Nanoscope Therapeutics to Present at the 4th Annual Dry AMD Therapeutic Development Summit
DALLAS, Nov. 9, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Dr. Samarendra Mohanty, President and Chief Scientific Officer will participate in a panel and present at the 4th Annual Dry AMD Therapeutic Development Summit taking place from November 14-16, 2023 in Boston, Massachusetts. Details for the panel and presentation are as follows:
Title: Overcoming the Limitations of Dry AMD not having a Recognised Disease Model
Session: Navigating how to Optimize Current Dry AMD In Vivo & In Vitro Models to Improve Efficacy
Session Date: November 15, 2023
Session Time: 11:00 a.m. E.T.
Location: Hilton Boston Logan Airport
During the panel discussion, Dr. Mohanty will highlight the reasons why developing a relevant animal model for dry AMD remains a pertinent challenge. He will also explore ways to incorporate more NHPs in preclinical testing to better inform the safety profile of dry AMD therapeutics.
Title: Expanding Optogenetics Vision Restoration to GA via Laser Delivery of Multi-Characteristic Opsin
Session: Investigating the Latest Advancements for Improving the Delivery of Dry AMD Therapeutics
Session Date: November 16, 2023
Session time: 4:00 p.m. E.T.
Location: Hilton Boston Logan Airport
In his presentation, Dr. Mohanty will discuss the prospect of optogenetics to improve visual acuity in macular degeneration patients from proof-of-concept data obtained in humans, while assessing the latest preclinical data on the use of the laser delivery of Multi-Characteristic Opsin (MCO). He will also discuss the platform’s potential to treat different retinal degenerative conditions.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy, secondary to AMD.
Investor Contact:
Argot Partners
212-600-1902
[email protected]
SOURCE Nanoscope Therapeutics
Nanoscope Therapeutics Awarded ‘Best of Show’ for Retina at Eyecelerator@AAO 2023
DALLAS, TX (November 3, 2020) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), has been awarded “Best of Show for Retina” at Eyecelerator@AAO2023. The award was presented to Nanoscope’s Chief Medical Officer, Samuel Barone, MD, for his presentation during the event’s Retina Showcase. In his presentation Dr. Barone showcased the company’s Multi-Characteristic Opsin platform and its potential to restore vision based on recent clinical data in patients with advanced retinal degenerative diseases.
Sam Barone, MD, Chief Medical Officer of Nanoscope Therapeutics accepts the “Best of Show – Retina” award during Eyecelerator@AAO2023 in San Francisco, November 2, 2023
Recipient of the Best of Show Award is chosen by a vote of conference attendees. “We are thrilled and honored for this recognition,” said Sulagna Bhattacharya, CEO, “It is very special because it comes from our own retina community. We thank you all for the encouragement. It means a great deal to our entire team.”
“The patient experience was front and center in my presentation, and I appreciate the acknowledgment of our work to make a difference in their lives,” said Samuel Barone, CMO.
Nanoscope Therapeutics leadership celebrates “Best of Show” award at Eyecelerator. From left, Jared Stephens, VP Strategy & Business; Samarendra Mohanty, Co-Founder & President; Sam Barone, Chief Medical Officer; Sulagna Bhattacharya, CEO; Michael Marquez, CFO.
Eyecelerator@AAO2023 took place on November 2, 2023 in advance of the American Academy of Ophthalmology Annual Meeting in San Francisco. Eyecelerator focuses on emerging trends, advancements and innovations in eyecare.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics Announces Presentations at the American Academy of Ophthalmology Annual Meeting 2023
DALLAS, Oct. 30, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced presentations at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting and Eyecelerator @ AAO 2023 taking place in San Francisco, California. Details for the presentations are as follows:
Eyecelerator @ AAO 2023
Session Title: Retina Showcase
Session Date: November 2, 2023
Session Time: 12:40 p.m. P.T.
Location: San Francisco Marriott Marquis, Nob Hill Room
Presenter: Samuel Barone, M.D., Chief Medical Officer
During his presentation, Dr. Barone will showcase our Multi-Characteristic Opsin platform and its potential to restore vision based on recent clinical data in patients with advanced retinal degenerative diseases.
