Stealth BioTherapeutics Completes Phase 1 Trial of Bevemipretide Eye Drops for Dry AMD
核心洞察
Stealth BioTherapeutics (搜索) has completed enrollment in its Phase 1 dose escalation trial evaluating bevemipretide eye drops for dry age-related macular degeneration.
The trial assessed safety, tolerability, and pharmacokinetics of the mitochondrial-targeted therapeutic in healthy volunteers across single and multiple ascending dose cohorts.
Preliminary results will be presented at the 2026 ARVO Annual Meeting, with a Phase 2 trial planned for late 2026.
Stealth BioTherapeutics (搜索) has completed enrollment in its Phase 1 dose escalation trial evaluating bevemipretide eye drops for the treatment of dry age-related macular degeneration (AMD), marking a significant milestone in the development of a potential noninvasive therapy for this leading cause of blindness in older adults.
The biotechnology company announced that preliminary results from both the single ascending dose (SAD) and multiple ascending dose (MAD) components of the trial will be presented at the 2026 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, scheduled for May 3-7 in Denver, Colorado.
Novel Mitochondrial-Targeted Approach
Bevemipretide, described as a mitochondrial cardiolipin binder, represents Stealth's second-generation investigational mitochondrial-targeted therapeutic candidate. The drug is being developed as an eye drop formulation designed to reach key retinal cells, including the retinal pigment epithelium and photoreceptors, offering a noninvasive treatment approach for dry AMD.
"Vision loss in dry AMD is caused by progressive and irreparable photoreceptor loss, making early intervention critical for protecting vision," said David R. Lally, MD, FASRS, Director of the Retina Research Institute at New England Retina Consultants and Clinical Assistant Professor at the UMass Chan Medical School-Baystate. "A noninvasive eye-drop therapy has the potential to improve patient adoption and adherence earlier in trajectory of this progressive disease."
Phase 1 Trial Design and Results
The Phase 1 trial was conducted in healthy volunteers to determine an appropriate safe and tolerable concentration range of bevemipretide for use in subsequent clinical studies in patients with dry AMD and other ophthalmic conditions.
The study consisted of two parts: Part A included three SAD cohorts of six healthy volunteers each, receiving one drop of topical bevemipretide at concentrations of 1%, 2%, or 3%, or vehicle control, randomized to either the trial eye or fellow eye. Part B comprised three MAD cohorts of eight participants each, randomized in a 3:1 ratio to receive bevemipretide or vehicle into both eyes, administered once or twice daily over 28 days.
Safety assessments and pharmacokinetic samples were analyzed after each cohort to inform dosing regimens for subsequent phases of the study.
Addressing Unmet Medical Need
Dry AMD represents a significant unmet medical need, affecting more than 10 million individuals in the United States. The condition is characterized by symptoms including distorted vision, reduction in low luminance visual acuity, reduced overall visual acuity, and blurred vision. Currently, there are no FDA or EMA approved treatments specifically for dry AMD.
Mitochondrial dysfunction is believed to be a significant contributor to the progression of dry AMD, making the mitochondrial network an attractive therapeutic target to improve retinal function and mitigate disease burden in this patient population.
Development Timeline and Next Steps
Stealth BioTherapeutics (搜索) plans to present the preliminary Phase 1 results at ARVO 2026, with the poster presentation titled "Bevemipretide Eye Drops for Dry AMD: A Phase 1 Dose Escalation Trial to Evaluate Safety, Tolerability, and Pharmacokinetics" scheduled for May 3, 2026, from 8:00 AM to 9:45 AM in the Poster Hall.
The company expects final results from the Phase 1 trial to be presented at the 2026 American Society of Retina Specialists (ASRS) meeting. Based on these data, Stealth is actively working to initiate a Phase 2 trial of bevemipretide eye drops for dry AMD in late 2026.
"Completion of our bevemipretide Phase 1 dose escalation trial is a significant milestone as we work toward initiating development of bevemipretide eye drops for devastating ophthalmic conditions including dry AMD," said Reenie McCarthy from Stealth BioTherapeutics (搜索). "We are committed to advancing innovative mitochondrial-targeted treatments that have the potential to improve outcomes and quality of life for patients."
Company Background
Stealth BioTherapeutics (搜索) is a commercial-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. The company's first commercial product received accelerated approval from the U.S. Food & Drug Administration in September 2025.
Beyond bevemipretide, Stealth is studying elamipretide in additional indications, including dry AMD and primary mitochondrial disease due to nuclear DNA mutations. The company's preclinical pipeline includes candidates SBT-589 and SBT-255 (搜索), both progressing through developmental milestones.