Synthetic Biologics, a developer of pathogen-specific therapies for serious infections and diseases, with a focus on protecting the microbiome, announced that enrollment has initiated and the first patient was dosed in a phase 1a clinical trial of SYN-004, an investigational oral beta-lactamase enzyme for the prevention of Clostridium difficile (C. difficile) infection (CDI), antibiotic-associated diarrhoea (AAD) and secondary antibiotic-resistant infections in patients receiving intravenous (IV) beta-lactam antibiotic therapy.
The randomised, double-blind, placebo-controlled phase 1a study, which is now underway at Clinical Pharmacology of Miami, is designed to evaluate the safety, tolerability and pharmacokinetics of five single-ascending doses of oral SYN-004 in healthy volunteers. In all, up to 40 healthy adult volunteers will be enrolled into five cohorts, with approximately six participants receiving SYN-004 and two receiving placebo in each cohort. Before the end of the year, topline phase 1 data is expected to be reported and a phase 1b study evaluating multiple-ascending doses of SYN-004 is planned to begin.
"The initiation of the clinical programme for SYN-004 represents an important milestone for Synthetic Biologics and a key step towards the first potential point-of-care preventative therapy for C. difficile, the CDC's top-ranking public health threat," said Jeffrey Riley, chief executive officer of Synthetic Biologics. "We look forward to moving Synthetic Biologics' innovative therapeutic approach to prevent C. difficile infection through clinical development, and further validating the connection between protecting the gut microbiome and a variety of GI, metabolic and CNS disorders."
Currently, there is no vaccine or drug approved by the US Food and Drug Administration (US FDA) specifically for the prevention of C. difficile infection, which the US Centres for Disease Control (CDC) has identified as an "urgent public health threat" and occurs mostly in people who have had recent medical care with IV antibiotics. These antibiotics can create a harmful imbalance in the gut microbiome by killing "good" bacteria, giving C. difficile a chance to multiply and cause diarrhoea, which can lead to dehydration, fever, abdominal pain, cramping, nausea, colitis, and even death. In all, 24 million Americans receive IV antibiotics annually.
SYN-004 is Synthetic Biologics' oral drug candidate designed to be the first and only treatment intended to prevent the development of C. difficile infection, by binding with and neutralising certain common IV beta-lactam antibiotics in the gut. During 2012, 14.4 million patients in the US received approximately 117.6 million doses of SYN-004 susceptible IV antibiotics. SYN-004 is intended to block the unintended harmful effects of antibiotics within the gastrointestinal (GI) tract, maintaining the natural balance of the bacterial flora (gut microbiome), potentially preventing the 1.1 million C. difficile infections and 30,000 C. difficile-related deaths in the US each year.