Stockholm, Sweden (Press Release) – Oncopeptides AB (Nasdaq Stock­holm: ONCO) an­nounces to­day the final top­line re­­sults from the pivotal phase 2 HORIZON study eval­u­ating mel­flu­fen in re­lapsed re­frac­tory mul­ti­ple myeloma (RRMM) patients. These re­­sults will form the basis for the up­com­ing NDA for ac­cel­er­ated ap­­prov­al in the US. The appli­ca­tion is on track for a sub­mission to the FDA at the end of Q2 2020. Oncopeptides will host a webcast to­day to provide an up­date on the final study re­­sults at 14.00 (CET).

The final HORIZON re­­sults rep­re­sent an Over­all Re­sponse Rate (ORR) im­prove­ment for triple-class re­frac­tory RRMM patients com­pared to the interim data pre­sented at the American Society of Hema­tol­ogy (ASH) meeting in De­cem­ber 2019. The HORIZON re­­sults show a good ef­fi­cacy and safety profile for mel­flu­fen in dif­fi­cult to treat RRMM patients. The final HORIZON data reinforce Onco­peptides' view that mel­flu­fen could play an im­por­tant role in the treat­ment of patients with RRMM.

Primary end-point re­­sults

*Two unconfirmed re­sponders on Jan­u­ary­ 14th have later been con­firmed

• Data has also been reviewed by the independent review com­mit­tee (IRC).
• The safety profile was con­sis­tent with pre­vi­ous reports from the HORIZON study with primarily hema­to­logic Adverse Events (AE) and a low in­ci­dence of non-hematologic AEs.
• Full results will be disclosed in a future peer-reviewed pub­li­ca­tion.

Jakob Lindberg, CEO comments:

"The pre­sen­ta­tion of final data from our pivotal HORIZON study, with competitive re­­sults in triple-class re­frac­tory myeloma patients, rep­re­sents the most im­por­tant mile­stone for Onco­peptides to date. These data con­firm that mel­flu­fen has a good ef­fi­cacy and safety profile in triple-class re­frac­tory myeloma patients - a fast-growing patient pop­u­la­tion with sig­nif­i­cant unmet med­i­cal need and lack of ap­prov­ed treat­ments. The safety profile was con­sis­tent with pre­vi­ous mel­flu­fen stud­ies with good tol­er­a­bil­ity and a low rate of non-haematological ad­verse events. We firmly be­lieve that mel­flu­fen has the poten­tial to be­come an im­por­tant treat­ment op­tion for patients with re­lapsed re­frac­tory mul­ti­ple myeloma. Study physicians and clin­i­cal sites have been immensely sup­port­ive and with their help we are on schedule to submit the NDA for ac­cel­er­ated ap­­prov­al end of Q2 2020."

"Furthermore, with the strong final re­­sults from HORIZON our Peptide-Drug Conjugate (PDC) plat­form has been val­i­dated. In to­day's webcast we will describe the PDC pipe­line de­vel­op­ment to date and the possibilities this gives us", concludes Jakob Lindberg.

Oncopeptides will host a webcast to­day to present the data and provide a general clin­i­cal up­date at 14.00 (CET) that can be followed via the link:

https://tv.streamfabriken.com/oncopeptides-press-conference

Participants who would like to ask ques­tions can use the telephone num­bers below:

Sweden: + 46-8-50558358
Europe: + 44-3333009269
USA: + 1-8446251570

The pre­sen­ta­tion can be found at:

www.oncopeptides.com / Investor Relations / Presentations / Presentation webcast HORIZON Topline re­­sults /

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 March 26, 2020 at 09.30 (CET).

About the OP-106 HORIZON study

In the pivotal phase 2 HORIZON study 157 mul­ti­ple myeloma patients have been en­rolled and eval­u­ated. The study was fully recruited in Octo­ber 2019 and the final data cut was made on Jan­u­ary­ 14th. The patients in the study are 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 EMD and/or had cytogenetic high - risk features.

About Melflufen

Melflufen (INN mel­phalan flufenamide) is a first-in-class anti-cancer 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 cleaved 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.

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 anti-cancer 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 cur­rent 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.