Rafael Pharmaceuticals Crosses Enrollment of 100th Patient in Pivotal Phase 3 Trial (ARMADA 2000) of CPI-613® (Devimistat) for Relapsed or Refractory Acute Myeloid Leukemia (AML)

Rhea-AI Summary

Rafael Pharmaceuticals announced significant progress in its Phase 3 clinical trials, surpassing 100 patient enrollments in the ARMADA 2000 trial for relapsed or refractory acute myeloid leukemia (AML). This pivotal trial evaluates the efficacy of CPI-613® (devimistat) combined with high-dose cytarabine and mitoxantrone. Rafael has also enrolled 500 patients in a parallel trial for advanced pancreatic cancer. The company emphasizes patient safety during the COVID-19 pandemic, with active trials in over 60 sites globally. The FDA and EMA have granted orphan drug designation to devimistat for these conditions.

Positive

• Surpassed 100 patient enrollments in the ARMADA 2000 Phase 3 trial for AML.
• Achieved milestone of 500 enrollments in advanced pancreatic cancer trial.
• CPI-613® (devimistat) is being evaluated in multiple pivotal trials.
• FDA and EMA granted orphan drug designation for AML and pancreatic cancer.

Negative

• None.

Company continues meeting enrollment milestones across clinical trials amid the COVID-19 pandemic, putting patient safety first

CRANBURY, N.J., Oct. 27, 2020 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, announced today that it has crossed the enrollment of 100 patients in its Phase 3 clinical trial (Armada 2000) for patients with relapsed or refractory acute myeloid leukemia (AML). The multicenter, open-label, randomized pivotal trial is assessing the efficacy and safety of Rafael’s lead compound CPI-613® (devimistat) in combination with high dose cytarabine and mitoxantrone (CHAM) compared to high dose cytarabine and mitoxantrone (HAM) therapy in older patients. The announcement comes on the heels of the Company’s milestone enrollment of 500 patients in its Phase 3 clinical trial for advanced pancreatic cancer.  

“Our patients don’t have the time to wait for the pandemic to subside before seeking treatment since cancer doesn’t wait, and for many, this trial is their last chance at remission or improved outcomes,” said Sanjveev Luther, President and CEO of Rafael Pharmaceuticals. “We thank our colleagues and our partners who helped us achieve this significant milestone, and the FDA for working with us to find a process that allows us to safely keep our clinical trials open to care for these patients, and we will continue to implement the safety protocols moving forward.”  

The American Cancer Association estimates that in 2020 alone, there will be nearly 20,000 new cases of AML. AML accountly for only 1% of all cancers making the patient population rare and more difficult to understand and treat.   

“The five-year survival rate for adult patients with refractory or relapsed AML is 25% or less, and treatment options that have made a significant impact in improving these rates, have not been identified,” said Jorge Cortes, M.D., Director of the Georgia Cancer Center at Augusta University and principal investigator on the Phase 3 clinical trial. “I continue to remain hopeful with each patient enrolled that we can give those diagnosed more options to fight this devastating disease.”  

“We are grateful to have Dr. Cortes leading this trial,” said Timothy S. Pardee, M.D., Ph.D., Co-Chief Medical Officer of the Company. “It is largely due to his extensive background in clinical research that we have been able to continue meeting milestone enrollments like this, while still taking every precaution necessary to conduct the study safely during the pandemic.”  

The clinical trial is currently active in more than 60 sites, running in countries including the United States, Canada, Europe, South Korea and Australia.  

About CPI-613® (devimistat)  
CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which 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. Combination with devimistat represents a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. 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 granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia. 

About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals is a leader in the growing field of cancer metabolism. The company 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. Rafael Pharmaceuticals’ lead compound, CPI-613® (devimistat), is a highly selective, well-tolerated and effective anti-cancer agent that is being evaluated in ongoing and completed Phase 1, 2 and 3 clinical trials. Devimistat has been granted orphan drug status by the FDA for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and Burkitt’s and peripheral T-cell lymphomas. The Company's investors include Rafael Holdings, Inc. (NYSE AMERICAN: RFL). For more information, please visit www.rafaelpharma.com.   

Safe Harbor Statement
This press release contains forward-looking statements. These statements relate to future events or the company’s future financial performance. In some cases, you can identify forward-looking statements by terminology such as "may", "will", "should", "expect", "plan", "anticipate", "believe", "estimate", "predict", "potential" or "continue", the negative of such terms, or other comparable terminology. These statements are only predictions. Actual events or results may differ materially from those in the forward-looking statements as a result of various important factors. Although we believe that the expectations reflected in the forward-looking statements are reasonable, such statements should not be regarded as a representation by the company, or any other person, that such forward-looking statements will be achieved. The business and operations of the company are subject to substantial risks which increase the uncertainty inherent in forward-looking statements. We undertake no duty to update any of the forward-looking statements, whether as a result of new information, future events or otherwise.  In light of the foregoing, readers are cautioned not to place undue reliance on such forward-looking statements. 

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FAQ

What is the purpose of Rafael Pharmaceuticals' ARMADA 2000 trial?

The ARMADA 2000 trial assesses the efficacy and safety of CPI-613® (devimistat) in combination with chemotherapy for patients with relapsed or refractory acute myeloid leukemia.

How many patients have been enrolled in the ARMADA 2000 trial?

More than 100 patients have been enrolled in the ARMADA 2000 trial.

What is CPI-613® (devimistat) used for?

CPI-613® (devimistat) is used to target cancer cell metabolism and is being tested in trials for various cancers, including AML and pancreatic cancer.

What is the significance of orphan drug designation for devimistat?

Orphan drug designation helps facilitate the development of treatments for rare diseases, offering benefits like tax credits and market exclusivity.

Where are the clinical trials for Rafael Pharmaceuticals currently active?

The clinical trials are active in over 60 sites across countries including the United States, Canada, Europe, South Korea, and Australia.