Oculis (OCS) H.C. Wainwright 28th Annual Global Investment Conference summary

Clinical development strategy

• No major changes in clinical or financial criteria for advancing programs after DIAMOND trial; focus remains on execution and leveraging validated biomarkers for patient selection.

• Licaminlimab and Privosegtor target different biological pathways, with Licaminlimab using TNFR1 biomarker enrichment for precision medicine in dry eye disease.

• PREDICT-1 trial is enriched for TNFR1-positive patients, aiming for clinically meaningful improvements in ocular discomfort, with 4-8 point VAS score differences considered competitive.

• Positive PREDICT-1 results would require at least one additional trial in signs before NDA submission, aligning with FDA expectations.

• Restricting indication to biomarker-positive patients is viewed as an advantage, supporting a precision medicine approach and potentially higher revenues.

Diagnostic and regulatory considerations

• Saliva-based qPCR assay for TNFR1 genotype is simple, rapid, and cost-effective, with turnaround as fast as one day in clinical practice.

• FDA does not require a companion diagnostic, but ongoing validation in phase III trials is planned for future clinical use.

PIONEER program and expansion plans

• PIONEER-1 focuses on optic neuritis, requiring rapid coordination among ER, neuro-ophthalmologists, and neurologists; lessons from phase II informed site activation.

• Primary endpoint for regulatory approval is low contrast visual acuity (LCVA); structural markers like GCIPL and neurofilament strengthen the neuroprotection profile for future applications.

• PIONEER-2 will focus on MS patients with acute relapses, aiming to expand the indication to broader acute MS relapses, increasing patient reach significantly.

• Safety data from PIONEER-1 will be leveraged for acute MS relapse programs, with FDA support for using the same dose and regimen.