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Is it possible that the EMA would expedite their approval process for Anavex since the United States has approved Leqembi?
Seems to me that the EMA has already demonstrated their willingness to find a safe AD drug by accepting our small P2b/3 trial (n= 509) as opposed to the typical double P3 trial.
Wow...that's a powerful video. Thanks.
Excellent post, sokol. Thanks for sharing all of this DD!
Good for you, sab. Having grandchildren sure makes life more enjoyable. Always puts a smile on my face when I talk with them. (I have 3 grandkids too)
Hey Doc.
I was hoping to get your opinion on the recent PR. Pretty impressive, aye?
Anavex Initiates Regulatory Submission of Oral Blarcamesine for Alzheimer's Disease to European Medicines Agency (EMA)
NEW YORK, Nov. 20, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders announced today that representatives of Anavex met with team members of the European Medicines Agency (EMA).
These meetings discussed the debilitating pathology of Alzheimer's disease and Anavex's blarcamesine (ANAVEX(R)2-73) Alzheimer's disease clinical program results, including data obtained in the ANAVEX(R)2-73-AD-004 study.
Pursuant to discussion at the meetings, Anavex initiated the process for submitting a Marketing Authorisation application to the EMA with the submission of the Centralised Procedure request with the goal of the Authorisation allowing direct access to the market of the European Union for oral blarcamesine for the treatment of Alzheimer's disease.
Anavex's goal is to take care of patients in a patient-centric way with the preference for convenient oral treatment options for Alzheimer's disease not requiring complex logistics resources and added personnel for drug administration and monitoring for brain edema and brain bleeds.
Severe symptoms in relation to Amyloid-Related Imaging Abnormalities (ARIA) is a known risk factor for Alzheimer's patients taking the class of drugs called monoclonal antibodies, and requires constant and repeated MRI examination, for which not all regions in Europe are currently sufficiently prepared and equipped for in addition to the requirement to address affordability and inequalities in patient access within European Union countries.1,2
"We look forward to working together with the team from EMA," said David Goldberger, RPh, MLS, Senior Vice President Regulatory Affairs at Anavex. "We continue to work towards fulfilling our purpose of improving patients' lives with oral blarcamesine not requiring any complex additional procedures for the treatment of people with Alzheimer's disease."
There are an estimated 7 million people in Europe with Alzheimer's disease, a number expected to double by 2030, according to the European Brain Council.3
The World Health Organization (WHO) estimated the cost in Europe of caring for people with dementia, including Alzheimer's disease, at $439 billion, or $31,144 per person in 2019. That includes hospital care, medicines, diagnostics, informal caregiver time, community services and long-term care facility costs.4,5
"There remains an urgent need for convenient once-daily oral treatment options for Alzheimer's disease, and Anavex is moving forward to potentially addressing the preference for simple patient-centered administrations and shared decision-making," said Christopher U Missling, PhD, President and Chief Executive Officer at Anavex.
In addition to significant improvement in dementia symptoms, blarcamesine demonstrated reduction of pathological aggregation of amyloid in early Alzheimer's disease as well reduction of brain volume loss, a well-known marker of neurodegeneration.
Data from the blarcamesine in Alzheimer's disease Phase 2b/3 randomized clinical trial will be published in an upcoming peer-reviewed journal.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX(R)2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX(R)2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX(R)2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX(R)2-73 for the treatment of Parkinson's disease. ANAVEX(R)3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX(R)3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter, Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company's most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
It's my understanding that once you submit your New Drug Application (NDA) to the FDA for approval, they will respond within 30 - 60 days as to whether or not they will accept the application.
If they find something wrong, they will send you a rejection letter and explain why they rejected your NDA...which could vary from a minor correction to a complete failure.
On the other hand, if everything is in order they will acknowledge your NDA within the 30 - 60 day period and the procedural evaluation and approval process begins.
So, I'm wondering if the TGD has already submitted the NDA for Rett syndrome; and is now waiting the 30 - 60 days to confirm that the FDA has accepted our New Drug Application?
Now that would be a great surprise to everyone, no?
SAB...correct me if I'm wrong, but the 6-month chart looks like an inverse Head & Shoulders...which would be very bullish, no?
I did the same thing...so you're in good company. Although I added shares, not options.
Remember, the EMA was given all of the data from the P2b/3 study. That's why they accepted the application from Anavex for Alzheimer's Disease. Your opinion is based on the PRs...which only highlights certain parts of the trial. Do you really think the PR below is supposed to include all of the data from the P2b/3 trial?
Anavex's Phase 2b/3 Trial of Blarcamesine (ANAVEX(R)2-73) in Patients with Alzheimer's Disease Shows Robust Clinical Efficacy and Slows Neurodegeneration
NEW YORK, Sept. 14, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders announced today that a follow-on analysis of the landmark Phase 2b/3 study to treat early Alzheimer's disease with the investigational drug blarcamesine (ANAVEX(R)2-73) did demonstrate a statistically significant slowing in cognitive decline associated with Alzheimer's disease.
The clinical effect was complemented by two independent biomarkers: A significant reduction in pathological amyloid beta levels in plasma1, as well as a significant slowing in the rate of pathological brain atrophy2 on MRI (Magnetic Resonance Imaging)3 scans.
