Breakthrough Therapy Designation granted by FDA for Alkeus Pharmaceuticals’ ALK-001 Stargardt disease treatment

Alkeus Pharmaceuticals, Inc, announced today that the FDA has granted Breakthrough Therapy Designation (BTS) for ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt disease.

A chemically-modified form of Vitamin A created to treat multiple retinal regenerative diseases, ALK-001 is a once-a-day oral drug.

Results from clinical data show that ALK-001 safely slows the progression of Stargardt while simultaneously preserving the normal visual cycle. It is also the only drug to receive BTS for the disease, according to a company news release.

The latest news follows the FDA’s reviewal of data from Alkeus Pharmaceuticals’ phase 2, double-masked, randomized, placebo-controlled trial in Stargardt disease.

“The results from our Phase 2 trial provide a strong basis for regulatory filing and approval of ALK-001 for the treatment of Stargardt disease,” said Leonide Saad, PhD, CEO of Alkeus Pharmaceuticals, in the release. “We look forward to working with the FDA and other regulatory agencies so that we can bring ALK-001 to patients as quickly as possible.”

Ongoing clinical trials for ALK-001 to treat patients with dry age-related macular degeneration (AMD) are also underway. The company expects to report data from both trials—as well as other ongoing studies — later this year.