Ultragenyx Pharmaceuticals shares fell 44.7% after its experimental Angelman syndrome treatment failed to meet primary and secondary endpoints in Phase 3 testing.
Ultragenyx Pharmaceuticals shares fell 44.7% to $14.67 in premarket trading after the company disclosed that its pivotal Phase 3 Aspire study evaluating apazunersen (GTX-102) failed to meet its primary and key secondary endpoints. The study evaluated the drug's effectiveness in treating Angelman syndrome, a condition characterized by developmental delays, balance problems, and speech impairment.
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Investigators found no meaningful differences between the treatment and placebo groups on either the primary endpoint, which measured the change from baseline in the Bayley-4 cognitive raw score, and the key secondary endpoint, which assessed net response using the Multidomain Responder Index. Following the results, several analysts revised their ratings and price targets. Evercore ISI downgraded the stock from Outperform to In Line and reduced its price target to $16. Baird lowered its rating from Outperform to Neutral and cut its price target to $16. JPMorgan downgraded the stock from Overweight to Neutral and reducing its price target to $36. William Blair downgraded the shares to Market Perform and removed the contribution from GTX-102 from its pipeline valuation.
Sources
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'Fall From Grace' — Biotech Crashes 47% To A Record Low
Investor's Business Daily
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Ultragenyx Shares Fall After Phase 3 Aspire Study Misses Primary Endpoint
Yahoo Finance