DALLAS, April 27, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced the presentation of key data from its Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010 at the ARVO annual meeting. MCO-010 is an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic therapy for vision restoration in advanced retinitis pigmentosa (RP), irrespective of gene mutation. Composite efficacy data validate MCO-010 as a potential treatment for RP. 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. Results showed vision function improvements after treatment with MCO-010 consistent with previous studies as well as a favorable safety profile. Key efficacy measures included Multi-Luminance Y-Mobility Test (MLYMT, vision-guided mobility), Multi-Luminance Shape Discrimination Test (MLSDT, near-field object recognition), and Best-Corrected Visual Acuity (BCVA) scores. 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.

Composite outcomes in key efficacy measures at 12-months were:

• 18 of 18 (100%) MCO-010 treated patients showed vision improvement in the MLYMT, MLSDT or BCVA compared to 5 of 9 (55.6%) receiving placebo (p=0.007);
• 17 of 18 (94.4%) MCO-010 treated patients showed vision improvement in the MLYMT or BCVA compared to 4 of 9 (44.4%) receiving placebo (p=0.008);
• 16 of 18 (88.9%) MCO-010 treated patients demonstrated a 2 or more luminance level improvement in the MLYMT or MLSDT compared to 4 of 9 (44.4%) receiving placebo (p=0.02); and
• 14 of 18 (77.8%) MCO-010 treated patients showed vision improvement in the MLSDT or BCVA compared to 3 of 9 (33.3%) receiving placebo (p=0.04).

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. 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.

These RESTORE results are consistent with those observed in the earlier Phase 1/2 trial (NCT04919473) in which 9 of 11 (82%) of subjects demonstrated 2 luminance level improvements in vision-guided mobility or 0.3 logMAR in visual acuity. In this Phase 1/2 study, a favorable safety profile was observed, with no serious or severe adverse events. People with severe vision loss due to RP currently have no available treatments that can improve their vision.

“I am honored to present the RESTORE trial results at ARVO. I have had the privilege of treating severely blind patients, enrolled in MCO-010 clinical trials, and have observed some improvement in their visual function. The fact that we see any gains in vision after a single intravitreal injection is remarkable,” said David Boyer, MD, Retina-Vitreous Associates Medical Group in Beverly Hills, CA and Nanoscope Clinical Advisory Board member. “Some participants who were living with severe vision impairment due to RP have noticed improvement in visual function. In addition, MCO-010 had a favorable safety profile. It is an honor to have been a part of the first randomized controlled trial to show a visual improvement in a profoundly visually impaired population.”

“RP patients with severe vision loss are a heterogeneous population with different genotypes and phenotypic manifestations of degeneration in their macula and peripheral retina. For this reason, we strongly believe that no single assessment can adequately capture clinically important changes in vision across this broad population. Composite endpoints can be used to evaluate overall vision function changes in a single measurement when the individual tests assess different aspects of vision performance, such as vision-guided mobility, object recognition and visual acuity. Across composite endpoints in the RESTORE study, significantly more MCO-010 treated patients experienced clinically significant vision improvements. This randomized controlled trial provides compelling evidence that MCO-010 optogenetic therapy, as a mutation-agnostic treatment, can improve vision in patients with advanced RP,” said Samarendra Mohanty, Co-founder, President and Chief Scientific Officer of Nanoscope.

“The treatment landscape for severe vision loss due to RP is one of no approved therapies and lacking a pre-defined single endpoint for approval. The improvements we are seeing in MCO-010 treated patients across the key composite measures of efficacy make it a promising candidate for the treatment of patients with severe vision loss due to RP,” added Sulagna Bhattacharya, Co-founder and Chief Executive Officer of Nanoscope. “We look forward to our upcoming conversations with the FDA on the totality of this data regarding an expeditious path to market for this exciting therapy. It truly brings us joy to see the impact and difference MCO-010 is making in the lives of patients, and we are grateful to all participants and investigators for being a part of the success of the RESTORE trial.”

