The US Food and Drug Administration has extended a clinical hold on a proposed Phase 3 clinical trial of a microbiome-based therapeutic developed by MaaT Pharma SA of France because it requires more information about the composition of the faecal-derived product. MaaT Pharma announced the clinical hold extension on 10 August.
Alnylam Pharmaceuticals Inc has reported positive Phase 3 data for its RNAi therapeutic patisiran which is being developed for ATTR amyloidosis with cardiomyopathy, a disease caused by the build-up of abnormal proteins in tissues of the heart. The therapy works by silencing the transthyretin messenger RNA, thereby inhibiting the synthesis of transthyretin protein. The disease affects the heart’s ability to function properly leading to shortness of breath and an irregular heart beat.
A small molecule drug designed to improve lung function has met the primary and secondary endpoints in a Phase 3 trial of patients with chronic obstructive pulmonary disease (COPD), the developer Verona Pharma Plc announced on 9 August. The drug, ensifentrine, is a dual inhibitor of the enzymes phosphodiesterase 3 and 4, a mode of action that enables it to combine both bronchodilator and anti-inflammatory effects in a single compound.
A Phase 3 trial of a combination therapy intended to improve the standard of care for the most common type of liver cancer failed to meet its dual primary endpoints, Merck & Co Inc and Eisai Co Ltd announced on 3 August. The trial was investigating Keytruda (pembrolizumab) and Lenvima (lenvatinib) as a first-line treatment for patients with unresectable hepatocellular carcinoma versus Lenvima alone. In the trial, there were trends towards an improvement in overall survival and progression-free survival but these trends did not meet statistical significance, the companies said.
A Phase 3 trial of an experimental checkpoint inhibitor for head and neck cancer has been ended for lack of efficacy, the developer Innate Pharma SA of France announced on 1 August. The drug, monalizumab was being studied in combination with cetuximab (Erbitux) versus placebo plus cetuximab in patients with metastatic disease who had previously had chemotherapy and another checkpoint inhibitor. Despite promising signals in an earlier Phase 1b/2 study, the Phase 3 trial did not meet a pre-defined threshold for efficacy.
NOXXON Pharma NV has updated its drug development strategy in light of further positive data for its aptamer drug NOX-A12 in patients with glioblastoma. The company’s resources will now be fully deployed to advance NOX-A12 towards registration. Other research projects will be put on hold, the company announced on 23 June 2022.
Faron Pharmaceuticals Ltd of Finland has reported top-line, 12 month survival data for its antibody drug bexmarilimab showing that a significant number of patients who benefited from the treatment were still alive at 12 months. The Phase 1/2 MATINS study is investigating the safety and efficacy of bexmarilimab monotherapy in 10 different metastatic or inoperable solid tumour cohorts.
A Phase 2 study of an experimental Alzheimer’s disease treatment designed to slow or prevent cognitive decline in people carrying a specific genetic mutation failed to meet its primary endpoints, the Roche Group announced on 16 June. The placebo-controlled trial was evaluating the monoclonal antibody crenezumab as a potential treatment for early-onset Alzheimer’s in people carrying the Presenilin 1 E280A mutation.
A France-based company advancing a therapy for both solid tumours and haematologic malignancies has raised €96 million in a Series C financing round to establish clinical proof-of-concept for its lead antibody molecule. ImCheck Therapeutics SAS is located in Marseille and supported by a large group of investors including Andera Partners, Earlybird Venture and a philanthropy fund managed by the US Leukemia and Lymphoma Society.
New data for Kymriah (tisagenlecleucel), the first chimeric antigen receptor (CAR) T cell therapy to be approved for a cancer, have shown durable remission and long-term survival for the children and young adults treated with the therapy. The developer, Novartis, presented the data at the 2022 European Hematology Association Hybrid Congress on 12 June.