Notre pipeline
Notre pipeline
Amyloïdose à transthyrétine (ATTR)
AKCEA-TTR-LRx for the treatment of ATTR
We are co-developing AKCEA-TTR- LRx with Ionis Pharmaceuticals to inhibit the production of transthyretin, the same protein inhibited by inotersen. We plan to develop AKCEA-TTR-LRx for patients with both the hereditary and the wild type form of the disease. AKCEA-TTR-LRx is planned to enter clinical development in 2018.
Cardiometabolic lipid disorders
Volanesorsen for the treatment of FCS
Akcea’s lead program, volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for rare metabolic disorders including familial chylomicronemia syndrome (FCS).
The Phase 3 study in patients with FCS, called APPROACH FCS, met its primary endpoints of triglyceride reduction and showed a statistically significant decrease in pancreatitis attacks in FCS patients with a history of high frequency pancreatitis. We are in the process of preparing the regulatory filing as well as commercial build-out to support the anticipated launch of this program.
Patients with FCS who have completed or meet the study criteria for the APPROACH and COMPASS studies can enroll in an open-label extension (APPROACH OLE) study. For more information on this study, please visit www.clinicaltrials.gov.
Volanesorsen for the treatment of FPL
Akcea’s lead program, volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for rare metabolic disorders including familial chylomicronemia syndrome (FCS).
The Phase 3 study in patients with FPL, called BROADEN FPL, is currently enrolling and we plan to report data from this study in 2019. For more information on this study, please visit www.clinicaltrials.gov.
Patients in the BROADEN study are also eligible to roll over into an open-label extension study upon completing dosing in the pivotal study.
AKCEA-APO(a)-LRx for the treatment of CVD driven by hyperlipoproteinemia(a)
We are 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. For more information on this study, please visit www.clinicaltrials.gov. This program is part of our exclusive, worldwide option and collaboration agreement with Novartis.
AKCEA-ANGPTL3-LRx for the treatment of rare hyperlipidemias
We are 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.
The congenital absence of ANGPTL3 is associated with lower lipid levels and reduced risk of insulin resistance, diabetes mellitus and cardiovascular disease relative to individuals with normal production of ANGPTL3 protein.
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). For more information, please visit www.clinicaltrials.gov.
AKCEA-ANGPTL3-LRx for the treatment of NAFLD with metabolic complications
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). For more information on this study, please visit www.clinicaltrials.gov.
AKCEA-APOCIII-LRx for the treatment of CVD driven by high triglycerides
We are 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 on this study, please visit www.clinicaltrials.gov. This program is part of our exclusive, worldwide option and collaboration agreement with Novartis.
Les produits énumérés sur cette page n'ont pas été approuvés.
Vision et mission
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Essais cliniques
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