American Academy of Ophthalmology (AAO) 2023 Annual Meeting
Title: MCO-010 optogenetic therapy for vision loss in Stargardt disease: Topline data from the Phase 2 STARLIGHT trial
Session Title: RET14 (Section XI: Late Breaking Developments, Part II)
Session Date: November 4, 2023
Session Time: 9:10 a.m. P.T. (In Person, Live Broadcast, On Demand)
Location: Moscone Center, West Building – Rm. 3004
Presenter: Stephen H. Tsang, M.D., Ph.D.
During his presentation, Dr. Tsang will discuss end-of-study data from our Phase 2 STARLIGHT trial of MCO-010 therapy in patients with severe vision loss due to Stargardt disease.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics Strengthens Research and Development Leadership with Appointment of Najam Sharif, PhD, DSc.
DALLAS, Oct. 26, 2023 / — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced the appointment of Najam Sharif, PhD, DSc, a veteran in drug/device discovery and development, as Vice President of Global Research and Development.
“We are delighted to welcome Naj to the Nanoscope team,” said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope. “His extensive experience in ophthalmology and pharmaceutical drug discovery and development is a natural fit for our organization, and we look forward to drawing on his clinical and regulatory expertise in particular at this pivotal time for MCO-010.”
“I am honored to join Nanoscope, a company that I see as transformative in the field of ophthalmology. As their lead asset, MCO-010, advances toward FDA approval, I look forward to the opportunity to positively impact the lives of people living with inherited retinal diseases, and to bring treatment options to a population for which there are no established cures,” said Dr. Sharif.
Dr. Naj Sharif brings over three decades of experience in pharmaceutical drug/device discovery and development to Nanoscope, including specialized expertise in ophthalmology and neuroscience. Throughout his career, he contributed to the discovery and development of several FDA-approved commercial drugs, including anti-glaucoma agent Travatan® and anti-allergic/inflammatory drugs such as Patanol® /Pataday® and Emedine®. In addition, Dr. Sharif was instrumental in guiding three drugs through to FDA approvals: Simbrinza® for glaucoma, IZBA® for the treatment of ocular hypertension and glaucoma, and PAZEO™ for the treatment ocular allergies. Most recently, Dr. Sharif served as Vice President, Global Biomedical Sciences & Head of Glaucoma and Neuroprotection at Santen, where he managed the company’s Clinical Sciences, Data Sciences, and Biometrics departments, and where he also established and directed Global Alliances & External Research activities. Prior to this, he held positions related to R&D and regulatory affairs at Alcon Research-Novartis, Synaptic Pharmaceutical Corp, and Syntex Pharma / Roche Pharmaceutical Corp. He is widely published with an extensive number of peer-reviewed publications, edited books and holds multiple patents for mitigation of neurological and ophthalmic diseases. He has been an invited organizer, chairman and speaker at multiple national and international conferences.
In addition to his professional experience, Dr. Sharif has served on numerous boards and committees. Dr. Sharif is an elected Fellow of multiple learned societies such as Association for Research in Vision and Ophthalmology, Association for Ocular Pharmacology and Therapeutics and British Pharmacological Society. Currently he serves on the Scientific Advisory Board of the Glaucoma Foundation and as an adjunct professor for the Duke-NUS Medical School in Singapore, and Institute of Ophthalmology at University College London. He is the recipient of several honors, including the Ernst H. Barany prize for “Outstanding Contributions to Ocular Pharmacology”, the first and inaugural “Dr. Roger Vogel Award” for Pharmaceutical Research, and the Sir James Black Award for “Contributions to Drug Discovery”.
Dr. Sharif received a PhD in neuroscience, DSc (by thesis) in ophthalmology, and dual bachelor’s degree in physiology and biochemistry from the University of Southampton (UK).
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics to Present at the 13th The Eye and the Chip World Research Congress
DALLAS, TX (October 9, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), today announced that Dr. Arup Roy, and Michael Carlson will present at the 13th The Eye and The Chip World Research Congress taking place from October 8-10 in Southfield, Michigan. Details for the presentations are as follows:
Title: Development and Validation of the Multi-Luminance Y-Mobility Test (MLYMT)) for Assessment of Vision-Guided Mobility in Patients with Profound Vision Loss
Session Title: Session Ten: Psychophysics continued
Session Date: October 10
Session Time: 10:00-10:30 a.m. E.T.
