Our clinical development program will provide us with a broad set of data and information about melflufen’s (INN melphalan flufenamide) efficacy in various patient groups. On June 30, 2020, we submitted a New Drug Application (NDA) to the US Food & Drug Administration (FDA) for accelerated approval based on final clinical data from the HORIZON study.
The clinical development program
We are currently conducting five clinical studies to characterize melflufen in multi-refractory multiple myeloma patients: OCEAN (OP-103), HORIZON (OP-106), ANCHOR (OP-104), BRIDGE (OP-107) and PORT (OP-109).
The program will provide a clear picture of how melflufen can be used for relapsed refractory multiple myeloma (RRMM) patients in various stages of the disease. This has lowered the development risk and given rise to several potential paths for obtaining approval for melflufen.
Melflufen has previously undergone both preclinical studies and clinical phase 1 and 2 studies with positive results in terms of both safety and efficacy in patients with multiple myeloma. Based on these results, the next logical step was to further develop melflufen through the studies OCEAN, HORIZON, ANCHOR and BRIDGE, PORT and the planned additional pivotal combination study LIGHTHOUSE.
Our pivotal phase 2 study, HORIZON and phase 3 study, OCEAN, are key studies for the submission of an NDA/MAA to potentially obtain marketing authorization for melflufen in the US and the EU for the treatment of RRMM. In addition to evaluating melflufen’s efficacy in relation to the existing standard treatment for RRMM (meaning pomalidomide), as evaluated by OCEAN, the development program also aims to demonstrate, through HORIZON, the activity of melflufen in patients with relapsed refractory multiple myeloma whose disease is triple-class refractory (i.e. refractory to at least one IMiD, one proteasome inhibitor and one anti-CD38 monoclonal antibody). Our phase 1/2 study, ANCHOR, is aimed at demonstrating how melflufen can be administered in combination with other multiple-myeloma drugs. BRIDGE is a phase 2 pharmacokinetic study to study melflufen’s safety in patients with reduced renal function. We are also preparing to start a pivotal phase 3 study called LIGHTHOUSE, which is expected to start during the fall 2020.
The regulatory path ahead
The submission in June 2020, for accelerated approval in the US for melflufen for the treatment of RRMM patients with triple-class refractory disease, is the first step in building a potential label for melflufen within myeloma. A potential accelerated approval results in a regulatory approval that later needs to be confirmed with clinical data from a randomized study. Both OCEAN and LIGHTHOUSE can independently act as confirmatory studies for a potential accelerated approval. Additionally, both OCEAN and LIGHTHOUSE – assuming positive outcome from the studies – can result in broadening of the label into less advanced RRMM patient populations (both studies) as well as in combination with daratumumab (LIGHTHOUSE).
Oncopeptides has collaborated with leading experts and held discussions with governing medical agencies and professional bodies in the US and Europe to create the development program for melflufen in RRMM. Upon receiving approval of the phase 3 OCEAN study design through the FDA Special Protocol Assessment in August 2016, detailed preparations commenced for the development program of melflufen. The program aims to fully characterize melflufen in the treatment of RRMM and thereby maximize the product candidate’s market potential.
In the OCEAN clinical phase 3 study, the efficacy of Oncopeptides’ product candidate, melflufen, is compared with pomalidomide, both are administered in combination with the steroid dexamethasone.