Acute Myeloid Leukemia Phase III Clinical Trials Activates 25 Sites and Gains Momentum with Prominent Leukemia-Focused Investigator at Helm

Oct 30, 2019 | Acute Myeloid Leukemia (AML), Bone Marrow, Cancer, Clinical Investigators, Oncology

Acute Myeloid Leukemia Phase III Clinical Trials Activates 25 Sites and Gains Momentum with Prominent Leukemia-Focused Investigator at Helm

Rafael Pharmaceuticals reported that 25 sites have been activated in the pivotal Phase III ARMADA 2000 clinical trial of CPI613® (devimistat) in older patients with relapsed or refractory acute myeloid leukemia (AML). Dr. Jorge Cortes, Director of Georgia Cancer Center at Augusta University leads as the principal investigator.

Acute Myeloid Leukemia (AML)

AML is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells or platelets. With different subtypes of AML, risk factors include smoking, previous chemotherapy treatment, and exposure to radiation according to the National Cancer Institute. With a recent estimate of 21,450 new cases per annum, those surviving 5 years currently equals 28%. Recent data shows there is approximately 62,000 living with AML in the United States and the average age of diagnosis is 68—most will pass within 5 years and many earlier.

The Study

The ARMADA 2000 Phase III study evaluates the safety and efficacy of CPI-613 in combination with High Dose Cytarabine and Mitoxantrone in comparison with high dose Cytarabine and Mitoxantrone in older patients with relapsed/refractory AML. The study commenced November 2018 and runs through till March 2023. The study sponsor seeks a total of 500 participants across 28 clinical investigational sites according to Clinicaltrials.gov.

With 25 sites up and running out of a total published target of 28 the sponsor appears to have sufficient interest from investigators for this class of treatment. The study is active in the U.S., Austria, Spain and South Korea and it would appear they will target more site openings to ensure achieving the 500 patient milestone.   

Outcome Measures

Primary Outcome Measures include Complete Remission (CR) with a time-frame of 8 months and Secondary Outcome Measures include A) Overall Survival of 12 months and B) CRh (time frame 12 months)—CR with partial hematological recovery. (CRh). This latter measure according to a recent academic report on response criteria for AML is defined as <5% blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (  platelets > 50 × 109/L and ANC > 0.5 × 109/L). 

The Principal Investigator

Dr. Jorge Cortes, a renowned clinical investigator, leads this pivotal study. When the study started Dr. Cortes was with MD Anderson however several months ago he was recruited to join Augusta University’s Georgia Cancer Center and associated Medical College of Georgia. Dr. Cortes is noted to be an international leader in leukemia research. His name is “globally synonymous with advances in the treatment of chronic and acute leukemia” reported Dean of Medical College of Georgia Dean Dr. David Hess.

The Sponsor

Rafael Pharmaceuticals is a leader in the growing field of cancer metabolism and is developing a new, first-in-class category of metabolic oncology therapeutics that attack hard-to-treat cancers by targeting the metabolic processes the disease needs to survive, grow and proliferate. The Company’s investors include Rafael Holdings, Inc. (NYSE AMERICAN: RFL).

About CPI-613 (devimistat)

CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael that targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects.

The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, and Burkitt’s lymphoma. The EMA has also granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

Call to Action

Do you or a loved one know someone in their 60s+ that has been diagnosed with Acute Myeloid Leukemia (AML)? If so, at least review this study and consider having their oncologist look at this study for purposes of relevance of participation.

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