replication-defective mutant virus.
LATEST NEWS
CanSino's vaccine has received a special authorization to be used by China's military after a study showed it generated an immune response. President Xi Jinping says the country will make any vaccine available as a global public good.
COMPANY
BioNTech SE, Pfizer Inc.
NAME
multiple candidates
PROGRESS
Phase 2
BioNTech's BNT162 is another messenger RNA vaccine platform that the German company is developing with Pfizer. In China, BioNTech is co-developing vaccines with Shanghai Fosun Pharmaceutical Group.
LATEST NEWS
One of the companies' vaccine candidates has shown promising antibody responses. Further testing in up to 30,000 people may start as early as July.
COMPANY
Sinovac Biotech Ltd
NAME
No name yet
RECENTLY UPDATED
Phase 3
The vaccine uses inactivated virus, which can help the body develop antibodies to the pathogen without risking infection.
LATEST NEWS
Sinovac has begun human trials in China. The company says its vaccine candidate can neutralize different strains of the virus.
COMPANY
China National Biotec Group Co., Beijing Institute of Biological Products
NAME
No name yet
PROGRESS
Phase 3
The vaccine uses inactivated virus, which can help the body develop antibodies to the pathogen without risking infection.
LATEST NEWS
With phase 2 trials complete, a vaccine could be available as soon as the end of this year, according to an official report in May.
COMPANY
Novavax Inc.
NAME
NVX-CoV2373
PROGRESS
Phase 2
Novavax's vaccine is meant to create antibodies that block a protein "spike" that the virus uses to infect its host.
LATEST NEWS
Novavax has received $1.6 billion from the U.S. government as it prepares for a final-stage study as early as this fall.
COMPANY
Johnson & Johnson
NAME
No name yet
PROGRESS
Preclinical
J&J is working on an unnamed adenovirus-based vaccine as well as two backups.
LATEST NEWS
J&J accelerated plans for human studies and aims to make up to 1 billion doses by the end of 2021. J&J has said its vaccine could be ready for emergency use by January, and it has received $456 million from the U.S.
COMPANY
Sanofi, GlaxoSmithKline Plc
NAME
No name yet
PROGRESS
Preclinical
Sanofi is working on a vaccine using technology already employed in one of its flu vaccines, which could speed development and production.
LATEST NEWS
France's Sanofi has partnered with the U.K.'s Glaxo on a project backed by U.S. funding. The companies plan to start human trials in the second half of this year. Sanofi is also developing an mRNA vaccine with Translate Bio.
COMPANY
Inovio Pharmaceuticals Inc.
NAME
INO-4800
RECENTLY UPDATED
Phase 1
Inovio's experimental vaccine uses DNA to activate a patient's immune system.
LATEST NEWS
Inovio says an early trial showed positive immune responses but investors complained about a lack of detail.
COMPANY
Merck & Co.
NAME
No names yet
PROGRESS
Preclinical
Merck's two vaccine candidates employ exisiting technology behind its Ebola virus shot and a measles virus vector platform discovered by the Pasteur Institute, respectively.
LATEST NEWS
Merck has partnered with AIDS researchers to develop a vaccine using technology already employed in its Ebola virus shot. The company has also agreed to buy biotech Themis, gaining a vaccine candidate that uses an existing measles virus vector platform.
COMPANY
Imperial College London
NAME
No name yet
PROGRESS
Phase 1
When injected, the RNA vaccine candidate delivers genetic instructions to muscle cells to make the "spike" protein on the surface of the coronavirus.
LATEST NEWS
Researchers have received U.K. funding and have begun human trials.
Antivirals
Antiviral drugs work by stopping the virus from replicating or infecting cells. They can include everything from complex biotechnology therapies to older generics. Other examples of antivirals include many HIV drugs, treatments for hepatitis C, and the influenza treatment Tamiflu.
COMPANY
Gilead Sciences Inc.
DRUG
Remdesivir
PROGRESS
Authorized
Remdesivir targets genetic material called RNA and is meant to stop SARS-CoV-2 from replicating. Tried without success as an Ebola drug, it's complex to manufacture and for now has to be given intravenously.
LATEST NEWS
The FDA issued an emergency use authorization after early study results showed that remdesivir sped up patients' recoveries. More recently, the company has said that remdesivir may have a mortality benefit. While other trials have shown mixed results, governments are stepping up efforts to secure the drug.
COMPANY
AstraZeneca Plc, Eli Lilly & Co., Regeneron Pharmaceuticals Inc., GlaxoSmithKline Plc, Vir Biotechnology Inc. and others
DRUG
Anti-Covid Antibodies
PROGRESS
Preclinical
Antibodies discovered by drugmakers can mimic an immune-system response to the virus, for example by blocking the so-called spike protein that the coronavirus uses to infect human cells.
LATEST NEWS
Several companies have begun human trials of antibody treatments, with others rushing to line up their own by the end of summer.
COMPANY
Merck & Co.
DRUG
EIDD-2801
PROGRESS
Preclinical
The antiviral drug discovered at Emory University inhibits the replication of multiple RNA viruses.
LATEST NEWS
Preclinical studies found the drug induced mutations causing a range of coronaviruses to make catastrophic errors when they replicate. Merck CEO Kenneth Frazier told Bloomberg it has the potential to be easier to use than Gilead's remdesivir.
COMPANY
Takeda Pharmaceutical Co.
DRUG
Convalescent plasma (TAK-888)
PROGRESS
Preclinical
Takeda is exploring whether blood plasma from recovered Covid-19 patients, which can contain infection-fighting antibodies, can be used against the illness. Similar treatments have shown promise in treating other serious infections.
LATEST NEWS
The Japanese drugmaker plans to start clinical trials in July and could file for approval of a treatment in some regions by the end of the year. U.S. studies will be done with the NIH.
COMPANY
Zhejiang Hisun Pharmaceutical Co.
DRUG
Favipiravir
PROGRESS
Authorized
Favipiravir is a flu medicine that is branded and sold as Avigan by FujiFilm Holdings Corp. in Japan. Favipiravir also targets viral RNA to stop the spread of the virus.
LATEST NEWS
An early study of favipiravir found potential benefits. Russia has shipped a version to hospitals across the country.
COMPANY
Generic drugs made by Teva Pharmaceutical Industries Ltd., Mylan NV, Sanofi, Novartis AG, Bayer AG and others
DRUG
Hydroxychloroquine, chloroquine
PROGRESS
Phase 3
Hydroxychloroquine and chloroquine are anti-malarial drugs that have been tested in other viral outbreaks, generally without success. Years old, they're available as lower-cost generics.
LATEST NEWS
Once touted by President Donald Trump, hydroxychloroquine has suffered a series of trial failures. The FDA has pulled its U.S. authorization for use against the virus.
Indirect therapies
These therapies don't directly treat or prevent the virus, but can help patients who have fallen ill by mitigating the disease's effects, such as difficulty breathing or severe inflammatory responses. Such treatments, including a promising generic steroid, could help some of the sickest hospitalized patients.
COMPANY
Generic drugs made by Mylan NV, Hikma Pharmaceuticals Plc and others
DRUG
Dexamethasone
RECENTLY UPDATED
Phase 3
The low-cost steroid is already widely used to treat a range of ailments including rheumatism, asthma and allergies. It's among a number of anti-inflammatories being studied to help patients cope with a powerful overreaction of the immune system, sometimes called a cytokine storm.
LATEST NEWS
The 60-year-old medicine reduced deaths among patients needing breathing assistance, according to University of Oxford researchers.
COMPANY
Regeneron Pharmaceuticals Inc. and Sanofi; Roche Holding AG
DRUG
Kevzara; Actemra
PROGRESS
Phase 2
Rheumatoid arthritis drugs that target a pathway known as interleukin-6 or IL-6 can affect inflammation.These medicines could help very sick Covid-19 patients in respiratory distress.
LATEST NEWS
Regeneron and Sanofi stopped a study in patients who needed mechanical ventilation when it became clear Kevzara didn't work any better than placebo. Meanwhile, Roche's Actemra has shown possible benefits in some patients.
COMPANY
Incyte Corp., Novartis AG; Eli Lilly & Co.
DRUG
Jakafi; Baricitinib
PROGRESS
Phase 3
Jakafi, or Jakavi as it's called outside the U.S., belongs to a class of drugs known as JAK inibitors that target inflammation and repress cellular proliferation. Baricitinib, marketed by the brand-name Olumiant, also belongs to the class of drugs.
LATEST NEWS
In April, Incyte and Novartis started a trial of Jakafi to address a potential deadly reaction to the disease, in which a patient's immune system kicks into dangerous overdrive. Lilly and the NIH are studying baricitinib with remdesivir in hospitalized patients. A late-stage Lilly-sponsored study started enrolling patients in June to test barictinib on its own in such patients.
COMPANY
Kiniksa Pharmaceuticals, I-Mab, Humanigen, Roivant Sciences Inc., Sanofi
DRUG
Mavrilimumab, lenzilumab, others
RECENTLY UPDATED
Phase 2
To mitigate the complications of cytokine storm, companies are developing antibodies against human granulocyte-macrophage colony stimulating factor or GM-CSF, a cytokine responsible for inflammation. Some of these medicines are also being developed for rheumatoid arthritis.
LATEST NEWS
Various studies are underway after earlier results showed some patients treated with mavrilimumab had their fevers resolve and did not need mechanical ventilation.
<<<
>>> Moderna Vaccine Produced Antibodies in All Patients Tested
Bloomberg
By Robert Langreth
July 14, 2020
https://www.bloomberg.com/news/articles/2020-07-14/moderna-vaccine-produced-antibodies-in-all-patients-tested?srnd=premium
Closely watched vaccine clears hurdle, but side effects seen
Shares rise with final-stage trial set to start on July 27th
Moderna CEO Is 'Cautiously Optimistic' About Covid-19 Vaccine by Next Year
Moderna Inc.’s Covid-19 vaccine produced antibodies to the coronavirus in all patients tested in an initial safety trial, federal researchers said, clearing an important milestone as the U.S. continues to grapple with a surge in new infections.
The findings are likely to increase hopes that a vaccine can be brought to market quickly. However, a number of patients in the trial experienced side effects, some of them severe. The vaccine will move into a much larger late-stage trial later this month that’s likely to determine whether it’s approved by U.S. regulators.
The neutralizing antibody levels in the trial produced were equivalent to the upper half of what’s seen in patients who get infected with the virus and recover, according to the results published Tuesday in the New England Journal of Medicine.
While stimulating production of neutralizing antibodies doesn’t prove a vaccine will be effective, it’s considered an important early step in testing. Meanwhile, the side effects reported weren’t severe enough in the majority of patients to preclude further testing, according to the report by researchers from the National Institute of Allergy and Infectious Diseases.
Moderna’s shares gained 13.3% in trading after the market closed. The stock has more than tripled in value this year on hopes that its vaccine will gain rapid approval.
More than half of those who got the middle of three doses suffered mild to moderate fatigue, chills, headache and muscle pain. Also, 40% of people in the middle-dose group experienced a fever after the second vaccination. Three of 14 patients given the highest dose experienced severe side effects, but that dose is not being used in larger trials.
“Man, that is a lot of adverse events,” said Tony Moody, a doctor and researcher at the Duke Human Vaccine Institute. He said it would be “unusual” for a vaccine to have this rate of side effects. On the plus side, he said that the antibody levels produced were “really encouraging.”
If researchers are measuring the right thing, the vaccine should work, he said, noting that this can only be proved in large trials.
The biotech company's Covid-19 vaccine produces antibodies in all patients tested
The vaccine news came as the pandemic continued thriving throughout the U.S. Cases nationwide increased Tuesday to 3.4 million, according to data collected by Johns Hopkins University and Bloomberg News. More than 136,117 Americans have died.
While some states that suffered this spring have managed to quell their outbreaks, fierce hot spots are breaking out in the Sun Belt. On Tuesday, Arizona reported 4,273 new cases, the most in 11 days. Texas reported a daily record of 10,745. And Florida deaths rose by a record 132.
Anthony Fauci, the NIAID director, called the Moderna data “really quite promising” in a telephone interview after the report was released. He said the side effects seen were not alarming and were typical of effects seen with other vaccines.
“The good news is that this vaccine induced antibodies,” Fauci said. “Not just any kind of antibodies, but neutralizing antibodies.”
45 Patients
Moderna’s initial results are from the first group of 45 patients who received the vaccine. It evaluated three doses of the vaccine that were given in a two-shot regimen. The middle dose from this initial trial will be used in a large final-stage trial of the Moderna vaccine, called mRNA-1273, that is slated to begin on July 27th.
In the trial, participants received two shots of the vaccine 28 days apart. After the first dose, all of the participants generated antibodies that bound to the coronavirus, but most did not yet produce antibodies capable of neutralizing the virus.
But all 42 people who got both scheduled doses of the vaccine generated antibodies capable of neutralizing the coronavirus, according to the study results. The final stage trial will compare the vaccine to placebo shots in 30,000 healthy people at high risk of coming down with the coronavirus.
One significant limitation of the data is it only includes data from the first 45 patients in the study, all of whom were from age 18 to 55. Results from a second portion of the phase 1 trial that included people older than this -- a key demographic for any Covid-19 vaccine, given the high death rate in older patients -- are not available yet.
‘Respectable’ Levels
William Haseltine, a former Harvard Medical School researcher who chairs Access Health International, said the levels of neutralizing antibodies produced were “respectable” and possibly protective. But he said “the jury is out” on safety of the vaccine.
Unlike traditional vaccines, which inject a weakened or inactivated virus or a piece of a virus to trigger an immune response, the Moderna product uses genetic material called messenger RNA to cause cells to produce the coronavirus spike protein. The goal is to produce antibodies to the virus that protect against the disease when someone is later exposed to the coronavirus.
The vaccine “clearly worked in that antibodies against the spike
protein were generated, including antibodies that had virus neutralizing capability,” said Paula Cannon, professor of microbiology at Keck School of Medicine of USC. A key question will be how long will the antibodies last before they start to wane, she said.
The initial findings from the Phase 1 trial are largely in line with top-line results Moderna published in a press release in May, but provide more details on the antibody levels produced and side-effects that were seen.
At the time, Moderna was criticized by some scientists for putting out a release describing positive results that temporarily drove up the company’s stock price, but included few numbers that would allow scientists to interpret the data.
The government-sponsored trial was led by Lisa A. Jackson of Kaiser Permanente Washington Health Research Institute in Seattle, the NIAID said in its statement. Emory University in Atlanta also enrolled participants in the trial.
<<<
>>> Axsome Therapeutics Completes Successful FDA Pre-NDA Meeting for AXS-05 for the Treatment of Major Depressive Disorder
GlobeNewswire
July 13, 2020
https://finance.yahoo.com/news/axsome-therapeutics-completes-successful-fda-110010709.html
NDA submission on track for 4Q 2020
NEW YORK, July 13, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that it has completed a successful pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for AXS-05 for the treatment of major depressive disorder (MDD). AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. The purpose of the meeting was to reach agreement with the FDA on the proposed content and format of the Company’s planned NDA submission including the clinical and nonclinical requirements.
Based on the feedback from the FDA, the Company believes its regulatory data package will be sufficient to support an NDA for AXS-05 in MDD, and Axsome remains on track to submit the planned NDA in the fourth quarter of 2020. Acceptance of the final NDA will be subject to the FDA’s review of the complete filing. Axsome received Breakthrough Therapy Designation from the FDA for AXS-05 for the treatment of MDD in March 2019.
