Jakob Lindberg, CEO of Oncopeptides @oncopeptides explains the combination of the pivotal phase 2 study, HORIZON and phase 3 study, OCEAN, becoming key studies for melphalan flufenamide in the US and the EU.
Oncopeptides highlighted its topline results for the pivotal HORIZON study: melflufen (INN melphalan flufenamide) showing a 26% overall response rate in triple-class RRMM patients, a patient population who currently have limited treatment options.
Of note, these results will form the basis for the accelerated NDA in the US, with the application on track for a submission to the FDA at the end of Q2 2020. For further background on this news, Oncopeptides hosted a webcast today to provide an update on the final study results and it can be found on-demand here.
"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.