privosegtor (OCS-05) / Accure Therap, Oculis - LARVOL DELTA

Oculis Announces Positive FDA Pre-IND Feedback Supporting Privosegtor Development Strategy for Treatment of Acute Multiple Sclerosis Relapses (GlobeNewswire) - "In the previously completed Phase 2 ACUITY study, Privosegtor showed a clear neuroprotective effect in optic neuritis, improving visual function, preserving retinal ganglion cell (RGC) layer integrity, and reducing neurofilament levels, a biomarker of axonal injury....It also supported a primary endpoint as early as 3 months, with the same 3mg/kg dose administered daily for 5 days as in PIONEER-1. Oculis anticipates an IND submission for the treatment of acute MS relapses in the fourth quarter of 2026."

FDA event • IND • Multiple Sclerosis

Oculis Holding AG…announced an upcoming presentation at Clinical Trials at the Summit 2026, taking place on June 12-13, 2026, in Las Vegas, Nevada. (GlobeNewswire) - "The presentation will highlight the development journey of Privosegtor, a peptoid small molecule that can cross both the blood-brain and retinal barriers and has the potential to become the first neuroprotective therapy for optic neuritis (ON), with potential broad applicability in other neuro-ophthalmic and neurological diseases....Following the successful Phase 2 ACUITY trial and the granting of Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for ON and PRIME (PRIority MEdicines) designation from the European Medicines Agency (EMA), Oculis is advancing the PIONEER registrational program for Privosegtor in optic neuropathies, with the PIONEER-1 trial in optic neuritis....Privosegtor is currently being evaluated in Oculis' PIONEER (Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research) program, which includes two registrational trials..."

Clinical • Optic Neuritis

PIONEER-1: Privosegtor Investigation in Optic Neuropathies Efficacy Evaluation Research (clinicaltrials.gov) - P3 | N=210 | Recruiting | Sponsor: Oculis

New P3 trial • Ocular Inflammation • Ophthalmology • Optic Neuritis

Privosegtor optic neuritis clinical trial design gains FDA agreement (Multiple Sclerosis News Today) - "The FDA has formally agreed that the design and planned analysis of an upcoming Phase 3 trial testing privosegtor in people with optic neuritis could support a future application seeking the therapy’s approval if the trial succeeds...PIONEER-1 and PIONEER-2 are intended to support an approval application for optic neuritis....PIONEER-3, will evaluate the therapy in NAION, a condition in which the optic nerve is damaged due to reduced blood flow"

New P3 trial • Optic Neuritis

Reduction in retinal ganglion cell loss with privosegtor in acute optic neuritis: post-hoc analysis of OCT scans (ARVO 2026) - P2 | "However, measurement of larger areas of the macula still showed statistically robust findings due to reduced variability. Based on the observed neuroprotection and significant improvements in visual function demonstrated in the ACUITY trial, privosegtor potentially represents a novel first-in-class neuroprotective treatment for sparing neurons in optic nerve disease."

Retrospective data • Ocular Inflammation • Ophthalmology • Optic Neuritis

Improved Low Contrast Visual Acuity, Reduction in Retinal Ganglion Cell Loss, and Neurofilaments-light with Privosegtor in Acute Optic Neuritis: Results from a Multicenter, Randomized, Placebo-controlled, Double-masked Trial (AAN 2026) - "Objective:To evaluate the efficacy, safety, and tolerability of privosegtor in acute optic neuritis (AON).Background:Privosegtor (OCS-05), a peptoid small molecule with neuroprotective activity, is being studied as a treatment for AON.Design/The ACUITY phase 2 multi-center, randomized, double-masked, placebo-controlled study evaluated privosegtor (2 or 3 mg/kg IV daily for 5 days) as an addition to methylprednisolone IV in patients with AON of less than 12 days duration, excluding AQP4+ patients. Privosegtor demonstrated significant improvement in visual function, reduced retinal ganglion cell damage, and lowered NfL changes, suggesting neuroaxonal preservation. The drug was well-tolerated by participants."

Clinical • Ocular Inflammation • Ophthalmology • Optic Neuritis • Plasma NfL

Oculis Announces European Medicines Agency PRIME Designation for Privosegtor, Advancing a Potential First‑in‑Class Neuroprotective Candidate for Optic Neuritis (The Manila Times) - "The EMA’s PRIME designation is supported by compelling visual-function results from the Phase 2 ACUITY trial in ON...Following a successful meeting with the FDA in the fall of 2025, Oculis is now advancing the PIONEER program, which includes three global registrational trials in ON and a second rare neuro-ophthalmic disease, non-arteritic anterior ischemic optic neuropathy (NAION). The first registrational trial in the program, PIONEER-1 in ON, was initiated in Q4 2025, with clinical site activation progressing as planned."

European regulatory • Trial status • Optic Neuritis

Oculis Announces U.S. FDA Breakthrough Therapy Designation Granted to Privosegtor for Treatment of Optic Neuritis (The Manila Times) - "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."

