Abstract
In this review period, several intraocular gene therapy products for retinal disease have completed milestones in their clinical development. The efficacy of low-dose ocular atropine for slowing pediatric myopia has been positive in some recent trials and negative in others. One firm with a stable low-dose atropine product received a positive opinion from the European Medicines Agency (EMA). Several firms presented results from key Phase 2 and Phase 3 studies.
Ophthalmical Pharmaceuticals and Biologics
ANI Pharmaceuticals received an expanded U.S. label for its Iluvien (fluocinolone acetonide intravitreal implant) that includes an indication for chronic non-infectious posterior uveitis (March 2025). Belite Bio announced a decision by the Data Safety Monitoring Board for the Phase 3 DRAGON trial to continue the trial of tinlarebant for the treatment of Stargardt’s disease (February 2025). Nicox completed dosing in its Whistler phase 3b trial of NCX470 to lower IOP (March 2025). Opthea announced results from its phase 3 clinical trial COAST (Combination OPT-302 with Aflibercept Study) of sozinibercept (a VEGF-C/D trap) in patients with wet AMD (March 2025). Perfuse Therapeutics presented results from its Phase 1/2 trial of its PER-001 intravitreal implant in patients with glaucoma (March 2025). Sydnexis received a positive opinion from the EMA Committee for Medicinal Products for Human Use for its low-dose atropine formulation, SYD-101 (Ryjunea), for slowing the progression of pediatric myopia. Santen has licensed this product in selected countries (April 2025). Unity Biotechnology announced results from its Phase 2b ASPIRE clinical trial of intravitreal UBX1325 in patients with diabetic macular edema (DME, March 2025).
Gene and Cell Therapy
4D Molecular Therapeutics (4DMT) commenced enrollment in its 4FRONT-1 phase 3 clinical trial evaluating 4D-150, its intravitreal gene therapy, for the treatment of wet age-related macular degeneration (AMD. March 2025). Atsena Therapeutics received U.S. Fast Track designation for its gene therapy product candidate, ATSN-201, for the treatment of X-linked retinoschisis (XLRS, March 2025). Luxa Biotechnology announced clinical data from its phase 1/2a clinical trial evaluating RPESCRPE-4W, a proprietary retinal pigment epithelium (RPE) cell therapy for patients with dry AMD (March 2025). Opus Genetics announced results from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label trial for LCA5-related inherited retinal disease (IRD, April 2025). Splice Bio dosed the first patient in its Phase 1/2 ASTRA study of SB-007, a dual-AAV gene therapy for Stargardt Disease (March 2025).
Regulatory, Government, and Pharmaceutical Industry
Alcon announced acquired a majority interest in Aurion Biotech, a clinical-stage company developing cell therapies to treat eye diseases (March 2025). Legal suits continue over the FDA’s judgment that the GLP-1 treatments are not in shortage, thus precluding compounding pharmacies from preparing and selling these products (February 2025). The American Diabetes Association made a policy statement on this issue.
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Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmical, pharmaceutical, and medical device firms. A complete listing of all of G.D.N.’s consulting affiliations can be found online.
Funding Information
No funding was received for this article.