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Oculis Announces Positive OCS-05 Phase 2 ACUITY Trial in Acute Optic Neuritis, Met Primary Safety Endpoint and Key Secondary Efficacy Endpoints Opening Development Pathways as a Potential First-in-Class Neuroprotective Therapy

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Oculis (Nasdaq: OCS) announced positive topline results from its Phase 2 ACUITY trial for OCS-05, a neuroprotective therapy candidate for acute optic neuritis. The trial met its primary safety endpoint and achieved statistical significance on key efficacy endpoints. The study demonstrated a 43% improvement in GCIPL thickness and 28% improvement in RNFL thickness with OCS-05 (3mg/kg/day) plus steroid versus placebo at 3 months.

The trial involved 36 randomized patients, with 33 included in the modified intent-to-treat analysis. Visual function improvements showed approximately 18 letters improvement at month 3 and 15 letters at month 6. No drug-related serious adverse events were reported, with headache and acne (10.5% each) being the most common side effects.

OCS-05 has received orphan drug designation from both FDA and EMA, and its IND application has been cleared by the FDA. The company reports approximately $105-110 million in cash and equivalents entering 2025.

Oculis (Nasdaq: OCS) ha annunciato risultati positivi dalla sua fase 2 dello studio ACUITY per OCS-05, un candidato terapia neuroprotettiva per la nevrite ottica acuta. Lo studio ha raggiunto l'obiettivo primario di sicurezza e ha ottenuto significativit脿 statistica sui principali endpoint di efficacia. Lo studio ha dimostrato un miglioramento del 43% nello spessore del GCIPL e un miglioramento del 28% nello spessore del RNFL con OCS-05 (3mg/kg/giorno) pi霉 steroide rispetto al placebo dopo 3 mesi.

La sperimentazione ha coinvolto 36 pazienti randomizzati, con 33 inclusi nell'analisi modificata di intent-to-treat. I miglioramenti della funzione visiva hanno mostrato un miglioramento di circa 18 lettere al mese 3 e 15 lettere al mese 6. Non sono stati riportati eventi avversi gravi correlati al farmaco, con mal di testa e acne (10,5% ciascuno) come effetti collaterali pi霉 comuni.

OCS-05 ha ricevuto la designazione di farmaco orfano sia dalla FDA che dall'EMA, e la sua applicazione IND 猫 stata approvata dalla FDA. L'azienda riporta circa 105-110 milioni di dollari in contante e equivalenti all'inizio del 2025.

Oculis (Nasdaq: OCS) anunci贸 resultados positivos de la fase 2 del ensayo ACUITY para OCS-05, un candidato a terapia neuroprotectora para la neuritis 贸ptica aguda. El ensayo cumpli贸 con su principal objetivo de seguridad y logr贸 significancia estad铆stica en los criterios clave de eficacia. El estudio demostr贸 una mejora del 43% en el grosor del GCIPL y una mejora del 28% en el grosor del RNFL con OCS-05 (3 mg/kg/d铆a) m谩s esteroide en comparaci贸n con el placebo a los 3 meses.

El ensayo involucr贸 a 36 pacientes aleatorizados, con 33 incluidos en el an谩lisis de intenci贸n a tratar modificado. Las mejoras en la funci贸n visual mostraron aproximadamente 18 letras de mejora al mes 3 y 15 letras al mes 6. No se informaron eventos adversos graves relacionados con el f谩rmaco, siendo los efectos secundarios m谩s comunes el dolor de cabeza y el acn茅 (10,5% cada uno).

OCS-05 ha recibido la designaci贸n de medicamento hu茅rfano por parte de la FDA y la EMA, y su solicitud IND ha sido aprobada por la FDA. La empresa informa de aproximadamente 105-110 millones de d贸lares en efectivo y equivalentes a principios de 2025.

Oculis (Nasdaq: OCS)電 旮夓劚 鞁滌嫚瓴届椉鞐 雽頃 鞁犼步 氤错樃 鞖旊矔 頉勲炒鞚 OCS-05鞚 2靸 ACUITY 鞁滍棙鞐愳劀 旮嶌爼鞝侅澑 斓滌 瓴瓣臣毳 氚滍憸頄堨姷雼堧嫟. 鞚 鞁滍棙鞚 欤检殧 鞎堨爠靹 氇╉憸毳 於╈”頄堨溂氅, 欤检殧 須姤 氇╉憸鞐愳劀 韱店硠鞝 鞙犾潣靹膘潉 雼劚頄堨姷雼堧嫟. 鞚措矆 鞐瓣惮電 OCS-05(3mg/kg/鞚)鞕 鞀ろ厡搿滌澊霌 鞖旊矔鞚 氤戫枆頄堨潉 霑, 鞙勳暯鞐 牍勴暣 GCIPL 霊愱粯鞐愳劀 43%鞚 臧滌劆鞚 氤挫棳欤检棃鞙茧┌, RNFL 霊愱粯鞐愳劀 28%鞚 臧滌劆鞚 氤挫榾鞀惦媹雼.