Breakthrough therapy • Optic Neuritis

Reduction in Retinal Ganglion Cell Loss and Improved Low Contrast Visual Acuity with Privosegtor in Acute Optic Neuritis: Results from a Multicenter Randomized Placebo-Controlled Double-Masked Trial (ECTRIMS 2025) - "Introduction: Privosegtor (OCS-05), a peptoid small molecule, has shown neuroprotective activity in several experimental animal models and is being studied as a treatment for acute optic neuritis (AON)... This phase 2 multi-center, randomized, double-masked, placebo-controlled study (ACUITY) tested privosegtor (2 or 3 mg/kg IV daily for 5 days) as add-on to standard of care (methylprednisolone IV) in patients with AON of <12 days duration, excluding AQP4+ patients... Privosegtor as add-on to standard of care was well-tolerated in AON and showed reduced retina damage suggesting neuroaxonal preservation, and significant improvement in visual function by end of follow-up. Disclousure of interest: Céline Louapre and Sophie Bonnin contributed equally. Céline Louapre has received speaker or consultancy fees from Biogen, Sanofi, Novartis and Merck, not related to the present study, and is consultant for Oculis SA."

Clinical • Late-breaking abstract • CNS Disorders • Movement Disorders • Multiple Sclerosis • Ocular Inflammation • Ophthalmology • Optic Neuritis • Parkinson's Disease

Oculis Hosts R&D Event Today to Showcase Progress on All Three Pipeline Assets (GlobeNewswire) - "The event will feature several world-renowned key opinion leaders (KOLs), and the presentations will cover: An update on the execution of the two Phase 3 DIAMOND trials of OCS-01 eye drops in diabetic macular edema (DME), with enrollment for both studies completed (>800 patients) and topline data anticipated in Q2 2026; The genotype-based development plan to drive a personalized approach in dry eye disease (DED) and Phase 2/3 study design for Licaminlimab (OCS-02) is anticipated to start in 2H 2025; An expanded data analysis from the ACUITY Phase 2 trial and future development plans for Privosegtor (OCS-05) in acute optic neuritis (AON). Two new programs will be announced utilizing Privosegtor as a neuroprotective treatment for an orphan condition, non-arteritic anterior ischemic optic neuritis (NAION), and for the acute treatment of relapses in multiple sclerosis (MS) patients."

Clinical data • Clinical protocol • Pipeline update • Diabetic Macular Edema • Dry Eye Disease • Multiple Sclerosis • Optic Neuritis

Oculis Announces Pricing of $100 Million Oversubscribed Offering of Ordinary Shares (GlobeNewswire) - "Oculis Holding AG...announced the pricing of an underwritten offering of 5,000,000 of its ordinary shares, CHF 0.01 nominal value per share, at a price of $20.00 per share, for total gross proceeds of $100 million, before deducting underwriting discounts and commissions and offering expenses (the 'Offering')....Oculis intends to use the net proceeds from the Offering to advance and accelerate its clinical development pipeline, particularly the development of its novel neuroprotective clinical candidate Privosegtor (OCS-05), as well as for working capital and general corporate purposes."

Commercial • CNS Disorders

ACUITY: OCS-05 in Patients with Acute Optic Neuritis (clinicaltrials.gov) - P2 | N=36 | Completed | Sponsor: Oculis | Recruiting ➔ Completed | Trial completion date: Mar 2024 ➔ Sep 2024 | Trial primary completion date: Oct 2023 ➔ Sep 2024

Trial completion • Trial completion date • Trial primary completion date • Ocular Inflammation • Ophthalmology • Optic Neuritis

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 (GlobeNewswire) - P2 | N=42 | ACUITY (NCT04762017) | Sponsor: Oculis | "Oculis Holding AG...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....Primary Endpoint was Safety-Based: 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%."

P2 data • Optic Neuritis

A Phase 1 randomized study on the safety and pharmacokinetics of OCS-05, a neuroprotective disease modifying treatment for Acute Optic Neuritis and Multiple Sclerosis. (PubMed, Sci Rep) - P2 | "OCS-05 (aka BN201) is a peptidomimetic that binds to serum glucocorticoid kinase-2 (SGK2), displaying neuroprotective activity...OCS-05 administered as 2-h i.v. infusions of multiple doses up to 3.0 mg/Kg daily for up to 5 consecutive days was safe and well tolerated. Based on this safety profile, OCS-05 is currently being tested in a phase 2 trial in patient with acute optic neuritis (NCT04762017, date registration 21/02/2021)."

Clinical • Journal • P1 data • PK/PD data • CNS Disorders • Multiple Sclerosis • Ocular Inflammation • Ophthalmology • Optic Neuritis

"@AccureTherapeut ACT-01" (,@Fran_Vimianzo)

ACUITY: ACT-01 in Patients With Acute Optic Neuritis (clinicaltrials.gov) - P2 | N=36 | Recruiting | Sponsor: Accure Therapeutics | Trial completion date: Dec 2022 ➔ Mar 2024 | Trial primary completion date: Aug 2021 ➔ Oct 2023

Trial completion date • Trial primary completion date • Ocular Inflammation • Ophthalmology • Optic Neuritis

[VIRTUAL] Synergistic effects of ACT-01 with dimethyl-fumarate or fingolimod for combination therapy in the EAE animal model of MS (ECTRIMS 2021) - "ACT-01 in combination with fingolimod or DMF shows synergistic clinical efficacy in the EAE animal model of MS."

