Multiple myeloma - March 2021

Multiple myeloma - March 2021

FDA approved Oncopeptides´ PEPAXTO (melphalan flufenamide) for patients with relapsed triple-class refractory multiple myeloma

01 Mar 2021

PEPAXTO (melphalan flufenamide/ melflufen – Peptide-drug conjugate) – Oncopeptides

  • FDA has approved PEPAXTO in combination with dexamethasone for the treatment of 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
  • PEPAXTO was granted accelerated approval based on the Phase 2 HORIZON study
  • Trial details: HORIZON; Phase 2 single-arm study in multiple myeloma; N= 157; PEPAXTO 40 mg Day 1 + dexamethasone 40 mg on Days 1, 8, 15 and 22 of each 28-day cycle; Primary outcomes: ORR; PCD: Sep’20; Active, not recruiting; Location: US, EU
  • Results: (N= 157)
    • The overall response rate was 23.7%
    • The median duration of response was 4.2 months
    • Melflufen in combination with dexamethasone demonstrated activity in a subset of patients with Extra Medullary Disease (EMD) (41%), an aggressive and resistant disease associated with a poor prognosis

PEPAXTO will be commercially available in the US in ~ two weeks

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CI Scientists Remarks:

  • Oncopeptides is a company focused on the development of targeted therapies for difficult-to-treat hematological diseases based on its proprietary Peptide Drug Conjugate platform (PDC)
  • About the drug: Melflufen flufenamide is a first in class peptide-drug conjugate that targets aminopeptidases and releases alkylating agents into tumor cells
  • Within the US, Oncopeptides plans to commercialize PEPAXTO on its own with launch expected in 2 weeks
  • PEPAXTO is positioned as a drug for patients who are refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody as “Triple Class Refractory”
  • Future strategy: Oncopeptides plans to expand the label into 3L+ RRMM as a single agent (OCEAN study) and 2L+ RRMM in combination (ANCHOR study)
  • Other indications:

  • PEPAXTO + Dexa + Borte (Phase 2)

    Soft tissue EMD disease

    Expected initiation: H2’21

    PEPAXTO + BiTe or CAR-T (Phase 2/3)

    Multiple myeloma

    Expected initiation: 2022

    PEPAXTO (Phase 1/2)

    Relapsed AML

    Expected initiation: H2’21/Q1’22

    PEPAXTO (Phase 1/2)

    Relapsed high-risk NHL/DLBCL

    Expected initiation: H2’21

– Dr. Kowndinya, CI Scientists

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