Akcea Therapeutics has announced it had completed enrollment of a phase 2b clinical study of investigational drug AKCEA-APO(a)-LRx. Akcea is conducting the study in patients with high Lp(a) and established cardiovascular disease (CVD) to determine the dose level and frequency of administration for a future planned phase 3 cardiovascular outcome study and to determine the safety and tolerability profile of AKCEA-APO(a)-LRx.

Akcea plans to report top-line data from the phase 2b study in the second half of 2018. Akcea is an affiliate of Ionis Pharmaceuticals Inc. focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders.

AKCEA-APO(a)-LRx is part of a strategic collaboration with Novartis. If Novartis exercises its option to license AKCEA-APO(a)-LRx after successful completion of the phase 2b study, it will be responsible for all future development activities for AKCEA-APO(a)-LRx including a global phase 3 cardiovascular outcome study and, if approved, global commercialization activities. As part of the agreement, Akcea retains the right to co-commercialize globally.

“Elevated Lp(a) is a genetic trait that is present in approximately 20-30% of the population and is recognized as a risk factor for cardiovascular disease.  There are currently no treatment options available that specifically target Lp(a). AKCEA-APO(a)-LRx is the only programme in clinical development for this indication that has been shown to substantially lower Lp(a),” said Mustafa Noor, MD, chief development officer of Akcea Therapeutics. “We now look forward to completing the study and beginning analysis of the study data. The results of this study will help us better understand the efficacy and safety profile of AKCEA-APO(a)-LRx, so that this program can advance into a pivotal cardiovascular outcome study to determine the potential benefit of lowering Lp(a).”

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 lipoprotein(a), or Lp(a).  Lp(a) is made up of apo(a) protein bound to LDL cholesterol resulting in an atherogenic and thrombogenic lipoprotein that has been genetically validated as an independent risk factor for coronary artery disease, heart attack, stroke, calcific aortic valve disease and peripheral arterial disease. phase 1 studies of all three of Akcea’s LICA drugs have shown that doses up to 30-fold lower than non-LICA drugs result in consistent target reductions and a favorable safety and tolerability profile.

The randomized, double-blind, placebo-controlled, dose-ranging phase 2b study is evaluating the safety and efficacy of different doses of AKCEA-APO(a)-LRx. The study enrolled over 270 patients with high Lp(a) and established cardiovascular disease.