The trial was a Multicenter (52 medical research centers/hospitals in 5 countries), randomized, double-blind, placebo-controlled, 48-week phase 2b/3 trial that enrolled 508 participants with early symptomatic Alzheimer disease (mild cognitive impairment/mild dementia). Participants were randomized to receive blarcamesine (n = 338) or placebo (n = 170) oral capsules once daily for 48 weeks.
All prespecified clinical endpoints were analyzed using a mixed model for repeated measures (MMRM). The MMRM analysis method is the convention used for regulatory filings and discussions with regulatory authorities are in preparation.
The trial is successful in meeting the co-primary endpoints if the significance of each endpoint is P < 0.05, or if the significance of only one co-primary endpoint is P < 0.025. If only one primary endpoint is significant at an α level of 0.025, then the secondary endpoint will be evaluated at the same level of 0.025. The trial was successful, since the differences in the least-squares mean (LSM) change from baseline to 48 weeks between the blarcamesine and placebo groups were â?'1.783 [95% CI, â?'3.314 to â?'0.251]; (P = 0.0226) for ADAS-Cog13, and â?'0.456 [95% CI, â?'0.831 to â?'0.080]; (P = 0.0175) for CDR-SB in patients with early Alzheimer's disease.
In addition, validated biomarkers of amyloid beta pathology, plasma AB42/40 ratio increased significantly (P = 0.048), demonstrating strong anti-amyloid effects of blarcamesine in Alzheimer's disease patients, while MRI revealed significant reduction in brain volume loss, including whole brain (P = 0.0005), comparing treatment to placebo. In the respective safety population, common treatment-emergent adverse events included dizziness, which was transient and mostly mild to moderate in severity, and occurred in 120 participants (35.8%) during titration and in 76 participants (25.2%) during maintenance with blarcamesine and 10 (6.0%) during titration and 9 (5.6%) during maintenance with placebo.
"There is hope that new therapies for Alzheimer's that target the disease beyond amyloid that may slow progression of the disease for many people with the earliest forms of the disease," said Marwan Noel Sabbagh, MD, Professor of Neurology and Chairman of the Scientific Advisory Board. "The advantage of blarcamesine (ANAVEX(R)2-73) is that it is a small oral molecule that exerts clinical benefits on cognition and neurodegeneration and could be appealing because of its route of administration and excellent safety profile."
This is among the first drugs to prospectively demonstrate efficacy on biomarkers of neurodegeneration.
"These data are very exciting, particularly in a study that can demonstrate objective slowing of markers of neurodegeneration," says Michael Weiner MD, Professor of Radiology and Biomedical Imaging, Medicine, Psychiatry, and Neurology at the University of California, San Francisco (UCSF) and Principal Investigator of the Alzheimer's Disease Neuroimaging Initiative (ADNI).
"Alzheimer's disease is such a devastating disease that affects tens of millions worldwide, and Anavex's clinical development is a testament to our determination to follow the science," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "We like to thank all the people involved in the study for their invaluable contributions and we look forward to advancing blarcamesine as a potential new convenient orally available treatment option for Alzheimer's disease."
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer's disease, Parkinson's disease, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex's lead drug candidate, ANAVEX(R)2-73 (blarcamesine), has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer's disease, a Phase 2 proof-of-concept study in Parkinson's disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX(R)2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX(R)2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX(R)2-73 for the treatment of Parkinson's disease. ANAVEX(R)3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX(R)3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the Company on Twitter,Facebook, Instagram, and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company's most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
Mutual agreements between the EMA and the TGA
AUSTRALIA
The European Medicines Agency and the European Commission have had confidentiality arrangements with the Therapeutic Goods Administration (TGA) of the Australian Government Department of Health and Aging since 2012, to allow the exchange of information between the parties as part of their regulatory and scientific processes. The European Union (EU) and Australia also have a mutual recognition agreement (MRA) in place on good manufacturing practice (GMP) compliance.
Confidentiality arrangement
EMA, the European Commission and TGA signed a confidentiality arrangement in 2012.
The confidential information that EMA and TGA can share includes:
guidance documents, policies, procedure and other technical documents;
post-authorisation pharmacovigilance data, particularly in relation to adverse drug reactions, as well as safety concerns arising from periodic safety update reports and post-authorisation obligations and commitments;
applications for scientific advice, orphan designation, marketing authorisation or post-authorisation activities of significant public health interest and complementary medicine listings;
cases, or possible cases, of counterfeit therapeutic goods and medicines;
risk management plans;
administrative arrangements including fees and charges;
information technology systems supporting regulatory processes.
Exchange of letters
PDF icon Confidentiality arrangement: Letter from EMA to TGA of the Australian Government Department of Health and Ageing (1/10/2012)
PDF icon Confidentiality arrangement: Letter from TGA of the Australian Government Department of Health and Ageing to EMA (1/10/2012)
MRA and collaboration on GMP compliance
The EU and Australia have signed an MRA on GMP. This allows EU authorities and their Australian counterparts to:
rely on each other's GMP inspections of manufacturers in their respective territories;
waive batch testing of products on entry into their territories;
share information on inspections and quality defects.
