October 11, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Genentech's Itovebi FDA Approved, Myrtelle's NAA Reduction, Pfizer's TALZENNA® Success

1. Myrtelle announces significant reduction in N-Acetylaspartate in phase 1/2 trial for Canavan disease

   • Myrtelle's phase 1/2 trial shows over 80% reduction in N-Acetylaspartate (NAA) in patients with Canavan disease.
   • The trial uses rAAV-Oligo001-ASPA gene therapy targeting oligodendrocytes to restore ASPA function.
   • MRI results indicate increased brain white matter and myelin volume post-treatment.
   • The therapy has received multiple designations from the FDA and other regulatory bodies.
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2. Arialys therapeutics begins phase 1 trial of ART5803 for autoimmune neuropsychiatric diseases

   • Arialys Therapeutics has started dosing healthy volunteers in a Phase 1 trial of ART5803, a monoclonal antibody targeting NMDA receptor autoantibodies.
   • The trial is a double-blind, placebo-controlled, single-ascending dose study in collaboration with Nucleus Network in Melbourne, Australia.
   • The study will assess the safety, tolerability, and pharmacokinetics of ART5803 in approximately 40 subjects.
   • Pending successful results, a Phase 2a trial for anti-NMDA receptor encephalitis (ANRE) is planned for the second half of 2025.
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3. FDA approves Genentech’s Itovebi for advanced breast cancer with PIK3CA mutation

   • The FDA approved Itovebi (inavolisib) in combination with palbociclib and fulvestrant for HR-positive, HER2-negative breast cancer with a PIK3CA mutation.
   • Approval is based on the Phase III INAVO120 study, showing a 57% reduction in disease progression risk compared to standard treatment.
   • The Itovebi regimen received FDA Priority Review and Breakthrough Therapy Designation.
   • Data from INAVO120 are being used for submissions to other global health authorities, including the European Medicines Agency.
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4. Pfizer’s Talzenna in combination with Xtandi prolongs overall survival in phase 3 TALAPRO-2 trial

   • Pfizer announced positive results from the TALAPRO-2 trial, showing Talzenna combined with Xtandi significantly improves overall survival in metastatic castration-resistant prostate cancer (mCRPC).
   • The trial included 1,035 patients across multiple regions, with two cohorts: all-comers and those with HRR gene mutations.
   • The combination therapy is already approved in over 35 countries, including the US and EU, for mCRPC treatment.
   • Detailed results will be presented at an upcoming medical congress and shared with global health authorities for potential label updates.
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5. Arrivo BioVentures begins phase 1 study of SP-624 for women with major depressive disorder

   • Arrivo BioVentures has initiated a Phase 1 study of SP-624, a SIRT6 activator, in healthy volunteers and patients with major depressive disorder (MDD).
   • The study aims to evaluate the epigenetic mechanism of SP-624 and its impact on neurological pathways and cognitive domains.
   • SP-624 is also in a Phase 2b study focusing on efficacy in females with MDD, following promising results from an earlier Phase 2 study.
   • The study will use Firefly Neuroscience’s BNA technology and Cambridge Cognition’s CANTAB Cognitive Test Battery to assess target engagement and cognitive changes.
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6. Oncoinvent doses first patient in phase 2 trial of Radspherin in ovarian cancer

   • Oncoinvent has initiated a Phase 2 trial for Radspherin in ovarian cancer patients with peritoneal carcinomatosis.
   • The trial is a randomized controlled study focusing on progression-free survival post-surgery and chemotherapy.
   • Radspherin is an alpha-radiation therapy designed for targeted treatment of cancers in body cavities.
   • The study is being conducted across six centers in the US, UK, Norway, Spain, and Belgium.
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7. Supernus Pharmaceuticals to host webcast on SPN-820 Phase 2a study for major depressive disorder

   • Supernus Pharmaceuticals will hold a webcast on October 17, 2024, to discuss the open-label Phase 2a study of SPN-820.
   • The study focuses on SPN-820 for the treatment of major depressive disorder.
   • Participants can pre-register for the webcast and will receive a personalized conference code.
   • A replay of the webcast will be available on the company's website for 60 days.
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