Nanoscope website
Nanoscope Therapeutics Inc. on Thursday announced that its MCO-010 therapy to restore vision to those impacted by a group of rare eye diseases has received fast-track designation from the U.S. Food and Drug Administration after its most recent clinical trial demonstrated positive and “unprecedented” results.
The results were consistent with those observed in earlier trials, demonstrating improvements in functional vision in the majority of patients suffering from retinitis pigmentosa, according to a press release. In addition to the evidence of a meaningful effect, MCO-010 showed no serious or severe ocular or systemic adverse effects.
“I have had the privilege of observing substantial improvements in visual function in several patients who have enrolled in MCO-010 clinical trials over the past year,” said Victor H. Gonzalez, founder of Valley Retina Institute of McAllen. “The fact that we see sustained and sometimes transformative gains in vision function, allowing them to walk in the clinic with more certainty, after a single treatment is remarkable and unprecedented.”
There are currently no available treatments for advanced RP. The company anticipates presenting its results at upcoming medical conferences.
Nanoscope, founded in 2009 by Samarendra Mohanty and Sulagna Bhattacharya, is a clinical-stage biopharmaceutical company developing gene therapies to cure genetic diseases that cause vision impairment and blindness. In addition to retinitis pigmentosa, these diseases include dry age-related macular degeneration, and Stargardt disease. The company had previously received grants of $3.5 million from the National Institutes of Health. It had also sought to raise $16 million in equity funding.
Nanoscope Therapeutics is a TechFW member, a 501(c)(3) nonprofit startup accelerator and business incubator.
In the trial, 18 patients with severe vision impairment because of RP received a single intravitreal injection of MCO-010 while nine received a sham intravitreal injection procedure.
The primary outcome measure was mean change in vision-guided mobility score against the placebo. Other key efficacy assessments included near object recognition and the best possible vision with the use of glasses or contact lenses.
“We are thrilled to see such encouraging results,” said Sulagna Bhattacharya, company CEO. “These results suggest that MCO-010 provides substantial benefit to patients with severe vision loss due to advanced RP. We are looking forward to engaging with the FDA and other regulatory agencies on the future of MCO-010, with the goal of expeditiously getting this novel therapy to patients. We sincerely thank the trial participants and families as well as the investigators and all who contributed to this groundbreaking trial.
Said Mohanty, president and chief scientific officer: “These results … represent more than a decade of work by many dedicated individuals involved in the discovery and development of this unique fast, broadband, and highly sensitive platform.”