Clinical Trial News

FDA approves neffy (epinephrine nasal spray) 2 mg for Type I Allergic Reactions, including anaphylaxis, in adults and children ≥30 kg, marking the first needle-free epinephrine treatment innovation in over 35 years.

FDA approves Lymphir (denileukin diftitox-cxdl) immunotherapy for relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. Lymphir targets the interleukin-2 (IL-2) receptor on malignant T-cells and Tregs, offering rapid skin relief without cumulative toxicity. The approval is based on a Phase 3 study showing a 36.2% Objective Response Rate (ORR) with 8.7% achieving Complete Response (CR), and a median time to response of 1.4 months.

WestGene Biopharma's mRNA therapeutic cancer vaccine, WGc-043, receives dual IND approvals from China's NMPA and the US FDA, marking the world's first for EBV-related cancers. Featuring AI-assisted antigen screening and an advanced delivery system, WGc-043 targets EBV-positive solid tumors and hematologic malignancies, with promising safety and efficacy data from previous trials.

A federal mandate in 2022 required all state Medicaid programs to cover routine costs of clinical trial participation, aiming to improve equity in study participation. A study found that states with such mandates saw a 5% increase in Black or Hispanic patient enrollment in oncology trials after Medicaid expansion under the ACA. The study emphasizes the need for collaboration between states and frontline clinicians to ensure awareness and implementation of the policy change, potentially improving cancer clinical trial diversity.

Novartis receives FDA accelerated approval for Fabhalta (iptacopan) to reduce proteinuria in primary IgA nephropathy (IgAN), targeting the alternative complement pathway. Approval based on Phase III APPLAUSE-IgAN study interim analysis showing 44% reduction in proteinuria at 9 months vs. 9% with placebo. Continued approval contingent on eGFR data expected in 2025.

Aldeyra Therapeutics announces achievement of primary endpoint in Phase 3 dry eye disease trial for reproxalap, showing statistical superiority over vehicle for ocular discomfort (P=0.004).

Merck discontinues Phase 3 KeyVibe-008 trial evaluating a fixed-dose combination of vibostolimab and pembrolizumab in extensive-stage small cell lung cancer (ES-SCLC) patients due to meeting pre-specified futility criteria for overall survival (OS). Patients in the combination arm experienced higher rates of adverse events (AEs) and immune-related AEs compared to the control arm. Ongoing comprehensive analysis and further clinical development program expansions are noted.

FDA approves Zurnai (nalmefene hydrochloride) auto-injector for emergency treatment of opioid overdose, indicated for adults and patients aged 12+ with respiratory/CNS depression. Zurnai, containing 1.5 mg nalmefene per dose, is designed for immediate administration in opioid-present settings, not a substitute for emergency care. Contraindicated in hypersensitive patients. Purdue Pharma aims to provide Zurnai at no profit, addressing the opioid crisis.

FDA approves Crexont, an extended-release capsule of carbidopa and levodopa for Parkinson's disease treatment, offering more 'Good On' time with less frequent dosing and a safety profile consistent with IR CD/LD.

Johnson & Johnson announces the publication of positive Phase 2 UNITY study results of nipocalimab in The New England Journal of Medicine for treating alloimmunized pregnant individuals at risk of early onset severe hemolytic disease of the fetus and newborn (HDFN), showing 54% achieved live birth at or after 32 weeks gestational age without intrauterine transfusion (IUT), suggesting nipocalimab could be the first non-surgical treatment for high-risk HDFN pregnancies.