by Blood Cancer United
WASHINGTON – The U.S. Food and Drug Administration has approved ziftomenib (Komzifti™) for the treatment of adults with an advanced form of acute myeloid leukemia (AML) with a mutation in a gene called NPM1.
About three in 10 people with newly diagnosed AML have the NPM1 mutation. Seven in 10 will experience relapse within three years, most within the first year.
In a clinical trial, 21.4% of patients treated with ziftomenib went into a full remission or remission with partial recovery of blood counts, which means there were no signs of cancer cells in the bone marrow, but not all blood counts had returned to normal. The median duration of the response was 5 months with a range of 1.9 to 8.1 months.
“People with all forms of AML, including those with the NPM1 mutation, are facing a serious form of blood cancer with limited treatment options, making it crucial that we continue to find new, effective treatments like ziftomenib” says Lore Gruenbaum, Ph.D., Chief Scientific Officer of Blood Cancer United®—formerly The Leukemia & Lymphoma Society.
Ziftomenib is the first and only once-daily oral menin inhibitor approved for these patients. Many of these patients are older and cannot tolerate other available treatments, including intensive chemotherapy or stem cell transplants.
Years of research have shown that AML is not just one disease. There are more than 10 types of AML and other rare mutations. Dr. Gruenbaum explained that different treatments will likely be needed to target each type of AML and help more patients.
A long-term commitment to improving AML treatment
Blood Cancer United has more research funding committed to AML than to any other form of blood cancer. This includes decades of funding that supported the scientific development of ziftomenib and menin inhibitor drugs in general. The menin inhibitor revumenib recently received a second FDA approval for the treatment of AML.
In the early 2000s, Blood Cancer United provided funding to a group of scientists who identified the role of the menin complex in the devastating effects of certain forms of leukemia.
Over the next several years, Blood Cancer United provided more than $6 million through its venture philanthropy initiative, the Therapy Acceleration Program® (TAP), to scientists at the University of Michigan. They discovered compounds that could inhibit the interaction of two proteins, menin and KMT2A/MLL, that together drive progression of AML.
With TAP funding, the scientists demonstrated the activity of their compounds in the laboratory. To facilitate translation of the scientists’ progress in the laboratory to patients, Blood Cancer United introduced the scientists to Kura Oncology, a company with the right skills to keep the research moving forward.
“What you see in this stream of funding from Blood Cancer United is how we support research at every stage, helping to accelerate new treatments for the patients who need them,” says Dr. Gruenbaum. “It’s not just a financial investment either. We also share our expertise based on decades of leadership in blood cancer. We will never stop in our quest for new safe and effective treatments.”
In addition to past support through grants and the TAP program, Blood Cancer United together with Kura Oncology is now evaluating ziftomenib in infants and children in the Blood Cancer United Pediatric Acute Leukemia (PedAL) Master Clinical Trial.
PedAL is the first integrated, global, pediatric acute leukemia master clinical trial. PedAL is designed to connect children with new and more effective treatments and accelerate future medical advances.
Cancer treatments are usually evaluated in adults first and if successful, then children. This has resulted in a nearly decade-long delay between when therapies are first available for adults compared to children. PedAL is changing the paradigm, providing the framework that has reduced the gap between the start of testing in adults and children.
About the Blood Cancer United Therapy Acceleration Program® (TAP)
TAP is a strategic venture philanthropy initiative that seeks to accelerate the development of innovative blood cancer therapeutics and change the standard of care. TAP partners with biotech companies to support the development of novel platforms, first-in-class assets addressing unmet medical needs, emerging patient populations, and even rare blood cancers. TAP has an open partnering cycle all year long and accepts applications from companies with innovative science that has high potential to improve patient lives. To learn more, visit https://bloodcancerunited.org/tap.