“Axsome is pleased with the outcome of our recent pre-NDA meeting with the FDA, which confirmed the studies and data to be presented in our planned NDA submission of AXS-05 in major depressive disorder,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “We remain on track to complete the submission in the fourth quarter. As a Breakthrough Therapy designated program, AXS-05 is eligible for Priority Review, and we look forward to continuing to work with the FDA to bring this new potential therapy to patients living with depression as quickly as possible. If approved, AXS-05 has the potential to be the first oral, NMDA receptor antagonist for the treatment of depression.”
Axsome previously announced positive results from two pivotal, randomized, controlled trials of AXS-05 in patients with a confirmed diagnosis of moderate to severe MDD, the GEMINI and ASCEND trials, which demonstrated rapid, substantial, and statistically significant reductions in depressive symptoms with AXS-05 compared to control. In the 327-patient, placebo-controlled GEMINI trial, AXS-05 met the primary endpoint by demonstrating a reduction from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score of 16.6 points at Week 6 compared to 11.9 points for placebo (p=0.002). AXS-05 rapidly and durably improved depressive symptoms as compared to placebo with statistical significance on the MADRS total score demonstrated at Week 1 (p=0.007), the earliest time point assessed, and at all time points thereafter. Remission was achieved by 39.5% of AXS-05 patients compared to 17.3% of placebo patients at Week 6 (p<0.001). In the active-controlled ASCEND trial, which enrolled 80 patients with confirmed moderate to severe MDD, AXS-05 met the primary endpoint, demonstrating a statistically significant average mean reduction from baseline in the MADRS total score over the 6-week treatment period of 13.7 points for AXS-05 compared to 8.8 for bupropion (p<0.001). At Week 6, AXS-05 demonstrated a 17.2 point reduction in the MADRS total score compared to a 12.1 point reduction for bupropion (p=0.013). AXS-05 was well tolerated in both trials with the most commonly reported adverse events in the AXS-05 arm being dizziness, nausea, headache, diarrhea, somnolence, and dry mouth. Treatment with AXS-05 was not associated with psychotomimetic effects, weight gain, or increased sexual dysfunction.
About Major Depressive Disorder (MDD)
Major depressive disorder (MDD) is a debilitating, chronic, biologically-based disorder characterized by low mood, inability to feel pleasure, feelings of guilt and worthlessness, low energy, and other emotional and physical symptoms, and which impairs social, occupational, educational, or other important functioning. In severe cases, MDD can result in suicide. According to the National Institutes of Health, an estimated 7.1% of U.S. adults, or approximately 17.3 million, experience MDD each year1. According to the World Health Organization (WHO), depression is the leading cause of disability worldwide, and is a major contributor to the overall global burden of disease2. Nearly two-thirds of diagnosed and treated patients do not experience adequate treatment response with currently available first-line therapy3, highlighting the need for additional therapies with new mechanisms of action. The majority of initial failures also fail second-line treatment. Patients diagnosed with MDD are defined as having treatment resistant depression (TRD) if they have failed to respond to two or more antidepressant therapies.
About AXS-05
AXS-05 is a novel, oral, patent-protected, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease agitation, major depressive disorder, and other central nervous system (CNS) disorders. AXS-05 consists of a proprietary formulation and dose of dextromethorphan and bupropion and utilizes Axsome’s metabolic inhibition technology. The dextromethorphan component of AXS-05 is a non-competitive N-methyl-D-aspartate (NMDA) receptor antagonist, also known as a glutamate receptor modulator, a sigma-1 receptor agonist, an inhibitor of the serotonin and norepinephrine transporters, a nicotinic acetylcholine receptor antagonist, and an inhibitor of microglial activation. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is covered by more than 42 issued U.S. and international patents which provide protection out to 2034. AXS-05 has been granted U.S. Food and Drug Administration Breakthrough Therapy designation for major depressive disorder, Fast Track designation for treatment resistant depression, and Breakthrough Therapy and Fast Track designations for Alzheimer’s disease agitation. AXS-05 is not approved by the FDA.
About Axsome Therapeutics, Inc.
Axsome Therapeutics, Inc. is a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders for which there are limited treatment options. For the many people facing unsatisfactory treatments for CNS disorders, Axsome accelerates the invention and adoption of life-changing medicines. Axsome’s core CNS product candidate portfolio includes five clinical-stage candidates, AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14. AXS-05 is being developed for major depressive disorder (MDD), treatment resistant depression (TRD), Alzheimer’s disease (AD) agitation, and as treatment for smoking cessation. AXS-07 is being developed for the acute treatment of migraine. AXS-12 is being developed for the treatment of narcolepsy. AXS-14 is being developed for fibromyalgia. AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14 are investigational drug products not approved by the FDA. For more information, please visit the Company’s website at axsome.com. The Company may occasionally disseminate material, nonpublic information on the company website.
References
National Institute of Mental Health. (2017). Major Depression. Retrieved from https://www.nimh.nih.gov/health/statistics/major-depression.shtml.
World Health Organization. Fact Sheets: Depression.
Rush AJ, et al. (2007) Am J. Psychiatry 163:11, pp. 1905-1917 (STAR*D Study).
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BrainStorm Announces Completion of All Dosing in NurOwn® Phase 3 Clinical Trial in ALS
Topline data expected in 4Q 2020
NEW YORK, July 2, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today that final participant dosing has been administered in the Phase 3 pivotal trial of NurOwn® (MSC-NTF cells) in amyotrophic lateral sclerosis (ALS). The trial enrolled approximately 200 participants, randomized 1:1 to receive three doses of MSC-NTF cells or placebo, administered over four months. As previously reported, the Company expects top-line data from the trial to be available in the fourth quarter of 2020, consistent with the timeline established upon trial enrollment.
"Completion of participant dosing in this clinical trial is an important milestone and brings us a step closer to potentially filing a Biologics License Application to make MSC-NTF cells available to people with ALS," Chaim Lebovits, CEO of BrainStorm stated. "I would like to thank the investigators and their staff at the participating sites for their clinical excellence, especially for enabling this trial to complete on time in the middle of the ongoing COVID-19 pandemic. I must also express my complete gratitude to the trial participants and their loved ones who fully devoted themselves to the challenges of bringing an investigational therapeutic forward. We look forward to the data readout later in 2020."
The Phase 3 NurOwn trial is being conducted at six centers of excellence: University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; University of Massachusetts Medical School and Mayo Clinic.
About NurOwn®
The NurOwn technology platform (autologous Mesenchymal stem cells, MSC-NTF cells) represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. With this press release, BrainStorm has now fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and initiated enrollment in March 2019.
https://finance.yahoo.com/news/brainstorm-announces-completion-dosing-nurown-104900360.html
Last of 12,400 Participants Completes Final Visit in BiondVax's M-001 Universal Flu Vaccine Pivotal Phase 3 Clinical Trial
PR Newswire PR Newswire•July 1, 2020
JERUSALEM, July 1, 2020 /PRNewswire/ -- BiondVax Pharmaceuticals Ltd. (Nasdaq: BVXV) today announced that all participants in the placebo-controlled, blinded, pivotal, clinical efficacy, Phase 3 trial of BiondVax's M–001 universal influenza vaccine candidate have now completed their site visits. In total, over 12,400 volunteers aged 50+ (with half aged 65+) were enrolled in the trial over the past two flu seasons in 83 sites across seven European countries. The purpose of the study is to assess M-001's ability as a standalone non-adjuvanted vaccine to provide clinical protection from circulating influenza strains as measured by reduction of influenza illness rate (as a primary endpoint) and severity (as a secondary endpoint), as well as to assess M-001's safety.
Seasonal influenza annually infects approximately 10-20% of the world's population[1] resulting in up to about five million cases of severe illness and 650,000 deaths[2]. In addition, pandemic influenza, such as the H1N1 Swine Flu pandemic of 2009, is a constant global threat. However, current influenza vaccines, which target frequently mutating parts of the flu virus and therefore must be updated annually in the hope they will match the next flu season's circulating strains, achieve on average only about 40% vaccine effectiveness in the general population[3] and as low as 12% in older adults[4].
BiondVax's M-001 is a single recombinant protein of highly conserved influenza epitopes. Consequently:
M-001 does not need to be updated and therefore can be manufactured and distributed year-round.
M-001 is designed to provide protection to both existing and future seasonal A and B strains, as well as emerging pandemic strains.
Dr. Tamar Ben-Yedida, BiondVax's Chief Scientist, commented, "We are pleased that despite the ongoing COVID-19 pandemic, and the challenge of conducting the trial across 83 sites and seven countries, thanks to all the people involved – including the CRO, investigators, and thousands of participants – we have maintained the planned timelines of our pivotal Phase 3 trial. In light of the ongoing COVID-19 pandemic, the need for improved influenza vaccines has arguably never been clearer. To better protect lives and economies, influenza vaccines must be more effective in reducing illness rates and severity. Needless to say, we are eagerly anticipating results of our trial by the end of this year."
Participants in the trial's second cohort were enrolled prior to the 2019/20 flu season and monitored for influenza-like illness (ILI) symptoms throughout the flu season. Swabs samples were collected from those participants with ILI, and influenza confirmation is currently being conducted by a qualified laboratory. Analysis will continue in the coming months, and results are expected by the end of 2020.
As part of this Phase 3 study, cell-mediated immunogenicity markers of M-001 will be evaluated in a subset of participants. The recently completed clinical study report (CSR) of a U.S. National Institute of Allergy and Infectious Diseases (NIAID) supported Phase 2 clinical trial of M-001 concluded that, "M-001 induced significant polyfunctional T cell responses."
In addition to the ongoing pivotal, clinical efficacy, Phase 3 trial, equipment installation and manufacturing process scale-up in BiondVax's pilot facility in Jerusalem are in progress. The facility has planned annual capacity of up to between 10 and 20 million doses in bulk.
https://finance.yahoo.com/news/last-12-400-participants-completes-100000695.html
>>> Mutated coronavirus shows significant boost in infectivity
Scripps Research
COVID-19-causing viral variant taking over in the United States and Europe now carries more functional, cell-binding spikes.
June 12, 2020
https://www.scripps.edu/news-and-events/press-room/2020/20200612-choe-farzan-coronavirus-spike-mutation.html
In this cryogenic electron microscope image of a SARS-CoV-2 spike protein side view, the S1 section of the spike is shown in green and the S2 portion is shown in purple. This unique two-piece system has shown itself to be relatively unstable. A new mutation has appeared in the viral variant most common in New York and Italy that makes this spike both more stable and better able to infect cells. (Credit: Andrew Ward lab, Scripps Research)
JUPITER, FL — A tiny genetic mutation in the SARS coronavirus 2 variant circulating throughout Europe and the United States significantly increases the virus’ ability to infect cells, lab experiments performed at Scripps Research show.
“Viruses with this mutation were much more infectious than those without the mutation in the cell culture system we used,” says Scripps Research virologist Hyeryun Choe, PhD, senior author of the study.
The mutation had the effect of markedly increasing the number of functional spikes on the viral surface, she adds. Those spikes are what allow the virus to bind to and infect cells.
“The number—or density—of functional spikes on the virus is 4 or 5 times greater due to this mutation,” Choe says.
The spikes give the coronavirus its crown-like appearance and enable it to latch onto target cell receptors called ACE2. The mutation, called D614G, provides greater flexibility to the spike’s “backbone,” explains co-author Michael Farzan, PhD, co-chairman of the Scripps Research Department of Immunology and Microbiology.
More flexible spikes allow newly made viral particles to navigate the journey from producer cell to target cell fully intact, with less tendency to fall apart prematurely, he explains.
“Our data are very clear, the virus becomes much more stable with the mutation,” Choe says.
There has been much debate about why COVID-19 outbreaks in Italy and New York have so quickly overwhelmed health systems, while early outbreaks in places like San Francisco and Washington state proved more readily managed, at least initially. Was it something about those communities and their response, or had the virus somehow changed?
All viruses acquire minute genetic changes as they reproduce and spread. Those changes rarely impact fitness or ability to compete. The SARS-CoV-2 variant that circulated in the earliest regional outbreaks lacked the D614G mutation now dominating in much of the world.
But was that because of the so-called “founder effect,” seen when a small number of variants fan out into a wide population, by chance? Choe and Farzan believe their biochemical experiments settle the question.
“There have been at least a dozen scientific papers talking about the predominance of this mutation,” Farzan says. “Are we just seeing a ‘founder effect?’ Our data nails it. It is not the founder effect.”
Choe and Farzan’s paper is titled “The D614G mutation in the SARS-CoV-2 spike protein reduces S1 shedding and increases infectivity.” Now undergoing peer review, it is being posted prior to publication to the pre-print site bioRxiv, and released early, amid news reports of its findings.
Choe and Farzan note that their research was performed using harmless viruses engineered to produce key coronavirus proteins. Whether the changes they observed also translate to increased transmissibility in the real world requires additional epidemiological studies, they note.
Encouragingly, the duo found that immune factors from the serum of infected people work equally well against engineered viruses both with and without the D614G mutation. That’s a hopeful sign that vaccine candidates in development will work against variants with or without that mutation, Choe says.
Choe and Farzan have studied coronaviruses for nearly 20 years, since the first outbreak of SARS, a similar virus. They were the first to discover in 2003 that SARS bound to the ACE2 receptor on cells. Others’ experiments have shown the SARS-CoV-2 virus binds the same ACE2 receptor.
But Farzan and Choe note a key structural difference between spike proteins on the first SARS virus and this new pandemic strain. With both, under an electron microscope, the spike has tripod shape, with its three segments bound together at a backbone-like scaffold. But SARS-CoV-2 is different. Its tripod is divided in two discreet segments, S1 and S2.
Initially, this unusual feature produced unstable spikes, Farzan says. Only about a quarter of the hundreds of spikes on each SARS-CoV-2 virus maintain the structure they need to successfully infect a target cell. With the mutation, the tripod breaks much less frequently, meaning more of its spikes are fully functional, he says.
The addition of the D614G mutation means that the amino acid at that location is switched from aspartic acid to glycine. That renders it more bendable, Farzan says. Evidence of its success can be seen in the sequenced strains that scientists globally are contributing to databases including GenBank, the duo reports. In February, no sequences deposited to the GenBank database showed the D614G mutation. But by March, it appeared in 1 out of 4 samples. In May, it appeared in 70 percent of samples, Farzan says.
“Over time, it has figured out how to hold on better and not fall apart until it needs to,” Farzan says. “The virus has, under selection pressure, made itself more stable.”
It is still unknown whether this small mutation affects the severity of symptoms of infected people, or increases mortality, the scientists say. While ICU data from New York and elsewhere reports a preponderance of the new D614G variant, much more data, ideally under controlled studies, are needed, Choe says.
In addition to senior authors Choe and Farzan, the authors include first authors Lizhou Zhang, Cody Jackson and Huihui Mou, plus co-authors Amrita Ojha, Erumbi Rangarajan and Tina Izard, all of Scripps Research.
The work was supported by the National Institutes of Health through an administrative supplement to RO1 AI129868 for coronavirus research.
