Abstract
In this review period, there were a large number of press releases relating to the scientific meetings held in the second quarter of 2025. Notable are U.S. regulatory approvals for a pharmacological treatment using a new mechanism of action for dry eye disease (DED), as well as expanded indications for approved retinal pharmacotherapies. Clinical studies are ongoing for new chemical entities and reformulated drugs for both anterior and posterior segment diseases. As well, gene therapy continues to be an area of activity. While most of these therapies are local ocular therapies, systemic delivery is also being evaluated.
Ophthalmical Pharmaceuticals and Biologics
Alcon received U.S. FDA approval for its Tryptyr™ (acoltremon ophthalmic solution), formerly known as AR-15512, for the treatment of signs and symptoms of DED. This molecule was originally developed by Avizorex (May 2025).
Aldeyra Therapeutics announced results of a Phase 3 randomized, double-masked, vehicle-controlled trial of its 0.25% reproxalap ophthalmic solution (May 2025).
Ashvattha Therapeutics announced results from a phase 2 study of its migaldendranib, an anti-angiogenic molecule injected subcutaneously, for the treatment of DME and wet AMD (May 2025).
Cognition Therapeutics reported results from its Phase 2 COG2201 ‘MAGNIFY’ trial of zervimesine (CT1812) in adults with geographic atrophy (GA, May 2025).
D. Western Therapeutics Institute, Inc. presented results of a Phase 2B study of its topical ocular H-1337 kinase inhibitor in the treatment of glaucoma and ocular hypertension (May 2025).
Entod Pharmaceuticals received marketing authorization from the Central Drugs Standard Control Organisation (CDSCO) in India for 0.05% atropine eye drops (Myatro XL) developed to slow the progression of myopia in children age 6 to 12 (April 2025).
EyePoint completed enrollment in its Phase 3 LUGANO Trial of its Duravyu™ (vorolanib intravitreal insert) for treatment of Wet AMD (May 2025).
Genentech received FDA approval for an expansion to the label for its Susvimo™ (ranibizumab injection) for the treatment of diabetic retinopathy (DR, May 2025).
Grifols cleared an investigational new drug (IND) application with the FDA for its GRF312 Ophthalmic Solution (immunoglobulin) for the treatment of DED. This product is jointly developed with Selagine (May 2025).
Isarna Therapeutics presented results from its phase 2 BETTER of its intravitreal ISTH0036, a selective TGF-β2-blocking antisense oligonucleotide, in patients with wet AMD and DME (May 2025).
Kodiak Sciences presented results from several clinical studies using its Antibody Biopolymer Conjugate platform for uveitis and selected retinal diseases (May 2025).
Nordic Pharma announced a partnership with Harrow to launch a generic version of Maxitrol™ (Neomycin and Polymyxin B Sulfates and Dexamethasone ophthalmic suspension) for the treatment and relief of bacterial eye infections (April 2025).
Ocular Therapeutix completed enrollment in its SOL-R registrational trial of Axpaxli in treatment of wet AMD (May 2025).
Oculis completed enrollment in two Phase 3 trials of its OCS-01 (dexamethasone) eye drops in the treatment of DME (DIAMOND-1 and DIAMOND-2, April 2025).
Okyo Pharma received FDA fast track designation for its OK-101 (urcosimod) for the treatment of ocular pain (May 2025).
Optigo presented preclinical results of the pharmacokinetics of its drug delivery system for aflibercept for the treatment of retinal diseases (May 2025).
PYC Therapeutics is continuing its development of VP-001, an RNA therapeutic, to treat Retinitis Pigmentosa type 11 (April 2025).
Pykus Therapeutics presented clinical data from an ongoing pilot study evaluating its PYK-2101, a focal hydrogel retinal sealant, in patients undergoing surgery for detached retina (May 2025).
SpyGlass Pharma announced results from a first-in-human study of its delivery system of a prostaglandin analog in patients with concomitant glaucoma and cataract (April 2025).
Tenpoint Therapeutics submitted a New Drug Application (NDA) to the FDA for its brimocohol PF for the treatment of presbyopia (April 2025).
Gene and Cell Therapy
4D Molecular Therapeutics received the Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its 4D-150 for the treatment of diabetic macular edema (DME). This adds to the existing RMAT for the treatment of wet age-related macular degeneration (AMD). D-150 is an intravitreal vector, R100, and a transgene cassette that expresses both aflibercept and a vascular endothelial growth factor (VEGF)-C inhibitory RNA interference (RNAi, May 2025).
Atsena Therapeutics received RMAT designation from the FDA for its ATSN-201 gene therapy for the treatment of X-linked retinoschisis (XLRS, April 2025).
Johnson & Johnson announced results from its LUMEOS trial of its botaretigene sparoparvovec (bota-vec) gene therapy in patients with X-lined retinitis pigmentosa. This product is an adeno-associated virus with a functional copy of the retinitis pigmentosa GTPase regulator (RPGR) gene (May 2025).
Medical Devices
Luminopia received US FDA clearance expanding its digital treatment for amblyopia treatment to patients aged 8 to 12 years (April 2022).
LumiThera announced results from the LIGHTSITE 3B extension trial, an open-label trial in dry age-related macular degeneration (AMD) subjects of its Valeda Light Delivery System for the treatment of dry AMD (May 2025).
Regulatory, Government, and Pharmaceutical Industry
Reports of manufacturing issues with over-the-counter tear supplements continue (May 2025).
The U.S. FDA
The Center for Drug Evaluation and Research announced the use of artificial intelligence (AI) in the review of an application. The issue of confidentiality and the nature of the application were not disclosed (April 2025).
The reduction in force in the FDA has impacted its ability to screen advisory committee members from conflict of interest screening (May 2025).
As part of its quality review, FDA noticed a vendor used by Sponsors that has serious data integrity issues (May 2025).
Continued efforts to reduce the use of animal testing (April 2025).
Has a joint initiative with the National Institutes of Health (NIH) to evaluate nutrition science (May 2025).
Oncology advisory committee recommended against an oncology product based upon non-US clinical data, which was not judged generalizable to the U.S. population (May 2025).
Has delayed the review date (Prescription Drug User Fee Act, PDUFA) for several New Drug Applications (May 2025).
The U.S. NIH Is
Adopting a new initiative to expand human-based science while reducing animal use in research (April 2025).
Accelerating a previous ruling regarding public availability of published articles (April 2025).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmical, pharmaceutical, and medical device firms. A complete listing of all of Dr. Novack’s consulting affiliations can be found online.
Funding Information
No funding was received for this article.