For more information on MRAs and the scope of the EU-Australia MRA, see Mutual recognition agreements.
EMA also participates in initiatives with partner authorities, including the TGA, on GMP inspections which aim to better distribute inspections capacity, allowing more sites to be monitored and reducing unnecessary duplication. For more information, see International collaboration on GMP inspections.
Cluster activities
The agencies, together with other non-EU regulators, hold regular meetings by phone or videoconference in so-called 'clusters'.
The clusters are areas of cooperation focusing on special topics and therapeutic areas identified as requiring an intensified exchange of information and collaboration.
https://www.ema.europa.eu/en/partners-networks/international-activities/bilateral-interactions-non-eu-regulators/australia
Thanks for your Technical Analysis...and based on today's SP it looks like it's spot on.
FYI...I posted that Barron's article to show everyone that the entire biotech sector is getting pummeled. (Most of us already know that...but some of the naysayers here still want to put all the blame on Dr. Missling)
Once we announce the results of the Excellence trial, I think that will stabilize our SP until we file the NDA. Once the NDA is filed, I think our SP will get a pretty good bump.
*****************************************************************************************************
Biotech Stocks Are Rallying, but the Outlook Isn't Good -- Barrons.com
Biotech stocks are climbing again, but it isn't because the outlook for the sector has gotten any brighter.
Share prices have been dropping consistently since early February of 2021, when the air started coming out of the pandemic-era biotech bubble. The SPDR S&P Biotech ETF (ticker: XBI), which tracks the sector, is down nearly 60% since then, and has fallen almost 15% this year. The S&P 500 is up about 17%.
Biotech Stocks Are Rallying, but the Outlook Isn't Good -- Barrons.com
Biotech stocks are climbing again, but it isn't because the outlook for the sector has gotten any brighter.
Share prices have been dropping consistently since early February of 2021, when the air started coming out of the pandemic-era biotech bubble. The SPDR S&P Biotech ETF (ticker: XBI), which tracks the sector, is down nearly 60% since then, and has fallen almost 15% this year. The S&P 500 is up about 17%.
The XBI has been climbing this month, however, and on Tuesday jumped 5.4% to close at $70.91.
From the first trading day in November through the close of the market on Tuesday, the XBI has slightly outperformed the S&P 500, climbing 8.8% while the S&P 500 has climbed 7.9%.
The biotech bump hasn't been tied to any broader rally among healthcare stocks. The Health Care Select Sector SPDR Fund (XLV), which tracks the sector broadly, is up 2.9% this month, while the VanEck Pharmaceutical ETF (PPH), which tracks U.S. and global drugmakers, is up 2.4%.
Instead, Mizuho healthcare equity strategist Jared Holz told Barron's, the gains are tied to a growing consensus that interest rates won't climb any higher, and a broader rally among small-cap stocks. The Russell 2000, which tracks small-cap stocks, climbed 5.4% on Tuesday.
The Tuesday consumer price index report from the Bureau of Labor Statistics, which showed inflation is slowing in the U.S., served to bolster a growing consensus that the Federal Reserve won't raise interest rates in December. The current high interest rates have been challenging for biotechs and growth stocks, both because they increase companies' funding costs and because higher yields on investments such as Treasury debt reduce the current discounted value of future earnings.
Goldman Sachs analyst Asad Haider wrote in a note in early October that the XBI "has largely become an inverse rates proxy."
A temporary reprieve from rate hikes won't be a long-term salve for the XBI, however. As Haider wrote in his early October note, Goldman Sachs analysts expect "an uneven path for interest rates in the month ahead," and that "the longer-term direction of travel could remain uneven and challenging."
Holz told Barron's on Wednesday that the XBI's Tuesday jump has nothing to do with the fundamentals of the biotech sector itself.
In fact, much of the news from biotech companies over the past few weeks has been bad. Sarepta Therapeutics (SRPT) shares fell 40% on Oct. 31 after the company's gene therapy for Duchenne muscular dystrophy, Elevidys, failed to demonstrate significant improvement over a placebo in the primary measure used in a confirmatory trial. Shares of Verve Therapeutics (VERV) fell 41% on Nov. 13 over safety worries in what the company presented as a successful early trial of its gene editing technology.
The setup leaves little hope for a broader, sustained biotech rebound. The early November bump hasn't made much of a dent in the XBI's 2023 losses, not to mention the slide it has sustained since early 2021.
Share prices of many of the XBI's constituents have been decimated, including Coherus BioSciences (CHRS), down 77% this year, and Vir Biotechnology (VIR), a pandemic-era darling. That stock is down 61% this year, partly because a trial of an influenza prophylaxis it is developing failed over the summer.
Even so, trading on Wednesday started off strong for biotech stocks. The XBI was up 2% in morning trading.
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
This content was created by Barron's, which is operated by Dow Jones & Co. Barron's is published independently from Dow Jones Newswires and The Wall Street Journal.
(END) Dow Jones Newswires
November 15, 2023 12:28 ET (17:28 GMT)
Copyright (c) 2023 Dow Jones & Company, Inc.
