Wells Fargo reduced its price target for Ultragenyx Pharma to $18 from $50, citing the failure of the Phase 3 ASPIRE study for its experimental therapy GTX-102 in Angelman syndrome. The downgrade represents a 64% reduction in the prior target and places the new estimate 32% below Ultragenyx’s closing share price of $26.53 on September 1.
Analyst Ben Burnett at Wells Fargo removed GTX-102 from the firm’s financial model following the trial’s outcome, which failed to achieve its primary endpoint—the Bayley-4 cognitive gross score—and its main secondary endpoint, the MDRI. The company reported no positive trends in evaluated subscales, and Wells Fargo adjusted its operating expense assumptions accordingly.
The setback also raised concerns about the prospects for Ultragenyx’s AURORA study, which targets patients with specific mutations. Wells Fargo described the AURORA program as riskier in light of the ASPIRE results. Prior to the announcement, Burnett assigned a consensus-implied probability of success for GTX-102 of below 50%.
Other firms followed with similar downgrades. JPMorgan reduced its price target to $36 from $80, while Baird cut its target to $16 from $40 and Evercore ISI lowered its target to $16 from $34. All three firms also adjusted their ratings to neutral or in line, reflecting heightened uncertainty around the program’s viability.
Ultragenyx’s financial position remains under pressure, with negative free cash flow of $489 million and an EBITDA loss of $465 million over the trailing twelve months. Shares closed at $26.53 on September 1, up from $25.81 the prior session.
