The designation is intended to accelerate a candidate therapy’s development and expedite its approval.
“Receiving Fast Track designation, especially during a pandemic that has created significant challenges for many trials across the globe, is a testament to the dedicated work of the Rafael team,” Sanjeev Luther, company president and CEO, said in a press release.
“We would not be here without the support of the FDA, our doctors, our patients, and all who are invested in the hope of finding a successful treatment for this hard-to-treat cancer,” he added.
CPI-613 is a first-in-class molecule that blocks enzymes that play a role in the metabolism of cancer cells. These enzymes are located in mitochondria, which generates most of the energy needed to power biochemical reactions in cells, by converting nutrients into energy for normal cell functioning.
In cancer cells, changes in the regulation of these enzymes cause the mitochondria to shift their function and convert nutrients into the building blocks of the next cancer cell. By targeting these enzymes, CPI-613 is thought to inhibit mitochondria in cancer cells, impairing their ability to resist chemotherapy.
The dual action of CPI-613 together with chemotherapy suggests it could be used to lower the chemotherapy doses currently used, potentially reducing adverse events experienced by patients.
The open-label Phase 3 ARMADA 2000 trial (NCT03504410) is investigating a combination of CPI-613 plus high-dose chemotherapy (a mix of cytarabine and mitoxantrone) versus high-dose chemotherapy alone in patients, ages 50 and older, with relapsed or refractory AML. The trial is currently recruiting, and more information can be found here.
The trial’s main (primary) goal is to assess whether CPI-613 plus chemotherapy increases the number of patients achieving a complete response (or the complete disappearance of all cancer signs), compared with the chemotherapy alone. Additional (secondary) measures include overall survival and complete response rate, with or without full recovery of blood cell counts.
Top-line findings of the study, which has enrolled more than half of the participants for a first interim analysis, are expected by October 2022.
The fast track designation “underscores the pressing need to find new ways to combat this aggressive disease,” said Jorge Cortes, MD, director of the Georgia Cancer Center at Augusta University and principal investigator of the trial. “It brings hope not only to clinicians, but to patients who hear that they have been diagnosed.”
CPI-613 was also recently granted fast track designation for the treatment of metastatic pancreatic cancer. It’s been named an orphan drug for the treatment of AML, pancreatic cancer, myelodysplastic syndrome, peripheral T-cell lymphoma, Burkitt lymphoma, and soft tissue sarcoma, which is a rare type of cancer affecting the tissues that support several of the body’s structures.
“We would not be here today without our principal investigator Jorge Cortes, M.D., the FDA, and leadership at Rafael who remain focused on patient care,” said Timothy S. Pardee, MD, PhD, co-chief medical officer of Rafael. “We are a community coming together to fight a common enemy, and I believe we are gaining ground in this battle every day.”
We are sorry that this post was not useful for you!
Let us improve this post!
Tell us how we can improve this post?