• Five Medicare Administrative Contractors (MACs) retired their local coverage determinations (LCDs) for minimally invasive glaucoma surgery (MIGS) that were scheduled to take effect on Jan. 29, 2024. The five MACs — WPS Government Health Administrators, Palmetto GBA, Celerian Group Company, National Government Services and Noridian Healthcare Solutions — have issued statements that the final LCDs will not go into effect. The MACs previously published LCDs on MIGS, which identified certain procedures as investigational for glaucoma management in patients over the age of 18. Sight Sciences released a statement supporting the decision, which impacts canaloplasty in combination with trabeculotomy ab interno, a procedural description associated with the company’s OMNI Surgical System. Iridex Corp. says that the company, along with other stakeholders opposing the LCDs, engaged with the MACs throughout the comment period to advocate for continued patient access to these glaucoma treatments. The decision effectively retains reimbursement and full access for Medicare patients to these procedures, including the cyclophotocoagulation procedures performed by Iridex’s G6 laser system and probes. 

• Harrow partnered with three health-care technology platforms to expand US availability of VEVYE (cyclosporine ophthalmic solution) 0.1%, a patented, non-preserved, twice-daily dosed, ophthalmic solution prescription drug based on a “water-free” semifluorinated alkane eyedrop technology. VEVYE, dispensed in a 10 microliter drop, is the first cyclosporine-based product indicated for treating both signs and symptoms of dry eye disease. The platforms include PhilRx, a patient access platform that provides end-to-end visibility into the entire prescription life cycle, starting when the physician writes a VEVYE prescription electronically to PhilRx directly from their EHR; Apollo Care, a comprehensive program designed to establish and manage copay program deployment, optimizing patient access and affordability of VEVYE; and PARx Solutions, a web-based technology stack that helps prescribers — free of charge — overcome challenges resulting from prior authorizations.

• Lupin Limited received approval from the FDA for its abbreviated new drug application for loteprednol etabonate ophthalmic suspension, 0.2%, to market a generic equivalent to the reference listed drug Alrex Ophthalmic Suspension, 0.2% (Bausch & Lomb). Loteprednol etabonate ophthalmic suspension, 0.2%, is indicated for the temporary relief of the signs and symptoms of seasonal allergic conjunctivitis. The product will be manufactured at Lupin’s Pithampur facility in India.

• The FDA granted regenerative medicine advanced therapy (RMAT) designation to Ocugen Inc’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations. RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400-101 Phase 1/2 clinical trial as measured by BCVA, Low Luminance Visual Acuity and Multi-Luminance Mobility Test. Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization. Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.

• The FDA granted orphan drug designation to Cellusion Inc.’s regenerative medicine product “iPS cell-derived corneal endothelial cell substitute” (CLS001) for the treatment of bullous keratopathy. CLS001 is alternative cell to corneal endothelial cells produced by efficiently differentiating iPS cells, which can be differentiated into various types of cells, based on Cellusion’s proprietary technique. Cellusion is currently preparing for corporate clinical trials in Japan and global clinical trials. With this designation, the company will further accelerate the global development of CLS001.

• Melt Pharmaceuticals received a written response from the FDA regarding its planned MELT-300 Phase 3 program. Based on the FDA’s response, Melt expects to begin Phase 3 program activities, which will consist of a single pivotal study comparing MELT-300 to sublingual midazolam and placebo in subjects undergoing cataract surgery, in the first quarter of 2024. MELT‑300, a non-IV, non-opioid tablet that combines fixed doses of midazolam (3 mg) and ketamine (50 mg), is administered sublingually using Catalent Inc.’s proprietary fast‑dissolving Zydis delivery technology to rapidly dissolve the tablet for absorption across the very thin sublingual mucosa.

• Oculis Holding AG announced First Patient First Visit in the OCS-01 Phase 3 OPTIMIZE-2 trial for the treatment of inflammation and pain following cataract surgery. Data from the Phase 3 OPTIMIZE-2 trial is intended to support the company’s NDA submission to the FDA. If approved, OCS-01 has the potential to be the first once-daily, topical, preservative-free corticosteroid for treating inflammation and pain following ocular surgery. Patients in the second Phase 3 OPTIMIZE-2 trial will be treated with once-daily OCS-01 post-cataract surgery vs vehicle for 2 weeks. Primary endpoints are the absence of anterior chamber cells (inflammation) on Day 15 and absence of pain on Day 4. OCS-01 is also being evaluated for the treatment of diabetic macular edema in the DIAMOND program and for cystoid macular edema in the LEOPARD trial. 

