Oculis Announces U.S. FDA Breakthrough Therapy Designation Granted to Privosegtor for Treatment of Optic Neuritis

Rhea-AI Summary

Oculis (Nasdaq: OCS) announced the U.S. FDA granted Breakthrough Therapy Designation to Privosegtor for treatment of optic neuritis on January 6, 2026. The designation is supported by Phase 2 ACUITY data showing an average +18‑letter gain in Low Contrast Visual Acuity (LCVA) at month 3 versus IV steroid alone, plus anatomical preservation and reduced neurofilament release. Oculis launched the registrational PIONEER program after a 2025 FDA meeting; PIONEER includes three pivotal trials and PIONEER‑1 in optic neuritis was initiated in Q4 2025 with global site activation and enrollment expected to begin shortly. The company cites a U.S. market opportunity of $7 billion for key optic neuropathies.

Positive

• FDA Breakthrough Therapy Designation granted (Jan 6, 2026)
• LCVA +18 letters vs steroid alone at month 3 in Phase 2 ACUITY
• PIONEER program launched with three pivotal trials; PIONEER‑1 initiated Q4 2025

Negative

• Drug‑related adverse events: headache and acne in 10.5% of participants

Key Figures

U.S. optic neuropathy market $7 billion Potential market for key optic neuropathies in the U.S.

LCVA gain vs. steroids 18 letters at 3 months Average Low Contrast Visual Acuity improvement in ACUITY trial

Clinically meaningful threshold 15-letter gain Approximate three-line LCVA improvement considered meaningful

Privosegtor dose 3 mg/kg/day Dose used with IV methylprednisolone in ACUITY trial

Common AEs rate 10.5% Headache and acne, each in two participants

PIONEER pivotal trials 3 trials PIONEER-1, -2 in ON and PIONEER-3 in NAION

Registered indication Optic neuritis Breakthrough Therapy Designation treatment indication

Neuroprotective target diseases 2 optic neuropathies Optic neuritis and NAION in the PIONEER program

Market Reality Check

$20.90 Last Close

Volume Volume 34,879 is well below the 104,050 20-day average (relative volume 0.34x). low

Technical Shares at $19.75 are trading above the $18.74 200-day moving average.

Peers on Argus

Several biotech peers were also lower: ABUS -1.26%, EYPT -0.63%, QURE -2.33%, TSHA -10.28%, UPB -3.47%, suggesting broader biotech weakness even as OCS reported positive FDA news.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 09	Managers’ transactions	Neutral	+0.5%	RSU vesting and settlement disclosures for several company directors.
Dec 03	Managers’ transactions	Neutral	+1.1%	Notification of RSU vesting and settlement for a single director.
Nov 28	Managers’ transactions	Neutral	+0.0%	Routine disclosure of RSU vesting for a company director.
Nov 25	Clinical program update	Positive	-2.2%	Positive FDA meeting enabling Privosegtor PIONEER registrational program.
Nov 18	Insider purchase	Positive	-0.1%	Director share purchase disclosed via managers’ transaction filing.

Pattern Detected

Recent news flow was dominated by routine managers’ transactions and one positive development update; the notable FDA/PIONEER announcement in late November coincided with a -2.17% move, showing at least one instance where positive clinical/regulatory news aligned with short-term weakness.

Recent Company History

Over the past few months, Oculis news centered on director RSU vesting disclosures on Nov 28, Dec 3, and Dec 9, 2025, which saw minimal share-price impact. A Nov 18, 2025 filing highlighted a board member share purchase. More strategically, a Nov 25, 2025 update outlined a positive FDA meeting enabling the PIONEER registrational program for Privosegtor, with three pivotal trials planned into 2026. Today’s Breakthrough Therapy Designation builds directly on that FDA dialogue and the Phase 2 ACUITY data already referenced in prior filings.

Regulatory & Risk Context

Active S-3 Shelf Registration 2025-11-10

An effective Form F-3 dated Nov 10, 2025 registers the resale of up to 494,259 ordinary shares issuable under a warrant. Oculis is not selling shares in this prospectus and would only receive proceeds upon any cash exercise of the warrant.

Market Pulse Summary

This announcement highlights FDA Breakthrough Therapy Designation for Privosegtor in optic neuritis, underpinned by Phase 2 ACUITY results showing an 18-letter LCVA gain at three months and targeting a potential $7 billion U.S. market across two optic neuropathies. It extends prior disclosures about the three‑trial PIONEER registrational program. Investors may watch enrollment progress, safety signals, and consistency of neuroprotective biomarkers while keeping in view recent financings supporting development.

