Stockholm, Sweden (Press Release) – Oncopeptides AB (publ) (Nasdaq Stock­holm: ONCO) to­day an­nounces that the US Food and Drug Admin­istra­tion, FDA, has granted priority re­view for Oncopeptides' New Drug Appli­ca­tion seek­ing ap­prov­al of mel­flu­fen (INN mel­phalan flufenamide), in com­bi­na­tion with dexa­meth­a­sone for the treat­ment of adult patients with mul­ti­ple myeloma whose dis­ease is re­frac­tory to at least one pro­te­a­some in­hib­i­tor, one immuno­modu­la­tory agent and one anti-CD-38 mono­clonal anti­body, (i.e., triple-class re­frac­tory mul­ti­ple myeloma patients). The FDA has set a PDUFA-date (Prescription Drug User Fee Act), which is the target date for their re­view of the New Drug Appli­ca­tion, to Feb­ru­ary 28, 2021.

The sub­mission is based on the re­­sults from the pivotal phase 2 study HORIZON, eval­u­ating in­tra­venous mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone in patients with re­lapsed re­frac­tory mul­ti­ple myeloma, (RRMM).

"This is very ex­cit­ing news. It is an im­por­tant mile­stone for Oncopeptides, and a major step in making mel­flu­fen avail­able for patients with mul­ti­ple myeloma, who des­per­ately need new treat­ment op­tions", says Marty J Duvall, CEO of Oncopeptides AB. "I am looking for­ward to a continuing dialogue with the FDA while we make the prod­uct avail­able to RRMM patients in the US through an ex­panded access pro­gram, in an FDA ap­prov­ed trial called sEAPort."

A Priority Review desig­na­tion means that FDA's goal is to take action on an appli­ca­tion within 6 months (compared to 10 months under stan­dard re­view). They will direct over­all resources to the eval­u­a­tion of appli­ca­tions for drugs that, "if ap­prov­ed, would be sig­nif­i­cant im­prove­ments in the safety or ef­fec­tiveness of the treat­ment, diag­nosis, or prevention of serious con­di­tions when com­pared to stan­dard appli­ca­tions".

The in­for­ma­tion in the press re­lease is in­for­ma­tion that Oncopeptides is obliged to make pub­lic pur­su­ant to the EU Market Abuse Regulation. The in­for­ma­tion was sub­mitted for pub­li­ca­tion on August 29, 2020 at 10.00 (CET).

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.