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BioMarin (BMRN) Options Positioning Ahead of Key Phase 3 Analysis

by | Jan 6, 2022

BioMarin (BMRN) expecting key data in early 2022 with two-year analysis of their Phase 3 trial for valoctocogene roxaparvovec (BMN 270) in hemophilia A. A positive read will help drive a re-filing of their BLA in the US and a CHMP on the EU filing sometime in Q2. The company would then be on pace for a US approval by Q4. The readout also comes at a time when BMRN is in the early stages of launch for Voxzogo and significant inflection towards their long-term goal of $8B to $10B in revenue. The Phase 3 analysis expected soon looks at a 134-participant study into the gene therapy and its safety/efficacy in hemophilia A, a hereditary bleeding disorder caused by a lack of blood clotting factor VIII. The condition can lead to chronic joint pain while bleeds in head can lead to longer-term problems like seizures and potentially death. The current standard of care is treating the missing clotting factor with concentrates through injections. BMN 270 is an AAV5 gene therapy that contains the gene for factor VIII. Early studies showed the expression of therapeutic amounts of the factor to stop bleeding and they announced positive Phase 3 data in early 2021. Factor replacement drugs or treatments have not been popular in treating hemophilia A with Roche’s standard Hemlibra quickly becoming the top treatment in the space but many physicians say they would switch – a Citi study in 2020 noted that 45% of surveyed physicians would switch to factor replacement treatments in the future. Piper bullish on 1/4 noting that while investor focus remains singularly on the upcoming Valrox two-year Phase 3 data, its notable that management took a number of U.S. price increases across the commercial portfolio that seem a bit more aggressive than years past and these price increases could provide for some moderate upside as 2022 BioMarin’s progresses. BMRN will be at the Goldman CEOS unscripted conference today and the JP Morgan Healthcare conference next week.

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