BioMarin Pharmaceutical Inc. (BMRN) PT Lowered to $59 at H.C. Wainwright

H.C. Wainwright analyst Mitchell Kapoor lowered the price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) to $59.00 (from $60.00) while maintaining a Neutral rating.

The analyst commented: "BioMarin agreed to acquire Alesta for $275M upfront + up to $215M in development and regulatory milestones, with all non-ALE1 assets spun out before close and no Alesta employees transferring. ALE1-101 is an adaptive Phase 1/2a study in healthy adults and adults with hypophosphatasia (HPP) focused on safety, tolerability, PK/PD and dose selection; under the agreement, ALE1-101 does not qualify as a “Phase 2 Trial” for milestone purposes. BioMarin is paying $275M upfront for ALE1 before a dedicated Phase 2 efficacy dataset, with the transaction therefore leaning heavily on the size and validation of the HPP market. AstraZeneca’s (AZN; not rated) Strensiq generated ~$1.7B globally in 2025, demonstrating that HPP can support blockbuster revenue. We think that commercial precedent helps explain the price BioMarin is willing to pay, but ALE1 still needs patient efficacy data to establish whether an oral PPi-lowering approach can capture a meaningful share of that market. We assign no ALE1 economics because current human testing remains focused on safety, PK/PD and dose selection. Recall, BioMarin also paid ~$270M for Inozyme in July 2025 and then BMN 401 was discontinued in August 2026 after Phase 3 ENERGY increased PPi but missed its RGI-C clinical co-primary endpoint. Therefore, we worry about the quality of BioMarin's development-stage BD transactions. The $275M upfront equals ~31% of BioMarin’s $874M 2Q26 cash balance. After adding the $275M as an IPR&D charge, we are lowering our 12-month PT to $59 from $60. We reiterate Neutral."

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