Oculis Holding AG’s OCS-05 Shows Promise in Treating Acute Optic Neuritis

OCS-05 has received FDA IND clearance and shows safety and efficacy in clinical trials.

Overview of the Phase 2 ACUITY Trial

Oculis Holding AG reported encouraging findings from its Phase 2 ACUITY trial for OCS-05, a candidate treatment for acute optic neuritis. The trial indicated OCS-05 not only had a commendable safety profile compared to a placebo but also delivered notable enhancements in both retinal thickness and visual function. Improvements noted over a six-month period point to a potential neuroprotective effect. Following FDA IND clearance, Oculis intends to start clinical development in the U.S. and investigate the drug’s potential for treating other neurodegenerative disorders. A detailed discussion of these findings will be available during a webcast on January 6, 2025.

Strengths of OCS-05

• OCS-05 exhibited a favorable safety and tolerability profile when compared to a placebo, highlighting its potential for clinical use.
• Statistically significant improvements were recorded in crucial secondary efficacy endpoints, suggesting OCS-05’s strong therapeutic potential.
• The FDA’s IND clearance allows for clinical development in the U.S., positioning OCS-05 for a broader global market reach.
• The company aims to advance OCS-05 as a first-in-class neuroprotective therapy, demonstrating commitment to addressing needs in ophthalmology and neurology.

Concerns About OCS-05

• The Phase 2 ACUITY trial involved a small group of only 36 patients, which may affect the reliability of the results.
• Despite some statistically significant improvements, secondary efficacy findings may not be extensive enough to stimulate strong investor interest without further confirmation.
• OCS-05 currently remains an investigational drug with no official regulatory approval for commercial use, which introduces uncertainties regarding its market viability.

Frequently Asked Questions

What were the findings of the OCS-05 Phase 2 ACUITY trial?

The trial revealed favorable safety outcomes and significant efficacy, improving retinal thickness and visual function for patients with acute optic neuritis.

How is OCS-05 designed as a neuroprotective therapy?

OCS-05 aims to protect retinal ganglion cells and enhance visual function in conditions such as acute optic neuritis.

What regulatory approvals has OCS-05 secured?

OCS-05 has received orphan drug designations from both the FDA and EMA, and the FDA has cleared its IND application.

When is the planned webcast for investors and analysts?

The webcast is set for January 6, 2025, at 8:00 AM Eastern Time.

What other applications might OCS-05 have?

OCS-05 could offer treatment possibilities in broader neurodegenerative diseases beyond just acute optic neuritis.

Disclaimer: This summary reflects information from a press release by GlobeNewswire. Errors may exist, and the full release can be accessed for comprehensive details.

Institutional Activity for $OCS

In the latest quarter, 3 institutional investors increased their holdings in $OCS stock, while 3 others reduced their positions.

To explore hedge funds’ stock portfolios, visit Quiver Quantitative’s institutional holdings dashboard.

Full Release

ZUG, Switzerland, Jan. 06, 2025 (GLOBE NEWSWIRE) —

• 
  OCS-05 exhibited a strong safety and tolerability profile compared to placebo
  
• 
  Statistically significant results were achieved on key secondary efficacy endpoints, including objective measures of retinal thickness and visual improvement
  
• 
  FDA IND clearance enables new clinical developments in the U.S. as part of a global strategy
  
• 
  Plans for OCS-05 include positioning it as a potential first-in-class neuroprotective therapy for acute optic neuritis, with evaluations for expansions into other neurodegenerative diseases
  
• 
  Company to host a webcast for investors and analysts on January 6, 2025, at 8:00 AM Eastern Time
  

Oculis Holding AG (Nasdaq: OCS / ICX: OCS.IC) is dedicated to improving sight and eye care. The company announced promising topline results from its Phase 2 ACUITY trial, successfully meeting both safety and efficacy benchmarks in patients with acute optic neuritis.

