January 9, 2025 - 🧬 [nGram] Today’s News Scoop: Rapport's CNS Breakthrough, Alchemab & Lilly's ALS Collaboration, Newron's South Korea Deal

1. Rapport Therapeutics announces new phase 1 data for RAP-219 in CNS disorders

   • RAP-219 achieved target receptor occupancy in the PET trial, supporting the dosing regimen for the ongoing Phase 2a trial in focal epilepsy.
   • The MAD-2 trial showed RAP-219 was well tolerated with faster titration and higher exposures than the initial MAD trial.
   • No serious adverse events were reported, and the drug maintained a favorable tolerability profile.
   • Topline data from the ongoing Phase 2a trial in focal epilepsy is expected in mid-2025.
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2. Alchemab Therapeutics collaborates with Lilly for ALS therapies

   • Alchemab Therapeutics partners with Lilly to develop up to five novel therapeutic antibodies for ALS.
   • The collaboration leverages Alchemab's platform to identify antibodies from resilient ALS patients.
   • Alchemab will receive an undisclosed upfront payment and potential milestone payments and royalties.
   • The partnership aims to address the significant unmet medical need in ALS treatment.
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3. Newron and Myung In Pharm announce license agreement for evenamide in South Korea

   • Newron Pharmaceuticals and Myung In Pharm have entered a license agreement for evenamide in South Korea.
   • Myung In Pharm will contribute 10% of the patient population for Newron's Phase III trial and cover related costs.
   • The Phase III trial will begin in H1 2025, involving 600 patients with treatment-resistant schizophrenia.
   • Newron will receive upfront and milestone payments, plus royalties on net sales in South Korea.
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4. Cyrus Biotechnology selects CYR212 for chronic IgG-driven autoimmune diseases

   • Cyrus Biotechnology has chosen CYR212, an engineered IgG protease, as its clinical candidate for chronic IgG-driven autoimmune diseases.
   • Preclinical studies showed CYR212's extended half-life and reduced immunogenicity compared to the wild type IdeS protease.
   • CYR212 aims to address over $20 billion in potential market opportunities, including diseases like myasthenia gravis and immune thrombocytopenia.
   • Cyrus plans to initiate clinical studies for CYR212, targeting indications where current anti-FcRn therapies are insufficient.
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5. Mytos announces manufacturing collaboration with Aspen Neuroscience in Parkinson’s disease

   • Mytos partners with Aspen Neuroscience to automate the manufacturing of autologous dopaminergic neuronal precursor cells (DANPCs) for Parkinson’s disease therapy.
   • Aspen will integrate Mytos’ iDEM automated cell culture technology into its new facility in Torrey Pines, California.
   • The collaboration aims to improve efficiency and scalability in cell therapy production, potentially reducing labor and increasing capacity.
   • This partnership could lead to substantial payments to Mytos over the coming years.
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6. Arkuda Therapeutics announces option exercise and asset purchase

   • Johnson & Johnson has exercised its option to acquire Arkuda's portfolio of lysosomal function enhancers.
   • Arkuda will receive an upfront payment and potential milestone payments based on development and commercial achievements.
   • The acquisition is part of an agreement with Janssen Pharmaceuticals, a Johnson & Johnson company.
   • The collaboration aims to advance small molecule enhancers of lysosomal function for neurodegenerative diseases.
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