SAN FRANCISCO – As California’s year-long drought continued, Regeneron Pharmaceuticals Inc. refreshed investors at the 32nd Annual J.P. Morgan Healthcare Conference (JPM) with news of a new deal for a combination therapy in wet age-related macular degeneration (AMD).
SAN FRANCISCO – As Wall Street calmed in the wake of Alnylam Pharmaceuticals Inc.’s double shot of news – a $700 million expansion of the pact with Sanofi SA’s Genzyme unit, and the $175 million buyout of RNAi peer Sirna Therapeutics Inc. – investor eyes turned to the prospects of ALN-TTR-sc, the subcutaneous therapy targeting transthyretin (TTR), in Phase II trials for familial amyloid cardiomyopathy (FAC).
SAN FRANCISCO – “Really great momentum” going into the J.P. Morgan Healthcare Conference this year seems likely to persist, at least through 2014, said Thomson Reuters Recap analyst Laura Vitez.
Wall Street rejoiced over Intercept Pharmaceuticals Inc.’s stoppage for efficacy of the FLINT Phase IIb trial with obeticholic acid (OCA) for the treatment of non-alcoholic steatohepatitis (NASH) – “the coming tsunami of liver disease,” said CEO Mark Pruzanski.
In its attempt to “replicate the Gleevec story in other tumor types,” Blueprint Medicines Inc.’s Series B round brought in $25 million that will fund the push led by “an elite group of people who have specialized in developing selective kinase inhibitors to known or very clear genomic drivers of cancers,” said Kyle Kuvalanka, chief business officer.
The rebound story of the week came via Epizyme Inc., which climbed mightily on news from the Phase I trial with its EPZ-5676 DOT1L histone methyltransferase (HMT) inhibitor, hitting a proof-of-concept milestone and bringing a $25 million payment from collaborator Celgene Corp.
Genetic profiling company 23andme Inc., facing a stern letter from the FDA and a separate lawsuit that describes the saliva-derived data as “meaningless,” will not provide health information to new customers until the regulatory matter is handled.
Neuralstem Inc.’s $19.65 million from a stock offering will help advance the lead compound NSI-566 in amyotrophic lateral sclerosis (ALS), which is “about halfway through” a Phase II trial, said CEO Richard Garr.
In Phase III with its quest for a topical Botox, Revance Therapeutics Inc. filed for an initial public offering (IPO) to raise as much as $86.25 million, cash that would help efforts with RT001, a physician-applied topical formulation of botulinum toxin type A, most advanced for lateral canthal lines (crow’s feet), but undergoing tests in other indications as well.
Cardium Therapeutics Inc.’s first data from the first stage of its Phase III trial with its gene therapy Generx (alferminogene tadenovec, Ad5FGF-4) in patients with myocardial ischemia due to arteriosclerosis were favorable enough that the firm is accelerating the study, with interim data in the first half of next year.
