The FDA has accepted ONL Therapeutics’ investigational new drug (IND) application for ONL1204 ophthalmic solution for the treatment of patients diagnosed with macula-off rhegmatogenous retinal detachment (RRD).
Tell me about ONL1204.
ONL1024 is a novel, first-in-class small peptide molecule Fas inhibitor that protects key retinal cells (photoreceptors) from cell death that takes place in various retinal diseases and conditions—and, as a result, leads to vision loss.
Of note: the compound has been granted orphan drug designation by FDA.
Any clinical trials?
ONL plans on launching a phase 2 study assessing ONL1204 in patients with macula-off RRD at U.S. sites in the second quarter of 2023.
The primary objective of the study will be to evaluate the safety and efficacy via a dose escalation model (50 μg and 200 μg) of a single intravitreal injection of ONL1204 as an adjunct to standard-of-care (SOC) surgical repair in subjects with a macula-off RRD.
Anything else?
The company is also studying ONL1204 in ongoing phase 1b studies for geographic atrophy associated with age-related macular degeneration (AMD) (NCT04744662) and progressing open-angle glaucoma (NCT05160805) as part of its later-stage clinical development program.
Significance?
According to ONL’s Chief Scientific Officer, David N. Zacks, MD, PhD, protecting patients’ photoreceptors after diagnosis but before surgery could potentially have a major role in delivering better visual outcomes following reattachment surgery.