Dimension Therapeutics Inc.'s $20 million up-front gene therapy deal – with the potential for $232 million in milestone payments – comes less than a year after the company was founded and draws to the table Bayer Healthcare Pharmaceuticals Inc., which gains rights to "the strategic must-have for them to maintain an ongoing presence in the hemophilia A space," said Dimension's CEO and co-founder Thomas Beck.
Taking advantage of the immuno-oncology updraft, Kite Pharma Inc. priced an initial public offering (IPO) of 7.5 million shares at $17 each, for gross proceeds of $127.5 million that will help advance its anti-CD19 chimeric antigen receptor (CAR) T-cell therapy.
Sold as a nutritional supplement – before it was ruled a drug by the FDA and went through phase II trials in the hands of Biostratum Inc. – pyridoxamine dihydrochloride entered a pivotal program overseen by Nephrogenex Inc., which is testing the derivative of vitamin B6 (pyridoxine) in diabetic nephropathy.
Insmed Inc.'s eventful ride with Arikayce (inhaled liposomal amikacin) took an upswing with word from the FDA that the compound has achieved breakthrough designation in nontuberculous mycobacterial (NTM) lung disease, despite missing its primary endpoint in a phase II trial in that indication.
Ipierian Inc. spinout True North Therapeutics Inc. will use $22 million from its series A round to push TNT009, its lead candidate, through early stage studies in autoantibody-driven rare diseases mediated by the complement system.
Gene therapy at last is coming into its own with a new kind of "p" value, said Bluebird Bio Inc. CEO Nick Leschly. "Instead of publications, it's now about patients," he said, commenting on positive results from a phase I/II trial with a gene therapy against beta-thalassemia major – data that started followers of Bluebird speculating on whether the approach will work in sickle cell disease (SCD), too.
Fresh from a prostate cancer deal with Johnson & Johnson unit Janssen Biotech Inc. and in the midst of ushering an immunotherapy bid against pancreatic cancer through phase IIb trials, Aduro Biotech Inc. nailed down $55 million in a series C round that should see the privately held company into 2016.
Half of all multiple sclerosis (MS) patients in the nearly $16 billion (and growing) market will be taking oral drugs two years from now, and they'll cycle from one to another, just as they've done in the past with other therapies, predicted Receptos Inc. CEO Faheem Hasnain.
Bolstering its hepatitis C virus (HCV) position as patent skirmishes continue in the space, Merck & Co. Inc. snatched up Idenix Pharmaceuticals Inc. for $24.50 in cash per share, or about $3.85 billion, in a 300 percent-plus premium deal approved by the boards of both companies and set to close during the third quarter of this year.
Jitters over prospects with any candidate in Alzheimer's disease (AD) failed to deter investors from putting up to $30.8 million into Transition Therapeutics Inc., which not only has AD work ongoing but also a phase II trial in type 2 diabetes with heavyweight partner Eli Lilly and Co.
