Assembling my biofolio...
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
SELB -
ARNA -
ARNA -
ARNA -
ARNA 1Q17 CC note
Looks like etrasimod may be the key asset for ARNA now with Phase 2 data due around the end of the year. (There is P2 data coming on their PAH drug in July so that is a more near-term binary (no clue on odds of success).) Etrasimod is an S1P receptor modulator. ARNA claims the drug is differentiated from the competition as it only hits the 1, 4, and 5 receptor sub-types and spares 2 and 3. They think this will lead to a safety advantage. Anyone with any comments on the likelihood of this?
FGEN 1Q17 CC notes
1. CEO believes that roxadustat is 6 or 7 years ahead of the closest competitor in China.
2. In distinguishing roxa P3 trials from others, CEO noted that FGEN is testing roxa against placebo in non-dialysis patients whereas others are testing against active comparator and he is not sure of the logic of the latter approach.
3. In discussing potential additional indications for pamrevlumab, CEO believes they are just scratching the surface and will depend in part on "if and when" they partner the drug.
4. Believe that pamrevlumab awakens T cells in solid tumors.
5. At the 46 minute mark of the call, CEO noted that KOLs in pancreatic cancer believe pamrevlumab will become a "core therapy in pancreatic cancer soon."
TRIL/ARGX -
SMMT -
FGEN/AKBA -
FGEN/AKBA -
G1 IPO (better CDK4/6?)
https://endpts.com/hey-pfizer-novartis-and-eli-lilly-g1-therapeutics-says-it-can-beat-all-of-you-in-its-115m-ipo/
Hey Pfizer, Novartis and Eli Lilly. G1 Therapeutics says it can beat all of you in its $115M IPO
by john carroll
April 18, 2017 10:14 AM EDT
Updated: 02:51 PM
It’s been almost a year since G1 Therapeutics completed a $47 million crossover round. But now the cancer drug developer says it’s ready for a $115 million IPO, jumping into a growing queue on Wall Street with a plan to take on some of the giants in the biopharma business.
Research Triangle Park, NC-based G1 is aimed at a well known target in cancer research: CDK 4/6. Pfizer scored the pioneering OK for their blockbuster drug in 2015, fielding Ibrance. And Novartis followed up recently with Kisqali (peak sales potential around $2.5 billion), setting up a direct clash as Eli Lilly pursues its own program for abemaciclib.
So where does little G1 fit into this picture?
CEO Mark Velleca and the crew at G1 feel they have started down the path to developing CDK 4/6 drugs that can do much better than the first wave in tackling cancer. Ibrance is closely linked with neutropenia, forcing physicians to give the drug on an intermittent schedule with time off to help patients handle the side effects. G1 believes its IV CDK 4/6 is a first-in-class contender that can supersede the frontrunners on the market. And it has an oral CDK 4/6 in development for daily dosing with a preclinical therapy looking to enter the clinic soon.
These drugs, says G1 in the S-1, are also prime for partnering with a wide range of approved therapies.
The lead drug, trilaciclib, is in three clinical trials—two Phase Ib/IIa trials and a Phase II trial. G1 has burned through $65 million to get to this point.
Hatteras Venture Partners owns the biggest chunk of equity, at 19.8%. They are followed by MedImmune Ventures (16%), Eshelman (15%) and RA Capital (10%). Velleca owns a little less than 3% of the company.
CLLS -
SCLN - Zadaxin for CF?
http://www.medpagetoday.com/Pulmonology/CysticFibrosis/64504
[Questions for me are:
1. Hasn't Zadaxin had a pretty long checkered history, which explains why it has never been approved in the U.S.?
2. Since it is an old drug, presumably there isn't much patent life left for SCLN. What is the best they could hope for if Zadaxin pans out in CF?
3. Is there no one else targeting thymosin alpha 1 in general? Isn't it odd as long as Zadaxin has been around that there do not seem to be others that have, or are currently, pursuing this target?]
Peptide Drug Seen as Possible Cystic Fibrosis Tx
Polypeptide thymosin alpha-1 fixed molecular defect, cut inflammation
by Salynn Boyles
Contributing Writer
April 11, 2017
The naturally occurring polypeptide thymosin a1 (Ta1) effectively corrected a protein misfolding that drives cystic fibrosis, and reduced inflammation, according to preclinical studies.
