Oculis Holding AG announced its Q3 2024 financial results and provided updates on its clinical development programs, highlighting progress in its late-stage pipeline and preparations for commercialization. The company's focus remains on advancing its core programs, including OCS-01 for diabetic macular edema (DME) and OCS-05 for acute optic neuritis (AON).
Clinical Pipeline Advancements
Enrollment in the Phase 3 DIAMOND program for OCS-01 in DME has progressed significantly, with approximately 70% of patients enrolled in the DIAMOND-1 trial and 40% in the DIAMOND-2 trial by early October. OCS-01 is an OPTIREACH formulation of high-concentration dexamethasone eye drops. The company anticipates being NDA submission ready in Q1 2025 for OCS-01 for post-operative pain and inflammation, following a pre-NDA meeting with the FDA in August 2024.
Topline results from the Phase 2 ACUITY trial of OCS-05 in AON are expected in December 2024. This trial is a randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the safety and tolerability of OCS-05 in 36 patients with AON. The primary endpoint is safety, with exploratory measurements to assess the neuroprotective potential of OCS-05.
OCS-05: A Novel Neuroprotective Candidate
OCS-05 is a peptidomimetic serum glucocorticoid kinase-2 (SGK-2) activator with a novel mechanism of action, potentially offering neuroprotection in neuro-ophthalmic diseases. AON is a rare inflammatory condition affecting the optic nerve, leading to potential permanent visual impairment, primarily in adults aged 20-40, affecting up to 8 in 100,000 people worldwide. While corticosteroids are used to manage inflammation, there remains a significant unmet need for therapies that preserve vision or provide neuroprotection following an acute episode.
Preclinical studies in animal models of neuroinflammation and neurodegeneration have demonstrated OCS-05's neuroprotective activity, including preventing retinal ganglion cell damage in glaucoma and AON models, promoting axonal sparing, and reducing demyelination in AON models. A Phase 1 trial in 48 healthy volunteers showed no drug-related side effects.
Financial Position
As of September 30, 2024, Oculis reported cash, cash equivalents, and short-term investments of $125.0 million, providing a cash runway into the second half of 2026. Research and development expenses for Q3 2024 were $15.0 million, compared to $10.0 million in the same period in 2023, primarily due to increased clinical trial expenses for the OCS-01 DIAMOND Stage 2 and OCS-05 ACUITY trials.
Management Commentary
Riad Sherif M.D., Chief Executive Officer of Oculis, stated, "During the quarter, we achieved excellent momentum in product pipeline development. We continue to accelerate recruitment for both Phase 3 trials in our core DIAMOND program with OCS-01 in DME and expanded this program’s committees with several world-renowned retina experts. Looking ahead, we are excited for the upcoming topline readout from the OCS-05 Phase 2 ACUITY trial in AON, anticipated in December 2024. The results will provide us with meaningful insights about the safety and tolerability of OCS-05, and its potential as a neuroprotective candidate in acute optic neuritis and other neuro-ophthalmic diseases."
