• 09 Jan 2026
• Admin
• News Article

FDA Grants Breakthrough Therapy Designation to Oculis’ Privosegtor for Optic Neuritis

The US FDA has granted Breakthrough Therapy Designation to privosegtor, Oculis Holding AG’s neuroprotective drug candidate for optic neuritis (ON).

The designation reflects strong phase II data and accelerates regulatory interaction for a condition with no approved neuroprotective therapies.

Why This Matters: Optic Neuritis Has No Neuroprotective Treatment?

Optic neuritis is a sight-threatening inflammatory condition of the optic nerve. It often represents the first clinical sign of multiple sclerosis, especially in young adults.

Key challenges today:

• Vision loss can become permanent
• Current care focuses on inflammation, not nerve protection
• Neuroaxonal damage continues even after steroids

Privosegtor aims to change that.

Privosegtor: Designed to Protect Nerve Cells

Privosegtor is a novel peptoid small molecule engineered to:

• Cross the blood–brain barrier
• Cross the retinal barrier
• Directly protect nerve cells from damage

If approved, it could become the first neuroprotective therapy for optic neuropathies.

ACUITY Trial: Clinically Meaningful Vision Gains

The FDA’s decision was supported by results from the phase II ACUITY trial.

Key findings at three months:

• 18-letter average gain in low-contrast visual acuity
• Compared with IV steroids alone
• A 15-letter gain equals a two-fold improvement in visual resolution

This level of improvement is considered clinically meaningful for daily functioning.

Beyond Vision: Structural and Biological Protection

Privosegtor did more than improve eye charts. The trial also showed:

• Preservation of retinal and optic nerve anatomy
• Reduced neurofilament release, a marker of neuroaxonal injury
• Consistent neuroprotective signals across multiple measures

This combination is rare in neuro-ophthalmology trials.

Safety Profile Remains Clean

Privosegtor was well tolerated. Reported drug-related adverse events:

• Headache
• Acne

There were:

• No serious drug-related adverse events
• No treatment discontinuations

PIONEER Program: Moving Into Registrational Trials

Following FDA interactions in 2025, Oculis launched the PIONEER program.

The program includes:

• Three pivotal trials
• Optic neuritis (ON)
• Non-arteritic anterior ischemic optic neuropathy (NAION)

The first trial, PIONEER-1, was initiated in Q4 last year. Enrollment is expected to begin shortly across three continents.

A $7 Billion Unaddressed Market

ON and NAION together represent a potential $7 billion US market, driven by:

• High unmet need
• Lack of neuroprotective options
• Risk of permanent vision loss

Privosegtor is positioned as a platform neuroprotective therapy, not a single-indication asset.

Leadership Perspective

Oculis sees this as a turning point.

• Riad Sherif, CEO: The designation validates privosegtor’s potential to redefine treatment for neuroaxonal loss.

Clinical experts agree.

• Mark Kupersmith, MD: This is the first study showing functional, anatomical, and biological neuroprotection together.

Bottom Line

Breakthrough Therapy Designation puts privosegtor on a fast regulatory path.

If pivotal trials succeed, Oculis could deliver:

• The first neuroprotective drug for optic neuritis
• A new treatment paradigm for neuro-ophthalmic diseases
• A scalable platform for broader neurological conditions

2026 is shaping up to be a defining year for this program.