The Phase 2 ACUITY (

A

cute Opti

C

Ne

U

r

IT

is of Dem

Y

elinating Origin) trial was a randomized, double-blind, placebo-controlled, and multi-center study. It evaluated OCS-05 (2mg/kg/day or 3mg/kg/day) given intravenously once daily for five days to patients diagnosed with acute optic neuritis and also receiving steroid treatment. In total, 36 patients were randomized, and 33 were included in the modified intent-to-treat (mITT) analysis.

Results from the ACUITY trial were promising, showing OCS-05 met primary safety endpoints and demonstrated neuroprotective benefits while improving visual function in affected patients.

Primary Endpoint Focused on Safety:

The trial evaluated cardiac safety using electrocardiogram (ECG) parameters, measuring shifts from normal to abnormal after treatment until Visit 4 (Day 15). Results indicated no significant differences in abnormal ECG findings across treatment groups.

• Two patients in the OCS-05 group (2 and 3 mg/kg/day) and one in the placebo group presented with a shift from normal to abnormal ECG parameters, amounting to 12.5% in each group. The shifts for the OCS-05 patients were mild, transient, and deemed clinically insignificant by central review.

Evaluating Secondary Efficacy Endpoints in Retinal Structure:

Optical Coherence Tomography (OCT) imaging provided objective measures of retinal structural thickness in patients’ affected eyes, aiming to assess neuroprotective effects of OCS-05 versus placebo. The analysis focused on the Ganglion Cell-Inner Plexiform Layer (GCIPL) and the Retinal Nerve Fiber Layer (RNFL). Results indicated:

OCS-05 Achieves Promising Results in Clinical Trial for Acute Optic Neuritis

Significant Improvement in Retinal Thickness

• A 43% increase in the mean change of GCIPL thickness was observed with OCS-05 (3mg/kg/day) plus steroid compared to placebo plus steroid at month 3. This improvement was sustained through month 6, with p-values of 0.049 and 0.052 at 3 and 6 months, respectively.
• In terms of RNFL thickness, a 28% improvement from the baseline was noted at month 3, reaching 30% by month 6, with p-values of 0.045 and 0.033 at 3 and 6 months, respectively.

Secondary Efficacy Endpoint
Visual Function Improvement Assessed:

The trial evaluated changes in 2.5% ETDRS low contrast letter acuity (LCVA) to determine visual function improvement. The results indicated:

• A notable difference in LCVA mean change from baseline showed about an 18-letter improvement at month 3 and approximately 15 letters at month 6 for those receiving OCS-05 (3 mg/kg/day) plus steroid compared to placebo plus steroid, yielding p-values of 0.004 and 0.012 at 3 and 6 months, respectively.

Treatment-Emergent Adverse Events (TEAEs):

• No serious adverse events (SAEs) related to the drug were reported.
• No adverse events led to drug withdrawal or study discontinuation.
• The most commonly reported drug-related adverse events (AEs > 10%) in the OCS-05 (2 or 3 mg/kg/day) plus steroid group included headache (10.5%, 2 patients) and acne (10.5%, 2 patients).

Riad Sherif, MD, Chief Executive Officer of Oculis, commented: “These promising safety and efficacy results from ACUITY mark a significant step toward introducing the first potential neuroprotective treatment in ophthalmology to patients. The observed vision enhancement is encouraging, and the sustained improvement in retinal structure highlights OCS-05’s therapeutic potential across various ophthalmic and neurological conditions. We are eager to advance OCS-05’s development for acute optic neuritis and explore its potential in additional neuro-ophthalmic settings.”

Mark Kupersmith, MD, Professor and Vice Chair of Translational Research at Icahn School of Medicine, added: “These groundbreaking results signify important progress for patients with acute optic neuritis. While steroids have been used to reduce inflammation, they do not prevent ongoing visual impairments or structural loss. The potential neuroprotective properties of OCS-05 observed in the ACUITY trial could provide significant hope for these patients, with implications extending to conditions like MS, other optic nerve disorders, and glaucoma if confirmed in further studies.”

