September 4, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Roche’s MS Breakthrough, FDA Orphan Drug for ALS, Glioblastoma Therapy

1. IPAX-1 study of TLX101 investigational glioblastoma therapy published in Neuro-Oncology Advances

   • The IPAX-1 Phase I study of TLX101 in combination with external beam radiation therapy (EBRT) for recurrent glioblastoma (GBM) has been published in Neuro-Oncology Advances.
   • The study confirmed the safety and tolerability of TLX101, with a median overall survival of 13 months from treatment initiation.
   • Key findings include a 44.4% response rate at 3 months post-treatment and no radiation-based toxicity.
   • The results support further investigation of TLX101 plus EBRT, including its potential as a first-line treatment.
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2. Actimed Therapeutics receives orphan drug designation for S-oxprenolol in ALS

   • The US FDA has granted Orphan Drug Designation to Actimed Therapeutics' S-oxprenolol (ACM-002) for treating amyotrophic lateral sclerosis (ALS).
   • ALS is a progressive neuromuscular disease leading to muscle atrophy, weakness, and eventual paralysis, with no current cure.
   • Pre-clinical models show S-oxprenolol may slow disease progression, improve survival, and protect motor neurons.
   • Orphan Drug Designation offers benefits like development cost reductions, increased FDA interaction, and seven-year market exclusivity post-approval.
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3. Roche’s fenebrutinib shows promising results in relapsing multiple sclerosis

   • New Phase II data shows fenebrutinib suppresses disease activity and disability progression for up to 48 weeks.
   • 96% of patients were relapse-free with an annualized relapse rate of 0.04.
   • MRI scans revealed 99% of patients were free of T1 gadolinium-enhancing lesions.
   • The safety profile was consistent with previous trials, with common adverse events including urinary tract infection and COVID-19.
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4. Obsidian Therapeutics receives FDA RMAT designation for OBX-115 for advanced melanoma

   • Obsidian Therapeutics announced FDA RMAT designation for OBX-115 for treating unresectable or metastatic melanoma resistant to immune checkpoint inhibitor therapy.
   • OBX-115 is an engineered tumor-derived autologous T cell immunotherapy, regulated using the FDA-approved drug acetazolamide.
   • Initial Phase 1 data presented at the 2024 ASCO Annual Meeting showed a favorable safety profile and encouraging efficacy in heavily pre-treated patients.
   • RMAT designation provides benefits like early and frequent FDA interactions, intensive guidance, and potential eligibility for Accelerated Approval and Priority Review.
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5. Prilenia’s pridopidine for Huntington’s disease accepted for European marketing authorisation review

   • Prilenia Therapeutics' Marketing Authorisation Application (MAA) for pridopidine has been accepted by the European Medicines Agency (EMA).
   • Pridopidine is a highly selective sigma-1 receptor agonist aimed at treating adults with Huntington’s disease (HD).
   • The MAA is based on extensive safety and efficacy data from pridopidine’s development program.
   • Prilenia is also in discussions with the FDA for potential approval in the U.S. and plans to consider regulatory submissions in other regions.
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6. NH TherAguix announces continuation of the phase II NANOBRAINMETS trial in the treatment of brain metastases

   • The DSMB has validated the continuation of the Phase II NANOBRAINMETS trial after a futility analysis.
   • No serious adverse events related to AGuIX® were reported, indicating a favorable safety profile.
   • 96 out of 134 patients have been randomized in the study so far.
   • Next interim analysis is expected by the end of 2024 to assess the clinical efficacy of AGuIX®.
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7. Vesper Bio announces successful Phase I study for potentially disease-modifying treatment for frontotemporal dementia

   • VES001 is a first-in-class oral, brain-penetrant, small molecule sortilin inhibitor for treating FTD(GRN).
   • Phase I study in 78 healthy volunteers showed excellent safety, tolerability, and target engagement.
   • Data demonstrated dose-proportionality and significant elevations in progranulin levels in both plasma and CNS.
   • Clinical trial application filed for Phase IIa study, with first dosing targeted for Q4 2024.
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8. Avenzo Therapeutics announces clinical study collaboration with Gilead Sciences to evaluate AVZO-021 and Trodelvy in HR+/HER2- metastatic breast cancer

   • Avenzo Therapeutics and Gilead Sciences enter a clinical study collaboration to evaluate AVZO-021 and Trodelvy.
   • The study will focus on HR+/HER2- metastatic breast cancer.
   • Gilead will provide Trodelvy, while Avenzo will conduct and sponsor the study.
   • The Phase 1b portion of the ongoing clinical study is set to begin in Q4 2024.
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9. Exciting developments in clinical research: Axcellant's groundbreaking global study

   • Axcellant, a European CRO, has launched a groundbreaking clinical trial in nuclear medicine.
   • The trial aims to innovate diagnostics for over 20 million patients in the US and over 200 million worldwide.
   • The study will be conducted at about 20 sites across the US, including the East Coast and Midwest.
   • FDA has approved the clinical development plan, and the first manufacturing sites will be activated in the coming months.
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10. Immunic enrolls first patient in phase 2 trial of vidofludimus calcium for post COVID syndrome

    • The trial is sponsored by Goethe University Frankfurt and funded by a German government grant.
    • Vidofludimus calcium aims to suppress Epstein-Barr virus reactivation and reduce fatigue symptoms.
    • The study will enroll 376 patients across 11 clinical sites in Germany.
    • Primary endpoint: change in physical function as measured by SF-36-PF from baseline to day 56.
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