A new company focused on obesity and cardiometabolic disorders was launched on 9 January with $411 million in a Series A venture financing. Named Verdiva Bio Ltd the company is registered in the UK with leadership in Europe and North America and a start-up portfolio from China. The company’s financing syndicate is co-led by Forbion of the Netherlands and the US private equity group General Atlantic with five other participants including Lilly Asia Ventures.
Demand for antibody-drug conjugates (ADCs) took a leap on 9 January after Netherlands-based Synaffix BV disclosed two new deals for its proprietary ADC technology. The first agreement is with Mitsubishi Tanabe Pharma Corp of Japan and the second with Boehringer Ingelheim GmbH of Germany. The value of the Mitsubishi deal was not disclosed, however that with Boehringer involves a potential milestone payment of up to $1.3 billion in addition to an undisclosed upfront payment.
The Belgian biotech Galapagos NV is to separate into two publically listed biotech entities with a focus on cell therapy and medical innovation. Announced on 8 January, the transaction will create an incubator company, provisionally called SpinCo, and a reconfigured Galapagos with a focus on cell therapies. SpinCo will be capitalised with approximately €2.45 billion of cash from Galapagos to investigate new medicines in oncology, immunology and/or virology. It will seek a listing on the Euronext stock exchange.
Zolgensma (onasemnogene abeparvovec), the gene therapy developed by Novartis for spinal muscular atrophy (SMA), has shown efficacy in a Phase 3 study of patients aged two to less than 18 years who are able to sit but have never walked independently. SMA is a genetic neuromuscular disease caused by the lack of a functional SMN1 gene that affects muscle functions and basic movement. Zolgensma was first approved in the US in 2019, and one year later in the EU, as a paediatric drug for children less than two years old and administered as a single, one-time infusion.
A kinase inhibitor for the treatment of lung diseases has been recommended for use in children and adolescents from the age of 6 years by the European Medicines Agency. The treatment, Ofev (nintedanib), was authorised for adults in 2014 and subject to approval by the European Commission, will be available for children in 2025. The indication is progressive fibrosing interstitial lung disease which includes a group of rare respiratory disorders characterised by the build-up of scarring in the lungs which can lead to breathing difficulties.
A small molecule drug, Welireg (belzutifan), has been given conditional authorisation by the European Medicines Agency for tumours associated with von Hippel-Lindau disease and previously treated advanced kidney cancer. The opinion, announced on 13 December, expands the availability of the drug to Europe from the US where it was approved in 2021 by the Food and Drug Administration. The developer is Merck & Co Inc which submitted data to the EMA from two clinical studies showing efficacy.
A new cell therapy has been approved by the US Food and Drug Administration to treat paediatric patients from two months of age who experience complications from a stem cell transplant for blood cancer or an immune system disorder. The treatment, Ryoncil (remestemcell-L rknd), is an allogeneic therapy made up from mesenchymal stromal cells and isolated from the bone marrow of healthy adult donors. It is indicated for the treatment of acute graft-versus-host disease in patients undergoing transplants who do not respond to corticosteroids.
A treatment for classic congenital adrenal hyperplasia (CAH) has been approved by the US Food and Drug Administration – the first new drug in decades for a genetic disorder involving the adrenal glands. The drug, Crenessity (crinecerfont), is a small molecule to be used as an adjunct to glucocorticoids. In two randomised, placebo-controlled trials in adults and children Crenessity was able to reduce an overproduction of androgen in adults and children relative to the production of cortisol, an essential hormone. This was achieved without the need for high levels of glucocorticoids.
A second candidate drug for Parkinson’s disease has failed to meet the primary endpoint in a clinical study of patients with early-stage disease. The Roche Group announced the results of its Phase 2b PADOVA study on 19 December, only days after UCB SA said its medication for early Parkinson’s disease had failed to show efficacy. Roche said it will continue to evaluate data from the main trial and an ongoing extension study in deciding what to do. The candidate drug, prasinezumab, is a monoclonal antibody designed to bind to alpha-synuclein protein and reduce neuronal toxicity.
Merck Inc has entered into a licensing deal with the Chinese pharmaceutical group Hansoh Pharma to gain access to a preclinical obesity asset, bringing it into a field currently dominated by Eli Lilly and Co and Novo Nordisk A/S. The asset, HS-10535, is an oral small molecule glucagon-like peptide-1 receptor agonist.