Melflufen induces response in triple-class refractory multiple myeloma
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Approximately one-quarter of patients with triple-class refractory multiple myeloma responded to treatment with melflufen, according to final topline results from the pivotal phase 2 HORIZON study released by the agent’s manufacturer.
The data will form the foundation for a new drug application expected to be filed with the FDA by the end of June.
Melflufen (Oncopeptides) is a peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. The agent has exhibited cytotoxic activity against myeloma cell lines resistant to other treatments.
The HORIZON study included 157 patients with multiple myeloma who failed immunomodulatory drugs and proteasome inhibitors, and they were refractory to pomalidomide (Pomalyst, Celgene) and/or daratumumab (Darzalex; Genmab, Janssen). The study included subgroups of patients who were triple-class refractory, had extramedullary disease and/or had cytogenetic high-risk features.
Results per independent review committee showed a 30% overall response rate in the intention-to-treat population, a 26% ORR among triple-class refractory patients (n = 119), and a 27% ORR among patients with extramedullary disease (n = 55).
Melflufen exhibited a safety profile consistent with prior reports from the HORIZON study. Most adverse events were hematologic in nature; incidence of nonhematologic adverse events was low, according to the press release.
Full results of the study will be shared in a peer-reviewed publication.
“These data confirm that melflufen has a good efficacy and safety profile [for patients with triple-class refractory myeloma], a fast-growing patient population with significant unmet medical need and lack of ... approved treatments,” Jakob Lindberg, CEO of Oncopeptides, said in the release. “We firmly believe that melflufen has the potential to become an important treatment option for patients with relapsed refractory multiple myeloma.”