Long Qt Syndrome 2

The majority of affected employees were notified yesterday, BioMarin said. BioMarin had already announced plans to cut its pipeline in half, but now the company has confirmed that 170 employees are also leaving the building as part of the R&D restructure. The Voxzogo and Roctavian maker revealed last month that it was halting four programs including BMN 331, a gene therapy being assessed in a phase 1/2 trial for patients with hereditary angioedema, and BMN 255, which was being evaluated in a phase 1 trial for metabolic dysfunction-associated steatohepatitis (MASH). Two preclinical programs were also terminated: a monoclonal antibody called BMN 355 designed to treat long-QT syndrome types 2 and 3; and BMN 365, which was targeted at plakophilin-2 mutations and arrhythmogenic right ventricular dysplasia/cardiomyopathy. Instead, the company is focusing on three programs that “met the highest bar for advancement.” They include BMN 351, a next-gen oligonucleotide in a phase 1/2 trial for patients with Duchenne muscular dystrophy, as well as BMN 349, a phase 1-stage oral small molecule for alpha-1 antitrypsin deficiency-associated liver disease. The final program to have been spared the ax is BMN 333, a preclinical C-type natriuretic peptide being developed for multiple growth disorders. The job cuts represent around 5% of the company’s workforce and are related to this “portfolio assessment,” a BioMarin spokesperson told Fierce Biotech in an emailed statement yesterday. The layoffs, which the company’s management agreed on last week, are likely to incur pre-tax charges of around $15 to $20 million during the second quarter as a result of severance and other employee termination benefits, BioMarin explained in a SEC filing (PDF). The majority of affected employees were notified yesterday, with the layoffs likely to be completed by the end of July, according to the filing. “We are grateful to the impacted employees for their dedication to our mission of developing transformative medicines for patients,” the spokesperson told Fierce Biotech. “BioMarin is committed to providing support through this transition for those employees impacted.” While BioMarin didn’t explicitly refer to any layoffs when the strategic rethink was outlined during first-quarter earnings in April, the company did say at the time that the pipeline restructure would save up to $60 million in R&D costs this year, offset by up to $20 million that will be spent on accelerating the prioritized programs.