Akcea and Ionis Report Positive Data from Phase 2 Study of AKCEA-APO(a)-LRx

Study demonstrates significant Lp(a) reduction, favorable safety and tolerability profile

Ionis Pharmaceuticals, Inc.


Largest and longest study of Ionis’ LICA technology

Data to be presented as a late-breaking clinical trial presentation at AHA on November 10 in Chicago

CAMBRIDGE, Mass. and CARLSBAD, Calif., Sept. 24, 2018 (GLOBE NEWSWIRE) --  Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced positive topline results from a Phase 2 clinical study of AKCEA-APO(a)-LRx in patients with established cardiovascular disease (CVD) and elevated levels of lipoprotein(a), or Lp(a).  Additional data from the Phase 2 study will be presented as a late-breaking clinical trial presentation at the American Heart Association Scientific Sessions in Chicago November 10-12, 2018.

The goal of the Phase 2 study was to characterize the safety and tolerability of AKCEA-APO(a)-LRx and to inform dose and dose frequency selection for the planned Phase 3 cardiovascular outcomes study.  The randomized, double-blind, placebo-controlled, dose-ranging study included 286 patients with established CVD and high Lp(a) (baseline mean of approximately 100 mg/dL [250 nmol/L] – more than three times the upper limit of normal). All patients were treated for at least six months, with some patients treated up to one year. Results from the study show:

  • Statistically significant dose-dependent reductions of Lp(a) compared to placebo at all dose levels, including low monthly doses of AKCEA-APO(a)-LRx.
  • Most patients in the active group achieved Lp(a) reductions below the established threshold of risk for CVD events.
  • Treatment emergent adverse events were balanced between the active and placebo groups.
  • Most common adverse event was injection site reactions (ISRs). ISRs were mostly mild and occurred in a minority of patients.
  • No patient in the study experienced a confirmed platelet level below 100,000/mm3. The incidence of platelet levels below normal (140,000/mm3) was comparable between the active (10.5%) and placebo (14.9%) groups.
  • Approximately 90% of patients completed treatment and the rate of treatment discontinuation was comparable between the active and placebo groups.

“These data represent an important step forward for patients who have significant risk of premature death from cardiovascular disease due to their high levels of Lp(a). In this large Phase 2 study AKCEA-APO(a)-LRx, robustly lowered Lp(a) with a favorable safety and tolerability profile. In addition, the data from this study support the potential to treat patients with convenient, low volume monthly doses,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “These results are also encouraging as we continue to develop our LICA pipeline.” 

Elevated Lp(a) is an independent, hereditary risk factor for CVD that cannot be well-controlled with lifestyle modifications, such as diet or exercise, or with treatment using existing cholesterol-lowering therapies. It is estimated that there are 8 to 10 million people living with established cardiovascular disease driven by elevated levels of Lp(a). The Phase 2 clinical study of AKCEA-APO(a)-LRx is the largest study conducted specifically for patients with elevated Lp(a).

“After my personal experience at age 39 with severe Lp(a)-induced heart disease and nearly having a heart attack myself, I have met many others living with this silent killer that is under-recognized in the wider population,” said Sandra Revill Tremulis, founder of the Lipoprotein(a) Foundation. “Even after diagnosis, with no treatment the increased risk of heart disease and heart attack remains a serious burden. This is an important advancement in the identification of a potential novel medicine for patients with Lp(a)-driven cardiovascular disease.” ...more here

 

Centers for Disease Control & Prevention (CDC) Approves Diagnosis Codes for Elevated Lipoprotein(a)

 

In response to the Lipoprotein(a) Foundation’s 2017 application, two International Classification of Diseases (ICD) 10 codes have now been approved by the CDC for elevated lipoprotein(a). These ICD-10 Diagnosis codes will help identify the global population at risk for early heart attacks, strokes, aortic valve disease and peripheral vascular disease and death due to elevated lipoprotein(a) – “a genetic, sticky, fatty, lipoprotein particle in the blood.”  They will encourage routine diagnosis, treatment, and family screening for elevated lipoprotein(a) before the first symptom, which may be death, while there is still time for prevention.  Elevated lipoprotein(a) is a genetic condition. A simple lipoprotein(a) blood test once in a person's life is required to diagnose a patient.  A healthcare provider cannot tell if a patient has elevated lipoprotein(a) by examining them.

The following codes will go into effect on October 1, 2018:


E78.41          Elevated lipoprotein(a)
Z83.430        Family history of elevated lipoprotein(a)


The need for new diagnostic codes was underscored in the article, “NHLBI Working Group Recommendations to Reduce Lipoprotein(a)-Mediated Risk of Cardiovascular Disease and Aortic Stenosis,” published in the January 2018 issue of the Journal of the American College of Cardiology. In response to advocacy by The Lipoprotein(a) Foundation, the NHLBI convened a strategic task force meeting to better characterize the role of Lp(a) in CVD and calcific aortic valve disease (CAVD). "Without an ICD-10 code for the diagnosis of elevated Lp(a), clinicians have no way to document elevated Lp(a) levels, except with the use of a generic hypercholesterolemia code. The lack of an ICD-10 code also limits research on Lp(a) using electronic health records,” said Sotirios (Sam) Tsimikas, MD, Director, Vascular Medicine, University of California San Diego, lead author of the JACC paper and a member of the Lipoprotein(a) Foundation’s Scientific Advisory Board.

 

"Importantly, more than 1 billion people globally have elevated levels of Lp(a). Convincing evidence has emerged from pathophysiological, epidemiological, and genetic studies that Lp(a) is causally related to both CVD, which includes myocardial infarction, stroke, peripheral arterial disease, and heart failure, and CAVD. Emerging therapies designed to significantly lower Lp(a) levels will allow us to test the hypothesis that reducing Lp(a) will reduce the risk of CVD and CAVD,” noted Henry Ginsberg, MD, Irving Professor of Medicine, Vagelos College of Physicians and Surgeons, Columbia University, New York, and Chief Medical Officer for The Lipoprotein(a) Foundation.


Sandra Tremulis, founder and CEO of the Lipoprotein(a) Foundation stated, "The codes used include monitoring of the incidence and prevalence of diseases, observing reimbursements and resource allocation trends, and keeping track of safety and quality guidelines. They also include the counting of deaths as well as diseases, injuries, symptoms, reasons for encounter, factors that influence health status, and external causes of disease. We want to give families a preemptive strike against their genetic cardiovascular disease destiny and catch it early before it becomes disabling or a tragedy.”

Stroke and Lipoprotein(a)

Young, Fit Football Player Suffering

a Massive Stroke

Justin's Story

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Heart Attack and Lipoprotein(a)

   Young, Fit, Female Having A Heart Attack

Sandra's Story

    

more here

Aortic Stenosis and Lipoprotein(a)

Young, Pregnant having a Heart Attack, Bypass and Valve Replacement Surgery

Tiffany's Story

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Media enquiries, please contact 650-995-3242 or serevill@lipoproteinafoundation.org to find out more about Lp(a).

 

 

 

 

 

 

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