Stockholm, Sweden (Press Release) – Oncopeptides AB (publ) (Nasdaq Stock­holm: ONCO) to­day an­nounces that the Com­pany submits a New Drug Appli­ca­tion (NDA) to the U.S. Food and Drug Admin­istra­tion, FDA, for ac­cel­er­ated 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-CD38 mono­clonal anti­body (i.e., triple-class re­frac­tory mul­ti­ple myeloma patients).

Melflufen is the lead can­di­date com­ing out of the Oncopeptides' pro­pri­e­tary PDC-platform. The prod­uct is a first-in-class aminopeptidase-targeting peptide-drug con­ju­gate that rapidly de­livers an al­kyl­at­ing pay­load into tumor cells. 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).

The re­­sults from 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). The re­sponses in the HORIZON study were durable and often deep­ened with pro­longed treat­ment, sug­gesting that patients could ben­e­fit from staying on treat­ment for as long as pos­si­ble.

"I am very proud and humbled by the or­ga­ni­za­tions ability to timely submit the NDA for ac­cel­er­ated ap­prov­al of mel­flu­fen. This is a major mile­stone for Oncopeptides and is a re­­sult of ded­i­cated re­search and de­vel­op­ment ac­­tiv­i­ties through­out the last decade", says Jakob Lindberg CEO of Oncopeptides. "I would like to ex­press my sincere gratitude to all patients, co-workers, in­ves­ti­ga­tors and share­holders who have provided relentless sup­port to enable a novel treat­ment op­tion for a fast-growing patient pop­u­la­tion with a sig­nif­i­cant unmet med­i­cal need".

Following the sub­mission to the FDA Oncopeptides will ini­ti­ate an Expanded Access Program (EAP) in the U.S. to enable mel­flu­fen treat­ment for patients with a sig­nif­i­cant unmet med­i­cal need.

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, through the agency of the contact person above, on June 30, 2020 at 08.00 (CET).

About the HORIZON study

In total 157 mul­ti­ple myeloma patients have been en­rolled and eval­u­ated in the pivotal phase 2 HORIZON study. The study was fully recruited in Octo­ber 2019, the final data cut was made on Jan­u­ary 14th and the final data was pre­sented at the EHA meeting in June. The patients in the study were re­frac­tory to poma­lido­mide and/or dara­tu­mu­mab after failing on immuno­modu­la­tory drugs (IMiDs) and pro­te­a­some in­hib­i­tors (PIs). The HORIZON study pop­u­la­tion in­cludes subgroups of patients who were triple-class re­frac­tory and/or had ex­tra­med­ul­lary dis­ease and/or had cytogenetic high-risk features.

Summary of re­­sults

All data were con­firmed by the Independent Review Com­mit­tee (IRC), with only minimal discordance.

About mel­flu­fen

Melflufen (INN mel­phalan flufenamide) is a first-in-class aminopeptidase-targeting peptide-drug con­ju­gate that rapidly de­livers an al­kyl­at­ing pay­load into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is im­medi­ately hydrolyzed by peptidases to de­liver an entrapped hydrophilic al­kyl­ator pay­load. Peptidases play a key role in pro­tein homeo­stasis and feature in cel­lu­lar processes such as cell-cycle pro­gres­sion and pro­grammed cell death. 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.

Expanded access policy

The preparations for an Expanded Access Program (EAP) in the US are well underway and the pro­gram will open in Q3. Oncopeptides en­cour­ages awareness of and par­tic­i­pa­tion in its clin­i­cal trials and be­lieves that participating in clin­i­cal trials is a good way for patients to access inves­ti­ga­tional drugs prior to regu­la­tory ap­prov­al. Individuals interested in participating in clin­i­cal trials for mel­flu­fen may visit https://www.oncopeptides.se/en/our-clinical-trials-summary/ for in­for­ma­tion about on­go­ing clin­i­cal trials. Patients are en­cour­aged to consult their physician re­gard­ing the possibility of participating in one of the on­go­ing clin­i­cal trials.

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 aminopeptidase-targeting peptide-drug con­ju­gate that rapidly de­livers an al­kyl­at­ing pay­load into tumor cells. Melflufen 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. Oncopeptides' head­quar­ters is in Stock­holm, Sweden with its U.S. head­quar­ters 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.