Abstract
In this review period, there were a wide range of announcements. An ophthalmic formulation of intravitreal bevacizumab, long used off-label for the treatment of wet age-related macular degeneration (AMD) was approved in Europe. A number of firms evaluating compounds with novel preclinical pharmacology announced Phase 2 clinical results—some positive and some negative. This confirms, in my opinion, the need for clinical trials to validate preclinical data—especially with novel targets. Several additional firms announced the start of controlled Phase 2 trials of novel therapeutics. Another positive sign is the start of several Phase 3 trials of novel products, suggesting that there were positive Phase 2 data, and the investment required for development of much needed additional therapies for ocular disease.
Ophthalmic Pharmaceuticals and Biologics
Apellis received an opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) regarding a Marketing Authorization Application (MAA) for its Syfovre™ (intravitreal pegcetacoplan) for the treatment of geographic atrophy (GA; June 2024).
Azura Ophthalmics announced commencement of enrollment in its “ASTRO” Phase 3 study of AZR-MD-001 (selenium sulfide ophthalmic solution) in the treatment of meibomian gland dysfunction (June 2024).
IVIEW Therapeutics began enrollment in its Phase 2 study of IVW-1001, a TRPM8 agonist in a lid wipe, for the treatment of dry eye disease (DED, June 2024).
Johnson & Johnson announced results for its Phase 2 “DAHLIAS” study of nipocalimab in the treatment of Sjögren’s disease (June 2024).
Melt Pharmaceuticals began enrollment in a Phase 3 study of its MELT-300, an oral fixed combination of midazolam and ketamine for pre-operative use in patients undergoing ocular surgery (June 2024).
Oculis Holding AG announced results from its Phase 2b “RELIEF” trial of its licaminlimab (OCS-02), an anti-TNFα biologic eye drop in patients with DED (June 2024).
Opthea announced that it has completed enrollment in its two Phase 3 studies (“COAST” and “ShORe”) for its sozinibercept in combination with standard-of-care anti-vasoactive endothelial growth factor (VEGF)-A therapies for the treatment of patients with wet AMD (May 2024).
Outlook Therapeutics has received European Commission Marketing Authorization for Lytenava (bevacizumab gamma), an ophthalmic formulation of bevacizumab for the treatment of wet AMD (May 2024).
Rezolute announced results from a Phase 2 study of RZ402, an oral, small molecule, plasma kallikrein inhibitor, in patients with diabetic macular edema (May 2024).
Skye Bioscience announced results from its Phase 2a clinical trial of SBI-100 ophthalmic emulsion, a cannabinoid CB1 receptor agonist, in patients with primary open-angle glaucoma or ocular hypertension (June 2024).
Vyluma announced that the EMA has validated for review its MAA for NVK002 (low-dose atropine) for slowing myopic progression. The firm also announced a meeting with Food and Drug Administration (FDA) regarding its U.S. marketing application (May 2024).
Gene and Cell Therapy
4D Molecular Therapeutics received FDA clearance of its investigational new drug application (IND) for its Phase 1 “GAZE” study of its 4D-175, an R100 vector-based intravitreal genetic medicine for the treatment of patients with GA (June 2024).
Aurion Biotech received Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation from the FDA for its AURN001, an allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease (June 2024).
Neurotech received submitted and received priority review status for a Biologic License Application for its NT-501 (revakinagene taroretcel), an intravitreal encapsulated cell therapy for the treatment of macular telangiectasia type 2 (June 2024).
Ocugen began enrollment in its “liMeliGhT” clinical trial for OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (June 2024).
Regulatory, Government, and Pharmaceutical Industry
FDA: Guidance for clinical pharmacology considerations for the development of oligonucleotides (June 2024).
The International Council for Harmonisation [ed stet] released guidance M12 for human drug interaction studies (May 2024).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, 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.