Clinical Trial News

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.

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.

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.

Axsome Therapeutics has successfully completed its Phase 3 clinical trial, ACCORD-2, for Alzheimer’s disease agitation, achieving both primary and key secondary endpoints with significant results compared to placebo.

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.

The U.S. FDA has approved the expanded use of TRIKAFTA for treating cystic fibrosis in patients aged 2 and older with specific CFTR gene mutations, making approximately 300 more people eligible for this treatment. The approval includes updates to safety information regarding liver injury and liver failure.

New data presented at the 66th ASH Annual Meeting highlights odronextamab's potential in treating various B-cell non-Hodgkin lymphoma subtypes, showing complete responses in untreated follicular lymphoma and durable responses in diffuse large B-cell lymphoma post CAR-T therapy.

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.

FDA approved CARVYKTI® for treating relapsed/refractory multiple myeloma, reducing disease progression or death risk by 59% vs standard therapies. Based on Phase 3 CARTITUDE-4 study, it's the first BCMA-targeted therapy for early relapse, offering a potential treatment-free period.

Verastem Oncology has announced that the FDA has accepted its New Drug Application for avutometinib in combination with defactinib for the treatment of recurrent low-grade serous ovarian cancer with a KRAS mutation, granting it Priority Review with a PDUFA target action date of June 30, 2025.