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Agios Announces Exclusive Worldwide License Agreement with Alnylam for Novel siRNA for the Potential Treatment of Polycythemia Vera

Key highlights:

Agios Pharmaceuticals, Inc. has entered into an exclusive worldwide license agreement with Alnylam Pharmaceuticals, Inc. under which Agios will acquire the rights to develop and commercialise Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV). This agreement combines Agios’ deep scientific expertise and capabilities in rare hematologic diseases with Alnylam’s industry-leading siRNA platform and strong track record of success.

PV is a rare hematologic disease with no disease-modifying treatments that affects approximately 100,000 patients in the U.S. PV is characterised by excessive production of red blood cells, which leads to increased blood volume and viscosity, and can result in thrombosis, cardiovascular events, and death.

“PV is a rare and potentially fatal hematologic disease for which phlebotomy is the standard of care,” said Brian Goff, chief executive officer at Agios. “We are pleased to license this program 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. This program is highly aligned with our core scientific expertise, clinical development and commercial capabilities in rare hematology as well as our business development strategy to expand beyond our industry-leading pipeline of PK activators. 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.”

The siRNA development candidate targets knockdown of TMPRSS6, a key driver of red blood cell production. This results in increased hepcidin, reducing iron available to the hematopoietic compartment, thereby reducing red blood cell production. This has the potential to maintain hematocrit within the normal range and reduce the risk of complications in individuals living with PV. TMPRSS6 siRNA has demonstrated low off-target activity, a favorable safety profile in rats, and a 90% knockdown of TMPRSS6 mRNA over 3 months in non-human primates, supporting the potential for an infrequent dosing regimen.

Under the terms of the agreement, Agios will make an upfront payment of $17.5 million to Alnylam for an exclusive global license to the TMPRSS6 siRNA program. In addition, Alnylam is eligible to receive up to $130 millionin potential development and regulatory milestone payments, in addition to sales milestones and tiered royalties. Agios will assume sole responsibility for all development, regulatory, and commercial activities and costs related to the program. Alnylam will provide manufacturing support for Phase 1, after which Agios will assume full responsibility for manufacturing.

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