Only a month ago, Opthea was making preparations for the potential launch of a new eye disease drug onto the market. Now, the candidate has failed to match Eylea in a phase 3 trial, leaving the Australian biotech to consider its own future.
The drug in question is sozinibercept, an inhibitor of vascular endothelial growth factors (VEGFs) C and D. Atea had been evaluating a 2-mg dose of sozinibercept every four or eight weeks in combination with Regeneron’s blockbuster VEGF inhibitor Eylea in patients with wet age-related macular degeneration (AMD) in a late-stage study.
Patients receiving the combination therapy every four weeks or eight weeks saw a mean change in best corrected visual acuity at Week 52 of 13.5 and 12.8 letters, respectively, while patients who received Eylea only saw a slightly higher improvement of 13.7 letters.
As well as failing on this primary endpoint, there was “no numerical difference observed in the key secondary endpoints,” Opthea noted.
The biotech has wasted no time in reaching out to investors to see whether it can avoid debts and additional charges being called in. It will also make a decision on the future of the Coast trial and the phase 3 Shore trial—which is assessing sozinibercept in combination with Novartis’ Lucentis and is due to read out this year.
“At this stage, no decision has yet been taken with respect to either trial, including whether to discontinue activities for the COAST trial or accelerate and unmask the ShORe trial,” Opthea said in the March 24 release. “Discussions continue with the DFA investors to determine the most appropriate course of action.”
With $113.8 million in the bank as of the end of February, “there remains material uncertainty as to Opthea's ability to continue as a going concern,” the company added. The biotech has halted trading of its stock on both the Australian and Nasdaq exchanges while it works out a plan.
The phase 3 fail marks an abrupt change in fortunes for Opthea, which had been confident enough in sozinibercept’s success to start gearing up its manufacturing activities in preparation for a potential commercial launch of the drug in wet AMD.
The company joined the Nasdaq via a $128 million IPO in 2020. Investment enthusiasm for sozinibercept was driven by a phase 2 trial in which the drug bested Lucentis in wet AMD as well as “observed evidence of improved clinical outcomes” in a phase 1b/2a study in combination with Eylea in patients with treatment-refractory diabetic macular edema.