Key Terms

breakthrough therapy designation regulatory

"Breakthrough Therapy Designation granted to Privosegtor, a neuroprotective candidate"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

optic neuritis medical

"for the treatment of optic neuritis Privosegtor is advancing in the registrational"

Inflammation of the optic nerve that can cause sudden vision loss, blurring, or eye pain, often like a camera lens fogging or an electrical wire briefly losing connection. It matters to investors because optic neuritis can signal safety issues for drugs or devices, affect regulatory approval, drive legal or clinical costs, and influence demand for treatments and diagnostic services—so reports of cases can move stock prices in healthcare and biotech names.

low contrast visual acuity medical

"achieved an average gain in Low Contrast Visual Acuity (LCVA) of 18 letters"

Low contrast visual acuity measures how well a person can see objects that are only slightly darker or lighter than their background, like reading faint print on gray paper. Investors care because it is a sensitive clinical endpoint in vision trials and product testing that can reveal meaningful benefit or harm not seen with standard high‑contrast tests, and those results can influence regulatory approval, market adoption, and commercial value of eye treatments or devices.

phase 2 medical

"supported by visual‑function results from the Phase 2 ACUITY trial in optic neuritis"

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

biomarker medical

"Additional analyses showed reduced neurofilament release, a biomarker of decreased"

A biomarker is a measurable indicator found in the body, such as in blood or tissues, that provides information about health, disease, or how the body responds to treatment. For investors, biomarkers can signal the potential success or risk of medical products or therapies, influencing the value of related companies and industry trends. They act like signals or clues that help assess the progress of medical advancements and their market impact.

neuroaxonal injury medical

"neurofilament release, a biomarker of decreased neuroaxonal injury seen in conditions"

Neuroaxonal injury is damage to the long, threadlike parts of nerve cells (axons) and the cells themselves that carry signals in the brain and nervous system. Think of it like frayed wiring in an electrical system: when these pathways are damaged, communication breaks down, which can lead to lasting symptoms or disability. For investors, such injury is important because it influences drug and device safety, trial outcomes, regulatory reviews, potential liability, and market demand for treatments or diagnostics.

adverse events medical

"The most common drug‑related adverse events (AEs) were headache and acne"

Adverse events are any harmful or unwanted medical occurrences experienced by people using a drug, device, or undergoing a treatment, whether or not the problem is caused by the product. Think of them as complaints or breakdowns noticed during a trial or after a product is on the market; regulators record and investigate them. Investors care because clusters or serious adverse events can delay approvals, trigger costly studies or recalls, change labeling, and quickly alter a company’s revenue and risk profile.

pivotal trials medical

"PIONEER program, which includes three pivotal trials to support registration plans"

Pivotal trials are the large, definitive clinical studies designed to show whether a drug or medical device works and is safe enough for regulatory approval. Investors watch them like a final exam for a product: passing typically clears the way to sell the therapy and generate revenue, while failing can delay or block approval and sharply reduce a company's value.

AI-generated analysis. Not financial advice.

ZUG, Switzerland, Jan. 06, 2026 (GLOBE NEWSWIRE) --

• Breakthrough Therapy Designation granted to Privosegtor, a neuroprotective candidate, for the treatment of optic neuritis

• Privosegtor is advancing in the registrational PIONEER program across 2 key optic neuropathies, representing an unaddressed potential market of $7 billion in the U.S.

• Privosegtor achieved an average gain in Low Contrast Visual Acuity (LCVA) of 18 letters compared to IV steroid alone at month 3 in the ACUITY trial
  

Oculis Holding AG (Nasdaq: OCS / XICE: OCS) (“Oculis”), a global biopharmaceutical company focused on breakthrough innovations to address significant unmet medical needs in neuro-ophthalmology and ophthalmology, today announced that its neuroprotective candidate Privosegtor was granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) for treatment of optic neuritis (ON).

Privosegtor, a novel peptoid small molecule designed to cross both the blood–brain and retinal barriers, has the potential to become the first neuroprotective therapy for optic neuropathies. These serious conditions carry a significant unmet need, because they can lead to permanent vision loss from nerve cell damage or death. There are no neuroprotective treatments currently available and together, they represent a potential market of $7 billion in the U.S. alone.

