Spectrum CTSA Pilot funding leads to FDA/EMA-approved Attruby^TM / Beyonttra^TM (Acoramidis,) for life-threatening heart disease

A pilot project supported by Spectrum CTSA’s SPARK program at Stanford has resulted in an FDA and EMA-approved drug that halts damaging deposits in the heart muscle. Attruby’s/Beyonttra’s development showcases the transformational impact of the CTSA-sponsored SPARK at Stanford program and its unique model of combining multidisciplinary mentoring and support with innovative education and training.

The therapeutic Attruby/Beyonttra (Acoramidis) is the result of an interdisciplinary collaboration between physician-scientist Isabella Graef, MD, former faculty member at Stanford University and CEO of Shenandoah Therapeutics, and Mamoun M. Alhamadsheh, PhD, a former medicinal chemist at Stanford University who is now a Professor of Pharmaceutical Chemistry at the University of the Pacific. Previously known as the Alhamadsheh-Graef molecule 10 (AG10), Attruby was designed and synthesized by Alhamadsheh. In 2013, Dr. Graef received a CTSA-funded pilot to develop a novel therapeutic for familial amyloidosis. This pilot, combined with SPARK’s innovative mentoring & education program, played a key role in the development of Acoramidis into a clinical candidate and empowering Drs. Graef and Alhamadsheh to co-found Eidos Therapeutics, which was later acquired by BridgeBio Pharma, Inc.

Attruby/Beyonttra (Acoramidis) stabilizes the transthyretin (TTR) protein in patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM), a severe and rare restrictive cardiomyopathy caused by accumulation of transthyretin fibrils in the myocardium. Attruby/Beyonttra (Acoramidis) halts the amyloid deposits that cause cardiac damage. Clinical trials demonstrated the drug’s exceptional efficacy, improving both survival rates and quality of life, offering renewed hope to thousands of patients worldwide who suffer from this rare disease.

SPARK at Stanford provides the knowledge and infrastructure to get academic discoveries across the "valley of death" to clinical trials and commercialization. Many innovative solutions discovered in academia never make it to patients due to a disconnect between academia and industry, but SPARK addresses the challenges associated with translating academic ideas and findings into therapeutics that benefit patients.

Dr. Graef commented, “This success is the result of years of dedication, collaboration, and a shared commitment to improving patients' lives. I am truly grateful that Dr. Alhamadsheh and I have been able to contribute to a therapy that can make a meaningful difference for those affected. We are grateful for SPARK’s mentoring and guidance, which was so important for us in developing Acoramidis.”