Floretina ICOOR 2024: Updates on Stargardt Disease, AMD, and Wet AMD Therapies
核心洞察
Alkeus Pharmaceuticals (搜索) shared results from TEASE studies, showing that gildeuretinol (ALK-001) significantly reduced the growth rate of retinal atrophic lesions in Stargardt disease (搜索) patients.
Annexon presented data from the Phase 2 ARCHER trial, indicating that ANX007 provides significant vision protection and photoreceptor preservation in dry AMD patients with geographic atrophy (搜索).
Opthea highlighted sozinibercept, currently in Phase 3 trials, as a potential therapy to enhance vision acuity gains in wet AMD (搜索) when combined with anti-VEGF-A (搜索) agents.
Ophthalmic companies presented updates from recent and ongoing trials at the Floretina International Congress on OCT Angiography, En Face OCT and Advances in OCT (ICOOR) 2024, held in Florence, Italy. The meeting, which took place from December 5-8, brought together clinicians and industry to discuss advancements in retina care.
Alkeus Pharmaceuticals: Gildeuretinol for Stargardt Disease
Alkeus Pharmaceuticals (搜索), Inc. presented findings from the Tolerability and Effects of ALK-001 on Stargardt diseasE (搜索) (TEASE) studies, which are evaluating oral gildeuretinol (ALK-001) for Stargardt disease. The TEASE-1 study demonstrated that gildeuretinol was well-tolerated and met its primary efficacy endpoint. Results showed a 21.6% reduction in the growth rate of retinal atrophic lesions area (square root) (P < .001), and a 29.5% reduction for untransformed areas of retinal atrophic lesions compared to untreated patients. The ongoing TEASE-2 trial is expected to release topline data in 2025. TEASE-3 and TEASE-4 are open-label studies, with TEASE-3 focusing on early-stage, genetically confirmed Stargardt disease patients without vision loss symptoms, and TEASE-4 serving as an open-label extension study.
Annexon: ANX007 for Dry AMD with Geographic Atrophy
Annexon shared key details from the Phase 2 ARCHER trial of its complement therapy ANX007, which is being evaluated for preventing visual acuity loss and preserving photoreceptors in patients with dry age-related macular degeneration (搜索) (AMD) and geographic atrophy (搜索) (GA). The presentation highlighted findings in patients with less advanced disease. ANX007, administered intravitreally, is a non-pegylated antigen-binding fragment designed to block C1q (搜索) locally in the eye. Ongoing trials have shown that ANX007 provides significant vision protection in both standard and low light conditions, along with significant preservation of photoreceptors in the fovea region, which is critical for visual acuity. Annexon is currently enrolling patients for the pivotal Phase 3 ARCHER II trial in GA, with data expected in the second half of 2026.
Opthea: Sozinibercept for Wet AMD
Opthea Limited announced that its lead product candidate, sozinibercept, was featured in an oral presentation on emerging therapies for wet AMD (搜索). Adnan Tufail, MBBS, MD, FRCOphth, presented on "Beyond VEGF-A (搜索) inhibition: Will blocking VEGF-C and VEGF-D lead to better outcomes?" Sozinibercept is currently being evaluated in two pivotal Phase 3 clinical trials, COAST (NCT04757636) and ShORe (NCT04757610). It is being investigated for use in combination with anti-VEGF-A therapies to improve overall efficacy and enhance vision acuity gains compared to standard-of-care anti-VEGF-A agents alone.