Early clinical data on a new cell therapy product to prevent organ rejection were reviewed in The Journal of Clinical Investigation on 1 May 2020 after a Phase 1b study showed the procedure to be safe and feasible. The therapy, MIC-Lx, is based on modified immune cells created from the white blood cells of a donor. It has been developed by the Heidelberg, Germany-based company TolerogenixX GmbH.
Newron Pharmaceuticals SpA has stopped development of sarizotan, a small molecule drug for Rett syndrome, after the Phase 3 STARS trial failed to meet its primary and secondary endpoints. Rett syndrome is a severe neurodevelopmental disorder, mainly affecting females, for which there are no approved treatments.
AstraZeneca Plc has partnered with the University of Oxford to develop, manufacture and distribute a candidate vaccine for COVID-19 that is based on technology originating from the university and its 2016 spin-out Vaccitech Ltd. The vaccine, ChAdOx1 nCoV-19, is in a Phase 1 clinical trial to test its safety and efficacy in healthy volunteers. Initial results from the study are expected to be reported in June.
A Phase 3 study of the antiviral remdesivir in patients with advanced COVID-19 has delivered positive results, the first indication that a treatment for the disease may be within reach. Preliminary data from the trial were disclosed on 29 April by the sponsor, the US National Institute of Allergy and Infectious Diseases.
A Phase 3 lung cancer trial of the checkpoint antibody Libtayo (cemiplimab) has been stopped early due to a significant improvement in overall survival, the developers Sanofi SA and Regeneron Pharmaceuticals Inc announced on 27 April. Libtayo is a programmed death receptor-1 (PD-1) blocking antibody that frees the immune system to fight cancer. The drug has already been approved in the US to treat advanced cutaneous squamous cell carcinoma.
Lynparza (olaparib) has demonstrated another benefit for patients with a specific genetic mutation – this time in a Phase 3 trial of men with metastatic castration-resistant prostate cancer. Lynparza is a poly ADP ribose polymerase (PARP) inhibitor that is already approved for ovarian, breast and pancreatic cancers.
A second pharma partnership is poised to start a human trial of a candidate vaccine for COVID-19 using messenger RNA (mRNA) technology. Germany-based BioNTech SE and Pfizer Inc will test BioNTech’s BNT162 programme in approximately 200 healthy subjects between the ages of 18 and 55. Permission to start the trial was granted by the German regulator, the Paul-Ehrlich-Institut, the companies announced on 22 April.
A partnership that brings together industry, pharmaceutical regulators and the US National Institutes of Health (NIH) is being created to speed up the development of therapies and vaccines for COVID-19. The planned alliance is the work of the NIH and its sister foundation, the Foundation for the NIH.
The German biotech BioNTech SE has joined forces with Pfizer Inc to co-develop a candidate vaccine against infection from the coronavirus SARS-CoV-2 which had affected 1.7 million people globally as of 12 April and resulted in 105,952 deaths.
A Phase 3 trial of the lung cancer drug Tagrisso (osimertinib) will be unblinded early on the recommendation of the data monitoring committee on evidence of overwhelming efficacy, the developer AstraZeneca Plc announced on 10 April. Tagrisso, a tyrosine kinase inhibitor targeting the epidermal growth factor receptor (EGFR), has already been approved for first and second-line use in patients with advanced non-small lung cancer. The Phase 3 trial, called ADAURA, was treating patients with early-stage disease. The primary endpoint of the trial is disease-free survival.