INmune Bio completes enrollment for phase 2 Alzheimer's trial
- INmune Bio has finalized the enrollment of 208 patients for its Phase 2 trial in early Alzheimer's disease.
- The trial, named AD02, is global, blinded, and randomized, focusing on patients with neuroinflammation.
- XPro is the treatment being tested, targeting glial cells to reduce neuroinflammation and improve cognitive function.
- Top line cognitive results are expected in the second quarter of 2025.
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PTC Therapeutics announces FDA approval of AADC deficiency gene therapy
- FDA granted accelerated approval for KEBILIDI, a gene therapy for AADC deficiency, marking the first gene therapy directly administered to the brain.
- The approval includes a broad label for both children and adults, with launch preparations underway.
- KEBILIDI's approval was based on safety and efficacy data from the ongoing global clinical trial PTC-AADC-GT-002.
- A Rare Disease Priority Review Voucher was also granted, which PTC plans to monetize.
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NurExone Biologic secures EMA orphan status for ExoPTEN in spinal cord injury
- NurExone Biologic's ExoPTEN therapy receives Orphan Medicinal Product Designation from the European Medicines Agency (EMA).
- ExoPTEN is designed for nerve regeneration and functional recovery in acute spinal cord injury using mesenchymal stem cell-derived extracellular vesicles.
- The designation offers incentives like 10 years of market exclusivity, access to grants, and reduced-cost scientific advice.
- Next steps include advancing into clinical trials and working with the EMA to accelerate ExoPTEN's development.
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Asha Therapeutics awarded grant to advance ALS treatment
- Asha Therapeutics received a grant from the ALS Association to develop ASHA-624, a novel inhibitor targeting SARM1 for ALS treatment.
- ASHA-624, designed using PRISM technology, aims to prevent axon degeneration by inactivating SARM1, showing promising preclinical results.
- The grant will support ASHA-624's progression towards first-in-human clinical trials as a potential disease-modifying therapy for ALS.
- Asha also appointed Disarm Therapeutics' founders to its Scientific Advisory Board to bolster expertise in SARM1 biology.
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Cognition Therapeutics reports Q3 2024 financial results and clinical update
- CT1812 showed a 95% slowing of cognitive decline in Alzheimer's patients with low plasma p-tau217 in Phase 2 SHINE study.
- Topline results from Phase 2 SHIMMER study in dementia with Lewy bodies expected by end of 2024.
- Continued enrollment in Phase 2 START and MAGNIFY studies for Alzheimer's and macular degeneration.
- Cash reserves of $22 million expected to fund operations into Q2 2025.
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