Buy Rating on Ultragenyx Pharmaceutical Bolstered by GTX-102’s Promise and Positive Valuation Outlook

Joseph Schwartz, an analyst from Leerink Partners, reiterated the Buy rating on Ultragenyx Pharmaceutical (RARE – Research Report). The associated price target remains the same with $98.00.

Joseph Schwartz has given his Buy rating due to a combination of factors related to Ultragenyx Pharmaceutical’s recent developments and future prospects. His positive outlook is influenced by the promising efficacy data of GTX-102 in treating Angelman syndrome, which met expectations with higher loading doses resulting in consistent or superior improvements across several endpoints. Furthermore, the continued progress observed up to day 758 reinforces the drug’s potential. Although the safety profile revealed additional serious adverse events (SAEs), the management’s response and the less severe nature of these events compared to previous ones suggest these can be managed effectively. Schwartz’s confidence is bolstered by the anticipation of a pivotal FDA meeting, which could lead to the initiation of a pivotal study by the end of 2024.
The valuation of Ultragenyx Pharmaceutical also plays a role in the Buy rating, with Schwartz estimating the GTX-102 program to potentially reach gross peak sales of approximately $1.2 billion in the US and EU markets, attributing a 40% probability of success. The upcoming end-of-Phase 2 meeting with the FDA is seen as a critical catalyst that could significantly impact the company’s shares. With these upcoming catalysts in mind, Schwartz maintains a favorable view of Ultragenyx Pharmaceutical’s shares, reiterating an Outperform rating with a price target of $98, suggesting confidence in the company’s growth trajectory and investment potential.

In another report released on April 16, Jefferies also maintained a Buy rating on the stock with a $129.00 price target.

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Ultragenyx Pharmaceutical (RARE) Company Description:

Ultragenyx Pharmaceutical, Inc. is a biopharmaceutical company, which engages in the identification, acquisition, development and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. Its product includes Mepsevii and Crysvita. Mepsevii is an intravenous, which is used for the treatment of Mucopolysaccharidosis VII. Crysvita is an antibody administered via subcutaneous injection used for the treatment of XLH. The company was founded by Emil D. Kakkis on April 22, 2010 and is headquartered in Novato, CA.