Amylyx Pharmaceuticals in the NEWS Amylyx Pharmaceuticals (AMLX) announced that the U.S. FDA has approved RELYVRIO™ (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS). The product, RELYVRIO, (previously known as AMX0035 in the U.S.) has significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled . . . This content is for paid …

Biogen & Eisai Co and Early Alzheimer's Disease  From Tokyo and Cambridge, Eisai Co., Ltd. and Biogen Inc. (BIIB) announced positive topline results from Eisai’s large global Phase 3 confirmatory Clarity AD clinical trial of lecanemab. The product is an investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of mild cognitive impairment (

FDA Approves bluebird bio Product Skysona for Early, Active CALD SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys with this devastating and fatal neurodegenerative disease. The U.S. FDA has granted Accelerated Approval of bluebird bio’s (BLUE) SKYSONA® (elivaldogene autotemcel), also known as eli-cel. SKYSONA® is indicated to slow the progression of neurologic dysfunction in boys 4-17 …

Intellia Therapeutics  Intellia Therapeutics.(NTLA) and Regeneron Pharmaceuticals (REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Rhythm Pharmaceuticals Rhythm Pharmaceuticals (RYTM): After soaring, based on great scientific and drug performance in treating genetic obesity, the selloff of the stock has occurred today after the firm’s announcement and pricing of its public offering of 4.8M shares of its common stock at $26 a share. Akero Therapeutics Akero Therapeutics (

Akero Therapeutics Akero Therapeutics (AKRO) - a clinical-stage company, today released topline data from HARMONY, a 24-week Phase 2b study evaluating the efficacy and safety of its lead product candidate efruxifermin (EFX) in patients with pre-cirrhotic nonalcoholic steatohepatitis (NASH), fibrosis stage 2 or 3 (F2-F3). The study met its primary endpoint for both the 50mg . . . This content is for paid subscribers. Please …

Amylyx Pharmaceuticals in the News Amylyx Pharmaceuticals (AMLX): The U.S. FDA Peripheral and Central Nervous System Drugs Advisory Committee has voted (7 yes votes and 2 no votes) that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol ) for the treatment of amyotrophic lateral sclerosis (ALS). The advisory committee’s decision was based on a review . . . This content is …

IVERIC Bio ~ September 6, 2022 IVERIC bio (ISEE) announced positive results from GATHER2 second Phase 3 clinical trial of Zimura® (avacincaptad pegol) - a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA). GATHER2 met its prespecified primary endpoint of mean rate of growth (slope) in GA area at 12 months with statistical significance and a . . . This content …