Advocacy in rare disease: A Fireside Chat with the U.S. Commissioner of Food and Drugs
15 NOV 2023
Science Webinars is pleased to welcome Robert M. Califf, Commissioner of Food and Drugs at the United States Food and Drug Administration (FDA), for a Fireside Chat about the intersection between the FDA and rare disease. This intimate and enlightening conversation will provide unique insights into the challenges and opportunities faced by individuals and organizations dedicated to advancing research and treatment options for rare diseases.
While each rare disease may affect only a small percentage of the population, collectively their impact is enormous, with more than 7,000 rare diseases affecting 1 in 10 people. Patients, families, caregivers, and advocacy groups work tirelessly to raise awareness, accelerate research, and navigate the complex regulatory landscape. In this Fireside Chat, we will explore how the FDA, as the regulatory authority for medical products in the United States, collaborates with these advocates to ensure timely and safe access to innovative therapies for rare disease patients.
Watching this Science Webinar, viewers will:
Learn about the FDA’s commitment to addressing rare diseases and how they incentivize the development of treatments
Hear the Commissioner speak to FDA collaborations with international regulatory agencies, patient advocacy groups, and individuals with rare disease
Explore recent technological advances that are streamlining the regulatory process.
https://www.science.org/content/webinar/advocacy-rare-disease-fireside-chat-commissioner-food-and-drugs
FDA’s Califf wants to get the public as excited about life expectancy as cures
by John Wilkerson 11/8/2023
WASHINGTON — It’s easy to get people worked up about cures, but they don’t seem to be bothered by falling life expectancy, and Food and Drug Administration Commissioner Robert Califf would like to figure out how to connect the two.
The United States has one of the lowest life expectancy rates among wealthy nations. Post-pandemic, the average American life span is down to 76.4 years. The average life expectancy in comparable countries is 80.3 years.
The issue may be too abstract for most, Califf suggested Tuesday at the Milken Institute Future of Health Summit.
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https://www.statnews.com/2023/11/08/fda-robert-califf-life-expectancy-cures/
Many FDA Initiatives Are Underway to Advance Development of Rare Disease Treatments
NATIONAL HARBOR, Maryland — Rare disease product development is challenging, requiring interdisciplinary collaboration and multiple strategies to produce viable solutions and treatments, officials said at a session presented at the 2021 World Orphan Drug Congress.
To streamline the drug development process, the US Food and Drug Administration (FDA) has reorganized the Office of New Drugs within the Center for Drug Evaluation and Research (CDER). A new rare disease hub is charged with advancing drugs for rare diseases, explained Janet Maynard, MD, deputy director of the Office of Rare Diseases. The division evaluates novel trial designs, approves orphan drugs and biologics for rare diseases, and assists in patient outreach and engagement.
According to Dr. Maynard, the number of orphan drug approvals has increased over the past decade, with 58% of all new drug approvals in the CDER falling in the orphan drug category. Compared to nonorphan new molecular entities (NME), 89% of orphan NMEs utilized expedited programs through the CDER and New Biologics Approvals to prioritize resources and accelerate approval timelines.
Dr. Maynard said many challenges exist in the field of rare disease drug development, including:
-Small (sometimes extremely small) patient populations
-Genotypic and phenotypic heterogeneity within a disease
-Poorly understood natural history
-Serious, often life-threatening, progressive childhood onset of many rare diseases
-Patient reluctance to participate in randomization into placebo arms of clinical trials
-Lack of or limited regulatory precedents
-Lack of drug development tools
-Need to uphold regulatory standards while allowing for regulatory flexibility.
The FDA has also established the Rare Disease Drug Development Council (RDDDC), a forum to discuss cross-cutting rare disease topics. This council, composed of 15 representatives from the CDER, serves in an advisory capacity.
Additionally, in December 2018, the FDA drafted the framework to establish a program to consider the potential of real-world evidence (RWE) as a basis for drug approval. RWE is defined by the FDA as “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data (RWD).”
RWD is defined by the FDA as “data relating to patient health status and/or delivery of health care routinely collected from a variety of resources.” These resources include electronic medical records, medical claims data, disease and product registries, and wearable devices, among others.
Dr. Maynard suggested that “well-designed, noninterventional studies relying on fit-for-purpose RWD when compared to a suitable control can be considered adequate and well-controlled under FDA regulations.”
The FDA has also undertaken an initiative called Patient-Focused Drug Development (PFDD), in which the perspectives of patients are considered when guiding treatment development. Patients can speak of the impact that disease symptoms have on their daily lives, as well as their current management of the disease using available treatments.
FDA Decision Date Approaching for First Potential ALGS Treatment
The FDA is currently establishing 4 guidances in which patient organizations may collaborate with the FDA on collecting and submitting patient data to guide product development.
Guidance 1, finalized in 2020, sets forth the methods with which to collect comprehensive and representative input. Guidance 2, drafted in 2019, details methods to identify what is most important to patients. Guidance 3, currently in progress, will discuss ways to select, develop, or modify fit-for-purpose clinical outcome assessments. Lastly, Guidance 4, also currently in progress, will discuss the incorporation of clinical outcome assessments into endpoints for regulatory decision-making.
