AMERICAN ACADEMY OF OPHTHALMOLOGY
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Established
- 1979-01-01
- Employees
- -
- Market Cap
- -
- Website
- http://www.aao.org/
Annexon, Inc. Advances in Neuroinflammatory Disease Treatments
Annexon, Inc. (ANNX) has made significant progress in developing treatments for neuroinflammatory diseases, with recent announcements including the expansion of its board of directors, positive results from clinical trials, and participation in major healthcare conferences.
Gildeuretinol Shows Promise in Preventing Stargardt Disease Progression in Early-Stage Patients
• Interim data from the TEASE-3 study reveals that oral gildeuretinol acetate prevented disease progression in early-stage Stargardt patients over multiple years.
• The study demonstrated stable visual acuity in patients treated with gildeuretinol, highlighting its potential to preserve vision in early-stage Stargardt disease.
• Alkeus Pharmaceuticals plans to submit an NDA to the FDA in 2025 for gildeuretinol as a treatment for Stargardt disease, addressing a critical unmet need.
• TEASE-3 is the first clinical trial to show no disease progression in early-stage Stargardt patients over multiple years, including preservation of visual acuity.
Acelyrin's Lonigutamab Shows Promise in Phase 2 Data for Thyroid Eye Disease
• Acelyrin's lonigutamab demonstrates a potential best-in-class efficacy and safety profile for treating Thyroid Eye Disease (TED), according to updated Phase 2 data.
• A virtual investor event on January 6, 2025, will feature clinician perspectives on unmet needs in TED and the design of the Phase 3 LONGITUDE program.
• The Phase 3 LONGITUDE program, developed after discussions with the FDA, aims to be the most inclusive registrational program in TED to date.
• Lonigutamab, a subcutaneously delivered monoclonal antibody targeting IGF-1R, offers potential for longer-term, convenient dosing, improving clinical response.
Ocugen's OCU400 Shows Promise in Retinitis Pigmentosa Treatment with Positive Clinical Data and Regulatory Milestones
• Ocugen's OCU400 gene therapy demonstrated a statistically significant improvement in low-luminance visual acuity in patients with retinitis pigmentosa (RP).
• The European Medicines Agency (EMA) has granted OCU400 an Advanced Therapy Medicinal Product (ATMP) classification, expediting its regulatory review process.
• Phase 1/2 trial results showed that 100% of treated patients experienced improved or preserved visual function over two years, regardless of the underlying genetic mutation.
• Ocugen's Phase 3 liMeliGhT clinical trial is ongoing, with plans for BLA/MAA submissions in the U.S. and Europe anticipated in the first half of 2026.
Eylea HD Shows Promise in Macular Edema Following Retinal Vein Occlusion
• Eylea HD (aflibercept 8 mg) demonstrated non-inferior visual acuity gains compared to Eylea (aflibercept 2 mg) in patients with macular edema following retinal vein occlusion.
• The Phase 3 QUASAR trial showed that Eylea HD, dosed every 8 weeks, achieved similar vision improvements to Eylea, dosed every 4 weeks.
• A significant portion of patients on Eylea HD were able to maintain an 8-week dosing regimen, potentially reducing the burden of frequent injections.
• Regeneron plans to submit these data to regulatory authorities, seeking approval for Eylea HD in this indication, offering a less frequent treatment option.
EMA Investigates Potential Link Between Semaglutide and Rare Eye Condition NAION
• The European Medicines Agency (EMA) is reviewing semaglutide, the active ingredient in Ozempic and Wegovy, following reports of a potential link to non-arteritic anterior ischemic optic neuropathy (NAION).
• The review was prompted by two studies from the University of Southern Denmark suggesting a doubled risk of NAION in patients using semaglutide.
• NAION is a rare eye condition involving reduced blood flow to the optic nerve, potentially leading to vision loss, and the EMA will assess all available data to determine if there is a causal relationship.
• The investigation follows increasing concerns and reports of NAION in semaglutide users, with the EMA's Pharmacovigilance Risk Assessment Committee (PRAC) evaluating the evidence.
PolyActiva's PA5108 Glaucoma Implant Demonstrates Promising IOP Reduction in Phase 2 Trial
• PolyActiva's PA5108 implant showed significant IOP reduction over 26 weeks in a Phase 2 trial, meeting key efficacy and safety endpoints.