氤 鞁滍棙鞐愲姅 36氇呾潣 氍挫瀾鞙 頇橃瀽臧 韽暔霅橃棃鞙茧┌, 33氇呾澊 靾橃爼霅 鞚橂弰 旃橂 攵勳劃鞐 韽暔霅橃棃鞀惦媹雼. 鞁滉皝 旮半姤 臧滌劆鞚 3臧滌洈歆胳棎 鞎 18鞛愳潣 臧滌劆鞚 氤挫榾瓿 6臧滌洈歆胳棎電 15鞛愳潣 臧滌劆鞚 氤挫榾鞀惦媹雼. 鞎诫瓿 甏霠悳 鞁皝頃 攵鞛戩毄鞚 氤搓碃霅橃 鞎婌晿鞙茧┌, 臧鞛 頋旐暅 攵鞛戩毄鞚 霊愴喌瓿 鞐摐毽(臧侁皝 10.5%)鞚挫棃鞀惦媹雼.

OCS-05電 FDA鞕 EMA搿滊秬韯 瓿犾晞鞎 歆鞝曥潉 氚涭晿鞙茧┌, FDA鞚 IND 鞁犾箔鞚 鞀轨澑霅橃棃鞀惦媹雼. 須岇偓電 2025雲 齑堨棎 鞎 1鞏 5氚彪鞐愳劀 1鞏 1觳滊 雼煬鞚 順勱笀 氚 順勱笀靹 鞛愳偘鞚 氤挫湢頃橁碃 鞛堧嫟瓿 氤搓碃頄堨姷雼堧嫟.

Oculis (Nasdaq: OCS) a annonc茅 des r茅sultats positifs pr茅liminaires de son essai de phase 2 ACUITY pour OCS-05, un candidat th茅rapie neuroprotectrice pour la n茅vrite optique aigu毛. L'essai a atteint son principal objectif de s茅curit茅 et a obtenu une signification statistique sur des crit猫res d'efficacit茅 cl茅s. L'茅tude a montr茅 une am茅lioration de 43% de l'茅paisseur du GCIPL et une am茅lioration de 28% de l'茅paisseur du RNFL avec OCS-05 (3 mg/kg/jour) plus st茅ro茂de par rapport au placebo apr猫s 3 mois.

L'essai a impliqu茅 36 patients randomis茅s, dont 33 inclus dans l'analyse modifi茅e d'intention de traiter. Les am茅liorations de la fonction visuelle ont montr茅 environ 18 lettres de progression au mois 3 et 15 lettres au mois 6. Aucun 茅v茅nement ind茅sirable grave li茅 au m茅dicament n'a 茅t茅 signal茅, les maux de t锚te et l'acn茅 (10,5% chacun) 茅tant les effets secondaires les plus courants.

OCS-05 a re莽u la d茅signation de m茅dicament orphelin de la part de la FDA et de l'EMA, et sa demande d'IND a 茅t茅 approuv茅e par la FDA. La soci茅t茅 indique avoir environ 105 脿 110 millions de dollars en liquidit茅s et 茅quivalents d茅but 2025.

Oculis (Nasdaq: OCS) hat positive Ergebnisse aus der Phase-2-Studie ACUITY f眉r OCS-05, einen neuroprotektiven Therapie-Kandidaten zur akuten Optikusneuritis, bekannt gegeben. Die Studie erreichte das prim盲re Sicherheitsziel und erzielte statistische Signifikanz bei den wichtigen Wirksamkeits-Endpunkten. Die Studie zeigte eine 43%ige Verbesserung der GCIPL-Dicke und eine 28%ige Verbesserung der RNFL-Dicke mit OCS-05 (3mg/kg/Tag) plus Steroid im Vergleich zu Placebo nach 3 Monaten.

