Trethera Corporation, a clinical stage biopharmaceutical company
committed to developing novel drugs targeting nucleotide metabolism
for the treatment of cancer and autoimmune diseases, announced
today that it was awarded a $2 million NIH Small Business
Innovation Research (SBIR) grant for preclinical studies treating
acute disseminated encephalomyelitis (ADEM), a neurologic disease
principally of children. Trethera’s clinical stage and
first-in-class drug, TRE-515, holds the only FDA Orphan Drug
designation for ADEM.
ADEM is an autoimmune disease that can present
with fever and difficulty walking as well as loss of consciousness
and coma. Viral infections, such as influenza, mumps, or COVID,
frequently precede the disease. ADEM is a rare disease, affecting
12,000 to 15,000 patients a year in the United States, with most
cases occurring in 6 to 8 year-old children. The grant will advance
preclinical studies to enable clinic entry for a disease where no
approved therapies currently exist.
TRE-515 is the only drug to receive an FDA
Orphan Drug designation for the treatment of ADEM, a designation
that confers substantial advantages, including FDA assistance in
designing clinical trials, access to the FDA Orphan Drug Grants
Program, exemption from the $4M drug approval application fee, and
eligibility for seven years of marketing exclusivity. Furthermore,
should the FDA approve TRE-515 for commercial use in ADEM, Trethera
would be eligible for a pediatric priority review voucher.
Combining the study data generated through this grant and the
Orphan Drug designation, Trethera plans to collaborate with the FDA
to define the most expedient clinical trial path to
commercialization.
“Achieving both NIH funding and FDA Orphan Drug
designation confers multiple external validations of our potential
to treat ADEM,” said Dr. Ken Schultz, principal investigator and
Trethera CEO. “Our team is highly motivated by the potential to
develop the first FDA approved treatment to save children’s lives
and improve outcomes in ADEM. Our strategy of combining TRE-515
commercial development for a rare disease with ongoing efforts to
treat more common diseases, such as solid tumors and Crohn’s,
provides multiple FDA approval opportunities, thereby reducing
overall development risk.”
“Approximately half of ADEM patients recover
with hospitalized intensive care. For others, ADEM can
be fatal or lead to lifelong disability. Any drug that could
improve these outcomes for patients would be groundbreaking.
TRE-515 could significantly benefit ADEM patients beyond the
available therapeutic options,” said Trethera Scientific Advisory
Board member Dr. Larry Steinman. Dr. Steinman is a distinguished
immunologist and pediatric neurologist at Stanford University.
“The pathology of ADEM involves a severe bout of
inflammation in the central nervous system that can include the
brain, spinal cord, and sometimes the optic nerves. The
inflammation damages myelin, the protective substance that coats
nerve fibers throughout the central nervous system. No medications
have been specifically approved by the FDA to treat ADEM,” said
UCLA’s Dr. Peter Clark, member of the Trethera Scientific Advisory
Board and grant co-investigator.
Figure 1: Representative stained
spinal cord sections from a mouse ADEM model. Arrows point to
regions of leukocyte infiltration.
A discussion summary from the independent panel
of NIH scientists and physicians that reviewed Trethera’s proposal
noted the “strong scientific premise and rigor…. established safety
in Phase I clinical trial for solid tumors… and commercialization
potential is high.”
Harvard’s Research Director of Neuroimmunology
and Trethera Scientific Advisory Board member, Dr. Michael Levy,
noted “The clinical treatment for an ADEM patient remains complex
on multiple levels. Treating a disease with a real mortality risk
in a vulnerable pediatric population creates a challenging role for
the attending physician especially given the lack of FDA approved
therapies. I look forward to the day TRE-515 can be FDA approved to
provide confidence to ADEM patients and their parents.”
Sources: J Neurol. 2020 Oct;267(10); Int Care
Med. 2008 Mar;34(3); Neuro. 2001 May 22;56(10)
About Trethera
Trethera is a clinical stage, privately held,
biopharmaceutical company dedicated to pioneering the development
of novel treatments for autoimmune diseases and cancers. Founded by
prominent UCLA scientists, Trethera is led by experienced
management and board members. Trethera's innovative approach to
targeting nucleotide metabolism led to the development of TRE-515,
an orally administered capsule twice designated by the FDA as an
Orphan Drug. TRE-515 is a first-in-class clinical stage drug that
inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in
the nucleoside salvage pathway, one of two biosynthetic pathways
that generate DNA precursors. It is believed that some forms of
cancer may be preferentially dependent on the salvage pathway to
support tumor growth, and certain autoimmune diseases might also
respond to TRE-515 treatment. Trethera is developing TRE-515 for
use as a monotherapy or in combination to precisely target a
metabolic vulnerability of cancer or autoimmune diseases that will
transform outcomes for patients.
For more information, please visit us at
trethera.com or e-mail Investor Relations at ir@trethera.com.
Note on Forward-Looking
Statements
All statements other than statements of historical facts
included in this press release that address activities, events or
developments that Trethera believes or anticipates will or may
occur in the future are “forward-looking statements,” which may
often, but not always, be identified by the use of such words as
"may," "might," "will," "will likely result," "would," "should,"
"estimate," "plan," "project," "forecast," "intend," "expect,"
"anticipate," "believe," "seek," "continue," "target" or the
negative of such terms or other similar expressions. Although
Trethera has a reasonable basis for the forward-looking statements
contained herein, Trethera cautions that such statements are based
on current expectations about future events and are subject to
risks, uncertainties and factors relating to medical and scientific
research, all of which are difficult to predict and many of which
are beyond Trethera’s control, that may cause actual results to
differ materially from those expressed or implied by the
forward-looking statements in this press release. These potential
risks and uncertainties include, without limitation: the extent to
which development of any novel cancer therapies or therapies for
autoimmune diseases succeeds; whether Trethera would obtain the
necessary regulatory approvals to commence human trials or
commercialize TRE-515 or any novel therapies resulting from such
research; Trethera successfully implementing its growth strategy,
including that relating to its disease therapies; the effects of
the global Covid-19 pandemic; changes in economic conditions;
competition; and risks and uncertainties applicable to the business
of Trethera. The statements in this press release speak only as of
the date hereof and Trethera does not undertake any obligation to
update, amend or clarify these forward-looking statements whether
as a result of new information, future events or otherwise. The
Company intends that all forward-looking statements be subject to
the safe-harbor provisions of the Private Securities Litigation
Reform Act of 1995.
The content of this press release is solely the views of its
authors and does not represent the official views of the NIH.
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