Clinical Trial News

FDA Approves Crexont (carbidopa and levodopa) Extended-Release Capsules for the Treatment of Parkinson’s Disease

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.

Groundbreaking Nipocalimab Study of Pregnant Individuals at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn Published in The New England Journal of Medicine

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.

July’s Molecules on the Move

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) for the Treatment of Grade 2 IDH-Mutant Glioma

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.

Safe and Intriguing ‘Nature’s Drugs’ Taking on Complex Diseases

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 Announces GMRx2 NDA Filing to FDA

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 Topical Atopic Dermatitis Candidate Significantly Inhibits Key Cytokines in Expansion Study of Flagship Formula

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 First Participant Dosed in a Phase 1 Clinical Trial of IBI3016

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 Advisory Committee Votes Favorably that the Data Support Arimoclomol as Effective Treatment for Patients with Niemann-Pick Disease Type C

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.

IDeate-Lung02 Phase 3 Trial of Ifinatamab Deruxtecan Initiated in Patients With Relapsed Small Cell Lung Cancer

The IDeate-Lung02 Phase 3 trial evaluating ifinatamab deruxtecan in relapsed small cell lung cancer has initiated, with the first patient dosed.
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