FDA grants fast track designation to Nanoscope Therapeutics for MCO-010 for the treatment of retinitis pigmentosa

MCO-010, an ambient-light activatable multi-characteristic opsin optogenetic monotherapy for vision restoration in blind patients, received fast track designation from the FDA.

Nanoscope Therapeutics announced that the FDA has granted Fast Track Designation (FTD) to MCO-010, an ambient-light activatable multi-characteristic opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of retinitis pigmentosa (RP) via intravitreal injection.

"The FDA's decision to grant FTD underscores the importance of MCO-010 to address a serious unmet need and validates its potential as an effective therapeutic for patients with RP," Sulagna Bhattacharya, CEO of Nanoscope, said in a news release. "We are proud to have the support of the FDA and look forward to collaboratively interacting with FDA to assess next steps in the clinical development and future regulatory review of MCO-010."

According to the company, in January, 27 RP patients with advanced vision loss were enrolled in a multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010. Top line data from the clinical trial are expected in H1 2023.

In September of this year, enrollment was completed in the Phase 2 open-label STARLIGHT clinical trial (NCT05417126) of MCO-010 in patients with advanced vision loss due to Stargardt disease. Six-month data from the clinical trial are expected in H1 2023.

“We are excited by the therapeutic potential of MCO-010, supported by robust data from previous preclinical and clinical studies,” Bhattacharya said in a news release at the time. “Completing the quick two-month enrollment of this Phase 2 trial, which will evaluate the safety and effects of a single intravitreal injection of MCO-010, brings us another major step forward in developing this novel therapy with broad therapeutic application.”

Nanoscope’s optogenetic therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells to enable vision in different color environments. The therapy is administered as a single intravitreal injection for in-office delivery without the need for any other devices or interventions. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA and is concurrently being evaluated in Nanoscope’s Phase 2b RESTORE trial in patients with RP.

The FDA's Fast Track designation is intended to facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the Fast Track designation, Nanoscope Therapeutics is eligible for more frequent regulatory meetings and communications with the FDA.