Alnylam Pharmaceuticals and Dicerna Pharmaceuticals are joining forces to develop and commercialize ribonucleicacid interference therapies (RNAi therapeutics) for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease).

As part of the alliance, Alnylam Pharmaceuticals’ ALN-AAT02 and Dicerna Pharmaceuticals’ DCR-A1AT, both investigational RNAi therapeutics will be evaluated in alpha-1 liver disease. Both the RNAi therapy candidates are currently in phase 1/2 development.

Dicerna Pharmaceuticals will be responsible for ALN-AAT02 and also DCR-A1AT at its price. The biotech company will have the option to advance either one or both of these candidates into clinical development for the treatment of alpha-1 liver disease.

Upon completion of the phase 3 development, Alnylam Pharmaceuticals will have a no-cost opportunity to opt-in to commercialize the selected RNAi therapeutic candidate in countries outside the US, where it already maintains commercialization infrastructure.

Each of the pharma companies will make pay tiered royalties to the other if Alnylam Pharmaceuticals decides to exercises its opt-in right. If the company waives its commercialization option, then Dicerna Pharmaceuticals will retain global rights for commercialization of the selected candidate(s) and will make milestone and royalty related payments to Alnylam Pharmaceuticals.

Apart from the RNAi therapeutics collaboration, the companies have signed a cross-license agreement of their respective intellectual property for Alnylam Pharmaceuticals’ lumasiran and Dicerna Pharmaceuticals’ nedosiran investigational programs for the treatment of primary hyperoxaluria.

John Maraganore – Alnylam Pharmaceuticals CEO said: “We look forward to collaborating with Dicerna to advance treatments for patients living with alpha-1 liver disease, where Dicerna will lead development and US commercialisation while Alnylam retains an ex-US commercialisation option, where the company already has the resources and experience to hit the ground running.

“Moreover, our cross-licence agreement for primary hyperoxaluria puts the needs of patients and the patient community first, and ensures freedom to operate for both companies for their respective RNAi therapeutic programmes in this ultra-rare orphan disease.”

  Alnylam Pharmaceuticals, alpha-1 liver disease, Dicerna Pharmaceuticals, John Maraganore, RNAi therapeutic, USA