Participated · Series C · Mar 2023

ONL Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapeutics to protect and improve vision in patients with retinal disease, specifically geographic atrophy (GA) associated with dry AMD. The firm targets a mechanism of action that prevents Fas-mediated death of retinal cells and inflammatory signaling pathways, which it cites as root causes of vision loss. Its lead candidate, ONL1204 Ophthalmic Solution, showed reductions in the rate of growth of GA lesions in a Phase 1b trial after six months with either a single injection or two injections 90 days apart versus sham. A consistent treatment effect was observed when comparing treated eyes to fellow eyes. The company is led by CEO David Esposito and co-founder and chief scientific officer David Zacks, M.D., Ph.D. ONL plans to use the new financing to expand development efforts and further advance its differentiated clinical program in GA. ONL Therapeutics is a clinical-stage biopharmaceutical company based in Ann Arbor, Michigan, developing first-in-class therapeutics to protect retinal cells from Fas-mediated cell death. Its lead asset, ONL1204 Ophthalmic Solution, is a novel small-molecule Fas inhibitor intended to protect photoreceptors and other retinal cells across a range of retinal diseases. ONL1204 has an active IND and has been granted orphan drug designation by the FDA for macula-off rhegmatogenous retinal detachment (RRD). The company is preparing to initiate a U.S.-based Phase 2 study in macula-off RRD next quarter and is conducting two ongoing Phase 1b studies in geographic atrophy (GA) associated with AMD and in progressing open-angle glaucoma (OAG) at sites in Australia and New Zealand. Preclinical work is ongoing to enable trials in other indications, including inherited retinal degeneration. The recently announced financing will support advancement of ONL1204 into Phase 2 and regulatory preparations for additional Phase 2 programs in GA and OAG. ONL Therapeutics, based in Ann Arbor, Michigan, is developing novel therapies to protect vision in patients with retinal disease. Its lead compound, ONL1204, is a novel small-molecule Fas inhibitor designed to protect key retinal cells, including photoreceptors, from Fas-mediated cell death. The company is building a platform of products intended for a range of blinding diseases, including retinal detachment, glaucoma, age-related macular degeneration (AMD), and inherited retinal degeneration (IRD). ONL1204 has been granted orphan drug designation by the U.S. FDA for retinal detachment, and the company has focused initial clinical plans on the acute indication of retinal detachment while pursuing preclinical work for chronic indications. Planned clinical activity includes completing a Phase 1 study in retinal detachment and initiating Phase 1b studies in open-angle glaucoma and dry AMD, plus a repeat-dose toxicology study to support accelerated chronic dosing. The company is led by CEO David Esposito and co-founder/CSO David Zacks, M.D., Ph.D. ONL Therapeutics is developing a platform of Fas inhibitors intended to protect key retinal cells and preserve vision across a range of retinal diseases. Its lead compound, ONL1204, is a novel, first-in-class small-molecule Fas inhibitor designed to protect photoreceptors and other retinal cells from cell death. ONL1204 has been granted orphan drug designation by the U.S. FDA for the treatment of retinal detachment. The company is preparing ONL1204 for a Phase I study in retinal detachment to be conducted in Australia later in 2019. ONL is pursuing a Series B to continue funding clinical development and to expand its Fas inhibitor pipeline into indications including glaucoma, age-related macular degeneration, and inherited retinal degeneration. Financially, ONL recently raised $3 million in a convertible note from a mix of current investors, management, and new investors to advance its clinical program. ONL Therapeutics is developing ONL1204, a novel first‑in‑class small‑molecule Fas inhibitor designed to prevent retinal cell death via direct and inflammatory signaling. The company is initially advancing ONL1204 toward clinical trials for retinal detachment, where the compound has received U.S. FDA orphan drug designation. Preclinical data and literature cited by the company support potential application of ONL1204 in glaucoma, wet and dry age‑related macular degeneration (AMD), non‑infectious uveitis and other retinal neuropathies. ONL closed a $4.25 million Series A and combined those proceeds with a recently announced $1.0 million grant from the National Eye Institute to finalize preclinical development. The financing also included conversion of a previously announced $1.0 million bridge loan. Funds will be used to complete preclinical work, prepare for first‑in‑human trials, and broaden research into other ocular indications with significant unmet need.