Oculis takes second eye disease therapy into pivotal trials
Swiss biotech Oculis is poised to start registration trials for privosegtor, its drug candidate for neuro-ophthalmic diseases, in the wake of encouraging phase 2 data.
The Zug-based company said privosegtor (OCS-05) will start two trials (PIONEER-1 and 2) in acute optic neuritis (AON) later this year and early in 2026, and a third (PIONEER-3) in non-arteritic anterior ischemic optic neuropathy (NAION) shortly before the end of next year.
The decision to move the drug into late-stage development follows encouraging phase 2 results in the ACUITY study of serum-glucocorticoid kinase (SGK-2) activator privosegtor in AON, a rare disease characterised by sudden inflammation of the optic nerve. The condition causes temporary vision loss and pain with eye movement and often is a warning sign for multiple sclerosis.
NAION, meanwhile, is a relatively rare sight-threatening disorder that, nevertheless, is the second-leading cause of optic nerve blindness after glaucoma. It has hit the headlines of late after a patient sued Novo Nordisk, claiming that his NAION was caused by the company's GLP-1 diabetes therapy Ozempic (semaglutide).
Privosegtor is Oculis' second ophthalmic drug to start a pivotal trials programme after OCS-01, a topical anti-TNF drug in an eyedrop formulation, which is in a pair of phase 3 trials in patients with diabetic macular oedema (DME), with results due in the second quarter of 2026 and regulatory filings scheduled for the latter half of the year.
PIONEER-1 and PIONEER-2 will look at privosegtor treatment after the onset of AON in patients with and without MS, with the primary endpoint low-contrast visual acuity (LCVA) at three months. At the moment, corticosteroids are used to treat the condition, but there are no approved neuroprotective therapies to protect or restore vision.
PIONEER-3 will enrol patients with acute-onset NAION, another disease that has no medical or surgical treatment shown to improve patients' prognosis.
All three trial protocols have been agreed with the FDA in end-of-phase 2 discussions, according to Oculis, which reckons it has enough cash on hand – around $182 million – to take privosegtor through the PIONEER programme and fund operations to mid 2027 without dipping into a recently announced loan facility with venture capital group BlackRock.
"The positive FDA meeting marks a significant milestone for our rapidly advancing neuroprotection candidate," commented the company's chief executive, Riad Sharif. "With this milestone and the upcoming top-line results from DIAMOND phase 3 DME programme, Oculis is now in a strong position with multiple pivotal studies, targeting multi-billion-dollar markets."
Other potential indications for privosegtor include geographic atrophy, neuropathic keratitis, and glaucoma.
