Agios Announces Exclusive Worldwide License Agreement for Novel siRNA for the Potential Treatment of Polycythemia Vera
03 Agosto 2023 - 7:30AM
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 www.agios.com.
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.
Contacts:
Agios Pharmaceuticals
Investor ContactChris Taylor, VP
Investor Relations and Corporate CommunicationsAgios
PharmaceuticalsIR@agios.com
Media Contact Dan Budwick1AB Media
dan@1abmedia.com
Agios Pharmaceuticals (NASDAQ:AGIO)
Gráfico Histórico do Ativo
De Ago 2024 até Set 2024
Agios Pharmaceuticals (NASDAQ:AGIO)
Gráfico Histórico do Ativo
De Set 2023 até Set 2024