Alkeus Pharmaceuticals Appoints Carlos Quezada-Ruiz as Chief Medical Officer to Lead Stargardt Disease Drug Development
核心洞察
Alkeus Pharmaceuticals (搜索) has appointed renowned retina specialist Carlos Quezada-Ruiz, M.D., F.A.S.R.S., as Chief Medical Officer to lead clinical development of gildeuretinol for Stargardt disease (搜索).
The company is accelerating preparations for a global Phase 3 registrational study of gildeuretinol, an oral therapy with breakthrough designation targeting ABCA4 (搜索)-related retinal degeneration.
Dr. Quezada-Ruiz brings extensive experience from 4D Molecular Therapeutics and Genentech/Roche, where he led development of treatments for inherited and neovascular retinal diseases.
Alkeus Pharmaceuticals (搜索) announced the appointment of internationally recognized ophthalmologist Carlos Quezada-Ruiz, M.D., F.A.S.R.S., as Chief Medical Officer, positioning the Cambridge-based biopharmaceutical company to advance its breakthrough-designated therapy for Stargardt disease (搜索) into Phase 3 clinical trials.
Dr. Quezada-Ruiz succeeds Seemi Khan, M.D., M.P.H., M.B.A., who has been promoted to the newly created role of Chief Development and Strategy Officer. The leadership transition comes as Alkeus accelerates preparations for a global Phase 3 registrational study of gildeuretinol in patients with advanced Stargardt disease (搜索).
Strategic Leadership for Rare Disease Development
"Dr. Quezada-Ruiz has extensive experience and a strong track record as a cross-functional leader bringing forward groundbreaking therapies that have helped transform the field of modern ophthalmology," said Michel Dahan, President and Chief Executive Officer of Alkeus Pharmaceuticals (搜索). "His strategic vision, profound clinical insight and passion for evidence-based innovation in ophthalmology will help us unlock the full potential of breakthrough-designated gildeuretinol."
Prior to joining Alkeus, Dr. Quezada-Ruiz served as Senior Vice President and Ophthalmology Therapeutic Area Head at 4D Molecular Therapeutics (4DMT), where he directed early- and late-stage gene-therapy programs for inherited and neovascular retinal diseases. His experience includes leading trial design and global regulatory alignment for the company's lead candidate.
Previously, Dr. Quezada-Ruiz held senior leadership positions at Genentech/Roche, where he played a pivotal role in the global development and regulatory approvals of treatments for neovascular (wet) AMD, diabetic macular edema (搜索) (DME) and diabetic retinopathy (搜索) (DR).
Addressing Unmet Medical Need in Stargardt Disease
"There is a significant unmet need for safe, effective, and convenient treatment options that can help preserve vision for people living with Stargardt disease (搜索)," Dr. Quezada-Ruiz said. "As a practicing retina specialist, I have seen firsthand the impact of progressive vision loss and the suffering it causes for patients with Stargardt disease and their families."
The new Chief Medical Officer emphasized the clinical potential of gildeuretinol, describing it as "an investigational oral therapy designed to target a primary driver of ABCA4 (搜索)-related retinal degeneration, with the goal of helping preserve retinal structure and stabilize visual function for people living with this inherited condition around the world."
Gildeuretinol's Regulatory Progress
Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. The therapy has received multiple regulatory designations from the U.S. Food and Drug Administration (FDA), including Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations for Stargardt disease (搜索).
The European Medicines Agency (EMA) has designated gildeuretinol as an orphan medicinal product for the treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 (搜索) gene, which includes Stargardt disease (搜索).
Building on TEASE Clinical Program
The upcoming Phase 3 study builds on the company's TEASE (Tolerability and Effects of ALK-001 on Stargardt diseasE (搜索)) clinical program, which consists of four independent clinical studies. TEASE-1 was a randomized, double-masked, placebo-controlled trial in patients with advanced Stargardt disease, while TEASE-2 was conducted in patients with moderate disease. Both studies have been completed.
TEASE-3 (NCT02402660) is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early-stage Stargardt disease (搜索) who have early signs of disease visible on retinal imaging but have not begun experiencing symptoms of vision loss. TEASE-4 (NCT04239625) serves as an open-label extension study.
Industry Recognition
Philip J. Ferrone, M.D., of Vitreoretinal Consultants of New York, praised the appointment: "Alkeus Pharmaceuticals (搜索) has made significant strides in innovative approaches to target a key mechanism underlying Stargardt disease (搜索), and the appointment of an internationally recognized expert like Dr. Quezada-Ruiz underscores the company's long-term, strong commitment to the retina community."
Dr. Quezada-Ruiz serves as Assistant Professor of Ophthalmology at the Instituto de Oftalmología Fundación Conde de Valenciana in Mexico City and as Chairman of the Scientific Committee of the Mexican Retina Society. He also serves as a scientific advisor to selected biotechnology companies advancing novel therapies and technologies for retinal diseases.
Organizational Growth
Dr. Khan, who served as Chief Medical Officer since the beginning of 2024, built the clinical foundation and development plans that positioned Alkeus to advance gildeuretinol into Phase 3 trials. "Since joining Alkeus two years ago, Dr. Khan has played an instrumental role in the transformation of Alkeus and in the development of our clinical and regulatory plans," Dahan said.
Alkeus Pharmaceuticals (搜索) is a private biopharmaceutical company backed by institutional investors led by Bain Capital Life Sciences (搜索), dedicated to preserving the sight of individuals impacted by retinal diseases.