Abstract
In this review period, the U.S. Food and Drug Administration (FDA) issued warnings about the marketing of unapproved ophthalmic drugs, including one which was also contaminated. Several firms released results of clinical trials covering a wide range of novel therapeutics from anterior to posterior, from re-purposed small molecules to new chemical entities to gene therapies. A novel systemic antibiotic was approved in the United States. Public health policy issues continue to be in the news, including the price of medications, public university financing, and documentation of research publications.
Ophthalmic Pharmaceuticals and Biologics
Amber Ophthalmics commenced subject enrollment in AMB-01-006 (NEXPEDE-1), a randomized, double-masked, vehicle-controlled phase 2/3 clinical trial evaluating two concentrations of Nexagon (lufepirsen ophthalmic gel) for the treatment of persistent corneal epithelial defects (August 2023).
Annexon announced results in its ARCHER trial of intravitreal ANX007, a Fab antibody fragment that selectively inhibits the complement component C1q, in the treatment of geographic atrophy (GA; August 2023).
Apellis provided an update on injection kits supplied by Apellis and an update on the rare events of retinal vasculitis reported in real-world treatment with Syfovre® (pegcetacoplan injection) for GA secondary to age-related macular degeneration (AMD; August 2023).
Clearside announced results of suprachoroidal axitinib for the treatment of wet AMD (August 2023).
Kiora Pharmaceuticals is developing its KIO-100 family of nonsteroidal, anti-inflammatory small molecules for local treatment of ocular inflammation (August 2023).
Novaliq announced that the European Medicines Agency (EMA) has for review its application for marketing authorization CyclASol (ciclosporin ophthalmic solution, August 2023).
Oculis announced results from its phase 3 OPTIMIZE trial with OCS-01 eye drops, a once-daily, high concentration, preservative-free, topical formulation of dexamethasone, for the treatment of inflammation and pain following ocular surgery (August 2023).
ONL Therapeutics completed enrollment in its phase 2 clinical trial of ONL1204 Ophthalmic Solution for the treatment of rhegmatogenous retinal detachment (August 2023).
Sandoz released positive results from its phase 3 confirmatory efficacy and safety study for its biosimilar aflibercept for wet AMD (August 2023).
Cell and Gene Therapy
Opus Genetics Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA (September 2023).
Otsuka Pharmaceutical and ShapeTX announced a multitarget collaboration to develop intravitreally delivered adeno-associated viruses for ocular diseases, with options to add additional targets and tissue types (September 2023).
ViGeneron received clearance from the EMA for a clinical trial of its VG901, a gene therapy to treat cyclic nucleotide gated channel subunit alpha 1-associated retinitis pigmentosa (August 2023).
Medical Devices
Myra Vision started a first-in-human clinical study of the Calibreye System, a glaucoma drainage device with titratable outflow control (September 2023).
Regulatory, Government, and Pharmaceutical Industry
Alimera acquired additional commercialization rights for Yutiq® (fluocinolone acetonide intravitreal insert) 0.18 mg for the treatment of chronic noninfectious uveitis affecting the posterior segment of the eye from Eyepoint (August 2023).
Among top U.S. public research universities, there is a call for more disclosure regarding spending increases (August 2023). 1
Entasis received U.S. FDA approval for its Xacduro (sulbactam for injection; durlobactam for injection), a new treatment for hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia, caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (August 2023).
Formosa Pharmaceuticals licensed to Eyenovia U.S. commercialization rights for its APP13007 (clobetasol proprionate ophthalmic nanosuspension 0.05%) for the Treatment of Inflammation and Pain Following Ocular Surgery (August 2023).
Frontiers publishers made an agreement with the University of California that allows faculty to publish in their journals without fees (August 2023).
Inotiv, a pharmaceutical testing company, faces an investigation into whether its monkey sourcing practices may have violated U.S. anti-foreign bribery law (August 2023). 2
Tessier-Lavigne withdrew a 2001 paper in Science that had been the subject of investigation. 3
Thea acquired ProQR Therapeutics late-stage ophthalmic assets, sepofarsen and ultevursen (August 2023).
The U.S. Centers for Medicare and Medicaid Services announced the first 10 medicines that will be subject to price negotiations with Medicare (August 2023).
The U.S. FDA:
○ Warned consumers not to purchase or use certain methylsulfonylmethane (MSM) eye drops due to contamination. MSM is an unapproved drug in the United States (August 2023).
○ Issued warning letters to eight companies for manufacturing or marketing unapproved ophthalmic drug products in violation of federal law. These warning letters are part of the agency's ongoing effort to protect Americans from potentially harmful ophthalmic products (September 2023).
○ Issued a notice of noncompliance for not reporting clinical trial results (August 2023).
○ Issued guidances on: Biosimilar User Fee Act updates on Performance Review Goals and Meeting, Institutional Review Boards, Real-World Data and Real-World Evidence (August 2023), and Human Factors Engineering Principles (September 2023).
Footnotes
Author Disclosure Statement
The author consults for numerous ophthalmic, pharmaceutical, and medical device firms. A complete listing of all of G.D.N.'s consulting affiliations can be found online.