In mouse and in vitro studies, Ta1 rectified multiple tissue defects in a mouse model of cystic fibrosis (CF), and in cells from people with the most common genetic mutation associated with cystic fibrosis, p.Phe508del, reported Allan Goldstein, PhD, of George Washington University in Washington, Enrico Garaci, MD, of the University of Rome Tor Vergata, and colleagues.
This mutation results in the production of a misfolded protein "with residual activity that is degraded by the ubiquitin-proteasome system during biogenesis," they wrote in Nature Medicine.
The results suggest that a Ta1-agent approved for use outside the U.S. may be a useful single-molecule-based treatment for the disease.
A synthetic version of the thymosin alpha 1 peptide, marketed as Zadaxin by California-based SciClone Pharmaceuticals, has been used for more than 15 years and is approved in close to three dozen countries for the treatment of viral infections, HIV, and other immunodeficiency diseases, and cancer.
Ta1 has been shown to have an excellent safety profile in clinical use when given as an adjuvant or immunotherapeutic treatment, Goldstein told MedPage Today. Goldstein is among the researchers who discovered Ta1, which was first isolated from the thymus, and synthesized it more than three decades ago.
Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) that compromise its ability to channel chloride from cell to cell.
"Because of the inherent complexity of the pathogenetic mechanisms involved in cystic fibrosis, which include impaired chloride permeability and persistent lung inflammation, a multidrug approach is required for efficacious cystic fibrosis therapy," the researchers wrote. "Corrector drugs rescue trafficking of p-Phe508del-CFTR to the plasma membrane by directly targeting the mutant protein."
"However, in site of their high efficacy in vitro, CFTR correctors show modest clinical benefits in individuals with CF who harbor the p-Phe508del mutation, even when these correctors are combined with the CFTR potentiator ivacaftor," they stated.
Ta1 has been shown to potentiate immune tolerance in the lung, reducing lung inflammation in some studies.
"This led us to hypothesize that administration of Ta1 could be beneficial in cystic fibrosis to alleviate inflammation at an early stage of the disease and/or in individuals who are newly diagnosed," the researchers wrote.
In the current studies, Ta1 was shown to improve inflammation and immune tolerance in a cystic fibrosis mouse model and in vitro, as well as improve the location and stability of mutant CFTR. The polypeptide was also shown to rescue CFTR protein and rescue p.Phe508del-CFTR in mice and human bronchial epithelial (HBE) cells.
"We found that intravenous administration of Ta1 corrected the misfolded (CFTR) protein that occurs because of this mutation which inhibits the ability of cells to regulate chloride ions," Goldstein said.
The researchers noted that while the corrector activity of Ta1 has yet to be verified in human studies, "the excellent safety profile and cost effectiveness of Zadaxin in adults and children suggest that Ta1 could be tested in clinical trials for possible pulmonary and extra-pulmonary benefits in individuals with cystic fibrosis."
Goldstein told MedPage Today that Garaci and others in Italy hope to soon obtain approval for a phase I study of the agent in patients with cystic fibrosis. If approved, the small study is expected to include adolescent (age 12 to 18) patients with cystic fibrosis and the study duration will be around 2 months, he said.
"Thousands of adults and children [outside the U.S.] have used this agent and it has a very good safety profile," Goldstein said. "I do expect this phase I trial to be approved."
He added that the first children were treated with Ta1 in the mid-1970s.
"It is exciting to think that all these years later, this synthetic peptide may prove effective as a one-drug treatment for both children and adults with this serious, deadly disease."
ACRS/(INCY) - ACRS patents re baricitnib
[I'm assuming for ACRS this still doesn't offset the concerns of baricitnib both being further ahead and already approved for other indications.]
ERYP.PA -
NWRN.SW -
CTMX -
Tocagen - files for IPO
http://www.nasdaq.com/article/head-case-brain-cancer-biotech-tocagen-files-for-a-86-million-ipo-cm758926
Head case: Brain cancer biotech Tocagen files for a $86 million IPO
March 09, 2017, 04:39:00 PM EDT By Renaissance Capital, Renaissance Capital
Tocagen, which is developing gene therapies for the treatment of brain cancer, filed on Thursday with the SEC to raise up to $86 million in an initial public offering.
