The Roche group has acquired commercialisation rights, outside the US, for an experimental gene therapy for Duchenne muscular dystrophy, a rare degenerative neuromuscular disorder. The deal was announced on 23 December and is with Sarepta Therapeutics Inc. It comes only days after Roche received regulatory clearance to acquire the gene therapy company Spark Therapeutics Inc.
Genmab A/S has expanded its antibody repertoire under a new research and collaboration agreement with CureVac AG to produce antibody therapeutics for cancer that have been developed using messenger RNA (mRNA) technology. The agreement includes an upfront payment of $10 million for the first product and a €20 million equity investment in CureVac, as well as options on three other programmes. The deal was announced on 19 December 2019.
Boehringer Ingelheim GmbH has encountered a setback in its plans to develop multiple products for non-alcoholic steatohepatitis (NASH). A small molecule drug in-licensed from Pharmaxis Ltd in Australia is being dropped from development because of the risk of drug interactions, the German company said on 18 December.
A large observational study of patients with atrial fibrillation treated with the cardiovascular drug Lixiana (edoxaban), has confirmed that the drug is noninferior to warfarin but generates fewer bleeding episodes, according to the developer, Daiichi Sankyo Co Ltd.
A combination therapy directed at patients with advanced non-squamous non-small cell lung cancer, failed to show a clinically meaningful overall response rate in a Phase 2 clinical trial, the developer Transgene SA announced on 12 December. Transgene has stopped further development of the compound TG4010, which is a cancer vaccine based on a modified Vaccinia virus.
The antibody-drug conjugate belantamab mafodotin has generated an overall response rate of 31% in a pivotal Phase 2 study of patients with relapsed/refractory multiple myeloma, GlaxoSmithKline Plc announced on 17 December. The UK company has made a regulatory submission for the drug with the US Food and Drug Administration.
The EU is to invest €10 million in a project to expand the use of structural biology in drug discovery and other disciplines. The project is being coordinated by Anastassis Perrakis from the Netherlands Cancer Institute and the Oncode Institute and will involve partners from other countries.
Novartis is to discontinue development of the small molecule drug fevipiprant as a treatment for asthma following the failure of two Phase 3 studies to show a reduction in the rate of moderate-to-severe exacerbation compared with placebo. A pooled analysis of the LUSTER-1 and LUSTER-2 studies did not meet the clinical threshold of efficacy for either of the two doses of the drug over a period of 52 weeks.
“The totality of these results do not support further development of fevipiprant in asthma,” the company said in a statement on 16 December.
Promethera Biosciences SA has raised an additional €7.5 million for its Series D financing round bringing total proceeds up to €39.7 million. The financing was led by new investors Sony Innovation Fund by IGV and Pegasus Tech Ventures. It will be used to further advance the company’s clinical programmes in non-alcoholic steatohepatitis (NASH) and acute-on-chronic liver failure as well as to support the company’s growth in Asia.
The US Food and Drug Administration has approved a new drug for Duchenne muscular dystrophy (DMD) on the basis of data showing that the medicine increased the production of the dystrophin protein in patients – a surrogate for improved motor function. The drug, Vyondys 53, was developed by Sarepta Therapeutics Inc, whose first DMD drug Exondys 51 was approved by the FDA in 2016, also on the basis of a surrogate endpoint.