Sparked by new 100-week data and discussions with the FDA, the company’s bid for approval in the United States will begin in Q1 2025.
Nanoscope Therapeutics (Dallas, TX, United States) announced today that it will submit its Biologics License Application (BLA) to the Federal Drug Administration (FDA) in Q1 2025 for its retinitis pigmentosa (RP) gene therapy candidate MCO-010.
The announcement comes after meetings with the FDA about the firm’s clinical program for MCO-010 and Nanoscope’s proposed roadmap for BLA submission. These next steps, according to the company, include a rolling BLA submission process based on the drug’s FDA Fast Track Designation.
MCO-010 is a gene therapy comprised of a light-activated opsin with bipolar cell targeting via mGluR6 promoter-enhancer. The gene therapy is delivered by the company’s proprietary AAV2 vector.
According to Nanoscope, its monotherapy does not involve targeting specific mutations in the outer retinal cells, making it potentially mutation agnostic.
Potential approval by the FDA would see MCO-010 join Luxturna (F. Hoffmann-La Roche AG; Basel, Switzerland) as the only FDA-approved drugs available for RP. Luxturna is currently only approved for confirmed biallelic RPE65 mutation-associated retinal dystrophy with viable retinal cells, and reportedly carries a bilateral treatment price tag of $850,000.
“Feedback from the FDA has informed Nanoscope on its BLA submission plan, thereby presenting the potential for a viable restorative option for patients whose vision has been lost to the array of progressive retinal degenerations that comprise RP,” said Dr. Allen Ho, director of retina research and co-director of the retina service at Wills Eye Hospital (Philadelphia, USA), and chief medical advisor of Nanoscope Therapeutics.
“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,” said Sulagna Bhattacharya, Nanoscope co-founder and chief executive officer.
“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.”
New trial data, the catalyst
The meetings with the FDA come on the heels of new 100-week data from the Phase 2b/3 RESTORE trial extension, presented by Dr. Michael Singer (USA) at the 2024 Annual Meeting of the American Society of Retinal Specialists in Sweden.
According to Dr. Singer, 40% of patients made a three-line gain in best-corrected visual acuity at 52 weeks. At 100 weeks, there was still a statistically significant improvement in visual acuity, with 30% of patients maintaining these three-line gains.
The drug’s safety profile was also tolerable, with only one patient on once-a-day topical steroids to control inflammation after 100 weeks.
“Preservation of baseline visual acuity over several years represents an important treatment effect that deviates from the expected natural history of RP.” said Dr. Ho on the potential impact of the drug on the RP treatment paradigm.
The RESTORE trial is now being extended into the REMAIN study, which will continue to track patients through five years.
Nanoscope Therapeutics is also investigating the potential applications of MC-010 in Stargardt disease, another sight-stealing affliction, via its Phase 2 STARLIGHT trial, which has already seen results published through 48 weeks.1
Reference
- Mahajan VB. Longitudinal BCVA Analysis of Patients With Stargardt Disease and Macular Degeneration Treated With MCO-010, a Mutation-Agnostic Optogenetic Therapy: 48-Week Results From a Phase 2a Clinical Trial (STARLIGHT). Invest. Ophthalmol. Vis. Sci. 2024;65(7):5266.