The FDA’s Breakthrough Therapy Designation for Privosegtor is supported by visual‑function results from the Phase 2 ACUITY trial in optic neuritis (ON), a rare, sight‑threatening neuro‑ophthalmic condition that is often the first clinical manifestation of multiple sclerosis. In the trial, Privosegtor delivered substantial improvement in LCVA along with consistent anatomical and biological benefits compared with placebo, reinforcing its potential as a neuroprotective treatment across both neuro‑ophthalmic and neurological diseases.

In the ACUITY trial, Privosegtor produced substantial vision improvements on the 2.5% ETDRS Low Contrast Letter Acuity chart. Patients receiving Privosegtor 3 mg/kg/day plus IV methylprednisolone gained an average of 18 letters at three months compared with placebo plus IV methylprednisolone. For context, a 15‑letter (three‑line) gain represents roughly a two‑fold improvement in visual resolution and is considered clinically meaningful for daily visual functioning.  Privosegtor also showed anatomical preservation of retinal and optic nerve structure, which are typically damaged during acute optic neuritis. Additional analyses showed reduced neurofilament release, a biomarker of decreased neuroaxonal injury seen in conditions such as multiple sclerosis. The most common drug‑related adverse events (AEs) were headache and acne (each in two participants; 10.5%). No drug‑related serious AEs or AEs leading to treatment or study discontinuations occurred.

Following a successful meeting with the FDA in 2025, Oculis launched the PIONEER program, which includes three pivotal trials to support registration plans for Privosegtor in ON and a second rare neuro-ophthalmic disease, NAION. These two optic neuropathies represent a potential market opportunity of potentially exceeding $7 billion in the U.S. alone, given the significant unmet medical need. The first trial in the program, PIONEER‑1 in ON, was initiated in Q4 last year. This global study spans three continents. Sites activation is underway, and enrollment is expected to begin shortly.

Riad Sherif, M.D., Chief Executive Officer of Oculis, stated, “Today’s Breakthrough Therapy Designation underscores Privosegtor’s significant potential as a first‑of‑its‑kind neuroprotective therapy for people living with optic neuritis, and highlights our commitment to redefining what’s possible for patients suffering from neuroaxonal loss. With the ACUITY results and Privosegtor now progressing as a neuroprotective platform across key neuro‑ophthalmic diseases, Oculis is uniquely positioned to reshape the treatment landscape in areas with substantial unmet needs, and 2026 is shaping up to be a milestone‑rich year across our late‑stage portfolio.”

Mark Kupersmith, M.D., Chief Medical Advisor, Neuro-Ophthalmology, added: “The ACUITY trial delivered truly groundbreaking results, demonstrating for the first time in a single study that a drug candidate consistently improves visual function alongside anatomical and biological evidence of neuroprotective benefit. Significant unmet medical needs remain, as patients with optic neuritis—more often young women and frequently experiencing the first sign of multiple sclerosis—are still at high risk of permanent visual loss.”

-ENDS-

About Privosegtor
Privosegtor, a novel peptoid small-molecule candidate that penetrates the blood-brain and retinal barriers, has the potential to become the first neuroprotective therapy for optic neuritis (ON) and other neuro-ophthalmic diseases. Positive results from the ACUITY Phase 2 trial demonstrated Privosegtor’s neuroprotective potential through anatomical preservation of the retina and improvements in visual function after an acute episode of optic neuritis. Consistent results were observed in animal models of neuroinflammation and neurodegeneration, where Privosegtor preserved retinal ganglion cell damage and was associated with improvements in mobility (clinical function disability). Privosegtor has received Breakthrough Therapy designation from the FDA and Orphan Drug designation from both the FDA and the EMA for ON and is now entering registrational trials for this indication, as well as a registrational trial in non-arteritic anterior ischemic optic neuropathy (NAION), as part of Oculis’ PIONEER (Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research) program. In addition to its potential neuroprotective effect on the optic nerve, Privosegtor could also have wide applicability in treating other neuro-ophthalmic and neurological indications.
Privosegtor is an investigational drug and has not received regulatory approval for commercial use in any country.

About Optic Neuritis
Optic Neuritis (ON) is a rare condition characterized by an acute inflammation of the optic nerve that can lead to permanent visual impairment. It affects up to 8 in 100,000 people worldwide with a U.S. incidence estimated to be >30,000 and often represents the first sign of multiple sclerosis¹. It mainly occurs in adults between the age of 20 and 40 years and is more frequent in women (2:1)². ON is a type of neuropathy (nerve disease) that happens when acute inflammation of the optic nerve affects the signals traveling from the eyes through the brain, causing pain, vision loss and other symptoms. The cells that make up the optic nerve have a lipid protective coating called a myelin sheath, which is preferentially damaged in ON. Without myelin, the optic nerve cells can’t send signals properly and axons can be irreversibly lost. To date there is no specific therapy approved for acute optic neuritis and the unmet needs remain for therapies that can prevent vision loss after an acute episode by reducing nerve cell permanent damage or death.

