The Medicines Company and Alnylam Pharmaceuticals Report Positive Final Results from ORION-1 Phase II Study of Inclisiran
Mar 17, 2017
—Inclisiran demonstrated significant and sustained reductions in LDL-C and high standards of safety and tolerability—
—Optimal starting dose regimen (300 mg injection administered on Day-1 and Day-90) lowered LDL-C by average of 52.6% (64 mg/dL) and up to 81% (122 mg/dL) at Day-180, and by time-adjusted mean of > 50% (63 mg/dL) for the six-month period from Day-90 through Day-270; every patient displayed significant response and mean LDL-C reductions over this time period were practically constant—
—Robust ORION-1 data reaffirm inclisiran's potential to address unmet needs with highly-differentiated, infrequent, low-volume dosing regimen of two or three injections per year—
—No material safety issues on inclisiran in ORION-1; overall incidence of adverse events for inclisiran was similar to placebo—
—The Company is actively preparing to advance inclisiran into
comprehensive, global Phase III development - initially focused on
—ORION-1 study presented today in Late-Breaking Clinical Trials
session at
—The Company to host conference call at
This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170317005437/en/
The detailed data from ORION-1 showed that inclisiran delivered significant and sustained reductions of LDL-C and high standards of safety and tolerability. Remarkably, every patient who received the two dose starting regimen displayed a significant response and the mean LDL-C reductions over time were practically constant. Inclisiran was well tolerated and no material safety issue was observed, including no investigational drug-related elevation of liver enzymes and no neuropathy, change in renal function, thrombocytopenia, or anti-drug antibodies.
"The groundbreaking results from ORION-1 are compelling and affirm
inclisiran's unique and highly-differentiated attributes, and its
game-changing potential to address the unmet needs of millions of
at-risk, often non-adherent, patients worldwide who continue to struggle
with high cholesterol given the limitations of available therapies,"
said
In ORION-1, the mean baseline LDL-C was approximately 130 mg/dL among 497 randomized and treated patients. The optimal starting dose regimen (300 mg injection administered on Day-1 and Day-90) achieved a mean LDL-C reduction of 52.6% and up to 81% at Day-180, and a time-adjusted mean of > 50% for the six month period from Day-90 through Day-270. For all dose groups, at all time points, differences in the primary (LDL-C) and secondary (PCSK9) endpoints between inclisiran and placebo were statistically significant (p < 0.0001).
The overall incidence of treatment emergent adverse events was 76% in both patients randomized to placebo and patients randomized to inclisiran, with no significant difference between inclisiran doses. Injection site reactions associated with inclisiran were infrequent (observed in 6.5% of patients given the two dose starting regimen and 3.8% of patients given the one dose starting regimen), mild or moderate, and transient.
The results from the Phase II study of inclisiran, published in today's
online issue of
Webcast Information for Late-Breaking Clinical Trial Presentations
Live audio and video of the "Late-Breaking Clinical Trials -
Conference Call and Webcast Details
|
|
(877) 359-9508 | ||||
| International: | (224) 357-2393 | ||||
| Conference ID: | 60380330 | ||||
The live webcast may be accessed in the "Investors-Events/Presentations"
section of
A taped replay of the conference call will be archived and available for approximately one week. The replay may be accessed as follows:
|
|
(855) 859-2056 | ||||
| International: | (404) 537-3406 | ||||
| Conference ID: | 60380330 | ||||
A replay of the webcast will also be archived and available after the conference call.
About ORION-1
ORION-1 is a placebo-controlled, double-blind, randomized Phase II study of single or multiple subcutaneous injections of inclisiran in a total of 501 patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents (e.g., diabetes and familial hypercholesterolemia) and elevated LDL-C despite maximum tolerated doses of LDL-C lowering therapies. The study compares the effect of different doses of inclisiran and evaluates the potential for an infrequent dosing regimen. The primary endpoint of the study is the percentage change in LDL-C from baseline at Day-180.
About Inclisiran
Inclisiran (formerly known as PCSK9si and ALN-PCSsc) is an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9 - a genetically validated protein regulator of LDL receptor metabolism - being developed for the treatment of hypercholesterolemia. In contrast to anti-PCSK9 monoclonal antibodies (MAbs) that bind to PCSK9 in blood, inclisiran is a first-in-class investigational medicine that acts by turning off PCSK9 synthesis in the liver.
About The Medicines Company
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,
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 Securities and Exchange Commission, including, without limitation, the risk factors detailed in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2017, which are incorporated herein by reference. The Company specifically disclaims any obligation to update these forward-looking statements.
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 RNAi therapeutics,
including inclisiran, its expectations regarding the timing of clinical
studies, its expectations regarding scientific and regulatory support
for inclisiran, 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 referenced in this news release relating to
inclisiran is preliminary and investigative. Inclisiran has not been
approved by the
View source version on businesswire.com: http://www.businesswire.com/news/home/20170317005437/en/
Media:
Vice President
margaret.langan@themedco.com
Investors:
Vice President, Investor Relations
krishna.gorti@themedco.com
or
Investors and Media:
Vice President
clindenboom@alnylam.com
Investors:
Associate Director
jbrodsky@alnylam.com
Source:
News Provided by Acquire Media
For Media Inquiries, please contact:
For Investor Inquiries, please contact:
MEDIA KIT
Essential assets and documents related to Alnylam
VIEW ALL PRESS RELEASES
FOLLOW US
