Ophthotech completes clean sweep of phase 3 trial flops

Ophthotech has posted negative results from a third phase 3 trial of its wet age-related macular degeneration (AMD) candidate. The failure of anti-PDGF drug Fovista to improve outcomes when added to Eylea or Avastin completes a clean sweep of failures for the Novartis-partnered program.

For the latest trial, investigators enrolled about 640 patients and randomized them either with Fovista or placebo on top of either Regeneron’s Eylea or Roche’s Avastin. The aim was to see whether the anti-PDGF, anti-VEGF combination improved visual acuity over 12 months. It didn’t.

The results from the two arms were nearly identical. Participants who received Fovista gained 9.42 letters of vision on average on the standard eye test. Without the addition of Fovista, Eylea and Avastin triggered an average improvement of 9.04 letters.

On one level, the setback is a positive for Novartis. The first two phase 3 failures showed there is no value in adding Fovista to Novartis’ Lucentis, eliminating the possibility of the Big Pharma using the combination to prop up sales of its waning blockbuster. If Fovista had then improved outcomes when added to Eylea or Avastin, Novartis would have paid hundreds of millions to Ophthotech to help bankroll a boost for its rivals, rubbing salt into the wounds while denting Lucentis’ prospects.

The failure also frees Ophthotech from a tricky decision. Collectively, the failure of the Lucentis trials and a Regeneron study combining Eylea and rinucumab suggest the anti-PDGF, anti-VEGF idea is a busted flush. But success in the latest phase 3 could have driven Ophthotech to pursue it further nonetheless.

As it is, Ophthotech can stick to the course it set following the earlier failures. Reading the writing on the wall, Ophthotech has already slimmed down, refocused and set about identifying assets to rebuild its battered business around.

"This outcome does not affect our strategy as the company moves forward with multiple ongoing or planned clinical programs in orphan retinal diseases coupled with multiple ongoing or planned clinical trials in back of the eye indications," Ophthotech CEO Glenn Sblendorio said in the statement.

With Fovista falling by the wayside, the focus is now on C5 complement inhibitor Zimura. Ophthotech is using the drug to spearhead its move into rare diseases while also rolling the dice on more AMD trials.