MIMICKING A NATURALLY PROTECTIVE APPROACH
Eidos Therapeutics is developing a small molecule named acoramidis (AG10), which was designed by researchers at Stanford University to potently stabilize TTR by mimicking the structure of the T119M TTR variant. T119M is known to protect carriers from developing ATTR due to the formation of unique bonds at the center of the molecule that stabilize the protein approximately 40-fold beyond the native structure.
In pre-clinical studies, acoramidis (AG10) was shown to bind TTR tetramers at the same central locations at which the T119M variant forms stabilizing bonds. This unique binding mode is thought to underlie the potent stabilization of TTR by acoramidis (AG10).
The safety and efficacy of acoramidis (AG10) have not been established. There is no guarantee that acoramidis (AG10) will receive health authority approval or become commercially available in any country for the uses being investigated.
Acoramidis (AG10) has demonstrated near-complete TTR stabilization in pre-clinical and clinical studies to date:
- In pre-clinical studies, acoramidis (AG10) demonstrated TTR stabilization across all TTR variants tested by fibril formation, Western blot, fluorescent probe exclusion, and sub-unit exchange assays1
- In a Phase 1 study in healthy volunteers, acoramidis (AG10) (800 mg twice daily) achieved >90% TTR stabilization at trough plasma concentrations and was well tolerated2
- In a Phase 2 study in patients with ATTR-CM, acoramidis (AG10) (800 mg twice daily) again achieved >90% TTR stabilization at trough plasma concentrations and adverse events were similar between placebo and the acoramidis (AG10) groups3
References: 1. Penchala SC et al. Proc Natl Acad Sci USA. 2013;110(24):9992-9997. 2. Fox JC et al. Clin Pharmacol Drug Dev. 2020;9(1):115-129. 3. Judge DP et al. J Am Coll Cardiol. 2019;74(3):285-295.
In the pipeline
Acoramidis (AG10) is currently being studied in a Phase 3 clinical trial in patients with ATTR-CM (ATTRibute-CM) and a Phase 3 clinical trial in patients with ATTR-PN (ATTRibute-PN).
Programs
Phase 1
Phase 2
Phase 3
Phase 3
Phase 3
About Eidos Therapeutics
Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the growing unmet need in diseases caused by transthyretin amyloidosis, or ATTR. ATTR is caused by the destabilization of TTR due to inherited mutations or aging and is commonly divided into 3 distinct categories: wild-type ATTR cardiomyopathy (wtATTR-CM), variant ATTR cardiomyopathy (vATTR-CM), and ATTR polyneuropathy (ATTR-PN). The estimated worldwide prevalence of each condition is 400,000 patients, 40,000 patients, and 10,000 patients, respectively. 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. We seek to treat this well-defined family of diseases at their collective source by stabilizing transthyretin.
Eidos is led by a team of highly experienced clinical researchers, physicians, and scientists who are responsible for developing more than 30 molecules through IND applications and more than 10 approved drugs. Together with patients and physicians, we aim to develop a safe, effective, and disease-modifying treatment for ATTR patients as expeditiously as possible.
EMA=European Medicines Agency; FDA=Food and Drug Administration; IND=Investigational New Drug; ODD=orphan drug designation.
The safety and efficacy of acoramidis (AG10) have not been established. There is no guarantee that acoramidis (AG10) will receive health authority approval or become commercially available in any country for the uses being investigated.
Parent Company
EIDOS IS A MEMBER OF THE BRIDGEBIO FAMILY
BridgeBio is a team of experienced drug discoverers, developers, and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.
