– Partnership Combines Alnylam’s Leadership in
RNAi Therapeutics with Roche’s Proven Track Record of Successfully
Developing and Launching Innovative Medicines Worldwide –
– Zilebesiran Represents a Potentially
Transformative Approach to Reducing CV Morbidity and Mortality in
Hypertension Patients at High CV Risk by Robustly and Durably
Lowering Blood Pressure –
– Alnylam will Receive an Upfront Cash Payment
of $310 Million and is Eligible to Receive Development, Regulatory,
and Sales Milestones, Including Substantial Near-Term Milestones,
for a Potential Deal Value of up to $2.8 Billion, as well as an
Equal Share of Profits and Losses in the United States and
Royalties on Net Sales Outside the U.S. –
– Alnylam will Lead Joint Clinical Development
Plan for First Indication, Including Cardiovascular Outcome Trial,
with Development Costs Shared Between the Companies –
– Alnylam and Roche will Co-Commercialize
Zilebesiran in the U.S., While Roche Obtains Exclusive Right to
Commercialize Zilebesiran Outside the U.S. –
Alnylam to Host Conference Call Today, Monday,
July 24, at 08:00 a.m. ET to Discuss Collaboration –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced it has entered into a
strategic agreement with Roche to develop and commercialize
zilebesiran, Alnylam’s investigational RNAi therapeutic for the
treatment of hypertension, which is currently in Phase 2 of
development. The partnership allows for a bold development plan
with the goal of disrupting the hypertension treatment paradigm
globally while advancing Alnylam’s P5x25 strategy.
Roche provides Alnylam the benefits of an outstanding partner
with a global footprint and a proven track record of developing and
commercializing novel therapies in complex markets. Roche has a
proven history of innovating and commercializing medicines building
upon their extensive global footprint which may potentially enable
zilebesiran to reach more patients with hypertension, a disease
that affects more than 1.2 billion patients globally.
“We are thrilled to announce this collaboration, as it combines
Alnylam’s proven track record in RNAi therapeutics with Roche’s
global commercial reach, commitment to innovation and desire to
transform the landscape for patients with severe cardiovascular
diseases,” said Yvonne Greenstreet MBChB, Chief Executive Officer
of Alnylam. “With this collaboration, we now can develop
zilebesiran in a more robust way, allowing us to have
cardiovascular outcomes data in hand at launch to ensure results
relevant not only for health authorities but also for access and
clinical practice in order to ultimately reach as many patients as
possible.”
“We are excited to work together with Alnylam and leverage our
strong R&D capabilities, our leadership in cardiovascular
diagnostics and our global commercial footprint to further develop
and provide this promising therapy with best-in-disease potential
to patients,” said Teresa Graham, CEO Roche Pharma. “Throughout our
history, we have redefined the standard of care across various
disease areas. Together with a strong partner like Alnylam, we are
looking forward to making a significant impact for patients living
with hypertension at high cardiovascular risk and potentially other
cardiovascular indications.”
In a Phase 1 study, zilebesiran, compared to placebo, was
associated with dose-dependent reductions in serum angiotensinogen
(AGT), achieving tonic blood pressure control with consistent and
durable blood pressure reduction throughout a 24-hour period,
sustained up to six months after single doses of ≥200 mg.
Zilebesiran also demonstrated an acceptable safety profile
supporting continued clinical development. The safety and efficacy
of zilebesiran are being investigated in Alnylam’s KARDIA Phase 2
clinical program either as a monotherapy (KARDIA-1) or in
combination with one of three standard-of-care antihypertensive
medications (KARDIA-2). Based on the positive Phase 1 data,
zilebesiran could potentially be a best-in-disease treatment and
provide transformational benefit, especially for patients with
hypertension at high cardiovascular risk. Zilebesiran also has the
potential to improve adherence to treatment due to its possible
biannual subcutaneous dosing regimen. In addition, zilebesiran may
be effective in additional potential cardiovascular indications
with high unmet need.
