New BioMarin CEO Prioritizes Three Programs, Axes Four Others

Rare Daily Staff

BioMarin Pharmaceutical said that after a strategic R&D asset review to maximize profitability, it will cut four programs from its pipeline to focus on three promising programs and accelerate commercial opportunities for its approved achondroplasia therapy Voxzogo.

“By advancing and focusing on these promising programs, we have taken decisive steps to drive value creation for all our stakeholders,” said Alexander Hardy, president and CEO of BioMarin, as part of the company’s reporting of financial results for the first quarter of 2024.

Hardy joined BioMarin in December 2023, replacing long-time Chairman and CEO Jean-Jacques Bienaime who retired.

The four programs to be discontinued include BMN 331, a phase 1/2 gene therapy for patients with hereditary angioedema; BMN 255, an oral therapy in phase 1b for metabolic dysfunction-associated steatohepatitis; BMN 355, a preclinical antibody treatment for long-QT syndrome types 2 and 3; and BMN 365, a preclinical asset targeting plakophilin-2 mutations and arrhythmogenic right ventricular dysplasia/cardiomyopathy. None of the programs were discontinued due to safety signals.

In addition to expanding indications for Voxzogo, BioMarin’s focus will concentrate on three pipeline programs: BMN 333, a preclinical biologic in development for multiple growth-disorders; BMN 349, a first-in-class, oral therapeutic for AATD-associated liver disease; and BMN 351, a next-generation oligonucleotide in phase 2 development for Duchenne muscular dystrophy.

BioMarin said the planned reduction in operating expenses for the discontinued early-stage R&D programs, are in the range of $50 million and $60 million, which will be partially off-set by planned increases in operating expenses for the accelerated programs, resulting in planned net reductions to 2024 operating expenses of $35 million to $40 million.