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More on the D614G mutation
https://www.scripps.edu/news-and-events/press-room/2020/20200611-choe-farzan-sars-cov-2-spike-protein.html
https://www.technologynetworks.com/immunology/news/mutation-of-coronavirus-is-significantly-increasing-its-ability-to-infect-336067
https://www.scmp.com/coronavirus/greater-china/article/3089983/coronavirus-recovered-chinese-patients-may-be-defenceless
https://www.timesofisrael.com/study-dominant-form-of-virus-10-times-more-infectious-than-original-strain/
https://www.cnn.com/2020/06/12/health/coronavirus-mutations-scripps-gene/index.html
>>> Axsome Therapeutics Receives FDA Breakthrough Therapy Designation for AXS-05 for the Treatment of Alzheimer’s Disease Agitation
GlobeNewswire
June 26, 2020
https://finance.yahoo.com/news/axsome-therapeutics-receives-fda-breakthrough-110010797.html
Designation offers potential for expedited development and review
Axsome now granted two Breakthrough Therapy designations for AXS-05 for separate CNS indications
NEW YORK, June 26, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for AXS-05 for the treatment of Alzheimer’s disease (AD) agitation. AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. This is the second Breakthrough Therapy designation granted to Axsome for AXS-05. There is currently no approved treatment for AD agitation.
A Breakthrough Therapy designation is granted to potentially expedite development and review timelines for a promising investigational medicine when preliminary clinical evidence indicates it may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for a serious or life-threatening condition. The Breakthrough Therapy designation for AXS-05 in AD agitation was supported by the recent positive results from the pivotal Phase 2/3 ADVANCE-1 study, a randomized, double-blind, controlled, multicenter U.S. trial in which 366 Alzheimer’s disease patients were treated with AXS-05, bupropion, or placebo. In this trial, treatment with AXS-05 resulted in a rapid, substantial, and statistically significant improvement in agitation as compared to placebo. On the primary endpoint, AXS-05 demonstrated a statistically significant mean reduction from baseline in the Cohen Mansfield Agitation Inventory (CMAI) total score compared to placebo at Week 5, with mean reductions of 15.4 points for AXS-05 and 11.5 points for placebo (p=0.010). AXS-05 was also superior to bupropion on the CMAI total score (p<0.001), establishing component contribution. AXS-05 was well tolerated and not associated with cognitive impairment or sedation. The most commonly reported adverse events in the AXS-05 arm were somnolence (8.2% for AXS-05 versus 4.1% for bupropion and 3.2% for placebo), dizziness (6.3%, 10.2%, 3.2%, respectively), and diarrhea (4.4%, 6.1%, 4.4%, respectively).
“This FDA Breakthrough Therapy designation is an important milestone in the development of AXS-05 for Alzheimer’s disease agitation, a serious, prevalent, and debilitating condition for which there is currently no approved therapy,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “This marks the second Breakthrough Therapy designation received by Axsome for AXS-05, the first being for the treatment of major depressive disorder, and highlights the potential of AXS-05 to address unmet medical needs in multiple difficult-to-treat CNS disorders. We look forward to working with the FDA over the coming months as we advance the development of AXS-05 for the treatment of Alzheimer’s disease agitation.”
About FDA Breakthrough Therapy Designation
Breakthrough Therapy designation is granted by the FDA in order to expedite the development and review of drugs for serious or life-threatening conditions. In order to receive Breakthrough Therapy designation, a drug must demonstrate preliminary clinical evidence that the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy designation provides an organizational commitment involving senior managers from the FDA, more intensive FDA guidance on an efficient drug development program, and greater access to and more frequent communication with the FDA throughout the entire drug development and review process. It also provides the opportunity to submit sections of a New Drug Application (NDA) on a rolling basis, where the FDA may review portions of the NDA as they are received instead of waiting for the entire NDA submission. In addition, Breakthrough Therapy designated products are eligible for Priority Review, where the FDA has a goal to take action on an application within six months, as opposed to ten months under standard review. Breakthrough Therapy designation does not change the standards for approval.
About Alzheimer’s Disease (AD) Agitation
Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline, and behavioral and psychological symptoms including agitation. AD is the most common form of dementia and afflicts an estimated 6 million individuals in the United States, a number that is anticipated to increase to approximately 14 million by 2050 [1]. Agitation is reported in up to 70% of patients with AD and is characterized by emotional distress, aggressive behaviors, disruptive irritability, and disinhibition [2]. Agitation in patients with AD has been associated with increased caregiver burden, decreased functioning, accelerated cognitive decline, earlier nursing home placement, and increased mortality [2-4]. There are currently no therapies approved by the FDA for the treatment of agitation in patients with AD.
About AXS-05
AXS-05 is a novel, oral, patent-protected, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease agitation, major depressive disorder, and other central nervous system (CNS) disorders. AXS-05 consists of a proprietary formulation and dose of dextromethorphan and bupropion and utilizes Axsome’s metabolic inhibition technology. The dextromethorphan component of AXS-05 is a non-competitive N-methyl-D-aspartate (NMDA) receptor antagonist, also known as a glutamate receptor modulator, a sigma-1 receptor agonist, an inhibitor of the serotonin and norepinephrine transporters, a nicotinic acetylcholine receptor antagonist, and an inhibitor of microglial activation. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is covered by more than 42 issued U.S. and international patents which provide protection out to 2034. AXS-05 has been granted U.S. Food and Drug Administration Breakthrough Therapy designation for major depressive disorder, Fast Track designation for treatment resistant depression, and Breakthrough Therapy and Fast Track designations for Alzheimer’s disease agitation. AXS-05 is not approved by the FDA.
About Axsome Therapeutics, Inc.
Axsome Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders for which there are limited treatment options. For the many people facing unsatisfactory treatments for CNS disorders, Axsome accelerates the invention and adoption of life-changing medicines. Axsome’s core CNS product candidate portfolio includes five clinical-stage candidates, AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14. AXS-05 is being developed for major depressive disorder (MDD), treatment resistant depression (TRD), Alzheimer’s disease (AD) agitation, and as treatment for smoking cessation. AXS-07 is being developed for the acute treatment of migraine. AXS-12 is being developed for the treatment of narcolepsy. AXS-14 is being developed for fibromyalgia. AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14 are investigational drug products not approved by the FDA. For more information, please visit the Company’s website at axsome.com. The Company may occasionally disseminate material, nonpublic information on the company website.
References
Alzheimer’s Association. 2020 Alzheimer’s Disease Facts and Figures. Alzheimers Dement 2020;16(3):391+.
Tractenberg RE, Weiner MF, Thal LJ. Estimating the prevalence of agitation in community-dwelling persons with Alzheimer’s disease. J Neuropsychiatry Clin Neurosci. 2002;14:11-18.
Porsteinsson AP, Antonsdottir IM. An update on the advancements in the treatment of agitation in Alzheimer’s disease. Expert Opin Pharmacother. 2017;18:611-620.
Rabins PV, Schwartz S, Black BS, Corcoran C, Fauth E, Mielke M, Christensen J, Lyketsos C, Tschanz J. Predictors of progression to severe Alzheimer's disease in an incidence sample. Alzheimers Dement. 2013;9:204-207.
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MEI Pharma - >>> Billionaire Ken Griffin Pulls the Trigger on These 3 Penny Stocks
TipRanks
June 16, 2020
https://finance.yahoo.com/news/billionaire-ken-griffin-pulls-trigger-133631229.html
Penny stocks, they divide market watchers like no other. Some investors steer clear of these tickers going for less than $5 apiece, as poor fundamentals or overwhelming headwinds could be keeping them down in the dumps.
On the other hand, penny stocks lure the more risk-tolerant. Not only does the bargain price tag mean you get more bang for your buck, but also even minor share price appreciation can yield huge percentage gains. The implication? Major returns for investors.
Based on the above, weeding out the long-term underperformers from the penny stocks going for gold can pose a significant challenge. In this case, the activity of legendary stock pickers can provide some inspiration.
Among these Wall Street titans is Ken Griffin. The guru, who began trading from his dorm room at Harvard University, went on to found hedge fund Citadel in 1990, with Griffin serving as CEO. Given that the fund now manages over $33 billion in capital, it’s no wonder investor focus locks in on Citadel when moves are made.
Currently, “we're in the moving business. So unless we think there's a very clear reason as to why an asset we own is going to appreciate soon, that's just not where we're going to be. And we drove our business away from balance-sheet intensive businesses to – in a sense – all skill-based businesses,” Griffin commented.
Taking all of this into consideration, we used TipRanks’ database to find out what the analyst community has to say about three penny stocks that Griffin's fund snapped up recently. It turns out that the analyst consensus has rated each a “Strong Buy." Not to mention substantial upside potential is also on the table.
Millendo Therapeutics (MLND)
Primarily focused on developing new treatments for endocrine diseases, Millendo Therapeutics wants to address the significant unmet medical needs of patients.
At $2.07, its share price could reflect the ideal entry point given everything the company has going for it.
Pulling the trigger for the first time, Griffen joined MLND’s growing list of fans. Recently, Citadel bought up 374,653 shares, with the value of this new holding coming in at just under $2 million.
Also singing the healthcare name’s praises is Roth Capital analyst Yasmeen Rahimi. At the beginning of May, MLND announced that it expects to have interim data in Q3 2020 from currently enrolled patients in the Phase 2b study evaluating nevanimibe (ATR-101), its ACAT1 inhibitor, in classic congenital adrenal hyperplasia (CAH), which was halted due to COVID-19. This development is especially exciting for the 5-star analyst as she believes success in this indication is very likely.
Currently, the only approved treatment option for patients with CAH are glucocorticoid and mineralocorticoid replacement therapies, but these can lead to overtreatment and growth retardation. “Thus, in our view, there remains an urgent need for interventions that can alleviate the serious health risks associated with cortisol deficiency and excessive androgen levels in CAH,” Rahimi said.
MLND tackles the condition from a different angle as “nevanimibe's mechanism of action (MOA) utilizes an elegant approach to CAH treatment by working upstream of these pathways and reducing the available reservoir of cholesterol esters, which are used in the synthesis of steroid hormones.” This allows for the suppression of excess steroid production as well as a more selective MOA.
Expounding on this, Rahimi noted, “In our view, nevanimibe’s selectivity gives it a major advantage in specifically reducing steroid synthesis in the adrenal cortex, which targets the root cause of CAH... Nevanimibe’s strength lies in its potential to offer an adrenal-targeted approach that can allow patients to better balance their control of excess androgen secretion with the required glucocorticoid replacement therapy.”
With data from nevanimibe’s Phase 2a trial demonstrating strong levels of both efficacy and safety, the deal is sealed for Rahimi.
To this end, the top analyst rates MLND a Buy along with a $6 price target. This target implies shares could climb 190% higher in the next twelve months. (To watch Rahimi’s track record, click here)
Looking at the consensus breakdown, most other analysts are on the same page. With 3 Buys and 1 Hold, the word on the Street is that MLND is a Strong Buy. The $5.25 average price target puts the upside potential at 153%. (See MLND stock analysis on TipRanks)
MEI Pharma (MEIP)
Hoping to build a top oncology franchise, MEI Pharma boasts a strong development pipeline that includes four compelling drug candidates.
Currently going for $3.49 apiece, the share price could present investors with the chance to get in on the action.
Not wanting to miss out on an opportunity, Citadel added a new MEIP holding to the fund. Griffin’s fund acquired 1,055,185 shares, with the value of the purchase landing at $1.7 million.
Turning now to the analyst community, Wells Fargo’s Jim Birchenough is also optimistic about MEIP’s long-term growth prospects. The 5-star analyst explains that a recent ASCO KOL investor event related to its lead candidate, ME-401, strengthened his bullish thesis.
“We continue to view ME-401 as a best-in-class PI3Kd inhibitor with superior safety and tolerability driving long-term dosing resulting in improved outcomes. We remain confident that the pivotal TIDAL follicular lymphoma (FL) trial will be positive and support product approval in 2021. The recent global alliance with Kyowa Kirin will accelerate development beyond 3rd line-plus FL into earlier stage disease and more broadly into other lymphomas in 2021,” Birchenough commented.
Looking at the trial’s design, it utilizes a 60mg once per day dose for two 28-day cycles, which is then followed by a 7-days on 21 days off intermittent dosing schema. This design is important as the intermittent dosing “mitigates against the risk for early discontinuation due to immune-related toxicity secondary to on-target inhibition of regulatory T cell function.” Additionally, should an 83% response rate be achieved, “both duration of disease and progression free survival will be superior to those for approved PI3K inhibitors, ZYDELIG, ALIQOPA and COPIKTRA.”
It should be noted that FL has a complicated treatment landscape. However, based on efficacy data for ME-401±rituximab, Birchenough argues that the therapy could be a second-line chemo-free option that’s more easily tolerated.
In line with his bullish take, Birchenough reiterated an Overweight call and $13 price target. Should the target be met, a twelve-month gain of 272% could be in store. (To watch Birchenough’s track record, click here)
Like Birchenough, other analysts also take a bullish approach. MEIP’s Strong Buy consensus rating breaks down into only Buys, 5, in fact. Given the $11.40 average price target, the upside potential lands at 204%. (See MEIP stock analysis on TipRanks)
Kezar Life Sciences Inc. (KZR)
Last but not least we have Kezar Life Sciences, which develops treatments for patients with autoimmune diseases and cancer. With encouraging new data supporting its most advanced asset, KZR-616, some think that its $4.96 share price looks like a steal.
This appears to be the stance taken by Griffin. Boosting its KZR holding by a whopping 1,338%, Citadel snapped up 859,760 shares. Now at 924,034 shares, the fund’s total stake in the company is valued at over $4 million.
Weighing in for H.C. Wainwright, 5-star analyst Raghuram Selvaraju says the recently published interim data from the Phase 1b part of the MISSION trial testing KZR-616 is incredibly promising. Pointing out this readout is most likely one of several data sets from interim analyses, he notes that “improvements were seen across seven measures of disease activity, and two out of two patients with lupus nephritis (LN) experienced a >50% reduction in proteinuria, a biomarker of disease severity.” On top of this, the candidate exhibited a robust safety and tolerability profile at the step-up dose, which could solve the tolerability issues produced by a higher dose.
Digging a bit deeper into the results, as of the data cutoff on May 4, the trial had 39 systemic lupus erythematosus (SLE) patients enrolled across five dose cohorts evaluating 45mg and step-up dosing to 60mg weekly for 13 weeks. Out of the patients that finished treatment in cohorts 2a and 2b, a majority saw all seven measures of disease activity improve.
Selvaraju added, “Improvement in disease activity persisted following completion of treatment, pointing to sustained impact with KZR-616. We are hopeful that this could prove a harbinger of disease-modifying potential.”
Even though COVID-19 has led to significant slowdowns and forced the company to fully pause some of its three KZR-616 studies, the situation may be improving soon, in Selvaraju’s opinion. KZR is still able to conduct trials at private clinics, and it already implemented an in-home nursing solution.
It should come as no surprise, then, that Selvaraju stayed with the bulls. In addition to keeping a Buy rating on the stock, the analyst bumped up the price target to $12. This new target suggests shares could soar 148% in the next year. (To watch Selvaraju’s track record, click here)
All in all, other analysts echo Selvaraju’s sentiment. 4 Buys and no Holds or Sells add up to a Strong Buy consensus rating. Based on the $15 average price target, which is more aggressive than Selvaraju’s, a twelve-month gain of 210% could be in the cards. (See KZR's stock-price forecast on TipRanks)
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>>> AstraZeneca is aiming to produce 2 billion doses of a coronavirus vaccine — and it could be ready by September
CNBC
JUN 5 2020
Lucy Handley
https://www.cnbc.com/2020/06/04/astrazeneca-is-set-to-make-two-billion-doses-of-a-coronavirus-vaccine.html
AstraZeneca plans to distribute 1 billion doses of a coronavirus vaccine to low and middle-income countries, with 400 million available this year.
