The clinical development of targeted therapies for difficult-to-treat hematological diseases and malignancies are based on our peptide-drug conjugate platform, PDC.
We are focusing on the late-stage development of our lead product candidate melflufen in multiple myeloma, we are enrolling patients in our AL-amyloidosis study, ASCENT, the first study outside myeloma, and we are preparing clinical studies for the second drug candidate coming out of our PDC-platform, OPD5.
Priority review by the FDA
Melflufen is a first in class peptide-drug conjugate that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. Our clinical studies generate a wealth of data about melflufen’s efficacy and side effect profile in various groups of patients with multiple myeloma. On June 30, we submitted a New Drug Application to the US Food & Drug Administration, FDA for accelerated approval based on the pivotal phase 2 HORIZON study. The FDA has granted our application a priority review and set a PDUFA date to February 28, 2021, when the review should be completed.
Robust clinical program
Melflufen is currently evaluated in a robust program. The clinical strategy has evolved over time, originally based on the results from our first clinical study O-12-M1, a phase 1/2 study in multiple myeloma conducted between 2013 – 2017. At the European Hematology Meeting in June 2020, we presented final results from our pivotal phase 2 HORIZON study in patients with triple-class refractory multiple myeloma, including a large cohort of patients with Extra Medullary Disease.
In September 2020 we completed the enrollment to the phase 3 OCEAN study including 495 patients. The primary end point is progression free survival. The data will be evaluated once 339 patients have reached progression. Top line results are expected during the first half of 2021. Other ongoing studies include the phase 2 studies ANCHOR, BRIDGE, PORT and ASCENT. First quarter 2021 we plan to start the comparative phase 3 LIGHTHOUSE study.
Clinical development strategy
Our clinical development strategy is to establish melflufen as a potential cornerstone in the treatment of multiple myeloma after the first line of therapy. To broaden the indication outside multiple myeloma, we have recently initiated a study in AL-Amyloidosis, ASCENT. The goal is to fully explore the benefit that melflufen can bring to patients with difficult to treat hematological disorders.
Next drug candidate in clinical development
Recently we have received an approval by the FDA to start clinical development of OPD5, the second drug candidate coming out of our PDC-platform.