• Annexon Inc. outlined its global registrational program for ANX007, a first-in-class C1q and classical complement inhibitor, for the treatment of patients with geographic atrophy. Annexon has gained alignment with the FDA on a Phase 3 registration program that includes using the prevention of ≥15-letter loss of BCVA as the primary outcome measure, as well as conducting a comparison of ANX007 to an injection agent, consistent with requests for trials across ophthalmic indications. The FDA has not required Annexon to study the slowing of lesion growth as measured by fundus autofluorescence. Annexon’s registration program will initiate first with ARCHER II, a global sham-controlled trial designed to confirm the results from the Phase 2 ARCHER trial, and potentially expedite the path to regulatory approval in Europe. ARCHER II is expected to begin enrollment in mid-2024, followed by ARROW in late 2024.

• MeiraGTx Holdings plc announced an asset purchase agreement with Janssen Pharmaceuticals (J&J), a Johnson & Johnson company, for the remaining interests in botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked RP, as well as a commercial supply agreement and a technology transfer agreement for bota-vec manufacturing. MeiraGTx will monetize potential future royalties and milestones related to bota-vec with J&J acquiring remaining rights for development, manufacturing and commercialization of bota-vec. In addition, MeiraGTx will receive $130 million in upfront and near-term milestone payments. The company will receive up to an additional $285 million in cash payments upon first commercial sales of bota-vec in the United States and EU and for manufacturing technology transfer. J&J will be responsible for any royalty or milestone amounts that become payable on bota-vec to UCL Business plc. 

• Visiox Pharmaceuticals and PowerUp Acquisition Corp. announced the execution of a definitive agreement for a business combination that would result in Visiox becoming a publicly traded company on the Nasdaq Capital Market. The business combination is expected to close in the first quarter of 2024. Upon closing, the combined company will be named Visiox Holdings with its common stock and warrants expected to be listed on Nasdaq under the ticker symbols VSXP and VSXPW, respectively. Visiox’s pipeline of an FDA-approved drug candidate and other late-stage clinical drug candidates includes treatments for patients with ocular hypertension, open-angle glaucoma and post-surgical inflammation and pain. 

• RetinalGeniX Technologies announced it has contracted with MEDsan Inc. to provide diagnostic testing services for its Institutional Review Board to conduct a study to personalize medical evaluations for patients receiving treatment for wet macular degeneration. Additionally, RetinalGeniX has started planning its pharmaceutical clinical studies for treating dry AMD and Alzheimer’s/dementia complex studies as part of its DNA/RNA GPS initiative. RetinalGeniX has also filed two provisional patents related to these studies and formed an institutional review board to launch a 390-patient clinical study intended to validate the relative suitability of anti-VEGF ocular injection treatments for patient candidates with wet AMD. In concert with its Pharmaco-Genetic Mapping, the company is developing a high-resolution retinal home and remote monitoring system that is intended to offer 24/7 real-time alerts to both physicians and patients and that does not require dilation of the pupil. 

• Prevent Blindness is participating in January’s National Glaucoma Awareness Month to provide patients, care partners and professionals with a variety of helpful resources to educate on glaucoma. In addition to a dedicated webpage, Prevent Blindness offers a variety of downloadable fact sheets, including a listing of glaucoma financial assistance resources, shareable social media graphics in English and Spanish, and educational videos. To help support patients and their care partners, Prevent Blindness and Responsum Health are offering “The Glaucoma Community,” a free, comprehensive platform available via a web browser or through a mobile app for Apple or Android users. Available in multiple languages, the program offers educational glaucoma content, a personalized newsfeed and the moderated “Community Chat.” New this year is The Glaucoma Community Promotional Toolkit for health-care and public-health professionals. In addition, Prevent Blindness will debut the “Focus on Eye Health Patient Story: Glaucoma” video, featuring Prevent Blindness Associate Director of Eye Health Services Tasha R. Lockridge sharing her personal journey with glaucoma. For more information on these resources, click here. 

Quick Notes is published weekly. Unless otherwise noted, the information presented is based on press releases. Find earlier editions here. To submit a press release to be considered for publication, click here.