Supernus Pharmaceuticals Inc. CEO Jack Khattar, in disclosing his firm’s plan to acquire Adamas Pharmaceuticals Inc., said the team there has made “significant progress in redirecting” the Parkinson’s disease (PD) drug Gocovri (amantadine), but plenty more potential remains on the table with the oral selective RET kinase inhibitor.
Allogene Therapeutics Inc. officials took many questions but had few answers during a conference call regarding the FDA’s clinical hold after the report of a chromosomal abnormality in ALLO-501A CAR T cells in a patient treated in the phase I/II Alpha2 study.
With its topical gel for primary axillary hyperhidrosis (excessive underarm sweating) in adults and children 9 years of age and older, Brickell Biotech Inc. is gearing up to challenge Journey Medical Corp.’s Qbrexza (glycopyrronium), an anticholinergic agent contained in a single-use, pre-moistened, medicated cloth.
The start of a phase Ib/II trial with nanatinostat by Viracta Therapeutics Inc. has brought new attention to the burgeoning field of synthetic lethality, where a number of players are piquing the interest of Wall Street.
In the year’s second biggest M&A deal, Merck & Co. Inc. will take over pulmonary and hematologic specialist Acceleron Pharma Inc. for $11.5 billion. The acquisition brings Merck a pair of potential blockbuster drugs, one of them already marketed. There is sotatercept, in development for treating pulmonary hypertension (PH), and also Reblozyl (luspatercept-aamt), the first and only erythroid maturation agent approved in the U.S., Europe and Canada for treating anemia in certain blood disorders.
As expected, Mirum Pharmaceuticals Inc. won FDA clearance for Livmarli (maralixibat), a minimally absorbed ileal bile acid transporter inhibitor, for cholestatic pruritus in patients with the rare liver disease Alagille syndrome, 1 year of age and older.
Amicus Therapeutics Inc.’s plan to spin off its gene therapy work by way of the combination with blank check firm Arya Sciences Acquisition Corp. IV “allows us to be laser-focused on maximizing the opportunity for Galafold [migalastat]” while setting up launch preparations for AT-GAA, said Bradley Campbell, chief operating officer.
Imugene Ltd.’s recent win with a patent granted in Japan with its HER-Vaxx immunotherapy, in development for HER2-positive gastric cancer, represented a smallish but significant win in the HER2/neu space, where plenty of other players are busy, with Greenwich Life Sciences Inc. a strong emerging name.
What one analyst characterized as “a little bit of an exotic or at least unusual statistical analysis on the phase II primary endpoint” may have dampened enthusiasm somewhat for Evelo Biosciences Inc.’s results in mild and moderate psoriasis with EDP-1815, but the firm is advancing the compound to registration studies.
Capricor Therapeutics Inc. CEO Linda Marban said the company’s CAP-1002 for Duchenne muscular dystrophy (DMD) should serve as “adjunctive to any of the therapies out there” – a handful are approved – and bring bonus favorable effects on cardiac function that one analyst called “a one-two punch” against the disease.
