Nanoscope Therapeutics Announces Positive Topline Results from Phase 2b RESTORE Trial of MCO-010 for the Treatment of Retinitis Pigmentosa

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Mar 30, 2023, 07:00 ET

88.9% (16/18) of MCO-010 treated patients experienced a clinically meaningful 2 or more luminance level improvement in vision-guided mobility or object recognition
Clinically meaningful visual acuity gains observed in several MCO-010 treated patients
Favorable safety profile for MCO-010 with no serious or severe adverse events

DALLAS, March 30, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc., today announced topline results from the Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation. MCO-010 has received both orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA).

In the RESTORE trial, 18 patients with severe vision impairment due to RP received a single intravitreal injection of MCO-010 while 9 received a sham intravitreal injection procedure. Results showed vision function improvements after treatment with MCO-010 consistent with previous studies as well as a favorable safety profile. The primary outcome measure was mean change in Multi-Luminance Y-Mobility Test (MLYMT, vision-guided mobility) score vs. placebo. Other key efficacy assessments included the Multi-Luminance Shape Discrimination Test (MLSDT, near object recognition) and Best-Corrected Visual Acuity (BCVA). For the MLYMT and MLSDT, a 2 or more luminance level change is considered clinically meaningful. For BCVA, a 0.3 LogMAR change is considered clinically meaningful, with negative change indicating improved visual acuity.

Key efficacy outcomes at 12-month were:

16 of 18 (88.9%) MCO-010 treated patients demonstrated a 2 or more luminance level improvement in MLYMT or MLSDT at 12 months compared to 4 of 9 (44.4%) receiving placebo (p <0.05);
12 of 18 MCO-010 treated patients improved by 2 or more luminance levels in the MLYMT compared to 3 of 9 receiving placebo;
10 of 18 MCO-010 treated patients improved by 2 or more luminance levels in the MLSDT compared to 2 of 9 receiving placebo;
7 of 18 MCO-010 treated patients improved by -0.3 LogMAR or more in BCVA compared to 1 of 9 receiving placebo; and
Change in MLYMT score (primary outcome), difference vs. placebo: +1.0 (95%CI 0.0, 3.0).

In addition to the evidence of a clinically meaningful effect, MCO-010 was well-tolerated with no serious or severe ocular or systemic adverse events reported. One SAE occurred in a placebo treated patient. There was a comparable incidence of treatment emergent adverse events (TEAEs) across study arms. The most common ocular TEAEs reported across treatment arms were anterior chamber cells, ocular hypertension, and conjunctival hemorrhage.

Today's RESTORE results are consistent with those observed in the earlier Phase 1/2 trial, demonstrating improvements in functional vision in the majority of patients treated with MCO-010 together with a favorable safety profile. People with severe vision loss due to RP currently have no available treatments to improve vision.

"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. 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," said Victor H. Gonzalez, MD, founder of Valley Retina Institute, McAllen, Texas and RESTORE investigator. "These participants who were living with severe vision impairment due to RP now have an improved quality of life, very different from before receiving MCO-010. In addition, MCO-010's favorable safety profile further strengthens my confidence in this ground-breaking treatment that I expect to become an important treatment option for people with advanced RP."

"These results are gratifying, as they demonstrate the potential of MCO-010 to restore vision and 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. This is a pivotal moment for the field of mutation-agnostic gene therapy and establishes optogenetics as a therapeutic modality that can restore functional vision in ambient light in patients with severe retinal degeneration. The RESTORE trial data further validate Nanoscope's MCO platform that is being applied across a growing pipeline of programs. Our approach demonstrates potential across a range of diseases and therapeutic areas and Nanoscope is uniquely poised to advance optogenetics to be a therapeutic reality for patients," added Samarendra Mohanty, Co-founder, President and Chief Scientific Officer of Nanoscope.

"We are thrilled to see such encouraging results from the RESTORE trial," said Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. "These results suggest that MCO-010 provides substantial benefit to patients with severe vision loss due to advanced RP, a condition for which there are currently no available treatments. 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. We would also like to thank our investors, the NIH and collaborators for diligently supporting us over many years in realizing the potential of MCO therapy in vision restoration for patients regardless of underlying genetic mutation."

The Company expects to present the RESTORE top-line results at upcoming medical conferences.

About Retinitis Pigmentosa
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina, and when activated by light they trigger the physiological process of vision. RP encompasses a group of rare genetic disorders in which the retina's photoreceptor cells degrade over time, leading to impaired vision and eventual blindness. These disorders are believed to be linked to over 100 different gene mutations. Approximately 100,000 people in the U.S. and an estimated 2 million people worldwide suffer from RP, making it the leading cause of inheritable blindness.

About MCO-010
Current gene therapies are aimed to treat patients with specific gene mutation in outer retinal cells, while ambient-light activatable MCO optogenetic monotherapy targeting abundant inner retinal neurons has the potential to restore vision lost due to advanced RP, with degenerated outer retinal cells. MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and most-light sensitive opsin currently in clinical trials. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase 1/2 trial of MCO-010 in advanced RP patients demonstrated improvement in vision-guided mobility, shape discrimination and visual acuity. A significant proportion of patients treated with MCO-010 in Phase 2b RESTORE trial exhibited improvements in functional vision assessed by vision-guided mobility, shape discrimination and visual acuity, along with a favorable safety profile.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure exists. The company's lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also fully enrolled a Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
[email protected]

SOURCE Nanoscope Therapeutics