Developing a targeted treatment for multiple myeloma

Oncopeptides lead compound, named melflufen, is a peptidase potentiated therapy within oncology. Melflufen is a very potent anti-angiogenetic compound that triggers rapid, robust, and irreversible DNA damage and exerts it cytotoxicity through alkylation of DNA.

Clinical Trials

Doctor talking smallerMelflufen is currently undergoing Phase 2 efficacy studies in patients with relapsed and relapsed-refractory multiple myeloma. The peptidase targeting causes melflufen and its metabolites to accumulate in the diseased cells resulting in targeted delivery of the active moieties to the cancer cells, and thereby better treatment of the disease.

In latest published clinical results in patients with late stage relapsed and relapsed-refractory (RRMM), melflufen plus dexamethasone showed an overall response rate (partial response or better) of 40% and clinical benefit rate (minimal response or better) of 63%. At the date of analysis, the median progression-free survival (PFS-50%) was 4.3 months and the median duration of response was equal to or greater than 7.7 months, these results are from efficacy evaluable patients according to the clinical trial protocol. It is worth noting that 43% were progression-free at 6 months and 12.5% at 12 months, with a PFS-25% of 9.7 months.

Similar results were seen across patient populations regardless of refractory status.

Based on the positive data and discussions with FDA a pivotal phase 3 superiority study is being planned in late-stage multiple myeloma. The multiple myeloma program also includes two additional phase 2 studies and one additional phase 1/2 study.

Oncopeptides Received Orphan Drug Designation for its lead candidate Melflufen in the treatment of multiple myeloma from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) in September 2014.

Phase 2 clinical trials


Dana-Farber Cancer Institute, MA, USA is the lead investigator of a Phase 2 multi-centre clinical trial in multiple myeloma in the USA. This is being conducted in both the US and Western Europe.

If you are interested in finding out more about this trial please email: