The cell-based gene therapy Breyanzi (lisocabtagene maraleucel) has received a positive opinion from the European Medicines Agency to treat patients with relapsed or refractory large B-cell lymphoma. Breyanzi was approved for the same indication in the US in 2021. It will be available to patients in the EU once the European Commission issues a marketing authorisation.
Data from four Phase 3 trials of faricimab, an experimental bispecific antibody for two eye diseases, were published online in The Lancet on 24 January, showing that the treatment delivered vision gains for patients that were at least as good as those achieved for aflibercept (Eylea), a widely prescribed ocular treatment. At the same time, faricimab required less frequent injections, according to the studies. Two of the trials enrolled patients with wet age-related macular degeneration and two enrolled patients with diabetic macular oedema.
UK-based CellCentric Ltd has secured a new investment from an arm of the American Cancer Society to progress a small molecule drug for multiple cancer indications. The investment, from BrightEdge, the cancer society’s philanthropic venture capital fund, will support clinical development of inobrodib, a candidate treatment for prostate and other cancers. Simultaneously, Morningside Investments Ltd, an existing investor, is increasing its financial support for the company. Both financing deals are equity investments, the size of which was not disclosed.
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.
Hal Barron, the chief scientific officer at GlaxoSmithKline Plc, is to become chief executive of a new regenerative medicine company that will seek to understand the process of cellular rejuvenation with the goal of slowing or even reversing the effects of disease. The company, Altos Labs Inc, will combine basic science with translational medicine across two institutes in the US and one in the UK. It is being launched with $3 billion of start-up capital, according to a statement issued on 19 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.
Belgium-based UCB SA is to acquire a US developer of drugs for rare diseases in a move that will both expand its existing epilepsy franchise and give it access to gene therapy technology. The proposed acquisition of Zogenix Inc of Emeryville, California is valued at approximately $1.9 billion (€1.7 billion) and has been approved by the boards of directors of both companies.
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.
Evotec SE is to collaborate with Eli Lilly and Co to discover up to five potential therapeutics for diabetes and chronic kidney diseases, leveraging its experience in metabolic disease discovery to investigate new approaches. Evotec has already worked with Novo Nordisk A/S on possible treatments for kidney diseases and has access to a kidney disease patient database to identify and validate possible drug targets. In the field of diabetes, it has a proprietary beta cell replacement therapy in preclinical development.