The wholesale acquisition cost for Amylyx Pharmaceuticals Inc.'s new U.S. FDA-approved amyotrophic lateral sclerosis (ALS) treatment, Relyvrio, has been set at about $158,000 for the first year’s treatment. It jumps to about $163,000 in the second year, the company said, a change that would move it closer to the cost of competitor Mitsubishi Tanabe Pharma Corp.'s Radicava (edaravone), which costs about $165,000 annually. A 28-day prescription will cost $12,504.
Solid Biosciences Inc. found a way to continue its Duchenne muscular dystrophy push, inking a deal to acquire privately held Aavantibio Inc., at the same time nailing down $75 million by way of a private placement with institutional investors.
A serious adverse event (SAE) in one participant has led the U.S. FDA to place a partial clinical hold on Avidity Biosciences Inc.’s lead program. The action is centered on the phase I/II Marina study of AOC-1001, an antibody oligonucleotide conjugate for treating myotonic dystrophy type 1, the most common form of muscular dystrophy in adults.
It failed to meet the primary endpoint at six months, but the European chief investigator for Biogen Inc.’s phase III trial of tofersen in treating amyotrophic lateral sclerosis (ALS) now describes the study as “trailblazing,” following a six-month open label extension.
Virios Therapeutics Inc. said most likely COVID-19 had a hand in the phase IIb failure of IMC-1 (famciclovir + celecoxib), a dual COX-2/COX-1 inhibitor for treating fibromyalgia. The drug failed to hit statistical significance in dampening pain severity when compared to placebo (p=0.302).
What a difference a U.S. FDA advisory committee meeting can make. In the wake of the Peripheral and Central Nervous System Drugs Advisory Committee voting 7-2 Sept. 7 to recommend approval of Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) candidate, shares of the Cambridge, Mass.-based company (NASDAQ:AMLX) more than regained the value they lost in March when the same committee voted against approval of AMX-0035.
Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis candidate, AMX-0035, will get a rare second bite at the adcom apple Sept. 7. This time around, the Cambridge, Mass.-based company is looking to improve on its first performance by stressing the survival benefit of its drug.
“No good data goes unpunished in this market,” H.C. Wainwright analyst Andrew Fein wryly noted in an Aug. 17 research report highlighting Wall Street’s dismal response to Blueprint Medicines Corp.’s positive top-line readout of the registrational Pioneer study, in which KIT inhibitor Ayvakit (avapritinib) met the primary and all key secondary endpoints in patients with non-advanced systemic mastocytosis.
Kringle Pharma Inc.’s phase II trial evaluating its recombinant human hepatocyte growth factor ligand, oremepermin alfa, failed to meet both primary and secondary endpoints in a study of its potential to help people with amyotrophic lateral sclerosis (ALS).
Amylyx Pharmaceuticals Inc. got good news when the Institute for Clinical and Economic Review posted a revised evidence report Aug. 4 that assessed the comparative clinical effectiveness and value of the company’s AMX-0035 and Mitsubishi Tanabe Pharma America Inc.’s Radicava (edaravone) in treating amyotrophic lateral sclerosis.