Global biopharmaceutical company Oculis Holding AG is accelerating its neuro-ophthalmic disease-targeted investigational candidate into a registrational program following a promising meeting with the FDA.
A neuro-ophthalmic asset? This is new …
Indeed. Especially considering our last coverage of Oculis was in regard to two other investigational assets—OCS-01 and OCS-02:
- The company announced patient enrollment completion for its phase 3 diabetic macular edema (DME) program evaluating OCS-01 (also under clinical evaluation for post-cataract surgery inflammation and pain)
- And before that, Oculis reported phase 2 data for its dry eye disease (DED) asset (OCS-02)
So tell me about the new candidate.
Its name: privosegtor (OCS-05).
- What it is: A novel peptoid small molecule asset—acting as a serum-glucose corticoid kinase-2 (SGK-2) activator—with the potential indication to become the first neuroprotective therapeutic for acute optic neuritis (AON) and non-arteritic anterior ischemic optic neuropathy (NAION)
To note: It’s already received Orphan Drug designation from the FDA (and the European Medicines Agency) for AON.
And its mechanism of action (MOA)?
This extends to providing anatomical preservation of the retina along with visual function improvements after an acute episode of ON.
How it does this: By activating the trophic factor pathways (insulin-like growth factor [IGF]-1 and brain-derived neurotrophic factor [BDNF]), both of which play key roles in the development and survival of neurons (such as retinal ganglion cells) involved in the visual system.
- The intended result: Protecting the nerve axons in neuro-ophthalmic diseases and preventing vision loss
For a deeper and more in-the-weeds dive on how privosegtor works, click here.
And how has it performed in clinical trials thus far?
Promising, to say the least. This is based on a data readout at the beginning of the year from the phase 2 Acute OptiC NeUrITis of DemYelinating Origin (ACUITY) trial (NCT04762017).
The crux of its setup: Privosegtor was evaluated for its safety and tolerability (versus a placebo) when administered via an intravenous (IVT) infusion to unilateral AON patients in one of two daily doses (2 mg/kg or 3 mg/kg) for 5 days in addition to corticosteroid standard of care.
And the findings: The asset met the study’s primary safety endpoint while also highlighting its neuroprotective structural benefit and ability to improve visual function among AON patients.
Nice! Now to these advancement plans.
Oculis is now reporting that, based on FDA feedback, it will kick off the PIONEER program—consisting of several global trials—to support registration plans for privosegtor’s potential indications for AON and NAION.
These trials (and their respective launch timeframes):
- PIONEER-1 for AON (Q4 2025)
- PIONEER-2 for AON (H1 2026)
- PIONEER-3 for NAION (mid-2026)
The intent: To run all three studies concurrently and “generate operational synergies, cost efficiencies, and speed up development timelines” for privosegtor.
So what do we know about the first of those trials?
We’ve actually got details on both PIONEER-1 and -2 (both targeting AON patients), and the former is kicking off before the end of the year.
The planned primary endpoint includes measuring low-contrast visual acuity (LCVA) at 3 months, with dosing and patient enrollment expected to mirror that of the ACUITY trial.
And looking at the big picture?
As Oculis CEO Riad Sherif noted, the upcoming PIONEER program is positioning the company as a leader in ophthalmic neuroprotection drug development.
- And with privosegtor, Oculis has "multiple opportunities to target devastating diseases with significant unmet medical needs in late-stage trials.”