While most of the health care industry was focused on megadeals, a few biotechs released some impressive clinical results last week. Isis Pharmaceuticals (IONS -1.52%) has posted two mid-stage wins in as many weeks. Hardly anybody noticed as Gilead Sciences' (GILD -0.56%) drug for respiratory syncytial virus pushed through a midstage trial. The biggest win, though goes to Intermune (ITMN.DL) which got its lead program back on track with US regulators.
Another first-in-class hopeful
Isis posted some great topline results from a novel blood thinner. Patients receiving its Factor XI inhibitor experienced a seven-fold decrease of life threatening thrombolic events compared to patients receiving Sanofi's lovenox.
Isis' unique blood thinner prevents the formation of clots -- in this case following knee surgery -- that can break loose and travel through the bloodstream. Thrombolic events that occur when one of these clots becomes trapped within a vital blood vessel can be fatal.
Blood thinners, like warfarin, prevent the formation of clots, but can also lead to equally threatening bleeding events. Creating safer, more convenient alternatives is a huge opportunity. Popularity is growing among new anticoagulants that stay in the sweet spot with longer dosage intervals. Johnson & Johnson has done well marketing Bayer's factor Xa inhibitor Xarelto in the US. Sales of the anticoagulant reached $319 million in the first quarter, more than doubling from the same period a year ago.
Isis has found that inhibiting clotting factor XI is more effective with less incidence of bleeding than Xa inhibitors, but its finding is limited to animal models. If Isis can eventually prove it to be the case in human subjects, it might have a very big winner on its hands.
Ahead of the pack... again
Last Tuesday, Gilead hit the mark in a mid-stage trial with S-5806. The antiviral fusion inhibitor significantly lowered viral load -- 99.9% decline -- among adult patients infected with respiratory syncytial virus (RSV).
Shares of GIlead hardly budged as the market appears focused on the company's record setting Sovaldi launch, but this could be another blockbuster in Gilead's antiviral lineup. RSV is the most common cause of lower respiratory tract infections among young children in the United States and worldwide. Currently there are no antiviral treatments, making Gilead the potential first mover in a field with millions of potential patients.
The relatively common virus can make healthy adults uncomfortable and contagious for about a week, but patients with weakened immune systems often require hospitalization. The virus leads to roughly 300,000 hospitalizations each year. With 1.5 million outpatient visits among children under five, an effective RSV antiviral could make a significant impact on public health spending. Of course, this was a mid-stage trial, so more data will be
Re-submission complete
Last week's biggest win goes to Intermune and its idiopathic pulmonary fibrosis (IPF) program. Late-stage candidate pirfenidone significantly reduced lung function decline. A 10% reduction in forced vital capacity is strongly associated with higher mortality risk. After a year just 16.5% of patients receiving pirfenidone experienced a 10% reduction versus 31.8 % in the placebo group. Pirfenidone patients also showed significantly improved six-minute walk distance, and progression-free survival.
Shares rose nearly 20% following the announcement, despite a relative lack of surprise. Pirfenidone won marketing approval in the EU years ago, but the FDA requested more data. Intermune recorded $70.3 million in pirfenidone sales last year, and $30.3 million in the first quarter of this year. While the EU region is performing well, exposure to the US market could more than double the company's top line. As a chronic condition with no available disease slowing therapies, the US IPF market represents an important opportunity for Intermune.
Unfortunately, the company may have to share the US IPF market with Boehringer Ingelheim. Results published earlier this month suggest that its drug nintedanib significantly slows progression of IPF. Both therapies could enter the US market next year.
Wasting no time, Intermune has already resubmitted its pirfenidone application to the FDA. The agency should issue a response by early August. If the resubmitted application is considered complete, an approval decision -- and potential launch -- could occur as soon as the first quarter of 2015.
