Data Show Melflufen Is Noninferior to Pomalidomide for Relapsed/Refractory Multiple Myeloma

Recently announced phase 3 data show similar progression-free survival results between melflufen and pomalidomide, the most used medicine for patients with relapsed or refractory multiple myeloma.

Results from the phase 3 OCEAN study (NCT03151811) found that melphalan flufenamide (melflufen; Pepaxto) was noninferior to pomalidomide (Pomalyst) when treating patients with relapsed or refractory multiple myeloma, according to a press release from Oncopeptides AB, the company responsible for the drug.

Pomalidomide is currently the most common medicine used to treat patients with relapsed or refractory multiple myeloma, with these results suggesting that melfufen may be a significant therapeutic option for patients who may need alternative treatment options moving forward.

“Following the accelerated approval of Pepaxto in the U.S. earlier this year, the positive topline results from the OCEAN study marks another major milestone for Oncopeptides. It is very exciting news for patients and indicates that melflufen has the potential to become part of the standard of care in relapsed refractory multiple myeloma,” Marty J. Duvall, Chief Executive Officer at Oncopeptides AB, said in a press release. “By demonstrating that melflufen is at least as efficacious as pomalidomide, we pave the way for a potential use of melflufen in earlier lines of therapy in a substantially larger patient population.”

The study enrolled 495 patients from over 100 hospitals across 21 countries to evaluate the safety and efficacy of melfufen versus pomalidomide. The primary end point was progression-free survival (PFS) as assessed by independent review committee.

The PFS assessed by independent review committee showed noninferiority of melflufen compared with pomalidomide (HR, 0.817, 95% CI, 0.659-1.012; P = .064). For investigator assessed PFS, the hazard ratio in favor of melflufen was 0.790 (95% CI, 0.639-0.976).

The median PFS when assessed by both independent review committee and investigators was 42% higher in the melflufen arm compared with the pomalidomide arm. The overall response rates observed were 32.1% and 26.5% for the melflufen and pomalidomide arms, respectively.

“The efficacy and safety data from the OCEAN study are very encouraging, and the results will be of importance in physicians’ treatment decisions for patients with relapsed and refractory multiple myeloma,” Pieter Sonneveld, MD, PhD, Professor of Hematology at the Erasmus University of Rotterdam, and Principal Investigator of the OCEAN study, said in a press release.“Melflufen has a unique mode of action, which in addition with its tolerability profile, makes the drug an attractive treatment option for many patients.”

The safety profile for melflufen was consistent with previously reported safety data and across age subgroups. For both the melflufen and pomalidomide arms, discontinuation rates due to adverse effects were similar.

In February of 2021, melflufen in combination with dexamethasone to treat adult patients with relapsed or refractory multiple myeloma following 4 or more prior lines of therapy was granted accelerated approval by the FDA based on phase 2 data.

Looking ahead, the global biotech company anticipates submitting a supplementary New Drug Application for melflufen later this year, with plans to present complete data from the OCEAN trial at an upcoming scientific conference.


Oncopeptides AB: Phase 3 OCEAN study demonstrates that melflufen is at least as efficacious as pomalidomide, the most used medicine in relapsed refractory multiple myeloma. News release. Oncopeptides AB. May 25, 2021. Accessed May 25, 2021.