AG10 Was Well Tolerated in Subjects Administered 400 mg
or 800 mg Twice Daily for 28 Days
Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced
positive results of its Phase 2 clinical trial studying AG10 in
subjects with symptomatic transthyretin (TTR) amyloidosis
cardiomyopathy (ATTR-CM). The data were presented in a
late-breaking Featured Science oral presentation at the American
Heart Association (AHA) Scientific Sessions. AG10 was well
tolerated, demonstrated >90% TTR average stabilization at day
28, and increased serum TTR concentrations, a prognostic indicator
of survival in ATTR-CM, in a dose-dependent manner. Subject to
discussions with regulatory agencies, these data support the
advancement of AG10 into Phase 3 pivotal trials planned to be
initiated in the first half of 2019.
“These data demonstrate that AG10 is well tolerated in
symptomatic patients with ATTR-CM with clear evidence of drug
activity in all actively treated subjects,” said Jonathan Fox, MD,
PhD, president and chief medical officer of Eidos. “The
consistently high levels of TTR stabilization, in all actively
treated subjects and across the entire dosing interval, were
correlated with statistically significant and dose-dependent
increases in serum TTR concentrations. We observed normalized serum
TTR levels in 100% of patients treated with AG10. We believe these
data provide clinical proof-of-concept for AG10 in ATTR-CM
patients. As reflected in these data, AG10’s TTR-stabilizing
properties continue to hold great promise that it could become a
best-in-class treatment for ATTR-CM.”
Phase 2 Clinical TrialThis Phase 2 clinical
trial was a randomized, double-blind, placebo-controlled,
multi-center study that enrolled patients with symptomatic ATTR-CM,
both wild-type and mutant. Eligible patients had confirmed ATTR-CM
and NYHA Class II or III symptoms, and at least one prior heart
failure hospitalization or active treatment for chronic heart
failure. The 49 enrolled subjects were randomly assigned in a 1:1:1
fashion to treatment with placebo, 400 mg AG10 twice daily, or 800
mg AG10 twice daily for 28 days. Results from the study showed the
following:
- The study met its primary objective of establishing that AG10
was well tolerated with no safety signals of potential clinical
concern related to the administration of AG10 in symptomatic
ATTR-CM patients. One serious adverse event (SAE) of dyspnea deemed
unrelated to study drug was observed in one actively-treated
subject (3%) and SAEs of atrial fibrillation, congestive heart
failure, and cellulitis were observed in two placebo-treated
subjects (12%). The overall rate of adverse events (AEs) was 69% in
subjects administered 800 mg bid AG10, 63% in subjects administered
400 mg bid AG10, and 88% in subjects administered placebo.
- As compared to placebo, subjects treated with AG10 demonstrated
a statistically significant increase in serum TTR concentrations
(p<0.0001), a prognostic indicator of survival in ATTR-CM
patients, in a dose-dependent manner. Subjects administered 800 mg
AG10 twice daily, 400 mg AG10 twice daily, and placebo exhibited
mean changes in TTR concentration from baseline of +50%, +36% and
-7%, respectively, at day 28.
- All subjects administered AG10 had serum TTR concentrations
within the normal range at day 28, whereas 31% of subjects
administered placebo had serum TTR concentrations below the normal
range on day 28.
- AG10 administration resulted in near-complete stabilization of
TTR at day 28 (>90%, on average), across the dosing interval in
all actively treated subjects as measured by established ex vivo
assays.
“We know that stabilizing TTR can lead to clinical benefit in
ATTR patients, that treatment with a stabilizer increases TTR
levels in ATTR-CM patients, and that higher serum concentrations of
TTR are associated with a better prognosis in ATTR-CM,” said Dr.
Daniel Judge, M.D., professor in the division of cardiology at the
Medical University of South Carolina. “These Phase 2 data provide
compelling evidence that AG10 stabilizes TTR to a high degree and
restores serum TTR levels to normal even in patients carrying
destabilizing mutations, suggesting the potential for the molecule
to become an effective disease-modifying therapy for ATTR
patients.”
Investor Conference Call and Webcast
DetailsEidos management will host an investor conference
call and webcast on Monday, November 12 at 8am ET to review the
Phase 2 data. To participate in the conference call, dial
+1-844-293-0174 (U.S. toll free) or +1-916-582-3546
(international), conference ID 8594856. 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. AG10 is currently being studied
in an open-label extension of a Phase 2 clinical trial in patients
with ATTR cardiomyopathy.
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 transthyretin amyloidosis (ATTR)
ATTR represents a significant unmet need of a comparatively
large patient population in the context of rare genetic diseases
with an inadequate current standard of care. There are three
distinct diseases that comprise the ATTR family: 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). 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, its potential
to become a best-in-class treatment for ATTR-CM, future clinical
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 our other product candidates through current and
future milestones, our ability to raise additional funding to
complete the development of AG10, our dependence on the success of
AG10, 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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