In der Studie waren 36 randomisierte Patienten beteiligt, wobei 33 in die modifizierte Intention-to-treat-Analyse einbezogen wurden. Die Verbesserungen der Sehfunktionen zeigten eine Verbesserung von etwa 18 Buchstaben nach 3 Monaten und 15 Buchstaben nach 6 Monaten. Es wurden keine schwerwiegenden unerw眉nschten Ereignisse im Zusammenhang mit dem Medikament gemeldet; Kopfschmerzen und Akne (jeweils 10,5%) waren die h盲ufigsten Nebenwirkungen.

OCS-05 hat von der FDA und EMA die Orphan-Drug-Auszeichnung erhalten, und der Antrag auf IND wurde von der FDA genehmigt. Das Unternehmen berichtet von ungef盲hr 105-110 Millionen US-Dollar in Bargeld und Barmitteln zu Beginn des Jahres 2025.

Positive
  • Met primary safety endpoint with no serious adverse events
  • 43% improvement in GCIPL thickness at 3 months (p=0.049)
  • 28-30% improvement in RNFL thickness at 3-6 months (p<0.05)
  • 18 letters improvement in visual function at 3 months (p=0.004)
  • FDA and EMA orphan drug designations received
  • Strong cash position of $105-110 million
Negative
  • Small trial size with only 33 treated patients
  • Some results showed borderline statistical significance (p=0.052)

Insights

The Phase 2 ACUITY trial results for OCS-05 represent a significant breakthrough in ophthalmic neuroprotection. The data shows compelling efficacy with a 43% improvement in GCIPL thickness and 28% improvement in RNFL thickness versus placebo at 3 months, maintained through 6 months. These structural improvements, coupled with an 18-letter gain in low contrast visual acuity, suggest robust neuroprotective effects.

For context, retinal layer preservation is important - think of it as preventing the "wiring" of the eye from degrading. The fact that both key retinal layers showed statistically significant preservation indicates OCS-05 may be effectively protecting nerve cells from damage. The clean safety profile, with no serious adverse events, positions this as a potentially transformative therapy for acute optic neuritis patients who currently have no approved treatments.

This trial success substantially de-risks OCS-05's development pathway and expands Oculis' market opportunity. With FDA orphan designation secured and IND clearance obtained, the company is well-positioned to advance into later-stage trials. The $105-110 million cash position provides adequate runway for continued development.

The potential first-in-class status in neuroprotection opens multiple value-creation opportunities: 1) Core acute optic neuritis indication, 2) Possible expansion into broader neurodegenerative conditions and 3) Potential partnering opportunities given the novel mechanism. The strong efficacy data and clean safety profile enhance the probability of regulatory success and future commercialization. For investors, this positive data point could serve as a significant catalyst for share price appreciation given the current $734 million market cap.

The acute optic neuritis market represents an attractive commercial opportunity given the complete lack of approved therapies. While steroids are commonly used, they don't prevent vision loss or provide neuroprotection - creating a clear unmet need that OCS-05 could address. The robust efficacy data, particularly the 18-letter improvement in visual acuity, suggests strong potential for market adoption if approved.

The broader opportunity in neurodegenerative diseases could significantly expand the addressable market. The company's strategic positioning in both ophthalmology and neurology, combined with first-mover advantage in neuroprotection, creates multiple paths to value creation. The positive trial results should help attract potential commercial partners and strengthen Oculis' negotiating position for future deals.

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

  • OCS-05 showed a favorable safety and tolerability profile compared to placebo
  • Achieved statistically significant results on key secondary efficacy endpoints compared to placebo, including objective structural measures of retinal thickness and visual improvement
  • IND clearance from FDA enables initiation of clinical development in the U.S as part of a global development program
  • Planning next steps for OCS-05 as a potential first-in-class neuroprotective therapy for acute optic neuritis, while evaluating potential expansion into neurodegenerative diseases in ophthalmology and neurology
  • Company to host investor and analyst webcast on January 6, 2025, at 8:00am Eastern Time


Oculis Holding AG (Nasdaq: OCS / ICX: OCS.IC) (鈥淥culis鈥), a global biopharmaceutical company purposefully driven to save sight and improve eye care, today announced positive topline results with OCS-05 in the Phase 2 ACUITY trial, which met the primary endpoint of safety and achieved statistical significance on several key efficacy-based secondary endpoints. The trial evaluated the safety, tolerability and efficacy of OCS-05, a neuroprotective candidate, in patients with acute optic neuritis.