The FDA has also initiated the Rare Disease Cures Accelerator (RDCA), which has funded $10 million in grants to the Critical Path Institute in collaboration with the National Organization for Rare Disorders (NORD) for investment and innovation in rare disease. The RDCA considers patient perspective in assessing clinical outcomes and standardization of data and analysis platforms to better characterize rare diseases.
Lastly, the FDA has created the CDER Pilot Grant Program to develop standard core clinical outcome assessments, which measure disease and treatment burdens and their related endpoints. These outcome measures are to be made available to the public at little to no cost.
[This article was written in August, 2021 but it's still has a lot of good info]
Maria Arini Lopez, PT, DPT, CSCS, CMTPT, CIMT | Publish Date August 25, 2021
Reference
Updates on clinical development for rare diseases: endpoints, real world evidence, patient voice. Presented at: World Orphan Drug Congress USA 2021; August 25, 2021; National Harbor, MD.
All of the articles that I've read requires the drug to be FDA-approved BEFORE they repurpose it for another disease...therefore, we have to wait until after the FDA approves our NDA for Rett.
However, once A2-73 is approved, Anavex will shift into high gear and start conducting P3 trials for each disease in our pipeline. This will be exciting because Anavex can become the go-to drug for neurological diseases. But we've got to get Rett over the finished line first. God willing.
Cheers.
According to the post below by Doc328, he claims (and I fully agree) that the FDA typically wants two successful P3 studies before granting approval for a new drug application (NDA).
But Doc328 also states (and I fully agree) that the FDA can also grant an NDA if we substitute one of the P3 studies with an FDA-approved drug...which will happen after we get A2-73 approved for Rett.
So, this should answer your question as to when Anavex is going to submit an NDA for Alzheimer's Disease.
******************************************************************************************************
Doc328
Monday, June 07, 2021
The FDA generally wants 2 large phase 2 [actually phase 3] studies to generate efficacy and safety data. I don't believe that single 450 patient 1 year study will be enough for the regulatory agencies. Sometimes, when a drug is already approved for one indication, they will allow additional indications with one large phase 3 study. So if Rett is approved there is a small possibility that this could occur...
Thanks bas.
Can someone cut-and-paste this article because Seeking Alpha requires membership to access it.
Thanks in advance.
Remarks by Commissioner Robert Califf to the 2023 Food and Drug Law Institute (FDLI) Annual Conference
May 17, 2023
[This is about 50% of his remarks. He gets very excited about finding an effective therapy for Alzheimer's Disease. See the portion in bold.]
By nearly any measure, it has been an extraordinary year for the FDA. For example, even as we continued to respond effectively to the Covid-19 pandemic, we approved a wide variety of safe and effective new therapies in 2022, including 37 novel drugs never before approved or marketed in the U.S., and numerous drugs for new uses and patient populations.
I am proud that more than half of the novel drug approvals last year were for patients with rare diseases. But even with this success, we recognize that thousands of rare diseases still do not have approved treatments. We are committed to supporting rare disease research, engaging patients and caregivers, and continuing to enhance our review processes to help advance medical products for rare disease patients.
In 2022, we approved the 40th biosimilar, a significant achievement in biosimilar product development. We also approved two interchangeable biosimilars, biological products that may (subject to state law) be substituted without the intervention of a prescriber, similar to how generic drugs are substituted for brand name drugs. There are now four approved interchangeable biosimilars.
I also call your attention to two areas that are shaping up possibly to be major game changers in our ongoing battle with chronic diseases that are causing the vast majority of death and disability in our country. We are awaiting final submissions, but if the submitted data matches up with the press releases, it is possible that we could see significant reductions in rate of progression in Alzheimer’s disease. [Note this was back in May, 2023]
Make no mistake … the U.S. continues to be the number one innovator in medical products, producing drugs, biologics, tests, and devices that fuel health care around the world. But to retain this leadership we need to strengthen our structures, methods, data collection, and science to allow us to be even more efficient, flexible, and effective in the exercise of our regulatory responsibilities.
Let me offer one small but important example -- the evolving role of advisory committees. The FDA has nearly 50 advisory committees and panels, which are convened to provide FDA with independent scientific, medical, and public health advice. While regulatory decision-making ultimately rests with FDA’s full time civil servants, the breadth and complexity of our responsibilities make the use of advisory committees an important tool for obtaining independent advice regarding scientific, technical, or policy questions to inform our critical work.
As a former long-term advisory committee member, I believe strongly in the value of interchange among our experts at the FDA and experts who work outside the agency. For certain complex issues, seeking the advice of an advisory committee not only makes our decisions better, but it also adds an element of transparent societal discussion that is important. But I also believe that our advisory committee system can be improved, to enable our experts to get the best advice possible. Stay tuned for developments in this area.
One of the biggest challenges we continue to face does not involve refinement of internal processes but rather how we respond to dangerous and corrosive external factors. I’m referring of course to the continuing and growing challenge of misinformation and disinformation. Since I raised this issue with you last year, there’s been an increasing understanding of the problem across government and the nation, but little in the way of real solutions.
