Isis Pharmaceuticals (ISIS) has long maintained that it intends to develop its triglyceride-lowering agent ISIS-APOCIIIRx without outside assistance, and a recent $170m equity offering will help it get part of the way there. The drug's startlingly positive phase II results revealed on Saturday ought to prompt generous partnership offers, though, and everyone has their price.
These presumed overtures may not be enough to induce the company to depart from its stated plans. But every clinical success further validates the antisense platform on which Isis has built its company, and with a pipeline featuring 18 candidates in phase II or III, Isis itself is starting to look like a takeout target.
Though highly positive, the data, presented at this week's meeting of the European Society of Cardiology in Amsterdam, are early and the trial size small. Top-line results of the monotherapy part of the ongoing 28-patient study showed that the highest dose of the drug, 300mg weekly for 13 weeks, caused a 79% drop in levels of ApoC-III and a 75% fall in triglyceride levels.
Taken alone these results would be encouraging but not conclusive. Added to earlier phase II results, though, a picture of an effective drug starts to take shape. The patients in this study had very high to severely high triglyceride levels, showing the drug to work well in a new population following its success in patients with type 2 diabetes and high triglycerides (ADA – Knockdown result boosts Isis and antisense alike, June 25, 2013).
Isis is now keen to push into phase III, saying that it will discuss plans with regulators and start a trial in patients with severely high triglycerides next year. The company has shown no reluctance to partner its other late-stage drugs; its flagship, Kynamro, was licensed to Sanofi (SNY) in phase III. But management has shown no signs of deviating from its intention to go it alone, despite the magnitude of the undertaking.
If big pharma is really keen to get hold of ISIS-APOCIIIRx, a more drastic move may be necessary. Isis's market cap of $3bn, while not chickenfeed exactly, would present no major problems if Sanofi or one of Isis's other partners – GlaxoSmithKline (GSK) or Biogen Idec (BIIB), for example – wished to acquire a company whose platform is looking increasingly promising.