Location: The Westin Southfield Hotel
Presenter: Arup Roy, Ph.D.
During his talk, Dr. Roy will discuss development and validation of novel mobility based functional vision assessment by MLYMT using longitudinal data from subjects with different levels of vision, evaluated by masked graders.
Title: Development and Validation of the Multi-Luminance Shape Discrimination Test (MLSDT) for Assessment of Near Object Recognition in Profoundly Vision Impaired Patients
Session Title: Group “A” Poster Presenters
Session Date: October 9
Session Time: 6:00-7:00 p.m. E.T.
Location: The Westin Southfield Hotel
Presenter: Michael Carlson
During his poster presentation, Mr. Carlson will discuss development and validation of novel near-object functional vision assessment by MLSDT using data from subjects with different levels of vision collected at multiple time points.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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]
Nanoscope Therapeutics Announces Presentations at The Retina Society 56th Annual Scientific Meeting
DALLAS, Oct. 9, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and macular degenerations, today announced presentations at The Retina Society 56th Annual Scientific Meeting taking place in New York, New York from October 11-14, 2023.
Details for the presentations are as follows:
Title: MCO-010 Optogenetic Vision Restoration in Retinitis Pigmentosa Patients with Profound Vision Loss: A Randomized, Sham-Controlled, Multi-center, Double-Masked Clinical Trial (RESTORE)
Session Title: Genetic Diseases, Dystrophies & Degenerations
Session Date: October 11, 2023
Session Time: 4:56 p.m. E.T.
Location: Intercontinental Barclay Hotel
Presenter: Christine Kay, M.D., Director of Electrophysiology, Retinal Genetics, and Clinical Trials at Vitreoretinal Associates in Gainesville, Florida, and Affiliate Assistant Professor at the University of South Florida.
In her presentation, Dr. Kay will discuss clinical data and analysis from Nanoscope’s Phase 2b RESTORE trial in patients with advanced retinitis pigmentosa.
Title: MCO-010 Optogenetic Therapy for Profound Vision Loss in Stargardt Disease: 12-Month Outcomes from the Phase 2 STARLIGHT Trial
Session Title: Imaging
Session Date: October 12, 2023
Session Time: 5:02 p.m. E.T.
Location: Intercontinental Barclay Hotel
Presenter: Allen Ho, M.D., Professor of Ophthalmology at Thomas Jefferson University and Attending Surgeon, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital.
In this late-breaking presentation, Dr. Ho will discuss recent 1 year data from Nanoscope’s Phase 2 STARLIGHT trial of MCO-010 therapy in patients with advanced Stargardt macular degeneration.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics Announces Presentations in Upcoming Conferences
DALLAS, Oct. 2, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and age-related macular degenerations (AMD), is scheduled to participate in the 2023 Ophthalmology Futures Retina Forum and Euretina Innovation Spotlight at the 23rd Euretina Congress, each taking place in Amsterdam, The Netherlands. Details for the presentations are below:
Ophthalmology Futures Retina Forum
Panel Discussion: Visual Preservation in Retinal Diseases: Will Gene Therapy Make a Dent?
Session Date: October 4, 2023
Session Time: 12:30-1:00 p.m. CEST
Location: Hyatt Regency Hotel
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer
On the panel, Dr. Mohanty will highlight gene-agnostic approaches that have the potential to affect large patient populations with retinal degeneration diseases.
Title: Pioneering a New Wave of Optogenetic Therapies for Vision Restoration
Session Date: October 4, 2023
Session Time: 2:38-2:45 p.m. CEST
Location: Hyatt Regency Hotel
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer
In this session, Dr. Mohanty will highlight Nanoscope’s Multi-Characteristic Opsin platform and recent clinical data from the clinical trials for patients with advanced retinitis pigmentosa and Stargardt disease.
Euretina Innovation Spotlight (EIS)
Title: Pioneering a New Wave of Optogenetic Therapies for Vision Restoration
Session Date: October 4, 2023
Session Time: 1:00 – 7:40 p.m. CEST
Location: Euretina Congress Centre (RAI) Amsterdam
Presenter: Sulagna Bhattacharya, Chief Executive Officer
In this poster session, Ms. Bhattacharya will highlight Nanoscope’s Multi-Characteristic Opsin and Non-viral Laser delivery platform technologies for advancing Nanoscope’s pipeline of inherited retinal disorders and advanced age-related macular degeneration.
