Ultragenyx Is Uniquely Positioned In Long Term, Remains On JPMorgan's Focus List

JPMorgan on Thursday shared updated thoughts on Ultragenyx Pharmaceutical Inc’s (NASDAQ:RARE) setrusumab program in osteogenesis imperfecta (OI).

In a fourth-quarter 2024 press release, the company said patients continue dosing in the Phase 3 Orbit and Cosmic trials, which evaluate setrusumab in pediatric and young adult patients with OI.

The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing toward the second interim analysis in mid-2025 or a final analysis in the fourth quarter of 2025.

Also Read: Ultragenyx’s Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside

Patients in the Cosmic study continue to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim and final analyses.

JPMorgan analyst Anupam Rama said Thursday, “Ultragenyx remains on the J.P. Morgan US Equity Analyst Focus List.”

Rama maintains the Overweight rating and writes, “We believe Ultragenyx is uniquely positioned in the long term with a broad and diverse pipeline of orphan disease assets and a highly regarded management team with a strong track record in the space.”

The analyst believes the second interim analysis (IA2) of ORBIT has a much better chance of achieving statistical significance for the primary endpoint, the annualized fracture rate (AFR), compared to the first interim analysis (IA1).

If IA2 meets statistical significance, the analyst expects Ultragenyx stock to rise to around the mid-$60s to low-$70s per share. If it falls short, the stock could drop to the mid-$20s to low-$30s, making the potential reward outweigh the risk.

JPMorgan increased the price target from $104 to $117 based on the updated setrusumab revenue build, UX111 in MPS3A addition to the model, and expense adjustments.

In February, the U.S. Food and Drug Administration accepted for review the Biologics License Application seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A, with a Prescription Drug User Fee Act action date of Aug. 18.

Price Action: RARE stock is up 2.6% at $38.98 at last check Thursday.

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