The Zolgensma gene therapy for spinal muscular atrophy, which was approved by the US Food and Drug Administration in May 2019, has now received a positive review by the European Medicines Agency. Pending approval by the European Commission, it is expected to be available to treat babies and young children across the EU soon. Spinal muscular atrophy is a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.
Llft BioSciences Ltd of the UK has teamed up with King’s College London to create a new version of its cell therapy for cancer which uses a special type of neutrophil to attack solid tumours. The cell therapy is still in preclinical development, but the company will work with the university to develop a new version using induced pluripotent stem cells (iPSCs). It believes the iPSC version will be easier to manufacture and will deliver significant cost savings to patients. Clinical trials of the new therapy are expected to start in 2022.
Evotec SE reported 19% higher revenue in 2019 to €446.4 million on the expansion of its drug discovery service business and milestones from its pharma partnerships. The company made a strategic move into biologics during the year with the acquisition of Just Biotherapeutics Inc in the US and also launched a new company to develop cancer therapies based on DNA damage repair.
Silence Therapeutics Plc has entered into a strategic collaboration with AstraZeneca Plc to develop and commercialise small interfering RNA (siRNA) therapeutics for a number of therapeutic areas – reviving a relationship that goes back more than 10 years. The two companies have agreed to develop siRNA molecules, which tell the body to eliminate or ‘silence’ instructions for making disease-causing proteins. These molecules will be directed against cardiovascular, renal, metabolic and respiratory disorders.
Positive data has been reported for the cancer drug Venclexta (venetoclax) in combination with azacitidine chemotherapy in a Phase 3 trial of patients with previously untreated acute myeloid leukaemia (AML), according to the co-developers AbbVie Inc and Roche. The trial, VIALE-A and a second study, VAILE-C, are being conducted in order to confirm an accelerated approval of Venclexta for AML which was issued by the US Food and Drug Administration in 2018.
Pharmaceutical regulators from 17 countries have agreed in principle on the criteria that must be met before developers of candidate vaccines for the coronavirus SARS-CoV-2 start human trials. The officials held a virtual meeting on 18 March, and a summary of their conclusions was released by the European Medicines Agency on 24 March.
Human macrophages that were genetically engineered with chimeric antigen receptors (CARs) have demonstrated an anti-cancer effect in mice, suggesting a new application for cell therapy in solid tumours. Results from preclinical studies of the new molecule were published in Nature Biotechnology on 23 March 2020.
The European Medicines Agency has issued a statement urging the research community to give priority to large randomised controlled studies when investigating potential treatments for COVID-19 and to include all EU countries in these trials.
New guidance on the conduct of clinical trials during the COVID-19 pandemic has been published by the European Commission, the European Medicines Agency and heads of national agencies in the EU. https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-10/guidanceclinicaltrials_covid19_en.pdf, Developers including Eli Lilly and Co, have already announced delays in the start of studies and a pause in enrollment of ongoing studies.
Seven scientists drawn from six EU member states met for the first time on 18 March to advise the European Commission on how to respond to the multitude of issues facing public health authorities during the coronavirus pandemic. Among the topics discussed were managing hospital capacity, the conduct of new clinical trials, and the protection of vulnerable populations such as refugees.