4D pharma plc (AIM: DDDD), a pharmaceutical company leading the development of live biotherapeutics, has today announced that the Company has submitted, and the U.S. Food and Drug Administration (FDA) has cleared, an Investigational New Drug Application for Blautix, the Company’s live biotherapeutic for the treatment of Irritable Bowel Syndrome (IBS). Enrolment in the Phase 2 study is expected to commence in the second half of 2018.
The double-blind, placebo-controlled multicentre Phase 2 study will evaluate the efficacy and safety of Blautix in patients with IBS with constipation (IBS-C) and IBS with diarrhoea (IBS-D). 4D has consulted with the FDA on the design of the study. Up to 500 patients will receive either Blautix or placebo daily for 8 weeks. The primary endpoint will be the Overall Response rate, defined as the proportion of patients reporting an improvement in their weekly cohort-specific symptoms (abdominal pain and stool frequency or consistency) for at least 4 out of 8 weeks. A range of secondary efficacy endpoints will also be assessed, as well as the effects of Blautix on the gut microbiome.
Dr. Alex Stevenson, 4D’s Chief Scientific Officer, commented: “We are delighted to advance Blautix into this important proof-of-concept study, which brings us another step closer to bringing a novel treatment regime to patients. Despite affecting up to 15% of the population, and a market forecast to be worth more than $2.5 billion by 2024*, IBS is poorly addressed with current therapies, which only target the symptoms of the disease rather than tackling the underlying causes. By targeting the microbiome, Blautix has the potential to make a real difference for patients and could represent a new era in the treatment of IBS.”
Blautix is a single-strain live biotherapeutic candidate in development for the treatment of IBS-C and IBS-D. The organism was originally isolated from a healthy individual and has a distinctive metabolism which utilises intestinal hydrogen as an energy source, reduces levels of hydrogen sulphide, and increases microbiota diversity and stability. Unlike existing therapies which only seek to address IBS symptoms, by targeting the microbiome Blautix has the potential to act directly on the underlying pathophysiology of disease to treat all IBS patients irrespective of traditional sub-types.