The checkpoint inhibitor Imfinzi (durvalumab), in combination with chemotherapy, reduced the risk of death by 20% in a Phase 3 trial of patients with advanced biliary tract cancer, the developer AstraZeneca Plc announced on 18 January. The multicentre trial compared Imfinzi and chemotherapy, with chemotherapy alone as a first-line treatment. Called TOPAZ-1, the trial enrolled 685 patients with unresectable cancer, including gallbladder cancer.
Asian patients with advanced hepatocellular carcinoma were able to live longer after being treated with Keytruda (pembrolizumab), one of the most widely prescribed checkpoint inhibitors for cancer. A Phase 3 trial of 453 patients with the disease achieved an overall survival rate of 21%, potentially acting as a confirmatory study for an accelerated approval given to Keytruda for this indication in the US, the developer Merck & Co Inc announced on 18 January.
ReNeuron Group Plc has taken a decision to out-license a clinical-stage cell therapy programme for retinitis pigmentosa (RP), an eye disease in which the retina is damaged, and focus instead on developing its exosome technology. The decision was announced to the London Stock Exchange on 18 January.
A Phase 2 trial of a small molecule drug for the treatment of sudden sensorineural hearing loss, or sudden deafness, failed to meet its primary efficacy endpoint, the developer Sensorion SA, announced on 17 January. The drug, SENS-401, was safe and well-tolerated but did not show a significant improvement in pure tone audiometry, a test used to measure hearing sensitivity. In the trial, it was compared with a placebo and administered to patients over a period of four weeks. The trial, AUDIBLE-S, enrolled 115 patients.
A monoclonal antibody developed by Novartis to treat chronic spontaneous urticaria, also known as chronic hives, did not show superiority to the marketed drug omalizumab in two Phase 3 trials. Announcing the trial results on 20 December, Novartis said that ligelizumab demonstrated superiority against placebo but not against omalizumab, known commercially as Xolair. Xolair, developed by Roche, has been approved for six indications in the US, including chronic idiopathic urticaria.
Bristol Myers Squibb Co has launched its second preclinical collaboration with Immatics NV in order to explore the potential of an antibody-like bispecific molecule as a treatment for solid tumours. The newest agreement, announced on 14 December, gives BMS exclusive rights to an engineered bispecific molecule consisting of a receptor that binds to a cancer target and a region that recruits T cells to the tumour site to become activated. The upfront payment is $150 million.
The US Food and Drug Administration has placed clinical holds on six trials of the HIV medicine islatravir after some trial participants showed a decrease in total lymphocyte and CD4+ T cell counts – a sign of weakened immune systems. Developed by Merck & Co Inc, islatravir is being studied for both the treatment and prevention of HIV-1 infection. “With the FDA’s clinical hold, no new studies may be initiated,” the company announced on 13 December.
The New England Journal of Medicine has published data from a Phase 3 trial of Dupixent (dupilumab), a widely prescribed anti-inflammatory drug which was recently approved by the US Food and Drug Administration for children with uncontrolled moderate-to-severe asthma. Data from the trial formed the basis of a US approval and have been included in a regulatory filing with the European Medicines Agency.
Merck & Co Inc paused enrolment on 6 December for two Phase 3 trials of its lead investigational treatment for HIV – a little more than two weeks after stopping dosing in a Phase 2 study of an HIV combination therapy. The measures were taken on the recommendation of the trials’ respective data monitoring committees. They affect MK-8507, a non-nucleoside reverse transcriptase inhibitor, and islatravir, a nucleoside reverse transcriptase translocation inhibitor. In both cases, the actions were taken in relation to total lymphocyte and CD4+ T cell counts in patients.
A stem cell-derived therapy has restored the function of pancreatic islet cells in a patient with Type 1 diabetes in what is being described as the first demonstration of a potentially curative treatment for the disease. The therapy is being developed by Vertex Pharmaceuticals Inc on the basis of research conducted by Douglas Melton, a professor at Harvard University and co-director of the Harvard Stem Cell Institute in Cambridge, US.