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.
Though not a huge surprise, the complete response letter (CRL) from the FDA for Lemtrada (alemtuzumab) sent a rumble through the multiple sclerosis (MS) therapy space, and shook investors in Genzyme Corp., which has been told by the agency that approval of the CD52-targeting antibody requires more trials.
With vaccines in the works for herpes simplex virus 2 (HSV-2) and pneumonia, Genocea Biosciences Inc. added to a banner year for initial public offerings (IPOs) in biotech, filing to go public and raise $75 million by way of selling an unspecified number of shares at an also-undisclosed price range.
Caught off guard by the FDA’s approval – and ahead of schedule, to boot – of Orenitram (treprostinil) extended-release tablets for pulmonary arterial hypertension (PAH), United Therapeutics Corp. plans to launch the product within the next six months.
Intrexon Corp.’s $26 million takeover of Medistem Inc. brings aboard endometrial regenerative cells (ERCs), derived from menstrual blood, for use with integrated synthetic biology platforms developed by Intrexon, opening potential for cell-based therapeutic candidates across a range of conditions.