In addition, the U.S. and U.K. are set to be sent 400 million vaccine doses between them, starting in September.
The vaccine, named AZD1222, was originally developed by Oxford University in the U.K.
Pharmaceutical company AstraZeneca is aiming to produce 2 billion doses of a coronavirus vaccine, including 400 million for the U.S. and U.K. and 1 billion for those in low- and middle-income countries.
It plans to start distributing the vaccine to the U.S. and U.K. in September or October, with the balance of deliveries likely to be made by early 2021, according to AstraZeneca CEO Pascal Soriot, on a call with journalists Thursday.
AstraZeneca said it had signed a licensing deal with the Serum Institute of India to provide 1 billion doses to low- and middle-income countries, with 400 million of those shots set to be delivered by the end of 2020, according to a press release Thursday.
The vaccine, named AZD1222, was originally developed by Oxford University in the U.K. and AstraZeneca is working with pharmaceutical industry partners to manufacture and distribute the drug.
Soriot said the distribution was dependent on clinical trials taking place by August. Clinical trials and manufacturing are set to occur concurrently, which is an unprecedented move for the pharmaceutical industry because of the risk of producing a drug that might not work.
“We are very focused and very committed. When you have something like this with this sort of pandemic and the tremendous impact it has on people, the economy, et cetera, you can’t second-guess what’s going to happen. You can’t spend your time figuring out is it going to work or not going to work, you just have to commit. … We come in and make a bet on some of these things,” Soriot said on the call.
AstraZeneca
?
@AstraZeneca
To further provide broad and equitable access to @UniofOxford potential #COVID19 vaccine, we've signed agreements with @CEPIvaccines, @gavi & @SerumInstIndia to supply low-and-middle income countries and beyond https://bit.ly/2Xv1Lu7
Oxford University’s Jenner Institute has worked with the Oxford Vaccine Group to develop the AZD1222 vaccine that includes a protein of the SARS CoV-2 virus strain, which causes the coronavirus disease known as Covid-19. It is currently testing the drug in around 10,000 adult volunteers. So far it has been “safe and well-tolerated,” according to AstraZeneca’s press release.
Asked whether the vaccine will work, Soriot said, “The chance of the vaccine working I would say we all have pretty good hope from what we’ve seen so far, but we can’t be sure of course,” adding that the company is creating a comprehensive database of safety information and expects to eventually have clinical trials with more than 50,000 volunteers taking part.
Trials are running concurrently with manufacturing to make the vaccine available as early as possible, according to Richard Hatchett, CEO of the Coalition for Epidemic Preparedness Innovations, which is working with AstraZeneca on the drug’s production. “Obviously, if the vaccine is successful, placing that early bet on the manufacturing gives a huge payoff because you end up with tens or even hundreds of millions of doses that become available at the earliest possible moment,” he said on the call with journalists Thursday.
“We believe we can get the vaccine to hundreds of millions of people around the world, importantly, including those in the countries with the lowest income. So our goal is really to not leave anybody behind,” Soriot said.
On Thursday, governments and businesses said they would give $8.8 billion to a vaccine alliance known as Gavi, which is backed by the Bill & Melinda Gates Foundation. The foundation and the World Health Organization have created a mechanism known as the Access to Covid-19 Tools Accelerator to make sure the vaccine is distributed fairly.
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Ken Griffith is bullish on MEIP. $13 price target.
https://finance.yahoo.com/news/billionaire-ken-griffin-pulls-trigger-133631229.html
Bladerunner
Oppenheimer & RBC initiate coverage of PRVB with $29 & $26 PT's, respectively.
Oppenheimer Releases a Buy Rating on Provention Bio (PRVB)
Catie Powers- June 15, 2020, 4:13 PM EDTSHARE ON:
Oppenheimer analyst Justin Kim assigned a Buy rating to Provention Bio (PRVB) today and set a price target of $29.00. The company’s shares closed last Monday at $15.64.
According to TipRanks.com, Kim is a 4-star analyst with an average return of 34.9% and a 55.6% success rate. Kim covers the Healthcare sector, focusing on stocks such as Aurinia Pharmaceuticals, Apellis Pharmaceuticals, and Aldeyra Therapeutics.
The word on The Street in general, suggests a Strong Buy analyst consensus rating for Provention Bio with a $26.00 average price target, implying an 86.0% upside from current levels. In a report issued on June 4, RBC Capital also initiated coverage with a Buy rating on the stock with a $25.00 price target.
The company has a one-year high of $18.50 and a one-year low of $4.72. Currently, Provention Bio has an average volume of 587.3K.
Based on the recent corporate insider activity of 20 insiders, corporate insider sentiment is positive on the stock. This means that over the past quarter there has been an increase of insiders buying their shares of PRVB in relation to earlier this year.
TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.
Provention Bio, Inc. is a clinical-stage biopharmaceutical company, which engages in the development and commercialization of novel therapeutics and solutions. Its products include PRV-031 for the interception of type 1 diabetes (T1D); PRV-015 for the treatment of gluten-free diet non-responding celiac disease; PRV-6527 for Crohn’s disease; PRV-300 for ulcerative colitis; PRV-3279 for the treatment of lupus; and PRV-101 for the prevention of acute coxsackie virus B (CVB), and the prevention of T1D onset. The company was founded by Francisco Leon and Ashleigh Palmer on October 4, 2016 and is headquartered in Lebanon, NJ.
Bladerunner
PRVB up 11% on Teplizumab data for T1D. Up another 3% in after hours. Presenting at ADA tomorrow.
Provention Bio up 11% on positive teplizumab data
Jun. 15, 2020 1:35 PM ET|About: Provention Bio, Inc. (PRVB)|By: Douglas W. House, SA News Editor
Provention Bio (PRVB +10.6%) is up on a 4x surge in volume in reaction to follow-up data from a Phase 2 study, "At Risk" TN-10, evaluating teplizumab for preventing/delaying the onset of type 1 diabetes (T1D) in relatives of type 1 diabetics deemed "at very high risk" of developing the condition.
Results showed that a single 14-day course of teplizumab significantly delayed the onset of T1D in presymptomatic patients by a median of three years compared to placebo.
In addition, declines in C-peptide levels (reductions indicate destruction of insulin-producing beta cells in the pancreas) in treated patients stabilized than reversed, suggesting possible restoration of insulin production by these cells.
Treatment with teplizumab resulted in 54% less risk of progressing to insulin-dependent T1D compared to placebo.
No new safety signals were observed.
The company expects to complete its U.S. marketing application in Q4.
Teplizumab is a CD3-targeted monoclonal antibody designed to slow the loss of insulin-producing beta cells in the pancreas while preserving beta cell function as measured by C-peptide.
Bladerunner
BrainStorm Granted SME Status by the European Medicines Agency
June 15 2020 - 09:00AM
GlobeNewswire Inc. Print
BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that it has been granted Small and Medium-Sized Enterprise (SME) status by the European Medicines Agency’s (EMA) Micro, Small and Medium-Sized Enterprise (SME) office.
The newly granted SME status allows BrainStorm to participate in significant financial incentives that include a 90% to 100% EMA fee reduction for scientific advice, clinical study protocol design, endpoints and statistical considerations, quality inspections of facilities as well as fee waivers for selective EMA pre and post-authorization regulatory filings, including Orphan Drug and PRIME designations.
Brainstorm is also eligible to obtain EMA certification of quality and manufacturing data prior to review of clinical data. Other incentives include EMA-provided translational services of all regulatory documents required for market authorization, further reducing the financial burden of the market authorization process.
“This is an additional and timely step in our global strategy as we continue to engage with the EMEA,” said David Setboun, Pharm.D, MBA, Chief Operating Officer of BrainStorm. “Our ALS investigational product received orphan drug status in Europe in July 2013 and we will benefit from the enhanced interaction and early dialogue allowing us to optimize development plans and speed up evaluation in Europe. EMA is facilitating pathways such as PRIME designation that enables critical medicines for major unmet medical need like NurOwn® to reach patients earlier.”
The EMA plays a central role in facilitating the development and authorization of medicines across Europe. The SME initiative promotes innovation from smaller companies such as BrainStorm to ensure Europe continues to be a favorable environment for preclinical and clinical development of promising new therapeutic options like NurOwn.
https://finance.yahoo.com/news/brainstorm-granted-sme-status-european-130010725.html
PRVB updates data showing significant delay in the onset of T1D
June 15, 2020 12:02 PM
PROVENTION BIO'S TEPLIZUMAB CONTINUED TO SIGNIFICANTLY DELAY THE ONSET OF INSULIN-DEPENDENT TYPE 1 DIABETES (T1D) IN PRESYMPTOMATIC PATIENTS
-One Course of Teplizumab Now Shown to Delay Insulin-Dependent T1D by a Median of Approximately Three Years Compared to Placebo- -Teplizumab Significantly Reversed the Decline in C-Peptide Levels Providing Further Evidence of Disease-Modifying Effect- -Extended Follow-up Results from the Pivotal "At-Risk" TN-10 Study Presented at the American Diabetes Association's 80th Scientific Sessions-
OLDWICK, N.J., June 15, 2020 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced extended follow-up data showing a single 14-day course of teplizumab (PRV-031) significantly delayed the onset of insulin-dependent type 1 diabetes (T1D) in presymptomatic patients by a median of approximately three years compared to placebo. These new data from the pivotal "At-Risk" TN-10 Study add one year to the two-year median delay that was previously reported in the New England Journal of Medicine and presented at last year's American Diabetes Association's (ADA's) Scientific Sessions. Teplizumab, Provention's lead drug candidate, is an anti-CD3 monoclonal antibody in development for the delay or prevention of insulin-dependent T1D in presymptomatic patients, defined by the presence of two or more T1D-related autoantibodies and dysglycemia.
The study was conducted by TrialNet, a network of the world's leading T1D researchers, and funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The findings were presented today at the ADA's 80th Scientific Sessions.
"Continuing to delay the onset of clinical-stage T1D with just a single course of teplizumab, now by approximately three years as compared to placebo, is profoundly relevant for patients facing a lifetime of insulin dependency, glucose monitoring, and lifestyle challenges to survive," said Ashleigh Palmer, CEO of Provention Bio. "We understand the urgency in fundamentally changing the progression of T1D in early-stage disease - before clinically relevant beta cell loss occurs - and remain steadfast in our commitment to bring teplizumab to the T1D community as quickly as possible."
Additional findings show C-peptide levels, a measure of a person's own insulin production, decreased in the placebo group, underscoring the destruction in beta cells that characterizes this disease. In contrast, teplizumab treatment stabilized and then significantly reversed the decline of C-peptide levels, suggesting a delay in the destruction of beta cells and restoration of insulin production by dysfunctional beta cells. Importantly, even in those who advanced to clinical-stage T1D, treatment with teplizumab resulted in a slower decline in C-peptide levels compared to placebo.
"C-peptide levels were stabilized and even improved with teplizumab treatment. We are highly encouraged to see these results that confirm beta cell function is being preserved and suggest that available beta cell function is being restored," said Colin Dayan, MA MBBS, FRCP, Ph.D., Professor of Clinical Diabetes and Metabolism, Cardiff University School of Medicine, who was not involved with the TN-10 study. "These data provide further evidence that, as a disease-modifying immunotherapy, teplizumab appears to be treating the root cause of T1D autoimmunity and provides potential benefit across several stages of the disease."
Presentations Highlights
The median time to clinical diagnosis of T1D after one course of teplizumab was approximately five years (an improvement of one year from previously published data) compared to approximately two years for the placebo group (unchanged from previously published data). Compared to placebo, teplizumab treatment resulted in a 54% reduction in risk of progressing to insulin-dependent T1D (hazard ratio 0.457, p=0.01). During this extended follow-up, nearly half of those treated with teplizumab are estimated to be free of clinical-stage disease at five years.
Teplizumab treatment was associated with a greater on-study C-peptide (p=0.009) compared to placebo. For both groups, C-peptide mean slopes preceding study entry were similar and declining. In the placebo group, this decline continued over the 6 months after study entry. By contrast, the teplizumab-treated group showed an increased C-peptide over this period (p=0.02 relative to study entry).
Teplizumab was well tolerated and the safety data is consistent with previous analyses.
Mr. Palmer added, "We remain on track to complete the Biologics License Application submission for teplizumab for the delay or prevention of insulin-dependent T1D in presymptomatic patients to the U.S. Food and Drug Administration (FDA) in the fourth quarter of this year. We look forward to working with the FDA as we advance the regulatory process under our Breakthrough Therapy designation."
About the Pivotal "At-Risk" TN-10 Study:
The "At-Risk" TN-10 Study, a pivotal Phase 2 clinical trial, evaluated teplizumab for the delay of insulin-dependent type 1 diabetes (T1D) in presymptomatic patients, defined by the presence of two or more T1D-related autoantibodies and dysglycemia (abnormal glucose metabolism). Seventy-six patients were enrolled ages 8 to 49, with 72% under the age of 18, and randomized to receive a single course of either teplizumab or placebo. Patients were followed in a blinded fashion until 40 of them developed clinical-stage T1D, and then indefinitely after the analysis of the randomized period data.
About Type 1 Diabetes (T1D):
Over 1.6 million Americans have type 1 diabetes (T1D), an autoimmune disease caused by the destruction of beta cells. T1D symptoms can take months or years to develop. The psychological impact of T1D is hard to quantity, but a diagnosis is life-altering, and regular monitoring and maintenance can be extremely stressful. T1D typically takes more than a decade off a person's life and life expectancy is reduced by 16 years on average for people diagnosed with T1D before the age of 10. Insulin is the current T1D treatment. It is necessary to keep patients alive, but it is a constant effort for patients. No disease-modifying treatments for T1D are currently available.
About Teplizumab (PRV-031):
Teplizumab is an anti-CD3 monoclonal antibody (mAb) being developed for the interception, delay, or prevention of type 1 diabetes (T1D). More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab has consistently demonstrated the capability of preserving beta-cell function and reducing the need for exogenous insulin usage. Teplizumab has been granted Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and PRIME designation by the European Medicines Administration. Provention Bio has initiated a rolling submission of the Biologic License Application for teplizumab for the delay or prevention of insulin-dependent T1D for use in presymptomatic patients and expects to complete the submission in Q4 2020. Provention is currently evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT Study).
About Provention Bio, Inc.:
Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company leveraging a transformational drug development strategy focused on the prevention or interception of immune-mediated disease. Provention's mission is to source, transform and develop therapeutic candidates targeting the high morbidity, mortality and escalating costs of autoimmune diseases. Provention's diversified portfolio includes teplizumab, a pre-commercial-stage candidate that has been shown to delay the onset of insulin-dependent type 1 diabetes (T1D) in at-risk patients during the presymptomatic phase of the disease. The Company's portfolio includes additional clinical product development candidates that have demonstrated proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus.
Bladerunner
>>> Coronavirus update: US reopening dogged by fears of new wave as pharma presses on with vaccine
by Anjalee Khemlani
Yahoo Finance
June 11, 2020
https://finance.yahoo.com/news/coronavirus-update-us-reopening-dogged-by-fears-of-new-case-spikes-as-pharma-presses-on-with-vaccine-180220247.html
A sudden spike in new coronavirus cases across key U.S. states put markets back on the defensive on Thursday, worrying public health experts as more regions continue to make tentative steps toward reawakening their economies.
The global case count is nearing 7.5 million as more than 477,000 have died. This week, the world’s largest economy set a new milestone of 2 million cases, with deaths climbing past 113,000.
