Ophthotech Refocuses After Trial Setback

Company will have $175 million for new initiatives.

Ophthotech Corporation is down but not out after the stunning failure of two major phase 3 clinical trials for the combination of Lucentis and its proprietary anti-PDGF drug Fovista in wet AMD. After accounting for the $100 million to $115 million in expenses associated with discontinued studies and drastic headcount reductions, the company said it will have at least $175 million to employ in new initiatives.

Speaking on a February 28 conference call, company Chairman David Guyer, MD, said Ophthotech would like to now leverage its deep experience in ophthalmology to license the rights to promising ophthalmic drugs, preferably but not limited to investigational back-of-the-eye therapies that have demonstrated encouraging results in early-stage clinical trials.

“It is early in the process, but all options are on the table,” said Dr. Guyer. “I think a lot of companies would like to collaborate with us, given our depth of experience in ophthalmolgy.” He also noted that Ophthotech has hired Leerink Partners as a financial adviser to explore “all strategic options.”

Dr. Guyer said a third phase 3 clinical trial in wet AMD using a combination of Fovista with either Eylea or Avastin would continue to completion later this year.

He said given that the two Fovista/Lucentis trials demonstrated no benefit in any subgroup, he thought it “unlikely” that the Eylea/Avastin study will succeed, but that “we have made a commitment to complete the trial.” He also noted that the Regeneron anti-PDGF drug had failed in combination with Eylea, creating more evidence that anti-PDGF might not be the way to go. In the case of Ophthotech’s other drug, Zimura, for both wet AMD and geographic atrophy, he noted that early results from a competitor’s phase 3 trial would be a key to whether to continue Zimura’s phase 2 study in geographic atrophy. The competitor is almost certainly Genentech’s promising drug lampilizumab.

Noting that the Ophthotech and competitor’s drugs both have similar mechanisms of action targeting complement factors, Dr. Guyer said success of the competitor’s drug in phase 3 would be a positive sign for continuing to study Zimura.

“It’s really a timing issue,” he said. “The competitor’s data will come out first and we will have a chance to see how it might relate to Zimura.”

The surprising failure of the two major phase 3 trials has played havoc with Ophthotech’s stock price, which has fallen from $38 a share to a current price of about $4 a share. However, the company has been quick to refocus and turn to possible new initiatives in medical retina, where new concepts abound.