BioMarin announced on Aug. 10 that it will discontinue investment in the investigational enzyme replacement therapy BMN 401, following disappointing results from a late-stage clinical trial earlier this year.
BMN 401 was being evaluated in the Phase 3 ENERGY trial for ENPP1 deficiency, a rare metabolic disorder characterized by symptoms such as early-onset osteoporosis, tendon calcification, and increased risk of cardiovascular death. The candidate was acquired by BioMarin in May 2025 through its $270 million acquisition of Inozyme Pharma. However, after one year, the asset met only one of two key endpoints in the ENERGY study—significantly lowering a key disease marker without demonstrating clinical benefit for patients.
According to BioMarin’s second quarter earnings report released Thursday, the company will halt development of BMN 401 across all indications. Previously, Inozyme had been testing the molecule for ABCC6 deficiency and calcific uremic arteriopathy—both rare diseases—but BioMarin no longer lists any active programs for these conditions.
Despite discontinuing BMN 401, BioMarin reported positive financial results this quarter with revenues increasing by 20% year-over-year to reach $990 million. The company’s product portfolio was led by Voxzogo, an achondroplasia treatment that generated $253 million in sales. Voxzogo sales grew by 14% compared to the same period last year despite competition from Ascendis Pharma’s Yuviwel, which received FDA approval for achondroplasia this past March and is expected to present its earnings on Aug. 13.
Stifel analysts said Friday, “Voxzogo continues to offset any impact with growth in the <2 [years] population and in the ex-US population, which comprises the majority of revenues. The next couple quarters will be key here in determining the competitive impact to BioMarin.”
In anticipation of Yuviwel’s approval last October, BioMarin withdrew its previously stated goal of reaching $4 billion in revenue by 2027 due mainly to potential competition facing Voxzogo. On Thursday’s report, however, BioMarin raised its full-year sales guidance from between $975 million and $1.02 billion to at least $1 billion with potential up to $1.05 billion.
