December 24, 2024 - 🧬 [nGram] Today’s Pharma Scoop: Blarcamesine's EMA Nod & More

1. Blarcamesine receives EMA filing acceptance for treatment of Alzheimer’s disease

   • The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for blarcamesine, an investigational drug for Alzheimer's disease.
   • The submission is based on positive results from the ANAVEX2-73-AD-004 Phase IIb/III trial, which showed that blarcamesine slowed clinical decline in early Alzheimer's patients.
   • Blarcamesine is an oral, once-daily small molecule that enhances autophagy, potentially offering a novel treatment option for Alzheimer's disease.
   • Next steps include continued engagement with the EMA to potentially bring this treatment to patients in Europe.
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2. Gain Therapeutics initiates Phase 1b clinical trial of GT-02287 in Parkinson’s disease

   • Gain Therapeutics has received approval in Australia to start a Phase 1b trial for GT-02287 in Parkinson’s disease.
   • The trial will assess safety, tolerability, and biomarkers over three months of dosing.
   • This follows a successful Phase 1 study showing safety and significant target engagement.
   • Enrollment is expected to complete by spring 2025, with data anticipated mid-2025.
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3. Ractigen therapeutics announces first patient dosed in phase I clinical trial for RAG-17 in SOD1-ALS

   • Ractigen Therapeutics has dosed the first patient in a Phase I trial for RAG-17, targeting ALS with SOD1 gene mutations.
   • The trial is a randomized, double-blind, placebo-controlled study assessing safety, pharmacokinetics, and preliminary efficacy.
   • RAG-17 has received Orphan Drug Designation from the FDA and IND approval from China's NMPA.
   • Promising data from an Investigator-Initiated Trial was presented at major neurology conferences, validating its potential.
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4. Fujirebio and Eisai collaborate on blood-based biomarkers for neurodegenerative diseases

   • Fujirebio and Eisai have signed a memorandum of understanding for joint research on blood-based biomarkers in neurodegenerative diseases.
   • The collaboration aims to develop and commercialize diagnostic methods, focusing on plasma phosphorylated tau 217 protein (p-Tau217).
   • The partnership will explore simple diagnostic methods and in vitro diagnostics for neurodegenerative diseases.
   • The goal is to establish diagnostic technologies globally, improving diagnosis and treatment options for these diseases.
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