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Rafael Pharmaceuticals Announces Initiation of Pivotal Phase 3 Trial (ARMADA 2000) of CPI-613 (devimistat) in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)
[November 29, 2018]

Rafael Pharmaceuticals Announces Initiation of Pivotal Phase 3 Trial (ARMADA 2000) of CPI-613 (devimistat) in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)


Newark, NJ, Nov. 29, 2018 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. (“Rafael”), a leader in the growing field of cancer metabolism-based therapeutics, today announced the initiation of a Phase 3, multicenter, open-label, randomized, pivotal trial (ARMADA 2000) to evaluate the efficacy and safety of its lead compound CPI-613 (devimistat) in combination with high dose cytarabine and mitoxantrone (CHAM) in older patients with relapsed or refractory acute myeloid leukemia (AML).

ARMADA 2000 will compare the efficacy and safety of high dose cytarabine and mitoxantrone (HAM, control arm) with CHAM.  Patients = 60 years old with relapsed or refractory AML and an ECOG performance status of 0 to 2 are eligible for enrollment. The primary efficacy endpoint will be Complete Remission (CR). Secondary endpoints include the evaluation of Overall Survival (OS) and Complete Remission with Partial Hematologic Recovery (CRh) as well as safety.

A total of 500 patients will be evaluated in ARMADA 2000 at approximately 87 sites in 12 countries. The interim analysis of the study is expected to be completed as early as Q1 2020.   More information on the trial is available at www.clinicaltrials.gov (NCT03504410).

An earlier Phase 1 dose escalation study (CCCWFU 22112) evaluated the safety, tolerability and efficacy of CHAM in patients with relapsed or refractory AML.  In total, 67 patients were enrolled and 62 were evaluable for response. The overall response rate was 50% with median survival of 6.7 months. In a subsequent Phase 2 study (CCCWFU 22215) of CHAM, a total of 48 patients were enrolled and all were evaluable for response. The overall response rate was 45% with median survival was 10.4 months.

In the combined data of these two trials, elderly patients (= 60 years) with relapsed or refractory AML dosed with 2,000 mg/m2 of CPI-613, exhibited 52% CR+CRi with median survival of 12.4 months.  In both studies, the treatment was well tolerated. (Frequent adverse events included Anemia, Neutropenia, Thrombocytopenia, Leukocytopenia and Lymphopenia) Given these results, further evaluation of CPI-613 in AML is warranted.

Howard Jonas, Chairman of Rafael Pharmaceuticals, said, “It is not only our goal to prolong life but to keep working towards a cure for AML and other difficult to treat cancers. We have great hope both for the success of this trial and even better results in years to come with our follow-on compounds. We are optimistic that our cancer metabolism drugs may create a new paradigm in oncology treatment.”

 Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals, commented, “Our motto, ‘To Save A Life Is To Save A Universe,’ reflects our commitment to develop potential treatments for patients with significant unmet clinical needs. Initiation of our ARMADA 2000 trial is a significant milestone in that direction. We believe that CPI-613 has the potential to change the course of treatment in elderly patients with AML. Given that the clinical development of CPI-613 started at Wake Forest Baptist Health, we wanted this to be the site to enroll the first patient in this Phase 3 trial. We are grateful to all the individuals that have been involved with clinical development of CPI-613.  Given the importance of this trial we have partnered with a reputable contract research organization, IQVIA, for study execution.”

Jorge Eduardo Cortes, MD, the Principal Investigator of this study commented: “This study explores a novel agent with a unique mechanism of action. The results from prior studies are very exciting and lead us to believethat CPI-613 may end up being a valuable addition to the AML armamentarium. Seeing the study started is inspiring.”



Timothy Pardee, MD, Ph.D. the Chief Medical Officer of Rafael Pharmaceuticals commented, “I am very excited to see the initiation of this phase 3 trial for fit, older, AML patients who have failed targeted or hypomethylator salvage therapy. Currently, there are limited therapeutic options for this population.”

Richard Staub, President of R&D Solutions at IQVIA, added, “We have a long-standing commitment to working with our biotech colleagues on the development of new therapies for patients with malignant disease. We are pleased that Rafael has placed their trust in us to work together on this important study.”


