Eidos Therapeutics Receives Positive Opinion for Orphan Designation from the European Medicines Agency for AG10, a Potent Oral Stabilizer for the Treatment of Transthyretin Amyloidosis
TTR normally circulates as a four-part molecule or tetramer, but in ATTR the molecule dissociates into individual monomers, which are unstable and aggregate as amyloid fibrils. Eidos’ lead product candidate, AG10, is designed to target ATTR at its source by stabilizing tetrameric TTR in the blood.
“We are pleased to have received Orphan Drug Designation from the
Orphan Designation by the
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 and symptomatic heart failure. Results from this trial will be presented on
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 also carrying a pathogenic, or disease-causing, mutation in the other copy of their 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.
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 future clinical milestones of AG10, including the completion of our ongoing Phase 2 clinical trial and availability of data therefrom, the initiation of Phase 3 clinical trials, the timing of these events, the indications we intend to pursue, the potential benefits available to us from orphan drug designations for AG10, 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 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
Source: Eidos Therapeutics, Inc.