A Phase 2 trial of a small molecule drug for patients with mild dementia due to Alzheimer’s disease has been stopped due to lack of efficacy, the developer Sage Therapeutics Inc announced on 8 October. The drug, dalzanemdor (SAGE-718), did not show a statistically significant difference in patients with the disorder, compared with a placebo, on the basis of a widely-used intelligence test.
A consortium of universities and hospitals, with financial support from the UK government and industry, is to undertake a four-year project to collect data on patients who have received immunotherapy for cancer to establish the benefits and risks of these treatments. The treatments, which include checkpoint inhibitors, have extended the lives of many patients by enabling the body’s immune system to recognise and destroy cancer cells.
AstraZeneca Plc is to pay $100 million upfront and up to $1.92 billion in milestone payments for rights to a pre-clinical lipid-lowering therapy from China. The developer, CSPC Pharmaceutical Group Ltd, is listed on the Hong Kong Stock Exchange and has R&D facilities in both China and the US. The asset being acquired is a small molecule lipoprotein inhibitor which is in pre-clinical development for patients suffering from dyslipidaemia, a disorder involving abnormal levels of lipids in the bloodstream. Dyslipidaemia is a risk factor for cardiovascular diseases.
An Italian biotech company, Aptadir Therapeutic Srl, which is developing a new class of RNA therapies, launched on 24 September with pre-seed funding of $1.6 million and plans to tackle cancer by blocking aberrant DNA methylation. DNA methylation is an essential biological process that enables the expression of genes in humans. But this process can be thrown off balance by external factors such as age or diets, leading to cancer and genetic disorders.
Novo Nordisk A/S is to pay an undisclosed amount of money to Evotec SE to gain access to the German company’s cell therapy technology and manufacturing capacity for its experimental therapies in a number of indications including diabetes. The collaboration, announced on 26 September, is an expansion of the Danish company’s platform technologies which are currently dominated by insulins and peptide therapies for obesity. Novo currently has cell therapy programmes under investigation for chronic heart failure, Parkinson’s disease and Type 1 diabetes.
Enara Bio Ltd, a UK biotech, has raised $32.5 million in Series B financing to build a pipeline of therapies for cancer that target peptide antigens in the dark genome. The financing round was co-led by the new investors Pfizer Ventures and M Ventures whose executives, respectively, Rana Al-Hallaq and Bauke Anninga, have joined the company’s board of directors.
A preclinical project designed to treat Huntington’s disease has received £35 million in Series A financing on early evidence that it can slow or even halt progression of the disease. Huntington’s is an inherited disorder that attacks areas of the brain that help control movement. The funding was awarded to LoQus23 Therapeutics Ltd, a biotech company based in Cambridge UK. The venture capital group Forbion led the round, alongside existing investors SV Health Investors’ Dementia Discovery Fund and the Novartis Venture Fund.
Mabylon AG, Swiss biotech company developing new antibody technologies, has received grant funding of more than CHF 1.3 million ($1.54 million) to discover a new approach for treating amyotrophic lateral sclerosis (ALS) and inflammatory bowel disease (IBD). The funding for ALS is being provided by the non-profit groups Target ALS and the ALS Association, while that for IBD is coming from Innosuisse, the Swiss Innovation Agency.
Mabylon develops intrabodies which are antibody fragments expressed inside a cell that bind to specific proteins within that cell.
A privately held Italian company has raised $52 million to progress development of an in vivo gene therapy to treat methylmalonic acidemia (MMA), a genetic disorder that is manifest in early infancy. Genespire Srl is a 2020 spin-out of the San Raffaele Telethon Institute for Gene Therapy, a cell and gene therapy research institute. The funds will support development of the company’s lead product, GENE202, up to a clinical Phase 1/2 trial.
An antibody-drug conjugate drug for breast cancer, jointly developed by AstraZeneca Plc and Daiichi Sankyo Co Ltd, failed to show statistical significance in overall survival at Phase 3 versus chemotherapy despite having achieved progression free survival for the same patient group. Announcing the results on 23 September, AstraZeneca said that the survival results were likely to have been affected by changes in the standard of care for breast cancer during the drug’s development period.