Memorial Day weekend, and growing public gatherings since then as summer approaches, have punctuated a concerning trend of rising COVID-19 hospitalizations and deaths in at least a dozen states. Texas and Florida, two of the U.S.’s most highly populated places, are among the most concerning cases — as is Arizona, which is also experiencing a jump in its daily counts.
Until recently, the U.S. trend had been leveling off, but is now starting to reflect the impact of what some fear may be a second wave — even though the daily count is largely flat at 20,000 new per day. That figure is hiding spikes that are worrying experts, just as New York and New Jersey — the two hardest-hit states — begin relaxing their lockdowns.
While increased testing has played a role in the rising case count, health officials are nervous about the increase in hospitalizations. While some reports classify these increases as a second wave, experts consider this part of the first wave as it was expected to hit different parts of the country at different times.
The relaxation of stay-at-home orders come amid fears that the virus persistence will undermine a nascent economic rebound, upsetting investors and fanning speculation that the U.S. could enter another lockdown.
In a briefing Wednesday, Federal Reserve Bank chair Jerome Powell said at least 15 million Americans will remain unemployed through 2020, even as jobless claims show signs of leveling off.
Treatments and vaccines advancing
Globally, policymakers and health experts are pinning their hopes on a vaccine, which is unlikely to materialize before next year.
The frontrunner in the U.S., Moderna (MRNA), announced Thursday it is on track to start Phase 3 trials in July. Meanwhile, Dr. Paul Stoffels, Johnson & Johnson (JNJ) Chief Scientific Officer, told Yahoo Finance on Thursday that the company also has an aggressive hopes for its COVID-19 vaccine.
The speed with which Moderna has advanced, and its meeting of endpoints and phases, has helped boost confidence in a vaccine being developed in potentially record time.
At the very least, the treatment could get an emergency use authorization by the end of the year. Indications of a successful vaccine are expected in September, at the earliest. And Eli Lilly (LLY) is reportedly eyeing September as the earliest one of its antibody treatments, currently in testing, could be authorized for emergency use.
Meanwhile, Regeneron (REGN) announced it was entering into clinical trials for a cocktail of antibody and antiviral treatments against Covid-19, Thursday.
That includes four different groups of participants being tested— hospitalized patients, symptomatic individuals who are not in the hospital, uninfected individuals on the front lines as well as uninfected individuals who live with a coronavirus patient.
George Yancopoulos, Regeneron’s cofounder, president and Chief Scientific Officer, said in a statement that the cocktail addresses two key issues with the virus.
The cocktail “could have a major impact on public health by slowing spread of the virus and providing a needed treatment for those already sick – and could be available much sooner than a vaccine,” he said, adding that the “world needs multiple solutions” to the COVID-19 crisis.
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Delirium, depression, anxiety, PTSD – the less discussed effects of COVID-19
Obviously, we don’t have long-term data for COVID-19, but looking at past epidemics can give us some idea of what to look out for in the next few months and years. When patients with Sars and Mers were assessed a few months later, 15% had depression and 15% had an anxiety disorder. Mental illness is common, though, so it’s hard to know how many of these people had longstanding problems or were struggling for other reasons.
What was really striking, though, was that rates of post-traumatic stress disorder (PTSD) were more than 30%. This is huge and hard to attribute to anything except the experience of the infection and its treatment. Fatigue also seemed to be a big problem: 19% of patients were still reporting this even months after “recovering”.
https://theconversation.com/delirium-depression-anxiety-ptsd-the-less-discussed-effects-of-covid-19-138671
IMHO one of the companies to benefit is BWAY
Results of Phase 3 Trial of NurOwn Cell Therapy Expected by End of Year
JUNE 5, 2020 Patricia Inacio, PhDBY PATRICIA INACIO, PHD IN NEWS.
Results of Phase 3 Trial of NurOwn Cell Therapy Expected by End of Year
The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced.
“BrainStorm made the decision to continue our Phase 3 trial and we have been strongly supported by our partners in the clinical community. Quick planning and coordination across our clinical sites and the support and direction of the U.S. FDA enabled our Phase 3 investigational trial for NurOwn to advance, with final dosing by July 2020,” Chaim Lebovits, CEO of BrainStorm, said in a press release.
The Phase 3 trial is fully enrolled with 200 participants across six sites in the United States, including the University of California, Irvine; Cedars-Sinai Medical Center, Los Angeles, California; California Pacific Medical Center; Massachusetts General Hospital, Boston; University of Massachusetts Medical School, and the Mayo Clinic.
All patients have received at least two of three NurOwn doses and top-line results will be announced later this year.
“Despite the challenges presented by COVID-19, patient dosing remains on track with all participants having received at least 2 treatments of NurOwn,” said Merit Cudkowicz, professor at Harvard Medical School and the director of the Healey Center for ALS and chair of Neurology at Massachusetts General. “Moving forward, only 20 patients are still to be dosed for the third and final time, and these final treatments remain on schedule.”
NurOwn is a cell-based therapy that uses mesenchymal stem cells (MSCs), which are extracted from a patient’s own bone marrow. These cells have the capacity to generate different cell types.
After extraction, MSCs are expanded in the lab and matured into cells that produce high levels of neurotrophic factors — compounds that promote nervous tissue growth and survival. The converted cells are then reintroduced into the body via an injection either into muscles or the spinal canal.
In a Phase 2 trial (NCT02017912) NurOwn was determined to be safe and to significantly slow disease progression in a subset of ALS patients with fast-progressing disease.
The current Phase 3 trial (NCT03280056) is assessing the safety and effectiveness of three administrations of NurOwn into the spinal canal when compared to a placebo, given every two months. The trial’s primary goal is to confirm the effectiveness of NurOwn as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R), a score of abilities such as swallowing, speech, handwriting, or walking.
During the COVID-19 pandemic, coordination between the trial’s clinical sites and the U.S. Food and Drug Administration allowed the trial to keep moving forward with only occasional scheduling changes to treatments. Moreover, the fact that the trial’s primary outcome measurement — ALSFRS-R — is validated by phone, allowed researchers to continue to collect data without exposing patients to potential COVID-19 infection.
“We are very fortunate to have developed an outstanding team of partners who are committed to BrainStorm’s investigational therapy NurOwn and share its promise in ALS research. This, along with the collective efforts of so many of our employees, partners, and patients, has enabled us to remain on track for study completion as planned in the fourth quarter of this year,” said Ralph Kern, MD, president and chief medical officer of BrainStorm.
https://alsnewstoday.com/2020/06/05/phase-3-trial-of-nurown-cell-therapy-for-als-on-track-and-results-by-end-of-year/
>>> AstraZeneca Approaches Gilead About Potential Merger
Bloomberg
By Ed Hammond, Aaron Kirchfeld, and Dinesh Nair
June 7, 2020
https://www.bloomberg.com/news/articles/2020-06-07/astrazeneca-is-said-to-approach-gilead-about-potential-merger?srnd=premium
U.K. drugmaker contacted Gilead last month; no formal talks
Deal with $96 billion U.S. firm would be record in health care
AstraZeneca Plc has made a preliminary approach to rival drugmaker Gilead Sciences Inc. about a potential merger, according to people familiar with the matter, in what would be the biggest health-care deal on record.
The U.K.-based firm informally contacted Gilead last month to gauge its interest in a possible tie-up, the people said, asking not to be identified because the details are private. AstraZeneca didn’t specify terms for any transaction, they said. While Gilead has discussed the idea with advisers, no decisions have been made on how to proceed and the companies aren’t in formal talks, the people added.
AstraZeneca, valued at $140 billion, is the U.K.’s biggest drugmaker by market capitalization and has developed treatments for conditions from cancer to cardiovascular disease. Gilead, worth $96 billion at Friday’s close, is the creator of a drug that’s received U.S. approval for use with coronavirus patients.
Gilead is not currently interested in selling to or merging with another big pharmaceutical company, preferring instead to focus its deal strategy on partnerships and smaller acquisitions, the people said. A representative for Gilead declined to comment. A spokesman for AstraZeneca said the company doesn’t comment on “rumors or speculation.”
Coronavirus Treatment
The overtures show how the pharmaceutical industry landscape could shift at a time when drugmakers are racing to find effective treatments for Covid-19. If a deal goes ahead, it would surpass Bristol-Myers Squibb Co.’s $74 billion takeover of Celgene Corp. last year as the biggest-ever health-care acquisition, according to data compiled by Bloomberg. It would also rank among the 10 biggest M&A transactions of all time.
Shares of AstraZeneca have risen about 41% over the past 12 months, making it the best performer on a Bloomberg Intelligence index of major Western pharmaceutical companies. Shares of Gilead gained about 19% over the period.
The 10 Largest Health-Care and Pharma Deals -
Celgene / Bristol-Myers Squibb (2019) - 87.6
Warner-Lambert / Pfizer (1999) - 87.3
Allergan / AbbVie (2019) - 83.8
Shire / Takeda (2018) - 81
Aventis / Sanofi (2004) - 73.5
SmithKline Beecham / Glaxo (2000) - 72.5
Express Scripts / Cigna (2018) - 68.4
Aetna / CVS (2017) - 68.1
Allergan / Actavis (2014) - 65
Pharmacia / Pfizer (2002) - 64.3
Source: Bloomberg Deal values include debt
Gilead has attracted investor interest as its antiviral drug for Covid-19, remdesivir, worked its way through clinical trials in recent months. The stock is still more than a third lower than its 2015 highs. The Foster City, California-based company has seen a steady decline in sales in its hepatitis C franchise and is trying to reinvigorate its drug-development pipeline.
Remdesivir, which has an emergency use authorization from the U.S. Food and Drug Administration, has been shown in some early studies to shorten hospital stays for people with Covid-19. SVB Leerink recently forecast that sales of the drug may reach $7.7 billion in 2022.
Tamiflu Developer
Gilead has been dispensing early rounds of the drug for free, leading some investors to question how the company plans to make money from it in the future. Chief Executive Officer Daniel O’Day has said the company may spend $1 billion on the treatment this year alone.
AstraZeneca is helping to manufacture a Covid vaccine developed at the University of Oxford. The U.S. has pledged as much as $1.2 billion to support the efforts as part of Operation Warp Speed, a push to secure vaccines for America. The shot is expected to enter final-stage clinical trials in June.
Gilead was founded in 1987 by Michael Riordan, a doctor with a Harvard MBA who aimed to discover treatments for viral infections after a bout with dengue fever acquired in southeast Asia. The company’s best-known successes include Tamiflu, the influenza treatment it helped develop.
The company also makes Truvada, a medicine that can help prevent HIV, as well as drugs for liver disease and inflammation. Gilead employs about 12,000 people, according to its website.
AstraZeneca is no stranger to large-scale, politically sensitive M&A. In 2014 it fended off a $117 billion approach from Pfizer Inc., a deal that attracted attention from U.S. lawmakers as it would have allowed New York-based Pfizer to lower its tax bill by redomiciling in the U.K.
Deal Slump
Health-care dealmaking has been a rare bright spot as the global pandemic and resulting lockdowns have doused the market for mergers and acquisitions. Global M&A volumes are down about 45% this year, according to data compiled by Bloomberg, and announced deals have been falling apart at a steady pace.
Excluding minority investments, dealmaking in April and May barely topped $100 billion in total, the data show, the lowest two-month period in at least 22 years.
AstraZeneca CEO Pascal Soriot, a former executive at oncology specialist Roche Holding AG, has transformed the company since taking the helm nearly eight years ago. At the time, it was struggling with an aging stable of drugs and a shortage of innovation.
He’s championed the development of Lynparza, which was initially approved for ovarian cancer but has also proved useful for treating other forms of the disease. AstraZeneca has since overtaken U.K. rival GlaxoSmithKline Plc in market value.
Last year, AstraZeneca sealed its biggest transaction in more than a decade, agreeing to pay as much as $6.9 billion to buy into a promising breast cancer treatment developed by Japanese drugmaker Daiichi Sankyo Co. The U.K. company reached a deal this month with Accent Therapeutics Inc. to potentially spend more than $1.1 billion collaborating on novel oncology therapies.
AstraZeneca shares have also been boosted by positive data from trials of its blockbuster lung cancer drug Tagrisso.
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re: MYOV
I established a position at $15.90. Comments?
Bladerunner
MEIP presents data at ASCO on its FOURTH clinical candidate for HER2-negative breast cancer. Comments welcome.
MEI Pharma Presents Clinical Results for ME-344 in Combination with Bevacizumab in Early HER2 Negative Breast Cancer Patients at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting
- Statistically significant biologic anti-tumor activity demonstrated as measured by a reduction in Ki67 in patients treated with ME-344 compared to an increase in patients receiving placebo -
MEI Pharma, Inc.
Jun 01, 2019, 09:00 ET
SAN DIEGO, June 1, 2019 /PRNewswire/ -- MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing potential new therapies for cancer, today announced data presented at ASCO 2019 from an investigator-initiated study of investigational ME-344 in combination with bevacizumab (marketed as Avastin®) in patients with early HER2-negative breast cancer. This study demonstrated proof of biologic anti-tumor activity as measured by a statistically significant reduction in Ki67, a measure of cell proliferation that is highly correlated with tumor response, in the group of patients treated with ME-344 compared to an increase in the group receiving saline.
"Data from this clinical study of ME-344 in combination with bevacizumab represents a potential novel approach to disrupting tumor metabolism and limiting tumor proliferation by inhibiting the heightened energy production necessary for cell division and cancer growth," stated the study principal investigator, Miguel Quintela-Fandino, M.D., Ph.D., Director of the Clinical Research Program, Centro Nacional De Investigaciones Oncologicas, Madrid, Spain. "These results offer evidence for the biologic antitumor activity of ME-344 in certain metabolic contexts and support further exploration of the mitochondrial inhibitor ME-344 in a therapeutic role, providing a potential novel mechanism that may improve patient outcomes in combination with antiangiogenic therapeutics such as bevacizumab."
The ME-344 ASCO 2018 poster can be accessed on the MEI Pharma website.
ME-344 Clinical Data
The clinical study was a multicenter, investigator-initiated, randomized, open-label trial evaluating ME-344 in a total of 42 patients with early HER2-negative breast cancer in combination with the vascular endothelial growth factor inhibitor bevacizumab. Patients were randomized one-to-one to either ME-344 plus bevacizumab or saline plus bevacizumab.
The primary objective of the study was to show proof of ME-344 biologic activity as measured by Ki67 reductions from day 0 to 28 compared to placebo. Secondary objectives included determining whether ME-344 biologic activity correlates with vascular normalization. The data demonstrate significant biologic activity in the ME-344 treatment group:
In ME-344 treated patients, mean absolute Ki67 decreases were 13.3 compared to an increase of 1.1 in the bevacizumab monotherapy group (P=0.01).
In ME-344 treated patients, mean relative Ki67 decreases were 23% compared to an increase of 186% in the bevacizumab monotherapy group (P < 0.01).
The mean relative Ki67 reduction in patients experiencing vascular normalization in the ME-344 treated patients was 33%, compared to an increase of 11.8% in normalized patients from the bevacizumab monotherapy group (P=0.09). Approximately one-third of patients in each arm had vascular normalization.
Treatment was generally well tolerated; two Grade 3 adverse events of high blood pressure were reported, 1 in each arm, and deemed related to bevacizumab.
About ME-344
ME-344 is a novel, tumor selective, isoflavone-derived mitochondrial inhibitor drug candidate. It directly targets the OXPHOS complex 1, a pathway involved in the production of adenosine triphosphate, or ATP, in the mitochondria. Treatment of tumor cells with ME-344 results in a rapid loss of ATP and cancer cell death. ME-344 demonstrated evidence of single-agent activity against refractory solid tumors in a Phase I study, and in preclinical studies, tumor cells treated with ME-344 resulted in a rapid loss of ATP and cancer cell death.
