Assembling my biofolio...
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Aprea (p53)
NTRP -
VSAR vs. ASND
Is there any obvious reason as to why ASND now trades at a sharply higher valuation than VSAR ($900M mkt cap vs. $600M)? Couple of comments/questions as I don't know growth hormone space well and only recently came across these names:
1. In general, I think these long-acting growth hormone drugs are more about convenience than any kind of advance in efficacy or safety versus the cheap daily injections. I don't know if that perhaps raises questions about pricing and reimbursement for them.
2. According to VSAR, they are now about a year ahead of ASND along with I believe PFE and NVO in bringing a long-acting growth hormone for growth hormone deficiency to market. Current treatment is a daily injection and VSAR drug, if successful in ongoing P3 trial for pediatric GHD that reads out around Sept. this year, would only require twice-monthly dosing. ASND version which, again, is a year or so behind, would require weekly injections. I get that both are meaningful improvements but, all things being equal, I would assume a child would prefer only two injections per month versus four.
3. Is there some issue with VSAR data to date vs. ASND data which can explain this discrepancy? I do see that VSAR drug was on a hold before (#msg-113418688 ) and CEO was replaced at same time so perhaps this could be part of it but hold was lifted two months later.
4. I do see that ASND does have a much broader pipe than VSAR, including early deals with SNY and Genentech, so perhaps this is part of the reason. See: http://ascendispharma.com/product-pipeline/overview/ vs. http://www.versartis.com/pipeline/ . Still, unless there is some obvious issue in data to date, VSAR GHD drug on the surface looks like it could be better and this is the lead asset for both companies.
ENTA -
SMMT @ BioCEO
1. Initial 24-week data from P2 trial of DMD drug ezutromid expected in 2Q or 3Q this year. Drug being tested in 40 DMD boys.
2. 3 buckets of endpoints for the trial: mechanism, muscle health, and functional tests.
3. For mechanism, there will be biopsies. Each boy will get 2 biopsies. All get biopsy at baseline and half get second biopsy at week 24 with other half getting biopsy at week 48.
4. For muscle health, there will be MRIs to look at disease progression, in particular the ratio of water to fat. As DMD progresses the ratio shifts more towards fat due to muscle breaking down and being replaced with fibrotic tissue.
5. For functional tests, SMMT will measure 6MWT and Northstar Ambulatory Assessment.
6. 30 boys will get the F3 formulation and 10 boys will get the F6 formulation. The F6 formulation previously showed a 6-fold increase in plasma levels compared to F3.
7. SMMT will also look at biomarkers of muscle regeneration through quantification of developmental myosin. If the drug works, there should be less regeneration of muscle fibers and therefore less developmental myosin in muscle fibers.
8. If data is positive, SMMT expects to start a P2 placebo-controlled trial in 2H17, which could support a potential filing in 2019.
GNRO - CRADA agreement for ALS
[Does anyone have any comments on likelihood of increased expression of HERV in various diseases, such as MS, which is lead indication for GNRO and P2B reads out end of year, being a fundamental driver of disease or is it more likely to be just a symptom tied to some other driver? I'm just assuming the latter given GNRO's valuation and general lack of interest in the company.]
http://www.geneuro.ch/data/news/GeNeuro-PR-NIH-CRADA-ENG.PDF
GeNeuro Signs CRADA Agreement with NIH to Develop Novel Antibody Treatment for ALS
? Partnership focuses on antibodies targeting HERV-K, a potential ALS causal factor
Geneuro, Switzerland, 7 February 2017 - GeNeuro (Euronext Paris: CH0308403085 – GNRO), a biopharmaceutical company developing new treatments for neurological disorders and autoimmune diseases, including multiple sclerosis (MS), announced today the signing of a Cooperative Research and Development Agreement (CRADA) with The National Institute of Neurological Disorders and Stroke (NINDS), part of the U.S. National Institutes of Health (NIH), to develop novel therapeutic antibodies for the treatment of amyotrophic lateral sclerosis (ALS). The research will evaluate the ability of these antibodies to neutralise a potential causal factor of ALS, the envelope protein of HERV- K (a family of Human Endogenous Retroviruses, HERVs).
Under the terms of the agreement, GeNeuro will provide antibodies designed to block the activity of HERV-K envelope protein. These candidate antibodies will be tested in cellular and animal models of HERV-K associated ALS by the NINDS with the aim to achieve preclinical proof-of-concept of this novel therapeutic avenue addressing ALS pathogenesis.
“This agreement truly combines the strengths of both parties; the pioneering work done by GeNeuro in the HERV field, especially in the development of antibodies able to neutralize HERV encoded proteins in associated diseases, and the excellent NIH research on the involvement of HERV-K in sporadic ALS led by Dr. Avindra Nath and his group,” said Hervé Perron, Chief Scientific Officer at GeNeuro. “With this partnership, we aim to show that blocking this pathogenic HERV protein could lead to a novel ALS treatment and, in time, expand the GeNeuro clinical pipeline into additional neurological disorders.”
Dr. Nath and his research group recently discovered the targeted expression and the pathogenic effects of the envelope protein from HERV-K in ALS1. Furthermore, the NIH team has developed cellular and transgenic mouse models that can be used to evaluate the anti-HERV-K antibodies as therapeutics candidates to treat ALS.
“We are excited about this collaboration as an initial step towards developing a therapeutic approach
for altering the course of the disease for patients with ALS,” said Dr. Nath, clinical director at the
National Institute of Neurological Disorders and Stroke (NINDS).
KTOV -
BIIB/Eisai -
MDCO -
PCSK9/MDCO -
BIIB/Eisai -