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Akcea and Ionis' Triglyceride-Lowering Drug Works in Mid-Stage Clinical Trial

By Brian Orelli, PhD - Updated Jan 22, 2020 at 4:54PM

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The drug will advance to studies in familial chylomicronemia syndrome and other diseases that result in high triglyceride levels.

Akcea Therapeutics (AKCA) and Ionis Pharmaceuticals (IONS -0.05%) reported positive results from a phase 2 clinical trial testing AKCEA-APOCIII-Lrx in patients with high triglyceride levels who either have cardiovascular disease or are at high risk for developing it. The results sent shares of Akcea up 11% on Wednesday, while the larger Ionis was up a more modest 2%.

In the 114-patient study, over 90% of patients taking the 50 mg once-monthly dose were able to lower their serum triglycerides to the target of 150 mg/dL or lower, while only 5% of patients taking a placebo met the goal. The patients started the study with an average triglyceride level of 285 mg/dL.

The biotech companies also noted that the drug was able to reduce risk factors for cardiovascular disease, including apoC-III, very low-density lipoprotein and remnant cholesterol, compared to placebo, although they didn't put numbers to the claims. The drug also increased high-density lipoprotein cholesterol, which is the good kind of cholesterol.

Blood vials on a diagnostic test order form

Image source: Getty Images.

AKCEA-APOCIII-Lrx works by targeting the mRNA that makes apolipoprotein C-III (apoC-III), a protein that regulates the level of serum triglycerides. The drug was discovered by Ionis and moved to Akcea when the company was spun off from Ionis.

Akcea plans to advance AKCEA-APOCIII-Lrx as a treatment for familial chylomicronemia syndrome, a genetic disease where the patients can't break down fat, resulting in increased levels of triglycerides. The company said it's also considering testing the drug in other diseases where patients have elevated triglyceride levels.

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