Swedish biotech company believes its treatment could be ‘canary in coal mine’ despite setback.
Advisory Committee on Oncology Medicines (ODAC) stated that they did not consider the treatment of patients with relapsed or refractory multiple myeloma (RRMM) to have a favorable benefit-risk profile.
Oncopeptides AB is testing Pepaxto in the 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. Patients also had to be refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD39-directed monoclonal antibody.
ODAC is part of the US Food and Drug Administration (FDA) and completed the discussion.
The majority of the panel felt that OCEAN did not demonstrate a favorable benefit-risk profile in the current indicated patient population.
In light of the results of the OCEAN trial, the FDA asked the ODAC to vote on the following question: “Given the potential harm to overall survival (OS), the inability to demonstrate a progression-free survival (PFS) benefit, and the lack of an appropriate dose, whether the benefit-risk profile of melphalan flufenamide is favorable for the current patient group?’ By a vote of 14 to 2, ODAC answered the question in the negative.
The canary in the coal mine
Jakob Lindberg, CEO of Oncopeptides, said: “We continue to trust our science and data. The heart of the ODAC discussion focused on the highly heterogeneous survival results in OCEAN across patient groups and how to interpret the subgroup data in light of the ITT OS outcome.
“One day, I believe OCEAN will be recognized as the canary in the coal mine for immunomodulatory drugs, which we believe is the main cause of OS heterogeneity and the dissociation between OS and OS in OCEAN, and show that Pepaxto has the potential to be a significant option treatment for elderly patients with RRMM.”
Cancer plasma cells
FDA will not issue a final decision on the issues discussed until input from the advisory committee process has been considered and all reviews have been completed.
Multiple myeloma affects the plasma cells in the bone marrow. The condition can cause severe fatigue and can also affect the bones and other organs such as the kidneys.
In multiple myeloma, altered cancerous plasma cells accumulate in the bone marrow, taking up space for healthy blood cells. Instead of making antibodies that protect against invading microbes, cancer cells produce 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 the Swedish Innovation Agency to develop a preclinical proof-of-concept (PoC) of a novel synthetic small polypeptide for the treatment of multiple myeloma.
https://www.labiotech.eu/trends-news/knockback-for-drug-to-treat-patients-with-relapsed-or-refractory-multiple-myeloma/ A setback for a drug to treat patients with multiple myeloma