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
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

DALLAS, April 13, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that its Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in three presentations at the ARVO Annual Meeting, taking place April 23-27, 2023 in New Orleans. The management team will also be present at the conference and available to meet at the Nanoscope Therapeutics booth #1056.

Details for the presentations are as follows:

Abstract Title: MCO-010 intravitreal optogenetic therapy in Stargardt disease. 6-month outcomes from the Phase 2 STARLIGHT trial
Abstract Number: 3546 – B0388
Session Type: Poster Session
Session Name: Advances in Ocular Inflammatory Disease Therapy
Session Date: Tuesday, April 25, 2023
Session Time: 3:30-5:15 p.m. CT
Location: Exhibit Hall, New Orleans Ernest N. Morial Convention Center
Presenter: Victor Hugo Gonzalez, M.D., Valley Retina Institute, McAllen, TX

Dr. Gonzalez will discuss Nanoscope’s 6-month safety and efficacy outcomes from the Phase 2 STARLIGHT trial for Stargardt disease. The outcomes will help inform the future clinical development of MCO-010 optogenetic therapy for vision restoration in Stargardt disease and other retinal degenerative conditions.

Abstract Title: Non-viral Targeted Laser Delivery of Multi-Characteristic Opsin Genes for Treatment of Geographic Atrophy
Abstract Number: 3861 – B0141
Session Type: Poster Session
Session Name: Gene and Cell Therapy
Session Date: Wednesday, April 26, 2023
Session Time: 10:30 a.m.-12:15 p.m. CT
Location: Exhibit Hall, New Orleans Ernest N. Morial Convention Center
Presenter: Samarendra Mohanty, Ph.D., President and Chief Scientific Officer of Nanoscope Therapeutics

Dr. Mohanty will detail the Optical Coherence Tomography guided non-viral laser approach that provides efficient, spatially targeted delivery of MCO-020 genes into retinal cells in geographic atrophy areas.

Abstract Title: Efficacy and safety of MCO-010 optogenetic therapy for vision restoration in patients with severe vision loss due to retinitis pigmentosa: A phase 2b randomized, sham-controlled, multi-center, multi-dose, double-masked clinical trial (RESTORE)
Abstract Number: 5443
Session Type: Paper Session
Session Name: Retinitis pigmentosa
Session Date: Thursday, April 27, 2023
Presentation Time: 1:30-1:45 p.m. CT
Location: 353-355, New Orleans Ernest N. Morial Convention Center
Presenter: David Boyer, M.D., Senior Partner, Retina-Vitreous Associates Medical Group in Southern California

Dr. Boyer will discuss Nanoscope’s 52-week safety and efficacy outcomes from the Phase 2b RESTORE trial, the Company’s most advanced clinical program. Efficacy and safety data from the RESTORE trial will provide evidence on the benefit/risk profile of MCO-010 in retinitis pigmentosa.

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
Nanoscope@argotpartners.com

• 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
Nanoscope@argotpartners.com

Nanoscope Therapeutics Appoints Industry Veteran Vicente Anido Jr., Ph.D., as a Strategic Advisor

DALLAS, March 20, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced the appointment of Vicente Anido Jr., Ph.D., as a Strategic Advisor to the Company’s Board and Management.

“We are pleased to have Vince join our team of advisors. His deep experience and accomplishments in building and commercializing profitable ophthalmic companies will provide valuable guidance to support our clinical and corporate strategy. We look forward to leveraging his expertise as we advance Nanoscope’s platform of optogenetic therapies to restore vision in sufferers of inherited retinal degenerative diseases,” said Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics.

“Nanoscope’s Multi-Characteristic Opsin optogenetic monotherapy, MCO-010, is a novel approach to treat inherited retinal diseases and I am eager to help the Company navigate through advanced clinical development and towards commercialization,” said Dr. Anido Jr. “MCO-010 demonstrates a compelling clinical profile with meaningful market opportunities. I look forward to working with the team in developing a successful clinical and corporate strategy to bring this potential sight-restoring technology to patients.”

