Leerink Partners Reiterates Outperform Rating on Ultragenyx Pharma (RARE)

Leerink Partners analyst Joseph Schwartz reiterated an Outperform rating and $85.00 price target on Ultragenyx Pharma (NASDAQ: RARE).

The analyst comments "● Bottom Line: Ultragenyx (RARE, OP) reported sales that were modestly above expectations for the third quarter, while investors eagerly await the potential interim analysis for setrusumab by year-end or early 2025. The company reported 3Q24 total revenue of $139.5M, versus our/consensus estimate of $135.5M/$135.2M; FY24 revenue guidance (which was raised last quarter) of $530-550M remains in place. Regarding the pipeline, investor focus remains primarily focused on setrusumab in osteogenesis imperfecta/OI, with a potential interim assessment from the Ph.3 portion of ORBIT by YE24/early 2025. Meanwhile, RARE continues to execute on the remainder of the pipeline, with several other milestones expected in the near term. These include (1) results from the Ph.3 COSMIC study with setrusumab in pediatric patients (2 to <7 years old) versus bisphosphonates, (2) longer-term data for GTX-102 (Angelman syndrome/AS) and starting patient recruitment in the pivotal program, (3) a BLA filing for UX111 (MPS IIIA) and DTX401 (glycogen storage disease type Ia/ GSDIa), and (4) the completion of enrollment in the Ph.3 Enh3ance study with DTX301 (ornithine transcarbamylase/ OTC deficiency). RARE ended the quarter with cash, cash equivalents, and marketable debt securities of $825M. Reiterate Outperform and $85 PT."

For an analyst ratings summary and ratings history on Ultragenyx Pharma click here. For more ratings news on Ultragenyx Pharma click here.

Shares of Ultragenyx Pharma closed at $52.18 yesterday.

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