Stockholm, Sweden (Press Release) – Oncopeptides AB (publ) (Nasdaq Stock­holm: ONCO) to­day an­nounces that the last patient has been suc­cess­fully en­rolled in the pivotal phase 3 study OCEAN in re­lapsed re­frac­tory mul­ti­ple myeloma (RRMM). The original en­roll­ment target of 450 patients was reached in May 2020, but an analysis in­di­cated that patients were staying longer on treat­ment than ini­tially esti­mated. Thus, a de­ci­sion was made to leave recruitment open to ensure that the num­ber of dis­ease pro­gres­sion events needed to com­plete the study would be reached within a reason­able time­frame. An addi­tional 45 patients have now been recruited. The com­pany has closed en­roll­ment and reiterates that the top­line re­­sults will be avail­able in the first half of 2021.

OCEAN is a ran­dom­ized, comparative study be­tween mel­flu­fen (INN mel­phalan flufenamide), and poma­lido­mide in patients with RRMM. The study was ini­ti­ated in 2017 and to­day in­cludes 495 patients from more than 100 hos­pi­tals around the world. The patients have pre­vi­ously been treated with at least an immunomodulator (IMiD) and a pro­te­a­some in­hib­i­tor (PI) and have all devel­oped re­sis­tance to their last line of ther­apy and to lena­lido­mide (IMiD), the most commonly used mul­ti­ple myeloma drug. The pri­mary end­point of the phase 3 OCEAN study is Progression Free Survival. The data will be eval­u­ated once 339 patients have progressed in their dis­ease.

"This is very ex­cit­ing news. Thanks to the ded­i­cated par­tic­i­pa­tion from patients, in­ves­ti­ga­tors and study teams around the world, we have man­aged to reach our extended en­roll­ment target faster than antic­i­pated, de­spite these chal­leng­ing times", says Marty J Duvall, CEO of Oncopeptides AB. "I am really impressed by the journey that Oncopeptides has made ever since the study start in 2017, from being a small Stock­holm based R&D com­pany, to becoming a fully- fledged, integrated, global bio­pharma com­pany, preparing the first com­mer­cial launch".

The U.S. Food and Drug Admin­istra­tion, FDA, has recently granted priority re­view for Oncopeptides' New Drug Appli­ca­tion seek­ing ap­prov­al of mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone for the treat­ment of adult patients with triple class re­frac­tory mul­ti­ple myeloma. The de­ci­sion was based on the re­­sults from the on­go­ing pivotal phase 2 study HORIZON, eval­u­ating mel­flu­fen in RRMM patients.

About mel­flu­fen

Melflufen (INN mel­phalan flufenamide) is a first in class peptide-drug con­ju­gate (PDC) that targets aminopeptidases and rapidly re­leases al­kyl­at­ing agents into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is im­medi­ately hydrolyzed by peptidases to re­lease an entrapped hydrophilic al­kyl­ator pay­load. Aminopeptidases are overexpressed in tumor cells and are even more pronounced in ad­vanced can­cers and tumors with a high mutational burden. In vitro, mel­flu­fen is 50-fold more potent in myeloma cells than the al­kyl­ator pay­load itself due to the in­creased intracellular al­kyl­ator con­cen­tra­tion. Melflufen dis­plays cyto­toxic ac­­tiv­ity against myeloma cell lines re­sis­tant to other treat­ments, in­clud­ing al­kyl­ators, and has also dem­onstrated in­hib­ition of DNA repair induction and angiogenesis in pre­clin­i­cal stud­ies. In the pivotal phase 2 HORIZON study mel­flu­fen plus dexa­meth­a­sone dem­onstrated en­cour­ag­ing ef­fi­cacy and a clin­i­cally man­ageable safety profile in heavily pre­treated patients with re­lapsed re­frac­tory mul­ti­ple myeloma, with primarily hema­to­logic Ad­verse Events (AE) and a low in­ci­dence of non-hematologic AEs.

About Oncopeptides

Oncopeptides is a pharma­ceu­tical com­pany focused on the de­vel­op­ment of targeted ther­a­pies for dif­fi­cult-to-treat hema­to­logical dis­eases. The com­pany is focusing on the de­vel­op­ment of the lead prod­uct can­di­date mel­flu­fen, a first in class peptide-drug con­ju­gate (PDC) that targets aminopeptidases and rapidly re­leases al­kyl­at­ing agents into tumor cells. Melflufen (INN mel­phalan flufenamide) is in de­vel­op­ment as a new treat­ment for the hema­to­logical malig­nan­cy mul­ti­ple myeloma and is cur­rently being tested in mul­ti­ple clin­i­cal stud­ies in­clud­ing the pivotal phase 2 HORIZON study and the on­go­ing phase 3 OCEAN study. Based on the re­­sults from the HORIZON study Oncopeptides has sub­mitted a New Drug Appli­ca­tion (NDA) to the U.S. Food and Drug Admin­istra­tion, FDA, for ac­cel­er­ated ap­prov­al of mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone for treat­ment of adult patients with triple-class re­frac­tory mul­ti­ple myeloma. Oncopeptides' global Headquarters is in Stock­holm, Sweden and the U.S. Headquarters is situated in Boston, Mass. The com­pany is listed in the Mid Cap segment on Nasdaq Stock­holm with the ticker ONCO. More in­for­ma­tion is avail­able on www.oncopeptides.com.

Source: Oncopeptides.