With the recent removal of its amyotrophic lateral sclerosis drug from the market, Amylyx Pharmaceuticals Inc. is looking to revive investor interest with interim data from its phase II Helios study testing the same drug, AMX-0035 (sodium phenylbutyrate plus taurursodiol), in Wolfram syndrome, a rare indication in which Amylyx could be leading the charge.
Following the U.S. FDA’s expansion of competing BCMA-targeting CAR T therapy Abecma (idecabtagene vicleucel) to include third-line and later treatment in multiple myeloma (MM) patients, the agency cleared Carvykti (ciltacabtagene autoleucel) from Legend Biotech Corp. and Johnson & Johnson’s Janssen unit for use in MM patients as early as second-line treatment. The label, which RBC Capital Markets analyst Leonid Timashev called a “best-case scenario,” includes no notable updates to the black box warning and should help accelerate and expand Carvykti’s update in the U.S., with 2024 revenues expected to top $950 million.
Less than a month after disclosing that its confirmatory phase III trial of Relyvrio (sodium phenylbutyrate plus taurursodiol) fell short of its endpoint, Amylyx Pharmaceuticals Inc. is withdrawing the amyotrophic lateral sclerosis (ALS) drug from the market.
Investors might not have been overly excited, but Genmab A/S executives enthused about the “complementarity” of its proposed acquisition of antibody-drug conjugate (ADC) specialist Profoundbio Inc. for $1.8 billion in cash. The deal, expected to close in the first half of 2024, marks the biggest by far for the Copenhagen, Denmark-based biopharma and the latest transaction for the red hot ADC space.
Acorda Therapeutics Inc. has filed for bankruptcy and agreed to sell its assets, including rights to its three commercialized drugs, to German biopharma Merz Therapeutics GmbH for $185 million. Palo Alto, Calif.-based Eiger Biopharmaceuticals Inc. also filed for bankruptcy.
The possibility for expanding Bristol Myers Squibb Co.’s S1P modulator, Zeposia (ozanimod), into Crohn’s disease took a hit on disappointing data from the initial analysis of its Yellowstone study, the first of two phase III trials. Results showed the study failed to meet the primary endpoint, defined as clinical remission as measured by the Crohn’s Disease Activity Index at week 12.
Boundless Bio Inc. got a lukewarm welcome on Nasdaq after pricing its IPO of 6.25 million shares at the midpoint price of $16 per share, raking in about $100 million in gross proceeds to advance its pipeline of ecDNA-driven drugs targeting oncogene-amplified tumors.
Merck & Co. Inc. is looking to a fast launch for sotatercept, its newly U.S. FDA-approved pulmonary arterial hypertension (PAH) drug. The agency cleared the drug, branded Winrevair, under priority review on its March 26 PDUFA date, marking the first in a new class of therapies the company has touted for its potential for disease modification.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria.
In what co-founder and CEO Claudia Ulbrich called the “right decision with right partner at the right point in time,” Cardior Pharmaceuticals GmbH agreed to an acquisition by Novo Nordisk A/S in a deal worth up to €1.025 billion (US$1.1 billion) that puts a potentially disease-modifying heart failure candidate in the hands of a big pharma player in the midst of establishing its presence in cardiovascular disease.
