Celladon

Celladon Corporation was a clinical-stage biotechnology firm that positioned itself at the forefront of cardiovascular gene therapy, targeting diseases with significant unmet medical needs. The company's primary focus was the development of treatments for heart failure and other conditions characterized by deficiencies in the SERCA family of enzymes, which are integral to regulating intracellular calcium.

The company's lead product candidate was MYDICAR, a genetically targeted enzyme replacement therapy developed for patients suffering from advanced heart failure due to systolic dysfunction. The scientific premise was based on addressing the deficiency of the SERCA2a enzyme, which becomes depleted in heart failure patients, leading to impaired cardiac function. Co-founder Dr. Roger J. Hajjar, Director of the Cardiovascular Research Center at Mount Sinai School of Medicine, was instrumental in developing the core science behind MYDICAR. The therapy consisted of a one-time, intracoronary infusion of an adeno-associated viral vector (AAV1) to deliver the SERCA2a gene directly to heart muscle cells, a procedure performed during a standard cardiac catheterization.

Under the leadership of CEO Krisztina Zsebo, Ph.D., Celladon advanced MYDICAR through clinical trials. The initial Phase 1/2 CUPID trial showed an acceptable safety profile and suggested potential efficacy, leading to further investigation. This early promise was recognized by the FDA, which granted MYDICAR Fast Track status in late 2011 and Breakthrough Therapy designation in April 2014, a status intended to expedite the development of treatments for life-threatening conditions. The company attracted significant investment from venture capital arms of major pharmaceutical companies, including Pfizer, Novartis, and Johnson & Johnson, to fund its pivotal Phase 2b trial, known as CUPID2.

The company's trajectory dramatically shifted in April 2015 when it announced that the 250-patient CUPID2 trial failed to meet its primary and secondary endpoints. The therapy did not demonstrate a significant effect in reducing heart failure-related hospitalizations or other major cardiovascular events compared to placebo. Following this definitive clinical failure, the company's stock value collapsed. Celladon subsequently halted all research and development, including preclinical programs, and significantly reduced its workforce. In November 2015, Celladon entered into a definitive reverse merger agreement with the privately-held Eiger BioPharmaceuticals. The transaction resulted in the combined entity being renamed Eiger BioPharmaceuticals, effectively marking the end of Celladon Corporation as an operating entity.

Keywords: Celladon, MYDICAR, gene therapy, heart failure, cardiovascular disease, SERCA2a, AAV1, Krisztina Zsebo, Roger J. Hajjar, CUPID trial, clinical trial failure, reverse merger, Eiger BioPharmaceuticals, biotechnology, enzyme replacement, cardiac gene therapy, systolic dysfunction, adeno-associated virus, clinical-stage, biopharmaceutical