Alnylam Initiates Phase 2 Clinical Study of Cemdisiran (ALN-CC5) in Patients with Atypical Hemolytic-Uremic Syndrome (aHUS)
September 26 2017 - 8:00AM
Business Wire
– Company Expects to Report Initial Patient
Data in 2018 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that it has initiated a Phase
2 clinical study with cemdisiran (formerly known as ALN-CC5), a
subcutaneously administered investigational RNAi therapeutic
targeting complement component C5 for the treatment of
complement-mediated diseases. The trial is being conducted in
patients with atypical hemolytic-uremic syndrome (aHUS) and will
evaluate the effect of C5 knockdown on hematologic response and
renal function, as well as safety and tolerability. The Company
expects to report initial clinical proof of concept data from this
trial in late 2018.
"C5 is a clinically validated target in patients with aHUS. We
believe cemdisiran can potentially offer clinical benefit for
patients and physicians with durable maintenance of disease
remission with an infrequent subcutaneous injection,” said Thomas
Hoock, Ph.D., Vice President, Program Lead, Cemdisiran at Alnylam.
"We believe that clinical activity for cemdisiran as monotherapy,
if demonstrated in the Phase 2 aHUS study, has the potential to
open broader opportunities for this investigational RNAi
therapeutic in other complement-mediated diseases, and we look
forward to initial data in 2018."
Cemdisiran Phase 2 Study DesignThe Phase 2 trial will
follow an adaptive study design with an initial cohort of 12 aHUS
patients who are not currently treated with an anti-C5 monoclonal
antibody. Patients will receive a 600 mg dose of cemdisiran once
every 4 weeks, with an option of reduced dose level and/or dose
frequency in a subsequent cohort. The primary endpoint of the Phase
2 study in aHUS patients is normalization of platelet counts, with
secondary endpoints including hematologic and thrombotic
microangiopathy (TMA) response, improvement in renal function,
safety and tolerability.
About CemdisiranFormerly known as ALN-CC5, cemdisiran
(pronounced “sem-DEE-si-ran”) is an investigational RNAi
therapeutic targeting the C5 component of the complement pathway in
development for the treatment of complement-mediated diseases. The
complement system plays a central role in immunity as a protective
mechanism for host defense, but its dysregulation results in
life-threatening complications in a broad range of human diseases
including paroxysmal nocturnal hemoglobinuria (PNH), atypical
hemolytic-uremic syndrome (aHUS), myasthenia gravis, neuromyelitis
optica, and membranous nephropathy, amongst others. Complement
component C5, which is predominantly expressed in liver cells, is a
genetically and clinically validated target; loss of function human
mutations are associated with an attenuated immune response against
certain infections and intravenous anti-C5 monoclonal antibody
(mAb) therapy has demonstrated clinical activity and tolerability
in a number of complement-mediated diseases. A subcutaneously
administered RNAi therapeutic that silences C5 represents a novel
approach for the potential treatment of complement-mediated
diseases. Cemdisiran utilizes Alnylam's ESC-GalNAc conjugate
technology, which enables subcutaneous dosing with increased
potency and durability and a wide therapeutic index.
The safety and efficacy of cemdisiran have not been evaluated by
the U.S. Food and Drug Administration or any other health
authority.
About RNAiRNAi (RNA interference) is a revolution in
biology, representing a breakthrough in understanding protein
synthesis in cells, and a completely new approach to drug discovery
and development. Its discovery has been heralded as "a major
scientific breakthrough that happens once every decade or so," and
represents one of the most promising and rapidly advancing
frontiers in biology and drug discovery today which was awarded the
2006 Nobel Prize for Physiology or Medicine. RNAi is a natural
process of gene silencing that occurs in organisms ranging from
plants to mammals. By harnessing the natural biological process of
RNAi occurring in our cells, the creation of 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, target the cause of
diseases by potently silencing specific mRNAs, with the goal of
preventing disease-causing proteins from being made.
About Alnylam PharmaceuticalsAlnylam (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 patients who have limited or inadequate treatment options.
Based on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of 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. 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 StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including without limitation, Alnylam's views with
respect to the potential for cemdisiran for the treatment of
patients with aHUS and potentially other complement-mediated
diseases, expectations regarding the timing for initial clinical
data from a Phase 2 clinical study of cemdisiran and expectations
regarding its "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
Quarterly Report on Form 10-Q 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.
Cemdisiran has not been approved by the U.S. Food and Drug
Administration, European Medicines Agency, 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, 617-682-4340or(Investors)Josh
Brodsky, 617-551-8276
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