Stockholm, Sweden (Press Release) – Oncopeptides AB (publ) (Nasdaq Stock­holm: ONCO) an­nounced to­day that the open-label Expanded Access Program, sEAPort, for eli­gible U.S. patients, is formally open. Melflufen (INN mel­phalan flufenamide), is cur­rently being eval­u­ated in sev­er­al clin­i­cal stud­ies as a treat­ment for patients with triple-class re­frac­tory mul­ti­ple myeloma. The sEAPort pro­gram is avail­able to adults, age 18 and older, who have re­ceived at least two prior lines of ther­apy and whose mul­ti­ple myeloma is re­frac­tory to at least one pro­te­a­some in­hib­i­tor, one immuno­modu­la­tory drug and one anti-CD38 mono­clonal anti­body, (i.e., triple-class re­frac­tory mul­ti­ple myeloma patients).

The Expanded Access Program was ini­ti­ated fol­low­ing the Com­pany's June 30 sub­mission of a New Drug Appli­ca­tion (NDA) to the U.S. Food and Drug Admin­istra­tion, FDA, for ac­cel­er­ated ap­prov­al of mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone for the treat­ment of adult patients with triple-class re­frac­tory mul­ti­ple myeloma. The NDA and the sEAPort pro­gram are primarily sup­ported by data from the pivotal phase 2 HORIZON study, which dem­onstrates that mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone, has a po­ten­tial to provide a thera­peutic op­tion for patients with re­lapsed re­frac­tory mul­ti­ple myeloma who are hard to treat and have a poor prog­nosis, in­clud­ing patients with triple-class re­frac­tory mul­ti­ple myeloma and patients with ex­tra­med­ul­lary dis­ease.

"Despite thera­peutic ad­vances, mul­ti­ple myeloma re­mains incurable," said Paula O'Connor, U.S. Head of Medical Affairs at Oncopeptides. "There is an urgent need for more ther­a­pies as patients be­come multi-resistant earlier in their treat­ment journey. Our Expanded Access Program enables us to provide access to mel­flu­fen as a po­ten­tial treat­ment for eli­gible patients while our appli­ca­tion is under re­view by the U.S. Food and Drug Admin­istra­tion."

EAPs are de­signed to provide patients living with serious or life-threatening con­di­tions access to inves­ti­ga­tional med­i­cines when no com­parable or sat­is­fac­tory treat­ment op­tions are avail­able, alter­na­tive ther­a­pies have been ex­hausted or the patient is in­eli­gible for on­go­ing interventional trials.

Forty to fifty med­i­cal sites in the U.S. are ex­pected to en­roll 100-200 patients in the sEAPort pro­gram.

More in­for­ma­tion about our clin­i­cal trials can be found at www.clinicaltrials.gov

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.