FDA Approves Oncopeptides´ PEPAXTO® (melphalan flufenamide) for Patients with Triple-Class Refractory Multiple Myeloma

Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO),  a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved PEPAXTO® (melphalan flufenamide), known during clinical development as melflufen, in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. This indication has been granted under accelerated approval based upon the HORIZON trial. PEPAXTO is the first anticancer peptide-drug conjugate approved in multiple myeloma.

“While the treatment landscape for multiple myeloma has dramatically improved in recent years, once patients become resistant to existing classes of therapy they can face a very guarded prognosis,” said Paul G. Richardson, MD, Clinical Program Leader and Director of Clinical Research at the Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute. “Research has shown melphalan flufenamide to be a novel and innovative therapeutic option, which is active in refractory disease and has manageable toxicity, with the convenience of being administered by infusion once a month. Based on our findings, melphalan flufenamide is an important addition to the treatment armamentarium, with the potential to meaningfully improve outcomes in an area of important unmet medical need.”

The HORIZON study, evaluating intravenous PEPAXTO in combination with dexamethasone, included heavily pre-treated patients with a poor prognosis. This multi-center single arm study evaluated 157 patients with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The Overall Response Rate for the patients within this group of patients with refractory multiple myeloma was 23.7 percent and the Median Duration of Response was 4.2 months. Of the subset of 97 patients, 41% had EMD (n=40), an aggressive and resistant disease associated with poor prognosis.

“We are proud to bring forward the first anticancer peptide-drug conjugate approved by the FDA for multiple myeloma,” said Jakob Lindberg, Chief Scientific Officer at Oncopeptides.  “PEPAXTO uses innovative technology that links a peptide carrier to a cytotoxic agent. The conjugated agent is a highly lipophilic compound, which allows it to be rapidly distributed into cells. The compound then leverages amino peptidases that are overexpressed in Multiple Myeloma cells, causing the release of the cytotoxic payload.”

Marty Duvall, Chief Executive Officer at Oncopeptides added, “The accelerated U.S. approval of PEPAXTO is a key step forward in fulfilling Oncopeptides’ core mission, to bring hope to patients in their battle against difficult-to-treat hematological diseases. Moving ahead, our focus is to further advance PEPAXTO.  We look forward to receiving top line data from the phase 3 OCEAN study in the second quarter.  This comparative study is designed to support a future supplementary New Drug Application to expand the label.”

PEPAXTO was approved under accelerated approval, which allows for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Oncopeptides expects PEPAXTO to become commercially available in the U.S. within approximately two weeks.

For more information, please see the full Prescribing Information and press release:  U.S. Press Release

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