As an ophthalmology industry leader, Dr. Anido Jr. brings over three decades of ophthalmic experience and will assist Nanoscope in its path towards the potential commercialization of MCO-010. He was most recently the Chief Executive Officer and Chairman at Aerie Pharmaceuticals. Before Aerie, he led several companies including his role as the Chief Executive Officer at ISTA Pharmaceuticals that was sold to Bausch & Lomb for $500 million in 2012. Dr. Anido Jr. holds a Ph.D. in Pharmaceutical Sciences from the University of Missouri, Kansas City and an M.S. and a B.S. in Pharmacy from West Virginia University.

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, is presently in RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. 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
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Announces World Health Organization Selection of “Sonpiretigene Isteparvovec” as International Nonproprietary Name for MCO-010

DALLAS, Feb. 28, 2023  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization (WHO) has selected “sonpiretigene isteparvovec” for the nonproprietary name of the Company’s MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease. MCO-010 has been granted orphan drug designation by the US Food and Drug Administration for both diseases and  multiple mid- to late-stage clinical trials are ongoing, including the Company’s Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial for RP. Top line data from RESTORE  are expected in Q1 2023.

The name “sonpiretigene isteparvovec” for MCO-010 was selected by the WHO Expert Advisory Panel of the International Pharmacopoeia and Pharmaceutical Preparations. MCO-010 reached the status of recommended INN after a period of public consultation and was included in the INN Recommended List 128, published in the No. 4 issue of the WHO Drug Information, Volume 36 in January 2023.

Dr. Samarendra Mohanty, President of Nanoscope, said “The WHO’s INN system has facilitated the name of sonpiretigene isteparvovec for the active pharmaceutical ingredient, which is the foundation of our clinical development in several retinal degenerative diseases with high unmet need. We believe that finalizing this name is an important step towards potential future commercialization of MCO-010.”

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, is presently in RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. 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
Nanoscope@argotpartners.com

Nanoscope Therapeutics CEO Sulagna Bhattacharya Joins BIO Health Section Governing Board

DALLAS, TX (February 13, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics, has been named to the Health Section Governing Board of the Biotechnology Innovation Organization (BIO) Board of Directors.

Ms. Bhattacharya was named to the BIO Board of Directors in February 2022 and currently serves on the Emerging Companies Section Governing Board, on which she will continue to serve in addition to her new role on the Health Section Governing Board. She has also been selected as a member of BIO’s Nominating Committee.

The BIO Health Section Governing Board is comprised of thought leaders who bring their expertise to major issues affecting their specific area of the biotechnology industry.

“Accelerating development of innovative therapies is a true passion of mine,” said Ms. Bhattacharya. “I look forward to working with the other biopharma leaders on the BIO Health Section Governing Board to promote policy strategies that enable this acceleration across the Biotech sector.”

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 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
Nanoscope@argotpartners.com

Nanoscope Therapeutics to Present at the BIO CEO & Investor Conference

DALLAS, Feb. 2, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Chief Executive Officer and Co-Founder of Nanoscope Therapeutics, will present at the BIO CEO & Investor Conference, taking place February 6-9, 2023, in New York.

Ms. Bhattacharya will provide a company update on Nanoscope’s clinical and corporate activities on Tuesday, February 7, from 2:00-2:15 p.m. Eastern Time in the Winter Garden Room (6^th floor) at the Marriott Marquis. Nanoscope leadership will be available for meetings during the conference from Monday, February 6 to Thursday, February 9. Those interested in scheduling a meeting may do so through the BIO One-on-One Partnering system: https://www.bio.org/events/bio-ceo-investor-digital-conference/partnering.

Ms. Bhattacharya presently serves on the board of directors of BIO. She is also a member of multiple BIO sub-committees for capital formation, strategy advocating for public policy beneficial to small-cap and private biotech companies.

