Truist Securities Reiterates Buy Rating on Ultragenyx Pharma (RARE)

Truist Securities analyst Joon Lee reiterated a Buy rating and $90.00 price target on Ultragenyx Pharma (NASDAQ: RARE)

The analyst comments "This AM, RARE disclosed that neither ORBIT nor COSMIC met the primary endpoint of fracture reduction, despite expected increases in BMD. We have been skeptical of setrusumab in OI, despite Ph2 results and KOLs' optimistic views as expressed in our prior note (HERE): • "We are admittedly a bit more skeptical on this program as the bone formed in OI patients are inherently defective. Therefore, we are not sure if forming more of the defective bone could actually result in fracture reduction" PR implies some numeric benefits with setrusumab and further analyses are underway. However, significant expense reductions are planned implying sunsetting of the OI program. The update puts the pipeline focus squarely back on Apazunersen (formerly GTX-102) in Angelman due to report topline from Ph3 Aspire in 2H26. Recall, the trial completed enrollment in July 2025, ~7 months ahead of schedule. Our diligence of the program, since its start in 2020, suggests Apazunersen has a much better probability of success in Angelman than setrusumab had in OI. Recently announced 2nd royalty deal with OMERS (private) for $400M upfront in exchange for an addt'l 25% royalty on sales of Crysvital in US/CAN should fund operations well-beyond Angelman topline readout in 2H26 and possibly into profitability (our prior note HERE). Our model assumptions are currently under review."

For an analyst ratings summary and ratings history on Ultragenyx Pharma click here. For more ratings news on Ultragenyx Pharma click here.

Shares of Ultragenyx Pharma closed at $34.19 yesterday.

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