The Phase 2 ACUITY (Acute OptiC NeUrITis of DemYelinating Origin) trial was a randomized, double-blind, placebo-controlled, multi-center trial, designed to evaluate OCS-05 (2mg/kg/day or 3mg/kg/day) administered intravenously once-daily for five days in patients with acute optic neuritis also receiving steroid. The study randomized 36 patients with recent onset (visual loss symptoms) of unilateral acute optic neuritis with a demyelinating origin, of which 33 patients received treatment and were included in the pre-specified modified intent-to-treat (mITT) analysis.

Positive results from the ACUITY trial showed that OCS-05 achieved primary safety endpoint in addition to highlighting neuroprotective structural benefit and the ability to improve visual function in patients suffering from acute optic neuritis.

Primary Endpoint was Safety-Based:

The percentage of patients with a shift from normal (baseline) to abnormal in electrocardiogram (ECG) parameters after study drug administration until Visit 4 (Day 15) was measured to evaluate cardiac safety. The results showed no difference in the percentage of patients with abnormal ECG parameters between the two treatment arms.

  • Two patients in the OCS-05 arms (2 and 3 mg/kg/day) and one patient in the placebo arm had a shift from normal to abnormal in any ECG measures between baseline and Visit 4 (Day 15), both equivalent to 12.5%. Events observed in the OCS-05 arms were mild and transient and qualified as not clinically significant by central review reading center.

Secondary Efficacy Endpoints Assessed Changes in Retinal Structure:

Optical Coherence Tomography (OCT) imaging was used to objectively measure the thickness of two different retinal segments in the affected eye to evaluate the potential neuroprotective effects of OCS-05 compared to placebo: 1) Ganglion Cell-Inner Plexiform Layer (GCIPL) and 2) Retinal Nerve Fiber Layer (RNFL). Results showed:

  • A 43% improvement in GCIPL thickness mean change from baseline in favor of OCS-05 (3mg/kg/day) + steroid compared to placebo + steroid at month 3 which was maintained through month 6 with p-values* of 0.049 and 0.052 at 3 and 6 months, respectively.
  • A 28% improvement in RNFL thickness mean change from baseline in favor of OCS-05 (3mg/kg/day) + steroid compared to placebo + steroid at month 3 reaching 30% improvement at month 6 with p-values* of 0.045 and 0.033 at 3 and 6 months, respectively.

Secondary Efficacy Endpoint Assessed Changes in Visual Function:

Changes in 2.5% ETDRS low contrast letter acuity (LCVA) were measured to assess visual function improvement. Results showed:

  • A favorable difference in LCVA mean change from baseline of approximately 18 letters at month 3 and approximately 15 letters at month 6 with OCS-05 (3 mg/kg/day) + steroid compared to placebo + steroid, with p-values^ of 0.004 and 0.012 at 3 and 6 months, respectively. This is demonstrated graphically in a figure in the attached PDF version of this press release.

Treatment emergent adverse events (TEAEs):

  • No drug-related serious adverse events (SAEs).
  • No AEs leading to drug withdrawal or study discontinuation.
  • Most frequently reported drug related AEs > 10% in the OCS-05 (2 or 3 mg/kg/day) + steroid treatment group were headache: 2 patients (10.5%), and acne: 2 patients (10.5%).

Riad Sherif, MD, Chief Executive Officer of Oculis, commented: 鈥淭hese positive safety and efficacy results from ACUITY represent a significant milestone in bringing the first potential neuroprotective treatment in ophthalmology to patients. The improvement in vision is especially encouraging, and the consistent improvement in retinal structure highlights the therapeutic potential of OCS-05 across multiple ophthalmic and neurological conditions. We are excited to further advance OCS-05鈥檚 development in acute optic neuritis, while actively exploring its potential in additional neuro-ophthalmic indications with the aim to deliver a first-in-class neuroprotective treatment option to patients.鈥

Mark Kupersmith, MD, Professor, Vice chair translational research, Chair NORDIC at Icahn School of Medicine at Mount Sinai Hospital, New York, added: 鈥淭hese groundbreaking results represent an important advancement for acute optic neuritis patients. Steroids have been used to treat the inflammation seen in acute optic neuritis, but don鈥檛 prevent persistent visual impairments or reduce structural loss. There remains a critical unmet need for neuroprotective therapies to preserve vision and the potential neuroprotective properties of OCS-05 observed in the ACUITY trial and its impact on visual function could offer significant hope for patients. These results, if replicated in larger clinical trials, could have profound implications, not only for this condition, but potentially for MS and other optic nerve disorders as well as glaucoma.鈥

