Clinical Trial News

Sanofi's cancer drug SAR444245, part of a $2.5B acquisition, returns to Phase 1/2 testing due to lower than expected efficacy, leading to a €1.6B impairment. The IL-2 based therapy aims for a safer, effective cancer treatment amidst competition from other biotechs innovating IL-2 therapies.

Jazz Pharmaceuticals completes BLA submission for zanidatamab, a HER2-targeted bispecific antibody, seeking accelerated approval for treatment of previously-treated HER2-positive metastatic biliary tract cancer. If approved, zanidatamab would be the first HER2-targeted treatment for this indication in the U.S. The BLA includes data from the Phase 2b HERIZON-BTC-01 trial showing a confirmed objective response rate of 41.3% in HER2-positive BTC patients, with a manageable safety profile.

Datopotamab deruxtecan's BLA accepted in the US for treating metastatic HR-positive, HER2-negative breast cancer, based on Phase III trial results showing significant PFS improvement over chemotherapy. FDA decision expected Q1 2025.

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.

FDA approved PharmaEssentia's Besremi, the first interferon therapy for polycythemia vera, a rare blood cancer. Besremi, an engineered interferon alpha, reduces bone marrow blood cell production. Approved after a 7.5-year study showing 61% achieved normal blood levels, it carries risks like neuropsychiatric disorders and fetal harm.

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.

The FACHILD study focuses on evaluating the modified Friedreich Ataxia Rating Scale (mFARS) in pediatric patients with Friedreich's ataxia (FRDA), a rare hereditary condition. The study highlights the rapid disease progression in children, the challenges in assessing disease status using mFARS, and the need for pediatric trials to explore therapeutic interventions. It also discusses the impact of the COVID-19 pandemic on data collection and the importance of FARS E (Upright Stability) as a predictor of disease progression.