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Optimistic Outlook for Ultragenyx Pharmaceutical Amid Promising Gene Therapy Developments

Optimistic Outlook for Ultragenyx Pharmaceutical Amid Promising Gene Therapy Developments

H.C. Wainwright analyst Ed Arce has maintained their bullish stance on RARE stock, giving a Buy rating today.

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Ed Arce has given his Buy rating due to a combination of factors related to Ultragenyx Pharmaceutical’s promising prospects. The acceptance of the BLA for UX111, a gene therapy aimed at treating Sanfilippo syndrome type A, by the FDA for Priority Review marks a significant milestone. This development is expected to boost the company’s trajectory towards achieving GAAP profitability by 2027, especially considering the absence of any planned advisory committee meeting for the application.
Additionally, the clinical data from the pivotal Transpher A study supports the efficacy of UX111, showing substantial reduction in cerebrospinal fluid heparan sulfate levels and significant cognitive improvements in patients. The valuation model used by Arce estimates a favorable price target based on the potential market performance of existing products and assets under development. These factors, combined with a risk-adjusted NPV valuation and an 11% discount rate, underscore a positive outlook for Ultragenyx’s stock, despite potential risks such as regulatory hurdles and market challenges.

Arce covers the Healthcare sector, focusing on stocks such as Arcturus Therapeutics, Ultragenyx Pharmaceutical, and Acurx Pharmaceuticals. According to TipRanks, Arce has an average return of 18.6% and a 48.50% success rate on recommended stocks.

In another report released today, Barclays also maintained a Buy rating on the stock with a $81.00 price target.

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