Melflufen

Overview

Melflufen is a peptide-conjugated alkylator (a new type of cancer therapy) that rapidly delivers a toxic drug payload into myeloma cells, resulting in myeloma cell death.

SparkCures ID 264
Generic Name Melflufen
Treatment Classifications

Clinical Trials

All Clinical Trials

View all active clinical trials around the US.

Early Relapse Multiple Myeloma

The following is a listing of clinical trials for patients with multiple myeloma who have received one to two prior lines of therapy.

Late Relapse Multiple Myeloma

The following is a listing of clinical trials for patients with multiple myeloma who have received three or more prior lines of therapy.

Smoldering Myeloma
Monoclonal Gammopathy of Undetermined Significance (MGUS)

Published Results

Data From Oncopeptides Melflufen Clinical Development Program Presented at SOHO 2019 Annual Meeting

September 11, 2019

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.

Oncopeptides looks forward to updates on myeloma drug at European hematology conference

June 11, 2019

According to an abstract of data from ANCHOR, of the 10 patients in the arm receiving melflufen with Darzalex there was a 100 percent overall response rate (ORR) at the 30mg dose cohort and a 50 percent response rate at the 40mg cohort. For the five receiving melflufen and bortezomib, the response rate was 100 percent in the 30mg cohort and zero percent at the 40mg dose.

Meanwhile, the company is planning to pursue accelerated FDA approval based on the HORIZON study, with a regulatory filing anticipated in early 2020. According to the EHA abstract for that study, the median PFS for the 95 patients treated as of the Feb. 6 cutoff was four months, with a 10 month median overall survival (OS) and an ORR of 30 percent. Data presented at ASH from a previous cutoff date showed a 33 percent ORR.

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