Two US institutions have taken steps to simplify the regulation of gene therapy, and bring the oversight of these technologies into the existing framework for monitoring experimental medicines. The shift in policy is described by Francis Collins, director of the National Institutes of Health, and Scott Gottlieb, commissioner of the Food and Drug Administration, in an article in the New England Journal of Medicine on 15 August 2018.
AstraZeneca Plc has announced that a Phase 3 trial of its proposed antibody treatment for systemic lupus erythematosus (SLE) failed to meet the trial’s primary endpoint of showing a statistically-significant reduction in disease activity as measured at 12 months by a responder index. Called TULIP 1, the trial was a double-blinded, 52-week placebo-controlled study evaluating the safety and efficacy of anifrolumab as a treatment for adult patients with moderate-to-severe SLE.
Pfizer has stopped two clinical studies that were evaluating a proposed antibody treatment for Duchenne muscular dystrophy (DMD) because of lack of efficacy. The first study was a Phase 2 trial testing the treatment for safety and efficacy, while the second was an open-label extension study, the company announced on 30 August.
The glucagon-like peptide-1 (GLP-1) analogue semaglutide has demonstrated an ability to promote weight loss in people without diabetes, in some cases up to 13.8%, when combined with diet and exercise. The data comes from a Phase 2 trial comparing semaglutide with liraglutide, another GLP-1 analogue, and with placebo.
Roche’s Genentech has committed funding of $96 million to Affimed NV for rights to the German company’s biologics platform for the production of cancer therapies targeting natural killer (NK) cells in the innate immune system. The platform can generate antibodies directed against NK cells as well as against T cells of the adaptive immune system.
A study in Plos Biology by researchers from the Helmholtz Zentrum München and the Ludwig-Maximilians-Universität München has described a metabolic network in mice that is not controlled by the brain but rather by the ‘circadian clock’ inside muscle cells.
The inference of this discovery, reported on 10 August, is that diseases like diabetes and obesity are influenced by the body’s internal clock rather than just the endocrine system or the brain.
Scientists in Vienna, Austria have used organoid technology to grow brain tumours in the laboratory, enabling them to replicate the process of brain carcinogenesis, potentially making the discovery of new biomarkers and treatments for these cancers easier.
Novartis has reported positive Phase 3 data from a trial investigating a new treatment for breast cancer targeting a protein in the PI3K pathway, a signalling pathway for human cancers. The compound, BYL719 (alpelisib), showed an improvement in progression-free survival.
AstraZeneca Plc’s treatment for chronic obstructive pulmonary disease (COPD), Bevespi Aerosphere, has shown non-inferiority but not superiority, on one respiratory measure in a Phase 3b trial in which it was compared with a similar treatment from GlaxoSmithKline Plc.
On a second respiratory measure, it failed to show non-inferiority.
Novo Nordisk A/S has entered a research alliance with Evotec AG to discover and develop new small molecule therapies for diabetes and obesity, diseases that affect an estimated one billion people worldwide. Financial terms of the agreement weren’t disclosed.