New antibacterial drug delivers safety, tolerability and efficacy data

In development for prevention of post-surgical staphylococcal infections

Destiny Pharma, a clinical stage biopharmaceutical company focused on developing antibacterial medicines, today announced the results of a US clinical trial of the novel preventive anti-bacterial drug exeporfinium chloride. The trial is sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the US National Institutes of Health, and was conducted at the NIAID Clinical Trials Unit at Case Western Reserve University in Cleveland and at Anaheim Clinical Trials in California.  

The two-stage clinical trial studied the safety, tolerability, and efficacy of intra-nasally applied exeporfinium chloride gels. Part one yielded safety data in eight volunteers and allowed progression to part two in 48 healthy volunteers with colonised nasal staphylococcus aureus (SA) bacteria. Part two was double-blinded, placebo controlled, and investigated two exeporfinium chloride concentration gels (0.5 & 2.0mg/g) and two viscosities (2 and 4%) applied into the nostrils for five days.  

Both concentrations were deemed safe and well-tolerated, and no drug was detected in the bloodstream. There was a similar safety profile observed in exeporfinium chloride and placebo-treated subjects, which is important because the product will be used in a preventive manner. In general, exeporfinium chloride demonstrated a rapid, anti-staphylococcal effect after one day, with the 2.0mg/g gel showing a statistical difference against placebo, which was sustained throughout dosing.  

Research shows that about one in five people carry SA in their nose, which increases the risk of a post-surgical SA infection by up to ten times. Using antibiotics to treat nasal SA carriage has been long practiced, but emergence of antibiotic resistant strains threatens the efficacy of the practice. In October 2015, the US Food & Drug Administration (FDA) recognised exeporfinium chloride as a Qualifying Infectious Disease Product (QIDP), and confirmed a new medical indication for the drug, namely for the ‘Prevention of Post-Surgical Staphylococcal Infections.’  

Hospital-acquired SA infection remains a major concern, particularly the methicillin resistant SA (MRSA). Infection prevention measures, including treatment of SA/MRSA ahead of surgery in at-risk patients, are now practiced in many countries.  

Still, there is an urgent global need for drugs that prevent SA infections but do not generate bacterial drug resistance. In the United States it is estimated that drug-resistant forms of SA such as MRSA result in 19,000 deaths per year. The annual estimated cost of SA infection in the US is $14.5 billion.

A report in The Lancet Infection Diseases in December 2015 estimates that between 38 and 51% of bacteria that cause post-surgical infections are resistant to traditional antibiotics. The report also estimates that a 30% reduction in antibiotic effectiveness could result in 120,000 additional post-surgical and chemotherapy related infections in the US.  

Compared to antibiotics, exeporfinium chloride has a novel structure and mechanism of action, killing SA bacteria rapidly without appearing to generate resistance. These features make it an ideal candidate for the prevention of post-surgical SA infection.  

Dr Bill Love, CEO of Destiny Pharma commented: "We are delighted with these results, which demonstrate that exeporfinium chloride is safe and well tolerated and has the potential to deliver the FDA/QIDP awarded US indication for the prevention post-surgical SA infection. The support from NIAID made this study possible and we plan to progress this important new drug into the next stage of development and thereon to market."