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.
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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.
With 70 partnered development programs, it is a chore to keep tabs on the progress at Ligand Pharma LGND. Continue reading below for a few updates from partner companies disclosed on 3q-2012 earnings conference calls.
Ligand Pharma $LGND has built a business around a large portfolio of partnerships in which other biotech and pharma companies develop drugs at no cost to LGND. The upside to shareholders from any one deal is lower, but the many "shots on goal" and growing royalties from PROMACTA and KYPROLIS offer a lower risk investment opportunity compared to many biotechs.
Continue reading below for a round-up of notes on LGND-partnered programs from a number of partners from 2q-2012 earnings conference calls.
Ligand and GlaxoSmithKilne $GSK will present early stage data for the use of PROMACTA (eltrombopag) in chemotherapy-induced thrombocytopenia at ASCO 2012 in June. Also, partner Merck will present phase 2 melanoma data for SCH727965. Finally, there will be several presentations from Onyx Pharma $ONXX related to carfilzomib. Abstracts will be released May 16th so stay tuned.
First quarter 2012 Partner Updates:
Merck $MRK and Baxter $BAX have partnered programs with LGND that were not discussed on their respective 1q-2012 earnings calls.
Partner Onyx Pharma $ONXX also has the multiple myeloma drug (which utilizes Ligand Captisol formulation technology) up for a FDA panel review and PDUFA date this summer. I have not seen a 1q-2012 conference call transcript or listened to the webcast, but I don't think there have been any material updates while we wait for those fey FDA events.
I speculate that evacetrapib is the undisclosed Eli Lilly $LLY programs slated to enter phase 3 in 2012 that utilizes LGND Captisol technology. As this drug is a member of the troubled CETP inhibitor class, I will carefully monitor to see if this phase 3 program actually begins (and to verify if this is indeed the program partnered with Ligand). With that caveat, here are the relevant excerpts from the conference call:
"We anticipate beginning Phase III trial for evacetrapib, our CETP inhibitor"
Q: "anything to update us on the timing and design of the evacetrapib studies?"
A: "As you know, we communicated our very promising data then in the fall and we are doing extensive planning them for the Phase III trial including interaction with regulators, which we are close to completing. We have also been preparing the CMC material for the trial. So we feel very good about the options we have to start the trial before the end of this year as we have communicated."