Clinical Trial News

A meta-analysis reveals that combining TACE with tyrosine kinase inhibitors (TKIs) and immune checkpoint inhibitors (ICIs) significantly improves treatment outcomes for hepatocellular carcinoma (HCC) patients, compared to TACE plus TKIs alone. This combination therapy enhances the objective response rate, progression-free survival, and overall survival, albeit with a higher incidence of hypertension.

CAR T cell therapy, initially successful against blood cancers, shows early promise in treating lupus, an autoimmune disease. A German study reported remission in lupus patients post-treatment, suggesting broader applications for CAR T therapies beyond cancer, with potential for lower doses and fewer side effects.

Supernus receives a Complete Response Letter from FDA for SPN-830, an apomorphine infusion device for Parkinson’s disease, citing product quality and infusion device master file issues. No clinical safety or efficacy issues identified. Company remains committed to addressing the CRL and resubmitting the NDA.

Stealth BioTherapeutics announces FDA acceptance of New Drug Application for elamipretide, a potential treatment for Barth syndrome, supported by positive Phase 3 data. The FDA plans to hold an advisory committee meeting to discuss the application. Elamipretide has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations.

The FDA accepted Shorla Oncology's NDA for SH-201, an oral leukemia drug, with a decision due by 30 November 2024. SH-201, acquired in July 2023, offers an alternative for patients struggling with traditional dosage forms. Shorla, bolstered by $35m in Series B funding, aims to expand its oncology portfolio, including SH-201 and previously approved SH-111.

UC Davis Health's cardiology team is among the first in the U.S. to treat tricuspid regurgitation using the Abbott TriClip™ system, a groundbreaking catheter-based device. This minimally invasive procedure, known as transcatheter edge-to-edge repair (TEER), offers patients a safer alternative to traditional surgery with significant improvements in quality of life.

Shorla Oncology announced FDA acceptance of its NDA for SH-201, a palatable oral liquid to treat certain leukemias and cancers, with a PDUFA action date of November 30, 2024. SH-201 aims to provide an alternative treatment for patients lacking oral liquid options, marking a significant step for the company and those affected by these diseases.

A phase 1 clinical trial of an mRNA-based cancer vaccine, autogene cevumeran, has shown potential in stimulating a lasting immune response against pancreatic cancer, with some patients showing no recurrence up to three years post-treatment. The vaccine, developed through a collaboration between BioNTech and Genentech, targets neoantigens specific to each patient's tumor, aiming to train the immune system to recognize and attack cancer cells. A phase 2 trial is underway to further evaluate the vaccine's efficacy and safety.

Stoke Therapeutics has achieved alignment with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the EMPEROR study, focusing on zorevunersen's effects on major motor seizure frequency, behavior, and cognition in children and adolescents.

PTC Therapeutics, Inc. has announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin, aimed at treating pediatric and adult patients with phenylketonuria (PKU). This marks a significant step towards providing a new treatment option for PKU patients, supported by promising clinical trial data.