A new epigenetic medicine has been approved in the US to treat epithelioid sarcoma, a rare sub-type of soft tissue sarcoma that affects young adults. The drug, Tazverik (tazemetostat), is a small molecule inhibitor of EZH2 methyltransferase which regulates gene expression. By blocking the activity of EZH2, the drug is expected to keep cancer cells from growing. The Food and Drug Administration announced the authorisation on 23 January.
A new drug has been approved in the US for thyroid eye disease, a rare condition in which the muscles and fatty tissues behind the eye become inflamed, causing the eyes to bulge. Tepezza (teprotumumab) is a monoclonal antibody that targets the insulin-like growth factor-1 receptor with the goal of modifying the course of the disease. In issuing the approval, the Food and Drug Administration said Tepezza is the first non-surgical treatment for thyroid eye disease, which can cause eye pain, double vision and sensitivity to light.
A Phase 3 trial of a lipid-lowering drug involving more than 13,000 patients has been discontinued after the study’s monitors concluded the medicine was unlikely to deliver benefits to patients. The sponsor, AstraZeneca Plc, was attempting to show that patients with mixed dyslipidaemia who are at risk of cardiovascular disease could be helped by Epanova (omega-3 carboxylic acids), a compound derived from fish oil. The patients were all on optimal statin therapy. But the independent data monitoring committee concluded that a benefit was unlikely.
Germany-based Medigene AG has reported positive top-line data from an early clinical study of a dendritic cell vaccine for cancer which showed both safety and tolerability as well as early signs of efficacy. The vaccine was administered to 20 patients with acute myeloid leukaemia, of whom half were 60 years of age or older. This patient cohort is often ineligible for haematopoietic stem cell transplantation and has poorer treatment outcomes, the company said. The balance of patients were younger than 60 years.
A Phase 3 study of the checkpoint antibody Tecentriq (atezolizumab) did not meet its primary endpoint of disease-free survival compared with observation in patients with muscle-invasive urothelial cancer, the developer Roche announced on 24 January.
An experimental treatment for Type 1 spinal muscular atrophy (SMA) has reached its primary endpoint in a pivotal trial of infants, paving the way for a US regulatory decision by late May, according to the developer Roche. The drug, risdiplam, is a small molecule that works by increasing the amount of protein produced by the survival motor neuron-2 (SMN2) gene.
Norway-based Targovax ASA has reported promising early data from a trial of its oncolytic virus ONCOS-102, in combination with chemotherapy, in patients with malignant pleural mesothelioma. Malignant mesothelioma is a tumour of the tissue surrounding the lungs, stomach and heart and is caused by exposure to asbestos fibres.
Adaptimmune Therapeutics Plc has returned to Nasdaq to raise new capital with an underwritten public offering of 21 million American depositary shares (ADSs) at a price of $4 per ADS. The issue raised a net $78.1 million after deducting the underwriting discount and other expenses. Adaptimmune made its initial public offering on Nasdaq in 2015.
Researchers from Cardiff University in the UK have described a new type of T cell receptor (TCR) that has been shown in laboratory studies to recognise and kill many human cancer types while ignoring healthy cells. T cells equipped with the new TCR were able to kill a host of malignancies including lung, skin, blood and colon cancers in mice engineered with human cancers and a human immune system. The research was published on 20 January 2020 in the journal Nature Immunology.
Germany-based BioNTech SE has taken steps to strengthen its position in personalised cancer therapies with the acquisition of Neon Therapeutics Inc of the US for $67 million. The Cambridge, Massachusetts-based company was founded in 2015 by experts in neoantigen biology. The company’s lead product, NEO-PTC-01, is a personalised neoantigen-targeted T cell therapy posed to enter Phase 1 in patients with metastatic melanoma who are refractory to checkpoint inhibitors. Neoantigens are newly formed antigens that have not been previously recognised by the immune system.