Oculis Holding AG

Recent Developments

Privosegtor, a novel small molecule peptoid that penetrates the blood-brain and retinal barriers, was selected by high-throughput screening for neurotrophic and neuroprotective properties. Following a successful meeting with the FDA in the third quarter of 2025, we advanced Privosegtor into the PIONEER registrational program for ON and NAION. PIONEER-1 is currently in the global site activation phase and commenced patient enrollment in August 2026, PIONEER-2 is planned to be initiated in the fourth quarter of 2026. Both trials will evaluate Privosegtor for the treatment of ON. The third trial in the PIONEER program, PIONEER-3, will evaluate Privosegtor after the acute onset of NAION and is expected to follow after PIONEER-1 and -2. Privosegtor was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency, a highly selective process to provide early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or provide benefits to patients without treatment options. Privosegtor has also been granted Breakthrough Therapy designation for the treatment of ON by the FDA, reinforcing global regulatory support for this unique neuroprotective asset. In May 2026, we received written agreement from the FDA under a Special Protocol Assessment (SPA) regarding PIONEER-1 to confirm that the design and planned analysis of the PIONEER-1 study are adequate to address the objectives necessary to support a future NDA submission, subject to a successful trial outcome and FDA review of the complete submission. In August 2026, the FDA’s Division of Neurology provided guidance that no additional preclinical studies will be required ahead of an Investigational New Drug (IND) submission for Privosegtor in acute MS relapses, cross-referencing the data from the open ON IND residing in the Division of Ophthalmology. Constructive FDA feedback also supports the proposed clinical development strategy and regulatory pathway, which will include MS patients with both optic neuritis and other relapses, such as ambulatory relapses, in future trials. Oculis expects IND clearance for the treatment of acute MS relapses in the fourth quarter of 2026.

Licaminlimab is a novel biologic treatment being evaluated for ocular inflammation, specifically as a treatment for TNFR1-mediated dry eye disease. We have completed the randomization of patients in the PREDICT-1 registrational trial in DED evaluating Licaminlimab as a precision medicine treatment, and expect to report topline results around year-end 2026.

We completed our OCS-01 DIAMOND Stage 2 program, which included two global pivotal Phase 3 clinical trials, DIAMOND-1 and DIAMOND-2, for the treatment of diabetic macular edema (“DME”). The topline results from the DIAMOND trials were announced in May of 2026. The primary endpoint, mean change in best corrected visual acuity early treatment diabetic retinopathy study (BCVA ETDRS) letter score at Week 52, was not met in both trials. The secondary endpoint of retinal thickness, as measured by OCT, showed a substantial and persistent reduction with OCS-01 vs. vehicle at all visits in DIAMOND-2 and at all visits except Week 52 in DIAMOND-1. The key secondary endpoint of the proportion of patients with ≥15-letter gain in BCVA was not met in both trials. OCS-01 was well tolerated, with no unexpected adverse events observed, and the overall safety profile was consistent with that of previous trials. Based on the results, at this time, we do not plan to pursue an FDA regulatory filing for OCS-01 in DME.