Nov 13, 2016 Press Release for Alnylam
Alnylam Pharmaceuticals and The Medicines Company Announce Publication of Phase 1 Clinical Data with Inclisiran (ALN- PCSsc) in the New England Journal of Medicine
Nov 13, 2016
Interim Results from ORION-1 Phase 2 Study of Inclisiran to be
Presented in Late-Breaking Clinical Trial Session at the American Heart
Association Scientific Sessions on
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Results from the study showed doses ≥300 mg (single or multiple doses) significantly reduced PCSK9 and LDL cholesterol for at least 6 months. Moreover, inclisiran was found to be generally well tolerated.
"The published findings from our Phase 1 trial with inclisiran add to
the clinical evidence supporting PCSK9 as a therapeutic target for
significantly lowering LDL cholesterol, as well as the ability of RNAi
therapeutic candidates to inhibit synthesis of liver-derived target
proteins in a potent and durable manner," said
Inclisiran is currently being studied in the ORION-1 Phase 2 study by
"We look forward to presenting the interim results of the ORION-1 Phase
2 data at AHA this week including Day 90 follow-up results for all 501
patients and a preliminary analysis of Day 180 follow-up for up to 200
patients," said
The NEJM publication highlights key results from the Phase 1 clinical trial of inclisiran, including safety and pharmacodynamic measures (PCSK9, LDL cholesterol, exploratory lipid parameters).
In the single-ascending-dose (SAD) phase, pharmacodynamic measures showed:
- Doses ≥300 mg reduced PCSK9 at day 84 (up to a least-squares mean (LSM) reduction of 74.5%);
- Doses ≥100 mg reduced LDL cholesterol at day 84 (up to a LSM reduction of 50.6%);
- Reductions in PCSK9 and LDL cholesterol were maintained at day 180 with little variation over the 6-month period for doses ≥300 mg.
In the multiple-dose (MD) phase, pharmacodynamic measures showed:
- Reduced PCSK9 (up to a LSM reduction of 83.8%) and LDL cholesterol (up to a LSM reduction of 59.7%) at day 84;
- Levels of PCSK9 and LDL cholesterol remained reduced in all the inclisiran cohorts at day 196.
Safety and side effect profile evaluations showed:
- Inclisiran was generally well tolerated following single and multiple subcutaneous dose administration;
- No serious adverse events (SAEs) or discontinuations due to AEs were reported;
- All observed adverse events (AEs) were mild or moderate in severity;
- There was one Grade 3 GGT elevation considered related to statin therapy.
The lead development responsibility for inclisiran transitioned from
Alnylam to
About the Inclisiran Phase 1 Study
The Phase 1 trial of inclisiran was conducted in the
About Hypercholesterolemia
Hypercholesterolemia is a condition characterized by very high levels of
cholesterol in the blood which is known to increase the risk of coronary
artery disease, the leading cause of death in the
About GalNAc Conjugates and Enhanced Stabilization Chemistry (ESC)-GalNAc Conjugates
GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology enables subcutaneous dosing with increased potency and durability, and a wide therapeutic index. This ESC-GalNAc-conjugate delivery platform is being employed in nearly all of Alnylam's pipeline programs, including inclisiran and several other programs in clinical development.
About RNAi
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off 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, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is leading the
translation of RNAi as a new class of innovative medicines. Alnylam's
pipeline of investigational RNAi therapeutics is focused in 3 Strategic
Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of
RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic
Disease, with a pipeline of RNAi therapeutics toward genetically
validated, liver-expressed disease targets for unmet needs in
cardiovascular and metabolic diseases; and Hepatic Infectious Disease,
with a pipeline of RNAi therapeutics that address the major global
health challenges of hepatic infectious diseases. In early 2015, Alnylam
launched its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics as a whole new class of
innovative medicines. Specifically, by the end of 2020, Alnylam expects
to achieve a company profile with 3 marketed products, 10 RNAi
therapeutic clinical programs - including 4 in late stages of
development - across its 3 STArs. The company's demonstrated commitment
to RNAi therapeutics has enabled it to form major alliances with leading
companies including Ionis, Novartis, Roche, Takeda,
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 the potential for inclisiran, including
the potential dosing regimen, the timing of clinical studies and the
presentation of clinical data, its expectations regarding its STAr
pipeline growth strategy, and 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 our 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-
The scientific information discussed in this news release relating to
inclisiran is preliminary and investigative. Inclisiran has not been
approved by the
About The Medicines Company
Forward Looking Statements
Statements contained in this press release that are not purely
historical may be deemed to be forward-looking statements for purposes
of the safe harbor provisions under The Private Securities Litigation
Reform Act of 1995. Without limiting the foregoing, the words
"believes," "anticipates," "expects," "potential," and similar
expressions are intended to identify forward-looking statements. These
forward-looking statements involve known and unknown risks and
uncertainties that may cause the Company's actual results, levels of
activity, performance or achievements to be materially different from
those expressed or implied by these forward-looking statements.
Important factors that may cause or contribute to such differences
include whether clinical trials for inclisiran, will advance in the
clinical process on a timely basis, or at all, or succeed in achieving
their specified endpoints; whether physicians, patients and other key
decision makers will accept clinical trial results; whether the Company
will make regulatory submissions for inclisiran on a timely basis, or at
all; whether its regulatory submissions will receive approvals from
regulatory agencies on a timely basis, or at all; and such other factors
as are set forth in the risk factors detailed from time to time in the
Company's periodic reports and registration statements filed with the
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(Investors and Media)
or
Investors
or
Media:
Vice President
margaret.langan@themedco.com
or
Investors:
Vice President, Investor Relations
Krishna.Gorti@themedco.com
Source:
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