Wolfe Research Reiterates Outperform Rating on BioMarin Pharmaceutical Inc. (BMRN)

Wolfe Research analyst Akash Tewari reiterated an Outperform rating and $95.00 price target on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN).

The analyst comments "Kuvan, generics, and Palynziq are the only FDA-approved therapies for PKU. Recall that BMRN's Kuvan was the first therapy approved for PKU, a rare genetic disease denoted by the accumulation of Phe. Specifically, Kuvan is a synthetic BH4, which serves as a cofactor for the PAH enzyme, aiding in the breakdown of Phe. Given its MoA, Kuvan and generics (entered US market in 2020) result in clinically meaningful reduction in Phe (≥30%) in PKU patients who retain some PAH function. Indeed, a medical geneticist who we consulted suggested that in their practice, all PKU patients are offered Kuvan or generics, and if a patient responds, they stay on therapy for life given Kuvan/generics' favorable pricing and tolerability profile. In contrast, classical PKU patients who completely lack PAH function don't respond to these therapies. Therefore, some of these classical PKU patients get on BMRN's ERT Palynziq, but treatment is burdensome. PTC's Sephience is expected to earn FDA approval this month. Sephience is a BH4 precursor that is mechansitically similar to Kuvan. Sephience received EMA approval last month and is expected to receive FDA approval (7/29 PDUFA) on the basis of data from the ph 3 APHENITY trial. In this trial, Sephience meaningfully reduced Phe across a broad range of PKU patients. Despite Sephience's touted utility in classical PKU, we believe that the way classical PKU was defined in the study was nonstandard. Moreover, from a MoA perspective, we question how Sephience could work in classical PKU patients who completely lack PAH function. Finally, PTC has messaged that it intends on pricing Sephience at a premium to Palynziq, which may hinder adoption. We remain buyers of BMRN. In our view, while Sephience may be able to capture a sliver of the PKU market, we continue to believe that Kuvan or generics more generally and Palynziq (WR $648M; cons $669M) in classical PKU will maintain their marketleading positions. See pages 3-10 for our detailed thoughts."

For an analyst ratings summary and ratings history on BioMarin Pharmaceutical Inc. click here. For more ratings news on BioMarin Pharmaceutical Inc. click here.

Shares of BioMarin Pharmaceutical Inc. closed at $58.23 yesterday.

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