Macular Dystrophy, Retinal, 2

Phase 2/3 of the DRAGON II trial will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan Phase 1b of DRAGON II trial to evaluate pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese subjects was recently completed Tinlarebant granted Orphan Drug and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1 Pivotal global Phase 3 trial of Tinlarebant in GA subjects (“PHOENIX”) is ongoing and more than 200 subjects have been enrolled Sept. 10, 2024 -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” or the “Company”), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the first patient has been dosed at the Tokyo Medical Center in the Phase 2/3 portion of its DRAGON II clinical trial evaluating Tinlarebant for the treatment of STGD1. Tinlarebant is a novel oral therapy designed to reduce the accumulation of vitamin A based toxins that cause retinal disease, and this Phase 2/3 study will evaluate the efficacy, safety, and tolerability of Tinlarebant in approximately 60 adolescent STGD1 subjects across the U.S., U.K., and Japan. “We are pleased to have successfully dosed the first subject in the Phase 2/3 portion of our DRAGON II trial. This milestone is a significant step forward in our mission to address the unmet needs of people living with Stargardt Disease,” said Dr. Tom Lin, Chairman and CEO of Belite Bio. “We recently completed a Phase 1b study of Tinlarebant in STGD1 patients at the Tokyo Medical Center and are happy to be continuing to partner with them for this next trial. With enrollment now also underway at sites in the U.S. and U.K., we are making meaningful progress toward our goal of delivering a life-changing oral therapy to those affected by this condition.” “DRAGON II is the first global Stargardt disease trial in Japan. As such, it provides the unprecedented opportunity for Japanese patients and their families to gain access to a potential therapy for this historically untreatable disease,” added Professor Kaoru Fujinami, Principal Investigator at National Hospital Organization, Tokyo Medical Center. “The level of interest we have received in this trial speaks to the urgency of the unmet need for patients living with STGD1. Following Tinlarebant’s receipt of Sakigake designation from Japan’s Ministry of Health, we feel well positioned to rapidly advance this trial and are working diligently to get this much needed therapy to patients.” The DRAGON II trial is a combination of Phase 1b open-label study in Japan to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of Tinlarebant, administered daily for 7 days in Japanese adolescent STGD1 subjects and a Phase 2/3, multicenter (U.S., U.K., and Japan), double-masked, placebo-controlled, randomized study designed to evaluate the efficacy, safety, and tolerability of Tinlarebant, administered daily for 24 months in adolescent STGD1 subjects. Approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects, are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization (Tinlarebant:placebo). The data from the Japanese subjects is intended to facilitate future NDA applications in Japan. Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S. Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. STGD1 is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, STGD1 and GA, or advanced Dry AMD, share a similar pathophysiology, which is characterized by the excessive accumulation of bisretinoids, retinal cell death, and progressive loss of vision. Vision loss occurs slowly, despite peripheral expansion of “dead retina,” until the disease reaches the center of the eye (the macula). Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt Disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. The content above comes from the network. if any infringement, please contact us to modify.

DALLAS, Sept. 9, 2024 /PRNewswire/ -- Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced presentations at the Retina Society 57th Annual Scientific Meeting and at Advanced Therapies Europe taking place during the 2nd week of September in Lisbon, Portugal. Details for the presentations are as follows: Title: BCVA Analysis of Patients Treated with Low- or High-Dose MCO-010 Mutation Agnostic Optogenetic Therapy for Retinitis Pigmentosa: 100-week TOPLINE Results from a Phase 2b/3 Randomized, Sham-Controlled Clinical Trial (RESTORE) Session Title: Genetic Diseases, Dystrophies & Degenerations Session Date: September 11, 2024 Session Time: 4:25 p.m. WEST Location: Pedro Leitao Ballroom, Four Seasons Hotel Ritz, Lisbon Presenter: Anat Loewenstein, MD, MHA, Professor and Director, Division of Ophthalmol., Tel Aviv University In her presentation, Dr. Loewenstein will highlight the end-of-study 100 week RESTORE data from mutation-agnostic Multi-Characteristic Opsin (MCO) therapy of severe vision loss due to retinitis pigmentosa. Title: Democratizing Gene Therapy for Neurodegenerative Diseases Session Title: Gene Therapy: Spotlight on Developments and Breakthroughs Session Date: September 11, 2024 Session Time: 4:15 p.m. WEST Location: Estoril Convention Center, Lisbon Presenter: Samarendra Mohanty, PhD, President & Chief Scientific Officer During his presentation, Dr. Mohanty will highlight the recent developments and breakthroughs on mutation-agnostic optogenetic gene therapy for vision restoration in inherited retinal dystrophies, and image-guided spatially targeted laser gene delivery platform for treating geographic atrophy. The presentation will be followed by a panel discussion at 4:45 pm. Dr. Mohanty will also chair a session titled "Flexibility in Action: Harnessing Adaptive Vector Platforms for Gene Therapy Manufacturing" on September 12, 2024, from 1:45 - 3: 00 p.m. WEST. 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 completed 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 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

