Results to be presented show a significant reduction of Lp(a) levels, favorable safety and tolerability profile
BOSTON and CARLSBAD, Calif.,
AKCEA-APO(a)-LRx is designed to inhibit production of apolipoprotein(a), or Apo(a) protein, thereby reducing systemic levels of lipoprotein(a), or Lp(a). 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 treatable patients living with cardiovascular disease and elevated levels of Lp(a).
Following is information about the late-breaking presentation of AKCEA-APO(a)-LRx:
- Safety and Efficacy of AKCEA-APO(a)-LRx to Lower Lipoprotein(a) Levels in Patients With Established Cardiovascular Disease: A Phase 2 Dose-Ranging Trial will be presented by Sotirios Tsimikas, M.D., FACC, FAHA, FSCAI, Univ California San Diego, and vice president of global cardiovascular development at
Ionis Pharmaceuticals, La Jolla, CA (Session LBS.02 – Late Breaking Clinical Trial: Novel Approaches to CV Prevention; Main Event 1)
Akcea will have an on-site presence at the meeting at booth 826 in the Science & Technology Hall where attendees can learn more about TEGSEDI™ (inotersen).
ABOUT AKCEA-APO(a)-LRx AND THE PHASE 2 STUDY
Akcea and Ionis announced positive topline results in
AKCEA-APO(a)-LRx is an antisense drug that uses Ionis’ advanced LIgand Conjugated Antisense, or LICA technology. AKCEA-APO(a)-LRx inhibits the production of apolipoprotein(a), or Apo(a), protein, thereby reducing Lp(a).
Lipoprotein(a), or Lp(a) is made up of apo(a) protein bound to LDL cholesterol and contains oxidized phospholipids, resulting in an atherogenic, pro-inflammatory and thrombogenic lipoprotein. Elevated Lp(a) is recognized as an independent, genetic cause of cardiovascular disease present in approximately 20-30% of the population. Lp(a) levels are determined at birth and, therefore, cannot be well controlled with lifestyle modifications, including diet and exercise.
For additional information about Lp(a), please see the Lipoprotein(a) Foundation at http://www.lipoproteinafoundation.org/.
In January 2017, Akcea entered into an exclusive, worldwide option and collaboration agreement with Novartis to develop and commercialize AKCEA-APOCIII-LRx and AKCEA-APO(a)-LRx. Along with the Phase 2 study of AKCEA-APO(a)-LRx reported here, Akcea is also conducting a Phase 2b dose-ranging study for AKCEA-APOCIII-LRx in patients with hypertriglyceridemia and established cardiovascular disease with data planned in 2019. The goal of both studies is to choose the optimal dose and evaluate alternative dose schedules, such as monthly dosing, for Phase 3 cardiovascular outcomes studies. Novartis has the option to license each drug after completion of the Phase 2 dose-ranging study and end-of-Phase 2 meeting with FDA. Upon option exercise for each drug, Novartis will pay Akcea a $150 million license fee of which 50% will be paid to Ionis. If licensed, Novartis plans to conduct a global Phase 3 cardiovascular outcome study in high-risk patients. Novartis will be responsible for worldwide development and, if approved, co-commercialization activities. As part of the collaboration, Akcea has the rights to co-commercialize Akcea-APO(a)-LRx in selected markets, on mutually agreed terms and conditions.
About AkceaTherapeutics, Inc.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA® (nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease.
Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world’s first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adult patients with hereditary transthyretin (TTR) amyloidosis (ATTR) that our affiliate, Akcea Therapeutics, is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.
To learn more about Ionis follow us on twitter @ionispharma or visit http://ir.ionispharma.com/.
*Spinraza is marketed by Biogen.
AKCEA AND IONIS FORWARD-LOOKINGSTATEMENT
This press release includes forward-looking statements regarding the business of
In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us” refers to
Ionis Pharmaceuticals™ is a trademark of
Akcea Investor Contact:
Vice President of Communications and Investor Relations
Akcea Media Contact:
Berry & Company
T: 212 253-8881
Berry & Company
T: 212 253-8881
Ionis Investor Contact:
D. Wade Walke, Ph.D.
Vice President, Investor Relations
Ionis Media Contact:
Source: Akcea Therapeutics, Inc.