BMRN: BMRN-401 Pediatric Study Looks Like a Failure

Research evidence excerpt

BMRN: BMRN-401 Pediatric Study Looks Like a Failure

Biotechnology Equity Research

Investment Thesis, Valuation and Risks

BioMarin Pharmaceutical, Inc. (BMRN)

Investment Thesis

We rate BMRN Overweight, driven by its strong, de-risked, and resilient enzyme replacement therapy (ERT) business, which should be

highly complementary to its new product launches and ultimately lead to margin expansion. We expect Voxzogo to benefit from its first-

mover advantage in achondroplasia, which should translate to a leading position in hypochondroplasia as well. We also expect BMRN to

generate margin expansion looking ahead.

Target Price Valuation for BMRN

We base our 12-month forward price target of $75 on our DCF analysis, where we use a discount rate of 8.5%. This reflects a lower cost

of capital relative to our SMid biotech coverage as a result of the relatively derisked ERT business. For BMRN, we assume a 0% terminal

growth rate, as we think this reflects the mature ERT business and that BMN333 is still in early-stage.

Risks to Our Price Target and Rating for BMRN

Downside Risks:

• Commercial headwinds that affect the growth and durability of ERT revenues, which requires additional costs to mitigate, affecting

margins as well.

• Indication expansion issues with Voxzogo and Roctavian as well as competition from other approved, or soon-to-be approved,

therapies. Competition and indication expansion issues can reduce growth potential, affecting sales.

• Discontinuation or timeline delays from the early-stage pipeline assets, questioning long-term development.

Companies Mentioned in Report

Company Name Ticker Last Price

(05/18/26)

BioMarin Pharmaceutical, Inc. BMRN $51.18

Source: Wells Fargo Securities LLC Estimates, FactSet

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