About CPI-613:
CPI-613 is a first-in-class drug developed based on Rafael Pharmaceuticals’ Altered Metabolism Directed (AMD) platform. CPI-613 targets altered regulation of metabolic processes specific to cancer cells. It is designed to be highly specific, simultaneously attack multiple targets, minimally toxic and have broad spectrum activity across a wide variety of cancers. CPI-613 is being or has been evaluated in 18 ongoing or completed trials as a single agent, as well as in combination with standard drug therapy for hematological malignancies and solid tumors. To date, over 300 patients have received one or more doses of CPI-613. In pancreatic cancer, CPI-613 in combination with modified FOLFIRINOX exhibited an objective response rate of 61%, median overall survival of 19.9 months and median progression free survival of 9.9 months. In elderly patients with AML, CPI-613 in combination with high dose cytarabine and mitoxantrone exhibited 52% CR+CRi and 12.4 months median overall survival.  In T-cell lymphoma, CPI-613 in combination with bendamustine exhibited a 75% overall response rate. CPI-613 has been granted orphan drug designation by the U.S. FDA for pancreatic cancer, AML, MDS, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to CPI-613 for pancreatic cancer and AML.

About Acute Myeloid Leukemia:
AML is a hematologic malignancy characterized by the accumulation of clonal myeloid progenitor cells (“blasts”) in the blood or bone marrow.1-3 Malignant hematopoietic stem and myeloid progenitor cells proliferate uncontrollably and disrupt normal hematopoiesis, resulting in impairments in the ability of these myeloid progenitor cells to differentiate.4 Current prevalence of AML in the U.S. is approximately 40,000 and mortality rates range from 25% to 75% in patients under 60 years of age and >90% in elderly patients.2 Many patients who achieve a remission and complete consolidation therapy still have a guarded prognosis. This is driven by the fact that over 50% of patients will experience a relapse, and most relapsed patients will die from AML within a year. There is no consensus standard treatment for relapsed or refractory disease, but most fit patients are treated with a high dose cytarabine (HiDAC)-based regimen.5

About Rafael Pharmaceuticals, Inc.:
Rafael Pharmaceuticals, Inc. is a privately held, clinical-stage, metabolic oncology therapeutics company committed to the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. Rafael’s primary objective is to develop innovative, highly selective, well tolerated and highly effective anti-cancer agents by selectively targeting altered metabolism in cancer cells. Rafael’s first-in-class clinical lead compound, CPI-613 is being evaluated in multiple Phase 1, 1/2, and 3 clinical studies. The Company's investors include Rafael Holdings, Inc. (NYSE American: RFL). For more information, visit http://www.rafaelpharma.com/.

References:

  1. Ferrara F, Schiffer CA. Acute myeloid leukaemia in adults. Lancet (London, England). 2013;381(9865):484-495.
  2. Gutierrez SE, Romero-Oliva FA. Epigenetic changes: a common theme in acute myelogenous leukemogenesis. Journal of hematology & oncology. 2013;6:57.
  3. Kubal T, Lancet JE. The thorny issue of relapsed acute myeloid leukemia. Current opinion in hematology. 2013;20(2):100-106.
  4. Pollyea DA, Kohrt HE, Medeiros BC. Acute myeloid leukaemia in the elderly: a review. British journal of haematology. 2011;152(5):524-542.

Disclosure:
Tim Pardee MD; Ph.D. was the site principal investigator for early Phase I/II clinical trials at Wake Forest Baptist Medical Center.
Bayard L. Powell, M.D., Section Chief, Hematology & Oncology, Associate Director for Clinical Research, Comprehensive Cancer Center, Professor, Hematology & Oncology, Wake Forest Baptist Medical Center serves as a member of Rafael Pharmaceuticals Medical Advisory Board.
Boris Pasche MD; Ph.D. Director, Comprehensive Cancer Center and Chairman, Department of Cancer Biology, Wake Forest Baptist Medical Center, serves as a Board Member to Rafael Holdings, Inc. (NYSE American: RFL) which is an investor in Rafael Pharmaceuticals, Inc.

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.

Contact
Sanjeev Luther
President & CEO Rafael Pharmaceuticals, Inc. 
sanjeev.luther@rafaelpharma.com

Jacob Jonas
Public Relations, Rafael Pharmaceuticals, Inc.
jacob.jonas@rafaelpharma.com

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