The San Diego, CA-based company was founded in 2007 and plans to list on the Nasdaq under the symbol TOCA. Leerink Partners, Evercore ISI and Stifel are the joint bookrunners on the deal. No pricing terms were disclosed.
The article Head case: Brain cancer biotech Tocagen files for a $86 million IPO originally appeared on IPO investment manager Renaissance Capital's web site renaissancecapital.com.
Chi-Med - positive P3 CRC data
http://www.chi-med.com/positive-ph3-fruquintinib-crc-fresco/
Chi-Med Announces Positive Top-Line Results for FRESCO, its Phase III Pivotal Registration Trial of Fruquintinib in Patients with Locally Advanced or Metastatic Colorectal Cancer
– Trial met all primary and secondary endpoints –
– Safety as expected –
– Progressing to China NDA submission mid-2017 –
– Full data to be reported at an upcoming scientific meeting in mid-2017 –
London: Friday, March 3, 2017: Hutchison China MediTech Limited (“Chi-Med”) (AIM/Nasdaq: HCM) today announces top-line results from FRESCO, its Phase III pivotal registration trial of fruquintinib in 416 patients with locally advanced or metastatic colorectal cancer (“CRC”) in China, who failed at least two prior chemotherapies, including fluoropyrimidine, oxaliplatin and irinotecan. The trial met its primary endpoint of demonstrating a clinically meaningful and a statistically significant increase in overall survival (“OS”), in the intention-to-treat (ITT) population of patients treated with fruquintinib plus best supportive care (“BSC”) as compared to patients treated with placebo plus BSC. Chi-Med is currently preparing to submit a new drug application (“NDA”) for fruquintinib to the China Food and Drug Administration.
In addition to OS, a statistically significant improvement in progression-free survival (“PFS”), a key secondary endpoint, was observed. The adverse events demonstrated in FRESCO did not identify any new or unexpected safety issues. Full detailed results are subject to ongoing analysis and are expected to be disclosed at an upcoming scientific meeting in mid-2017.
Simon To, Chairman of Chi-Med, said, “Well over a decade of effort and investment has now paid-off with these compelling Phase III top-line results. They reinforce fruquintinib’s potential to address major unmet clinical needs for patients in both China and around the world. They also open the way to our submitting a NDA on fruquintinib around the middle of this year.”
“The success of the FRESCO trial is an important milestone not just for CRC patients and Chi-Med, but also for Chinese innovation,” he added. “We believe this is one of the first home-grown, China-discovered and developed, mainstream innovation in the field of oncology to succeed in a pivotal Phase III registration trial. It shows that China has the resources, capability and perseverance to emerge as an innovator in the global oncology field. With eight small molecule drug candidates in over 30 clinical studies worldwide, Chi-Med is at the forefront of this important evolution.”
“We are pleased to be working with the innovative biopharmaceutical company, Chi-Med, on the development of fruquintinib,” said Kerry L. Blanchard, Senior Vice President of China Medicines Development Unit and External Innovation of Eli Lilly and Company (“Lilly”) China Drug Development. “This relationship highlights our commitment to help build a vibrant innovation ecosystem in China, and we look forward to our further collaboration to bring this novel medicine to patients.”
In addition to the FRESCO colorectal cancer trial, fruquintinib is being studied in China in a Phase III pivotal trial in non-small cell lung cancer (“NSCLC”), known as FALUCA; and a Phase II study using fruquintinib combined with Iressa® (gefitinib) in the first-line setting for patients with advanced or metastatic NSCLC. Other studies currently being planned, and soon to be initiated, include a Phase III study in gastric cancer in combination with paclitaxel in China, new studies in the U.S., and certain exploratory studies in combination with other oncology agents.
About VEGF and Fruquintinib
At an advanced stage, tumors secrete large amounts of vascular endothelial growth factors (“VEGF”), a protein ligand, to stimulate formation of excessive vasculature (angiogenesis) around the tumor to provide greater blood flow, oxygen, and nutrients to the tumor. VEGF and VEGF receptors (“VEGFR”) play a pivotal role in tumor-related angiogenesis, and the inhibition of the VEGF/VEGFR pathway. This represents an important therapeutic strategy in blocking the development of new blood vessels essential for tumors to grow and invade.