That lack of traction is due in part to the very nature of misinformation, which involves unaccountable or unreliable sources, often coming through unattributable social media sources. One of the most basic responsibilities of the FDA is to disseminate facts about science and medicine to the public, in order to help Americans make informed choices about their health. But by undermining confidence in science, purveyors of misinformation make this much more difficult by weakening faith in governmental and other institutions, including the FDA and, in turn, endangering the American public.
The American public is not an abstract concept—these are our family members and friends. The extraordinary tragedy that the vast majority of Americans dying from Covid are not up to date and vaccination and/or were not treated with an authorized antiviral is only the tip of a much larger iceberg at the core of our decline in life expectancy from drug overdose, suicide, gun violence and chronic diseases.
The FDA is working to aggressively to combat this, but we can’t do it alone. For instance, we recently relaunched a new updated FDA Rumor Control web page, which provides a place for people to find the facts on hot topics and learn more about how to spot and address misinformation. I encourage you to stop by our table at this meeting and take a look at the page so you can help disseminate these and other facts that counter misinformation. As members of the food and drug community and bar, you not only can play a role in doing something about misinformation but also, I would suggest, have a special responsibility to do so.
As you well know, the role and mission of the FDA to protect public health was created, defined, delineated, and mandated by Congress. The agency has made an extraordinary difference in the health and safety of American patients and consumers. And today, it is under attack like never before. Indeed, our fundamental authority to regulate the safety and efficacy of products is being challenged.
I’m not going to discuss any specific cases – I’m sure many of you follow them closely. What I want to ask you to do – as lawyers intimately familiar with, and occasionally advocates for the crucial role the FDA plays – is to stand strong for the work and principles of FDA – to not let them be distilled or destroyed through misinformation, disinformation, or partisan legal challenges.
Many of you have worked at the FDA, often in key positions. Some of you have litigated on behalf of the agency. I’ve participated in many discussions about the revolving door for biomedical scientists, but I’ve heard little discussion about the revolving door for lawyers, and while the considerations are not identical, there are significant similarities that deserve more attention and debate.
I’m not asking you not to be advocates for your clients; my hope, however, is that in your current work you go beyond simply abiding by the ethical rules governing lawyers who leave federal government practice for the private sector and seek to strengthen the environment in which we all operate.
Each of you have the ability to help shape the debate and discussion, to promote communication and collaboration, and to work productively to reinforce the importance of our institutions to progress, no matter which side of a legal proceeding you’re on. In taking this honorable path you can help to make sure that facts win out over misinformation and disinformation, that the principles we stand for are not eroded, and that our ability to protect patients and consumers is reaffirmed and preserved.
Thank you.
https://www.fda.gov/news-events/speeches-fda-officials/remarks-commissioner-robert-califf-2023-food-and-drug-law-institute-fdli-annual-conference-05172023
Doc328 would disagree with you!
Doc328
Monday, June 07, 2021
The FDA generally wants 2 large phase 2 studies to generate efficacy and safety data. I don't believe that single 450 patient 1 year study will be enough for the regulatory agencies. Sometimes, when a drug is already approved for one indication, they will allow additional indications with one large phase 3 study. So if Rett is approved there is a small possibility that this could occur...
Quote: "The apparent freeze on new trials bothers me."
It shouldn't bother you if you can see the big picture.
You see, if Anavex were to start new trials for Fragile X, Angelman Syndrome, or any other disease before Rett approval, they would have to spend millions of dollars and add years of research conducting P1, P2 and P3 trials for each disease.
However if the FDA approves A2-73 for Rett, Anavex can repurpose the drug and simply run one P3 trial for each disease.
So yes, there is a freeze...but there's a good reason for it.
See the big picture and relax.
6 Incredible Companies That Started in a Garage
From small, rented garages to multibillion-dollar companies, the following companies prove great ideas are still great ideas – no matter where they start.
Every company has an origin story, and many of those stories started from humble beginnings. From small, rented garages to multibillion-dollar companies, these companies prove great ideas are still great ideas, no matter where they started. Explore the fascinating origin stories of companies that began as ideas in a garage.
*Apple
For decades, Apple’s products have been winning over the hearts of consumers. The iPhone, which was introduced in 2007, is still one of the world’s most successful products. By 2018, the popularity and ubiquity of the iPhone helped Apple become a trillion dollar company, and today, Apple Inc. is a multinational company.
But at one time, the company was called Apple Computer, Inc. and focused solely on computers. It was founded on April 1, 1976 by two college dropouts. They wanted to design computers small enough for consumers to have them in their homes and offices. As the legend goes, the first Apple computers were made in their family's garage in Los Altos, California.
One of their first big orders was from a local retailer who ordered 50 computers at $500 each. Although they had already abandoned the business, they successfully reached their goal in just 30 days.
In 1980, sales jumped to $117 million and Apple went public. That garage is now listed as one of the city's historic properties.
Hewlett-Packard
Hewlett-Packard Co. is one of the world’s most successful companies that started in a garage. In 1939, two electrical-engineering graduates of Stanford, started their company in a one-car, 12-by-18-foot garage in the back of a house they were renting.
The company’s first product was an audio oscillator, and its first customer was Walt Disney Productions, which bought eight audio oscillators to use for certifying the surround sound systems installed in theaters for its first full-length film, Fantasia (1940).
