Chief Executive Emil Kakkis expressed disappointment, citing the discrepancy between the failed Aspire study and the promising data observed in earlier Phase 1/2 trials. The company is now reevaluating the entire apazunersen program and has signaled an immediate shift in corporate strategy to prioritize fiscal stability.
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Ultragenyx Shares Sink After Angelman Syndrome Drug Trial Fails
Shares of Ultragenyx Pharmaceutical plummeted 45% to $14.65 following news that its experimental drug apazunersen failed to reach primary and secondary endpoints in a Phase 3 trial. The treatment, intended for patients with the rare neuro-genetic disorder Angelman syndrome, failed to show the expected improvements in cognitive testing.
Management intends to implement significant expense reductions while pivoting focus toward the company's existing commercial business. Kakkis noted that Ultragenyx will prioritize expansion into new markets to reach profitability by next year, effectively sidelining development efforts that no longer align with its immediate financial goals.
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