Clinical program - on hold

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

Oncopepeptides has decided to reduce the activity level in the clinical development program with melflufen (INN melphalan flufenamide) to increase the company's cash runway and at the same time support the ongoing marketing authorization application process in Europe.

  • OCEAN study will continue with long-term follow-up and documentation in accordance to previous plans.
  • Patient recruitment has been completed in both PORT and BRIDGE and the studies can be closed with relevant scientific data sets.
  • ANCHOR will close without the last 10 previously planned patients in the bortezomib + melflufen study arm – data sets will be large enough to draw relevant scientific conclusions.
  • ASCENT, COAST and LIGHTHOUSE will close with incomplete number of patients, it will not be possible to draw any relevant scientific conclusions from these data sets.

Read the press release - Oncopeptides focuses clinical development efforts to increase cash runway

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

Our focus

The purpose of our clinical development program is to support the ongoing filing of melflufen in the EU and lay the foundation for the next generation of drug candidates from the PDC platform.

FDA approval and EMA submission

On February 26, 2021, PEPAXTO (melphalan flufenamide) in combination with dexamethasone was granted accelerated approval by the U.S. Food and Drug Administration, FDA, 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.

On October 22, 2021, the company decided to withdraw PEPAXTO from the market in the US following data from the phase 3 OCEAN study which showed an overall survival in the ITT population with a HR of 1.104.

Oncopeptides also announced that it will continue to support the filing for conditional marketing authorization of melflufen (melphalan flufenamide) in the EU to the European Medicines Agency, EMA.

January 21, 2022, Oncopeptides announced that the Company has contacted the US Food and Drug Administration and rescinded the October 22, 2021, letter requesting voluntary withdrawal of the NDA of Pepaxto® in the US.

Purpose of clinical program

The purpose of our clinical development program is to support the ongoing filing of melflufen in the EU and lay the foundation for the next generation of drug candidates from the PDC platform.

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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