FDA Grants Orphan Drug Designation to Eidos Therapeutics’ Product Candidate, AG10, for Treatment of Transthyretin Amyloidos...
October 03 2018 - 2:35PM
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage
biopharmaceutical company focused on addressing the large and
growing unmet need in diseases caused by transthyretin (TTR)
amyloidosis (ATTR), announced today that the U.S. Food and Drug
Administration (FDA) has granted the company Orphan Drug
Designation for AG10 for the treatment of ATTR.
TTR normally circulates in the blood as a four-part molecule or
tetramer, but in ATTR the tetramer destabilizes and dissociates
into individual monomers which aggregate as amyloid fibrils. AG10,
Eidos’ lead product candidate, is designed to target ATTR at its
source by stabilizing tetrameric TTR in the blood. Eidos is
investigating AG10 for the treatment of ATTR cardiomyopathy
(ATTR-CM) and ATTR polyneuropathy (ATTR-PN), both of which are
progressive, fatal diseases. The company plans to initiate Phase 3
studies in each indication in the first half of 2019.
“The granting of Orphan Drug status to Eidos’ lead development
candidate, AG10, for treating transthyretin amyloidosis is an
important step forward for the product and our company,” said
Jonathan Fox, M.D., Ph.D., president and chief medical officer of
Eidos. “We believe that AG10 holds great promise as a
disease-modifying therapy for patients with ATTR, and Orphan status
will help us develop it as quickly as possible.”
Orphan Drug Designation provides incentives and support for the
development of drugs for patients with rare diseases, specifically
to drugs that are intended for the treatment of diseases or
conditions that affect fewer than 200,000 people in the United
States. Orphan Drug Designation provides certain benefits,
including seven years of market exclusivity upon regulatory
approval, exemption from FDA application user fees and tax credits
for qualified clinical trial expenses.
About AG10
AG10 is an investigational, orally-administered small molecule
designed to potently stabilize tetrameric transthyretin, or TTR,
thereby halting at its outset the series of molecular events that
give rise to amyloidosis, or ATTR. AG10 is currently being examined
in a Phase 2 clinical trial in patients with ATTR cardiomyopathy.
Top-line results from this trial are expected to be reported by the
end of 2018.
AG10 was designed to mimic a naturally-occurring variant of the
TTR gene (T119M) that is considered a “rescue mutation” because it
has been shown to prevent ATTR in individuals carrying pathogenic,
or disease-causing, mutations in the TTR gene. To our knowledge,
AG10 is the only TTR stabilizer in development that has been
observed to mimic the “super-stabilizing” properties of this rescue
mutation.
About Eidos Therapeutics
Eidos Therapeutics is a clinical stage biopharmaceutical company
focused on addressing the large and growing unmet need in diseases
caused by transthyretin (TTR) amyloidosis (ATTR). For more
information, please visit www.eidostx.com.
Forward-Looking Statements
This release includes forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933 and Section
21E of the Securities Exchange Act. All statements other than
statements of historical facts, including the statements about the
clinical and therapeutic potential and future clinical milestones
of AG10, including the initiation of Phase 3 clinical trials, the
potential for AG10 to capture any expected benefits from Orphan
Drug Designation, the indications we intend to pursue and our
possible clinical or other business strategies, and the timing of
these events, are forward-looking statements. Forward-looking
statements can be identified by terms such as “believes,”
“expects,” “plans,” “potential,” “would” or similar expressions and
the negative of those terms. These forward-looking statements are
based on our management’s current beliefs and assumptions about
future events and on information currently available to
management.
Forward-looking statements involve known and unknown risks,
uncertainties and other factors that may cause our actual results,
performance or achievements to be materially different from any
future results, performance or achievements expressed or implied by
the forward-looking statements. These risks include, but are not
limited to, risks and uncertainties related to: our limited
operating history and historical losses, our liquidity to fund the
development of our other product candidates through current and
future milestones, our ability to raise additional funding to
complete the development and any commercialization of our product
candidates, our dependence on the success of our lead product
candidate, AG10, results from our clinical trials
and pre-clinical studies and those of third parties
working in the same area as our product candidate and our
dependence on third parties in connection with our manufacturing,
clinical trials and pre-clinical studies. Additional
risks and uncertainties that could affect our future results are
included in the section titled “Risk Factors” and “Management’s
Discussion and Analysis of Financial Condition and Results of
Operations” in our Quarterly Report on Form 10-Q for the quarter
ended June 30, 2018, which is available on the SEC’s website
at www.sec.gov and our website at eidostx.com. Additional
information on potential risks will be made available in other
filings that we make from time to time with the SEC. In addition,
any forward-looking statements contained in this press release are
based on assumptions that we believe to be reasonable as of this
date. Except as required by law, we assume no obligation to update
these forward-looking statements, or to update the reasons if
actual results differ materially from those anticipated in the
forward-looking statements.
Media Contact:
Carolyn Hawley, Canale Communications, (619) 849-5382,
carolyn@canalecomm.com
Investor Contact:
Alex Gray, Burns McClellan, (212) 213-0006,
agray@burnsmc.com
Eidos Therapeutics (NASDAQ:EIDX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Eidos Therapeutics (NASDAQ:EIDX)
Historical Stock Chart
From Apr 2023 to Apr 2024