Pablo Villoslada, M.D., Chair of the Department of Neurology at Hospital del Mar, and Oculis’ Scientific Advisory Board Member, remarked: “I am enthusiastic about the ACUITY results aligning with strong effects seen in animal models addressing neuroinflammation and neurodegeneration. OCS-05’s promise in enhancing vision is encouraging, as further studies can now be conducted for acute optic neuritis while exploring the full potential of this innovative neuroprotective candidate in both ophthalmology and neurology.”

OCS-05 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for acute optic neuritis. This condition typically affects young adults and is characterized by inflammation and demyelination of the optic nerve, which can lead to permanent visual impairment. While steroids are used to mitigate inflammation, they do not provide neuroprotection against vision loss.

Moreover, the Investigational New Drug (IND) application for OCS-05 has been approved by the FDA, allowing the start of clinical development in the United States. The ACUITY trial results, combined with Oculis’ strong development trajectory and solid financial position—approximately $105 to 110 million in cash and short-term investments—set a positive outlook as the company approaches 2025.

Analyst and Investor Call:

The Oculis management team will present an analyst and investor call today at 8:00 a.m. U.S. Eastern Time to discuss the trial results. Interested individuals can access the call via the following webcast here.

A recording of this event will be available for 90 days on the company’s website in the “Events and Presentations” section under “Investors and Media.”

-ENDS-

About OCS-05:

OCS-05 is an innovative small molecule candidate with the potential to be the first neuroprotective therapy for acute optic neuritis and other neuro-ophthalmic diseases. In animal studies, OCS-05 has shown promise in preventing retinal ganglion cell damage and has been linked to improvements in clinical function. Currently in Phase 2 development for acute optic neuritis, OCS-05 has received orphan drug designation from both the FDA and the EMA. The potential for a neuroprotective treatment could extend to various neuro-ophthalmic disorders, such as glaucoma and diabetic retinopathy.

OCS-05 is still investigational and has not gained regulatory approval for commercial use in any country.

About Acute Optic Neuritis:

Acute optic neuritis is a rare condition marked by inflammation of the optic nerve leading to possible long-term visual impairment. It affects up to 8 in every 100,000 people globally and often serves as the early sign of multiple sclerosis. This condition is more common in women and typically occurs in adults aged 20 to 40 years. Patients may experience ocular pain and vision loss, with inflammation resulting in damage to the optic nerve’s protective myelin sheath. Remyelination can happen post-inflammation, but it is often incomplete.

“`html

New Hope for Optic Neuritis: Oculis Trials Innovative Therapy

Recent research highlights the challenges faced by patients with acute optic neuritis, emphasizing the need for effective treatment options.


Overview of the ACUITY Trial

The Phase 2 ACUITY (

A
cute Opti

C
Ne

U
r

IT
is of Dem

Y
elinating Origin) trial was a comprehensive, randomized, double-blind, placebo-controlled study conducted across multiple centers in France. Its goal was to evaluate the effects of OCS-05, administered via daily intravenous infusions for five days, compared against a placebo. The trial focused on patients suffering from acute optic neuritis who were also receiving steroid treatment. Researchers aimed not only to assess safety but also to investigate OCS-05’s potential benefits for neuroprotection and visual function recovery. Among the 36 eligible participants aged 18 to 60, 33 were assigned doses of OCS-05 (2mg/kg/day or 3mg/kg/day) alongside steroids, included in the per-protocol analysis.

The primary safety assessment involved monitoring the percentage of patients whose ECG readings shifted from normal to abnormal from Day 1 through Day 15.

Key Secondary Efficacy Endpoints Included:

• Changes in ganglion cell and inner plexiform layer (GCIPL) thickness in the affected eye, measured via OCT at Day 5, Month 1, Month 3, and Month 6 against initial metrics.
  • This layer remains unaffected by edema during the inflammatory phase of acute optic neuritis.
    