About Non-arteritic Anterior Ischemic Optic Neuropathy
Non-arteritic anterior ischemic optic neuropathy (NAION) is an acute optic nerve disorder that causes permanent visual impairment in >60% of affected patients³. It is the most common cause of acute optic nerve injury in individuals over 50 years old⁴ and affects up to 10.2 per 100,000 people worldwide⁵ with a U.S. incidence estimated to be >30,000^4,6,7. In NAION, the optic nerve head region swells and there is painless sudden vision loss. The swelling eventually resolves, but the optic nerve axons and neuronal cell bodies (in the retina) are permanently lost, leading to significant irreversible visual impairment or even blindness⁸. There are no approved therapies for NAION and the unmet medical need is for therapies that preserve vision and provide neuroprotection for patients suffering from NAION.

About the ACUITY Trial Supporting Breakthrough Therapy Designation
The Phase 2 ACUITY (Acute OptiC NeUrITis of DemYelinating Origin) trial was a randomized, double-blind, placebo-controlled, multi-center trial, designed to evaluate a once-daily intravenous infusion of Privosegtor over five days compared with placebo, in patients with acute optic neuritis receiving steroids. In addition to safety, other secondary efficacy endpoints were measured to evaluate the potential of Privosegtor on neuroprotection and visual function improvement in acute optic neuritis patients. The study randomized 36 eligible patients aged between 18 to 60, with recent onset (visual loss symptoms) of unilateral acute optic neuritis with a demyelinating origin, of which 33 patients received Privosegtor 2mg or 3 mg/kg/day plus IV methylprednisolone, or placebo plus IV methylprednisolone for five days.

About Breakthrough Therapy Designation⁹
Breakthrough therapy designation is intended to expedite the review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. Approaches to demonstrating substantial improvement include the following:

• Direct comparison of the new drug to available therapy shows a much greater or more important response
• If there is no available therapy, the new drug shows a substantial and clinically meaningful effect on an important outcome when compared with a placebo or a well-documented historical control.
• The new drug added to available therapy results in a much greater or more important response compared to available therapy in a controlled study or to a well-documented historical control.
• The new drug has a substantial and clinically meaningful effect on the underlying cause of the disease, in contrast to available therapies that treat only symptoms of the disease, and preliminary clinical evidence indicates that the drug is likely to have a disease modifying effect in the long term (e.g., a sustained clinical benefit compared with a temporary clinical benefit provided by available therapies).
• The new drug reverses or inhibits disease progression, in contrast to available therapies that only provide symptomatic improvement.
• The new drug has an important safety advantage that relates to serious adverse reactions (e.g., those that may result in treatment interruption) compared with available therapies and has similar efficacy.

A breakthrough therapy designation conveys more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. FDA will review the full data submitted to support approval of drugs designated as breakthrough therapies to determine whether the drugs are safe and effective for their intended use before they are approved for marketing.

About Oculis

Oculis is a global biopharmaceutical company (Nasdaq: OCS; XICE: OCS) focused on breakthrough innovations to address significant unmet medical needs in neuro-ophthalmology and ophthalmology. Oculis’ highly differentiated late-stage clinical pipeline includes three core product candidates: Privosegtor, a breakthrough neuroprotective candidate in the PIONEER program which consists of studies intended to support registration plans for treatment in optic neuropathies like optic neuritis (ON) and non-arteritic anterior ischemic optic neuropathy (NAION), with potentially broad clinical applications in various other neuro-ophthalmic and neurological diseases; OCS-01, an eye drop in pivotal registration studies, aiming to become the first non-invasive topical treatment for diabetic macular edema (DME); and Licaminlimab, a novel, topical anti-TNFα in Phase 2, which is being developed with a genotype-based approach to drive precision medicine in dry eye disease (DED). Headquartered in Switzerland with operations in the U.S. and Iceland, Oculis is led by an experienced management team with a successful track record and supported by leading international healthcare investors.

