STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial — SkimNews
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Ultragenyx said Wednesday that GTX-102, its experimental Angelman syndrome therapy, showed no benefit compared to a sham treatment in a large Phase 3 trial.
- GTX-102 had previously shown powerful results in early trials, raising hopes among families affected by Angelman syndrome, a rare disease causing severe intellectual disabilities and developmental delays.
- Advocates for patients with other neurological conditions had hoped the drug could become the first of many medicines to improve cognition, communication, and other aspects of life for patients with intellectual disabilities.
- Investors had been banking on the Angelman drug as Ultragenyx's path to profitability, making the failure a significant blow to the company's business.
- Ultragenyx already has multiple approved medicines on the market, but they address mostly ultra-rare diseases.
Why it matters: The Phase 3 failure strips Ultragenyx of the growth asset investors were pricing into its path to profitability, while families affected by Angelman syndrome lose what early data had suggested was a promising therapeutic candidate. Beyond the company, GTX-102 had been watched as a potential proof-of-concept for cognition-improving medicines in broader intellectual-disability populations.
Ask SkimNews
