Clinical Trial News

The FDA issued a clinical hold on zelnecirnon trials due to a patient's liver failure, potentially linked to the drug. Dosing and new enrollments are paused, except for RAPT's tivumecirnon oncology trial. No liver toxicity was found in other participants or nonclinical studies. RAPT investigates the case, emphasizing patient safety and FDA collaboration.

Immuneering's stock rose 13% to $7.49 in premarket trading after receiving FDA fast-track designation for IMM-1-104, a treatment for pancreatic ductal adenocarcinoma, aimed at patients who have failed one line of treatment.

Mabwell's B7-H3 targeting ADC, 7MW3711, approved by FDA for advanced malignant solid tumor trials, shows promising tumor-killing effects and safety. Developed using IDDC™ platform, it features innovative components for specific tumor targeting and apoptosis induction. Mabwell, with a robust R&D system, focuses on innovative medicines across various therapeutic areas.

AstraZeneca's Tagrisso, approved by the FDA for use with chemotherapy, shows improved outcomes for EGFR-mutated non-small cell lung cancer, extending progression-free survival by nine months over monotherapy. New trial data supports its early use and effectiveness in unresectable cases, reinforcing its status as a key treatment.

Global vaccine safety study links COVID-19 vaccines to small increases in rare neurological, blood, and heart-related conditions, including heart inflammation from mRNA vaccines and brain blood clots from viral-vector vaccines.

AstraZeneca and Daiichi Sankyo's BLA for datopotamab deruxtecan (Dato-DXd) was accepted in the US for treating advanced nonsquamous NSCLC post-systemic therapy, based on TROPION-Lung01 Phase III trial results showing improved PFS over docetaxel. FDA decision expected Q4 2024. Ongoing trials aim to establish Dato-DXd as a new standard in NSCLC treatment.

BCMA-directed CAR T-cell therapy (NXC-201) for AL amyloidosis showed a 100% hematologic response rate in 9 patients, with 6 achieving complete responses. Median response time was under a month, with mild cytokine release syndrome. Despite 6 deaths at 7-month follow-up, the therapy's efficacy and safety profile suggest a promising 1-shot solution for durable responses, potentially improving organ responses and AL amyloidosis management.

Denali Therapeutics and Sanofi's ALS drug failed Phase 2 trial, missing the primary endpoint on the ALSFRS-R scale. Despite the setback, Sanofi continues testing the drug for multiple sclerosis. Denali is also developing another ALS drug, DNL343, targeting nerve cell death mechanisms.

Spectral Medical Inc. achieved a significant milestone by enrolling 90 patients in its TIGRIS trial, a step towards FDA approval for its sepsis treatment, PMX. This progress may trigger a milestone payment from Baxter, maintaining PMX's exclusive distribution rights. Spectral aims to introduce a new ICU care standard in the U.S.

A US-based study on 690 adults with chronic idiopathic constipation (CIC) found prucalopride significantly reduced constipation-related symptoms (abdominal pain, distension, nausea) and complications (hemorrhoids, intestinal obstruction, rectal bleeding) 6 months post-initiation. Significant improvements were noted in patients aged 18-64, but not in those ≥65.