November 20, 2024 - 🧬 [nGram] Today’s News Scoop: Neuron23's Parkinson's Trial & ALS Advancements

1. Neuron23 unveils groundbreaking phase 2 trial in early Parkinson’s disease

   • Neuron23 announced the global Phase 2 NEULARK trial for NEU-411, targeting LRRK2-driven Parkinson’s disease.
   • The trial uses a digital biomarker and patient stratification to identify those most likely to benefit from NEU-411.
   • NEU-411 showed safety and target engagement in Phase 1 trials, with potential benefits for a broader PD population.
   • The trial will utilize a novel assay and digital biomarker for precise monitoring and patient selection.
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2. Verge Genomics and Lilly advance ALS treatments with AI platform

   • Verge Genomics and Lilly are collaborating to develop ALS treatments using Verge's AI-enabled CONVERGE platform.
   • Lilly has chosen to develop two drug targets identified by Verge, triggering milestone payments to Verge.
   • The collaboration, initiated in 2021, could be worth up to $694 million, including potential downstream royalties.
   • Verge is also advancing its internal ALS drug candidate, VRG50635, currently in a Phase 1B study in Canada and Europe.
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3. Sangamo Therapeutics receives FDA clearance for ST-503 IND application

   • The FDA has cleared Sangamo's IND application for ST-503, targeting idiopathic small fiber neuropathy (iSFN).
   • ST-503 is an epigenetic regulator designed to treat chronic, intractable pain associated with iSFN.
   • The Phase 1/2 study will evaluate the safety and efficacy of ST-503, with patient enrollment expected in mid-2025.
   • ST-503 uses an AAV vector to target the SCN9A gene, reducing Nav1.7 sodium channel expression, crucial for pain signaling.
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4. Tiziana Life Sciences awarded ALS trial grant by ALS Association

   • Tiziana Life Sciences received a grant from the ALS Association for a 20-patient clinical trial.
   • The trial will evaluate intranasal foralumab, an anti-CD3 monoclonal antibody, for ALS treatment.
   • The study aims to assess safety and early-stage disease improvement parameters.
   • The trial is part of the Hoffman ALS Clinical Trial Awards Program, supporting early-stage ALS therapies.
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5. Sage Therapeutics announces topline results from the Phase 2 DIMENSION study of dalzanemdor in Huntington’s disease

   • The Phase 2 DIMENSION study evaluated dalzanemdor for cognitive impairment in Huntington’s Disease.
   • Dalzanemdor did not show a statistically significant difference from placebo on the primary endpoint, SDMT, at Day 84.
   • Secondary endpoints also showed no significant or clinically meaningful differences.
   • Sage Therapeutics will not pursue further development of dalzanemdor and will close the ongoing PURVIEW safety study.
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6. Ractigen Therapeutics receives FDA orphan drug designation for RAG-21 in ALS treatment

   • Ractigen Therapeutics' RAG-21, an siRNA therapy, has been granted FDA orphan drug designation for treating FUS-ALS, a severe ALS subtype.
   • RAG-21 targets the FUS gene, reducing toxic protein levels and addressing motor neuron degeneration.
   • Preclinical studies show RAG-21's efficacy and safety in mitigating FUS protein mis-localization and aggregation.
   • The orphan drug designation offers benefits like a 25% tax credit on clinical trials, seven-year marketing exclusivity, and a waiver of the New Drug Application fee.
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7. Neurizon's NUZ-001 reduces aggregation of key ALS disease target TDP-43 in preclinical study

   • NUZ-001 and its metabolite reduced TDP-43 aggregation by ~50% and ~55% in ALS motor neurons.
   • The treatment improved electrophysiological dysfunction in TDP-43 mutated motor neurons.
   • Findings support previous Phase 1 MEND study results and highlight NUZ-001's potential in ALS therapy.
   • Next clinical trial for NUZ-001 is planned for early 2025.
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8. Synapticure raises $25 million series A to expand virtual care for neurodegenerative diseases

   • Synapticure secured $25 million in Series A funding led by B Capital, with participation from CommonSpirit Health, CVS Health Ventures, and others.
   • The funding will be used to expand partnerships, enhance their technology platform, and fast-track clinical research.
   • Synapticure aims to provide nationwide access to expert neurologists and comprehensive care for neurodegenerative diseases.
   • Plans include scaling their national medical group and introducing new offerings through partnerships with patient advocacy organizations.
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9. Remedy Pharmaceuticals receives FDA guidance to advance CIRARA for treatment of severe stroke

   • Remedy Pharmaceuticals held a Type C meeting with the FDA on October 16, 2024, to discuss the Phase 3 trial design for CIRARA.
   • CIRARA is being developed for large hemispheric infarction (LHI), a severe form of ischemic stroke.
   • The FDA provided valuable feedback, reinforcing confidence in the trial design and CIRARA's potential.
   • Next steps involve advancing CIRARA to address the unmet needs of LHI patients.
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10. WideTrial supports NIH-funded expanded access program for investigational ALS drug

    • WideTrial is collaborating with the NIH to provide pre-approval access to ibudilast, an investigational drug for ALS.
    • The program is funded by a $22 million grant from the National Institute of Neurological Disorders and Stroke (NINDS).
    • Led by Dr. Bjorn Oskarsson at Mayo Clinic, the program aims to enroll 200 ALS patients across multiple centers.
    • The study will measure ibudilast's effect on ALS progression using neurofilament protein levels as a biomarker.
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