SAN FRANCISCO, Sept. 30, 2019 (GLOBE NEWSWIRE) -- Eidos Therapeutics, Inc. (Eidos) (EIDX), a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in transthyretin (TTR) amyloidosis (ATTR), today announced that an interim analysis of its ongoing Phase 2 open-label extension (OLE) study of AG10 in subjects with symptomatic ATTR cardiomyopathy will be presented in a late-breaking featured science oral presentation at this year’s American Heart Association (AHA) Scientific Sessions.
Daniel Judge, M.D., professor in the division of cardiology at the Medical University of South Carolina, will discuss the data in a presentation entitled “Long-term Safety and Efficacy of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Interim Analysis of the Ongoing Phase 2 Open-Label Extension Study” at 5:30 PM ET on November 16, 2019 as part of the Early Phase Science oral session.
In addition, Eidos will present several posters at AHA. The following summarizes Eidos’ presentations at the conference:
- Long-term Safety and Efficacy of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Interim Analysis of the Ongoing Phase 2 Open-Label Extension Study (Dr. Daniel Judge; Saturday, November 16, Oral Presentation: 5:30-5:38 PM)
- ATTRibute-CM: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center, Global Phase 3 Study of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) (Dr. Daniel Judge; Monday, November 18; Poster Display: 11:30-12:00 PM)
- Differential Transthyretin Binding, Kinetic Stability and Additive Ex Vivo Stabilization by AG10 Compared to Tafamidis (Dr. Uma Sinha; Monday, November 18; Poster Display: 1:30-2:00 PM)
- Differential Ex Vivo Stabilization of Transthyretin by AG10 and Tafamidis in Samples from Patients with Moderate or Severely Destabilizing Mutations (Dr. Uma Sinha; Monday, November 18; Poster Display: 1:30-2:00 PM)
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 TTR amyloidosis, or ATTR. In a Phase 2 clinical trial in patients with symptomatic ATTR-CM, AG10 was generally well tolerated, demonstrated greater than 90 percent 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 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 or ameliorate 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.
The Phase 3 ATTRibute-CM study of AG10 in patients with ATTR-CM is underway. Part A of the study will assess the change from baseline in 6-minute walk distance (6MWD) at 12 months. Part B of the study will evaluate reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations at 30 months. In addition, Eidos plans to initiate a Phase 3 study of AG10 in ATTR polyneuropathy (ATTR-PN) by the end of 2019.
About transthyretin amyloidosis (ATTR)
There is significant medical need in transthyretin amyloidosis (ATTR) given the large patient population and limited 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 untreated 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 for untreated patients. 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 is a BridgeBio Pharma subsidiary focused on addressing the large and growing unmet need 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.
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, our ability to enroll patients in and conduct the ATTRibute-CM trial and our planned Phase 3 clinical trial of AG10 in ATTR-PN 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 and our planned Phase 3 clinical trial of AG10 in ATTR-PN, 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 June 30, 2019, 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.