Denali Therapeutics announces topline results for Regimen G evaluating eIF2B agonist DNL343 in the Phase 2/3 HEALEY ALS platform trial
- The Phase 2/3 HEALEY ALS Platform Trial did not meet its primary endpoint of efficacy in slowing ALS progression.
- Primary and key secondary endpoints, including muscle strength and respiratory function, showed no statistical difference from placebo.
- DNL343 was found to be safe and well tolerated among participants.
- Further analyses, including neurofilament light (NfL) and other biomarkers, are expected later in 2025.
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Stoke Therapeutics aligns with global regulators for phase 3 study of zorevunersen
- Stoke Therapeutics will host a webcast to discuss regulatory alignment for a Phase 3 study of zorevunersen.
- Zorevunersen is being developed as a potential first disease-modifying treatment for Dravet syndrome.
- The webcast is scheduled for January 7, 2025, at 8:00am Eastern Time.
- Zorevunersen has received orphan drug designation from the FDA and EMA, and Breakthrough Therapy Designation from the FDA.
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BrainChild Bio to advance BCB-276 CAR T-cell therapy for pediatric brain tumors
- BrainChild Bio is advancing BCB-276, a CAR T-cell therapy targeting B7-H3, for diffuse intrinsic pontine glioma (DIPG).
- The company plans a pivotal Phase 2 trial, supported by FDA alignment, to accelerate the Biologics License Application process.
- Preliminary Phase 1 data from Seattle Children's shows promising safety and efficacy, with improved survival rates in DIPG patients.
- The trial aims to initiate by the end of 2025, focusing on children and young adults with this incurable brain tumor.
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BIAL announces first patient out in its phase 2 clinical trial of BIA 28-6156 for GBA1 Parkinson's disease
- BIAL's ACTIVATE Phase 2 study of BIA 28-6156 has seen its first patient complete the full dose regimen.
- BIA 28-6156 is a first-in-class, oral, allosteric activator of beta-glucocerebrosidase (GCase) for GBA1 mutation Parkinson's disease.
- The study is a multicenter, randomized, double-blind, placebo-controlled trial evaluating two dose levels (10mg/day and 60mg/day).
- Topline data from the study is expected in mid-2026.
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Vesper Bio initiates Phase Ib/IIa proof of concept study of VES001 in asymptomatic patients with gene mutations that cause frontotemporal dementia
- Vesper Bio has started dosing VES001 in a Phase Ib/IIa trial for patients with GRN mutations causing frontotemporal dementia (FTD).
- The trial, named SORT-IN-2, is open-label and conducted in the Netherlands and the UK, aiming to complete by mid-2025.
- VES001 is an oral treatment designed to elevate progranulin levels, crucial for neuronal health, in FTD(GRN) patients.
- The study will assess the safety, tolerability, and effect of VES001 on progranulin levels in cerebrospinal fluid and blood plasma.
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Neuraxpharm expands branded business with acquisition of narcolepsy treatments
- Neuraxpharm acquires Provigil (modafinil) and Nuvigil (armodafinil) to enhance its CNS portfolio.
- The acquisition covers most markets outside the US, with strong sales in Europe, Australia, and Mexico.
- This move aligns with Neuraxpharm's strategy to grow its CNS brands and enter new territories like Australia.
- No financial details of the transaction have been disclosed.
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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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