The garage was used as a research lab, development workshop, and manufacturing facility for nearly a year before the partners outgrew it and expanded to a bigger space. The company was incorporated in 1947 and 10 years later became a public company.
Today, that humble garage is a California Historical Landmark and has been restored to what it was like in 1939.
Amazon
In 1994, just four years after being named the youngest vice president of a successful New York hedge fund, this successful professional quit his job and drove to Bellevue, Washington. He rented a house and began developing software for what he believed was an untapped online retailing opportunity in the book industry. He spent a year programming the site, Amazon.com, which went live in July 1995.
Amazon started in a garage, and because its leader couldn’t have meetings in a garage, he would meet people at a nearby bookstore, where most of Amazon's first contracts were negotiated. Two years after the company started, Amazon went public. Today, the company sells a lot more than just books; it's the world's largest online retailer.
Google
In the mid-1990s, two up and coming professionals met at Stanford and decided to start a company. In 1998, they rented a 2,000-foot Menlo Park garage. The founders established their company’s headquarters in that space, and Google became another business that started in a garage.
Within a year, Google outgrew the garage and moved to an office in Palo Alto with its eight employees.
In early 2020, Alphabet, which owns Google, became the third American tech company – joining Apple and Microsoft .
The Walt Disney Co.
Did Disney start in a garage? Technically, the Cartoon Studio did in 1923. A few years prior, they formed Laugh-O-Gram Studio, making live-action animation. After a bad business deal, Disney had to file for bankruptcy in 1921.
In the summer of 1923, the founder of Disney was living in a one-car garage that belonged to his uncle. He set up Disney Brothers Cartoon Studio, and the company filmed the Alice Comedies, which would later inspire Disney's version of Alice in Wonderland.
A few months later, they moved to a bigger lot down the street from their uncle's house, which is where they signed a deal with Universal Studios to distribute the Alice Comedies.
The garage was located about 45 minutes from today's Disneyland Park in Anaheim, California. Disneyland opened in 1955, and Walt Disney World opened near Orlando, Florida, in 1971.
Mattel
Before Mattel became synonymous with the Barbie doll, the company’s founders were making picture frames in a Southern California garage in 1945.
From the scraps of the picture frames, which were made of Lucite and flocked wood, they started making dollhouse furniture. One of the owners, who worked at Paramount Pictures, is said to have landed her husband's first order by bringing a suitcase full of the dollhouse furniture to a store on Wilshire Boulevard. The success of the dollhouse furniture pushed them to focus solely on manufacturing toys.
In 1959, the Barbie doll was invented. In the over 60 years since, the company has reinvented Barbie time and again; she has been a flight attendant, an astronaut, a president, a surgeon, and more.
The Takeaway
The world was a very different place when Mattel and many of the other companies above started from garages. But one thing is constant: where you make something happen isn’t as important as making it happen. Whether it’s a food business or another home-based venture, you can turn nothing but an idea into something limitless.
A version of this article was originally published on June 2, 2014.
*There is some question as to whether Apple started in a garage or not.
You're kidding, right?
All of the naysayers have been questioning the results of the P2b/3 trial as faulty and suspicious. Having a respected journal give an independent, peer-reviewed paper confirming the results should give us a pretty good bump.
Let's face it, our company has been beaten to a pulp because so much FUD has questioned the legitimacy of our data.
Once the journal confirms our results, I expect other media outlets to shine a light on the report...which, in turn, should attract more investors.
Should be quite exciting, IMO.
That's not a fair comparison.
The only comparison for AVXL (or any biotech company) should be the XBI...not the DOW or the S&P.
You're missing my point, Mike.
Just about EVERY biotech company is hitting rock bottom right now because they are anchored to the XBI. Once they file an NDA everything changes.
This is part of being a biotech investor.
Looks like BIVI and ANVS hit new 52-week lows. Seems like we only hear about them when they go up, but crickets when they hit the bottom.
AVXL was down today...but it held above $5.60 despite the sell-off in the XBI and the overall market.
I never said that he was new. I was quoting Bourbon's post so he could reflect on his comments.
Don't take things out of context.
Hoping that the new guy is a lot better...is not exactly an enthusiastic reception.
"But in farness to Dr. M, Fadiran might have been the best he could attract at the time. Hope the new guy is a lot better, good he has corporate pharma experience."
Quote: "I've been complaining about useless FDA bureaucrats on the AVXL payroll for years."
Well, then you should applaud his departure and welcome his replacement. No?
I'm very impressed with Mr. Goldberg's background -- especially in Regulatory Affairs -- so, this is a deliberate change to increase the professionalism and efficiency of Anavex's management team.
Mr. Goldberger has most recently been with Otsuka where he led the Regulatory Affairs efforts to gain approval for multiple CNS products, including ABILIFY MAINTENA®, REXULTI®, new indications for ABILIFY® as well other product and therapeutic area approvals. Prior to Otsuka he held senior level positions in Global Regulatory Affairs for Johnson and Johnson (J&J) Pharmaceutical Research and Development in Global Regulatory Affairs. Throughout his career he has covered the full range of investigational products development and marketed products across multiple therapeutic areas including psychiatry and neurology. Mr. Goldberger has extensive experience in working with the U.S. FDA, EMA, Health Canada, and other global health authorities. In addition, he led CMC Regulatory Affairs, Labeling, Regulatory Operations/Technology and Medical Writing areas. Mr. Goldberger holds bachelor’s and master’s degrees from Rutgers University.
