A Phase 3 trial of the Type 2 diabetes drug Farxiga (dapagliflozin), which was being in tested in patients with chronic kidney disease, is to be stopped early on efficacy grounds. This follows a recommendation by the trial’s independent data monitoring committee. Announcing the decision on 30 March, AstraZeneca Plc said the committee had concluded that patients in the trial experienced “overwhelming benefit” from the drug.
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.
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.
A therapeutic cancer vaccine consisting of synthetic long peptides has delivered a meaningful survival benefit for cervical cancer patients when administered in combination with standard-of-care chemotherapy. The Phase 2 trial showed that the vaccine and carboplatin/paclitaxel chemotherapy were associated with prolonged survival.
A clinical study in which orally-delivered bacteria have been combined with the checkpoint inhibitor Keytruda has shown promising results in six patients with a variety of advanced cancers. The developer, 4D pharma Plc, said the data represent the first global confirmation that a live biotherapeutic product can initiate a response in cancer patients. It noted that a microbiome product combined with a checkpoint inhibitor might improve the efficacy of the checkpoint therapy.
Roche has announced plans to start a placebo-controlled Phase 3 trial of its rheumatoid arthritis drug Actemra (tocilizumab) in adults in hospital with pneumonia caused by the coronavirus. The trial is to be conducted in collaboration with the US Biomedical Advanced Research and Development Authority (BARDA). It will evaluate Actemra, plus a standard of care, compared with placebo plus a standard of care.