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.
Sage Therapeutics has successfully resolved a rare seizure disorder among the majority of patients in its small but closely watched Phase I/II study, adding more promising outcomes to the early evidence that helped inspire one of this year's most successful biotech IPOs. In the data update out Monday morning investigators say the 73% overall response rate in their study is also being mirrored among a small group of patients who are receiving the drug on an emergency-use basis.
Rare disease drugs can cost as much as $400,000 a year, and those hefty price tags have sparked a lively debate about the cost of treating rare diseases over the long term.
Some orphan drugs to treat rare diseases can cost as much as $400,000 a year. Those hefty price tags have sparked a lively debate in recent years about whether the American health care system can shoulder the burden of treating rare diseases over the long term.
Edison Pharmaceuticals' in-development rare disease treatment missed its primary endpoint in a mid-stage trial, denting the company's plans for an expansive take on CNS R&D.