Pharma Focus Asia

Mallinckrodt And Silence Therapeutics Announce Collaboration To Develop And Commercialize RNAi Therapeutics For Complement-Mediated Diseases

Friday, July 19, 2019

Mallinckrodt plc , a global biopharmaceutical company, and Silence Therapeutics plc, a leader in the discovery, development and delivery of novel RNA2 interference (RNAi) therapeutics for the treatment of serious diseases, today announced a collaboration that will allow the companies to develop and commercialize RNAi drug targets designed to inhibit or 'silence' the complement cascade, a group of proteins that are involved in the immune system and that play a role in the development of inflammation. These proteins are known to contribute to the pathogenesis of many diseases, including autoimmune diseases.

Under the terms of the agreement, Mallinckrodt will obtain an exclusive worldwide license to Silence's C33 complement asset, SLN500, with options to license up to two additional complement-targeted assets in Silence's preclinical complement-directed RNAi development program. Silence will be responsible for preclinical activities, and for executing the development program of each asset until the end of Phase 1, after which Mallinckrodt will assume clinical development and responsibility for global commercialization.

Mallinckrodt has agreed to provide Silence with an upfront payment of $20 million. Silence is also eligible to receive up to $10 million in research milestones for SLN500 and for each optioned asset, in addition to funding for Phase 1 clinical development including GMP4 manufacturing. Silence will fund all other preclinical activities. The collaboration provides for potential added clinical and regulatory milestone payments of up to $100 million for SLN500, as well as commercial milestone payments of up to $563 million for SLN500. Should Mallinckrodt opt to license one or two additional assets, Silence could receive up to $703 million in similar clinical, regulatory, and commercial milestone payments per asset. Silence would also receive tiered, low double-digit to high-teen royalties on net sales for SLN500 and each optioned asset.

"We are very excited by the significant potential offered by Silence's RNAi technology to meet the needs of underserved patients in a number of serious and critical conditions, and we look forward to collaborating with them to advance development of complement-targeted assets as new therapeutic options," said Steven Romano, M.D., Chief Scientific Officer and Executive Vice President of Mallinckrodt.

"This license provides Mallinckrodt with an expansion of early development opportunities and the value of applying RNA technology therapeutically to target a range of rare diseases as well as conditions of immune dysregulation," adds Ulrich H. von Andrian, M.D., Edward Mallinckrodt Jr. Professor of Immunopathology, Microbiology and Immunobiology, Harvard Medical School and Member of the Mallinckrodt Pharmaceuticals Scientific Advisory Council.

"This collaboration combines Silence Therapeutics' knowledge and world-class research and development in the field of RNAi with Mallinckrodt's expertise and resources as a global biopharmaceutical company with years of experience solving complex challenges," said Dr. David Horn Solomon, President and Chief Executive Officer of Silence Therapeutics. "The increasing number of clinical conditions and pathologies that involve the complement system has generated significant interest in the development of therapeutic options to inhibit complement activation, which holds great promise in treating a range of diseases. We look forward to working closely with Mallinckrodt to extend our capabilities with RNAi therapies that have the potential to transform patients' lives."  

The companies will work together to develop and commercialize this RNAi therapeutic target, with the possibility of additional assets, and will form a Joint Steering Committee to guide the asset's development program. Silence's proprietary technology is a highly specific and modular platform designed to inhibit or 'silence' the expression of disease-causing genes. The technology is appropriate to target any number of genes; however, the collaboration focuses on the complement cascade, which is implicated in a number of autoimmune diseases.

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