“A white space opportunity.” That’s how Enlaza Therapeutics Inc. co-founder and CEO Sergio Duron described to BioWorld the company’s efforts to develop the first covalent biologics, an endeavor that has gained the backing of an impressive group of investors in a recently closed $100 million series A round.
A week after Bristol Myers Squibb Co. disclosed a significant restructuring plan to focus on long-term growth drivers, the big pharma partnered with Repertoire Immune Medicines Inc. in an early stage, multiyear collaboration to develop T-cell targeted medicines for up to three autoimmune diseases, paying $65 million up front, with a potential $1.8 billion in development, regulatory and commercial milestones, along with tiered royalties.
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
Eli Lilly and Co. is planning to file for U.S. FDA approval later this year after reporting that tirzepatide met all primary and key secondary endpoints in two phase III trials in obstructive sleep apnea (OSA). Assuming approval, tirzepatide could become the first drug approved specifically for OSA, while providing potential entry access for Medicare Part D coverage, which is denied for the GLP-1 class of drugs approved as obesity medications.
Investors anxious to see data from Marinus Pharmaceuticals Inc.’s phase III Raise study testing intravenous ganaxolone in refractory status epilepticus (RSE) will have to wait a little longer, as the trial failed to meet the predefined criteria for stopping at the interim analysis, sending shares (NASDAQ:MRNS) down nearly 83%.
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.
