Ultragenyx: Refocusing on Apazunersen and Strengthened Balance Sheet Support Buy Rating Despite OI Setback

In a report released today, Joon Lee from Truist Financial maintained a Buy rating on Ultragenyx Pharmaceutical, with a price target of $90.00.

Joon Lee has given his Buy rating due to a combination of factors that, in his view, still support Ultragenyx’s long‑term value despite the recent trial setback. While the company reported that its ORBIT and COSMIC studies in osteogenesis imperfecta did not achieve the primary goal of reducing fractures, he had already viewed this program cautiously, given the biological challenges of improving bone quality in this patient population. The subsequent decision to significantly scale back spending on this indication realigns resources and management attention toward more promising assets, rather than continuing to fund a program with a questionable risk‑reward profile.

Lee’s positive stance instead centers on Apazunersen for Angelman syndrome, which he sees as the key value driver, underpinned by a Phase 3 trial that completed enrollment earlier than anticipated and, based on his prior due diligence, carries a higher likelihood of success. He also highlights the recent royalty financing with OMERS, which delivers $400M upfront in exchange for an additional royalty on Crysvita sales and should provide sufficient capital to fund the company well past the Angelman Phase 3 data readout and potentially to profitability. This combination of a derisked and strategically focused pipeline, a clearly defined late‑stage catalyst in 2H26, and an improved balance sheet underpins his decision to maintain a Buy rating, even as the model assumptions are being updated to reflect the OI outcome.

Based on the recent corporate insider activity of 42 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of RARE in relation to earlier this year.