In addition to single-agent activity, ME-344 may also have potential in combination with antiangiogenic therapeutics. While antiangiogenics reduce the rate of glycolysis in tumors as a mechanism to block growth, tumor metabolism often shifts to mitochondrial metabolism to continue energy production to support continued tumor proliferation. In such cases of tumor plasticity in the presence of treatment with antiangiogenics, targeting the alternative metabolic source with ME-344 may open an important therapeutic opportunity.
About MEI Pharma
MEI Pharma, Inc. (Nasdaq: MEIP) is a late-stage pharmaceutical company focused on developing potential new therapies for cancer. Our portfolio of drug candidates contains four clinical-stage assets, including one candidate in an ongoing global registration trial and another candidate in a Phase 2 clinical trial which may support an accelerated approval marketing application with the U.S. Food and Drug Administration. Each of our pipeline candidates leverages a different mechanism of action with the objective of developing therapeutic options that are: (1) differentiated, (2) address unmet medical needs and (3) deliver improved benefit to patients either as standalone treatments or in combination with other therapeutic options. For more information, please visit www.meipharma.com.
Bladerunner
MEIP crashes through its 52-week high today. Holding an analyst day tomorrow.
Bladerunner
>>> Novavax Jumps After Starting Covid-19 Vaccine Study
Bloomberg
By Cristin Flanagan
May 26, 2020
Vaccine to be tested on 130 healthy volunteers in Australia
Biotech’s vaccine is one of an estimated 10 being tested
https://www.bloomberg.com/news/articles/2020-05-25/novavax-starts-covid-19-study-and-targets-first-results-in-july?srnd=premium
Novavax Inc. jumped at much as 18% on Tuesday after initiating testing of its coronavirus vaccine candidate in people. The biotech anticipates providing a first look in July at what sort of immune responses are generated.
In the first part of the study, 130 healthy adult volunteers at two sites in Australia will get two doses of NVX-CoV2373, the biotech’s experimental vaccine. If the initial results look promising, the company plans to quickly move into the second phase of the trial, which would expand testing to other countries and age groups outside of 18 to 59.
Novavax’s valuation has swelled by more than 1,000% to about $2.7 billion since the start of the year despite having no products on the market. With about $388 million in funding from the Oslo-based Coalition for Epidemic Preparedness Innovations in its pocket, Novavax has said it is targeting emergency-use authorization and production could be scaled up to 100 million doses by year end.
Novavax among biotech stocks rallying on vaccine hopes
“Our investment in Novavax allows us to focus on manufacturing in parallel with the clinical development of the vaccine, so that if the vaccine is proven to be safe and effective, we can make doses available to those who need them without delay,” CEPI chief executive Richard Hatchett, said in a statement.
The Gaithersburg, Maryland-based company is one of an estimated 10 that have begun testing vaccines for the pandemic in people, according to the World Health Organization. WHO estimates there are more than 100 other vaccine candidates in earlier stages of development. Moderna Inc. reported the first Covid-19 vaccine results in humans last week.
Smaller vaccine developers are racing alongside larger, profitable drugmakers like Pfizer Inc., AstraZeneca Plc. as well as academic institutions. Even if a vaccine is eventually shown to be effective, there will be hurdles to manufacturing as well as distribution.
With Novavax shares at the highest in over a week, B Riley FBR’s Mayank Mamtani said the stock could return to its May 15 record high, the analyst recommended buying shares ahead of the July results.
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>>> Gamida Cell Ltd. (GMDA), a clinical stage biopharmaceutical company, focuses on developing cell therapies to cure cancer, and rare and serious hematologic diseases. The company's lead product candidate is NiCord, a nicotinamide (NAM)-expanded cord blood cell therapy that is in Phase III studies in patients with high-risk hematologic malignancies, as well as in Phase I/II studies in patients with severe aplastic anemia. It is also developing NAM-NK, an innate immunotherapy, which is in Phase I studies for the treatment of relapsed or refractory non-Hodgkin lymphoma and multiple myeloma. The company was founded in 1998 and is headquartered in Jerusalem, Israel.
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>>> BiondVax Pharmaceuticals Ltd. (BVXV), a clinical stage biopharmaceutical company, focuses on developing and commercializing immunomodulation therapies for infectious diseases primarily in Israel. Its product candidate is M-001, a synthetic peptide-based protein, which is in Phase III clinical trials that is used for treating seasonal and pandemic strains of the influenza virus. The company was founded in 2003 and is headquartered in Jerusalem, Israel. <<<
>>> Preliminary Data from NIH/NIAID-sponsored Phase 2 Clinical Trial of BiondVax’s M-001 Universal Influenza Vaccine Candidate Validates Results of Previous Clinical Trials
https://www.biondvax.com/2020/02/preliminary-data-from-nih-niaid-sponsored-phase-2-clinical-trial-of-biondvaxs-m-001-universal-influenza-vaccine-candidate-validates-results-of-previous-clinical-trials/
Data published on Clinicaltrials.gov
Jerusalem, Israel – February 5, 2020 – BiondVax Pharmaceuticals Ltd. (Nasdaq: BVXV) announced today that preliminary data from the Phase 2 clinical trial of BiondVax’s M-001 universal influenza vaccine candidate have been published. The trial was supported by the U.S. National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). The primary objectives of the trial were to assess safety and T-cell responses to M-001. The data, which are consistent with results of previous clinical trials of M-001, indicate that both primary objectives were achieved. Analysis of the data is ongoing, and the clinical study report (CSR) is expected in Q2 2020.
Dr. Tamar Ben-Yedidia, BiondVax’s Chief Scientific Officer, commented, “We are grateful for the opportunity to collaborate with NIAID and thank them for their professionalism and partnership. This was M-001’s first clinical trial in the US under the FDA’s Investigational New Drug program (IND). Data from this Phase 2 trial confirm results from six previously completed clinical trials of M-001. BiondVax’s universal flu vaccine candidate was found to be safe, well tolerated and induce statistically significant cellular immune responses.”
The Phase 2 clinical trial was conducted by NIAID-funded Vaccine and Treatment Evaluation Units (VTEUs). The trial enrolled 120 participants aged 18 to 49 years at the following VTEU sites: Baylor College of Medicine in Houston, Texas; the University of Iowa in Iowa City, Iowa; and Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio. Individuals were randomly assigned to receive either two doses of M-001 or two doses of a placebo, with the doses spaced three weeks apart. Later, near the beginning of the 2018/19 flu season, all participants were immunized with a currently marketed quadrivalent seasonal influenza vaccine. Laboratory analyses of vaccine immunogenicity were performed at the Baylor College of Medicine and Saint Louis University (St. Louis, Missouri) VTEU sites and at BiondVax Pharmaceuticals.
The published data are available at
https://clinicaltrials.gov/ct2/show/NCT03058692. The trial was supported through NIAID awards #HHSN272201300016I, HHSN272201300015I, HHSN272201300020I, and HHSN272201300021I.
In parallel, BiondVax’s pivotal, clinical efficacy, Phase 3 trial in Europe, involving 12,463 older adults, is ongoing. Results of that trial are expected by the end of 2020.
About BiondVax
BiondVax (NASDAQ: BVXV) is a Phase 3 clinical stage biopharmaceutical company developing a universal flu vaccine. The vaccine candidate, called M-001, is designed to provide multi-strain and multi-season protection against current and future, seasonal and pandemic influenza. BiondVax’s proprietary technology utilizes a unique combination of conserved and common influenza virus peptides intended to stimulate both arms of the immune system for a cross-protecting and long-lasting effect. In a total of 7 completed Phase 1/2 and Phase 2 clinical trials enrolling 818 participants, the vaccine has been shown to be safe, well-tolerated, and immunogenic. The ongoing pivotal Phase 3 clinical trial aims to assess safety and effectiveness of M-001 in reducing flu illness and severity. For more information, please visit www.biondvax.com.
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SunTrust Robinson Humphrey initiates coverage of MEIP with $16 PT.
Bladerunner
>>> Baker Brothers Snap Up These 3 Small-Cap Biotech Stocks
TipRanks
May 26, 2020
https://finance.yahoo.com/news/baker-brothers-snap-3-biotech-144317766.html
According to strategists from J.P. Morgan, who have been tracking biotech fund flows to gauge interest levels, the volatility exhibited over the last few years has persisted in 2020, but for now, flows have taken off in a single direction.
Momentum in the biotech space has fueled the charge forward, with the fund flow winning streak now at 8 weeks. On top of this, as of May 21, the capital taken in by the IBB ETF quarter-to-date has already hit more than $2 billion, and the material gain for this period lands at $289 million.
It should come as no surprise, then, that investor focus has locked in on the biotech sector. However, given the industry’s volatile reputation, market watchers are turning to the best in the biotech stock picking business. Enter the Baker Brothers.
Brothers Julian and Felix Baker have earned guru-like status on Wall Street, with the track record to back it up. Baker Bros. Advisors, the hedge fund founded by the brothers in 2000 that focuses primarily on biotech companies, grew $250 million in managed assets in 2003 into $16.2 billion worth of assets as of May 15, 2020. Not to mention back in October 2019, the fund raked in a $1.4 billion gain in just two weeks after positive catalysts for two of its holdings, Seattle Genetics and BeiGene, sent shares skyrocketing.
Looking to the legendary brothers for inspiration, we scanned the fund’s recent 13F filing to see if its activity would point us in the right direction, and zeroed in on three biotech stocks in particular. Running the tickers through TipRanks’ database, we found out that each has received Buy ratings from analysts and sports some impressive upside potential.
BioDelivery Sciences (BDSI)
Subscribing to a patients-first philosophy, BioDelivery Sciences focuses on developing cutting-edge therapies for patients with serious and debilitating chronic conditions. Based on the strength of its Belbuca product, its drug designed to help manage severe pain, this biotech has scored several fans.
Among those singing BDSI’s praises are the Baker brothers. Acquiring a new stake in the company, Baker Bros. Advisors snapped up 1,638,334 shares valued at $8.07 million.
Representing Cantor, analyst Brandon Folkes also likes what he’s seeing. Following its Q1 2020 earnings release, he tells clients that the primary driver of his bullish thesis is Belbuca.
Looking at Belbuca’s performance in the most recent quarter, the drug generated net sales of $33.5 million, a 79% increase from the prior-year quarter. In addition, total Belbuca Rxs came in at 99,486, with NBRx at 7.3%. As for total Rxs, they were up 52%, and Folkes points out that the net revenue per Belbuca Rx exceeded his original forecast. BDSI also notched a 28% year-over-year gain in unique prescribers.
During the quarter, coverage for Belbuca grew after it added more than 2 million Medicare patients. It also expanded coverage in two regional commercial plans, representing 900,000 patients. Not to mention Folkes argued, “With a strong balance sheet we believe BDSI is well positioned to continue to drive growth in 2020 and beyond.”
The story for BDSI doesn’t end there. The company’s other product, Symproic, also delivered a solid quarterly performance, with it acquiring 1,170 new prescribers. Adding to the good news, total Rxs reached 16,137, up 19% from the prior-year quarter. It was able to achieve an 11.7% total RX share and 13.3% net Rx share.
Folkes concluded, “We have viewed BDSI as a Belbuca execution story, and the company continues to exceed its execution goals as well as our expectations time after time for Belbuca and now has started to do the same with Symproic. BDSI continues to execute on its commercial plan to drive continued growth of Belbuca, such that we believe investors will be rewarded by the company's solid execution across both products in 2020 and beyond, which should drive upward earnings revisions and further upside in BDSI stock.”
To this end, Folkes kept his Overweight call and $8 price target as is. Should this target be met, a twelve-month gain of 62% could be in store. (To watch Folkes’ track record, click here)
Turning now to the rest of the Street, other analysts are on the same page. Only Buy ratings have been received in the last three months, 6 to be exact, so the consensus rating is a Strong Buy. In addition, the $8.04 average price target puts the upside potential at 63%. (See BioDelivery stock analysis on TipRanks)
IVERIC Bio Inc. (ISEE)
Focused on the discovery and development of treatment options for retinal diseases, IVERIC hopes to address significant unmet medical needs. While some investors have expressed concern regarding a recent trial delay, ISEE is still attracting significant support.
Back in March, management announced that it was going to push back the initiation of the pivotal Phase 3 program for Zimura, its lead asset, in Geographic Atrophy (GA) based on the potential patient safety issues stemming from COVID-19. The trial will feature elderly participants who are required to visit physician offices for treatment, which is problematic as they might be more susceptible to the virus, lending itself to potential trial disruptions.
Baker Bros. Advisors has been sitting in the bull camp. Even more positive on the stock now, the fund boosted its ISEE holding by a whopping 1,054%, or 1.5 million shares. At over 1.6 million shares, the total position is valued at $6.93 million.
Meanwhile, Cowen analyst Ken Cacciatore remains unphased by the trial delay. “By pausing now for the pandemic to normalize, this should ensure a more seamless, continuous study. Encouragingly, in the interim, the company continues to bring clinical sites on board, which should allow for accelerated enrollment once the study is initiated. And, we believe, the recently received Fast Track designation from the FDA and the potential eligibility for expedited priority review could offset these delays,” he commented.
Digging a bit deeper into Zimura, it is an anti-C5 aptamer that inhibits terminal complement activation implicated in the pathogenesis of GA, an advanced form of dry AMD. In the original Phase 2/3 study in GA, the therapy was able to meet its primary endpoint, and was generally safe without causing any cases of inflammation and endophthalmitis.
Additionally, Cacciatore noted, “Based on these results – and given the size and design – the FDA indicated that the Phase II/III trial could serve as one of two pivotal studies, which we believe substantially de-risks the Zimura development. If the next Phase III results are positive, we believe IVERIC should be able to file the Zimura NDA in H2:2022 with a potential U.S. launch in H2:2023.”
As 1.5 million people in the U.S. suffer from GA, with it causing 20% of blindness, the fact that there aren’t any therapies available creates a significant market opportunity. This prompted Cacciatore to state, “The unmet need in GA is clear, given the lack of FDA-approved therapies for dry AMD, and we believe Zimura represents a potential $750 million opportunity.”
With respect to its gene therapy programs, ISEE plans to submit the IND for IC-100 in Q4:2020 and initiate the Phase 1/2 study in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosain H1:2021.
Based on all of the above, Cacciatore stayed with the bulls. Along with an Outperform rating, the $15 price target remains unchanged. This target conveys his confidence in ISEE’s ability to skyrocket 254% in the next twelve months. (To watch Cacciatore’s track record, click here)
Looking at the consensus breakdown, it has been relatively quiet when it comes to other analyst activity. Only one other review has been published recently, but it was also bullish, so the consensus rating is a Moderate Buy. Given the $14 average price target, the upside potential lands at 230%. (See IVERIC stock analysis on TipRanks)
Minerva Neurosciences (NERV)
With its innovative therapies, Minerva Neurosciences’ goal is to dramatically improve the lives of patients battling central nervous system (CNS) diseases. Ahead of an upcoming unblinding of data for its lead candidate, roluperidone (MIN-101), the biotech has found itself on the Street’s radar.
The Baker Brothers recently decided to pull the trigger on this stock. Adding a NERV position to the fund, Baker Bros. Advisors bought up 688,531 shares. The hedge fund’s new stake boasts a value of $4,145,000.
