Ionis Pharmaceuticals ($IONS) announced that the pivotal Phase 3 APPROACH study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronemia syndrome. The trial was being carried out by the company’s wholly owned subsidiary Akcea Therapeutics. The average incoming triglyceride level of patients in the study was 2,209 mg/dL. For the primary endpoint of the study, volanesorsen-treated patients (n=33) achieved a statistically significant (p<0.0001) mean reduction in triglycerides of 77% from baseline after 3 months of treatment, compared to a mean increase of 18% in placebo-treated patients (n=33).
The company stock failed to react positively to the news. However, it has progressed well in the recent past as it gained over 6 percent this year so far. Ionis stock is up 28 percent in the past 12 months. It is currently trading close to its 52 weeks high of $57.
Ionis Pharmaceuticals Inc. also recently reported its financial results. The company said that
it outperformed its financial guidance by ending 2016 with pro forma operating income of $25.8 million and $665.2 million in cash, cash equivalents and short-term investments. Its GAAP loss from operations stood at $46.3 million. The company also reported that it announced positive clinical data from 11 studies with six drugs during the year.