A capital-efficient path to first-in-human.
From completed discovery and preclinical work to an active IND-enabling program and a planned Phase 1/2 in 2027, under the 505(b)(2) regulatory pathway.
From discovery to a planned first-in-human study.
505(b)(2), not a new molecular entity.
Both components are previously characterized. A 505(b)(2) application permits reliance, in part, on the FDA's prior findings for an approved drug, which for OF-01 means the systemic safety, toxicology and CMC record generated for the PBA + TUDCA combination (Relyvrio, NDA 216660, approved 2022). New work is ocular-specific. That is the source of the capital and timeline advantage.
The Relyvrio withdrawal.
Relyvrio was voluntarily withdrawn from the market in 2024 after its confirmatory trial in ALS did not meet its endpoint, and FDA approval was formally withdrawn in 2025. That outcome concerns efficacy in ALS, a different disease treated systemically. It does not remove the human safety, toxicology and CMC record that a 505(b)(2) filing references. OF-01 is a different indication, target tissue and route, and its own efficacy must be established in prospective clinical studies.
Near-term workstreams funded by this raise.
Formulation lock
Lead topical formulation lock and stability-indicating analytical methods.
GLP toxicology
GLP ocular tolerability and toxicity in rabbit and minipig models.
Ocular PK
Ocular pharmacokinetics and tissue distribution.
Bioanalysis
Bioanalytical method development for quantitation in ocular matrices.
GMP supply
GMP clinical supply manufactured for first-in-human dosing.
Pre-IND
Pre-IND regulatory interactions with health authorities.