A New and Different Approach to Treat Triple Refractory Multiple Myeloma

The Swedish company Oncopeptides announced interesting news, a few days ago, for those of us who have failed prior treatment with immunomodulators and proteasome inhibitors and who have also become refractory to Pomalyst (pomalidomide) and/or Darzalex (daratumumab). The company has recently completed a 157-patient study with their proprietary innovation of the  alkylating drug Melfufen (melphalan flufenamide) administered in combination with dexamethasone. This study not only targeted a patient population with few, if any, treatment options left as well as patients with ‘high risk’ multiple myeloma.

The innovation that Oncopeptides has brought into our future treatment armamentarium is that they have discovered a way to combine the drug Melflufen with a peptide. There is no need here to go into the fine points of peptide chemistry. Just think in terms of ‘small versions of proteins’ and know that we encounter peptides in the daily use of a host of cosmetic products. The combination of Melflufen-peptide (named a ‘drug conjugate’), administered to a patient is rapidly taken up into the myeloma cells where it is split apart. The Melfufen delivers a concentration of cancer killing drug that is 50 times more potent than melphalan delivered in traditional ways and goes to work rapidly to cause irreversible DNA-damage to the cancerous myeloma cells, leading to cell death. I suggest you take a look at a short video provided on the Oncopeptides website that shows the whole process in action.

The results of this clinical study mentioned earlier can be summarized as follows:

• Overall response rate in triple class refractory patients of 26%.
• The drug is tolerated well with primarily hematological side effects.

The company has indicated that it intends to file a New Drug Application with the US FDA before mid-year 2020. FDA has previously announced that this drug will be granted Orphan Drug Status which provides for a somewhat expedited review and approval process. I would expect to see this treatment option for desperate patients to come to market in the first quarter of 2021, assuming that the company meets all the standards of the US review process.