ATTRibute-CM study design, which incorporates
feedback from the FDA, has the potential to accelerate registration
with a 12-month primary endpoint of change in 6-minute walk
distance (6MWD), followed by a 30-month endpoint of mortality and
cardiovascular-related hospitalizations
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced the
initiation and design of its pivotal global Phase 3 trial
(ATTRibute-CM) of AG10 in patients with transthyretin (TTR) amyloid
cardiomyopathy (ATTR-CM). The design of the ATTRibute-CM study,
which incorporates feedback from FDA, includes two potentially
registrational endpoints. In Part A, benefit in change from
baseline in 6-minute walk distance (6MWD) will be evaluated at 12
months, potentially accelerating the time to registration. In Part
B, reduction in all-cause mortality and frequency of
cardiovascular-related hospitalizations will be evaluated at 30
months.
“ATTRibute-CM aims to generate registrational
evidence that AG10 provides meaningful benefit to ATTR-CM patients.
The trial is designed to evaluate preservation of function and
quality of life on an accelerated timeframe in addition to
evaluating longer-term benefit on mortality and
cardiovascular-related hospitalizations,” said Jonathan Fox, MD,
PhD, president and chief medical officer of Eidos. “If successful,
this trial could lead to an approval of AG10 for the treatment of
ATTR-CM, a disease with significant unmet medical need and limited
treatment options.”
The ATTRibute-CM study advances clinical
development of AG10 following positive results from a Phase 2 trial
in subjects with symptomatic ATTR-CM. In that study, AG10 was shown
to be generally well-tolerated and significantly increase serum
transthyretin, a biomarker associated with survival in a
retrospective study of ATTR-CM patients, in a dose-dependent
manner. All subjects treated with AG10 achieved normal serum
transthyretin levels within 28 days of treatment, even those whose
baseline levels were well below normal. Results of this study were
presented in a late-breaking Featured Science oral presentation at
the Annual Scientific Sessions of the American Heart Association in
November 2018. An open-label extension of this study is
ongoing.
ATTRibute-CM Design
ATTRibute-CM will 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 6MWD 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.
Investor Conference Call and Webcast
Details Eidos management will host an investor conference
call and webcast 8 am ET to review the Phase 3 trial
design. To participate in the conference call, dial +1-844-293-0174
(U.S. toll free) or +1-916-582-3546 (international), conference ID
7365928. The webcast will be available live and for replay on the
company’s website at ir.eidostx.com.
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. 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 (ATTRibute-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 transthyretin (TTR)
amyloidosis (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 registrational endpoints in the ATTRibute-CM trial, the
potential to accelerate registration of AG10, the design of the
ATTRibute-CM trial, our ability to enroll patients in and conduct
the ATTRibute-CM trial in accordance with our plans, future
clinical and regulatory milestones of AG10, 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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