MOGENRY earns Japan priority review for inherited retinal dystrophies

Japan's Pharmaceuticals and Medical Devices Agency (PMDA) has accepted the New Drug Application (NDA) for sonpiretigene isteparvovec (MOGENRY; Nanoscope Therapeutics), formerly MCO-010, for inherited retinal dystrophies (IRDs) and granted it priority review.¹ The Japanese filing seeks a disease-agnostic IRD indication, broader than the US Biologics License Application (BLA) for retinitis pigmentosa (RP) with severe vision loss now under FDA review.¹ Nanoscope announced the acceptance October 1 and expects potential decisions in both markets in the first half of 2027.¹

Priority review stems from the agent's Sakigake designation, Japan's expedited pathway for innovative medical products, and builds on orphan designation for IRDs in Japan. The filing follows FDA acceptance of the US BLA in September 2026. If approved, the agent would become the first disease-agnostic therapy in Japan to improve vision in patients with IRDs, according to the company.¹

What clinical data support the MOGENRY NDA in Japan?

The NDA draws on the phase 1/2a trial in RP (NCT04919473), the phase 2b/3 RESTORE trial in RP (NCT04945772), and the phase 2 STARLIGHT trial in Stargardt disease (NCT05417126). RESTORE, a multicenter, randomized, double-masked, sham-controlled trial, randomized patients with advanced RP to high-dose, low-dose, and sham arms of nine patients each.^1,3

At the week 52 primary end point, the high-dose arm gained a mean 0.337 logMAR vs sham (P = .021), and the low-dose arm gained 0.382 logMAR (P = .029).² At week 76, the key secondary end point, the high-dose arm reached a 0.539-logMAR mean gain (P = .001), while the low-dose gain of 0.374 logMAR did not reach significance (P = .065).²

Longitudinal analysis showed significant separation from sham at visits from weeks 36 through 88.² On a responder basis, 39% (7 of 18) of treated patients gained at least 0.3 logMAR at week 52, rising to 56% (10 of 18) at week 76.²

STARLIGHT, an open-label phase 2 study, treated six participants with Stargardt disease and severe vision loss, with no serious adverse events reported over 48 weeks. At week 48, mean best-corrected visual acuity (BCVA) improved 5.5 ETDRS letters without a wearable low-vision aid and 9.0 letters with one. Participants with atrophy confined to the macula gained 12.0 and 32.0 letters, respectively.⁴

Long-term durability and ocular safety of sonpiretigene isteparvovec

Most patients dosed in RESTORE continued into the REMAIN extension study (NCT06162585), and long-term follow-up data form part of the Japanese submission.¹ At the Retina Society 59th Annual Scientific Meeting in Los Angeles, Christine Kay, MD, a vitreoretinal surgeon and IRD specialist, reported 40% of treated patients gained 0.3 logMAR and maintained the gain through 4 years. Sham-arm patients remained stable or declined, and Kay noted 10- and 20-year outcomes remain unavailable.³

No serious adverse events were reported in the treatment arms through 2 years of RESTORE follow-up. Anterior chamber cell, reported in 44% of treated patients vs 22% with sham, and ocular hypertension, reported in 39% vs 11%, were the most common adverse events. These events resolved or were controlled with topical medication, according to the company.²

In a September interview on the US filing, Benjamin Bakall, MD, PhD, who directs the inherited retinal disease clinic at Associated Retina Consultants in Phoenix, Arizona, identified broader use as a key open question. "The question is whether this treatment can also benefit other forms of inherited retinal disease, Stargardt disease, and age-related macular degeneration (AMD) with geographic atrophy (GA)," Bakall told Modern Retina.

In a the pilot episode of The OT Roundup, Veeral Sheth, MD, a retina specialist at University Retina, described the practical effect of a mutation-agnostic approach on candidate selection. "You don't necessarily have to do genetic testing on these patients to determine whether they're a good treatment candidate," Sheth told Ophthalmology Times. He added genetic testing still informs prognosis, family counseling, and eligibility for future gene-specific therapies.

Voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics), approved by the FDA in 2017, remains limited to RPE65-mediated disease.³ Nanoscope plans a phase 3 registrational trial of the agent in Stargardt disease in 2026 and expects to start a phase 2 program for its MCO platform in geographic atrophy, according to the company.¹

References
1. Nanoscope Therapeutics announces PMDA acceptance and priority review of New Drug Application for MOGENRY in Japan for the treatment of inherited retinal dystrophies. News release. Nanoscope Therapeutics, Inc; October 1, 2026. Accessed October 1, 2026. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-announces-pmda-acceptance-and-priority-review-of-new-drug-application-for-mogenry-in-japan-for-the-treatment-of-inherited-retinal-dystrophies-302895292.html
2. Ho AC. Efficacy and safety of MCO-010 optogenetic therapy for vision restoration in patients with severe vision loss due to RP: two-year topline results from a phase 2b/3 randomized, sham-controlled clinical trial (RESTORE). Presented at: American Academy of Ophthalmology 2024 Annual Meeting; October 20, 2024; Chicago, IL.
3. Filkins K, Kay C. Retina Society 2026: MCO-010 optogenetic therapy maintains visual acuity gains through 4 years in advanced RP. Modern Retina. September 24, 2026. Accessed October 1, 2026. https://www.ophthalmologytimes.com/view/retina-society-2026-mco-010-optogenetic-therapy-maintains-visual-acuity-gains-through-4-years-in-advanced-rp
4. Lam B, et al. Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial. eClinicalMedicine. 2025. https://www.sciencedirect.com/science/article/pii/S2589537025003621