Cure Rare Disease Receives FDA Feedback on Gene Therapy Program for Limb-Girdle Muscular Dystrophy Type 2I/R9

Cure Rare Disease has completed a pre-IND meeting with the FDA for its LGMDR9 gene therapy program, CRD-003. The FDA provided feedback on CMC, nonclinical development, and clinical trial design, supporting CRD's approach while offering guidance to strengthen the IND submission. The agency agreed on a 4-month murine GLP toxicology study and endorsed the proposed clinical trial design and patient selection strategy.

NRx Pharmaceuticals (NASDAQ: NRXP) has filed for the FDA's new Commissioner's National Priority Voucher (CNPV) program for NRX-100, its preservative-free ketamine formulation. The CNPV program could reduce FDA review time from 10-12 months to 1-2 months. The company's application aligns with all required criteria, including addressing the US health crisis of suicidal depression and PTSD. NRx has already submitted CMC information and received FDA feedback, maintaining its timeline for potential approval by end of 2025. The company is targeting the current $750 million ketamine market, projected to reach $3-5 billion by 2033. NRx is also filing a citizen petition to withdraw preservative-containing ketamine forms and has filed a patent for its preservative-free manufacturing process. This filing represents a potentially significant regulatory development for NRx Pharmaceuticals' ketamine formulation (NRX-100) [1].

MBX Biosciences (Nasdaq: MBX) has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MBX 4291, a long-acting glucagon-like peptide-1 (GLP-1)/ glucose-dependent insulinotropic polypeptide (GIP) receptor co-agonist prodrug for the treatment of obesity. The company's novel PEP™ platform technology aims to create a once-monthly injectable with improved gastrointestinal tolerability and increased maximal weight loss. Preclinical studies show that MBX 4291's active component has a similar activity profile to tirzepatide and supports once-monthly administration [2].

Cure Rare Disease (CRD) has completed a pre-IND meeting with the FDA for its LGMDR9 gene therapy program, CRD-003. The FDA provided feedback on CMC, nonclinical development, and clinical trial design, supporting CRD's approach while offering guidance to strengthen the IND submission. The agency agreed on a 4-month murine GLP toxicology study and endorsed the proposed clinical trial design and patient selection strategy.

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References:
[1] https://www.stocktitan.net/news/NRXP/n-rx-pharmaceuticals-inc-nasdaq-nrxp-announces-filing-of-pbtmizq19xif.html
[2] https://www.biospace.com/press-releases/mbx-biosciences-announces-ind-submission-of-mbx-4291-its-long-acting-glp1-gip-receptor-co-agonist-prodrug-for-the-treatment-of-obesity