Oncopeptides has announced that the full top-line results from the phase 2 HORIZON study, evaluating intravenous melflufen in combination with dexamethasone in patients with relapsed refractory multiple myeloma (RRMM), have been presented at the ongoing 25th European Hematology Association meeting, EHA.
The results support the NDA submission to the US Food and Drug Administration, FDA, for accelerated approval of melflufen. The company is on track to submit the application to FDA by the end of Q2, 2020.
Melflufen is an anticancer peptide-drug conjugate that rapidly delivers an alkylating payload into tumor cells. The results from the HORIZON study demonstrates that melflufen in combination with dexamethasone, have the potential to provide a therapeutic option for patients with relapsed refractory multiple myeloma (RRMM) that are hard to treat and have a poor prognosis, including patients with triple class refractory myeloma and patients with Extramedullary Disease. Responses were durable and often deepened with prolonged treatment, suggesting that patients could benefit from staying on treatment for as long as possible. The results are consistent with previously reported data, while no new safety concerns were identified.
“The HORIZON data is an important milestone for Oncopeptides and further validates our Peptide-Drug Conjugate platform,” says Klaas Bakker, MD and CMO of Oncopeptides. “The results are in line with results from previous interim analyses. Notably, the PFS of 8.5 months in responding patients (both all treated and triple-class refractory), was materially higher than the DOR, which is explained by the relatively long-time it took for patients to respond (median 1.9 months). This is very encouraging for patients with an unmet need; ultimately this is the period patients benefit from treatment with melflufen. The fact that the treatment also seems to be well tolerable makes this a potentially attractive treatment option for a fast-growing patient population with a significant unmet medical need.”
Photo of Klaas Bakker: Oncopeptides