Stockholm, Sweden (Press Release) – Oncopeptides AB (Nasdaq Stock­holm: ONCO) an­nounced to­day that, after dis­cus­sions with the FDA, the com­pany has ini­ti­ated the preparation for submitting a New Drug Appli­ca­tion (NDA) for ac­cel­er­ated ap­­prov­al of mel­flu­fen for the treat­ment of patients with triple-class re­frac­tory mul­ti­ple myeloma. The com­pany targets to submit the appli­ca­tion in the first quarter of 2020.

During the spring, Oncopeptides has been engaged in dialogue with the FDA to explore whether mel­flu­fen could be eli­gible for ac­cel­er­ated ap­­prov­al based on the promising data gen­er­ated in the on­go­ing phase 2 HORIZON clin­i­cal trial. The target indi­ca­tion would be for the treat­ment of patients with re­lapsed re­frac­tory mul­ti­ple myeloma whose dis­ease is triple-class re­frac­tory (i.e. re­frac­tory to at least one IMiD, one pro­te­a­some in­hib­i­tor and one anti-CD38 mono­clonal anti­body). In the dis­cus­sions, the FDA has re­quested and re­ceived all avail­able clin­i­cal data at hand for mel­flu­fen.

As a re­­sult of the dialogue with the FDA, Oncopeptides has ini­ti­ated preparations for an NDA sub­mission based on the avail­able HORIZON data. The detailed plan for the filing process is still under devel­op­ment, but Oncopeptides cur­rently targets to submit the appli­ca­tion during the first quarter of 2020. This could then lead to the first mel­flu­fen mar­ket ap­­prov­al in the US in 2020.

"We are very ex­cited over the oppor­tu­ni­ty to help patients with triple-class re­frac­tory mul­ti­ple myeloma who cur­rently have very lim­ited treat­ment op­tions to poten­tially access a new treat­ment alter­na­tive that may have a large im­pact on their lives. The out­come of the regu­la­tory dis­cus­sions during the spring is even better than we antic­i­pated and is a major mile­stone for Oncopeptides as it means that we can start the appli­ca­tion process and poten­tially attain mar­ket ap­­prov­al sig­nif­i­cantly earlier than planned," says Jakob Lindberg, CEO of Oncopeptides.

"Potential ap­­prov­al will of course de­pend on the formal FDA re­view once we have sub­mitted the appli­ca­tion, but based on the dis­cus­sions and the data at hand, we be­lieve we have a very good chance to secure an ap­­prov­al, provided that the re­­sults gen­er­ated in the HORIZON trial con­tinue to be in line with the data we have seen so far. It will there­fore be very ex­cit­ing to present up­dated HORIZON data at the Euro­pean Hema­tol­ogy Asso­ci­a­tion (EHA) meeting in Amsterdam on June 16," concludes Jakob Lindberg.

Conference call for in­­vestors, analysts and the media

Oncopeptides will host a conference call and present an op­er­a­tional up­date on Tuesday May 21, 2019 at 10:00 (CET). It will be pre­sented by CEO Jakob Lindberg and members of the Oncopeptides man­agement team. The conference call will also be streamed via a link on the website: www.oncopeptides.com.

Phone num­bers for par­tic­i­pants from:
Sweden: +46-8-505-583-65
Europe: +44-3333-009-035
USA: +1-833-526-8381

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 May 20, 2019 at 18.00 (CET).

About mel­flu­fen

Melflufen is a lipophilic peptide-conjugated al­kyl­ator that rapidly de­livers a highly cyto­toxic pay­load into myeloma cells through peptidase ac­­tiv­ity. It belongs to the novel class of Peptidase Enhanced Cytotoxics (PEnC), which is a family of lipophilic peptides that exhibit in­­creased ac­­tiv­ity via peptidase cleavage and have the poten­tial to treat many can­cers. Peptidases play a key role in pro­tein homeo­stasis and feature in cel­lu­lar processes such as cell-cycle pro­gres­sion and pro­grammed cell death. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is im­medi­ately cleaved by peptidases to de­liver an entrapped hydrophilic al­kyl­ator pay­load. In vitro, mel­flu­fen is 50-fold more potent in myeloma cells than the al­kyl­ator pay­load itself due to the peptidase cleavage, and induces irreversible DNA damage and apop­tosis. 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.

About Oncopeptides

Oncopeptides is a pharma­ceu­tical com­pany devel­op­ing drugs for the treat­ment of can­cer. The com­pany is focusing on the devel­op­ment of the lead prod­uct can­di­date mel­flu­fen, a novel lipophilic peptide-conjugated al­kyl­ator, belonging to a new class of drugs called Peptidase Enhanced Cytotoxics (PEnC). Melflufen is in devel­op­ment as a new treat­ment for the hema­to­logical can­cer mul­ti­ple myeloma and is cur­rently being tested in a global pivotal phase 3 trial called OCEAN, a phase 2 trial called HORIZON and in two addi­tional sup­porting clin­i­cal trials. Oncopeptides' head­quar­ters is located in Stock­holm, Sweden and the com­pany is listed in the Mid Cap segment on Nasdaq Stock­holm with the ticker ONCO.

Visit www.oncopeptides.com for more in­for­ma­tion.

Source: Oncopeptides.