August 7, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Clene's ALS Data, Sangamo & Genentech Deal, Indapta's MS Trial

1. Clene announces new CNM-Au8 biomarker and clinical efficacy data submitted to FDA in support of treatment for ALS

   • Clene submitted new CNM-Au8 biomarker and clinical efficacy data to the FDA for ALS treatment.
   • Data includes post hoc analyses from two Phase 2 clinical trials, showing significant clinical improvements in survival and functional status.
   • CNM-Au8 NfL Responders demonstrated a 28% mean reduction in NfL levels and improved survival compared to non-responders.
   • Long-term treatment with CNM-Au8 30 mg showed significant survival benefits in the HEALEY ALS Platform and RESCUE-ALS trials.
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2. Sangamo Therapeutics announces global epigenetic regulation and capsid delivery license agreement with Genentech

   • Sangamo Therapeutics has entered into a license agreement with Genentech to develop genomic medicines for neurodegenerative diseases.
   • The agreement includes Sangamo’s zinc finger repressors targeting the tau gene and a second undisclosed neurology target.
   • Sangamo will license its neurotropic AAV capsid, STAC-BBB, known for its potent blood-brain barrier penetration.
   • Genentech will handle clinical development, regulatory interactions, manufacturing, and global commercialization.
   • Sangamo will receive $50 million in upfront license fees and milestone payments, with potential earnings up to $1.9 billion.
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3. Indapta Therapeutics announces FDA clearance of IND for Phase 1 trial of IDP-023 for progressive multiple sclerosis

   • Indapta Therapeutics received FDA clearance for an IND to initiate a Phase 1 trial of IDP-023 for progressive multiple sclerosis (MS).
   • The trial will be led by Stanford University and UCSF, combining IDP-023 with the anti-CD20 monoclonal antibody, ocrelizumab.
   • The study aims to explore the biologic effects of IDP-023, leveraging a translational program developed with Stanford and UCSF.
   • Indapta is also conducting a Phase 1/2 trial of IDP-023 in patients with non-Hodgkin’s lymphoma and multiple myeloma.
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4. Exelixis announces FDA acceptance of sNDA for cabozantinib in advanced neuroendocrine tumors

   • The FDA accepted Exelixis' supplemental New Drug Application (sNDA) for cabozantinib for treating advanced pancreatic and extra-pancreatic neuroendocrine tumors (NET).
   • Cabozantinib received orphan drug designation for pancreatic NET.
   • The sNDA is based on the phase 3 CABINET trial, which showed significant improvement in progression-free survival (PFS) for cabozantinib versus placebo.
   • The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of April 3, 2025.
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5. Alzamend Neuro partners with Massachusetts General Hospital for a Phase II clinical trial of AL001 for bipolar disorder

   • Alzamend Neuro collaborates with Massachusetts General Hospital to conduct a Phase II clinical trial of AL001 for bipolar disorder.
   • The trial aims to compare brain lithium levels from AL001 to commonly marketed lithium salts.
   • Dr. Ovidiu Andronesi from Harvard University will be the principal investigator.
   • The study seeks to meet FDA safety standards through the Section 505(b)(2) pathway.
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