Writing for BTIG, five-star analyst Thomas Shrader has high hopes for NERV as it gears up for the release of Phase 3 data evaluating roluperidone as a treatment of the negative symptoms (the loss of functions) associated with schizophrenia. He points out that the trial is modeled closely after the successful Phase 2b study, which was well-sized and held up to significant data reanalysis, with it also being deemed acceptable as an initial trial for approval if confirmed in the Phase 3 trial.
“Overall, we see roluperidone as having a unique place in the schizophrenia treatment landscape less prone to generics that overwhelmingly treat positive symptoms of the disease (things like hallucinations). A second drug in Phase 2 studies for insomnia provides modest downside protection in case of Phase 3 disappointment, but the roluperidone unblinding is the reason to own the stock,” Shrader commented.
Speaking to the available market, Shrader believes the commercial opportunity is “huge” as schizophrenia affects almost 1% of Americans and drugs in the space are both widely used as well as expensive, with the cost coming in at around $50 per day if patent protected. He added, “The lead drug roluperidone has shown the ability to treat the negative symptoms of schizophrenia making it essentially unique in the treatment landscape.”
While some have interpreted the Phase 2b results to mean that the placebo adjusted treatment effect was modest, Shrader calls this conclusion “premature”. To support this claim, he argues efficacy in the Phase 2a trial was close to producing results that make independent living more likely, efficacy was still building at nine months and the Phase 3 trial extension runs a full year and the treatment effect from baseline, not placebo, might be a more accurate metric.
If that wasn’t enough, its MIN-202 (seltorexant) therapy is another strong component of the development pipeline, with it achieving a rapid onset of action and a short half-life designed to work quickly but reduce the “hangover effects” that come with sleep aids. In addition, Phase 2b data showed superior and more sustained efficacy compared to Ambien. Going forward, Shrader sees the candidate as having the potential to be a major upside driver.
All of this prompted Shrader to join the bulls. To kick off his coverage, he published a Buy rating and set a $23 price target. A 61% twelve-month gain could materialize if the target is reached. (To watch Shrader’s track record, click here)
What does the rest of the Street think about NERV? It turns out that other analysts have also been impressed. 4 Buy ratings and no Holds or Sells have been assigned in the last three months, making the consensus rating a Strong Buy. The $20.33 average price target indicates 42% upside potential.
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Re: GMDA
GMDA yesterday at $4.57
re: GMDA
(I started buying GMDA yesterday at $4.57. It has jumped to over $5 now.)
https://finance.yahoo.com/news/3-biotech-stocks-under-5-132240271.html
Bladerunner
ALPS Medical Breakthroughs ETF (SBIO)
Performance over 1-Year: 20.9%
Expense Ratio: 0.50%
Annual Dividend Yield: 0.04%
3-Month Average Daily Volume: 66,070
Assets Under Management: $188.3 million
Inception Date: December 30, 2014
Issuing Company: ALPS
SBIO is a fund focused on the U.S. health and biotechnology sector, targeting companies with drugs in Phase II or Phase III clinical trials. This fund invests in firms with market caps of $200 million to $5 billion, so it contains micro-cap and mid-cap companies, as well as small-cap, in its portfolio.6? The fund's top three holdings include Immunomedics Inc. (IMMU), a pharmaceuticals company focused on cancer treatments; United Therapeutics Corp. (UTHR), a biotechnology company; and Acceleron Pharma Inc. (XLRN), a biopharmaceutical company
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https://www.investopedia.com/investing/small-cap-etfs/?utm_campaign=quote-yahoo&utm_source=yahoo&utm_medium=referral&yptr=yahoo
>>> Vaccine experts say Moderna didn’t produce data critical to assessing Covid-19 vaccine
Stat News
By HELEN BRANSWELL
MAY 19, 2020
https://www.statnews.com/2020/05/19/vaccine-experts-say-moderna-didnt-produce-data-critical-to-assessing-covid-19-vaccine/
Heavy hearts soared Monday with news that Moderna’s Covid-19 vaccine candidate — the frontrunner in the American market — seemed to be generating an immune response in Phase 1 trial subjects. The company’s stock valuation also surged, hitting $29 billion, an astonishing feat for a company that currently sells zero products.
But was there good reason for so much enthusiasm? Several vaccine experts asked by STAT concluded that, based on the information made available by the Cambridge, Mass.-based company, there’s really no way to know how impressive — or not — the vaccine may be.
While Moderna blitzed the media, it revealed very little information — and most of what it did disclose were words, not data. That’s important: If you ask scientists to read a journal article, they will scour data tables, not corporate statements. With science, numbers speak much louder than words.
Even the figures the company did release don’t mean much on their own, because critical information — effectively the key to interpreting them — was withheld.
Experts suggest we ought to take the early readout with a big grain of salt. Here are a few reasons why.
The silence of the NIAID
The National Institute for Allergy and Infectious Diseases has partnered with Moderna on this vaccine. Scientists at NIAID made the vaccine’s construct, or prototype, and the agency is running the Phase 1 trial. This week’s Moderna readout came from the earliest of data from the NIAID-led Phase 1.
NIAID doesn’t hide its light under a bushel. The institute generally trumpets its findings, often offering director Anthony Fauci — who, fair enough, is pretty busy these days — or other senior personnel for interviews.
But NIAID did not put out a press release Monday and declined to provide comment on Moderna’s announcement.
The n = 8 thing
The company’s statement led with the fact that all 45 subjects (in this analysis) who received doses of 25 micrograms (two doses each), 100 micrograms (two doses each), or a 250 micrograms (one dose) developed binding antibodies.
Later, the statement indicated that eight volunteers — four each from the 25-microgram and 100-microgram arms — developed neutralizing antibodies. Of the two types, these are the ones you’d really want to see.
We don’t know results from the other 37 trial participants. This doesn’t mean that they didn’t develop neutralizing antibodies. Testing for neutralizing antibodies is more time-consuming than other antibody tests and must be done in a biosecurity level 3 laboratory. Moderna disclosed the findings from eight subjects because that’s all it had at that point. Still, it’s a reason for caution.
Separately, while the Phase 1 trial included healthy volunteers ages 18 to 55 years, the exact ages of these eight people are unknown. If, by chance, they mostly clustered around the younger end of the age spectrum, you might expect a better response to the vaccine than if they were mostly from the senior end of it. And given who is at highest risk from the SARS-CoV-2 coronavirus, protecting older adults is what Covid-19 vaccines need to do.
There’s no way to know how durable the response will be
The report of neutralizing antibodies in subjects who were vaccinated comes from blood drawn two weeks after they received their second dose of vaccine.
Two weeks.
“That’s very early. We don’t know if those antibodies are durable,” said Anna Durbin, a vaccine researcher at Johns Hopkins University.
There’s no real way to contextualize the findings
Moderna stated that the antibody levels seen were on a par with — or greater than, in the case of the 100-microgram dose — those seen in people who have recovered from Covid-19 infection.
But studies have shown antibody levels among people who have recovered from the illness vary enormously; the range that may be influenced by the severity of a person’s disease. John “Jack” Rose, a vaccine researcher from Yale University, pointed STAT to a study from China that showed that, among 175 recovered Covid-19 patients studied, 10 had no detectable neutralizing antibodies. Recovered patients at the other end of the spectrum had really high antibody levels.
So though the company said the antibody levels induced by vaccine were as good as those generated by infection, there’s no real way to know what that comparison means.
STAT asked Moderna for information on the antibody levels it used as a comparator. The response: That will be disclosed in an eventual journal article from NIAID, which is part of the National Institutes of Health.
“The convalescent sera levels are not being detailed in our data readout, but would be expected in a downstream full data exposition with NIH and its academic collaborators,” Colleen Hussey, the company’s senior manager for corporate communications, said in an email.
Durbin was struck by the wording of the company’s statement, pointing to this sentence: “The levels of neutralizing antibodies at day 43 were at or above levels generally seen in convalescent sera.”
“I thought: Generally? What does that mean?” Durbin said. Her question, for the time being, can’t be answered.
Rose said the company should disclose the information. “When a company like Moderna with such incredibly vast resources says they have generated SARS-2 neutralizing antibodies in a human trial, I would really like to see numbers from whatever assay they are using,” he said.
Moderna’s approach to disclosure
The company has not yet brought a vaccine to market, but it has a variety of vaccines for infectious diseases in its pipeline. It doesn’t publish on its work in scientific journals. What is known has been disclosed through press releases. That’s not enough to generate confidence within the scientific community.
“My guess is that their numbers are marginal or they would say more,” Rose said about the company’s SARS-2 vaccine, echoing a suspicion that others have about some of the company’s other work.
“I do think it’s a bit of a concern that they haven’t published the results of any of their ongoing trials that they mention in their press release. They have not published any of that,” Durbin noted.
Still, she characterized herself as “cautiously optimistic” based on what the company has said so far.
“I would like to see the data to make my own interpretation of the data. But I think it is at least encouraging that we’ve seen immune responses with this RNA vaccine that we haven’t seen with previous RNA vaccines for other pathogens. Whether it’s going to be enough, we don’t know,” Durbin said.
Moderna has been more forthcoming with data on at least one of its other vaccine candidates. In a statement issued in January about a Phase 1 trial for its cytomegalovirus (CMV) vaccine, it quantified how far over baseline measures antibody levels rose in vaccines.
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>>> CytoDyn and the Mexican National Institutes of Health Participate in a Collaborative Study of Leronlimab for the Treatment of Severe/Critical COVID-19 Population
May 19, 2020
https://www.cytodyn.com/newsroom/press-releases/detail/433/cytodyn-and-the-mexican-national-institutes-of-health
The study is anticipated to consist of approximately thirty patients with potential involvement of the NIH of Mexico in other CytoDyn trials
VANCOUVER, Washington, May 19, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (“CytoDyn” or the “Company”), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, today announced it will be coordinating with the NIH of Mexico and providing leronlimab for a trial for the severe/critical COVID-19 population in Mexico with the potential to collaborate on further CytoDyn COVID-19 trials.
CytoDyn is currently enrolling a Phase 2b/3 clinical trial for 390 patients, which is a randomized, placebo-controlled with 2:1 ratio (active drug to placebo ratio). CytoDyn is also enrolling a Phase 2 randomized clinical trial with 75 patients in the mild-to-moderate COVID-19 population. CytoDyn has been granted more than sixty emergency Investigational New Drug (eIND) authorizations by the U.S. Food and Drug Administration (FDA) and plans to provide clinical updates for this patient population later in the week.
“We look forward to evaluating leronlimab as a treatment option for patients of COVID-19. We have seen the devastation of this disease on the citizens of Mexico and are looking forward to providing effective treatment options to mitigate the devastation of COVID-19,” said Dr. Gustavo Reyes Terán, head of the Coordinating Commission of National Institutes of Health and High Specialty Hospitals of Mexico, an organization that coordinates the main institutions of medical care and public research in the country.
“The NIH of Mexico is committed to help alleviate human suffering and mortality of Mexican citizens. The Metropolitan Area of the Valley of Mexico has a population of approximately 21.5 million people and the contagious nature of COVID-19 is relentless. We look forward to working with the NIH of Mexico to rapidly commence with the proposed study. We also believe that this study results, along with the ongoing Phase 2 study, could establish a path for quick approval in Mexico for use of leronlimab in COVID-19 patients,” said Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn.
About Coronavirus Disease 2019
CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.
SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.
About Leronlimab (PRO 140) and BLA Submission for the HIV Combination Therapy
The FDA has granted a “Fast Track” designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).
In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.
The Company filed its BLA for Leronlimab as a Combination Therapy for Highly Treatment Experienced HIV Patients with the FDA on April 27, 2020, and submitted additional FDA requested clinical datasets on May 11, 2020. After the BLA submission is deemed completed, the FDA sets a PDUFA goal date. CytoDyn has Fast Track designation for leronlimab and a rolling review for its BLA, as previously assigned by the FDA, and the Company plans to request a priority review for the BLA. A priority review designation means the FDA’s goal is to take action on the marketing application within six months of receipt (compared with 10 months under standard review).
In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.
The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted “orphan drug” designation to leronlimab for the prevention of GvHD.
About CytoDyn
CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn filed its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients, and submitted additional FDA requested clinical datasets on May 11, 2020. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at www.cytodyn.com.
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>>> Innovation Pharmaceuticals’ Brilacidin Reduces Viral Titer of SARS-CoV-2 (COVID-19) by 75 percent After Only 1 Hour of Preincubation in In Vitro Study at BSL-3 Facility; Demonstrates Potent and Rapid Virucidal Activity
GlobeNewswire
May 19, 2020
https://finance.yahoo.com/news/innovation-pharmaceuticals-brilacidin-reduces-viral-113010146.html
Experiment supports Brilacidin’s therapeutic potential as a COVID-19 treatment and as a prophylactic agent against COVID-19
WAKEFIELD, Mass., May 19, 2020 (GLOBE NEWSWIRE) -- Innovation Pharmaceuticals (IPIX) (“the Company”), a clinical stage biopharmaceutical company, announced today that its anti-SARS-CoV-2 (COVID-19) drug candidate, Brilacidin, in an in vitro experiment using VERO cells, reduced the viral titer (load) of SARS-CoV-2 by 75 percent after only 1 hour of preincubation prior to infection at a concentration of 10µM as compared to vehicle control.
These statistically significant lab results strongly support Brilacidin’s prophylactic treatment potential given the drug’s potent and rapid virucidal activity—a unique ability, different from any other known drug currently in development to treat COVID-19, to inactivate the novel coronavirus prior to host cell entry and subsequent viral replication. A majority of antiviral agents targeting SARS-CoV-2 attempt to inhibit viral replication rather than completely eliminating the virus (virustatic versus virucidal).
Additional time-dependent and concentration-dependent experiments are underway to further delineate Brilacidin’s virucidal properties against SARS-CoV-2.
The review article linked below more broadly supports the therapeutic and prophylactic potential of Host Defense Proteins/Antimicrobial Peptides and their mimetics, like Brilacidin, against coronaviruses.
Memariani H, Memariani M. “Therapeutic and Prophylactic Potential of Anti-Microbial Peptides Against Coronaviruses.” Ir J Med Sci. 2020 Apr 18: 1–2.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7165072/
Brilacidin anti-SARS-CoV-2 research is part of ongoing antiviral studies that are being conducted at a U.S. Regional Biocontainment Laboratory (RBL). An earlier 16-hour post-infection experiment, in VERO cells, showed Brilacidin exhibited a dose-dependent reduction in SARS-CoV-2 infectious viral titers.
The Company is encouraged by Brilacidin’s considerable antiviral potential and believes that its use as a possible potent prophylactic and therapeutic antiviral agent for SARS-CoV-2, and other viruses, is warranted.
Separately, the Company and laboratory researchers (from a different testing facility) agreed that Brilacidin as a vaccine would not be studied further at this time. While the vaccine pathway remains of interest, the Company is prioritizing Brilacidin’s clinical development as a potential novel COVID-19 treatment, and thus is taking steps to rapidly advance Brilacidin into human trials.