Pablo Villoslada, M.D., Chair of the Department of Neurology at Hospital del Mar, Pompeu Fabra University in Barcelona, Spain, Oculis鈥 Scientific Advisory Board Member, said: 鈥淚 am excited to see that the ACUITY results are consistent with the robust effects observed in animal models of neuroinflammation and neurodegeneration in the prevention of retinal ganglion cell damage and that OCS-05 has shown promising results in the improvement of vision. I look forward to the additional studies that can now be initiated for acute optic neuritis while further exploring the full potential of this promising neuroprotective candidate in both ophthalmology and neurology indications.鈥

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, a rare condition characterized by acute inflammation and demyelination of the optic nerve, often affecting young adults, in which retinal thinning is directly associated with vision loss and permanent visual impairment. There are currently no approved therapies specifically indicated for acute optic neuritis and despite steroids being used to treat inflammation and improve recovery, steroids are unable to provide neuroprotection to prevent vision loss.

In addition, the Investigational New Drug (IND) application for OCS-05 has also been cleared by the U.S. Food and Drug Administration (FDA), enabling the initiation of clinical development in the United States to support the global potential of OCS-05. The encouraging ACUITY trial results, Oculis鈥 positive development momentum and a solid balance sheet, including approximately $105 to 110 million in cash, cash equivalents and short-term investments, provide Oculis with a strong start in 2025 to focus on delivering additional development and business milestones.

Analyst and investor call

The Oculis management team will host an analyst and investor call today at 8:00am U.S. Eastern Time, to review the trial results.听听 Interested parties may participate in the call via the following webcast .

A replay of the webcast and accompanying slides will be available for 90 days following the event through the 鈥淓vents and Presentations鈥 page of the 鈥淚nvestors and Media鈥 section of the company鈥檚 website.

-ENDS-

About OCS-05

OCS-05 is a novel peptidomimetic small molecule candidate with the potential to become a first-in-class neuroprotective therapy for acute optic neuritis and other neuro-ophthalmic diseases. In animal models of neuroinflammation and neurodegeneration, OCS-05 has shown positive results in prevention of retinal ganglion cell damage and was associated with improvements in mobility (clinical function disability). OCS-05 is currently in Phase 2 development for the treatment of acute optic neuritis and has received orphan drug designation from both the FDA and the EMA. In addition to acute optic neuritis, a neuroprotective treatment could potentially have wide applicability in neuro-ophthalmic indications such as glaucoma, diabetic retinopathy and other vision-threatening diseases.

OCS-05 is an investigational drug and has not received regulatory approval for commercial use in any country.

About Acute Optic Neuritis

Acute optic neuritis 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 and often represents the first sign of multiple sclerosis. It mainly occurs in adults between the age of 20 and 40 years and is more frequent in women (2:1). The acute inflammatory process of acute optic neuritis leads to the loss of myelin covering the optic nerve and the axons. At the onset, patients often suffer from ocular pain that increases with eye movement and vision loss. Once the inflammation recedes, remyelination often occurs but it is incomplete. Without the myelin sheath protecting the axon, neurons located in demyelinated segments become fragile and prone to death. Unfortunately, damaged axons cannot regrow, leading to permanent visual impairment. To date there is no specific therapy approved for acute optic neuritis and unmet needs remain for therapies that can prevent vision loss after an acute episode of optic neuritis.

About ACUITY Trial

The Phase 2 ACUITY (Acute OptiC NeUrITis of DemYelinating Origin) trial was a randomized, double-blind, placebo-controlled, multi-center trial in France, designed to evaluate a once-daily intravenous infusion of OCS-05 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 OCS-05 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 OCS-05 2mg/kg/day, 3m/kg/day, or placebo for five days in addition to steroid and were included in the pre-specified mITT analysis.

The assessment of the primary safety endpoint was carried out through the percentage of patients with shift from normal (at baseline) to abnormal in any ECG parameter at any time from Day 1 (after study drug administration) to Day 15.

