Clinical Trial News

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.

George Medicines' GMRx2, a triple combination single pill for hypertension, achieved primary efficacy and safety endpoints in Phase III studies, aiming to transform treatment.

Turn Therapeutics announced positive data results of GX-03, showing significant inhibition of key cytokines in an eczema model, with a 67.7% reduction in IL-31 levels vs placebo. The candidate also demonstrated a significant reduction in ISGA score to 0.8, indicating clear to almost clear skin condition.

Innovent and SanegeneBio announce the first participant dosed in a Phase 1 clinical trial of IBI3016, an siRNA drug candidate targeting angiotensinogen (AGT) for hypertension treatment, demonstrating significant blood pressure reduction in preclinical studies. The collaboration aims to evaluate IBI3016's safety and efficacy in healthy volunteers and mild hypertension patients, with the potential to offer a more effective and safer treatment option for hypertension patients globally.

FDA's GeMDAC voted favorably (11 yes, 5 no) that arimoclomol is effective for Niemann-Pick disease type C (NPC). Zevra Therapeutics remains confident in arimoclomol's clinical benefit and its path to approval, with a PDUFA action date of September 21, 2024.