With two FDA meetings in its hip pocket to discuss maralixibat for treating pruritus associated with Alagille syndrome, Mirum Pharmaceuticals Inc. got a resounding boost from the market Monday as its stock (NASDAQ:MIRM) closed up 111%, at $17.28.
In August the FDA was skeptical about Sarepta Therapeutics Inc.’s injectable Vyondys 53 (golodirsen), but that changed swiftly Friday with the agency’s accelerated approval for the Duchenne muscular dystrophy (DMD) follow-on therapy, the first treatment specifically for this subtype.
Neurotrauma Sciences LLC and Emory University’s Institute for Drug Development entered a license agreement for patents and technology invented at the school, enlarging a deal the two cut about a year ago.
ORLANDO, Fla. – The world’s biggest and certainly most lavish hematology gathering, the 61st American Society of Hematology conference, just ended in Orlando, having brought 30,024 people from 25 countries to glory in Florida sunshine, if they got outside, but mostly to bask in the discipline’s most up-to-the-minute data. The amount of research was staggering, with 5,978 abstracts available for review. Key themes included work aimed at overcoming obstacles to CAR T therapy, new progress in preventing and treating venous thromboembolism, moves to address health care disparities and new developments in the care of sickle cell diseases. Late-breakers highlighted new data on Blincyto (blinatumomab, Amgen Inc.), Sanofi SA's sutimlimab, azacitidine and Darzalex (daratumumab, Janssen Biotech Inc.).
ORLANDO, Fla. – As the enormous American Society of Hematology annual meeting wound down, the Janssen Pharmaceutical Cos. of Johnson & Johnson released phase III data showing that adding Darzalex to carfilzomib and dexamethasone, compared to carfilzomib and dexamethasone alone, significantly improved progression-free survival in patients with relapsed/refractory multiple myeloma.
ORLANDO, Fla. – Data from two studies from Forty Seven Inc. announced at the American Society of Hematology (ASH) annual meeting moved the market in a major way Monday as the company stock (NASDAQ:FTSV) soared 111% to close at $30.43, up $15.99.
ORLANDO, Fla. – Not long past the opening of the American Society of Hematology’s (ASH) massive annual conference, Janssen Biotech Inc. released upbeat follow-up survivability data on Imbruvica (ibrutinib) from two studies and an integrated analysis evaluating Imbruvica in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
ORLANDO, Fla. – New phase I/II data from Autolus Therapeutics plc announced at the American Society of Hematology’s (ASH) annual conference show that AUTO-3, the first bicistronic CAR T targeting CD19 and CD22 followed by an anti-PD1, was well-tolerated in a phase I/II study.
