Press Release Details

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

Aug 03, 2023

– Preclinical siRNA Targeting TMPRSS6 is a Potential Disease-Modifying Treatment for Polycythemia Vera –

– Agreement Combines Agios’ Scientific Expertise and Capabilities in Rare Hematologic Diseases with Alnylam’s Industry-Leading siRNA Platform –

– Alnylam to Receive a $17.5 million Upfront Payment and is Eligible to Receive Potential Future Development and Commercial Milestone Payments and Royalties –

CAMBRIDGE, Mass., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) has entered into an exclusive worldwide license agreement with Alnylam Pharmaceuticals, Inc. under which Agios will acquire the rights to develop and commercialize 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 characterized 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 million in 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.

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Agios Pharmaceuticals

Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios has a PAH stabilizer in preclinical development as a potential treatment for phenylketonuria (PKU) and deep scientific expertise in classical hematology. For more information, please visit the company’s website at

Agios Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of, and plans relating to, Agios’ license agreement with Alnylam; the potential benefits of the licensed siRNA development candidate; the potential of TMPRSS6 as a therapeutic target; and the potential benefits of Agios’ strategic plans and focus. The words “aim,” “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that the licensed siRNA development candidate or any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of the COVID-19 pandemic or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to maintain key collaborations; the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.


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Source: Agios Pharmaceuticals, Inc.