The initial 750-liter cGMP production run on Fujifilm's pAVEway™ platform yielded an unprecedented 5.5 kilograms of >95% pure SYN-004 active pharmaceutical ingredient (API) drug substance, which will be used to support Synthetic Biologics' planned Phase I and II clinical trials, and continued research and development studies.
SYN-004 is believed to be the first and only therapy designed to neutralize intravenous (IV) antibiotics in the gut. It is intended to protect and maintain the balance of bacterial flora in the gastrointestinal (GI) tract, or gut microbiome, and to potentially prevent the devastating effects of C. difficile infection. Research continues to demonstrate that protection of the microbiome plays an increasingly important role in the prevention of a variety of GI, metabolic and CNS disorders. The U.S. Centers for Disease Control and Prevention (CDC) have classified C. diff as an "urgent public health threat", surpassing Methicillin-resistant Staphylococcus aureus (MRSA) as the number one hospital-acquired infection in the United States. C. difficile is a multidrug-resistant bacterium that infects 1.1 million U.S. patients annually, 1 and 30, 000 patients die with a C. difficile infection annually2.
"We are extremely impressed by the quality, quantity and efficiency of the initial SYN-004 API manufacturing work by Fujifilm, " stated Jeffrey Riley, Chief Executive Officer of Synthetic Biologics. "Fujifilm not only exceeded our yield expectations, but also provided exemplary customer service through its diligent approach and skillful execution in developing the cGMP process for SYN-004."
Mr. Riley concluded, "Importantly, with a solid new GMP manufacturing process, Synthetic Biologics remains on schedule to file an Investigational New Drug (IND) application for SYN-004, and initiate Phase Ia and Ib clinical studies for the prevention of C. difficile infection in the second half of 2014. Preliminary Phase I topline data is expected by year-end 2014, and a Phase II efficacy study of SYN-004 is planned to begin in the first half of 2015."