Research update

Breakthrough compounds for diabetes and inflammation

23 September, 2015 – C4X Discovery Holdings plc (“C4XD”), a leader in rational drug discovery and design, is pleased to provide the following research update on recent key milestones in its programmes for Type 2 diabetes and inflammation, two areas of significant unmet medical need that represent large pharmaceutical markets.

Breakthrough approach

C4XD’s technology has enabled rapid progress across its programmes in recent months, providing further exciting proof-of-concept for our novel approach to drug discovery based on conformational design of solution structures. We have now met significant milestones in Type 2 diabetes and inflammation, adding to the earlier success with our Orexin-1 programme, and further demonstrating C4XD’s ability to rapidly and intelligently develop new medicines for areas of high, unmet need.

C4XD expects to progress both the Type 2 diabetes and inflammation programmes into in vivo proof-of-principle testing in the next few months.

Type 2 Diabetes: targeting GPR142

C4XD has identified novel lead molecules that activate GPR142, a key factor in the production of insulin. Targeting GPR142 may stimulate insulin production in a glucose-dependent manner, avoiding the hypoglycaemia risk associated with existing diabetes therapies. GPR142 has recently become the focus of considerable research and patent activity within the pharma industry.

Using its proprietary technology, C4XD has identified critical drug design principles, enabling us to generate potent, orally available compounds in just a few months.


C4XD has designed novel activators for the NRF-2 pathway, which is important in mediating diseases such as Chronic Obstructive Pulmonary Disease (“COPD”) and Multiple Sclerosis (“MS”). NRF-2 is the subject of considerable investment by the pharma industry; for example, it is the target for the MS drug Tecfidera®, which generated 2014 revenues of $2.9 billion.

Existing approaches activate NRF-2 in a non-specific and/or irreversible way with potential for toxic side effects. C4XD’s technology has enabled us to rationally design and generate selective, reversible NRF-2 activators that may offer improved safety and efficacy.


C4XD’s most advanced compound, an oral Orexin-1 antagonist for the treatment of addiction, is in formal pre-clinical safety and toxicity studies prior to initiation of clinical trials. We expect to file a Clinical Trial Application to enable human studies at the end of 2016.

C4XD expects to be in a position to announce further news regarding novel targets in the coming weeks.

“We continue to make outstanding progress with our proprietary programmes, which are focused on areas of high unmet medical need, with large target markets” said Piers Morgan, CEO. “Our technology enables us to design novel, selective, potent leads and drug candidates in a fraction of the time and cost compared to conventional methods.”