"The worst disease you've never heard of." That's how patient advocacy and nonprofit group Debra (Dystrophic Epidermolysis Bullosa Research Association of America) refer to epidermolysis bullosa (EB), a rare genetic disorder characterized by fragile skin that it often leaves its sufferers with chronic open wounds and blisters.
An angina drug dating back to the 1970s until its use was curtailed due to toxicity is back in development, this time aimed at rare hereditary heart disease hypertrophic cardiomyopathy (HCM) in the hands of Heart Metabolics Ltd., which relaunched as an Irish firm and disclosed Thursday a $20 million series A round.
As the tag line for the upcoming Allicense meeting in San Francisco suggests, this year's focus is on the "next generation" of dealmaking. But that isn't just an industry buzzword.
Two more biopharma firms priced initial public offerings (IPOs), though Adamas Pharmaceuticals Inc. and Cerulean Pharmaceuticals Inc. received lukewarm welcomes on Nasdaq Thursday, as the Nasdaq Biotechnology Index – after a short-lived rally the day before – took another slide.
Deemed “fantastic” by a leukemia expert, phase I data presented by Agios Pharmaceuticals Inc. snagged the spotlight Sunday at the American Association for Cancer Research (AACR) meeting in San Diego, with isocitrate dehydrogenase-2 (IDH2)-targeting candidate AG-221 producing a complete remissions (CRs) in three of seven evaluable patients.
Halozyme Therapeutics Inc. said it is working quickly to assess an unexpected imbalance in thromboembolic event rate between treatment and control groups, which prompted a halt to its phase II trial testing PEGPH20 in pancreatic cancer, though the lack of detail left room for plenty of speculation.
Eleven months ago, Celgene Corp. tapped Forma Therapeutics Holdings LLC for a multicandidate collaboration targeting the relatively new biology of protein homeostasis. In a deal, disclosed Tuesday, the big biotech returned, inking a second, wider deal that brings Forma $225 million in up-front cash and sets the stage for two additional collaborations and a buyout option down the road.
Mention the term “hereditary ataxia” and the first disease likely to come to mind is Friedreich’s ataxia, a rare, genetic disorder that has nabbed the attention of several companies.
Briefing documents released ahead of the Endocrinologic and Metabolic Drugs Advisory Committee meeting Tuesday for Mannkind Corp.’s regulatory-plagued inhaled insulin candidate, Afrezza, indicate that executives of the Valencia, Calif.-based firm have their work cut out for them.
No news proved to be bad news for Exelixis Inc., which saw its shares drop 39.4 percent Wednesday after reporting that its phase III COMET-1 study of Cometriq (cabozantinib) will have to continue to its final analysis, disappointing investors who had hoped for the same early success seen with recently approved drugs in the prostate cancer space.