Under the terms of the agreement, Alnylam will receive an
upfront cash payment of $310 million and is eligible to receive
additional substantial near-term payments, including development
milestone payments over the next few years, as well as regulatory
and sales milestones, for a potential deal value of up to $2.8
billion. In addition, Alnylam is entitled to an equal profit share
in the U.S., where Alnylam and Roche will co-commercialize
zilebesiran. Roche obtained the exclusive right to commercialize
zilebesiran outside the U.S. in exchange for low double digit
royalties on net sales of zilebesiran outside of the U.S. Alnylam
believes that this partnership will allow the companies to pursue a
joint development plan and commercialization approach that has the
potential to unlock the full value of zilebesiran. Additionally,
Alnylam will lead a joint clinical development plan for the first
indication with Roche’s participation, which includes a
cardiovascular outcomes trial prior to submission of zilebesiran
for regulatory approval, with all development costs shared 40% by
Alnylam and 60% by Roche. Roche may lead development for additional
indications in the future.
Goldman Sachs & Co. LLC served as exclusive financial
advisor to Alnylam.
Alnylam Conference Call Information
Alnylam management will discuss the new collaboration via
conference call on Monday, July 24, 2023 at 8:00 am ET. To access
the call, please register online at
https://register.vevent.com/register/BIceae6347f7d14f5cb144ead1cf7cc974.
Participants are requested to register a minimum of 15 minutes
before the start of the call. A replay of the call will be
available two hours after the call and archived on the same web
page for six months.
A live audio webcast of the call will be available on the
Investors section of the Company’s website at
www.alnylam.com/events. An archived webcast will be available on
the Company’s website approximately two hours after the event.
About Zilebesiran
Zilebesiran is an investigational, subcutaneously administered
RNAi therapeutic targeting angiotensinogen (AGT) in Phase 2
development for the treatment of hypertension in high unmet need
populations. AGT is the most upstream precursor in the
Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a
demonstrated role in blood pressure (BP) regulation and its
inhibition has well-established anti-hypertensive effects.
Zilebesiran inhibits the synthesis of AGT in the liver, potentially
leading to durable reductions in AGT protein and ultimately, in the
vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes
Alnylam's Enhanced Stabilization Chemistry Plus (ESC+)
GalNAc-conjugate technology, which enables subcutaneous dosing with
increased selectivity and a wide therapeutic index. The safety and
efficacy of zilebesiran have not been established or evaluated by
the FDA, EMA or any other health authority.
About Hypertension
Hypertension is a complex multifactorial disease clinically
defined by most major guidelines as a systolic blood pressure (SBP)
of above 140 mm Hg and/or a diastolic blood pressure (DBP) greater
than 90 mm Hg, though AHA/ACC guidelines have a lower threshold of
a SBP above 130 mm Hg and/or a DBP greater than 80 mm Hg. More than
one billion people worldwide live with hypertension.1 In the U.S.
alone, approximately 47 percent of adults live with hypertension,
with more than half of patients on medication remaining above the
blood pressure (BP) target level. Despite the availability of
anti-hypertensive medications, there remains a significant unmet
medical need, especially given the poor rates of adherence to
existing daily oral medications and daily peak and trough effects,
resulting in inconsistent BP control and an increased risk for
stroke, heart attack and premature death.2 In particular, there are
a number of high unmet need settings where novel approaches to
hypertension warrant additional development focus, including
patients with poor medication adherence, difficult-to-treat and
resistant hypertension, and in patients with high cardiovascular
risk.
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 Alnylam Pharmaceuticals
Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation
of RNA interference (RNAi) into a whole new class of innovative
medicines with the potential to transform the lives of people
afflicted with rare and prevalent diseases with unmet need. Based
on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach yielding transformative
medicines. Since its founding in 2002, Alnylam has led the RNAi
Revolution and continues to deliver on a bold vision to turn
scientific possibility into reality. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), AMVUTTRA®
(vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and
Leqvio® (inclisiran), which is being developed and commercialized
by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of
investigational medicines, including multiple product candidates
that are in late-stage development. Alnylam is executing on its
“Alnylam P5x25” strategy to deliver transformative medicines in
both rare and common diseases benefiting patients around the world
through sustainable innovation and exceptional financial
performance, resulting in a leading biotech profile. Alnylam is
headquartered in Cambridge, MA. For more information about our
people, science and pipeline, please visit www.alnylam.com and
engage with us on Twitter at @Alnylam, on LinkedIn, or on
Instagram.
