Mar 12, 2018 Press Release for Alnylam
Alnylam Retains Global Rights to Lumasiran, an Investigational RNAi Therapeutic for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Mar 12, 2018
− Company Plans to
− Lumasiran Granted Breakthrough Therapy Designation by the
“We are extremely pleased to retain worldwide development and
commercialization rights for lumasiran, expanding the number of global
rare disease opportunities for Alnylam. Based on Phase 1/2 clinical data
presented to date, we believe lumasiran could be a transformative
treatment for patients with PH1, an ultra-rare disease with no approved
therapies, in which excess oxalate production leads to kidney failure
and significant morbidity and mortality,” said
The clinical dataset informing the Sanofi Genzyme decision consisted of
results from Parts A and B of the ongoing Phase 1/2 study of lumasiran,
recently presented
at the
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. The safety and efficacy of
lumasiran have not been evaluated by the
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.
Alnylam –
In
In
About
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
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 Alnylam’s global commercialization of
lumasiran, if approved, the continuing option rights of Sanofi Genzyme
through 2019 and potentially beyond, 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-
Lumasiran has not been approved by the
View source version on businesswire.com: http://www.businesswire.com/news/home/20180312005358/en/
Source:
Alnylam Pharmaceuticals, Inc.
Investors and Media
Christine
Regan Lindenboom, 617-682-4340
or
Investors
Josh Brodsky,
617-551-8276
For Media Inquiries, please contact:
Christine Lindenboom
SVP, Investor Relations & Corporate Communications media@alnylam.com 617-682-4340
For Investor Inquiries, please contact:
Josh Brodsky
VP, Investor Relations & Corporate Communications investors@alnylam.com 617-551-8276
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