As per the terms of the exclusive worldwide agreement, Agios will acquire the rights to develop and market Alnylam’s preclinical candidate that targets TMPRSS6, a key driver of red blood cell production.
Agios CEO Brian Goff said: “PV is a rare and potentially fatal hematologic disease for which phlebotomy is the standard of care.
“We are pleased to license this programme from Alnylam, the leading RNAi therapeutics company, with the goal of delivering a convenient, disease-modifying treatment option that addresses the underlying pathophysiology of PV and reduces or eliminates the need for phlebotomy.
“We look forward to initiating IND-enabling studies this year with the aim of delivering a transformative treatment option for this patient community with profound unmet need.”
Alnylam will receive an upfront payment of $17.5m, potential development and regulatory milestone payments of up to $130m from Agios along with sales milestones and tiered royalties.
Alnylam will provide manufacturing support for Phase I study and thereafter Agios will take full responsibility for manufacturing.
Agios will be responsible for development, regulatory and commercial activities along with costs related to the TMPRSS6 siRNA programme.
PV, which affects nearly 100,000 patients in the US, is characterised by excessive red blood cells production, which leads to high blood volume and viscosity.
Alnylam is also engaged in developing RNAi-based medicines for treating infectious, cardio-metabolic, central nervous system and ocular diseases.