Cambridge, Massachusetts-based Progentos Therapeutics has registered a Phase I/II first-in-human study of PRO-1562, an oral, central nervous system (CNS)-penetrant small molecule designed to induce remyelination, in healthy adults and patients with relapsing multiple sclerosis (RMS) who also have chronic optic neuropathy. The trial (NCT07826689) is listed as recruiting as of September 29, 2026, with a study start date of October 1, 2026. The registration marks Progentos's first entry into human testing since its 2024 launch.
The study runs in two parts: a randomized, double-blind, placebo-controlled single-ascending dose Phase I in healthy adults testing multiple dose levels at a single site, followed by a six-month, multi-center Phase IIa in adults aged 18–65 with RMS and chronic optic neuropathy. Total planned enrollment is 160 participants; Phase IIa patients continue stable disease-modifying therapy and receive once-monthly oral PRO-1562 or placebo, with the primary endpoint being change from baseline in P100 visual evoked potential (VEP) latency — a measure of optic nerve conduction speed — at six months. The sole registered site is CMAX Clinical Research in Adelaide, South Australia, with primary completion expected in December 2028.
Progentos describes its approach as developing small molecules intended to activate endogenous oligodendrocyte progenitor cells to generate new oligodendrocytes and remyelinate axons in patients with MS and other demyelinating diseases. The molecular target of PRO-1562 has not been publicly confirmed. Progentos has characterized PRO-1562 as a potential first-in-class small molecule designed to induce remyelination of axons affected by MS.
The Phase IIa portion will use change from baseline in P100 visual evoked potential (VEP) latency at six months as its primary endpoint, providing an objective electrophysiological measure associated with remyelination. A secondary endpoint will assess MRI-measured brain myelin content at six months.
Progentos launched in 2024 with a USD 65 million Series A financing led by Forbion to advance its lead remyelination program through human proof-of-concept studies. The underlying program was acquired from Frequency Therapeutics, an MIT spinout founded around discoveries from the laboratories of Robert Langer at MIT and Jeffrey Karp at Harvard Medical School and Brigham and Women’s Hospital.