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Ionis Pharma Lipid Disorder Candidate Positive in Phase III

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, Inc.IONS , recently announced positive results from a pivotal phase III study evaluating its pipeline candidate volanesorsen for treatment of patients with familial chylomicronemia syndrome (FCS). The aforesaid study is referred to as APPROACH.

Ionis Pharma's shares have significantly underperformed the Zacks classified Medical-Drugs industry so far this year. Its shares lost 2.8%, while the industry registered an increase of 7%.

APPROACH is a randomized, double-blind, placebo-controlled, 52-week phase III study. The study was conducted in 66 patients with FCS. The study met its primary end points and demonstrated 77% reduction in triglycerides of volanesorsen-treated patients (n=33) from baseline after three months of treatment compared to a mean increase of 18% in placebo-treated patients (n=33). The treatment effect was found to be sustainable over the 52-week treatment period.

The company plans to file for marketing authorization for FCS in the U.S., Europe and Canada in 2017.

Apart from the APPROACH study, Akcea is conducting two phase III studies of volanesorsen - BROADEN and COMPASS. The study in patients with familial partial lipodystrophy (FPL), referred to as BROADEN, is currently underway and data from this study is expected in 2019. The company has also conducted the COMPASS study in patients with triglycerides above 500 mg/dL to expand the exposure database for volanesorsen to support global regulatory filings. In Dec 2016, Akcea announced that the COMPASS study has met its primary endpoint.

We remind the investors that volanesorsen has been granted orphan drug status in both the U.S. and the EU for the treatment of FCS. Furthermore, volanesorsen has been granted orphan drug status in the EU for the treatment of FPL. The company is also in the process of applying for this status for this indication in the U.S. Akcea plans to globally commercialize volanesorsen for both FCS and FPL, if approved.

Per the company's press release, FCS is a rare disease affecting approximately 3,000-5,000 patients worldwide. Hence, there is a strong unmet need for drugs for the treatment of this disease.

We note that presently Ionis Pharma's lipid-lowering drug Kynamro is approved in the U.S. for cholesterol management in patients suffering from homozygous familial hypercholesterolemia. In May 2016, Ionis Pharma entered into an agreement with Kastle Therapeutics under which the later acquired the global rights to develop and commercialize Kynamro. The company previously licensed Kynamro to Genzyme, a Sanofi SNY company.

We expect investor focus to remain on pipeline updates from the company.

Ionis Pharmaceuticals, Inc. Price

Ionis Pharmaceuticals, Inc. Price | Ionis Pharmaceuticals, Inc. Quote

Zacks Rank & Key Picks

Ionis Pharma currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector include Celgene Corporation CELG and Sunesis Pharmaceuticals, Inc. SNSS . Each of these stocks carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here .

Celgene's earnings per share estimates increased from $6.52 to $6.60 for 2017 and from $8.15 to $8.16 for 2018 over the last 60 days. The company posted positive earnings surprises in three of the four trailing quarters with an average beat of 5.08%.

Sunesis' loss per share estimates narrowed from $2.57 to $2.44 for 2016 and from $2.16 to $1.97 for 2017 over the last 60 days. The company posted positive earnings surprises in three of the four trailing quarters with an average beat of 0.54%.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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