Clinical Trials

Clinical Trials

Akcea is developing therapeutic therapies in two general areas of serious and rare diseases: ATTR amyloidosis and cardiometabolic lipid disorders. Akcea focuses on disrupting the production of key proteins involved in disease processes. While traditional drugs modify proteins involved in diseases, the drugs that are being developed by Akcea actually decrease the production of these proteins in the first place. Learn more about the technology platform behind our pipeline of drug therapies.

ATTR amyloidosis

Inotersen for the treatment of patients with polyneuropathy due to hereditary ATTR amyloidosis (hATTR)

An open-label extension study, or OLE, is ongoing for patients who have completed the Phase 3 NEURO-TTR study, in which all patients are treated with inotersen.

An expanded access program (EAP) is also available for eligible patients with hATTR. For more information, please visit this link.

Akcea’s other therapy for ATTR amyloidosis, AKCEA-TTR-LRx, is in preclinical development.

Cardiometabolic lipid disorders

Cardiometabolic lipid disorders are complex, with a multitude of risk factors. There is a growing body of evidence through genomics and epidemiological studies supporting the need for new therapies targeting the protein and lipid risk factors associated with these cardiometabolic lipid disorders.

Lipid and lipoprotein risk factors, including LDL-C, triglycerides, apolipoprotein C-III (ApoC-III) and Lp(a), are related to many diseases, both rare and relatively common. They result in a variety of unmet medical needs, varying in severity, and manifesting in a range of clinical presentations including pancreatitis, diabetes, liver disease, and multiple forms of cardiovascular disease.

Akcea is advancing a mature pipeline of four novel investigational drugs with the potential to treat multiple indications. Volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, are all based on antisense technology developed by Ionis Pharmaceutics, Inc. Our most advanced drug, volanesorsen, is initially intended for two serious and rare lipid disorders, familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL).

While traditional drugs modify proteins involved in diseases, the drugs that are being developed by Akcea actually decrease the production of these proteins in the first place. Learn more about the technology platform behind our pipeline of drug candidates.

Volanesorsen for the treatment of FCS and FPL

Volanesorsen is an investigational new drug designed to reduce the amount of apolipoprotein C-III (ApoC-III), a protein that is a key regulator of triglyceride levels, in the bloodstream. Four global trials have formed the Phase 3 program for volanesorsen. Two of these studies – the APPROACH Study in patients with FCS and the COMPASS Study in patients with severe hypertriglyceridemia – both met their primary endpoints of reducing triglycerides in the respective patient populations and showed a statistically significant decrease in pancreatitis attacks in FCS patients with a history of high frequency pancreatitis. Two additional Phase 3 studies are ongoing:

Read more about the results of the APPROACH Study.

Following completion of the APPROACH clinical trial, the pivotal phase 3 study of volanesorsen in FCS, Akcea is now conducting the APPROACH Open Label Study, a multi-center study to further evaluate the efficacy of volanesorsen measured by percent change in fasting triglyceride levels from baseline and to assess the durability of the efficacy with extended administration of volanesorsen.

Akcea is conducting the BROADEN Study, a randomized, placebo-controlled, double-blind Phase 2/3 clinical trial for volanesorsen in the treatment of FPL, that is currently enrolling patients. The main purpose of the BROADEN Study is to assess the effects of volanesorsen on triglyceride levels and other metabolic derangements in people with FPL.

AKCEA APO(a) LRx for the treatment of CVD driven by hyperlipoproteinemia(a)

Akcea is developing AKCEA APO(a) LRx in multiple indications for patients who are at significant risk of CVD because of their elevated levels of Lp(a). AKCEA APO(a) LRx inhibits the production of the apolipoprotein(a), or Apo(a), protein, thereby reducing Lp(a), a very atherogenic (promoting the formation of plaques in the arteries) and thrombogenic (promoting the formation of blood clots) form of low density lipoprotein, or LDL.

We have initiated a Phase 2 study of AKCEA APO(a) LRx in patients with hyperlipoproteinemia(a) with established CVD.

AKCEA ANGPTL3 LRx for the treatment of rare hyperlipidemias

Akcea is developing AKCEA ANGPTL3 LRx to treat multiple lipid disorders, or rare hyperlipidemias. In preclinical studies, an analog of AKCEA ANGPTL3 LRx inhibited the production of the angiopoietin like 3, or ANGPTL3, protein in the liver, inhibiting liver fat accumulation and lowering blood levels of LDL C and very low density lipoprotein cholesterol, or VLDL C.

We have initiated an exploratory Phase 2 program of AKCEA-ANGPTL3-LRx which is planned to include three studies recruiting patients with one of three rare hyperlipidemias, including familial chylomicronemia syndrome (FCS), familial partial lipodystrophy (FPL), and homozygous familial hypercholesterolemia (HoFH).

AKCEA-ANGPTL3-LRx for the treatment of NAFLD

We have initiated a Phase 2b study that will evaluate the safety and efficacy of different doses of AKCEA-ANGPTL3-LRx in approximately 144 patients with hypertriglyceridemia, type 2 diabetes mellitus and nonalcoholic fatty liver disease (NAFLD).

AKCEA APOCIII LRx for the treatment of CVD driven by high triglycerides

Akcea is developing AKCEA APOCIII LRx to inhibit the production of ApoC III, the same protein inhibited by volanesorsen, for the broad population of patients who are at risk for cardiometabolic disease due to their elevated triglyceride levels.

We have initiated a Phase 2b study to evaluate the safety and efficacy of different doses and dose intervals of AKCEA-APOCIII-LRx in approximately 100 patients with hypertriglyceridemia and established cardiovascular disease (CVD).

For more information about Akcea clinical trials, please contact us at research@akceatx.com.