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Ultragenyx Pharmaceuticals: Buy Rating Supported by Promising Gene Therapy Trial Results and Regulatory Advancements

Ultragenyx Pharmaceuticals: Buy Rating Supported by Promising Gene Therapy Trial Results and Regulatory Advancements

Leerink Partners analyst Joseph Schwartz has reiterated their bullish stance on RARE stock, giving a Buy rating yesterday.

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Joseph Schwartz has given his Buy rating due to a combination of factors, including promising trial data and regulatory advancements with Ultragenyx Pharmaceutical’s gene therapy UX111. The data shows significant improvements in clinical function for patients with Sanfilippo Syndrome Type A, correlating with reductions in a key biomarker, heparan sulfate (HS), in cerebrospinal fluid. These results are supported by statistically significant findings from the Transpher A study and its long-term follow-up, with notable gains in cognitive and communication skills, as well as motor functions, compared to natural history data.
Furthermore, the company’s recent agreement with the FDA, recognizing CSF-HS reduction as a valid surrogate endpoint, supports the submission of a Biologics License Application for Accelerated Approval. This regulatory progress, combined with the positive clinical outcomes, reinforces the potential of UX111 and underpins Schwartz’s optimistic outlook on Ultragenyx’s stock, justifying the Buy rating.

In another report released yesterday, Morgan Stanley also maintained a Buy rating on the stock with a $64.00 price target.

Based on the recent corporate insider activity of 48 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of RARE in relation to earlier this year.

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