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 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
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Receives Fast Track Designation by the FDA for MCO-010 for the Treatment of Stargardt Disease

DALLAS, Jan. 30, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to MCO-010, an ambient-light activatable Multi-Characteristic Opsin (MCO) optogenetic monotherapy to restore vision in blind patients, for the treatment of Stargardt disease to improve visual function.

“We are pleased to receive the FDA’s decision to grant Fast Track Designation for the second indication of our vision restoring therapy, and we look forward to sharing six-month data from the Phase 2 STARLIGHT clinical trial of MCO-010 therapy in Stargardt patients this quarter,” said Sulagna Bhattacharya, CEO of Nanoscope. “Receiving Fast Track Designation reinforces the urgency of our mission to find an effective solution for Stargardt patients and is an acknowledgement of MCO-010’s potential to address this unmet medical need.”

In September 2022, enrollment was completed in the Phase 2 open-label STARLIGHT clinical trial (NCT05417126) of MCO-010 in six patients with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease. In this study, all subjects received the same single intravitreal dose of 1.2E11gc/eye of MCO-010. Six-month data from the STARLIGHT trial are expected in Q1 2023.

The FDA’s Fast Track designation is intended to facilitate the development and expedite the review of novel therapies to treat serious conditions for which there is unmet medical need. With the Fast Track designation, Nanoscope Therapeutics is eligible for more frequent regulatory meetings and communications with the FDA.

In February 2022, enrollment was completed in the Phase 2b multicenter, randomized, double-masked, sham-controlled RESTORE clinical trial (NCT04945772) of MCO-010. Top line data from the RESTORE trial are also expected in Q1 2023.

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation 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
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics Announces Presentations at the Optogenetics and Optical Manipulation Conference

DALLAS, Jan. 23, 2023  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced an oral presentation and a poster presentation at the Optogenetics and Optical Manipulation Conference as part of the SPIE Photonics West BiOS Program, taking place January 28-February 2, 2023 in San Francisco.

Details for the oral presentation are as follows:

Abstract Title: Functionalizing inner retina with multi-characteristic opsin arrested further degeneration and functional deterioration
Abstract Number: 12366-10
Session Name: 3. Clinical Applications
Session Date: Sunday, January 29, 2023
Presentation Time: 11:40 a.m.-12:00 p.m. PT
Location: Moscone Center, Room 101 (Level 1 South Lobby)
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer of Nanoscope Therapeutics

In addition to highlighting the benefits of MCO gene therapy, Dr. Mohanty will discuss the results of intravitreally delivered AAV-carried MCO-010 in multiple mice models of retinal degeneration, including retinitis pigmentosa and Stargardt disease, that showed arrest of further retinal degeneration as evaluated by optical coherence tomography (OCT), electroretinogram (ERG) and immuno-histochemistry (IHC).

Details for the poster presentation are as follows:

Abstract Title: OCT-guided variable spot electrophysiology platform for correlated structural and functional assessment of optogenetic intervention
Abstract Number: 12366-24
Session Name: BiOS poster session
Session Date: Sunday, January 29, 2023
Presentation Time: 5:30-7:00 p.m. PT
Location: Moscone Center, Level 2 West

Dr. Mohanty will detail the development of image-guided spatially resolved functional assessment of retina using OCT-guided variable-spot stimulated electroretinogram (vsERG). The OCT-vsERG platform is currently used to monitor diseased areas in retina of patients with Stargardt macular degeneration and to measure functional changes after optogenetic therapy.

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

SOURCE Nanoscope Therapeutics

Nanoscope Therapeutics to Present at the Next Generation Ophthalmic Drug Delivery Summit

DALLAS, Jan. 19, 2023  — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, President and Chief Scientific Officer of Nanoscope Therapeutics, will give a presentation titled “Pioneering Laser-Targeted Optical Delivery of Therapeutics to the Eye” and participate in a panel discussion at the Next Generation Ophthalmic Drug Delivery Summit, taking place January 24-26, 2023 in San Francisco.

