Why This Cardiovascular Disease Study Could Be Huge

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Akcea Therapeutics Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals Inc. (NASDAQ: IONS) each made handy gains on Wednesday after they reported midstage results for their cardiovascular disease study.

Specifically, the results come from the Phase 2 study of Akcea-Apociii-LRx in the treatment of patients with hypertriglyceridemia who are at risk for or have established cardiovascular disease.

Ultimately, the study met the primary endpoint of significant triglyceride lowering and multiple secondary endpoints with a favorable safety and tolerability profile.

The study demonstrated statistically significant dose-dependent reductions in fasting triglycerides compared to placebo at all dose levels and significant reductions in multiple additional risk factors.

Also, treatment-emergent adverse events were comparable between active and placebo groups. Approximately 85% of patients completed treatment and the rate of treatment discontinuation was comparable between the active and placebo groups.

Some quick background: Hypertriglyceridemia denotes high blood levels of triglycerides. Elevated levels of triglycerides are associated with atherosclerosis and predispose patients to cardiovascular disease. Note that this affects over 3 million Americans every year.

Louis O’Dea, M.D., chief medical officer at Akcea, commented:

We are very encouraged that patients achieved a significant reduction in apoC-III and triglyceride levels in this study, with the majority reaching triglyceride levels below the recognized threshold for cardiovascular risk. These data further validate the consistent efficacy and safety profile that we have seen across Ionis’ LICA technology platform. Based on the positive results from this study, we plan to rapidly pursue development of Akcea- Apocii-LRx for familial chylomicronemia syndrome or FCS. Because we were able to achieve substantial triglyceride lowering with this investigational medicine, we are also considering developing it for other rare and common diseases associated with elevated triglycerides. We are very grateful to the patients, families and physicians who participated in this study and are excited about the potential to bring this much needed therapy to patients.

Shares of Akcea were last seen up 9% at $18.89, in a 52-week range of $13.90 to $35.82. The consensus price target is $31.00.

Ionis was trading up almost 4%, at $62.33 in a 52-week range of $53.34 to $86.58. The consensus price target is $68.00.


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