23rd Euretina Congress
Abstract Title: MCO-010 Optogenetic Therapy to Treat Stargardt Disease: Safety and Efficacy through Week 48 in the Phase 2a STARLIGHT Clinical Trial
Session Title: Euretina Session 6 – Landmarks & Late Breakings
Session Date: October 6, 2023
Session Time: 12:15-1:15 p.m. CEST
Location: RAI Amsterdam
Presenter: Michael Singer, M.D., Clinical Professor of Ophthalmology at the University of Texas Health Science Center
In his session, Dr. Singer will discuss recent data from Nanoscope’s Phase 2 STARLIGHT trial of MCO-010 therapy in patients with advanced Stargardt macular degeneration.
Abstract Title: Optogenetic therapy with MCO-010 in severe sight loss due to retinitis pigmentosa: 12-month results from the RESTORE randomized, placebo-controlled double-masked clinical trial
Session Details: Available as an audio-narrated free paper viewable at the event or on the virtual congress platform at 4:00 p.m. CEST on October 5, 2023.
Location: RAI Amsterdam
Presenter: SriniVas R. Sadda, M.D., President and Chief Scientific Officer of the Doheny Eye Institute, the Stephen J. Ryan – Arnold and Mabel Beckman Endowed Chair, and Professor of Ophthalmology at the University of California – Los Angeles (UCLA), David Geffen School of Medicine
Dr. Sadda will discuss clinical data and analysis from Nanoscope’s Phase 2b RESTORE trial in patients with advanced retinitis pigmentosa.
Abstract Title: Design and Development of Two Novel Assessments of Vision Dependent Function in Patients with Severe Vision Impairment due to Retinitis Pigmentosa Receiving MCO-010
Session Details: Available as an audio-narrated free paper viewable at the event or on the virtual congress platform at 4:00 p.m. CEST on October 5, 2023.
Location: RAI Amsterdam
Presenter: Victor Gonzalez, M.D., Valley Retina Institute, McAllen, TX
Dr. Gonzales will discuss the validation of two novel functional vision endpoints, mobility and object recognition, suitable to assess low-vision patients such as those with advanced retinitis pigmentosa.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics to Present at the 4th Annual Gene Therapy for Ophthalmic Disorders Summit
DALLAS, TX (September 28, 2023) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced that Dr. Sam Barone, Chief Medical Officer, will give a presentation on the clinical development of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy platform at the 4th Annual Gene Therapy for Ophthalmic Disorders Summit taking place from October 3-5 in Boston, Massachusetts. Details for the presentation are as follows:
Title: Progressing Optogenetic Gene Therapies Through the Clinic
Session Title: Optogenetics & Mutation-Independent Vision Restoration in the Clinical Setting
Session Date: October 4, 2023
Session Time: 12:20 p.m. E.T.
Location: Hilton Boston Back Bay
Presenter: Sam Barone, M.D., Chief Medical Officer
During his presentation, Dr. Barone will introduce and highlight the benefits of Nanoscope’s proprietary MCO gene therapies for patients with inherited retinal dystrophies (IRDs). Additionally, Dr. Barone will discuss clinical data from RESTORE, a Ph2b multi-center, randomized controlled trial of lead asset MCO-010 in patients with advanced Retinitis. Pigmentosa. Recent clinical progress from STARLIGHT, a Ph2 multi-center open-label trial of MCO-010 in patients with advanced Stargardt Macular Degeneration will also be presented.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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]
Nanoscope Therapeutics Strengthens Clinical and Regulatory Expertise With Appointment of Samuel Barone, M.D., as Chief Medical Officer
- Former U.S. Food and Drug Administration (FDA) officer, practicing ophthalmologist and vitreoretinal surgeon to lead Nanoscope’s broad clinical pipeline of optogenetic gene therapies
DALLAS, Sept. 21, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, announced today the appointment of Samuel Barone, M.D., an accomplished leader in the pharmaceutical space and former FDA official, as Chief Medical Officer.
