ChromaDex plans to conduct additional studies
on NRC in anticipation of filing for an Investigational New Drug
Application (IND) for the treatment of AT
ChromaDex Corp. (NASDAQ:CDXC), the global authority on
nicotinamide adenine dinucleotide (NAD+) and healthy aging
research, today announced that the U.S. Food & Drug
Administration (FDA) granted Orphan Drug Designation (ODD) and Rare
Pediatric Disease (RPD) Designation for NRC, the company’s product
candidate for the treatment of Ataxia Telangiectasia (AT). Plans
are underway to file an Investigational New Drug (IND) application
with the U.S. FDA in anticipation of conducting human clinical
trials, which will be guided by Dr. Vilhelm (Will) Bohr, Prof.,
University of Copenhagen and Scientific Advisor to ChromaDex.
“Over 30 million people in the U.S. are impacted by more than
7,000 rare diseases, many of which are life-threatening and lack
effective treatments,” said Rob Fried, CEO of ChromaDex. “We
believe NRC has potential as a treatment for AT.”
“AT is a condition where children suffer from the adverse
effects of premature aging and face a very limited life
expectancy,” said Dr. Andrew Shao, ChromaDex Senior Vice President
of Global Scientific & Regulatory Affairs. “We are excited to
continue supporting the AT community and eagerly anticipate the
results from future research.”
About Ataxia Telangiectasia (AT)
Ataxia Telangiectasia (AT) is a rare, progressive disease that
typically presents in early childhood and is characterized by
neurological and immunological symptoms. Those with AT often
exhibit an unsteady gait (ataxia), impaired coordination of eye
movements (oculomotor apraxia), and involuntary movements
(choreoathetosis). AT leads to cerebellar degeneration and many
affected children become wheelchair-dependent. Currently, there is
no cure or FDA-approved treatment to slow the progression of AT,
with the average life expectancy being around 25 years for those
diagnosed in childhood.
There are many types of ataxia. Another form of ataxia is
Friedreich’s ataxia (FA), which is being addressed by Biogen and
Larimar Therapeutics. FA impacts 1 in 50,000 people in the U.S.
(NIH). Ataxia Telangiectasia (AT) impacts roughly 1 in 40,000
people in the U.S. (Riboldi et al., 2023; Tieve et al., 2015).
Clinical Research on NRC and AT
To date, NRC has been investigated in two third-party funded,
peer-reviewed published clinical trials for the treatment of AT.
The first study published in Movement Disorders demonstrated that
supplementation with NRC improved AT scores and increased
immunoglobulins, or antibodies, in the immune-compromised patients,
with AT score improvements reversing once supplementation concluded
(Veenhius et al., 2021).
The second phase II two-year long study, also published in
Movement Disorders, demonstrated that long-term NRC supplementation
increased whole blood NAD+ levels up to fourfold, and improved
neuromotor coordination and eye movements in 90% of participants
while maintaining biomarkers of stable liver and kidney function,
as compared to historical disease progression (Presterud et al.,
2023). Both studies reported no serious adverse events, with NRC
being generally well-tolerated.
Not associated with ChromaDex’s future NRC IND filing, there are
two additional investigator-initiated ongoing registered clinical
trials that will examine NR supplementation in AT patients. The
first is a continuation of Presterud et al. 2023 and will track AT
patients over the course of 8-10 years. Recently registered, the
second will be a single-arm open-label clinical trial scheduled to
commence this year in Australia.
Significance of Orphan Drug Designation (OOD) and Rare
Pediatric Disease Designation (RPD)
According to the FDA, there are too few treatments for rare
diseases because of high research and development costs, which
companies often cannot recoup as a result of small patient
populations. To incentivize companies to invest in bringing
treatments to market for rare diseases, in 1983, Congress passed
the Orphan Drug Act, which makes Orphan Drug Designation (ODD)
candidates eligible for tax credits, waives their user fees, and
may provide a period of exclusivity should the orphan drug be
subsequently approved by FDA.
Related to ODD, the FDA’s Rare Pediatric Disease (RPD)
designation, further incentivizes companies to invest in rare
childhood diseases by providing a voucher program to applicants
approved by September 30th, 2024. Through this program, companies
with RPD designation that ultimately obtain successful drug
approval for a rare pediatric disease are provided a voucher, which
can be used to expedite the FDA review of another drug candidate or
sold to other companies.
Future Clinical Trials on NRC and AT
With both designations granted, ChromaDex plans to file an
Investigational New Drug (IND) application for future human
clinical trials for the use of NRC in the treatment of AT. As a
leading expert on the cell biology and biochemistry of AT, Dr. Bohr
will serve ChromaDex as an advisor through this process.
