Oculis announces positive phase 2 trial results for OCS-05 in acute optic neuritis
- OCS-05 met primary safety endpoint and key secondary efficacy endpoints in the Phase 2 ACUITY trial.
- The trial showed significant improvements in retinal thickness and visual function compared to placebo.
- OCS-05 is being developed as a potential first-in-class neuroprotective therapy for acute optic neuritis.
- IND clearance from FDA allows for clinical development in the U.S. as part of a global program.
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NMD Pharma receives FDA orphan drug designation for NMD670 in Charcot-Marie-Tooth disease
- NMD670, a small molecule inhibitor, has been granted orphan drug designation by the FDA for Charcot-Marie-Tooth disease (CMT).
- The drug is currently in a Phase 2 clinical trial for CMT, involving 80 adult patients with genetically confirmed CMT1 or CMT2 subtypes.
- NMD Pharma is conducting three global trials for NMD670, including studies in spinal muscular atrophy and generalized myasthenia gravis.
- Data readouts from these trials are expected between the second half of 2025 and the first half of 2026.
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JCR Pharmaceuticals and Modalis Therapeutics advance gene therapy research
- JCR Pharmaceuticals and Modalis Therapeutics have validated the initial proof of concept for a novel gene therapy targeting a CNS disease.
- The collaboration will now enter a new phase focusing on pre-clinical studies using JCR's J-Brain Cargo technology and Modalis' CRISPR-GNDM platform.
- The goal is to develop a gene therapy with improved efficacy and safety, delivered via intravenous injection.
- This advancement is expected to have a minor impact on the financial results of both companies for the current fiscal year.
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