|Biotech Due Diligence
It has been awhile, so if you haven't explored my Sarepta Therapeutics SRPT resource page about the company, Duchenne muscular dystrophy (DMD), their drug eteplirsen as well as competitors, please do. One of the features is a collection of coverage of this story, from both the investor and patient/community perspectives. This post will feature a round-up of recent relevant content as we await the next updates from the company.
PS - check the Twitter folks who provided these links, all are key follows for those interested in this area.
PPS - as always, inclusion of a link here does not imply I endorse or agree with the views of the authors
Don't Take Your Vitamins
Paul Offit (New York TImes)
Profiles in Long-Termism: Sarepta Therapeutics CEO Chris Garabedian
Luke Timmerman (Xconomy)
Be sure to head over to our SRPT home page for extensive coverage of Sarepta and their DMD drug eteplirsen from investing, scientific, and patient perspectives.
Legislation would Create New Incentives, Penalties to Compel Reporting of Trials Results
Alec Gaffney (Regulatory Focus)
I can count on one hand the number of listings I have seen on Clinicaltrials.gov that had the "required" results published - hopefully the situation will be improved somehow.
Master protocol’ aims to revamp cancer trials
Heidi Ledford (Nature)
FDA Issues Final Orphan Drug Regulations
Michelle Butler (FDA Law Blog)
Opinion recap: No patent on natural gene work
Lyle Denniston (SCOTUS Blog)
Solid summary of the Myriad Genetics MYGN patent case ruling re BRCA1/BRCA2.
RNA and Oligonucleotide Therapeutics
April 10 - 13, 2013
Cold Spring Harbor Laboratory (CSHL)
Conference homepage link
Ryszard Kole, Sarepta Therapeutics
Adrian Krainer, Cold Spring Harbor Laboratory
Art Krieg, RaNA Therapeutics
Bruce Sullenger, Duke University
There was a great deal of discussion around this conference, and particularly the data presentation by GSK/Prosensa for their Duchenne Muscular Dystrophy DMD drug drisapersen. This drug is of course the key competitor to eteplirsen from Sarepta Therapeutics SRPT. Key links and info are archived in this post.
There is much discussion recently about the side effect of proteinuria (protein in the urine, a potential indicator of kidney damage) in Duchenne Muscular Dystrophy trials from Sarepta Therapeutics SRPT (eteplirsen) and GSK/Prosensa (drisapersen). An early stage trial of the Prosensa drug showed proteinuria in all patients, but the actual raw lab values for the small number of patients with further follow-up were quite modest (see details below). Clearly, in later trials these adverse effects were much more serious, as it has been reported that multiple buys required hospitalization.
I came across a few links in reading about this topic so I thought I would share them here in one place.
Also visit the index of all of the SRPT research on BiotechDueDiligence.
(continue reading for full post)