Clinical-stage biotechnology firm Nanoscope Therapeutics has received Fast Track designation (FTD) from the US Food and Drug Administration (FDA) for its optogenetic monotherapy, MCO-010, to treat retinitis pigmentosa (RP).
MCO-010, the lead asset of the company, is an ambient-light activatable multi-characteristic opsin (MCO) monotherapy, which has been designed to restore vision in blind patients.
It has also received orphan drug designations from the US FDA for the treatment of RP and Stargardt.
Nanoscope Therapeutics CEO Sulagna Bhattacharya said: “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.
“We are proud to have the support of the FDA and look forward to collaboratively interacting with FDA to assess the next steps in the clinical development and future regulatory review of MCO-010.”
Currently, the therapy is in a randomised, multicentre, sham-controlled, double-masked Phase IIb clinical trial dubbed RESTORE for RP in the US.
The study has been designed to evaluate the safety and efficacy of a single intravitreal injection of virally carried MCO-010.
Nearly 27 RP patients with advanced vision loss were enrolled in the study in January this year, with top-line data anticipated in the first half of next year.
In September, Nanoscope also completed subject enrolment in the Phase II open-label STARLIGHT clinical trial of optogenetic therapy in patients with advanced vision loss caused due to Stargardt disease.
Six-month data from the study are anticipated in the first half of next year.