A Swedish biotech company says it believes its treatment could be the ‘canary in the coalmine’ despite a knockback.
The Oncologic Drugs Advisory Committee (ODAC) has said it does not consider the treatment for patients with relapsed or refractory multiple myeloma (RRMM) favorable in a benefit-risk profile.
Oncopeptides AB has been trialing Pepaxto in the OCEAN trial, which is indicated in combination with dexamethasone for the treatment of adults with RRMM who have received at least four prior lines of therapy. The patients’ condition had also to be refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD39-directed monoclonal antibody.
Pepaxto benefit-risk profile
ODAC is part of the U.S. Food and Drug Administration (FDA) and finalized the discussion.
A majority of the panel considered that OCEAN did not confirm a favorable benefit-risk profile in the current indicated patient population.
In the light of the outcome of the OCEAN trial, the FDA has asked the ODAC panel to vote on the following question: “Given the potential detriment in overall survival (OS), failure to demonstrate a progression-free survival (PFS) benefit, and lack of an appropriate dose, is the benefit risk profile of melphalan flufenamide favorable for the currently indicated patient population?” In a 14 to 2 vote, the ODAC answered no to the question.
Canary in the coal mine
Jakob Lindberg, CEO of Oncopeptides, said: “We still have confidence in our science and data. The heart of the ODAC discussion was focused on the highly heterogenous survival result in OCEAN across patient groups and how to interpret the subgroup data considering the ITT OS result.
“One day I believe that OCEAN may be recognized as the canary in the coal mine for immunomodulatory drugs that in our view is the main cause behind the OS heterogeneity and dissociation between PFS and OS in OCEAN and show that Pepaxto has the potential to become a meaningful treatment option for elderly patients with RRMM.”
Cancerous plasma cells
The FDA will not issue a final determination on the issues discussed until input from the advisory committee process has been considered and all reviews have been finalized.
Multiple myeloma affects plasma cells in the bone marrow. The condition can cause extreme tiredness and can also affect the bones and other organs, such as the kidneys.
In multiple myeloma, the altered, cancerous plasma cells build up in the bone marrow, taking away space for healthy blood cells. Instead of making antibodies that protect from invading germs, the cancerous cells make an abnormal protein called monoclonal immunoglobulin, or monoclonal protein (M-protein).
Earlier this month, Oncopeptides AB received a SEK 5 million ($465,000) research grant from Sweden’s Innovation Agency to develop a preclinical proof of concept (PoC) for a novel synthetic small polypeptide for the treatment of multiple myeloma.