− Company Intends to Advance Lumasiran to a
Phase 3 Study in Late 2018 −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, announced today that the European
Medicines Agency (EMA) has granted access to its Priority Medicines
(PRIME) scheme for lumasiran (ALN-GO1), an investigational RNAi
therapeutic targeting glycolate oxidase for the treatment of
Primary Hyperoxaluria Type 1 (PH1). The purpose of the PRIME
initiative is to bring treatments to patients faster by enhancing
the EMA's support for the development of medicines for diseases
where there is an unmet medical need and where early clinical data
show potential to benefit patients. Lumasiran was recently granted
Breakthrough Therapy Designation by the United States Food and Drug
Administration (FDA).
Promising results from Parts A and B of the ongoing Phase 1/2
study of lumasiran, recently presented at the American Society of
Nephrology (ASN) Kidney Week 2017 Annual Meeting in November 2017,
formed the basis of the application for PRIME.
“We are very pleased to have lumasiran accepted into the PRIME
program. Based on the preliminary clinical data presented to date,
we believe lumasiran could potentially be a transformative
treatment option for patients with PH1, an ultra-rare, debilitating
condition that presents in early childhood and is associated with
an enormous disease burden,” said Pritesh Gandhi, PharmD., Vice
President and General Manager, Lumasiran program at Alnylam. “We
look forward to collaborating with the EMA to optimize the
development and accelerate the assessment of lumasiran, with the
goal of advancing this investigational medicine into a Phase 3
trial in late 2018 and to the market as rapidly as possible.”
About Lumasiran
Lumasiran (formerly known as ALN-GO1) is an investigational RNAi
therapeutic targeting glycolate oxidase (GO) in development for the
treatment of Primary Hyperoxaluria Type 1 (PH1). Lumasiran is
designed to reduce the hepatic levels of the GO enzyme, thereby
depleting the substrate necessary for oxalate production, which
directly contributes to the pathophysiology of PH1. Lumasiran
utilizes Alnylam's Enhanced Stabilization Chemistry
(ESC)-GalNAc-conjugate technology, which enables subcutaneous
dosing with increased potency and durability and a wide therapeutic
index. Lumasiran has received both U.S. and EU Orphan Drug
Designations, and a Breakthrough Therapy Designation from the U.S.
Food and Drug Administration (FDA). The safety and efficacy of
lumasiran have not been evaluated by the FDA, EMA or any other
health authority.
About Primary Hyperoxaluria Type 1 (PH1)
PH1 is an ultra-orphan disease in which excessive oxalate
production results in the deposition of calcium oxalate crystals in
the kidneys and urinary tract and can lead to the formation of
painful and recurrent kidney stones or nephrocalcinosis. Renal
damage is caused by a combination of tubular toxicity from oxalate,
calcium oxalate deposition in the kidneys, and urinary obstruction
by calcium oxalate stones. Compromised kidney function exacerbates
the disease as the excess oxalate can no longer be effectively
excreted, resulting in subsequent accumulation and crystallization
in bones, eyes, skin, and heart, leading to severe illness and
death. About 50 percent of patients will have kidney failure by age
15, and about 80 percent will have end stage renal disease by age
30. Current treatment options for advanced disease are very limited
and include frequent renal dialysis or combined organ
transplantation of liver and kidneys, a procedure with high
morbidity that is limited due to organ availability. Although a
small minority of patients respond to Vitamin B6 supplementation,
there are no approved pharmaceutical therapies for PH1.
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 major new class of medicines, known as RNAi therapeutics,
is on the horizon. 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 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 (Nasdaq: ALNY) is leading 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 genetic, cardio-metabolic, and hepatic infectious diseases.
Based on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of severe and debilitating diseases. Founded in 2002, Alnylam
is delivering on a bold vision to turn scientific possibility into
reality, with a robust discovery platform and deep pipeline of
investigational medicines, including four product candidates that
are in late-stage development. Looking forward, Alnylam will
continue to execute on its "Alnylam 2020" strategy of building a
multi-product, commercial-stage biopharmaceutical company with a
sustainable pipeline of RNAi-based medicines to address the needs
of patients who have limited or inadequate treatment options.
Alnylam employs over 700 people in the U.S. and Europe and 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 or on LinkedIn.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's views with respect to potential for lumasiran to be a
transformative treatment for patients with PH1, the plans and
timing to advance lumasiran into a pivotal study, expectations
regarding regulatory review and Alnylam’s global commercialization
of lumasiran, if approved, and expectations regarding "Alnylam
2020" guidance for the advancement and commercialization of RNAi
therapeutics, constitute forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. 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, Alnylam's ability
to discover and develop novel drug candidates and delivery
approaches, successfully demonstrate the efficacy and safety of its
product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to
occur in other subjects or in additional studies or otherwise
support further development of product candidates for a specified
indication or at all, actions or advice of regulatory agencies,
which may affect the design, initiation, timing, continuation
and/or progress of clinical trials or result in the need for
additional pre-clinical and/or clinical testing, delays,
interruptions or failures in the manufacture and supply of its
product candidates, obtaining, maintaining and protecting
intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, competition from others using technology
similar to Alnylam's and others developing products for similar
uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business
activities, and establish and maintain strategic business alliances
and new business initiatives, Alnylam's dependence on third parties
for development, manufacture and distribution of products, the
outcome of litigation, the risk of government investigations, and
unexpected expenditures, as well as those risks more fully
discussed in the "Risk Factors" filed with Alnylam's most recent
Annual Report on Form 10-K filed with the Securities and Exchange
Commission (SEC) and in other filings that Alnylam makes with the
SEC. 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.
Lumasiran has not been approved by the FDA, EMA, or any other
regulatory authority and no conclusions can or should be drawn
regarding the safety or effectiveness of this investigational
therapeutic.
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Alnylam Pharmaceuticals, Inc.Investors and
Media:Christine Regan Lindenboom, 617-682-4340orInvestors:Josh
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