Marrow Peak falls on four-year data for its Huntington's gene therapy
Disease progression slowed by 41% compared with a matched external group, down from 60% at the three-year mark.
By Tomás IbarraPublished · 1 min read
Shares in Marrow Peak fell by a fifth on Saturday's disclosure that the benefit of its one-time gene therapy for Huntington's disease appeared to narrow in a fourth year of follow-up.
Patients given the high dose declined 41% more slowly than a matched group drawn from a natural history study. A year earlier the difference was 60%.
The company said the change reflected the small number of patients, twelve, who have reached the four-year visit, and that a biomarker of nerve cell damage remained below baseline. It still plans to seek accelerated approval early next year.
Investors had hoped for evidence that the effect was holding steady, said one analyst, since durability is the central argument for a treatment delivered by brain surgery.