Dr. Bohr remarked, “This is a significant step towards providing
treatment for AT, a disease with no cure. It is an honor to work as
an advisor on such pivotal research, and we are committed to
advancing the science behind NRC to meet the urgent needs of this
rare disease community.”
“We are excited at the thought of embarking on these important
future clinical trials for NRC as we are committed to advancing
this to provide hope and relief for those suffering from this
debilitating disease,” commented Dr. Susan Perlman, MD, Clinical
Professor of Neurology and Director of the Ataxia Center at the
UCLA Medical Center in Los Angeles, and ChromaDex Clinical
Consultant.
For additional information on the science supporting NRC and for
future updates visit www.chromadex.com.
Forward-Looking Statements:
This release contains forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933, as amended,
and Section 21E of the Securities and Exchange Act of 1934.
Statements that are not a description of historical facts
constitute forward-looking statements and may often, but not
always, be identified by the use of such words as “expects,”
“anticipates,” “intends,” “estimates,” “plans,” “potential,”
“possible,” “probable,” “believes,” “seeks,” “may,” “will,”
“should,” “could” or the negative of such terms or other similar
expressions, and include the statements regarding the potential
benefits and development of NRC as a treatment for AT or other
diseases, including statements regarding clinical trials and
obtaining IND Designation from the FDA. These forward-looking
statements are based on the Company’s current expectations and are
subject to risks and uncertainties that may cause actual results to
differ materially, including unanticipated developments in and
risks related to: the ability to continue to pursue additional
studies, human trials, and to obtain an IND Designation from the
FDA; whether the potential benefits of NRC can be further
supported; further research and development and the results of
clinical trials possibly being unsuccessful or insufficient to meet
applicable regulatory standards or warrant continued development;
the ability to enroll sufficient numbers of subjects in clinical
trials; determinations made by the FDA and other governmental
authorities; our ability to maintain sales, marketing and
distribution capabilities; changing consumer perceptions of our
products; our reliance on a single or limited number of third-party
suppliers; and the risks and uncertainties associated with our
business and financial condition. More detailed information about
ChromaDex and the risk factors that may affect the realization of
forward-looking statements is set forth in ChromaDex's Annual
Report on Form 10-K for the fiscal year ended December 31, 2023,
ChromaDex's Quarterly Reports on Form 10-Q and other filings
submitted by ChromaDex to the SEC, copies of which may be obtained
from the SEC's website at www.sec.gov. Readers are cautioned not to
place undue reliance on these forward-looking statements, which
speak only as of the date hereof, and actual results may differ
materially from those suggested by these forward-looking
statements. All forward-looking statements are qualified in their
entirety by this cautionary statement and ChromaDex undertakes no
obligation to revise or update this release to reflect events or
circumstances after the date hereof.
About ChromaDex:
ChromaDex Corp. (NASDAQ:CDXC) is the global authority on
nicotinamide adenine dinucleotide (NAD+), with a focus on the
science of healthy aging. The ChromaDex team, comprised of
world-renowned scientists, works with independent investigators
from esteemed universities and research institutions around the
globe to uncover the full potential of NAD+. A vital coenzyme found
in every cell of the human body, NAD+ declines with age and
exposure to other everyday stressors. NAD+ depletion is a
contributor to age-related changes in health and vitality.
Setting the benchmark as the gold standard in scientific rigor,
safety, quality, and transparency, ChromaDex is the innovator
behind its clinically proven flagship ingredient, Niagen® (patented
nicotinamide riboside, or NR), the most efficient and
superior-quality NAD+ booster available.
ChromaDex’s robust patent portfolio protects NR or nicotinamide
riboside chloride (NRC) and other NAD+ precursors. ChromaDex
maintains a website at www.chromadex.com, to which ChromaDex
regularly publishes copies of its press releases, news, and
financial information.
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version on businesswire.com: https://www.businesswire.com/news/home/20240607042855/en/
ChromaDex Media Contact: Kendall Knysch, Senior Director
of Media Relations & Partnerships 310-388-6706 ext. 689
kendall.knysch@chromadex.com
ChromaDex Investor Relations Contact: Ben Shamsian Lytham
Partners 646-829-9701 shamsian@lythampartners.com
ChromaDex (NASDAQ:CDXC)
過去 株価チャート
から 12 2024 まで 1 2025
ChromaDex (NASDAQ:CDXC)
過去 株価チャート
から 1 2024 まで 1 2025
