Clinical Trial News

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.

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

FDA approves Jemperli plus chemotherapy for all adult patients with primary advanced or recurrent endometrial cancer, including those with MMRp/MSS tumors, based on RUBY phase III trial results showing significant OS benefit.

BioNTech announces positive Phase 2 results for mRNA immunotherapy BNT111 in advanced melanoma patients, showing significant improvement in ORR when combined with cemiplimab vs. historical control, with both monotherapies active. BNT111, part of BioNTech's FixVac platform, targets four tumor-associated antigens and has received FDA Fast Track and Orphan Drug designations for melanoma treatment.

Vertex's suzetrigine, an oral NaV1.8 pain signal inhibitor, has been accepted by the FDA for priority review to treat moderate-to-severe acute pain, with a PDUFA target action date of January 30, 2025.

FDA approves Darzalex Faspro (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for induction and consolidation in newly diagnosed multiple myeloma (NDMM) patients eligible for autologous stem cell transplant (ASCT), supported by Phase 3 PERSEUS study data showing significant improvement in progression-free survival (PFS) and deeper responses with D-VRd compared to VRd.

The FDA extended the PDUFA action date for Syndax Pharmaceuticals' NDA for revumenib, a treatment for relapsed or refractory KMT2Ar acute leukemia, to December 26, 2024, due to a major amendment submission. Revumenib aims to address significant unmet needs in KMT2A-rearranged acute leukemia patients.

FDA approves Zunveyl (benzgalantamine) for mild-to-moderate Alzheimer's disease, a novel oral therapy with a dual mechanism of action designed to improve tolerability and efficacy, addressing therapy adherence issues due to GI side effects and insomnia.

Nuvalent's NVL-520, an oral, brain-penetrant, TRK-sparing ROS1 kinase inhibitor, entered Ph. II of ARROS-1 trial for advanced ROS1-positive NSCLC, showcasing a potential best-in-class profile.