4D pharma plc a pharmaceutical company in the development of Live Biotherapeutic products (LBPs) – a novel class of drug derived from the microbiome, today announces additional positive data from its completed Phase II trial of LBP Blautix® in subjects with irritable bowel syndrome with constipation (IBS-C) or with diarrhoea (IBS-D).
The data has been presented in a poster session at Digestive Disease Week (DDW), on May 22, 2021, by Professor Eamonn Quigley, M.D., Head of Gastroenterology and Hepatology at Houston Methodist Hospital, Professor of Medicine at Weill Cornell Medical, and Chief Investigator of the study.
4D pharma previously announced topline efficacy and safety results from the trial, conducted in the US, UK and Ireland, in October 2020. Further analysis of the data has revealed strong and statistically significant activity on the key symptom of bowel habit, a potential approvable primary endpoint per regulatory guidelines. In addition, analysis of the data by geographical region shows that earlier topline results were impacted by an unusually high placebo response in patients in the UK and Ireland, and enhanced positive signals were seen in the larger US population.
Single strain LBP Blautix® is the first therapeutic globally to have demonstrated efficacy in both IBS-C and IBS-D. The clinically meaningful overall response rates and improvements in bowel habit in both IBS-C and IBS-D in this signal finding Phase II trial, in spite of an unexpectedly high placebo response in certain patient groups, are highly encouraging for subsequent larger studies with increased statistical power.
“Following the announcement of the topline results in October, 4D pharma has been able to review the data in more detail. We have also discussed the results with internationally renowned key opinion leaders and patient groups. We are encouraged by the positive outcomes of this additional analysis, and we strongly believe that this signal finding study supports the continued development of Blautix as a novel treatment for IBS,” said Dr. Alex Stevenson, Chief Scientific Officer, 4D pharma. “The Phase II results, in conjunction with regulatory guidance and KOL discussions, provide a clear and viable path forward for 4D pharma to continue to develop Blautix to address a significant unmet need. Topline results were significantly impacted by an unusually high placebo response in certain geographical patient groups. Despite this, the activity of Blautix relative to placebo in this Phase II study is competitive with approved IBS therapeutics. We are pleased to now share our additional positive findings following more detailed analysis of the clinical data. With the vital learnings we have gained, we are now even more enthusiastic about the chances of success in subsequent, larger, well-powered studies.”
“The idea of a single, safe, effective therapeutic able to address multiple sub-types of IBS is a hugely exciting proposition for both patients and clinicians. The level of efficacy achieved by Blautix® in IBS-C and IBS-D is very encouraging for larger pivotal studies,” said Professor Eamonn Quigley, Chief Investigator of the study. “Importantly, the Phase II study used robust enrolment criteria and endpoints, setting the program up well for any pivotal studies. The efficacy and clean safety profile of Blautix® presents an attractive package and a competitive position compared to what is currently available to prescribe to IBS patients.”
The signal finding Phase II study was a multi-centre, randomised, double-blind, placebo-controlled study. It enrolled 353 patients in the US, UK and Ireland with IBS-C or IBS-D as defined by the Rome IV criteria, and moderate or severe IBS symptom severity score (IBS-SSS) of ≥175 at screening (158 IBS-C and 195 IBS-D patients). Each sub-type cohort was randomized 1:1 to receive oral Blautix or placebo, two capsules twice daily, for 8 weeks. The study was designed to generate signals of activity in both IBS-C and IBS-D and to define key parameters for a pivotal program.
The previously announced overall responder rate (ORR) primary endpoint was assessed in all randomized subjects (N=353). ORR was also assessed in all patients evaluable for efficacy at eight weeks (N=316). Bowel habit and abdominal pain, the two components of the overall responder endpoint, were also analyzed independently. Post-hoc analyses of efficacy readouts by geographic region and gender subsets were also conducted.
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