Priority review granted to possible LHON treatment

Chiesi Global Rare Diseases announced that the FDA has granted priority review for its regulatory submission for idebenone for treatment of Leber hereditary optic neuropathy. The target action date for a decision is February 28, 2026. According to the company’s press release, if approved, idebenone would be the “first and only clinically proven therapy for LHON in the United States.” The treatment is already available in several countries outside the U.S., marketed under the brand name Raxone. A Phase 3 study evaluating the therapy found statistically significant improvements in overall change in best visual acuity and change in visual acuity in the best eye with idebenone compared to placebo. Post-hoc analysis of responders found that a higher proportion of patients in the idebenone group had clinically meaningful improvements in visual acuity as well, according to the press release.

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Update on drug in development for neuropathic corneal pain

Okyo Pharma shared that it established a registration pathway, the next stage of clinical development, for its lead drug candidate, urcosimod, for treatment of neuropathic corneal pain. The company is developing a multiple-ascending dose clinical trial that will enroll about 100 patients with neuropathic corneal pain at multiple U.S. sites. The trial will be randomized, placebo-controlled, and double masked with the intent of identifying the registration dose for urcosimod for future Phase 3 trials. The topline results from the ascending dose trial are expected in 2026.

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FDA approves generic bimatoprost

Amneal Pharmaceuticals announced FDA approval of the Abbreviated New Drug Application for its bimatoprost ophthalmic solution 0.01%, indicated for IOP reduction in patients with open-angle glaucoma or ocular hypertension. The product is considered a generic equivalent to LUMIGAN (AbbVie).

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Phase 1/2a clinical results for first-in-human retinal pigment epithelial stem cell therapy

Luxa Biotechnology announced publication of data from its Phase 1/2a first-in-human clinical trial for retinal pigment epithelial (RPE) stem cells as a therapy for patients with dry AMD. The low-dose cohort (50,000 cells) in this study evaluating RPESC-RPE-4W found that patients who had severe vision loss gained an average of 21.67 ETDRS letters, which was maintained through 12 months, according to the company’s press release. Patients who had better baseline vision gained 3 letters at 6 months. In terms of safety, the company reported no tumor formation, no severe inflammation, or other serious therapy-related adverse events. The data is published in the journal Cell Stem Cell.

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Adult dosing complete in trial for X-linked retinoschisis therapy

Atsena Therapeutics announced that it completed dosing of the adults enrolled in part B of its Phase 1, 2, 3 trial evaluating subretinal injection of ATSN-201 for X-linked retinoschisis. The preliminary data from the adult cohort will inform dosing of pediatric patients, which is expected to begin in the fourth quarter of this year. Nine adults were enrolled in Part B of this ongoing, multicenter, clinical trial, receiving either low volume ATSN-201, high volume ATSN-201, or control. Patients in the control group will be observed for 1 year and then have the option to receive ATSN-201 treatment.

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Company news

• Optos is collaborating with Toku to bring the latter company’s BioAge into its Optos Daytona, Monaco, MonacoPro, and California devices in the U.S. This AI solution estimates a person’s biological age by evaluating vascular and metabolic indicators in the retina, according to the Optos press release. This, with the ultra-widefield retinal images, will allow eyecare providers to provide patients with information about both their ocular health and systemic wellness, according to the company.

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ASCRS news and events

• Business of Refractive Cataract Surgery (BRiCS) Summit: This course, taking place October 3–5, in Chicago, Illinois, along with its extensive, take-home Resource Library, will help practices gain the navigational tools needed to increase premium IOL adoption and lead patients effectively on their refractive surgery journey. Learn more.
• ASCRS Live!: This educational dinner series is heading to several cities across the U.S. in 2025. Registration is open for the next event in Orlando on October 16.
• 2026 ASCRS Annual Meeting: Hotel room blocks are open for the 2026 ASCRS Annual Meeting, taking place in Washington, D.C., April 10–13, 2026.

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Research highlights

• The prevalence and patient/surgeon characteristics for IOL exchange in the U.S., as well as surgical complication rates, among Medicare beneficiaries was the subject of a retrospective cross-sectional analysis published in the Journal of Cataract & Refractive Surgery. The study included data from Medicare beneficiaries (48,967 patients) who had IOL exchange (52,583 exchanges) between 2011 and 2020, as well as complete demographic information and follow-up data. The authors found that the number of exchanges increased over the study timeframe, from 4,621 in 2011 to 6,114 in 2019. According to the study, most patients were white and lived in cities. Similarly, most of the surgeons practiced in cities; most were also male and had an average of 24.7±11.2 years between medical school and the last surgery performed in the study period. The most common reasons for IOL exchange were lens failure (51.4% of cases) and lens displacement (21.1%). Complications that occurred during the IOL exchange procedure were epiretinal membrane (6.8%), mechanical lens complication (5.9%), and replacement IOL dislocation (4.9%). The authors noted in the conclusion that rates of corneal decompensation, retinal detachment, and endophthalmitis were low in IOL exchange procedures.
• A randomized clinical trial published in JAMA Ophthalmology evaluated the benefit of adjunctive topical corticosteroids (difluprednate) and/or crosslinking with riboflavin, in addition to topical antibiotics, on outcomes for treatment of bacterial keratitis. Patients (n=280) with smear-positive bacterial corneal ulcers were randomized into three groups: 1) topical moxifloxacin 0.5%, with topical placebo plus sham crosslinking; 2) topical moxifloxacin 0.5%, difluprednate 0.05%, and sham crosslinking; and 3) topical moxifloxacin 0.5%, difluprednate 0.05%, and crosslinking. The authors reported, after controlling for baseline, there was no difference in visual acuity at 6 months with the addition of topical steroids with crosslinking or topical steroids alone, compared to placebo. Scar size was not improved with the addition of topical steroids either, and scar size increased in the group that received crosslinking and steroids. The authors concluded that “adjunctive topical corticosteroids were not superior to placebo, and adjunctive CXL had increased scar size, suggesting that these alternative therapies, on average, may not be superior to topical antibiotics alone for treatment of bacterial keratitis.”

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Product news

• Topcon Healthcare launched the TERA Dry Eye Imager, which can detect, grade, and manage dry eye disease, according to the company, with high-resolution imaging, validated clinical grading scales, assessment of both signs and symptoms, and guidance for treatment and follow-up.

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