AAO 2024: Emerging Data in Retinal Therapies for Geographic Atrophy, Stargardt Disease, and Retinitis Pigmentosa
核心洞察
Alkeus Pharmaceuticals will present results from clinical studies of oral gildeuretinol for geographic atrophy (搜索) secondary to age-related macular degeneration (SAGA) and Stargardt disease (搜索) (TEASE).
Annexon will share new analyses of ANX007 from the Phase 2 ARCHER trial in geographic atrophy (搜索), highlighting clinical results and anatomical changes.
Nanoscope Therapeutics (搜索) is set to share topline data from the RESTORE trial of MCO-010 therapy in patients with severe vision loss due to retinitis pigmentosa (搜索) (RP).
The American Academy of Ophthalmology (AAO) 2024 meeting in Chicago is set to showcase a range of emerging data from clinical trials focused on retinal therapies. Several companies are preparing to present updates on investigational treatments for diseases including geographic atrophy (搜索) (GA), Stargardt disease (搜索), X-linked retinoschisis (搜索) (XLRS) and retinitis pigmentosa (搜索) (RP).
Alkeus Pharmaceuticals: Gildeuretinol for Geographic Atrophy and Stargardt Disease
Alkeus Pharmaceuticals will present findings from its clinical studies of gildeuretinol, an investigational oral therapy, for both geographic atrophy (搜索) secondary to age-related macular degeneration (SAGA study) and Stargardt disease (搜索) (TEASE study). The SAGA study is a 2-year, randomized, double-masked, placebo-controlled trial. The TEASE study investigates the potential of deuterated vitamin A to preserve vision in Stargardt disease.
Annexon: ANX007 for Geographic Atrophy
Annexon will present new analyses of ANX007 from the completed Phase 2 ARCHER trial in geographic atrophy (搜索) (GA). ANX007 is a first-in-kind, non-pegylated antigen-binding fragment (Fab) designed to block C1q (搜索) locally in the eye via intravitreal formulation. The presentation will cover clinical results and anatomical changes observed in the ARCHER trial.
Atsena Therapeutics: ATSN-201 for X-Linked Retinoschisis
Atsena Therapeutics (搜索) will present safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (搜索) (XLRS). ATSN-201 utilizes AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina, aiming to avoid surgical risks associated with foveal detachment.
Avirmax Biopharma: Gene Therapy Programs for Ocular Diseases
Avirmax Biopharma (搜索) will showcase the progress of its ABI gene therapy ocular programs. Their lead candidate, ABI-110 for wet AMD, is currently enrolling patients for Phase I/IIa trials in the US (NCT06550011). Preclinical data has demonstrated improved tropism to the macular retina compared to wildtype serotypes and AAV2.7m8 in animal models. Additionally, Avirmax is developing ABI-201 programs for geographic atrophy (搜索) (GA), dry AMD, proliferative diabetic retinopathy (PDR), and the ABI-902 pipeline targeting multiple types of glaucoma.
Beacon Therapeutics: AGTC-501 for X-Linked Retinitis Pigmentosa
Beacon Therapeutics (搜索) will present 24-month interim safety and efficacy results from the Phase 2 SKYLINE trial of AGTC-501 in patients with X-linked retinitis pigmentosa (搜索) (XLRP). The data showed a 57% response rate (4/7) in study eyes treated with a high dose (6.8 E+11 vg/eye) of AGTC-501, defined as an improvement in retinal sensitivity of at least 7 decibels (dB) in at least 5 loci, as assessed by microperimetry. The high-dose cohort also exhibited a robust improvement in mean retinal sensitivity. The low-dose cohort (7.5 E+10 vg/eye) showed a 25% response rate (1/4). No clinically significant safety events were reported.
Belite Bio: Tinlarebant for Adolescent Stargardt Disease
Belite Bio (搜索) will present data from its completed Phase II trial of tinlarebant (LBS-008) in adolescents with Stargardt disease (搜索).
Eyenovia: Clobetasol Propionate for Post-Surgical Inflammation and Pain
Eyenovia will present results from the successful Phase 3 study (CPN-302) of clobetasol propionate suspension 0.05% (APP13007), which led to its FDA approval for inflammation and pain following ocular surgery. The company launched clobetasol in the US on September 26, 2024.
LENZ Therapeutics: LNZ100 for Presbyopia
LENZ Therapeutics (搜索) will present on LNZ100, a preservative-free, single-use, once-daily eye drop containing aceclidine, as a potential therapy for the treatment of presbyopia. The candidate met all primary and secondary endpoints in the registration-enabling Phase 3 CLARITY study.
Nanoscope Therapeutics: MCO-010 for Retinitis Pigmentosa
Nanoscope Therapeutics (搜索) is set to share end-of-study topline data from the RESTORE trial, a randomized-controlled trial (RCT) of MCO-010 therapy in patients with severe vision loss due to retinitis pigmentosa (搜索) (RP). The company reports that RESTORE is the first RCT to demonstrate statistically significant visual acuity improvement in this patient population and is a key component of their upcoming BLA submission for the RP program.
ZEISS: Digital Enhancements and Surgical Solutions
ZEISS (搜索) Medical Technology will showcase digital enhancements and surgical solutions at the AAO conference, focusing on innovations across cataract, corneal refractive, retina, and glaucoma workflows.