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Investing.com - Wolfe Research has reiterated an Outperform rating for BioMarin Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ:BMRN) with a price target of $95.00, citing the company’s strong position in the phenylketonuria (PKU) treatment market. InvestingPro data shows the company is currently undervalued, with a strong financial health score of "GREAT" and an impressive perfect Piotroski Score of 9, indicating robust operational efficiency.
The research firm believes BioMarin’s established PKU treatments will maintain their market-leading positions despite upcoming competition from PTC Therapeutics (NASDAQ:PTCT)’ Sephience, which has a PDUFA date of July 29. Sephience, a BH4 precursor mechanistically similar to BioMarin’s Kuvan, received European Medicines Agency approval last month. The company’s strong market position is supported by impressive financials, including an 81% gross profit margin and 19.4% revenue growth in the last twelve months.
Wolfe Research questions Sephience’s efficacy claims for classical PKU patients who completely lack PAH enzyme function, noting that the clinical trial definition of classical PKU was "nonstandard." The firm also points out that PTC (NASDAQ:PTC)’s intention to price Sephience at a premium to BioMarin’s Palynziq may limit market adoption.
BioMarin’s PKU portfolio includes Kuvan, which faces generic competition in the US since 2020, and Palynziq, an enzyme replacement therapy primarily used for classical PKU patients. According to a medical geneticist consulted by Wolfe Research, patients who respond to Kuvan or its generics typically remain on these therapies for life due to favorable pricing and tolerability.
Wolfe Research projects Palynziq sales of $648 million, compared to consensus estimates of $669 million, and maintains its bullish stance on BioMarin shares despite the potential for Sephience to capture "a sliver of the PKU market." InvestingPro subscribers have access to 10 additional key insights about BioMarin, including detailed analysis of its competitive position and growth prospects. Get the full picture with the comprehensive Pro Research Report, available exclusively on InvestingPro.
In other recent news, BioMarin Pharmaceutical Inc. has completed its $270 million acquisition of Inozyme Pharma, adding the late-stage enzyme replacement therapy INZ-701 to its portfolio. This acquisition aims to enhance BioMarin’s offerings for treating ENPP1 Deficiency, a rare genetic condition. Additionally, BioMarin’s hemophilia A gene therapy, ROCTAVIAN, demonstrated sustained efficacy and safety five years post-treatment, with 81.3% of participants remaining off prophylactic treatment. The therapy received FDA approval in June 2023, further solidifying BioMarin’s position in gene therapy advancements.
Morgan Stanley (NYSE:MS) initiated coverage on BioMarin with an Overweight rating, citing the strong progress of Voxzogo and the company’s diversified pipeline as key growth drivers. Jefferies maintained a Buy rating, acknowledging BioMarin’s regulatory challenges concerning Voxzogo’s market exclusivity against Ascendis Pharma (NASDAQ:ASND). Meanwhile, Stifel also upheld a Buy rating, emphasizing BioMarin’s growth potential and strategic moves, including the recent acquisition and plans for further business development. These developments reflect BioMarin’s ongoing efforts to strengthen its market position and expand its therapeutic offerings.
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