Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for the Treatment of Acute Myeloid Leuk...
December 15 2020 - 9:00AM
Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader
in the growing field of cancer metabolism-based therapeutics,
announced today that the U.S. Food and Drug Administration (FDA)
has granted Fast Track designation for the Company’s lead compound,
CPI-613® (devimistat), for the treatment of acute myeloid leukemia
(AML).
“Receiving Fast Track designation, especially
during a pandemic that has created significant challenges for many
trials across the globe, is a testament to the dedicated work of
the Rafael team,” said Sanjeev Luther, President
and CEO of Rafael. “We would not be here without the support of the
FDA, our doctors, our patients, and all who are invested in the
hope of finding a successful treatment for this hard-to-treat
cancer.”
While one of the most common types of leukemia
in adults, AML accounts for only 1% of all cancers.
“This designation underscores the pressing need
to find new ways to combat this aggressive disease,” said
Jorge Cortes, M.D., Director of the Georgia Cancer
Center at Augusta University and principal investigator on the
Phase 3 clinical trial. “It brings hope not only to clinicians, but
to patients who hear that they have been diagnosed.”
“We would not be here today without our
principal investigator Jorge Cortes, M.D., the FDA, and leadership
at Rafael who remain focused on patient care,” said Timothy
S. Pardee, M.D., Ph.D., Co-Chief Medical Officer of the
Company. “We are a community coming together to fight a common
enemy, and I believe we are gaining ground in this battle every
day.”
This announcement comes on the heels of the
Company receiving Fast Track designation for devimistat for the
treatment of metastatic pancreatic cancer, in November. The company
has continued to reach milestones throughout the year, including
the recent Orphan Drug Designation for the treatment of soft tissue
sarcoma for devimistat, and the initiation of a Phase 2 clinical
trial of devimistat in combination with hydroxychloroquine in
patients with clear cell sarcoma of soft tissue.
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, 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) and Burkitt’s,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 events or the company’s future financial
performance. In some cases, you can identify forward-looking
statements by terminology such as "may", "will", "should",
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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
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###
Rafael Media Contact:
Vanessa Donohue
rafael@antennagroup.com
(201) 465-8036
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