Fruquintinib (HMPL-013) is a highly selective small molecule drug candidate that has been shown to inhibit VEGFR 24 hours a day via an oral dose. It is currently under the joint development in China by Chi-Med and its partner Lilly. Two clinical studies are ongoing in lung cancer, including a late stage, pivotal Phase III registration study (FALUCA). In addition, fruquintinib is also in clinical development for the treatment of gastric cancer.
About FRESCO and Colorectal Cancer
The FRESCO trial is a randomized, double-blind, placebo-controlled, multicenter, Phase III pivotal trial in patients with locally advanced or metastatic CRC who have failed at least two prior systemic antineoplastic therapies, including fluoropyrimidine, oxaliplatin and irinotecan. No drugs have been approved in third-line CRC in China, with BSC being the general standard of care. Enrollment was completed in May 2016. 416 patients were randomized at a 2:1 ratio to receive either: 5mg of fruquintinib orally once per day, on a three-weeks-on / one-week-off cycle, plus BSC; or placebo plus BSC. The primary endpoint is OS, with secondary endpoints including PFS, objective response rate (“ORR”), disease control rate (“DCR”) and duration of response (“DoR”). Additional details of the FRESCO study may be found at clinicaltrials.gov, using identifier NCT02314819. Full results from the FRESCO study are planned to be published at a scientific event in mid-2017.
CRC is the second most common cancer type in China, with about 380,000 new cases per year, according to CA Cancer Journal for Clinicians 2016. There were approximately 1.5 million new CRC cases globally in 2015 which are expected to increase to approximately 1.7 million new cases per year by 2020, according to Frost & Sullivan.
About Fruquintinib in Lung and Gastric Cancer
Lung: The FALUCA trial is a randomized, double-blind, placebo-controlled, multi-center, Phase III registration study targeted at treating patients with advanced non-squamous NSCLC, who have failed two lines of systemic chemotherapy. Enrollment began in December 2015. Patients are randomized at a 2:1 ratio to receive either: 5mg of fruquintinib orally once per day, on a three-weeks-on / one-week-off cycle, plus BSC; or placebo plus BSC. The primary endpoint is OS, with secondary endpoints including PFS, ORR, DCR and DoR. Chi-Med plans to enroll approximately 520 patients in about 45 centers across China. Additional details about this study can be found at clinicaltrials.gov, using identifier NCT02691299. Topline results from the FALUCA study are expected to be released in early 2018.
In January 2017 Chi-Med initiated a multi-center, single-arm, open-label Phase II study of a combination therapy using fruquintinib and Iressa® (gefitinib) in the first-line setting for patients with advanced or metastatic NSCLC. The objectives are to evaluate the safety and tolerability as well as preliminary efficacy of the combination therapy in the first-line setting for advanced or metastatic non-squamous NSCLC patients with epidermal growth factor receptor (EGFR) activating mutations. Additional details about this study may be found at clinicaltrials.gov, using identifier NCT02976116.
Gastric: Chi-Med completed a Phase I/II dose finding study of fruquintinib in combination with paclitaxel, which established a combination regimen that was well tolerated. Results of this study were published at the 2017 Gastrointestinal Cancers Symposium sponsored by the American Society of Clinical Oncology in January 2017. Additional details about this study can be found at clinicaltrials.gov, using identifier NCT02415023. A pivotal Phase III registration study is expected to start during the first half of 2017.
About Chi-Med
Chi-Med is an innovative biopharmaceutical company which researches, develops, manufactures and sells pharmaceuticals and healthcare products. Its Innovation Platform, Hutchison MediPharma Limited, focuses on discovering and developing innovative therapeutics in oncology and autoimmune diseases for the global market. Its Commercial Platform manufactures, markets, and distributes prescription drugs and consumer health products in China.
Chi-Med is majority owned by the multinational conglomerate CK Hutchison Holdings Limited (SEHK: 0001). For more information, please visit: www.chi-med.com.
FGEN -