News

Biopharma executives in Europe and Asia, together with their financial supporters, have launched a new company to develop therapies that will both activate and inhibit the function of regulatory T cells (Tregs). Tregs are white blood cells that play a key role in regulating the immune system to ensure that the body responds appropriately to foreign antigens and neoantigens. Announced on 18 October, Regimmune/Kiji TX is the result of a merger between Kiji Therapeutics of France and Regimmune Ltd of Taiwan. The Japan based venture capital group DCI Partner Co Ltd helped execute the merger.

Cambridge, US, based Wave Life Sciences Ltd reported positive proof of mechanism data on 16 October from a Phase 1b/2a study of an RNA editing oligonucleotide drug which restored levels of a critical protein in patients with alpha-1 antitrypsin deficiency, a genetic disease affecting the lung and liver. The estimated prevalence of the disease, with the mutation studied in the trial, is 200,000 in the US and Europe. 

For the first time, a drug for diabetic macular oedema has been tested and shown to be effective in a cohort of individuals from minority groups who are disproportionately affected by the disease, the developer Roche announced on 18 October. Vabysmo (faricimab) is a bispecific antibody that was first approved in 2022 to treat age-related macular degeneration and diabetic macular oedema. Since that time it has also been authorised for retinal vein occlusion.

A retrospective analysis of biopsies taken from cancer patients who had undergone treatment with a checkpoint inhibitor and subsequently received an mRNA vaccine for coronavirus disease has shown an association between the vaccination and immune response to the therapies. Results of the analyses were presented at the 13 to 17 September meeting of the European Society for Medical Oncology. The lead author of the paper is Adam Grippin, a resident in radiation oncology at the University of Texas MD Anderson Cancer Center, US.

A new oral medicine for schizophrenia was approved by the US Food and Drug Administration on 27 September introducing a mechanism of action for treating the disease – a leading cause of disability worldwide. The drug, Cobenfy (xanomeline and trospium chloride), is the first antipsychotic drug approved to treat schizophrenia that targets cholinergic receptors as opposed to dopamine receptors, which is currently the standard of care. The drug was developed by Bristol-Myers Squibb Co.

H. Lundbeck A/S is to acquire Longboard Pharmaceuticals Inc of the US for $2.6 billion in a transaction that will significantly expand the Danish company’s neurology pipeline. Longboard has a small molecule asset in Phase 3 that is being developed for the treatment of seizures associated with a group of rare epilepsies – a market segment not currently covered by the Danish company. The drug, bexicaserin, is currently in a study for Dravet syndrome, a rare form of epilepsy that begins in infancy.

Itovebi (inavolisib), a kinase inhibitor for use in combination with two other drugs to treat breast cancer, has been approved by the US Food and Drug Administration. This follows a priority review and breakthrough therapy designation for the combination with the marketed breast cancer drugs palbociclib (Ibrance) and fulvestrant (Faslodex). Itovebi targets the PI3K enzyme, a product of the PIK3CA gene. According to the US National Cancer Institute, mutations in the PIK3CA gene have been identified in multiple cancers including breast, lung, ovary, stomach and brain.

Immatics NV, a clinical stage biotech company, has announced the pricing of a new share offering in the US which is expected to raise $150 million for the development of T cell receptor (TCR) therapies for cancer. The underwritten public offering of 16,250,000 ordinary shares was priced at $9.25 per share. The offering is expected to close on 15 October.

This year’s Nobel Prize in Chemistry has been awarded to David Baker, Demis Hassabis, and John Jumper for their work in the field of protein science. Dr Baker, a professor at the University of Washington, US, has been recognised for his pioneering research in computational protein design. Drs Hassabis and Jumper, both from Google DeepMind, are credited with developing AlphaFold2, an artificial intelligence model that predicts the structure of proteins with great accuracy.

Purespring Therapeutics Ltd, a gene therapy company developing treatments for kidney diseases, has raised £80 million from private investors to bring its lead product into clinical development. This is the latest in a series of transactions among biotech companies in the kidney disease sector but the first for a preclinical gene therapy. Purespring’s product candidate, PS-002, is an adeno-associated viral vector with a gene payload for the treatment of IgA nephropathy (IgAN), a chronic kidney disease primarily affecting young adults.