Billion-dollar gamble

Last December, Forbes published a feature article about Megan Baldwin’s work  to improve the antiangiogenic treatment of eye diseases. I hope she wins her billion-dollar gamble not only because the drug in question was invented here at the University of Helsinki. The drug is called OPT-302 or with its INN name  sozinibercept. The patent  lists Kari Alitalo and me as the inventors. The drug was initially designed (under the name VGX-300) for cancer treatment, but - perhaps luckily - never really took off as such because it inhibits not only VEGF-D but also VEGF-C, which is needed in the body’s immune response against the cancer. So far, nobody has been able to separate the prometastatic and the proimmunogenic propeties of VEGF-C.

However, VEGF-C and VEGF-D have been suspected to be responsible for neovascular eye diseases  (such as wet age-related macular degeneration, AMD) that fail to respond to anti-VEGF-A treatments (such as aflibercept or bevacizumab). Unsurprisingly, Sozinibercept is used in combination with anti-VEGF-A drugs. The results from clinical phase I and II trials have been very encouraging, but typically only the phase III trials answer the question of whether the drugs work well enough to justify asking the FDA and EMA for a market authorization. The first results for the OPT-302 phase III studies  are expected for Q2/2025, and the share price of Opthea  has started to incrementally recover since last summer, indicating that at least some investors think that this gamble could have a happy ending. Helsinki University sold the IP for this drug to Circadian in exchange for shares. That might or might not have been a good decision depending on the outcome of the phase III trials. I keep my fingers crossed as I am still waiting to receive my 15% inventor’s share from my university, which the Finnish law allocates as an incentive for innovation to inventors employed by universities.

UPDATE On the 24th of March 2025, we got to know the disappointing Phase III results for sozinibercept (OPT-302): Both pivotal trials (COAST and ShORe) failed to meet their primary visual-acuity endpoints. The company has since halted development of the program. The University of Helsinki lost millions of Euros as they were clinging to the Opthea stocks instead of hedging their bets by selling half of them before the phase III trials were announced. It’s never good to be too greedy. Investors and clinicians alike are now reassessing the future of combination approaches in age-related macular degeneration. For me, as the inventor of sozinibercept, the question remains whether focusing on VEGF-C/D was the correct secondary target. It is also unclear whether the aflibercept/sozinibercept combination could still improve anti-angiogenesic tumor therapy, which is a different situation compared to neovascular eye diseases. Many outlets have reported on the failure: https://www.google.com/search?q=sozinibercept+opt-302+phase+3&oq=sozinibercept+opt-302+phase+  .