Combination therapy • Preclinical • CNS Disorders • Immune Modulation • Immunology • Infectious Disease • Inflammation • Multiple Sclerosis • Pulmonary Disease • Respiratory Diseases • Tuberculosis • GSK3B • NDRG1

[VIRTUAL] Synergistic effects of ACT-01 with dimethyl-fumarate or fingolimod for combination therapy in the EAE animal model of MS (ECTRIMS 2021) - "ACT-01 in combination with fingolimod or DMF shows synergistic clinical efficacy in the EAE animal model of MS."

Combination therapy • Preclinical • CNS Disorders • Immune Modulation • Immunology • Infectious Disease • Inflammation • Multiple Sclerosis • Pulmonary Disease • Respiratory Diseases • Tuberculosis • GSK3B • NDRG1

[VIRTUAL] Acute optic neuritis as a clinical model to assess efficacy and safety of ACT-01, a novel neurotrophin-like molecule with potential for disease-modifying treatment (ECTRIMS 2021) - "The ACUITY trial aims to assess ACT-01 safety and preliminary efficacy in reducing retinal damage due to AON. Moreover, this trial is a proof-of-concept for the validity of the neuroprotective approach for disease-modifying treatment of demyelinating diseases."

Clinical • Immunology • Ocular Inflammation • Ophthalmology • Optic Neuritis • NDRG1

[VIRTUAL] Phase 1 safety, tolerability and pharmacokinetics of ACT-01, a neuroprotective small molecule for the disease modifying treatment of Acute Optic Neuritis and MS (ECTRIMS 2021) - "ACT-01 administered with 2h i.v. infusions of single doses up to 3.2 mg/Kg and of multiple doses up to 3.0 mg/Kg daily for up to 5 consecutive days was safe and well-tolerated. ACT-01 displayed linear pharmacokinetics with respect to C(max) and AUC and no significant accumulation occurred with multiple doses over 5 days"

Clinical • P1 data • PK/PD data • Ocular Inflammation • Ophthalmology • Optic Neuritis • GSK3B • MRI • NDRG1

[VIRTUAL] Extended neuroprotective efficacy of ACT-01 in the animal model of lysolecithin induced demyelination in optic nerve (ECTRIMS 2021) - "ACT-01 provides continuous and sustained neuroprotective effects after a short course administration in the animal model of lysolecithin-induced demyelination, thus suggesting long-term benefits after an inflammatory insult of the CNS."

Preclinical • Ocular Inflammation • Ophthalmology • Optic Neuritis • GSK3B • NDRG1

[VIRTUAL] Acute optic neuritis as a clinical model to assess efficacy and safety of ACT-01, a novel neurotrophin-like molecule with potential for disease-modifying treatment (ECTRIMS 2021) - "The ACUITY trial aims to assess ACT-01 safety and preliminary efficacy in reducing retinal damage due to AON. Moreover, this trial is a proof-of-concept for the validity of the neuroprotective approach for disease-modifying treatment of demyelinating diseases."

Clinical • Immunology • Ocular Inflammation • Ophthalmology • Optic Neuritis • NDRG1

[VIRTUAL] Phase 1 safety, tolerability and pharmacokinetics of ACT-01, a neuroprotective small molecule for the disease modifying treatment of Acute Optic Neuritis and MS (ECTRIMS 2021) - "ACT-01 administered with 2h i.v. infusions of single doses up to 3.2 mg/Kg and of multiple doses up to 3.0 mg/Kg daily for up to 5 consecutive days was safe and well-tolerated. ACT-01 displayed linear pharmacokinetics with respect to C(max) and AUC and no significant accumulation occurred with multiple doses over 5 days"

Clinical • P1 data • PK/PD data • Ocular Inflammation • Ophthalmology • Optic Neuritis • GSK3B • MRI • NDRG1

[VIRTUAL] Extended neuroprotective efficacy of ACT-01 in the animal model of lysolecithin induced demyelination in optic nerve (ECTRIMS 2021) - "ACT-01 provides continuous and sustained neuroprotective effects after a short course administration in the animal model of lysolecithin-induced demyelination, thus suggesting long-term benefits after an inflammatory insult of the CNS."

Preclinical • Ocular Inflammation • Ophthalmology • Optic Neuritis • GSK3B • NDRG1

ACUITY: ACT-01 in Patients With Acute Optic Neuritis (clinicaltrials.gov) - P2; N=36; Recruiting; Sponsor: Accure Therapeutics

Clinical • New P2 trial • Ocular Inflammation • Ophthalmology • Optic Neuritis