01/09/2026
A new oral drug could offer hope for people facing one of the most aggressive forms of multiple myeloma.
Some people with multiple myeloma have a genetic change called t(4;14). This mutation is associated with faster disease progression and fewer treatment options over time. Until now, there have been no therapies that directly target this genetic driver, leaving many patients with limited options after relapse.
At the 2025 American Society of Hematology Annual Meeting, IMF's Scientific Advisory Board member Dr. Saad Usmani shared updated results from a Phase 1 clinical trial studying KTX-1001, a first-in-class oral therapy designed for high-risk multiple myeloma. KTX-1001 was developed to specifically shut down the activity of the protein that fuels cancer growth in myeloma with the t(4;14) mutation. This makes it the first drug designed to target this high-risk driver at its source.
Even in this heavily treated group, researchers observed encouraging signs of anti-myeloma activity. About 40 percent of participants achieved disease control, meaning their cancer responded or remained stable, and some responses lasted a year or longer.
Because these early results are encouraging, the research is now moving forward to study KTX-1001 in combination with other therapies, including carfilzomib and the investigational drug mezigdomide. These next steps will help determine how this therapy may strengthen existing treatments and improve outcomes for people with high-risk disease.
For people living with relapsed or refractory multiple myeloma, especially those with the t(4;14) mutation, treatment options often become more limited over time. A targeted oral therapy that addresses the genetic driver of the disease could represent an important step forward and help people live longer.
This is the type of research the International Myeloma Foundation champions through our Scientific Advisory Board, the International Myeloma Working Group, and in close partnership with leaders like Dr. Saad Usmani. Together, we help guide the science that shapes care for people with myeloma worldwide.
To learn more about the latest research directly from the IMF Scientific Advisory Board, visit: https://mmsm.link/4boNwxd
