HC Wainwright assumed coverage on Ultragenyx Pharmaceuticals, Inc. (NASDAQ:RARE) on Monday, representing an attractive opportunity at current levels.

Analyst Raghuram Selvaraju notes that Ultragenyx stock is down 35% year to date, compared to a 2.7% decline in the benchmark XBI index.

“Intriguing buying opportunity in one of biopharma’s most diversified rare disease-focused companies,” the analyst said.

HC Wainwright assumed coverage at a Buy rating with a price target of $80.

The analyst says Ultragenyx shares have primarily come under pressure due to investor disappointment in the interim readout from the pivotal Orbit trial of UX143 (setrusumab) for osteogenesis imperfecta (OI or brittle bone disease).

In July, Ultragenyx and Mereo BioPharma Group plc (NASDAQ:MREO) announced the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with OI is progressing toward a final analysis around the end of the year, consistent with the original plan.

The second interim analysis did not meet the minimal p-value threshold of p<0.01 to end the study early.

Also Read: Analyst Bullish On Ultragenyx, Poised For Growth With Six Drug Approvals Expected By 2028

Selvaraju says the Orbit trial has not failed, and the final results are expected to be positive because the risks have been minimized. This should support a U.S. regulatory filing for setrusumab next year.

The analyst also writes the company’s current commercial portfolio alone should support fair value in the roughly $35-$40 per share range without crediting the pipeline.

HC Wainwright highlights Ultragenyx’s flagship Crysvita franchise, which could achieve peak annual sales of over $3 billion.

In July, the U.S. Food and Drug Administration issued a Complete Response Letter for Ultragenyx’s Biologics License Application (BLA) for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA). 

In the CRL, the FDA requested that the company provide additional information and improvements related to specific aspects of CMC and observations from the recently completed manufacturing facility inspections.

“In our view, Ultragenyx should be able to resubmit the BLA later this year, with potential approval in mid-2026. We remind investors that the Sanfilippo syndrome indication, while a small target market, represents an area of high unmet need with no currently-approved medications,” HC Wainwright wrote in an investor note on Monday.

RARE Price Action: RARE stock is up 0.93% at $27.59 at publication on Monday.

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