August 8, 2024 - 🧬 [nGram] Today’s Neurology Scoop: Neurogene's RMAT for Rett, Amneal's FDA Approval, ANEW's Patents

1. Neurogene announces RMAT designation for NGN-401 investigational gene therapy for Rett syndrome

   • NGN-401 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for Rett syndrome.
   • The designation is based on preliminary clinical evidence from the ongoing Phase 1/2 trial showing potential to address unmet medical needs.
   • RMAT designation includes benefits like early communications with FDA, intensive guidance, and eligibility for Accelerated Approval and Priority Review.
   • Interim efficacy data from the low-dose cohort is expected in Q4 of this year, with additional data from the high-dose cohort anticipated in the second half of 2025.
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2. Amneal receives U.S. FDA approval for IPX203 for treatment of Parkinson’s disease

   • Amneal Pharmaceuticals received FDA approval for CREXONT (carbidopa and levodopa) extended-release capsules for Parkinson’s disease.
   • CREXONT combines immediate-release granules and extended-release pellets, providing longer 'Good On' time with less frequent dosing.
   • The RISE-PD clinical trial showed CREXONT offers 0.5 hours more 'Good On' time per day compared to immediate-release CD/LD.
   • Amneal plans to launch CREXONT in the U.S. in September 2024.
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3. ANEW Medical announces patents issued in major Asian markets for genetic therapy in Alzheimer's and ALS

   • Patents issued in mainland China, Hong Kong, and Shanghai protect ANEW's Klotho protein and gene delivery system.
   • The patents cover treatments for Alzheimer's, Parkinson's, and ALS.
   • ANEW plans to begin human testing of these therapies in late 2025.
   • The intellectual property was licensed from Universitat Autònoma de Barcelona and ICREA.
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4. Rapport Therapeutics reports second quarter financials and provides business update

   • Completed IPO, raising $174.4 million in gross proceeds to fund clinical development.
   • Phase 2a trial of RAP-219 in focal epilepsy initiation on track, with topline data expected in mid-2025.
   • Two additional proof of concept trials for RAP-219 in peripheral neuropathic pain and bipolar disorder planned for 2H 2024 and 2025, respectively.
   • Ended the quarter with $336.1 million in cash, expected to fund operations through the end of 2026.
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5. Cognition Therapeutics reports financial results for Q2 2024 and provides business and clinical update

   • Phase 2 SHINE trial of CT1812 in mild-to-moderate Alzheimer's disease showed ~40% mean improvement in ADAS-Cog 11 vs placebo.
   • Topline results from SHIMMER study in mild-to-moderate dementia with Lewy bodies expected by year-end 2024.
   • Continued progress in Phase 2 START study of CT1812 in early Alzheimer's disease and Phase 2 MAGNIFY study in geographic atrophy secondary to dry AMD.
   • Cash and cash equivalents as of June 30, 2024, were approximately $28.5 million, with sufficient funds to operate into Q2 2025.
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6. ProMIS Neurosciences announces second quarter 2024 financial results and recent highlights

   • Reported positive topline data from Phase 1a clinical trial of PMN310 for Alzheimer’s disease, meeting objectives for tolerability, safety, and pharmacokinetics.
   • Secured up to $122.7 million in private placement financing to advance Phase 1b study of PMN310 in Alzheimer’s disease patients in the second half of 2024.
   • Phase 1a trial showed PMN310 to be generally safe and well-tolerated, with no treatment-emergent serious adverse events observed.
   • ProMIS continues to advance its amyloid beta vaccine program in Alzheimer’s disease and presented preclinical data at the Alzheimer’s Association International Conference.
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7. Trevena reports second quarter 2024 results and provides business update

   • TRV045 demonstrated sustained long-term analgesic effect and differentiated mechanism of action in preclinical models.
   • Completed $12 million financing, including a non-dilutive $2 million tranche and $10 million reduction in liabilities.
   • Ongoing strategic review of OLINVYK, exploring potential alternatives such as sale, license, or discontinuation of US commercial sales.
   • Reported a net loss of $4.9 million for Q2 2024, compared to $8.0 million in Q2 2023.
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