Ultragenyx Stays On JPMorgan’s Focus Record As Analyst Highlights Robust Lengthy-Time period Positioning – Ultragenyx Pharmaceutical (NASDAQ:RARE)

JPMorgan on Thursday shared up to date ideas on Ultragenyx Pharmaceutical Inc’s RARE setrusumab program in osteogenesis imperfecta (OI).

In a fourth-quarter 2024 press launch, the corporate stated sufferers proceed dosing within the Part 3 Orbit and Cosmic trials, which consider setrusumab in pediatric and younger grownup sufferers with OI.

The randomized, placebo-controlled Part 3 portion of the Orbit examine is progressing towards the second interim evaluation in mid-2025 or a ultimate evaluation within the fourth quarter of 2025.

Additionally Learn: Ultragenyx’s A number of Business Belongings And Candidates Impress Analyst; Sees Strong Upside

Sufferers within the Cosmic examine proceed to be handled with both setrusumab or intravenous bisphosphonates (IV-BP) remedy and will probably be evaluated in parallel with the Orbit interim and ultimate analyses.

JPMorgan analyst Anupam Rama stated Thursday, “Ultragenyx stays on the J.P. Morgan US Fairness Analyst Focus Record.”

Rama maintains the Obese ranking and writes, “We imagine Ultragenyx is uniquely positioned in the long run with a broad and numerous pipeline of orphan illness property and a extremely regarded administration workforce with a robust observe document within the area.”

The analyst believes the second interim evaluation (IA2) of ORBIT has a a lot better likelihood of attaining statistical significance for the first endpoint, the annualized fracture charge (AFR), in comparison with the primary interim evaluation (IA1).

If IA2 meets statistical significance, the analyst expects Ultragenyx inventory to rise to across the mid-$60s to low-$70s per share. If it falls brief, the inventory may drop to the mid-$20s to low-$30s, making the potential reward outweigh the chance.

JPMorgan elevated the value goal from $104 to $117 based mostly on the up to date setrusumab income construct, UX111 in MPS3A addition to the mannequin, and expense changes.

In February, the U.S. Meals and Drug Administration accepted for overview the Biologics License Utility in search of accelerated approval for UX111 (ABO-102) AAV gene remedy as a remedy for sufferers with Sanfilippo syndrome kind A, with a Prescription Drug Consumer Price Act motion date of Aug. 18.

Value Motion: RARE inventory is up 2.6% at $38.98 at final verify Thursday.

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