Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a
leader in the growing field of cancer metabolism-based
therapeutics, today announced it will initiate a Phase 2 clinical
trial of CPI-613®(devimistat) in combination with
hydroxychloroquine in patients with clear cell sarcoma of soft
tissue. In partnership with Sara’s Cure and Sarcoma Alliance for
Research through Collaboration (SARC), Rafael will begin enrolling
patients into the Simon two-stage design trial at sites across the
United States.
“Our daughter’s battle with clear cell sarcoma
was very eye opening around the lack of treatment options for
patients with clear cell sarcoma, largely due to the rarity of the
disease,” said Lennie Woods, Founder and Executive
Director of Sara’s Cure. “When we learned about Rafael’s dedication
to fighting rare diseases, we immediately connected to discuss if
devimistat can potentially be a solution in treating this disease.
We are so grateful to have a partner like Rafael that truly cares
about helping people like our daughter, and reminds our entire
community that we are not alone in this fight.”
Woods founded Sara’s Cure after Sara, her
teenage daughter, was diagnosed with clear cell sarcoma. Traveling
to the top hospitals across the country, Woods and her family
discovered how difficult it is to treat this disease, and even once
it is successfully treated, it often returns. Clear cell sarcoma is
most often found in teens and young adults in their 20s.
“We are honored to partner with Lennie and
Sara’s Cure to fight this disease and launch one of the very few
trials for clear cell sarcoma, as part of our continued mission to
provide hope for patients with hard-to-treat cancers,” said
Sanjeev Luther, President and CEO of Rafael
Pharmaceuticals. “Both Sara’s Cure and SARC share our passion in
being a voice for those battling this rare disease.”
“This study is an outstanding example of the
value of collaborative efforts among advocacy, biotech and
academia. Recognizing the need to better understand and treat clear
cell sarcoma, SARC, as an academic research consortium will
leverage the strengths of SARC to assist with this important
trial,” said Steven Young, President and CEO of
Sarcoma Alliance for Research Collaboration. SARC has been
instrumental in assisting with the study design, correlative
studies, identifying key sites to run the trial as well as
coordinating with Sara’s Cure.
“Clear cell sarcoma most often affects young
adults, but it is seen in all ages,” said Robin
Jones, M.D., of the Institute of Cancer Research and
principal investigator on the trial. “The opening of this trial not
only has the potential to provide a promising treatment option for
those affected by this disease, but it sparks the hope that many of
us have been waiting for, for a long time.”
Being part of this trial gives me hope that we
are truly turning a corner in how we treat this childhood-stealing
disease,” said Matteo Trucco, M.D., Director of
the Children’s Cancer Innovative Therapy Program of the Cleveland
Clinic. “I’m honored to be part of this journey.”
“Clear cell sarcoma is truly one of the most
challenging sarcomas to treat,” said Rashmi Chugh,
M.D., medical oncologist and Professor of Internal Medicine at
University of Michigan. “Our hope is that with the initiation of
this important study, we will learn some critical lessons about how
to successfully treat this aggressive disease.”
The Company is deeply appreciative for the many
principal investigators who will be part of this critical trial. In
the United States, sites to open include Seattle Children’s
Hospital, Indiana University Melvin and Bren Simon Cancer Center,
Cleveland Clinic Taussig Cancer Center and University of Michigan.
Additional sites are also planned to be opened in the United
Kingdom and Scotland.
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 patients’ 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, Burkitt’s lymphoma and soft
tissue sarcoma. 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), Burkitt’s and peripheral
T-cell lymphomas and soft tissue sarcoma. The Company's investors
include Rafael Holdings, Inc. (NYSE AMERICAN: RFL). For more
information, please visit www.rafaelpharma.com.
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Actual events or results may differ materially from those in the
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In light of the foregoing, readers are cautioned
not to place undue reliance on such forward-looking statements.
###
Rafael Media Contact:
Vanessa Donohue
rafael@antennagroup.com
(201) 465-8036
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