Clinical Trial News

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.

In May 2024, major transactions advanced neuroplastogens, KRAS G12C inhibitors, Pompe disease treatments, and molecular glues. Positive clinical readouts were seen for TTR stabilizers, Hedgehog pathway inhibitors, plasma kallikrein inhibitors, DPP-1 inhibitors, and PCSK9 inhibitors.

FDA approves Voranigo (vorasidenib), an IDH1 and IDH2 inhibitor, for treating Grade 2 IDH-mutant glioma in patients 12+ years old post-surgery. Voranigo offers a once-daily pill for disease management, with Phase 3 INDIGO trial results showing significant progression-free survival extension and safety.

Intrinsic Medicine, led by CEO Alex Martinez, focuses on developing human milk oligosaccharides (HMOs) to treat chronic diseases like Parkinson’s and IBS, addressing the high cost of drug development and the shift towards orphan drugs. Their lead compound, 2’ Fucosyllactose (2’FL), has received approval in Australia for a phase 2 trial in Parkinson’s and IBS, leveraging the gut immune brain axis (GIBA) for disease treatment.