Agios Announces Exclusive Worldwide License Agreement with Alnylam for Novel siRNA for the Potential Treatment of Polycythemia Vera
– 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
PV is a rare hematologic disease with no disease-modifying treatments that affects approximately 100,000 patients in the
“PV is a rare and potentially fatal hematologic disease for which phlebotomy is the standard of care,” said
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
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.
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
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
Source: Agios Pharmaceuticals, Inc.