Alnylam Forward Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. All statements
other than historical statements of fact regarding Alnylam’s
expectations, beliefs, goals, plans or prospects including, without
limitation, expectations regarding Alnylam’s aspiration to become a
leading biotech company and the planned achievement of its “Alnylam
P5x25” strategy, Roche’s participation in the development and
commercialization of zilebesiran, the potential for zilebesiran to
disrupt the treatment paradigm in hypertension, Alnylam’s
expectations regarding the receipt of upfront cash, as well as
potential development, regulatory and sales milestones and
royalties from Roche, Alnylam’s ability to obtain approval for new
commercial products or additional indications for its existing
products, and Alnylam’s projected commercial and financial
performance, should be considered forward-looking statements.
Actual results and future plans may differ materially from those
indicated by these forward-looking statements as a result of
various important risks, uncertainties and other factors,
including, without limitation: the direct or indirect impact of the
COVID-19 global pandemic or any future pandemic on Alnylam’s
business, results of operations and financial condition and the
effectiveness or timeliness of Alnylam’s efforts to mitigate the
impact of the pandemic; Alnylam’s ability to successfully execute
on its “Alnylam P5x25” strategy; Alnylam's ability to discover and
develop novel drug candidates and delivery approaches and
successfully demonstrate the efficacy and safety of its product
candidates; the pre-clinical and clinical results for Alnylam’s
product candidates, including patisiran and vutrisiran; actions or
advice of regulatory agencies and Alnylam’s ability to obtain and
maintain regulatory approval for its product candidates, including
patisiran and vutrisiran, as well as favorable pricing and
reimbursement; successfully launching, marketing and selling
Alnylam’s approved products globally; delays, interruptions or
failures in the manufacture and supply of Alnylam’s product
candidates or its marketed products; delays or interruptions in the
supply of resources needed to advance Alnylam’s research and
development programs, including as may arise from recent
disruptions in the supply of non-human primates; obtaining,
maintaining and protecting intellectual property; Alnylam’s ability
to successfully expand the indication for ONPATTRO or AMVUTTRA in
the future; Alnylam's ability to manage its growth and operating
expenses through disciplined investment in operations and its
ability to achieve a self-sustainable financial profile in the
future without the need for future equity financing; Alnylam’s
ability to maintain strategic business collaborations; Alnylam's
dependence on third parties for the development and
commercialization of certain products, including Roche, Novartis,
Sanofi, Regeneron and Vir; the outcome of litigation; the potential
impact of a current government investigation and the risk of future
government investigations; and unexpected expenditures; as well as
those risks more fully discussed in the “Risk Factors” filed with
Alnylam's 2022 Annual Report on Form 10-K filed with the Securities
and Exchange Commission (SEC), as may be updated from time to time
in Alnylam’s subsequent Quarterly Reports on Form 10-Q and in its
other SEC filings. In addition, any forward-looking statements
represent Alnylam's views only as of today and should not be relied
upon as representing its views as of any subsequent date. Alnylam
explicitly disclaims any obligation, except to the extent required
by law, to update any forward-looking statements.
1pertension. World Health Organization.
https://www.who.int/news-room/fact-sheets/detail/hypertension.
Published September 2019. Accessed November 2021.
2rey, R. M., Muntner, P., Bosworth, H. B.,
& Whelton, P. K. (2018). Prevention and Control of
Hypertension: JACC Health Promotion Series. Journal of the American
College of Cardiology, 72(11), 1278–1293.
https://doi.org/10.1016/j.jacc.2018.07.008
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Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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