    ¹
    

• Changes in retinal nerve fiber layer (RNFL) thickness in the affected eye, also measured with OCT at the specified intervals.
  • RNFL thickness can increase initially due to inflammation but later decreases, indicating axonal loss. Since RNFL is unmyelinated, its thinning provides direct evidence of this loss.
    
    ^2,3
    

• Changes in the 2.5% ETDRS low contrast letter acuity (LCVA) assessed at various intervals, compared to baseline.
  • LCVA effectively reflects persistent visual decline post-acute optic neuritis, overcoming limitations found in standard high contrast visual acuity measurements.
    
    ⁵
    

For further details about the study, please visit ClinicalTrials.gov
here

.


About Oculis

Oculis, a global biopharmaceutical company (Nasdaq: OCS / ICX: OCS.IC), is committed to enhancing eye care and preserving vision. Their diverse pipeline includes several innovative candidates, such as OCS-01 for diabetic macular edema and post-cataract surgery complications, OCS-02 (licaminlimab) targeting dry eye disease, and OCS-05, which focuses on neuroprotection for acute optic neuritis. Headquartered in Switzerland with branches in the U.S. and Iceland, Oculis aims to improve patient outcomes globally, supported by a seasoned management team and prominent healthcare investors.

For more information, please visit:

www.oculis.com


Oculis Contacts

Ms. Sylvia Cheung, CFO

sylvia.cheung@oculis.com


Investor Relations

LifeSci Advisors

Corey Davis, Ph.D

.

cdavis@lifesciadvisors.com

1-212-915-2577


Media Relations

ICR Healthcare

Amber Fennell / David Daley / Sean Leous

oculis@icrhealthcare.com


Cautionary Statement on Forward-Looking Statements

The financial projections within this release are preliminary and based on the available data at this time. The final financial results for the quarter are pending and may differ from these estimates. This preliminary information has not been audited by an external accounting firm.

This release also includes forward-looking statements about OCS-05’s potential effects, including patient impacts, market opportunities, and overall research strategies. As clinical trial results are often preliminary, they may not reflect outcomes in future trials. Many factors can affect actual results, and these forward-looking statements should not be considered guarantees of performance.

“`

Oculis Provides Insights on Financial Risk Factors

Oculis emphasizes important financial risks outlined in their annual report, available for review on the SEC’s website.

Many risks affecting Oculis are outside the company’s control. For a thorough understanding of these factors, stakeholders should refer to 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). These reports can be accessed at www.sec.gov. Oculis does not commit to updating these risk statements after this release, unless legally required to do so.


¹

Toosy, A. T., et al. (2014). Optic neuritis. The Lancet Neurology, 13(1), 83-99.


²

Trip, S. A., et al. (2005). Retinal nerve fiber layer axonal loss and visual dysfunction in optic neuritis. Annals of Neurology, 58(3), 383-391.


³

Costello, F., et al. (2006). Quantifying axonal loss after optic neuritis with optical coherence tomography. Annals of Neurology: Official Journal of the American Neurological Association and the Child Neurology Society, 59(6), 963-969.


⁴

Henderson, A. P., et al. (2010). A serial study of retinal changes following optic neuritis with sample size estimates for acute neuroprotection trials. Brain, 133(9), 2592-2602.


⁵

Petzold, A., et al. (2020). Case for a new corticosteroid treatment trial in optic neuritis: review of updated evidence. Journal of Neurology, Neurosurgery & Psychiatry, 91(1), 9-14.

* Mixed Model for Repeated Measures (MMRM) Least-Squares Mean Change from Baseline: (nominal directional p-value), mITT population (study eye)

^ Mixed Model for Repeated Measures (MMRM) Least-Squares Mean Change from Baseline: (nominal p-value), mITT population (study eye)

• Oculis Holding AG – Press Release

This article was originally published on Quiver News. For the full story, please visit their website.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.