“I am very excited to be working with the Anavex portfolio, which has great potential to significantly impact the treatment of patients with neurodevelopmental, neurodegenerative and psychiatry disorders,” said Mr. Goldberger. “I believe that I can help build upon the work done to date and move the products toward regulatory approval for the patients who need them.”
Anavex will be releasing their 10k sometime this month...followed by a Q & A conference call. It would not surprise me if they release the TLD the day before the 10k.
Hang in there, PW.
Good post, tred.
Seems like there's endless chorus of the same song.
Quote: "I most want to know when Fragile X and PD and other pivotal trials will commence."
I would think that pivotal trials on Fragile X, etc will start after they announce a successful P 2/3 trial with Excellence Rett. Once that is achieved, they can submit the NDA and get approval.
Once approved, they can repurpose A2-73 and run a simple P3 trial for efficacy on each disease.
Pushing for a PR based on your own desires is an uphill battle that usually ends in frustration. TGD knows that the shareholders are antsy for news regarding Rett...and he has reiterated his 2H commitment. So I'm fine with that goal.
There's an old saying in the marketing industry that professional copy writers use when creating their promotions:
The more you tell, the more you sell!
And thus, I would rather wait for a full TLD rather than one that allows people to poke holes in it.
Our patience will have a happy ending. You'll see.
You'll note that this stock was at $8/share six months ago...and then the MMs took it down to $3.53 just before the company announced a successful P3 TLR.
This is exactly how the MMs work, folks. I've seen it happen dozens of times.
We're going through the same manipulation. Just hold tight and we'll be rewarded.
Gradually moving up. Did someone turn on the HF switch for us?
Nah, just following the XBI upward.
The bottom line is that every member of the FDA Advisory board has to be accountable for their decision...and therefore, it makes their decision a lot easier if they know that a respected journal has evaluated the data and given it a positive endorsement.
So easy to rebut your rebuttal...
So easy to rebuttal....
First time leading a company...and yet, he single-handedly saved it from BK and is now debt-free with $150 Million in the bank That makes a great CFO....not CEO That doesn't change the fact that he single-handedly rescued the company from BK. Furthermore, he is responsible for building the company from 1 employee to 40 employees...creating the management structure...and attracting some of the best Biotech executives in the industry. THAT makes a good CEO.
First time doing trials...and yet, every trial has been a success. Success??? Then where is the TLD on all those successful trials??? You're taking things out of context. Most of those trials were P1s and P2s. The only P3s were the adult Rett AVATAR trial and the P2b/3 AD trial. The adult Avatar P3 is the first half of the NDA and the pediatric Excellence P2/3 is the second half. (Can't put the horse before the cart) The NDA for the P2b/3 AD trial will be submitted after the Rett trial is submitted.
First time trying to commercialize...and yet, we are 6-12 months away from commercializing A2-73 for Rett. How do you know that? We haven't seen the TLD yet??? If the Excellence trial had failed, that would be a material event and we would know that by now.
First time dealing with regulators...and yet, he was smart enough to hire the top-dog from the FDA to ensure our approval process. You got to be kidding...How many years has it been since he hired his first ex-FDA'er? Everyone was touting back then that would expedite trials...LOL...nothing was expedited...Lets hope his 3RD ex-FDA-er works out. Once again you are taking things out of context. Those other "ex-FDA'ers" were hired during the P1 and P2 trials. We just finished the AVATAR trial last year...and that is the first half of the NDA for Rett. TGD hired Dr. Kun Jin in January just after the P2b/3 AD trial ended. Everything is in place for success.
First time dealing directly with shareholders...as it turns out, the largest shareholders of AVXL are institutions. Yet the share price is controlled by the fudster bas with his daily hype (except when he is selling and reloading). If tutes were committed (besides Index funds) then we wouldn't be at $5. More likely $25. The entire market has been selling off. Until we submit an NDA we will continue to follow the XBI - which is at a 52-week low right now.
Quite an accomplishment, no? Funny things happen when one is so heavy invested that they fail to see reality. I see reality just fine. There's probably 100 biotech companies that would love to have $150 Million in the bank and debt-free. We are in great shape for the future. Not too many biotech companies can say that.
Well, if I were on the FDA Advisory board I would be more likely to approve a drug that has been accepted and published in a respected journal. (Here in the U.S., we call that CYA)
First time leading a company...and yet, he single-handedly saved it from BK and is now debt-free with $150 Million in the bank
First time doing trials...and yet, every trial has been a success
First time trying to commercialize...and yet, we are 6-12 months away from commercializing A2-73 for Rett
First time dealing with regulators...and yet, he was smart enough to hire the top-dog from the FDA to ensure our approval process
First time dealing directly with shareholders...as it turns out, the largest shareholders of AVXL are institutions.
Quite an accomplishment, no?