“We are thrilled to welcome Dr. Barone to the Nanoscope team. The regulatory expertise garnered during his tenure at the FDA will be instrumental as we continue to advance our pipeline through late-stage clinical development and navigate the regulatory process,” said Sulagna Bhattacharya, Co-Founder and Chief Executive Officer of Nanoscope. “Dr. Barone will support our mission to bring sight-restoring therapies to patients with inherited retinal diseases in an expeditious manner.”
“Nanoscope has the potential to impact the treatment landscape with its gene-agnostic, sight-restoring optogenetic therapies and bring these transformative therapeutic options to patients with severe vision loss due to inherited retinal diseases, for which there are no restorative therapies available,” said Dr. Barone. “I look forward to taking MCO-010 through approval and advancing Nanoscope’s follow-on assets to the clinic.”
Dr. Samuel Barone is a board-certified ophthalmologist and vitreoretinal surgeon with clinical development and regulatory experience across a range of indications in ophthalmology, dermatology, rare diseases, and cell and gene therapy. He was most recently Chief Medical Officer at Gemini Therapeutics prior to its merger with Disc Medicine, where he oversaw the development of novel therapeutics targeting the complement system to treat genetically defined age-related macular degeneration and systemic renal disease. Prior to that he was Chief Medical Officer for Veloce BioPharma, a topical therapeutics company focusing on unmet clinical needs in dermatology, ophthalmology and supportive oncology. He was also Chief Medical Officer for Avalanche Biotechnologies and then Senior Vice President, Clinical Development, for Adverum Biotechnologies. Both roles included supervision and implementation of the clinical development of AAV-based gene therapy products targeting unmet medical needs in ophthalmology and rare diseases. Early in his career, he served on active duty as a flight surgeon for the United States Air Force.
As a Senior Medical Officer at the FDA in the Office of Cellular, Tissue and Gene Therapies (now the Office of Therapeutic Products), he served as a subject matter expert and developed FDA Guidance Documents. He has global experience in overseeing clinical trials in the U.S. as well as in Australia and India. He was involved with Nanoscope for the Phase 1/2a clinical trial design for MCO-010.
Dr. Barone received an M.D. from The Pennsylvania State University College of Medicine. He completed a residency in ophthalmology at The New York Eye and Ear Infirmary and a medical and surgical retina fellowship at the University of California, San Diego.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal 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 recently completed their end-of-study visit for the 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 retinitis pigmentosa 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
Nanoscope Therapeutics Unveils Clinical Trial Results for MCO-010 in Treating Stargardt Disease
DALLAS, Aug. 9, 2023 — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company focused on gene therapies for retinal degenerative diseases, presented key results from its Phase 2 clinical trial, STARLIGHT, at the American Society of Retina Specialists (ASRS) annual meeting in Seattle last week. The trial evaluated the effects of their novel therapeutic, MCO-010, for patients with Stargardt disease, a condition currently lacking available treatments.
Key results from the STARLIGHT clinical trial:
- Patients treated with MCO-010 demonstrated clinically meaningful improvements in best-corrected visual acuity (BCVA)
- An approximate 3 dB gain in mean sensitivity, as measured by Octopus visual field perimetry
- No serious adverse events (SAEs) were observed in patients treated with MCO-010 in this study
“I appreciated the opportunity to present on the promising results of Nanoscope’s Phase 2 STARLIGHT clinical trial. With no current treatment options, MCO-010 may bring hope to those patients with severe vision loss due to Stargardt disease by restoring broadband light sensitivity,” said Dr. Allen Ho, Professor of Ophthalmology at Thomas Jefferson University and Attending Surgeon, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital.
“We are thrilled to have had Dr. Allen Ho present the Phase 2 STARLIGHT clinical trial data at this year’s ASRS. MCO-010 is advancing our proprietary AAV gene therapy platform with the potential to improve vision for patients with Stargardt disease and other inherited retinal diseases (IRDs). These results offer promising prospects for addressing this challenging disease,” said Sulagna Bhattacharya, Chief Executive Officer & Co-Founder of Nanoscope.
About Stargardt Disease
Stargardt disease is a rare inherited eye disorder caused by fatty deposits in the macula, leading to center field loss of vision. It affects approximately 8,000-10,000 patients in the U.S., often with symptoms beginning in childhood or adolescence. Currently, there are no FDA-approved treatments.
