News

A second Phase 3 trial of ensifentrine, a candidate product for chronic obstructive pulmonary disease (COPD), has met its primary endpoint, paving the way for a regulatory submission in the US in the first half of 2023, the developer Verona Pharma Plc announced on 20 December. Ensifentrine is a dual inhibitor of the enzymes phosphodiesterase 3 and 4, a mode of action that enables it to combine both bronchodilator and anti-inflammatory effects in a single compound.

The US Food and Drug Administration has approved a gene therapy for bladder cancer, the second authorisation of a novel gene treatment within a month. The new drug, Adstiladrin (nadofaragene firadenovec), is indicated for the treatment of patients with bladder cancer which hasn’t yet invaded the muscle and is unresponsive to the Bacillus Calmette-Guérin (BCG) immunotherapy. The decision comes not long after the agency approved a gene therapy for haemophilia B.

An experimental messenger RNA (mRNA) cancer vaccine has generated positive data in combination with Keytruda (pembrolizumab) in patients with advanced melanoma, the developers Moderna Inc and Merck & Co announced on 13 December.

Plans to develop a candidate therapy for solid tumours using a T cell receptor (TCR) approach were disclosed on 2 December by Immunocore Holdings Plc, which has concluded an oncology collaboration with Gadeta BV of the Netherlands. The two companies are at different stages of development, but they share an interest in using TCRs as agents for fighting disease.

TCRs are a group of proteins found on T cells that bind to certain antigens on cancer cells causing T cells to attack the malignancy.

An allogeneic dendritic cell vaccine for patients who have residual cancer after previous successful treatments with chemotherapy has shown positive survival data, according to the developer Mendus AB. The treatment, DCP-001, is being evaluated in a Phase 2 trial as a maintenance therapy in acute myeloid leukaemia (AML) for patients with measurable residual disease. Fresh data from the trial, ADVANCE II, was reported at the American Society of Hematology Annual Meeting on 12 December.

A competition between US and French multinationals for ownership of Horizon Therapeutics Plc ended on 12 December with an all-cash offer of $27.8 billion from Amgen Inc for the Ireland-based company. The deal value is the largest healthcare merger of 2022, according to The Wall Street Journal. Sanofi SA, which also held talks with Horizon, dropped out of the discussions saying that the target company’s price expectations were too high.

A Phase 3 trial of a small molecule drug designed to treat patients with metastatic non-small cell lung cancer failed to meet its primary endpoint of overall survival, the sponsor Ipsen SA announced on 8 December. Cabozantinib was being investigated in 366 patients with both squamous and non-squamous non-small cell lung cancer whose disease had progressed after treatment with a checkpoint inhibitor and chemotherapy.

Belgium-based UCB SA has reported positive Phase 3 data for its monoclonal antibody bimekizumab in hidradenitis suppurativa, a chronic inflammatory skin disease that is estimated to affect about one percent of the population in countries where the disease has been identified. Data from the two studies is expected to form the basis of global regulatory applications starting in the third quarter of 2023.

C4X Discovery Holdings Plc of the UK has secured a licensing deal with AstraZeneca Plc to apply its small molecule drug discovery technology to identify new treatments for inflammatory and respiratory diseases. The initial plan is to develop a product to treat chronic obstructive pulmonary disease (COPD).

Cancer Research Horizons, a division of the UK charity Cancer Research UK, is to work with the Karolinska Institutet over the next five years to identify new opportunities for cancer treatments and move these projects forward through industrial partnerships.