Some cancers and hematological conditions can be difficult to treat. But biotechnology company Oncopeptides AB is working to address this issue for patients with relapsed or refractory (R/R) multiple myeloma. In a press release, the company shared that its therapy PEPAXTO (melphalan flufenamide), in conjunction with dexamethasone, is now FDA-approved for patients with R/R multiple myeloma who have been treated with at least four previous therapies. Additionally, their condition must be resistant to a CD-38 directed monoclonal antibody, immunomodulatory agent, and proteasome inhibitor. As a result, PEPAXTO offers a treatment option which fills an unmet need in this patient community.

PEPAXTO

So what is PEPAXTO? This anticancer peptide-drug conjugate is the first of its kind to be approved for this indication. Each month, PEPAXTO is administered as an infusion. Because it only takes about 30 minutes, it offers an efficient and accessible treatment option. The therapy binds a peptide carrier and cytotoxic agent, which then causes the release of a cytotoxic payload. Ultimately, this causes cancerous cell death.

PEPAXTO’s approval hinges, in part, from data from the Phase 2 HORIZON clinical trial. 157 patients enrolled. 40 patients had extremely aggressive cancer. During the trial, patients were treated intravenously with PEPAXTO and dexamethasone. Approximately 23.7% of patients responded to treatment. The drug combination was relatively safe and well-tolerated. However, some side effects did occur. These included:

• Fatigue
• Nausea and diarrhea
• Fever
• Respiratory tract infections
• Thrombocytopenia (low platelet count)
• Leukopenia (low leukocyte / white blood cell count)
• Lymphopenia (lowered lymphocyte counts)
• Neutropenia (low neutrophil count)
• Anemia (low red blood cell count)
• Myelodysplastic syndromes and/or acute myeloid leukemia (MDS/AML)

Altogether, PEPAXTO should be available for patients within the next 2 weeks.

Multiple Myeloma

While the exact cause of multiple myeloma is not known, researchers do know that this rare cancer forms in plasma cells, a type of white blood cell. Normally, plasma cells fight infections by creating antibodies which attack foreign invaders. However, multiple myeloma cells crowd out healthy blood cells and create high levels of M proteins, causing issues. Risk factors for multiple myeloma include age, family history, or biological sex (male). Symptoms include:

• Appetite loss
• Nausea
• Unintended weight loss
• Fatigue
• Constipation
• High calcium levels in the blood
• Excessive thirst
• Frequent infections
• Bone pain (frequently in the chest or spine)
• Confusion
• Frequent bone fractures
• Anemia (low red blood cell count)
• Thrombocytopenia (low platelet count)

Learn more about multiple myeloma here.