Oncopeptides (STO:ONCO) announced today that it received FDA approval for its Pepaxto treatment for multiple myeloma.
Waltham, Mass.-based Oncopeptides’ Pepaxto (melphalan flufenamide) received approval in combination with dexamethasone for treating adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one CD38-directed monoclonal antibody, according to a news release.
The company touts Pepaxto, which was approved based on results from its Horizon trial, as the first anticancer peptide-drug conjugate approved for multiple myeloma.
Pepaxto uses technology that links a peptide carrier to a cytotoxic agent, resulting in a lipophilic compound that helps to deliver Papxto into cells to leverage aminopeptidases that are overexpressed in multiple myeloma cells and cause the release of the cytotoxic agents. It is administered once per month in a 30-minute infusion process.
The Horizon trial evaluated 157 patients with relapsed or refractory multiple myeloma, with 97 of the patients falling under the triple-class refractory category with at least four prior lines of treatment. The overall response rate for that group was 23.7% and the median duration of response was more than four months.
Oncopeptides expects the Pepaxto treatment to be made commercially available in the U.S. within approximately two weeks.
“We are proud to bring forward the first anticancer peptide-drug conjugate approved by the FDA for multiple myeloma,” Oncopeptides chief scientific officer Jakob Lindberg said in the news release. “Pepaxto uses innovative technology that links a peptide carrier to a cytotoxic agent. The conjugated agent is a highly lipophilic compound, which allows it to be rapidly distributed into cells. The compound then leverages aminopeptidases that are overexpressed in multiple myeloma cells, causing the release of the cytotoxic payload.”
Pepaxto received FDA approval under an accelerated timeline based on the results of the Horizon trial.
“The accelerated U.S. approval of Pepaxto is a key step forward in fulfilling Oncopeptides’ core mission, to bring hope to patients in their battle against difficult-to-treat hematological diseases,” CEO Marty Duvall added. “Moving ahead, our focus is to further advance Pepaxto. We look forward to receiving top-line data from the phase 3 OCEAN study in the second quarter. This comparative study is designed to support a future supplementary New Drug Application to expand the label.”