Nanoscope Announces BLA Submission Plans for Retinitis Pigmentosa Gene Therapy MCO-010 Following Meeting With FDA
Nanoscope intends to go forward with the BLA submission in the first quarter of next year.
Nanoscope Therapeutics has stated that it plans to file a biologics license application(BLA) for MCO-010 (sonpiretigene isteparvovec), its investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy intended to treat retinitis pigmentosa (RP), following a meeting with the FDA that it characterized as “productive”.1
The company noted that it intends to pursue a rolling BLA submission for MCO-010 in severe vision loss due to RP, with reference to the fast track designation that the program previously has received from the FDA. Nanoscope intends to go forward with the BLA submission in the first quarter of next year. The company additionally noted that the FDA had provided regulatory feedback in the meeting and acknowledged the planned subsequent steps.
“We are pleased with the positive interactions we have had with FDA as a result of the exceptional expertise and tireless commitment of the Nanoscope team,” Sulagna Bhattacharya, B. Tech, MBA, the cofounder and CEO of Nanoscope, said in a statement.1 “Our shared goal is to change lives, and together, we have advanced MCO-010 to the point of BLA submission. With every step forward, we are focused on the patients who are waiting for meaningful sight restoration. Our team looks forward to continuing the important work we have begun, along with our partners, to bring this therapy to patients who have significant unmet need.”
MCO-010 is currently being evaluated in the phase 2b/3 RESTORE clinical trial (NCT04945772). Results from this study were presented earlier this year at at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.2 With regard to best-corrected visual acuity (BCVA), it was reported that the low dose cohort had mean improvements of 0.160 (P against sham = .2767), 0.191 (P = .1945), 0.431 (P = 0.0058), and 0.332 LogMAR (P = .0290) at weeks 16, 24, 36, and 52, respectively. The high dose cohort had mean improvements of 0.022 (P against sham = .8403), 0.197 (P = .0772), 0.214 (P = .0549), and 0.287 (P = .0104) at weeks 16, 24, 36, and 52, respectively.
“Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP,” Allen C. Ho, MD, FACS, FASRS, the director of retina research and codirector of the Retina Service at Wills Eye Hospital, and chief medical advisor of Nanoscope, added to the statement.1 “Feedback from the FDA has informed Nanoscope’s BLA submission plan, thereby presenting the potential for a viable, restorative option for patients whose vision has been lost due to the array of progressive retinal degenerations that comprise RP.”
MCO-010, which consists of an adeno-associated virus (AAV2) vector delivering multicharacteristic opsin and is administered via an intravitreal injection, was granted fast track designation by the FDA in October 2022 for the treatment of retinitis pigmentosa (RP) via intravitreal injection.3 Notably, in January 2023, MCO-010 was also granted fast track designation by the FDA for the treatment of Stargardt disease.4 Nanoscope is evaluating MCO-010 for the treatment of Stargardt disease in the phase 2 STARLIGHT clinical trial (NCT05417126).1 A pivotal phase 3 trial in Stargardt is planned for the first quarter of next year.
“This productive meeting with the FDA also follows our recent End of Phase 2 meeting for our Stargardt macular degeneration program, which is advancing to a phase 3 registrational trial,” Samarendra Mohanty, PhD, the president and chief scientific officer of Nanoscope, added to the statement.1 “The evidence of improvements in visual acuity over 3 years across the RESTORE and REMAIN studies reinforces the strength of our commitment to bring this transformative therapy to patients.”
