Belgium-based UCB SA has negotiated a partnership with Novartis to co-develop a small molecule treatment for Parkinson’s disease which it in-licenced in 2014 from Neuropore Therapeutics Inc. The candidate treatment, UCB0599, targets alpha-synuclein, a key protein involved in the disease’s pathology. The compound is currently in a Phase 2 study in patients with early-stage disease.
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.
Vifor Pharma Group has expanded its presence in nephrology with the acquisition of two companies with therapies addressing specific aspects of vascular calcification. The companies are Sanifit Therapeutics SA of Spain and Inositec AG of Switzerland.
Merck & Co Inc and its partner Ridgeback Biotherapeutics LP published new efficacy data for their Covid-19 treatment on 26 November showing that the anti-viral drug reduced the risk of hospitalisation or death by 30%, compared with a placebo, instead of the earlier reported 50%. The updated efficacy figure is based on additional data presented to the US Food and Drug Administration. The FDA’s Antimicrobial Drugs Advisory Committee was scheduled to meet on 30 November to evaluate the latest information.
A cell therapy company launched by Syncona Ltd in 2019 has completed an oversubscribed Series B financing round to start the clinical development of a compound to prevent organ rejection in liver transplant patients. Quell Therapeutics Ltd raised $156 million from a consortia co-led by Jeito Capital, Ridgeback Capital Investments, SV Health Investors and Fidelity Management & Research Company LLC. Syncona also participated in the round.
Quell is developing engineered T regulatory (Treg) cells as therapies.
Finland-based Valo Therapeutics Oy has raised €11 million in an over-subscribed financing round to support a first clinical trial of it lead cancer drug – an antigen-coated oncolytic virus. The financing, announced on 15 November, was led by the corporate advisory firm Ironside Capital. Existing investor Freeman Road Pty Ltd participated in the round as did new investor MMJ Group Holdings of Australia. The round is being described as a prelude to an initial public share offering.
GlaxoSmithKline Plc has negotiated rights to a candidate treatment for non-alcoholic steatohepatitis (NASH) from Arrowhead Pharmaceuticals Inc which uses RNA interference (RNAi) technology to reduce levels of the liver enzyme ALT and address the disorder. The candidate therapy, ARO-HSD, targets the protein-coding gene HSD17B13 which is involved in the metabolism of hormones, fatty acids and bile acids. NASH is a disorder in which excessive fat builds up in the liver, causing inflammatory damage and fibrosis.
Novo Nordisk A/S is to acquire Dicerna Pharmaceuticals Inc, a company developing candidate RNA interference (RNAi) therapeutics for multiple diseases including cardiometabolic, viral, and chronic liver indications. Under the terms of the agreement, Novo will acquire all of the outstanding shares of Dicerna for $38.25 per share for a total equity value of $3.3 billion.
Sanofi SA has announced an equity investment of $180 million in Owkin, a company with an AI platform which enables scientists to conduct research on diseases without having to pool data centrally. The model, called ‘federated learning,’ enables data scientists to securely connect to decentralised, multi-party data sets and create AI models for targeted drug discovery. Owkin is based in New York City, US but has scientific roots in France.
A preventive treatment for migraine, Vyepti (eptinezumab), has received a positive opinion from the European Medicines Agency – the first step towards getting an authorisation for the EU market. If approved, it will be the first and only intravenous treatment for migraine prevention in the region, the developer H. Lundbeck A/S, announced on 12 November.