Roche has stopped dosing patients in a Phase 3 study of an investigational drug for Huntington’s disease, a rare genetic condition that causes nerve cells in the brain to break down. The drug, tominersen, is an antisense therapy designed to reduce the production of all forms of huntingtin protein, including its mutated variant. The trial was the largest study to date in patients with this disease.
An independent data and safety monitoring board (DSMB), which had oversight over AstraZeneca’s latest Covid-19 vaccine trial, has expressed concern that the company may have included outdated information from the trial when it reported an efficacy rate of 79% for the vaccine on 22 March. The DSMB concern was cited in a press release issued by the National Institute of Allergy and Infectious Diseases (NIAID), an agency of the US National Institutes of Health, on 23 March.
The Astra Zeneca/Oxford University vaccine (AZD1222) has shown an efficacy rate of 79% in preventing symptomatic Covid-19 disease and 100% efficacy at preventing severe disease and hospitalisation, AstraZeneca announced on 22 March. The interim data come from a Phase 3 trial conducted in the US which enrolled 32,449 participants. Speaking to reporters, company executives said data from the trial will be filed shortly with the Food and Drug Administration with a view to gaining an emergency authorisation and starting US distribution soon thereafter.
A Phase 3 trial of the checkpoint inhibitor Libtayo (cemiplimab) is to be stopped early after showing an improved overall survival in patients with cervical cancer, reducing the risk of death by 31% compared with chemotherapy, Regeneron Pharmaceuticals Inc and Sanofi SA announced on 15 March. The companies plan regulatory submissions in 2021.
One of several clinical studies of canakinumab in non-small cell lung cancer has failed to meet its primary endpoint, but other studies in the programme are continuing, Novartis announced on 9 March 2021. Canakinumab is a monoclonal antibody designed to neutralise the bioactivity of human interleukin-1beta (IL-1beta). The Phase 3 CANOPY-2 trial, which was evaluating canakinumab in combination with the chemotherapy agent docetaxel, did not meet its primary endpoint of overall survival.
An experimental oncolytic virus therapy in clinical development by Targovax ASA has continued to show a survival benefit for patients with malignant pleural mesothelioma 21 months after treatment. At follow-up, half of the patients in the first-line treatment group were still alive and median overall survival had not been reached, the company announced on 23 February.
A small molecule drug being developed by Immunic Inc has shown evidence of clinical activity in hospitalised patients with moderate Covid-19 disease, the company announced on 17 February. The finding, from a Phase 2 trial, suggests that the drug, IMU-838, may have application in patients aged over 65 years, as well as preventing long-term Covid-19 symptoms such as fatigue.
Lynparza (olaparib) has passed an important threshold in a Phase 3 trial of patients with early breast cancer with the result that it will undergo a primary analysis for efficacy earlier than planned. This follows a recommendation by the trial’s independent data monitoring committee which concluded that the drug has the potential to achieve a sustainable and clinically relevant treatment effect in patients, according to the sponsors AstraZeneca Plc and Merck & Co Inc.
Bluebird bio Inc announced on 16 February the temporary suspension of two gene therapy trials following reports of two cases of cancer. The trials were being conducted in support of a prospective gene therapy for sickle cell disease. The company is investigating the cause of the cancers in order to determine if there is any relationship with the lentiviral vector used in the studies.
A vaccine developed by Russia to prevent coronavirus disease has been shown to be 91.6% effective, according to data from a Phase 3 trial published in The Lancet on 2 February 2021. The vaccine, Gam-COVID-Vac (Sputnik V), is a combined vector vaccine carrying the gene for the SARS-CoV-2 full length glycoprotein S. It is intended to elicit antibodies to the virus glycoprotein as well as antigen-specific cellular immunity.