Stockholm, Sweden (Press Release) – Oncopeptides AB (publ) (Nasdaq Stock­holm: ONCO) to­day an­nounces that the Com­pany has in­formed the Euro­pean Medicines Agency, EMA, about its intention to submit an appli­ca­tion for a con­di­tional mar­ket­ing autho­ri­za­tion of mel­flu­fen (INN mel­phalan flufenamide) in the EU, based on the pivotal phase 2 HORIZON study in re­lapsed re­frac­tory mul­ti­ple myeloma (RRMM).

The de­ci­sion to submit an appli­ca­tion for con­di­tional ap­prov­al has been grounded on an in-depth analysis of the regu­la­tory en­viron­ment and is endorsed by key opinion leaders in the EU. Previously the Com­pany in­tended to await the re­sults from the on­go­ing ran­dom­ized, phase 3 OCEAN study be­fore submitting an appli­ca­tion for mar­ket­ing autho­ri­za­tion. Upon com­ple­tion, the out­come from the OCEAN study com­par­ing mel­flu­fen and poma­lido­mide in patients with RRMM, will be sub­mitted to regu­la­tory author­i­ties to po­ten­tially ex­pand the label of mel­flu­fen.

The HORIZON study dem­onstrates that mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone has a po­ten­tial to pro­vide a thera­peutic op­tion for patients with RRMM that are hard to treat and have a poor prog­nosis, in­clud­ing patients with triple-class re­frac­tory myeloma and patients with ex­tra­med­ul­lary dis­ease (EMD).

"Key opinion leaders and clinics across Europe have gained extensive ex­peri­ence of mel­flu­fen from our clin­i­cal de­vel­op­ment pro­gram in mul­ti­ple myeloma. We share a mutual interest to enable early access to this rapidly grow­ing patient pop­u­la­tion in des­per­ate need of new treat­ment op­tions", says Klaas Bakker, CMO of Oncopeptides.

According to the Euro­pean Medicines Agency, med­i­cines are eli­gible for con­di­tional ap­prov­al if they are aimed at treating or preventing seriously debilitating or life-threatening dis­eases. Conditional mar­ket­ing autho­ri­za­tions may be granted if; the ben­e­fit-risk bal­ance of the prod­uct is pos­i­tive, com­pre­hen­sive data can be pro­vided, there is an unmet med­i­cal need, and the ben­e­fit to pub­lic health of making the prod­uct avail­able outweighs the risks due to need for addi­tional data.

The US Food and Drug Admin­istra­tion, FDA, has granted priority re­view to Oncopeptides' New Drug Appli­ca­tion of mel­flu­fen in com­bi­na­tion with dexa­meth­a­sone for treat­ment of patients with mul­ti­ple myeloma. The FDA has set a target date for the re­view of the New Drug Appli­ca­tion, to Feb­ru­ary 28, 2021.

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.