Amid news of the splashy $4.8 billion Amicus buy, BioMarin has slipped in the discontinuation of a genetic liver disease candidate it had once hoped would become a best-in-class treatment.
The oral small molecule, coded BMN 349, was being studied in a phase 1 trial for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease. The inherited disorder elevates patients' chances of developing certain diseases, including liver scarring.
But BioMarin has ended development of the oral therapeutic, according to a one-line document filed with the Securities and Exchange Commission on Dec. 22. The decision was “announced” on Dec. 19, according to the document—the same day the company broadcast its Amicus acquisition, which is the largest transaction in the company’s 28-year history. No press release about the discontinuation has been publicly posted.
“BioMarin regularly assesses all programs in our portfolio to ensure strategic fit and to allocate our resources to developing and advancing medicines with the potential to have the greatest clinical impact for people with serious medical conditions,” a spokesperson told Fierce Biotech, citing that process as reasoning behind the discontinuation.
“This was purely a strategic decision as part of our regular portfolio review process and was not related to the safety or efficacy of BMN 349,” the spokesperson added.
Employees who were working on BMN 349 have been moved to other programs within the company, according to the spokesperson.
BMN 349’s safety and tolerability was being evaluated in a phase 1 study that enrolled about 12 patients, according to a ClinicalTrials.gov page last updated in September. The participants either had the most common severe deficiency genotype known as PiZZ, the milder genotype PiMZ or metabolic dysfunction-associated steatohepatitis (MASH).
The trial’s primary measures included reports of adverse events, lab test abnormalities, and heart rate or lung function abnormalities.
Participants were slated to receive one dose of either BMN 349 or placebo.
As recently as October, the biopharma had planned on launching a phase 2 proof-of-concept study for the asset in the first half of 2026, according to a third-quarter earnings presentation.
The discontinuation hit is padded by BioMarin’s buy of Amicus and its Fabry disease drug Galafold and Pompe disease combination treatment Pombiliti-Opfolda. The California company also gains U.S. rights to DMX-200, a potential first-in-class small molecule in phase 3 development for the rare kidney disease focal segmental glomerulosclerosis.
The acquisition is BioMarin’s second key buy of the year after the drugmaker purchased Inozyme for $270 million in the spring. The May deal brought aboard enzyme replacement therapy INZ-701, which is in late-stage development with topline data expected early next year.
Zooming out, the tossing of BMN 349 follows in the footsteps of numerous other programs BioMarin has recently discarded.
In October, the pharma said it was hoping to divest in hemophilia A gene therapy, Roctavian. Earlier in 2025, in August, BioMarin axed a preclinical phenylketonuria drug once touted as a potential successor to its approved med Palynziq.
In 2024, BioMarin shook up its corporate structure, separating into three business units—one of which was based solely around Roctavian—while also laying off 7% of its global workforce. The company also stopped work on candidates for hereditary angioedema, metabolic dysfunction-associated steatohepatitis, long QT syndrome and cardiomyopathy.
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