Brilacidin and COVID-19
Brilacidin is one of the few drugs targeting COVID-19 that has been tested in Phase 2 human trials for other clinical indications, providing an established safety and efficacy profile, thereby potentially enabling it to rapidly help address the emerging worldwide coronavirus crisis. Lab testing conducted at a U.S.-based Regional Biocontainment Laboratory (RBL) supports Brilacidin’s antiviral activity in directly inhibiting SARS-CoV-2 in cellular assays, with a molecular screening study of 11,552 compounds also supporting it as a promising novel coronavirus treatment. Additional pre-clinical and clinical data support Brilacidin’s potential to inhibit the production of IL-6, IL-1b, TNF-a and other pro-inflammatory cytokines and chemokines (e.g., MCP-1), which have been identified as central drivers in the worsening prognoses of COVID-19 patients. Brilacidin’s antimicrobial properties might also help to fight secondary bacterial infections, which can co-present in up to 20 percent of COVID-19 patients. These data collectively support Brilacidin as a particularly promising and unique (3 in 1 combination: antiviral, immune/anti-inflammatory, and antimicrobial) anti-COVID-19 therapeutic candidate.
For researchers and institutions interested in collaborating on Brilacidin for COVID-19, please send inquiries to: covid19@ipharminc.com
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About Innovation Pharmaceuticals
Innovation Pharmaceuticals Inc. (IPIX) is a clinical stage biopharmaceutical company developing a world-class portfolio of innovative therapies addressing multiple areas of unmet medical need, including inflammatory diseases, cancer, infectious disease, and dermatologic diseases. Brilacidin, a versatile compound with broad therapeutic potential, is in a new chemical class called defensin-mimetics. A Phase 2 trial of Brilacidin as an oral rinse for the prevention of Severe Oral Mucositis (SOM) in patients with Head and Neck Cancer, met its primary and secondary endpoints, including reducing the incidence of SOM. The Company plans to advance Brilacidin oral rinse into Phase 3 development, subject to available financial resources. Positive results were also observed in a Phase 2 Proof-of-Concept trial treating patients locally with Brilacidin for Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS). Brilacidin for UP/UPS was licensed to Alfasigma S.p.A. in July 2019. A Phase 2b trial of Brilacidin showed a single intravenous dose of the drug delivered comparable outcomes to a seven-day dosing regimen of the FDA-approved blockbuster daptomycin in treating Acute Bacterial Skin and Skin Structure Infection. Kevetrin is a novel anti-cancer drug shown to modulate p53, often referred to as the “Guardian Angel Gene” due to its crucial role in controlling cell mutations and has successfully completed a Phase 2 trial in Ovarian Cancer. More information is available on the Company website at www.IPharmInc.com.
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>>> MIT Professor’s Moderna Stake on Brink of Topping $1 Billion
Bloomberg
Devon Pendleton and Tom Maloney
May 18, 2020
https://finance.yahoo.com/news/mit-professor-moderna-stake-brink-205502161.html
(Bloomberg) -- Another early investor of Moderna Inc. is on the cusp of owning a stake worth at least $1 billion after the biotech firm reported encouraging early trial results for an experimental Covid-19 vaccine.
The value of board member Bob Langer’s 3.2% holding, including stock options, rose to $934.3 million Monday, as the shares surged 20% to a record $80 each. Langer, a professor at the Massachusetts Institute of Technology, would be at least the third individual with Moderna holdings topping $1 billion, joining Chief Executive Officer Stephane Bancel and Harvard University professor Timothy Springer.
Moderna, whose shares have more than quadrupled this year, released interim data from a small phase 1 trial showing positive early signs that the vaccine can create an immune system response to the virus in humans. The news helped fuel a broader surge in stocks, with the S&P 500 Index advancing 3.2%, the most since April 8.
The company priced a stock offering to fund manufacturing of its coronavirus vaccine at $76 a share, 5% below the last close, people familiar with the deal said after Monday’s market trading.
Langer, 71, has licensed or sub-licensed patents to more than 400 biotech, pharmaceutical, chemical and medical companies, according to his biography at MIT’s Langer Lab.
Bancel and Springer own stakes worth $2.45 billion and $1.38 billion, respectively, according to the Bloomberg Billionaires Index. The biggest beneficiary of the stock surge is top shareholder Flagship Pioneering Inc., a firm started by Moderna co-founder Noubar Afeyan. Flagship distributed 10 million shares to investors last week, leaving it with an 11% stake worth $3.27 billion. Afeyan declined to elaborate when asked about how much of the stock he owns individually.
“The broader market reaction is a measure of the need people have to perceive that there’s a scientific, technological solution to this kind of battle,” Afeyan said in a phone interview. “We’ve increased expectations, but that hasn’t changed what we do.”
‘Warp Speed’
Bancel and other Moderna executives, including outgoing Chief Financial Officer Lorence Kim and President Stephen Hoge, have been selling shares, some through prearranged trading plans. Bancel has sold about 200,000 shares since Feb. 21, data compiled by Bloomberg show.
Moncef Slaoui, who owned 82,508 shares as of Feb. 21, stepped down from Moderna’s board last week and plans to divest his stake to help lead “Operation Warp Speed,” a Trump administration effort involving the private and public sectors to accelerate the development of a vaccine, the company said in an emailed statement.
Slaoui intends to donate the incremental value accrued from his Moderna stake since the May 14 close to cancer research, Caitlin Oakley, a spokeswoman for the U.S. Department of Health & Human Services, wrote in an email.
Moderna Insiders Get Fresh Chance to Cash Out After 200% Surge
Other major shareholders include AstraZeneca Plc and Theleme Partners, the hedge fund of Patrick Degorce, a former partner of the Children’s Investment Fund.
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>>> 'Something we've never seen before': Scientists still trying to understand baffling, unpredictable coronavirus
by Mark Johnson
Milwaukee Journal Sentinel
5-18-20
https://www.msn.com/en-us/health/medical/something-weve-never-seen-before-scientists-still-trying-to-understand-baffling-unpredictable-coronavirus/ar-BB14eZ3B?li=BBnb7Kz&ocid=mailsignout
MILWAUKEE – The new coronavirus has spread like wildfire, killed — and spared — people of all ages and all health conditions, baffled doctors, defied guidance and conventional wisdom, and produced an unprecedented array of symptoms.
There's never been a virus like it.
"This gets into every major biological process in our cells," said Nevan J. Krogan, a molecular biologist at the University of California, San Francisco, who has studied HIV, Ebola, Zika, dengue and other viruses over the last 13 years.
"At the molecular level, it's something we've never seen before, and then look at what it does to the body — the long list of symptoms — we've never seen that before."
As Americans debate the reopening of businesses, bars, schools and other aspects of everyday life, it's important to understand the virus we are up against and why it has sown so much suffering and confusion.
At first, the virus was thought to be mostly a risk to older adults and people with chronic illnesses; its primary point of attack, the lungs. Then 30- and 40-years-olds with the virus began dying of strokes. Recently, a small number of infected children have died of a mysterious illness resembling Kawasaki disease.
Symptoms of COVID-19 range from fever, coughing and shortness of breath to the loss of smell and taste and the angry red swelling that has come to be known as "COVID toes." Studies have found that damage from SARS-CoV-2, the virus that causes the disease COVID-19, isn't limited to the lungs; it can include the heart, liver, kidneys, gastrointestinal system and bowels.
To understand a virus' "motivation" — why it does what it does — keep in mind that it is a parasite. It lives inside its human or animal host taking what it needs at the expense of the host.
As long as it finds hosts without immunity, and as long as its own mutations do not weaken its ability to spread and multiply, the virus thrives.
Key benchmarks of a virus are how widely it spreads and how deadly it is to those it infects.
In the five months since it was first identified in Wuhan, China, SARS-CoV-2 has infected more than 4.5 million people across the globe, killing more than 300,000.
"The thing that strikes me about the clinical aspect is the shear amount of transmissibility," said Megan Freeman, a virologist and specialist in pediatric infectious diseases at UPMC Children's Hospital of Pittsburgh.
"There are very few viruses that are more contagious than this one," agreed Mark Schleiss, an investigator for the Institute of Molecular Virology at the University of Minnesota.
A single COVID-19 patient spreads the disease to a median of 5.7 people, making it twice as contagious as the 1918 Spanish flu, according to a report in the journal Emerging Infectious Diseases.
The rate of infection in the U.S. — what experts are referring to when they talk about "flattening the curve" — has slowed. Even so, the nation is still averaging about 1,000 COVID-19 deaths a day, roughly two 9/11 attacks every week.
Thousands of the world's best scientists have worked with remarkable speed seeking to understand the new coronavirus. They deciphered its genetic code in barely a week and have produced scores of papers suggesting possible treatments and vaccines.
"The canvas we call COVID-19 was blank 16 weeks ago," said Gregory Poland, director of Mayo Clinic's Vaccine Research Group. "We've filled in a lot of dots, but we have so much more to go."
"We don't understand HIV that well in my opinion," Krogan said, "and we've been studying that for decades."
Seeking the Achilles' heel of the virus
Scientists know some but not all of the reasons the new coronavirus spreads so easily. Freeman points to several factors, including one that distinguishes SARS-CoV-2 from the virus it closely resembles, Severe Acute Respiratory Syndrome (SARS).
SARS attacks the lower respiratory system, whose job it is to pull in air from the upper respiratory system.
SARS-CoV-2, however, attacks the upper respiratory system, the pathway that allows air to travel in and out of the lungs as we breathe. The upper airway is also the system involved when we cough.
The upper respiratory system offers a more efficient means of spreading, Freeman said.
"That's why it is more transmissible. ... When this emerged probably there was some mutation that adapted it to use the upper airway, and the upper airway has made it very successful."
A second difference between SARS and SARS-CoV-2 involves the bond that allows viral cells to attach to human cells and infect them. With both viruses, this bond forms between the Spike Protein on the virus and a region on the outside of the human cell called the ACE-2 receptor.
Both viruses use this bond to enter cells, but the bond is much stronger with the new coronavirus than it was with SARS. That is why many of the potential treatments so far are designed to undermine that bond.
"You always want to target the Achilles' heel of the virus, something the virus does not have the luxury of changing too much" explained Maria Elena Bottazzi, a professor of pediatrics at Baylor College of Medicine and co-developer of a potential SARS vaccine that is going to be tested on SARS-CoV-2.
Although public health leaders compared the new coronavirus to seasonal flu early on, coronaviruses have a special feature that separates them from other viruses like flu. They have their own proofreading system that helps limit mistakes in their genetic code known as mutations.
"Flu viruses make six and a half times more mistakes than coronaviruses do," Freeman said.
Even as the spread of the virus slows in the United States, some researchers fear what will happen if major outbreaks hit poorer, densely populated cities in Africa.
"Look at the problems we've had," Poland warned. "Now amplify that through areas that have civil wars, that have corruption, that have scarce resources. That's a humanitarian disaster in the making."
A remarkable destroyer
The virus' skill at spreading poses one challenge; what it does inside the human body poses another.
Much of what scientists have learned so far about the new coronavirus suggests that it is a remarkable destroyer at both the micro and macro levels, decimating individual cells and entire organs.
At the molecular level, the virus disrupts some of the most fundamental functions of life: cell division; the system cells use to talk with one another; and their ability to make proteins.
The proteins our bodies make help us carry out almost every human action from eating and walking to breathing and thinking.
Once a person ingests the new coronavirus, it enters the lungs and directly infects the air sacs, the microscopic workhorses that take in the air we breathe. In serious cases, the air sacs fill with fluid, leaving less and less room for oxygen. This is a feature of what is known as COVID-19 pneumonia.
The pneumonia can lead to Acute Respiratory Distress Syndrome, a severe breathing condition that deprives the vital organs of oxygen.
A healthy blood oxygen level is usually between 95% and 100%. Below 90% is low. Some COVID-19 patients have been found to have blood oxygen levels below 65%.
As the air sacs are infected and damaged, this triggers the immune system, which can lead to a dangerous condition called a cytokine storm. In a cytokine storm, the immune system goes into overdrive and winds up killing both healthy and diseased cells.
One of the most subtle and deadly offshoots of low blood oxygen is a condition called silent hypoxia.
As described by emergency room doctor Richard Levitan in a commentary in the New York Times, silent hypoxia allows patients to develop low blood oxygen levels without realizing the problem until it dips into dangerous territory. They breathe faster to compensate for the lack of oxygen but are unaware they are breathing faster.
Levitan suggested that people can bypass long waits for coronavirus tests, using a simple device called a pulse oximeter as an early warning system for detecting COVID-19 pneumonia. The device, which fits over your finger, measures blood oxygen levels, and can be bought at pharmacies without prescription for about $30.
Unfortunately, SARS-CoV-2 does not restrict its damage to the lungs.
The virus also causes blood clots, which have led to people in their 30s and 40s dying from strokes.
And then there are the recent cases in New York and Paris of children with COVID-19 who develop symptoms closely resembling Kawasaki disease. The disease begins with a rash and fever, inflames blood vessels, and eventually can damage the coronary arteries that deliver blood to the heart.
"What is it about COVID-19 that it produces Kawasaki disease?" said Schleiss, at the University of Minnesota, who estimates he has seen more than 1,000 Kawasaki cases in his medical career.
"I don't think SARS-CoV-2 is a cause of Kawasaki disease, but it is the cause of something very similar."
Every few weeks, SARS-CoV-2 seems to reveal new and disturbing oddities.
A recent study from China published in the journal JAMA Network Open, reported finding the new coronavirus in the semen of six of 38 infected men, raising concern that it may be possible to transmit the virus through sexual contact.
The virus also has been found in patient stool samples and untreated wastewater. The Centers for Disease Control and Prevention has reported that standard methods at wastewater treatment plants should be effective enough to protect workers.
Such discoveries, however, raise fundamental questions, as we try to return to our old lives. Have we yet determined all of the ways the virus can spread from one person to another? Are there activities that may pose risks we have not anticipated?
At the University of California, San Francisco, Krogan said it will be important to examine the genetic scripts of both those who suffer severe cases of COVID-19 and those who get mild or no disease at all.
"There are 30-year-olds and some of them are asymptomatic and others are on respirators. What the hell is that all about?"
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>>> Moderna's stock soars on positive early-stage data for its coronavirus vaccine candidate
MarketWatch
May 18, 2020
By Jaimy Lee
https://www.marketwatch.com/story/modernas-stock-soars-on-positive-early-stage-data-for-its-coronavirus-vaccine-candidate-2020-05-18?siteid=bigcharts&dist=bigcharts
Shares of Moderna Inc. MRNA, +3.29% jumped 25.1% in premarket trading on Monday after the biotechnology company announced positive results from a Phase 1 clinical trial for its experimental mRNA coronavirus vaccine. The trial was done in partnership with the National Institute of Allergy and Infectious Diseases.
Within 43 days and after two doses, the participants taking one of two dosing levels of the vaccine candidate reported the same or higher level of antibodies as in blood samples gathered by patients who have recovered from COVID-19, the company said. There were four adverse events during the trial, including one participant who reported a severe skin reaction where the investigational vaccine was administered.
"These interim Phase 1 data, while early, demonstrate that vaccination with mRNA-1273 elicits an immune response of the magnitude caused by natural infection starting with a dose as low as 25" micrograms, Moderna chief medical officer Dr. Tal Zaks said in a news release.
The next step is a Phase 2 trial, which has been approved to move forward by the Food and Drug Administration. It will focus on two dosing levels (50 microgram and 100 microgram). The early-stage trial had a third dosing level, 250 micrograms.
Moderna's vaccine candidate is widely viewed as a frontrunner in the effort to develop the first vaccine for the virus. The preclinical company said earlier this month that the Phase 2 trial will begin "shortly," and on Monday it said it expects a Phase 3 trial to begin in July, if the vaccine is successful in the mid-stage trial. Moderna's stock has soared 240.9% year-to-date. The S&P 500 SPX, +0.39%, in comparison, is down 11.3%.
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