• The implant, designed for sustained latanoprost delivery, addresses patient adherence issues by providing consistent medication over an extended period.
• Clinical trials suggest minimal adverse effects, with no evidence of inflammation or corneal endothelial cell changes observed during the study.
• A Phase 2b study is planned in the US, with Phase 3 trials expected to begin soon, potentially leading to market availability by 2029.
ABBV-RGX-314 Demonstrates Potential as One-Time Treatment for Bilateral Wet AMD in Phase 2 Study
• Phase 2 study of ABBV-RGX-314 shows a 97% reduction in annualized anti-VEGF treatment burden in patients with bilateral wet AMD.
• 78% of patients were completely injection-free at 9 months post-administration of ABBV-RGX-314 in the fellow eye.
• The study demonstrated sustained best-corrected visual acuity (BCVA) and central retinal thickness (CRT) at 9 months.
• ABBV-RGX-314 was well-tolerated, with no drug-related serious adverse events observed in the treated fellow eye.
Stem Cell Therapy Shows Promise in Early Trial for Retinitis Pigmentosa
• A phase 1 clinical trial at UC Davis Health demonstrated the safety of injecting CD34+ stem cells into the eyes of retinitis pigmentosa (RP) patients.
• The study confirmed the feasibility of isolating high-quality CD34+ cells from patient's bone marrow for therapeutic use in RP.
• Four out of seven patients experienced measurable improvements in vision, suggesting potential therapeutic benefits of the stem cell therapy.
• Researchers plan to conduct larger trials to further evaluate the safety and efficacy of CD34+ stem cell therapy for retinitis pigmentosa.
FDA Issues Complete Response Letter for Astellas' Avacincaptad Pegol; Aldeyra's Reproxalap NDA Accepted
• The FDA issued a Complete Response Letter for Astellas Pharma's avacincaptad pegol intravitreal solution, indicating the sNDA cannot be approved in its current form.
• Aldeyra Therapeutics' resubmitted NDA for topical ocular reproxalap, a first-in-class investigational candidate for dry eye disease, has been accepted by the FDA.
• Aldeyra Therapeutics is expanding its option agreement with AbbVie, securing additional funding to support the commercialization of reproxalap.
• Ocular imaging in emergency departments, utilizing non-mydriatic fundus cameras, enhances timely and accurate diagnoses, benefiting both patients and on-call ophthalmologists.
Gildeuretinol Receives FDA Rare Pediatric Disease and Fast Track Designations for Stargardt Disease
• Gildeuretinol (ALK-001) has been granted Rare Pediatric Disease and Fast Track designations by the FDA for treating Stargardt disease, an inherited retinal disease.
• The designations were based on data from the TEASE-1 trial, which demonstrated a statistically significant slowing of lesion growth in late-stage Stargardt disease patients.
• Interim data from the TEASE-3 study indicated that early-stage Stargardt disease patients treated with gildeuretinol showed no disease progression over 2-6 years.
• Alkeus Pharmaceuticals plans to apply for a Priority Review voucher upon submission of the New Drug Application (NDA) for gildeuretinol.
Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) received Rare Pediatric Disease and Fast Track Designations from the FDA for Stargardt disease treatment.
• TEASE-1 trial data showed a 21.6% reduction in retinal atrophic lesion growth rate with gildeuretinol compared to the untreated arm.
• TEASE-3 data suggests early treatment with gildeuretinol may prevent progressive central vision loss in Stargardt disease patients.
• Alkeus Pharmaceuticals' CEO highlights these designations as recognition of the significant unmet need in Stargardt disease.
Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) has been granted Rare Pediatric Disease and Fast Track designations by the FDA for treating Stargardt disease, a rare inherited retinal disease.
• Clinical data from the TEASE-1 trial showed gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients (p < .001).
• Interim data from the TEASE-3 study suggests early treatment with gildeuretinol may prevent disease progression and maintain visual acuity in early-stage Stargardt patients.
• Stargardt disease affects an estimated 30,000 to 87,000 people in the US, and gildeuretinol is being developed to reduce vitamin A dimerization, a key factor in the disease.
Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) receives FDA Rare Pediatric Disease and Fast Track designations for Stargardt disease, a progressive condition causing severe vision loss.
• Clinical data from the TEASE-1 study showed gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients.
• Interim data from the TEASE-3 study indicated that early-stage Stargardt disease patients treated with gildeuretinol showed no disease progression over 2-6 years.
• Gildeuretinol has Breakthrough Therapy and Orphan Drug designations, highlighting its potential as a groundbreaking therapy for Stargardt disease.
Alkeus Pharmaceuticals' Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) receives Rare Pediatric Disease and Fast Track designations from the FDA, highlighting the urgent need for Stargardt disease treatments.
• TEASE program data presented at the American Academy of Ophthalmology showed gildeuretinol slowed the growth rate of atrophic retinal lesions in Stargardt patients.
• Early-stage Stargardt patients treated with gildeuretinol in the TEASE-3 study showed no disease progression and stable visual acuity over two to six years.
• Alkeus plans to apply for a Priority Review Voucher, potentially expediting the development of other clinical programs.
Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) received Rare Pediatric Disease and Fast Track designations from the FDA for treating Stargardt disease, a progressive condition causing vision loss.
• Clinical data from the TEASE-1 study demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients (p<0.001).
• Interim data from the TEASE-3 study showed that early-stage Stargardt disease patients treated with gildeuretinol experienced no disease progression over 2-6 years.
• Gildeuretinol has Breakthrough Therapy and Orphan Drug designations, potentially expediting its development and review for this condition with no approved treatments.
Gildeuretinol Receives FDA Rare Pediatric Disease and Fast Track Designations for Stargardt Disease
• Gildeuretinol (ALK-001) has been granted Rare Pediatric Disease and Fast Track designations by the FDA, highlighting the urgent need for Stargardt disease treatments.
• Clinical data from the TEASE program demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions in Stargardt disease patients.
• Early-stage Stargardt disease patients treated with gildeuretinol in the TEASE-3 study showed no disease progression and stable visual acuity over two to six years.
• Alkeus plans to apply for a Priority Review Voucher, potentially expediting the development of other clinical programs.
Gildeuretinol Receives FDA Fast Track and Rare Pediatric Disease Designations for Stargardt Disease
• Gildeuretinol (ALK-001) receives Rare Pediatric Disease and Fast Track designations from the FDA for Stargardt disease treatment, highlighting its potential as a groundbreaking therapy.
• Clinical data from the TEASE program demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients.
• Early-stage Stargardt disease patients treated with gildeuretinol in the TEASE-3 study showed no disease progression and maintained stable visual acuity.
• Gildeuretinol has a favorable safety profile, with no adverse events related to hyper- or hypo-vitaminosis A reported in clinical trials.
AAO 2024 Highlights: Aging Clinicians, Presbyopia Drop, Modern Anesthesia, and AMD Advancements
• Alfredo Sadun, MD, addressed the challenges of aging clinicians in ophthalmology, highlighting the complexities of assessing competency and avoiding ageism.
• Jennifer Loh, MD, presented data on CSF-1, a low-dose pilocarpine drop, demonstrating a 30% mean reduction in pupil size and improved near visual acuity without affecting distance vision.
• Brent Kramer, MD, discussed the benefits of the MKO Melt for cataract surgery, emphasizing improved patient and team experience due to its opioid-free, needle-free approach.
• Researchers at Wilmer Eye Institute found that increased ANGPTL4 may counteract anti-VEGF therapy in wet AMD and that experimental drug 32-134D could enhance treatment efficacy.
OTX-TKI Implant Shows Promise in Reducing Injection Frequency for Wet AMD
• A phase I study of OTX-TKI, an intravitreal implant containing axitinib, demonstrated a significant reduction in injection frequency for wet AMD patients.
• The OTX-TKI implant resulted in a nearly 90% reduction in injection burden compared to aflibercept over a 12-month period.
• Visual acuity and central subfield thickness outcomes were stable with OTX-TKI, showing comparable efficacy to aflibercept.
• Phase III trials (SOL-1 and SOL-R) are underway to assess the durability, safety, and efficacy of repeat dosing of OTX-TKI.
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