Alexion Pharmaceuticals is no longer a one-product company, winning tandem European approvals for two rare disease drugs it hopes will mimic the success of Soliris, the world's most expensive treatment.
Amicus Therapeutics has stepped in to snap up the late-stage rare disease biotech Scioderm, beefing up its orphan drug pipeline in exchange for $229 million in stock and cash along with a promise of up to $618 million more for meeting a slate of milestones.
Boston-based Rhythm has gone back to the venture well to draw up a fresh $40 million in backing from some A-list investors to fuel a mid-stage program for rare genetic disorders linked with a lethal level of obesity.
AstraZeneca CEO Pascal Soriot has long argued that shedding some of the company's noncore businesses could help jump-start sales, adding cash to its reservoirs as it chases its lofty sales goals. Now, the British drugmaker is taking another step in that direction as it passes off one of its rare cancer drugs to Sanofi, getting $300 million in the process.
Three years ago, BioMarin top scientist Henry Fuchs pitched BMN-111 as one of the company's most promising early-stage drug prospects, with stellar mouse data to suggest that it was on track to develop a therapy to correct the genetic mutation behind dwarfism. On Wednesday evening, the San Rafael, CA-based biotech outlined Phase II data from 26 children that provided ample proof-of-concept human data that Fuchs was well justified in his belief.
Developing new drugs for depression has become one of the most frustrating tasks in biotech as companies have struggled to demonstrate a positive effect against hard-to-control placebo responses in clinical trials or prove that dramatic and quick responses in small groups can translate into safe and broadly used drugs. Sage Therapeutics, though, is touting the results it's seen in just four patients.
Would-be makers of rare disease meds, rejoice: A 6-month exclusivity extension, proposed last year in Congress, has returned. The 21st Century Cures Act, now under consideration in the House of Representatives, once again includes a measure that would prolong competition-free marketing for some meds repurposed to treat rare diseases.
Shuffling a thick deck of preclinical programs, Moderna Therapeutics has dealt out a fresh biotech hand for the third in its series of venture units tasked with recruiting a new team to shepherd messenger RNA programs into the clinic.
Alexion Pharmaceuticals has turned to Big Data specialist BioXcel for help identifying its next blockbuster rare disease drug. BioXcel is applying its rare disease database to the collaboration, which aims to figure out which indications are most likely to benefit from various therapeutics.
Cashing in on the current enthusiasm in venture circles for gene therapy upstarts, ReGenX has snagged a $30 million round that will finance a round of new hires as it sets sail for the clinic with lead programs for rare diseases of the central nervous system.