Secondary efficacy endpoints included:

  • Change in ganglion cell and inner plexiform layer (GCIPL) thickness in the affected eye measured with OCT at central reading center at Day 5, Month 1, Month 3 and Month 6 compared to baseline of the affected eye
    • The retinal ganglion cell layer, or retinal ganglion cell and inner plexiform layers combined (GCIPL), the 2 layers are difficult to discriminate by OCT, is not affected by disk swelling during the inflammation phase of acute optic neuritis.1
  • Change in retinal nerve fiber layer (RNFL) thickness in the affected eye as measured with OCT at central reading center at Day 5, Month 1, Month 3 and Month 6 compared to baseline of the affected eye.听
    • The RNFL comprises of unmyelinated axons originating from the retinal ganglion cell bodies. During acute optic neuritis onset, RNFL thickness first increases due to optic nerve swelling during inflammation, but post-inflammation reduction in thickness indicates axonal loss.听 Because RNFL is not myelinated, its thinning is direct evidence of axonal loss, as opposed听to a potential demyelination and loss of oligodendrocytes.2,3,4
  • Change in the 2.5% ETDRS low contrast letter acuity (LCVA) measured at Day 15, Month 1, Month 3, and Month 6 in the affected eye compared to baseline.听
    • LCVA better captures the persistent decrement in low contrast after acute optic neuritis and overcomes the ceiling effect observed with high contrast visual acuity (HCVA) / best corrected visual acuity (BCVA) measurement.5

More information about the study is available on ClinicalTrials.gov .

About Oculis

Oculis is a global biopharmaceutical company (Nasdaq: OCS / ICX: OCS.IC) purposefully driven to save sight and improve eye care. Oculis鈥 highly differentiated pipeline comprises multiple innovative product candidates in development. It includes OCS-01, a topical eye drop candidate for diabetic macular edema (DME) and for the treatment of inflammation and pain following cataract surgery; OCS-02 (licaminlimab), a topical biologic anti-TNF伪 eye drop candidate for dry eye disease (DED) and for non-infectious anterior uveitis; and OCS-05, a candidate designed to be neuroprotective for acute optic neuritis. Headquartered in Switzerland and with operations in the U.S. and Iceland, Oculis鈥 goal is to improve the health and quality of life of patients worldwide. The company is led by an experienced management team with a successful track record and is supported by leading international healthcare investors.

For more information, please visit:

Oculis Contacts
Ms. Sylvia Cheung, CFO

Investor Relations
LifeSci Advisors
Corey Davis, Ph.D.

1-212-915-2577

Media Relations
ICR Healthcare
Amber Fennell / David Daley / Sean Leous

Cautionary Statement Regarding Forward Looking Statements

This financial information in this release reflects the Company鈥檚 preliminary estimate, based on currently available information. Financial closing procedures for the quarter are not yet completed and final results may therefore vary from this estimate. This preliminary estimate has not been audited by our independent registered public accounting firm.

This press release contains forward-looking statements and information. For example, statements regarding the potential effects of OCS-05, including patient impact and market opportunity; the potential of OCS-05 to be a neuroprotective therapy or treatment for acute optic neuritis and other neuro-ophthalmic diseases; the potential of OCS-05 to promote neuronal survival and repair; the potential neuroprotective effects of OCS-05 on preserving retinal thickness in acute optic neuritis patients; the potential of OCS-05 to prevent vision loss; the Company鈥檚 estimated cash, cash equivalents and short-term investments; and Oculis鈥 research and development programs, regulatory and business strategy, future development plans, and management, are forward-looking. Certain clinical trial results presented in this press release are topline and preliminary and subject to change, as analysis is ongoing. These topline results may not be reproduced in subsequent patients and clinical trials. 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鈥 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鈥 annual report on Form 20-F and any other documents filed with the U.S. Securities and Exchange Commission (the 鈥淪EC鈥). Copies of these documents are available on the SEC鈥檚 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.

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

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

3 Costello, F., and 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., and 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., and 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)

Attachment


FAQ

What were the key efficacy results of OCS-05 in the Phase 2 ACUITY trial?

OCS-05 showed 43% improvement in GCIPL thickness, 28% improvement in RNFL thickness at 3 months, and approximately 18 letters improvement in visual function compared to placebo plus steroid.

How many patients participated in Oculis' Phase 2 ACUITY trial for OCS-05?

The trial randomized 36 patients, with 33 patients receiving treatment and included in the modified intent-to-treat analysis.

What is the current cash position of Oculis (OCS) entering 2025?

Oculis reported approximately $105-110 million in cash, cash equivalents and short-term investments entering 2025.

What regulatory designations has OCS-05 received for acute optic neuritis?

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

What were the main side effects reported in the OCS-05 ACUITY trial?

The most frequently reported drug-related adverse events were headache and acne, each affecting 10.5% of patients in the treatment group.

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