Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside

In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc RARE.

The analyst keeps an Overweight rating on Ultragenyx with a price target of $102 and says the company is uniquely positioned in the long term with an expansive and diverse pipeline of orphan disease assets and highly regarded management. JP Morgan sees multiple value-creating catalysts over the next six to 18 months.

• Ultragenyx’s recent meeting focused on setrusumab and the ongoing Phase 3 ORBIT trial. The analyst noted that the company remains confident in the trial’s success, with a second interim analysis expected by mid-2025 and final results by the end of 2025.
• At current share levels, the analyst noted the market is not fully valuing the setrusumab program and anticipates positive trial data could push Ultragenyx stock price to a ~$7-handle.
• Ultragenyx emphasized that moving forward to the second interim analysis does not change the study’s likelihood of success. Instead, the additional time allows for clearer differentiation between the setrusumab and placebo groups, with the minimum follow-up increasing from 7 months in the first interim analysis to 12 months in the second.
• On the commercial front, Ultragenyx plans to leverage its existing Crysvita infrastructure in the U.S. for setrusumab, given overlapping call points.
• Approval could also make the company eligible for a Priority Review Voucher, providing a potential source of non-dilutive capital. In Europe, where Mereo owns the rights, Ultragenyx will lead the regulatory filing process and anticipates submitting around 2-3 months after the U.S. filing.

On Sunday, Ultragenyx reported preliminary 2024 revenue of $555 million—$560 million, compared to the consensus of $543.02 million.

Crysvita’s revenue for 2024 is estimated to be $405 million—$410 million, and Dojolvi’s revenue is estimated to be $87 million—$89 million.

The company expects 2025 sales of $640 million—$670 million versus a consensus of $648.54 million.

Price Action: RARE stock is up 4.31% at $41.75 at last check Wednesday.

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