The Medicines Company Presents New Data at the National Lipid Association Scientific Sessions
–Data shows inclisiran has a one-size-fits-all dosing regimen of 300 mg on Day-1, Day-90 and every six months thereafter across a wide range of dyslipidemia patient populations and sub-groups–
The Medicines Company (NASDAQ:MDCO) presented important new data and analyses from the ORION development program for inclisiran at the National Lipid Association (NLA) 2018 Scientific Sessions held in Las Vegas, NV. Multiple studies in the ORION development program demonstrate that the proposed dosing regimen is likely to be the same for a wide range of dyslipidemia patient populations, including those hard-to-treat patients with homozygous familial hypercholesterolemia (HoFH) and other sub-groups.
Dr. David Kallend, MBBS, Chief Medical Officer of The Medicines Company, presented a range of new data and analysis during a Late Breaker session titled “The Efficacy and Safety of Inclisiran, An RNAi Therapeutic Targeting PCSK9, in Different Patient Populations”, demonstrating that the inclisiran dosing regimen (300 mg injection administered on Day-1, Day-90 and then every six months thereafter) achieved substantial PCSK9 and low-density lipoproteins cholesterol (LDL-C) lowering in patients with HoFH and primary dyslipidemia, as well as in various sub-groups, such as patients with renal impairment and diabetes. The data showed that inclisiran lowered LDL-C by more than 50% across a wide range of dyslipidemia patient populations and sub-groups, and by up to 44% in HoFH patients.
Commenting on the data, Principal Investigator for the ORION-1 and ORION-11 trials, Professor Kausik Ray, Professor of Public Health, Imperial College London, United Kingdom, and honorary consultant cardiologist, Imperial College NHS Trust, said, “The data demonstrate, yet again, inclisiran’s emerging, best-in-class therapeutic profile, as well as its 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.”
John J.P. Kastelein, M.D., Ph.D., Professor of Medicine and Chairman of the Department of Vascular Medicine at the Academic Medical Center of the University of Amsterdam, and Chairman of the ORION-1 and ORION-9, ORION-10 and ORION-11 Steering Committees, said, "New therapies are needed to treat HoFH who are refractory or intolerant to current approaches for the management of their LDL-C levels. The simplicity and convenience of a one-size-fits-all dosing regimen, without the necessity of dose adjustment, is a highly-attractive therapeutic option for health care professionals, and could make a significant difference in busy clinical practice.”
Dr. Kallend added, “The data presented confirm the consistent efficacy of inclisiran’s dosing regimen across all patient populations and sub-groups, with no safety concerns observed in any patient population.”
ORION-1 was a placebo-controlled, double-blind, randomized Phase II trial 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 trial compared the effect of different doses of inclisiran and evaluated the potential for an infrequent dosing regimen. The primary endpoint of the trial was the percentage change in LDL-C from baseline at Day-180.
ORION-2 is an ongoing single-arm, open-label pilot study of inclisiran in patients with genetically-confirmed homozygous familial hypercholesterolemia. The primary endpoint is the percentage change in LDL-C from baseline to Day-90 and Day-180.
ORION-7 was a Phase I, open-label, parallel-group trial to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of a single 300 mg dose of inclisiran in patients with mild, moderate and severe renal impairment, compared to patients with normal renal function. The primary endpoint of the trial was to evaluate the impact of renal impairment on the pharmacokinetics of inclisiran and establish dosing recommendations for these patients.
Inclisiran is an investigational GalNAc-conjugated RNA interference therapeutic, which inhibits the synthesis of PCSK9 protein in liver cells, thereby reducing liver cell LDL-receptor turnover, and lowering plasma LDL-C.
The Medicines Company and Alnylam Pharmaceuticals, Inc. are collaborating in the advancement of inclisiran pursuant to their 2013 agreement. Under the terms of that agreement, Alnylam completed certain pre-clinical studies and the Phase I clinical study, with The Medicines Company leading and funding the development of inclisiran from Phase II forward, as well as potential commercialization.
About The Medicines Company
The Medicines Company is a biopharmaceutical company driven by an overriding purpose – to save lives, alleviate suffering and contribute to the economics of healthcare. The Company’s goal is to create transformational solutions to address the most pressing healthcare needs facing patients, physicians and providers in cardiovascular care. The Company is headquartered in Parsippany, New Jersey.
Statements in this presentation about The Medicines Company (the Company), the Company’s products and product candidates, the timing of clinical trial results, regulatory submissions, product or indication launches, the Company’s strategy, future financial results and operations, and future opportunities for the Company, that are not purely historical, and all other statements 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," “plans,“ “expects," “intends," “estimates," “potential," “outlook,” “may,” “will,” “would,””could,” and similar expressions are intended to identify forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward looking statements, including: whether the Company’s product candidate will advance in the clinical trials process on a timely basis or at all; whether clinical trial results will warrant submission of applications for regulatory approval; whether the Company’s product candidate will receive approvals from regulatory agencies; the extent of the commercial success of the Company’s product candidate, if approved; and such other factors as are set forth in the risk factors detailed from time to time in the Company’s periodic reports filed with the Securities and Exchange Commission (SEC) including, without limitation, the risk factors detailed in the Company’s Annual Report on Form 10-K filed with the SEC on March 1, 2018, which are incorporated herein by reference. The Company specifically disclaims any obligation to update these forward-looking statements whether as a result of new information, future events or otherwise.
The Medicines Company
Krishna Gorti, M.D.
Vice President, Investor Relations