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]
REFERENCE: Ho, et al. MCO-010 optogenetic therapy for severe vision loss in Stargardt disease: 6-month outcomes from the Phase 2 STARLIGHT trial. ASRS 2023.
SOURCE Nanoscope Therapeutics
Nanoscope Therapeutics to Present at the 2023 American Society of Retina Specialists (ASRS) Annual Scientific Meeting
DALLAS, TX (July 21, 2023) — 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 a presentation at the 2023 ASRS Annual Scientific Meeting taking place from July 28-August 1, 2023 in Seattle, Washington. The management team will be in attendance at the conference and accessible for meetings.
Details for the presentation are as follows:
Abstract Title: MCO-010 Optogenetic Therapy for Severe Vision Loss in Stargardt Disease: 6-Month Outcomes from the Phase 2 STARLIGHT Trial
Paper Type: Late Breaking Presentation
Session Title: Hereditary Retinal Disease & Genetics Symposium
Session Date: Monday, July 31, 2023
Session Time: 4:30 PM PT
Presenter: Allen C. Ho, MD, FASRS, Professor of Ophthalmology at Thomas Jefferson University and Attending Surgeon, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital
Dr. Ho will present the safety and efficacy results obtained from the Phase 2 STARLIGHT trial focusing on Stargardt disease. These outcomes will play a vital role in shaping the future clinical development of MCO-010 optogenetic therapy, aimed at restoring vision in patients with Stargardt macular degeneration and other retinal degenerative diseases.
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]
Nanoscope Therapeutics to Participate in the Baird 2023 Biotech Discovery Series
DALLAS, June 12, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Sulagna Bhattacharya, Co-Founder and Chief Executive Officer, Dr. Samarendra Mohanty, Co-Founder and President, and Dr. Victor Hugo Gonzalez, Valley Retina Institute, will participate in a fireside chat at Baird’s Biotech Discovery Series. The fireside chat is scheduled to be held on Friday, June 23, 2023, at 10:30 a.m. ET.
To register for the live webcast, please contact your Baird representative. A replay of the webcast will be available through Baird for a limited time following the event.
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]
Nanoscope Therapeutics Announces Participation in Upcoming Conferences
DALLAS, June 8, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, is scheduled to participate in the following conferences:
Title: The Restore Study: MCO-010 Optogenetic Therapy for Vision Restoration in Retinitis Pigmentosa
Session Date: June 10, 2023
Session Time: 4:43 p.m. MDT
Location: Park City, UT
Presenter: Michael Singer, M.D., Clinical Professor of Ophthalmology at the University of Texas Health Science Center
Dr. Singer will present 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.
Retinal Imaging Biomarkers & Endpoints Summit
Title: Leveraging Retinal Imaging Modalities to Measure Success of Optogenetic Therapy
Session Date: Wednesday, June 14, 2023
Session Time: 2:25 p.m. ET
Location: The Colonnade Hotel, Boston, MA
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer
Dr. Mohanty will lead a discussion on deploying fluorescence-biomarker imaging to quantify opsin expression in retina, and OCT-guided multi-color variable spot ERG for measuring spatially selected and integrated signal improvement due to optogenetic therapy, while leveraging OCT to quantify attenuation of degeneration in optogenetically treated retina, and assessing functional activities of optogenetically-sensitized retinal cells upon light stimulation in a label-free manner.
Panel Discussion: Developing Clinical Endpoints Based on Current Standards of Approval & Steering Guidance for Future Investment
Session Date: Thursday, June 15, 2023
Session Time: 9:00 a.m. ET
Location: The Colonnade Hotel, Boston, MA
Presenter: Dr. Samarendra Mohanty, President and Chief Scientific Officer
On the panel, Dr. Mohanty will highlight the novel endpoints Nanoscope is developing to measure vision function and functional vision.
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
Nanoscope Therapeutics to Present at the BIO International Convention
DALLAS, June 5, 2023 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that management will present at the BIO International Convention, taking place June 5-8, 2023, in Boston, MA. In addition to presenting, management will be available for virtual and in person one-on-one meetings with qualified members of the investor community who are registered to attend the conference.
