Clinical Development

The clinical development of targeted therapies for difficult-to-treat hematological diseases and malignancies are based on our peptide-drug conjugate platform, PDC.

Investigators and Patients should be aware that there is a partial clinical hold in place for Oncopeptides’ Clinical Trials, which means that we are not currently enrolling new patients. If patients are receiving a clinical benefit from melphalan flufenamide they may continue treatment provided they are informed of the risks and sign a revised written informed consent.

Melflufen Clinical Development plan

OPD5 Clinical development plan

Our focus

We are focusing on the late-stage development of our lead product candidate melflufen in multiple myeloma, we are enrolling patients in our AL-amyloidosis study, ASCENT, the first study outside myeloma, and we are preparing clinical studies for the second drug candidate coming out of our PDC-platform, OPD5.

FDA approval and EMA submission

Melphalan flufenamide, also known as melflufen, is a first-in-class peptide-drug conjugate that targets aminopeptidases and rapidly releases alkylating agents inside cancer cells. Our clinical studies generate a wealth of data about melflufen’s efficacy and side effect profile in various groups of patients with multiple myeloma. February 26, 2021, the US Food & Drug Administration, FDA, approved PEPAXTO® (melphalan flufenamide, also known as melflufen),based on the pivotal phase 2 HORIZON study.  On April 16, we submitted a New Drug Application to the European Medicines Agency, EMA, for conditional marketing authorization of melflufen (melphalan flufenamide) in the EU.

Robust clinical program

Melflufen is currently evaluated in a robust program. The clinical strategy has evolved over time, originally based on the results from our first clinical study O-12-M1, a phase 1/2 study in multiple myeloma conducted between 2013 – 2017. 

Clinical development strategy

Our clinical development strategy is to establish melflufen as a potential cornerstone in the treatment of multiple myeloma after the first line of therapy. To broaden the indication outside multiple myeloma, we have recently initiated a study in AL-Amyloidosis, ASCENT. The goal is to fully explore the benefit that melflufen can bring to patients with difficult to treat hematological disorders.

Next drug candidate in clinical development

Our goal is to establish a stream of new clinical candidates going forward and we expect to nominate new candidate drugs on a yearly basis.

 

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.

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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.

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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.

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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.

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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.

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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.

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PORT

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

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ASCENT

The phase 1/2 ASCENT study is the first study with melflufen outside multiple myeloma. The ASCENT study is an open-label study with melflufen and dexamethasone in patients with AL-amyloidosis, who have undergone at least one previous line of therapy.

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