Clinical Trial News

Agios Pharmaceuticals shifted focus to rare genetic diseases, leading to FDA approval of Pyrukynd, the first treatment for pyruvate kinase deficiency anemia. Pyrukynd, a twice-daily tablet, showed efficacy in Phase 3 trials, offering a new option for patients previously reliant on blood transfusions. Agios plans further trials in children and other anemia types, maintaining Pyrukynd's price for five years.

Iwilfin (eflornithine), recently FDA-approved, reduces relapse risk in high-risk neuroblastoma patients. Its EMA approval is pending, with potential availability in Europe by mid-2025. Patients can access it via clinical trials or Named Patient Import before official approval.

IAG933, a novel YAP-TEAD interaction disruptor, shows promise in preclinical trials for treating Hippo pathway-altered cancers, including mesothelioma, lung, pancreatic, and colorectal cancers. It induces cell death, reduces tumor growth, and enhances efficacy when combined with other therapies. Clinical trials are underway.

An ALS drug candidate, DNL788/SAR443820, by Sanofi and Denali Therapeutics failed a Phase 2 trial, missing the main endpoint on the ALS Functional Rating Scale-Revised. Despite strong Phase 1 results, the failure questions RIPK1 inhibition's efficacy for ALS treatment. Further insights may come from an ongoing 52-week extension study. Meanwhile, other RIPK1 inhibitors and ALS treatments continue development.

Phase 3 BOND-003 study results highlight cretostimogene monotherapy's efficacy in high-risk BCG-unresponsive NMIBC, showing a 75.2% complete response rate and durable responses over 12 months. With no Grade 3+ TRAEs, it offers a bladder-sparing option, potentially improving patient outcomes and quality of life.

FDA rejected Regeneron's applications for accelerated approval due to the absence of a confirmatory study, signaling potential use of new 2022 authority, possibly facing criticism for delaying serious disease treatments.

Milestone Pharmaceuticals resubmits NDA for etripamil, a calcium channel blocker nasal spray for treating PSVT, addressing FDA concerns. If approved, etripamil could offer a self-administered treatment option for PSVT and AFib-RVR, potentially impacting millions of patients in the U.S.

Aldeyra Therapeutics plans to resubmit a New Drug Application for reproxalap, a RASP modulator for dry eye disease, with a dry eye chamber trial in H1 2024, aiming for NDA resubmission in H2 2024, contingent on trial success.

Enfortumab vedotin plus pembrolizumab significantly improves survival in untreated advanced urothelial cancer, offering a potential first-line alternative to chemotherapy. China's NMPA accepted the sBLA, marking a step towards a new treatment paradigm.

Applied Therapeutics announces FDA extension of review period for govorestat NDA by three months, new PDUFA target action date set for November 28, 2024, for treatment of Classic Galactosemia. Govorestat, an investigational ARI, demonstrated rapid and sustained reduction in galactitol with favorable safety profile in Phase 3 studies. If approved, it would be the first medication for Galactosemia.