FRIDAY, Sept. 6, 2024 -- Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report. The illness is caused by mutations in the GUCY2D gene, which is critical to producing proteins that enable vision. People with this form of Leber congenital amaurosis (LCA1) typically lose their vision in early childhood. Just under 100,000 people are thought to be affected worldwide. However, a team of researchers at the University of Pennsylvania say that a new gene therapy injected under the retinas of children and adults with LCA1 improved vision by 100 to 10,000 times. “Even though we previously predicted a large vision improvement potential in LCA1, we did not know how receptive patients’ [eye] photoreceptors would be to treatment after decades of blindness,” noted study co-lead author Artur Cideciyan , a research professor of ophthalmology at the university. “It is very satisfying to see a successful multi-center trial that shows gene therapy can be dramatically efficacious.” The findings were published Sept. 5 in The Lancet journal. A total of 15 LCA1 patients -- 12 adults and three children -- participated in the new trial. All had vision that was equal to or below 20/80, meaning that objects a normally sighted person could see at 80 feet could only be clear at 20 feet away for a person with LCA1. Glasses were of little help, because glasses only address the eye's focusing ability, not any underlying medical cause driving vision loss. The new gene therapy is called ATSN-101 and was developed by Atsena Therapeutics, which funded the new trial. This was a small phase 2 trial, meant to gauge a treatment's safety and explore differences when various dosages are used. The injected therapy was tried at three different dosages. Three adult patients each received low, medium and high doses of ATSN-101. Once the treatments' safety was assessed, six participants (including the three children) received high-dose ATSN-101. According to the research team, vision began to improve quickly in all patients, usually within a month of treatment. Improvements have lasted for at least a year. Patients' vision was tested in various ways, including their ability to move through a sometimes dimly-lit pathway, eye charts and "light flash" tests conducted in a dark environment. Two of nine patients who got the high-dose treatment had a 10,000-fold improvement in their sight, Cideciyan's group reported. “That 10,000-fold improvement is the same as a patient being able to see their surroundings on a moonlit night outdoors as opposed to requiring bright indoor lighting before treatment,” said Cideciyan, who also co-directs the Center for Hereditary Retinal Degenerations at UPenn. “One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire.” Side effects could happen, and were usually related to the surgery used to implant the gene therapy. These included tiny hemorrhages within the eye that later healed, or transient inflammation of the eye, treated with steroids. The patients' vision is being tracked going forward, the researchers said, and a bigger, randomized trial is being planned as a requirement of any U.S. Food and Drug Administration approval of the new therapy. The new work follows on the team's prior success using gene therapy to reverse another form of Leber congenital amaurosis, this time connected to a mutation in the CEP290 gene. “The treatment success in our most recent clinical trials together with our earlier experience brings hope for a viable treatment for about 20 percent of infantile blindness caused by inherited retinal degenerations,” study co-author Dr. Tomas Aleman said in a UPenn news release. He is research professor in ophthalmology and co-director of the Center for Hereditary Retinal Degenerations. “The focus now is on perfecting the treatments and treating earlier manifestations of these conditions once safety is confirmed," he said. "We hope similar approaches will lead to equally positive outcomes in other forms of congenital retinal blindness.” Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.