AG10 demonstrated equivalent, near-complete stabilization of TTR
in wild-type and mutant subjects with TTR amyloid cardiomyopathy
(ATTR-CM).
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), today announced the
publication of its Phase 2 clinical trial studying AG10 in subjects
with symptomatic TTR amyloid cardiomyopathy (ATTR-CM) in the
Journal of the American College of Cardiology (JACC) entitled
“Transthyretin Stabilization by AG10 in Symptomatic Transthyretin
Amyloid Cardiomyopathy”.
Eidos also presented subgroup analyses from the same Phase 2
clinical study in an oral presentation at this year’s American
College of Cardiology (ACC) Scientific Sessions. Stephen Heitner,
M.D., director of the hypertrophic cardiomyopathy clinic at the
Oregon Health & Science University, presented these findings in
an oral presentation entitled “AG10 Consistently Stabilizes
Transthyretin to a High Level in Both Wild Type and Mutant Amyloid
Cardiomyopathy: Responder Analyses From a Phase 2 Clinical
Trial”.
“These data confirm the potential ability of AG10 to treat
ATTR-CM, regardless of mutational status,” said Jonathan Fox, MD,
PhD, president and chief medical officer of Eidos. “The observed
near-complete stabilization of TTR in all actively treated subjects
and across the dosing interval provide clinical proof-of-concept
for AG10 in both wild-type and mutant ATTR-CM. These Phase 2
results support further investigation of AG10 in the ongoing
ATTRibute-CM Phase 3 study.”
The primary results from the Phase 2 study were presented at the
2018 American Heart Association (AHA) Annual Meeting in Chicago, IL
on November 10, 2018.
About ATTRibute-CM
ATTRibute-CM (NCT03860935) is expected to enroll approximately
510 subjects with symptomatic ATTR-CM, associated with either
wild-type or mutant TTR, with New York Heart Association Class
I-III symptoms. Subjects will be randomized 2:1 between treatment
(AG10 800 mg) and placebo twice daily. In Part A, change in
six-minute walk distance at 12 months will be compared between
treatment and placebo groups as the first registrational primary
endpoint. In Part B, the study will continue for a total duration
of 30 months, at which point all-cause mortality and frequency of
cardiovascular-related hospitalizations will be compared between
treatment and control groups. Secondary endpoints include quality
of life as assessed by the Kansas City Cardiomyopathy
Questionnaire, safety parameters, serum TTR levels, and TTR
stabilization. In Part B, concomitant use of approved, indicated
therapies may be allowed. The design of ATTRibute-CM was reviewed
with the U.S. Food and Drug Administration (FDA) and with the
European Medicines Agency (EMA).
About AG10
AG10 is an investigational, orally-administered small molecule
designed to potently stabilize TTR thereby halting at its outset
the series of molecular events that give rise to ATTR. In a Phase 2
clinical trial in subjects with symptomatic ATTR-CM, AG10 was
generally well tolerated, demonstrated >90% average TTR
stabilization at day 28, and increased serum TTR concentrations, a
prognostic indicator of survival in a retrospective study of
ATTR-CM patients, in a dose-dependent manner. AG10 is currently
being studied in an open-label extension of a Phase 2 clinical
trial in patients with ATTR-CM and sites are currently being
activated for a Phase 3 clinical trial of AG10 in patients with
ATTR-CM.
AG10 was designed to mimic a naturally-occurring variant of the
TTR gene (T119M) that is considered a rescue mutation because
co-inheritance has been shown to prevent ATTR in individuals also
inheriting a pathogenic, or disease-causing, mutation in the TTR
gene. To our knowledge, AG10 is the only TTR stabilizer in
development that has been observed to mimic the stabilizing
structure of this rescue mutation.
About transthyretin amyloidosis (ATTR)
ATTR represents a significant unmet medical need with a large
patient population and an inadequate current standard of care. ATTR
is caused by the destabilization of TTR due to inherited mutations
or aging and is commonly divided into three distinct categories:
wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR
cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The
worldwide prevalence of each disease is approximately 400,000
patients, 40,000 patients and 10,000 patients, respectively.
All three forms of ATTR are progressive and fatal. For patients
with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in
life (age 50+), with median survival of three to five years from
diagnosis. ATTR-PN either presents in a patient's early 30s or
later (age 50+), and results in a median life expectancy of five to
ten years from diagnosis. Progression of all forms of ATTR causes
significant morbidity, impacts productivity and quality of life,
and creates a significant economic burden due to the costs
associated with progressively greater patient needs for supportive
care.
About Eidos Therapeutics
Eidos Therapeutics is a clinical stage biopharmaceutical company
focused on addressing the large and growing unmet need in diseases
caused by ATTR. Eidos is developing AG10, a potentially
disease-modifying therapy for the treatment of 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
potential therapeutic and clinical benefits of AG10, the potential
for AG10 to be a safe and effective treatment for all forms of
ATTR-CM, the potential registrational endpoints in the ATTRibute-CM
trial, the design of the ATTRibute-CM trial, our ability to enroll
patients in and conduct the ATTRibute-CM trial in accordance with
our plans, the timing of these events, the indications we intend to
pursue and our possible clinical or other business strategies, 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 AG10 through current and future milestones, our
ability to raise additional funding to complete the development of
AG10, our dependence on the success of AG10, our ability to enroll
patients in the ATTRibute-CM trial, results from our clinical
trials and pre-clinical studies and those of third
parties working in the same area as our product candidate, our
ability to advance AG10 in clinical development in accordance with
our plans, 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 September 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
Communications619-849-5382Carolyn@canalecomm.com
Investor Contact:Alex GrayBurns
McClellan212-213-0006agray@burnsmc.com
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