Details for the presentations are as follows:
Title: Multi-Characteristic Opsins – A new wave of optogenetic therapeutics for vision restoration
Session Date: Wednesday, June 7, 2023
Session Time: 11:45 a.m. ET
Location: Boston Convention & Exhibition Center, Room 104C
Presenter: Sulagna Bhattacharya, Co-Founder and Chief Executive Officer
In her presentation, Ms. Bhattacharya will give an overview of the company, including its proprietary Multi-Characteristic Opsin (MCO) gene therapy platform, currently in development for vision restoration in patients blinded by inherited retinal degenerative diseases, such as retinitis pigmentosa (RP) and Stargardt macular degeneration, regardless of genetic mutations.
Title: Former FDA Reviewers Panel: Perspectives on Cell and Gene Therapy Development from Inside and Outside the FDA
Session Date: Wednesday, June 7, 2023
Session Time: 11:00 a.m. ET
Location: Boston Convention & Exhibition Center, Room 206AB
Presenter: Dr. Massoud Motamed, Vice President of Quality Assurance and Analytics
Dr. Motamed will serve on a panel of former FDA experts in cell and gene therapy and discuss solutions to some of the major challenges faced in the field. Dr. Motamed’s deep FDA experience and his experience with Nanoscope offer an insightful perspective on best practices regarding how to bring breakthrough therapies to patients.
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
Nanoscope Therapeutics Announces Presentations at the World Orphan Drug Congress
DALLAS, May 22, 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 multiple presentations at the World Orphan Drug Congress, taking place May 23-25, 2023 at Gaylord National Resort & Convention center in Washington, DC.
Details for the presentations are as follows:
Title: Pioneering mutation-agnostic gene therapy for retinal disorders
Session Title: Advanced Therapies Clinical Development
Session Date: Wednesday, May 24, 2023
Session Time: 3:20-3:40 PM ET
Location: Theatre 3
Presenter: Samarendra Mohanty, Ph.D., Co-Founder, President & Chief Scientific Officer
Dr. Mohanty will discuss Nanoscope’s mutation-agnostic optogenetic therapy for treatment of various retinal degenerative diseases. Current gene therapies are intended to treat patients with specific gene mutations 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 retinitis pigmentosa (RP) and Stargardt macular degeneration, 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. Efficacy and safety data from the RESTORE and STARLIGHT trials are consistent with those observed in the earlier Phase 1/2 trial (NCT04919473), and thus provide compelling evidence on the benefit/risk profile of MCO-010.
Title: Multi-Characteristic Opsins – A new wave of optogenetic therapeutics for vision restoration
Session Title: Pitch and Partner
Session Date: Thursday, May 25, 2023
Session Time: 1:00 PM ET
Location: Theatre 6
Presenter: Sulagna Bhattacharya, Co-Founder & Chief Executive Officer
Ms. Bhattacharya will provide high level update on Nanoscope’s corporate and development path to commercialize MCO-010 for advanced RP, for which there is presently no approved treatment available. A significant proportion of patients treated with MCO-010 in randomized, double-masked, multicenter RESTORE trial exhibited improvements in functional vision assessed by vision-guided mobility, shape discrimination and visual acuity, along with a favorable safety profile. An observational study (NCT05820100) is ongoing to provide supportive evidence to validate the novel functional vision endpoints.
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
Nanoscope Therapeutics Announces Presentation of Key Results from Phase 2b RESTORE Trial of MCO-010 for the Treatment of Retinitis Pigmentosa at the ARVO Annual Meeting
- 100% (18/18) MCO-010 patients experienced a clinically meaningful improvement in vision-guided mobility (MLYMT), near-field object recognition (MLSDT) or visual acuity (BCVA) (p=0.007 vs sham)
- 94.4% (17/18) MCO-010 patients showed vision improvement in the MLYMT or BCVA (p=0.008 vs sham)
- 88.9% (16/18) of MCO-010 patients experienced a clinically meaningful 2 or more luminance level improvement in MLYMT or MLSDT (p=0.02 vs sham)
- Favorable safety profile for MCO-010 with no serious or severe adverse event
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
[email protected]
SOURCE Nanoscope Therapeutics
Nanoscope Therapeutics Announces Presentations at the ARVO Annual Meeting
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
[email protected]
Nanoscope Therapeutics Announces Positive Topline Results from Phase 2b RESTORE Trial of MCO-010 for the Treatment of Retinitis Pigmentosa
- 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]
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.
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
[email protected]
SOURCE Nanoscope Therapeutics
