Antisense specialist Isis Pharmaceuticals shares leapt this week after the company reported positive data with its triglyceride-lowering drug candidate.
ISIS-APOCIII Rx achieved a spectacular reduction in levels of triglyceride – a marker for heart disease, diabetes, obesity and stroke – at the highest dose tested in a phase II study reported at the European Society of Cardiology (ESC) annual meeting in Amsterdam, prompting a 13 per cent rise in its share price over the last couple of days.
Top-line data from the small 28-patient study showed that single-agent therapy with ISIS-APOCIII Rx at a weekly dose of 300mg caused triglycerides to drop 75 per cent, while levels of another cardiovascular biomarker – apolipoprotein CIII (apoCIII) – fell 79 per cent after 13 weeks’ treatment, with no non-responders.
“The most important new observation from the new study … is that ISIS-APOCIII Rx is showing good efficacy as a single agent in patients with very high to severely high triglycerides,” said Isis’ head of development Richard Geary on a conference call to discuss the results.
The company estimates there are approximately a 100,000 patients in the US and Europe who have severely high triglycerides despite currently available triglyceride-lowering therapies.
“Standard therapies on the market today like fibrates are not able to lower triglyceride sufficiently in these patients to bring them to recommended triglyceride levels,” said Geary, adding that this leaves them at elevated risk of cardiovascular disease.
Isis is already planning a phase III trials programme on the strength of the latest data and earlier phase II studies which showed the drug was effective in patients with diabetes and high triglycerides and also when given in combination with fibrate drugs such as fenofibrate.
Advancing the programme to pivotal trials is a significant milestone for the company, as ISIS-APOCIII Rx is the first drug that it intends to develop without a larger commercial partner. Current estimates are that the pivotal trial programme would take about two years, setting up a filing for the drug in 2016 or 2017.
The data is positive news for Isis after the disappointment of having to discontinue a rheumatoid arthritis candidate – ISIS-CRP Rx – after disappointing phase II results last month.
A phase II trial in patients with high triglyceride levels caused by a very rare condition known as familial chylomicronaemia syndrome (FCS) is due to be presented later this month. FCS affects around 3,000 to 5,000 patients worldwide.
Isis’ lead programme at the moment is Kynamro (mipomersen), an antisense drug for elevated cholesterol that is partnered with Sanofi and was approved in the US in January but rejected a month earlier by the European Medicines Agency (EMA).
With Isis’ antisense pipeline starting to deliver the goods, analysts have suggested that the company could become a takeover target for a larger pharmaceutical company.