United Kingdom

Profile: Akcea Therapeutics Inc (AKCA.OQ)

AKCA.OQ on NASDAQ Stock Exchange Global Select Market

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Akcea Therapeutics, Inc., Incorporated on December 22, 2014, is a biopharmaceutical company. The Company is focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. The Company's drugs, volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, are all based on antisense technology developed by Ionis Pharmaceuticals, Inc. (Ionis). The Company's volanesorsen drug has completed a Phase III clinical program for the treatment of familial chylomicronemia syndrome (FCS) and is currently in Phase III clinical development for the treatment of familial partial lipodystrophy (FPL). The Company's clinical pipeline contains drugs with the potential to treat inadequately addressed lipid disorders beyond elevated LDL-C that are contributing to the dramatic increase in the incidence of cardiometabolic disease, such as elevated triglycerides, oxidized phospholipids and other lipoproteins, such as lipoprotein(a), or Lp(a).

Each of the four drugs in the Company's pipeline targets the specific ribonucleic acid, or RNA, that encodes for a unique protein associated with lipid dysfunction, robustly and selectively inhibiting the production of such protein. Additionally, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx utilize Ionis' advanced Ligand Conjugated Antisense (LICA) technology. The Company has completed a Phase I/II study with AKCEA-APO(a)-LRx in patients with hyperlipoproteinemia(a). The Company is engaged in developing AKCEA-ANGPTL3-LRx to treat multiple lipid disorders. The Company is engaged conducting a Phase I/II program for AKCEA-ANGPTL3-LRx in people with elevated triglycerides. The Company is also engaged developing AKCEA-APOCIII-LRx to inhibit the production of ApoC-III, the same protein inhibited by volanesorsen, for the broad population of patients having cardiometabolic disease due to their elevated triglyceride levels. The Company is engaged conducting a Phase I/II study of AKCEA-APOCIII-LRx.

The Company competes with uniQure N.V., Regeneron Pharmaceuticals, Inc. and Gemphire Therapeutics, Inc.

Company Address

Akcea Therapeutics Inc

55 Cambridge Pkwy Ste 100
CAMBRIDGE   MA   02142-1219
P: +1760.6032732
F: +1302.6555049

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