Indicates encouraging safety profile and non-futility of study
Leeds, UK, April 17, 2020 - 4D pharma plc (AIM: DDDD), a pharmaceutical company leading the development of Live Biotherapeutics, today announces the key findings of a planned interim analysis in its BHT-II-0002 study in IBS.
BHT-II-0002 is 4D Pharma’s phase II, randomized, double blind, placebo-controlled, multicenter study. It is evaluating the safety and efficacy of repeated oral doses of the company’s Live Biotherapeutic, Blautix, in adult subjects with irritable bowel syndrome (IBS) subtypes IBS-C and IBS-D.
“4D Pharma has continued to progress its phase II study of Blautix, whilst continuing development of its broad pipeline of therapies for a range of conditions,” said Duncan Peyton, CEO, 4D Pharma. “This pipeline is focused on oncology, asthma and neurodegeneration, enabling 4D Pharma to develop potential therapies for a number of serious conditions. Our internal discovery platform, Micro Rx, continues to uncover new approaches to areas of significant unmet need. Given the strong safety profile we have seen across our programs, our work with Live Biotherapeutics enables 4D Pharma to develop potential therapies for a number of serious conditions for patients with considerable underserved needs with a strong safety profile.”
The BHT-II-0002 study interim analysis, aimed to support clinical and commercial development plans, has been conducted on a total of 246 IBS patients (118 IBS-C and 128 IBS-D). Data on around 78 additional patients who have all finished drug treatment and primary endpoint evaluations for all patients are expected to be included in the full study analysis and trial data. 4D Pharma expects to report on this full data set as planned in Q3 2020.
The interim analysis demonstrates that Blautix has a safety profile comparable to placebo, with 3 percent vs 2 percent patient discontinuation in the drug group vs. placebo due to adverse events (AE). There was one recorded severe adverse event (SAE) in each arm with no relation to study drug, and mild or moderate adverse events reported in 24 percent of subjects for both Blautix and placebo arms, indicating that the treatment has no increased adverse effects compared to placebo. The interim analysis of the primary endpoint of the IBS study (described more fully below) shows that the study is not futile and 4D pharma plc is encouraged to continue the analysis of the full trial data.
“The interim analysis of the Blautix phase II results in IBS is encouraging,” says Dr. Alex Stevenson, Chief Scientific Officer, 4D Pharma. “IBS is a disease that causes significant morbidity and has a high unmet medical need. 4D Pharma identified the potential of the live biotherapeutic Blautix as a new approach to treat IBS and address both IBS-C and IBS-D with the same therapy. The interim analysis supports and informs 4D Pharma’s future development and commercialization plans with partners.”
Blautix is a single strain live biotherapeutic product, being developed as a single treatment for both IBS-C and IBS-D. Pre-clinical studies demonstrated its ability to address visceral hypersensitivity and other symptoms of IBS and increase microbiome diversity. A phase I study in healthy volunteers and IBS patients showed that Blautix was well tolerated and could increase microbiome diversity and stability in IBS patients. An improvement in symptoms was also reported relative to placebo. Further information on the phase II study can be found at ClinicalTrials.gov Identifier: NCT03721107.
IBS has an incidence of 11 percent internationally, ranging from 10 percent to 25 percent in different regions. Around 30 percent of people who experience the symptoms of IBS will consult physicians for their IBS symptoms. IBS has different subtypes, IBS-C, IBS-D and IBS-M, which comprise in the US 38.5 percent, 32.5 percent and 29.0 percent of diagnosed cases respectively. Existing approved treatments are focused on addressing the specific symptoms of disease subtypes, rather than the underlying disease and often have significant side effects and modest levels of efficacy. Research indicates that the microbiome is altered in IBS patients compared to healthy controls, but that the alteration is consistent across the different sub-types, suggesting the possibility that a microbiome based intervention could address multiple subtypes.
About the BHT-II-0002 study
This study has recruited over 300 patients in 35 investigator sites in the USA and the UK. The interim analysis included evaluation of the primary endpoint of the study, which is based on the number of overall responders in each group. The definition of an overall responder is IBS subtype specific and consists of two components. For both IBS subtypes, the first component is the weekly average of abdominal pain intensity. Improvement is defined as a decrease of at least 30 percent compared with baseline. The second component is different for the two IBS subtypes and relates to stool frequency per week for IBS-C and a reduction in incidence of loose stool consistency for IBS-D compared with baseline. In order for a subject to be classed as a responder, both components must show improvement in the same week for at least 50 percent of the treatment period of 8 weeks.
Founded in February 2014, 4D pharma is a world leader in the development of Live Biotherapeutics, a novel and emerging class of drugs, defined by the FDA as biological products that contain a live organism, such as a bacterium, that is applicable to the prevention, treatment or cure of a disease. 4D has developed a proprietary platform, MicroRx(R), that rationally identifies Live Biotherapeutics based on a deep understanding of function and mechanism.
4D's Live Biotherapeutic Products are orally delivered single strains of bacteria that are naturally found in the healthy human gut. The Company has five clinical studies in progress, namely a Phase II clinical study of BLAUTIX(R) in Irritable Bowel Syndrome (IBS), a Phase I/II study of MRx0518 in combination with KEYTRUDA(R) (pembrolizumab) in solid tumours, a Phase I study of MRx0518 in a neoadjuvant setting for patients with solid tumours, a Phase I study of MRx0518 in patients with pancreatic cancer and a Phase I/II study of MRx-4DP0004 in asthma. Preclinical-stage programmes include candidates for CNS disease such as Parkinson's disease and other neurodegenerative conditions. The Company has a research collaboration with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ, USA, to discover and develop Live Biotherapeutics for vaccines.
For more information, refer to https://www.4dpharmaplc.com.
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