Melbourne biopharmaceutical company Opthea has abandoned efforts to develop its therapy sozinibercept for neovascular age-related macular degeneration (nAMD), with a second pivotal trail now failing to meet its primary endpoint.
It comes after the ASX- and Nasdaq-listed firm revealed on 24 March 2025 that the Phase 3 COAST (Combination of OPT-302 with Aflibercept Study) trial was not able to show a mean change in best corrected visual acuity (BCVA) from baseline to week 52.
The “negative result” prompted the company to say that its solvency was now in question.
Opthea then brought forward the announcement of topline results from its second Phase 3 trial ShORe (Study of OPT-302 in combination with Ranibizumab) for nAMD.
On 31 March, it revealed this also failed to meet its primary endpoint.
Both COAST and ShORe were evaluating the efficacy and safety of 2 mg sozinibercept – a novel VEGF-C/D ‘trap’ fusion protein – in combination with aflibercept and ranibizumab, respectively, compared with standard-of-care monotherapy.
“We are disappointed that COAST and ShORe did not demonstrate the improvements in vision with sozinibercept combination therapy compared to standard-of-care that we had hoped for,” said Opthea CEO Mr Frederic Guerard.
“We are grateful to all patients, clinical investigators and their staff around the world who participated in the sozinibercept Phase 3 clinical program, and for their contributions in investigating new treatments for wet AMD.”
Opthea, along with its Development Funding Agreement (DFA) investors, have now decided to “discontinue the development of sozinibercept in wet AMD with immediate effect”.
Management were ‘confident of success’
The company’s future still remains uncertain, in news that came a a big surprise to the industry and investors.
As recently as December 2024, Guerard was quoted saying he was “fairly confident” the therapy would be on sale and in paying customers’ eyes by early 2027, but warned biotech can be tough business.
One of its biggest backers, Regal Partners, has now written off hundreds of millions of dollars it had invested in Opthea.
“The company’s management team were so confident of success they were able to attract some of the leading experts of the industry to join the company in the last 12 months,” co-founder Mr Phil King said in a letter to investors, according to the AFR.
He added that earlier trial results had been “highly encouraging, producing statistically significant results above the standard of care, reinforcing our confidence”.
Given these developments, Opthea said it remained possible that under the DFA it could be required to “pay a multiple of the amount funded by the DFA investors” that would have “a material adverse impact on the solvency of the company”.
“As previously disclosed, termination can be triggered by a range of events, including, among other things, Opthea’s insolvency, in which case Opthea will be obligated to pay a multiple of the amounts funded by the DFA investors,” a statement said.
Opthea is continuing “to explore possible options to deliver the best outcome for the company and its shareholders”.
The company estimates it will have unaudited cash and cash equivalents of US$100 million (AU$166 m) at the end of March 2025.
“In light of these matters, there remains material uncertainty as to Opthea’s ability to continue as a going concern. As noted above, discussions with the DFA Investors are ongoing and Opthea cannot be certain as to the outcome of those discussions or when that outcome may become known.”
Trading in Opthea shares will be suspended on the ASX until it’s in a position to provide the market more clarity on these issues and the impact of its financial position.
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