Back at the beginning of 2014, a fast-growing Regeneron revealed that it had been building a new genetics research center that would zero in on carefully targeted diseases as it built new drug development programs from the ground up. Eighteen months later the big biotech says the effort--led by some top researchers wooed in to run the operation--has paid off with a clear target for a rare genetic bone disease. And they have an antibody in preclinical testing that could correct the disorder.
Sage Therapeutics is pressing forward in the field of tremor treatment after its lead drug notched a positive--though not statistically significant--effect on the debilitating condition in Phase II, results the company believes will help light the way for a bigger trial.
LimmaTech Biologics, a spillover from the $190 million (€169 million) takeover of GlycoVaxyn by GlaxoSmithKline, has opened its doors. The biotech is starting life with GlycoVaxyn's research operation and a 5-year agreement to pump novel bioconjugate antigen‐based vaccines into GSK's pipeline.
Amgen is buying into Novartis' early-stage BACE program for Alzheimer's, paying an unspecified upfront and milestones in exchange for a cut of the program, which looks to slash the production of a key toxic protein widely viewed as a prime suspect in the development of the memory-wasting ailment.
Sanofi is backing out of a collaboration with Ardelyx that could have paid out nearly $200 million, returning a handful of candidate treatments for high phosphates in patients with kidney disease.
Trevena's treatment for pain following abdominoplasty met its goals in a Phase IIb study, clearing the way for late-stage development and sending the company's share price soaring.
A new drug for blood cancer from Bristol-Myers Squibb and AbbVie is on the fast track to FDA approval, winning a priority review to treat multiple myeloma.
Late last year San Francisco-based gene therapy upstart Audentes Therapeutics gathered a $42.5 million venture round from some A-list investors with plans to use the money to bankroll some new deals. Today, it made good on the plan and pulled back the covers from a buyout that gives the little biotech player a third preclinical program as it tackles a hot field.
Now that Amgen and the Regeneron/Sanofi team have scored pioneering approvals of two leading PCSK9 therapies, the race is already on to top the leaders with a new wave of even better drugs that can slash levels of bad cholesterol more efficiently. And Alnylam, alongside its partners at The Medicines Company, has staked out a claim to an RNAi treatment that could beat them all--some 5 years down the road.
The FDA has awarded priority review status to Sarepta Therapeutics' eteplirsen. The regulatory go sign means Sarepta is still tucked in just behind BioMarin in the race to bring a therapy for Duchenne muscular dystrophy to market, with analysts tipping the agency to make a decision on both drugs by the end of the year.