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 the
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 the 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.
Safe Harbor StatementThis press release contains
forward-looking statements. These statements relate to future
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cases, you can identify forward-looking statements by terminology
such as "may", "will", "should", "expect", "plan", "anticipate",
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result of various important factors. Although we believe that the
expectations reflected in the forward-looking statements are
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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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Rafael Media Contact:
Vanessa Donohue
rafael@antennagroup.com
(201) 465-8036
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