Summary of HORIZON Interim Data

Melflufen continues to demonstrate promising activity in patients with RRMM, many of whom also have EMD. According to these interim study results, patients demonstrated an ORR of 28% and a clinical benefit rate (CBR) of 40%.

Median PFS is 4.0 months in the ongoing study and duration of response (DOR) is 4.4 months. The majority of patients evaluated (86%) achieved SD or better.

Treatment was generally well tolerated with manageable toxicity, nonhematologic AEs were infrequent and the rate of discontinuation due to AEs was low. Treatment-related SAEs occurred in 20% of patients and were most commonly febrile neutropenia (5%) and thrombocytopenia (2%).

The median age of patients in the clinical study was 64 years. 62% of patients in the study had high-risk cytogenetics, 29% of patients were ISS stage III and 60% of the patients had EMD. The median number of prior lines of therapy was five and the median time since initial diagnosis was 6.2 years.