Details for the presentation and panel are as follows:

Pioneering Laser-Targeted Optical Delivery of Therapeutics to the Eye
Date and Time: Thursday, January 26, 2023; 11:45 a.m. PT
Location: Hyatt Centric Fisherman’s Wharf

In his presentation, Dr. Mohanty will discuss the application of laser-targeted optical delivery as a therapeutic platform, and he will detail Nanoscope’s proof-of-concept to deliver genes and other molecules.

Panel Discussion: What is the Ideal System to Test Durable Ophthalmic Therapies? Discussing Desired
Attributes for a Benchmark System
Date and Time: Thursday, January 26, 2023; 9:15 a.m. PT
Location: Hyatt Centric Fisherman’s Wharf

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope’s Top Executive Team to Participate in Advanced Therapies Week

DALLAS, TX (January 11, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Dr. Samarendra Mohanty, President and Chief Scientific Officer, will give a presentation titled “Pioneering a New Wave of Gene-Agnostic Gene Therapy” in the session “The Next Wave: Cell and Gene Therapy’s Commercial Shift” at Advanced Therapies Week, taking place January 17-20, 2023 in Miami. He will also participate in the closing panel discussion.

Details for the talk and panel are as follows:

Title: Pioneering a New Wave of Gene-Agnostic Gene Therapy
Session: The Next Wave: Cell and Gene Therapy’s Commercial Shift
Date and Time: Thursday, January 19, 2023; 3:35 p.m.- 4:05 p.m. ET (talk) and 4:35 p.m.-5:00 p.m. ET (panel)

Location: Miami Beach Convention Center

Advanced Therapies Week brings together industry leaders in healthcare for knowledge sharing and relationship building to advance the next pillar of medicine.

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com

Nanoscope Therapeutics Appoints Massoud Motamed, PhD, as VP of Quality

DALLAS, TX (January 5, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced the appointment of Massoud Motamed, PhD, as Vice President of Quality. Dr. Motamed brings nearly a decade of CGMP quality and compliance experience, including extensive experience in manufacturing and regulatory review of gene therapy products.

“Massoud’s experience leading quality and compliance in CGMP manufacturing for gene therapies will be valuable as we progress in our Chemistry, Manufacturing and Controls (CMC) capabilities. This appointment supports the continued advancement of our optogenetic therapies to restore vision in millions of people blinded by retinal diseases and highlights our evolution as a company,” said Sulagna Bhattacharya, CEO of Nanoscope.

“I am thrilled to join the highly motivated team at Nanoscope in the development of its novel approach to treating retinal degenerative disease,” said Dr. Motamed. “I look forward to helping Nanoscope navigate advanced clinical development and upcoming regulatory submissions as the Company continues to progress in its clinical trials.”

Dr. Motamed was most recently a CMC reviewer for the Gene Therapy Branch at the Center for Biologics Evaluation and Research (CBER), the center within the US Food and Drug Administration (FDA) that regulates biological products for human use under applicable federal laws. Dr. Motamed has received many awards including FDA’s Outstanding Service award from the Office of Regulatory Affairs. Previously, he served as Head of Quality for the Viral Vector Core at UT Southwestern Medical Center. Prior to this, Dr. Motamed held appointments as Head of Auditing and Compliance at Cipla, a global pharmaceutical company, as well as an FDA inspector for pharmaceutical quality and compliance. He holds a PhD in Biochemistry from UT Southwestern Medical Center, an MS in Organic Chemistry from the University of California, Berkeley and a BS in Biochemistry from Southern Methodist University.

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, is presently in Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trials in the U.S. for retinitis pigmentosa (NCT04945772) with top-line data expected Q1 2023. The company has also fully enrolled a Phase 2 trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designation for RP and FDA orphan drug designations for RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.

Investor Contact:
Argot Partners
212-600-1902
Nanoscope@argotpartners.com