|Biotech Due Diligence||
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.
Fourth Quarter and Year-end 2012 earnings season is past now form Ligand Pharma as well as big pharma/biotech players. I assembled the latest (and historical) sales and royalty information for LGND's key product PROMACTA (eltrombopag) and it's sole competitive, NPLATE from Amgen AMGN.
(continue reading the full post for the figures and commentary)
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)
Following a joint venture spin-off from Alnylam ALNY and Isis Pharma ISIS, Regulus Therapeutics was born to focus on drug discovery using microRNA technology. Their 9/7/2012 revised S-1 filing revealed a planned price range $10-12 (but the offering was actually priced at $4 and Regulus now trades as ticker RGLS). Continue reading below for some highlights from the prospectus about the company and its research programs.