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Melflufen for triple-class refractory multiple myeloma | Final topline results from the phase II HORIZON study released

Apr 28, 2020

The final topline results from the phase II HORIZON ( NCT02963493) study have recently been released. The trial investigated the efficacy and safety of melphalan flufenamide (melflufen) plus dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma (RRMM). 1 The results demonstrate favorable efficacy and safety profiles for melflufen in patients with RRMM, including those with triple-class refractory- and extramedullary disease ( Table 1).The data from this study are set to form the foundations of a new drug application (NDA) for accelerated approval to the U.S. Food & Drug Administration (FDA). 1

Table 1. Primary endpoint results of the HORIZON study

EMD, extramedullary disease; IRC, independent review committee; ITT, intention to treat; ORR, overall response rate

*Two unconfirmed responders on January 14 have later been confirmed.

Primary endpoint, %

Investigator assessed (January 14)

IRC (January 14)

Including unconfirmed responses at time of data cut-off *                                                 

ORR in the ITT population (n = 157)



31 (investigator and IRC)

ORR in triple-class refractory patients (n = 119)




ORR in patients with EMD (n = 55)




  A previous analysis of the HORIZON study was presented by Paul Richardson at the 24 thCongress of the European Hematology Association (EHA). Read the MM Hub coverage here .

Expert Opinion

  1. AB O. Oncopeptides Announces 26% overall response rate of melflufen in triple-class refractory multiple myeloma patients from the pivotal HORIZON study. PRNewswire. Published Mar 26, 2020. Accessed Apr 27, 2020.