New biotherapeutics drug for Crohn's to enter trials

A UK based company specialising in new drugs which use live bacteria to treat serious bowel complaints is causing excitement among investors.

4D pharma's share price rose 10 per cent on Tuesday on news that it will commence clinical trials of its two lead candidates in 2015.

Blautix is for the treatment of Irritable Bowel Syndrome (IBS) and Thetanix is for the Paediatric Crohn's Disease (PCD), and both use biotherapeutics – the use of live bacteria – to help treat the underlying causes of the diseases.

The Manchester-based firm says biotherapeutics has the potential to fundamentally change the approach to treating these diseases, and others. The therapies are based on research into the 'microbiome'- the interplay of many different types of bacteria which exist naturally in the human body.

There is growing evidence that changing the balance of 'good' and 'bad' bacteria in the gut microbiome can also improve health in many different ways - reducing inflammation which causes bowel complaints, but also alleviating depression and anxiety, for example.

4D pharma believes treatments using the approach could show a greatly improved safety profile and greater efficacy over existing treatments, and could also target multiple pathways in a single disease.

For IBS, there are currently few treatment options, and in Crohn's disease in children, treatment options depend on severity. Corticosteroids, immunomodulators and anti-TNF therapies are often used, but are associated with a range of side effects, from infection to certain cancers. Surgery may also be necessary for some children. Around 20% of patients with Crohn's disease present when they are younger than 20 years and it is estimated that there are around 41,000 children in the USA with PCD.

Despite the company's confidence in their approach, the drugs are yet to prove their potential in human trials. The first clinical studies in 2015 will mainly look at safety and tolerability, with clinical effects a secondary endpoint.

The FDA recently granted orphan status to Rosburix for the treatment of paediatric ulcerative colitis, which makes it one of the leaders in the emerging field of biotherapeutics.

Dr. Alex Stevenson, 4D's chief scientific officer commented: "The Blautix and Thetanix trials will represent important milestones for the company and a new class of therapeutic, designed to treat the underlying causes of diseases rather than just the symptoms. In addition, 4D continues to generate a strong pipeline of live biotherapeutics for other important diseases, using its proprietary MicroRx platform."

Other leading companies in the field include Microbiome Therapeutics, which in June presented its first clinical trial data of its lead 'microbiome modulator' NM504. The drug is designed to treat imbalances associated with type 2 diabetes, and preliminary results showed the treatment lowered blood glucose levels, and even lowered cholesterol levels.

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