Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, today announces that three abstracts with data on melflufen (INN melphalan flufenamide) in relapsed refractory multiple myeloma, RRMM, have been accepted by the 2021 American Society of Clinical Oncology, ASCO, and have now been published online. The clinical data presentations include updates on Oncopeptides' ANCHOR and LIGHTHOUSE studies as well as a pooled analysis of the O-12-M1 and HORIZON studies in patients who have been exposed to or become refractory to prior alkylators.

"The phase 1/2 ANCHOR study of melflufen plus dexamethasone in combination with either daratumumab or bortezomib shows encouraging clinical activity in heavily pre-treated patients with relapsed refractory multiple myeloma, and the optimal dose of melflufen has now been established for both regimens," says Klaas Bakker, MD, PhD, Executive Vice President and Chief Medical Officer, Oncopeptides AB. "We will now complete patient enrolment in the bortezomib arm, while the daratumumab arm is already fully accrued, as presented at the American Hematology Association meeting in December 2020, where updated safety and efficacy data from ANCHOR were presented. The data presented here at ASCO data clearly support further development of melflufen in triplet regimens and reinforce the rational for the ongoing phase 3 LIGHTHOUSE study, comparing melflufen plus dexamethasone in combination with subcutaneous daratumumab with daratumumab alone."

Below is a brief description of the abstracts that have been accepted by the ASCO. 

1. ANCHOR (OP-104): MELFLUFEN PLUS DEXAMETHASONE AND BORTEZOMIB IN RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS

The ANCHOR study determined that the optimal dose of melflufen is 30 mg plus dexamethasone and bortezomib and the results showed clinical activity in heavily pretreated RRMM patients. Recruitment for this study is ongoing and updated data including efficacy and safety will be presented at ASCO.

2.  LIGHTHOUSE (OP-108): A PHASE 3 STUDY OF MELFLUFEN IN COMBINATION WITH DEXAMETHASONE AND DARATUMUMAB IN RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS

The phase 3 LIGHTHOUSE study is a randomized, controlled, open-label study of melflufen plus dexamethasone in combination with daratumumab vs daratumumab alone in patients with RRMM previously treated with an immunomodulatory agent and a proteasome inhibitor, similar to the indication for daratumumab monotherapy. The primary objective is superiority of PFS. Key secondary endpoints include ORR (≥ PR), DOR, and safety. Patient recruitment is ongoing with a planned enrolment of 240 patients.

3. A POOLED ANALYSIS OF THE O-12-M1 AND HORIZON STUDIES: MELFLUFEN PLUS DEXAMETHASONE IN RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS (RRMM) WHO ARE EXPOSED OR REFRACTORY TO PRIOR ALKYLATORS

This pooled analysis of the O-12-M1 and HORIZON studies showed that melflufen in combination with dexamethasone showed meaningful efficacy and demonstrated a clinically manageable safety profile in patients with RRMM who had been exposed or become refractory to prior alkylators.

PEPAXTO? (melphalan flufenamide, also known as melflufen), in combination with dexamethasone, was granted accelerated approval by the FDA on February 26, 2021, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.