SYN-004 Receives Breakthrough Therapy Designation from FDA

Synthetic Biologics announced the U.S. Food and Drug Administration (FDA) granted a Breakthrough Therapy Designation for SYN-004 (ribaxamase) for the prevention of Clostridium difficile infection. SYN-004 (ribaxamase) is the company's oral enzyme designed to protect the gut microbiome from disruption caused by certain intravenous (IV) beta-lactam antibiotics.

The Breakthrough Therapy Designation is based on data from the successful Phase 2b clinical trial of ribaxamase, which met its primary endpoint of significantly reducing CDI. FDA Breakthrough Therapy Designation is intended to expedite development and review timelines when preliminary clinical evidence indicates that a drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for serious or life threatening diseases. If approved by the FDA, SYN-004 would be the first available drug designed to prevent Clostridium difficile infection by protecting the gut microbiome from antibiotic-mediated dysbiosis.

"We are delighted by the FDA's recognition of ribaxamase's potential to prevent CDI, and the dire need to fill the current void of an approved intervention," Jeffrey Riley, President and Chief Executive Officer said. "Following this announcement, we have been asked and anticipate requesting a Type-B multidisciplinary meeting with the Agency for a comprehensive discussion on the overarching, high-level drug development plan and pathway to licensure for ribaxamase. We look forward to working closely with the FDA throughout the development and review process and remain dedicated to bringing this potentially paradigm-shifting approach to antibiotic therapy to patients in critical need."

Clostridium difficile infection is the number one hospital acquired infection in the U.S., with more than 453,000 patients diagnosed annually. CDI results in approximately 29,000 deaths, $1.5 billion in additional healthcare costs, as well as significant and sometimes prolonged illness.