Clinical Program

Clinical program

The purpose of our melflufen clinical development program is to support regulatory approval processes globally and explore safe and effective use in different patient populations, use in combination with other myeloma treatments and administration with both central and peripheral infusion, and real-world use in Germany.

Effective July 8, 2021, the U.S. Food and Drug Administration, FDA, requested a partial clinical hold of the clinical program with melflufen and OPD5 in the US, prompting Oncopeptides to refocus its clinical program to support the marketing authorization process in Europe that led to the approval of melflufen by the European Medicines Agency, EMA, and UK’s Medicines and Healthcare products Regulatory Agency, MHRA, in Aug and Nov 2022 respectively.

O-12-M1

O-12-M1 is a completed international, open-label phase 1/2 study to establish the dose of melflufen in combination with dexamethasone and investigate the treatment response in patients with relapsed refractory multiple myeloma, who were refractory to the last line of therapy.

HORIZON

The phase 2 HORIZON study is a pivotal, single-arm study, evaluating the safety and efficacy of melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma.

OCEAN

The phase 3 OCEAN study is a randomized, head-to-head study, evaluating the efficacy and safety of melflufen and dexamethasone, versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma.

ANCHOR

ANCHOR is a phase 1/2 open label multicenter study evaluating the safety and efficacy of melflufen plus dexamethasone in combination with either daratumumab or bortezomib in patients with relapsed refractory multiple myeloma.

BRIDGE

The phase 2 BRIDGE study is an open-label, single-arm study, designed to evaluate the pharmacokinetics, safety and efficacy of melflufen in combination with dexamethasone in multiple myeloma patients with reduced renal function.

PORT

The phase 2 PORT study is an open-label, randomized, cross-over study which compared pharmacokinetics, safety and tolerability of peripheral or central intravenous administration of melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma.

LIGHTHOUSE

The phase 3 LIGHTHOUSE study is a randomized, open-label, phase 3 study of melflufen and dexamethasone in combination with daratumumab compared to daratumumab alone in patients with relapsed and refractory multiple myeloma.

ASCENT

ASCENT was a Phase 1/2 study with the objective of exploring the safety and tolerability of melflufen in patients with AL amyloidosis following at least one prior line of therapy. The study was prematurely terminated on 04 November 2021.

HARBOUR

HARBOUR, OP-113 is a non-interventional study (NIS), of melphalan flufenamide (melflufen, Pepaxti^®) in combination with dexamethasone in patients with relapsed and/or refractory multiple myeloma (RRMM) according to the approved label.

COAST

COAST is an open label, phase 1/2 study assessing safety, tolerability and efficacy of OPD5 in combination with dexamethasone for patients with Relapsed refractory multiple myeloma (RRMM). The protocol was approved by health authorities and ethics committees, but the study was terminated before including any patients.

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Clinical Development