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

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

Axonal and Myelin Neuroprotection by the Peptoid BN201 in Brain Inflammation. (PubMed, Neurotherapeutics) - "In vivo, BN201 prevents axonal and neuronal loss, and it promotes remyelination in models of multiple sclerosis, chemically induced demyelination, and glaucoma. In summary, we provide a new promising strategy to promote neuroaxonal survival and remyelination, potentially preventing disability in brain diseases."

Journal • CNS Disorders • Glaucoma • Immunology • Multiple Sclerosis • Ophthalmology • Solid Tumor

New Spanish firm Accure Therapeutics launched (The Pharma Letter) - "...Accure Therapeutics, a new pharma R&D player in the central nervous system (CNS) field, announces its launch with a Series A round of funding totaling 7.6 million euros ($8.1 million). The funding round was led by Alta Life Sciences and supported by the Center for Technological and Industrial Development (CDTI)....The company will start work on three distinct new medicine programs focused on innovative targets....The initial assets, ACT-01 ready for testing in a clinical Phase II study and both ACT-02 and ACT-03 in preclinical experiments, have been sourced from two Spanish R&D companies (Bionure and Iproteos) and academic research centers with a strong scientific background in neurobiology."

Financing • New P2 trial • Preclinical • CNS Disorders

A unique internal ribosome entry site representing a dynamic equilibrium state of RNA tertiary structure in the 5'-UTR of Wheat yellow mosaic virus RNA1. (PubMed, Nucleic Acids Res) - "Cytosines (C55, C66, C105 and C108) in H1 and H2 and guanines (G73, G79 and G85) in LR1 form discontinuous and alternative base pairing to maintain the dynamic equilibrium state, which is used to elaborately regulate translation at a suitable level. The WYMV RNA1 5'-UTR contains a novel IRES, which is different from reported IRESes because of the dynamic equilibrium state. It is also suggested that robustness not at the maximum level of translation is the selection target during evolution of WYMV RNA1."

Journal

BN201 SAD MAD Study in Healthy Subjects (clinicaltrials.gov) - P1; N=48; Completed; Sponsor: Bionure Farma SL; Recruiting ➔ Completed

Clinical • Trial completion