Rafael Pharmaceuticals Receives Approvals to Open Trial Sites in India and Canada for Pivotal Phase 3 Trial (ARMADA 2000) of CPI-613® (devimistat) in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

Patient enrollment will begin only once local health officials determine that it is safe, in light of the COVID-19 pandemic

Newark, New Jersey, UNITED STATES

Cranbury, NJ, April 23, 2020 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, today announced that the Central Drugs Standard Control Organization (CDSCO), the national regulatory body for Indian pharmaceuticals and medical devices, granted approval for the opening of sites in India for the company’s Phase 3 clinical trial in the treatment of acute myeloid leukemia (AML). The company has also received approval by Health Canada to open sites for the trial in Canada.

ARMADA 2000 is a multicenter, open-label, randomized pivotal trial for evaluating the efficacy and safety of Rafael’s lead compound CPI-613® (devimistat) in combination with high dose cytarabine and mitoxantrone (CHAM) compared with high dose cytarabine and mitoxantrone (HAM)  in older patients (50 years-old and older) with relapsed or refractory AML.

“This week, we recognized AML World Awareness Day, when the world joins forces to address the importance of supporting and advocating for better treatments for AML patients,”
said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “But at Rafael, this is our mission every single day. With over 50 activated sites, including sites in the United States, Europe, Australia and South Korea, we are continuing to expand the trial in order to ultimately deliver more effective treatments. We are now bringing our trial into India, with its population of more than 1.3 billion people, as well as to Canada, to address the significant unmet needs for AML treatments in these markets.”

In light of the COVID-19 pandemic, the sites will not immediately be open for patient enrollment. Rafael will stay in close contact with the trial sites and local health officials to determine when patients can begin enrolling safely. The safety and wellbeing of patients is Rafael’s foremost priority.

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 represent 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.


Rafael Media Contact:
Holly Dugan
(201) 465-8019