For more information, please visit: www.oculis.com

Oculis Contact
Ms. Sylvia Cheung, CFO
sylvia.cheung@oculis.com

Investor Relations
LifeSci Advisors
Corey Davis, Ph.D.
cdavis@lifesciadvisors.com

Media Relations
ICR Healthcare
Amber Fennell / David Daley / Sean Leous
oculis@icrhealthcare.com

Cautionary Statement Regarding Forward Looking Statements

This press release contains forward-looking statements and information. For example, statements regarding the potential benefits of the Company’s product candidates, the initiation, timing, progress and results of current and future clinical trials, Oculis’ research and development programs, regulatory and business strategy, including planned interactions with the FDA and potential benefits of breakthrough therapy designation; Oculis’ future development plans; the timing or likelihood of regulatory filings and approvals; statements about market opportunity, and the Company’s expected financial position and cash runway, are forward-looking. All forward-looking statements are based on estimates and assumptions that, while considered reasonable by Oculis and its management, are inherently uncertain and are inherently subject to risks, variability, and contingencies, many of which are beyond Oculis’ control. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, assurance, prediction or definitive statement of a fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. All forward-looking statements are subject to risks, uncertainties and other factors that may cause actual results to differ materially from those that we expected and/or those expressed or implied by such forward-looking statements. Forward-looking statements are subject to numerous conditions, many of which are beyond the control of Oculis, including those set forth in the Risk Factors section of Oculis’ annual report on Form 20-F and any other documents filed with the U.S. Securities and Exchange Commission (SEC). Copies of these documents are available on the SEC’s website, www.sec.gov. Oculis undertakes no obligation to update these statements for revisions or changes after the date of this release, except as required by law.

References:

1. Martínez-Lapiscina EH, et al. (2014): Is the incidence of optic neuritis rising? Evidence from an epidemiological study in Barcelona (Spain) 2008-2012. J Neurol. 2014 Apr; 261(4): 759-767.
2. Pérez-Cambrodí RJ, Gómez-Hurtado Cubillana A, Merino-Suárez ML, Piñero-Llorens DP, Laria-Ochaita C. Optic neuritis in pediatric population: a review in current tendencies of diagnosis and management. J Optom. 2014 Jul-Sep;7(3):125-30.
3. Sing Hayreh S. (2008): Nonarteritic anterior ischemic optic neuropathy: natural history of visual outcome. Ophthalmology. 2088 Feb;115(2):298-305.
4. https://www.aao.org/eyenet/article/naion-diagnosis-and-management
5. Kupersmith, MJ et al. (2024): Ophthalmic and Systemic Factors of Acute Nonarteritic Anterior Ischemic Optic Neuropathy in the Quark207 Treatment Trial. 2024 July;131(7):790-802.
6. Hattenhauer M G et al. (1997): Incidence of nonarteritic anterior ischemic optic neuropathy. American Journal of Ophthalmology. 1997 Jan;123(1):103-7.
7. Lee M S et al. (2011): Incidence of nonarteritic anterior ischemic optic neuropathy: increased risk among diabetic patients. Ophthalmology 2011 Mar 24;118(5):959-963
8. North American Neuro-Ophthalmology Society website: https://www.nanosweb.org
9. U.S. Food and Drug Administration. “Guidance for Industry: Expedited Programs for Serious Conditions - Drugs and Biologics, 2014”. Available at https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics

FAQ

What did Oculis announce about Privosegtor on January 6, 2026 (OCS)?

Oculis announced FDA Breakthrough Therapy Designation for Privosegtor for optic neuritis on Jan 6, 2026.

What were the Phase 2 ACUITY results for Privosegtor reported by Oculis (OCS)?

In ACUITY, Privosegtor plus IV methylprednisolone achieved an average +18‑letter LCVA gain at three months versus placebo plus steroid.

How does the FDA designation affect Oculis' development plans for Privosegtor (OCS)?

The designation follows a 2025 FDA meeting and supports Oculis' PIONEER registrational program of three pivotal trials to seek approval.

When did PIONEER‑1 for optic neuritis start and when will enrollment begin (OCS)?

PIONEER‑1 was initiated in Q4 2025; global site activation is underway and enrollment is expected to begin shortly.

What safety signals did Oculis report for Privosegtor in ACUITY (OCS)?

The most common drug‑related adverse events were headache and acne (each in 10.5%); no drug‑related serious AEs or discontinuations occurred.

What market opportunity did Oculis cite for Privosegtor in the U.S. (OCS)?

Oculis cited a potential U.S. market opportunity of approximately $7 billion across key optic neuropathies.