RNAi Biotech Deal is Sealed
The UK-based biotech Silence Therapeutics has signed a deal with UK pharma company Mallinckrodt to develop RNA interference (RNAi) therapies for autoimmune diseases.
Mallinckrodt will pay Silence Therapeutics €17.8M ($20M) upfront, and up to €600M ($673M) in developmental milestone payments. In exchange, it will get a license for an RNAi treatment for diseases involving the innate immune system, such as autoimmune diseases.
Silence Therapeutics develops treatments that deliver RNAi molecules into cells, which then silence the expression of proteins causing a disease. The preclinical candidate that Mallinckrodt has licensed silences a protein that stimulates the immune system. By silencing this protein, the drug could treat diseases involving inflammation and the immune system, such as autoimmune diseases.
In addition to Silence’s preclinical drug, Mallinckrodt will have the option to license other programs from Silence Therapeutics, paying an additional amount of up to €626M ($703M) in developmental milestone payments per treatment.
Silence Therapeutics’ other programs are progressing through the preclinical pipeline. The two most advanced therapies, for the genetic conditions beta-thalassemia and myelodysplastic syndrome, are now ready to enter phase I.
RNAi therapies have been in the works for more than 20 years, and the journey has been slowed by safety concerns, and even patient deaths in several trials. However, the field appears to be moving forward, with the first RNAi therapy approved in the US and Europe for the treatment of a rare inherited disease last year.