Multiple Myeloma Coverage from Every Angle

Melflufen-Based Combination Therapy for Resistant Myeloma

By: Kelly M. Hennessey, PhD
Posted: Friday, October 18, 2019

Melflufen (a peptidase-enhanced cytotoxic agent that delivers melphalan to cancer cells) plus dexamethasone appear to be active in patients with relapsed or refractory multiple myeloma with and without extramedullary disease, according to the results of the HORIZON phase II, single-arm, open label, multicenter study. The interim results were presented by Paul G. Richardson, MD, of Dana-Farber Cancer Institute, Harvard Medical School, Boston, and colleagues at the 2019 International Myeloma Workshop (IMW) in Boston (Abstract OAB-86).

Included in the HORIZON trial were 136 patients, 44 of whom had a confirmed diagnosis of extramedullary disease at baseline. The overall response rate for patients with extramedullary disease was 23%, compared with 27% in patients without extramedullary disease, with a median duration of response of 3.4 and 4.4 months, respectively. Of the 44 patients with extramedullary disease who responded to treatment, the median overall survival was 18.5 months, compared with 5.1 months for those who did not respond to treatment. In patients without extramedullary disease who responded to melflufen plus dexamethasone, the median overall survival was 17.2 months, compared with 8.5 months for patients who did not respond.

“We are experiencing a significant increase in the number of relapsed and refractory myeloma patients with extramedullary disease and without effective treatment options,” explained Dr. Richardson in a press release. “The observation in the HORIZON trial, that melflufen shows activity in these patients and may impact favorably on survival outcome, is very encouraging. In my view, given the relative lack of therapeutic alternatives for these patients, moving rapidly into melflufen-based combination strategies in patients with extramedullary disease, as well as exploring earlier lines of therapy should be the next priority.”

Disclosure: The study authors’ disclosure information can be found at

By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.