Our board provides executive leadership for the business

Read more: Board of Directors

Meet the people who take charge of day-to-day operations at Vernalis

Read more: Senior management

The lead molecule arising from our Fatty Acid Amide Hydrolase research programme, V158866 completed a phase II study in spinal cord injury patients in August 2015.  It is now available for partnering.


The FAAH enzyme metabolises the endocannabinoids (ECs) which are neurotransmitters acting on a range of different receptors. Inhibit FAAH, and elevated EC levels appear in the nervous system. Amongst the receptors which the ECs act upon are the CB1, CB2 and TRPV1 receptors, stimulation of which has been shown to relieve pain. ECs also act on the PPARα receptor which is known to reduce inflammation.

  • Generated from in-house research programme
  • Potentially profound analgesic response
  • Effective in pre-clinical pain models

FAAH inhibitors

May cause powerful analgesic response

Since endocannabinoids are only produced on demand, it may be that FAAH inhibitors, such as V158866, can selectively increase the local levels of neurotransmitter in tissues. This could deliver a powerful analgesic and anti-inflammatory response, without the side effects that more widespread cannabinoid receptor activation can generate.


Vernalis identified Fatty Acid Amide Hydrolase as a target that might be particularly amenable to our research techniques back in 2007. V158866 was the lead molecule to emerge from this research programme, demonstrating our ability to generate promising candidates that go on to full pipeline development.

Leading players in the pharmaceutical industry are paying particular interest to FAAH inhibitor molecules, with a number of them currently in development by the major companies. Additional possible indications for the class include the treatment of anxiety and depression.

Pre-clinical studies

V158866 has been shown to be a potent and selective slowly reversible inhibitor of human FAAH1 (IC50 24nM). It has been evaluated in a series of pre-clinical pain models and has been shown to have efficacy in:

  • Carageenan-induced thermal hyperalgesia
  • Carageenan-induced inflammation
  • Mono-iodoacetate model of osteoarthritis
  • Chronic constriction injury model of neuropathic pain


It has no significant effect on locomotor function in pre-clinical models, even at high doses.

A range of pre-clinical safety and pharmacokinetic studies were conducted on V158866.

Clinical studies

Positive results were announced in September 2011 for V158866 in a Phase I SAD and MAD study. The double-blind, placebo controlled study investigating single and multiple ascending doses was conducted in healthy male volunteers. The objective of the study, to identify doses of V158866 that are both safe and well tolerated and to measure the compound's effect on FAAH activity and endocannabinoid levels, was achieved. The study has confirmed V158866 is a potent inhibitor of human FAAH and has identified once daily doses to be tested in future studies in pain or other indications.

In August 2015, the Company announced results from a Phase II POC study in which the programme was being investigated in patients with neurophathic pain as a result of spinal cord injury.  Although the dosing of V158866 resulted in elevated endocannabanoid levels, on an intent-to-treat basis, the study failed to meet its pain reduction primary endpoint. Treatment did show a trend towards efficacy on a per protocol basis and was generally well tolerated.

We do not plan to make any further investment in this programme and seek to realise its potential value through partnering.

Project Status

Pre-clinical sudies completed, clinical studies were initiated in March 2011 and the positive results of the Phase I SAD and MAD study announced in September 2011. We initiated a Phase II proof-of-concept study in 2013 and clinical study results were announced in August 2015.

We do not plan to make any further investment in this programme and seek to realise its potential value through partnering

A novel, recombinant human thrombolytic protein, V10153 is being developed for the potential treatment of acute ischaemic stroke.

  • A modified form of human plasminogen
  • Only activated to plasmin by the presence of thrombin on newly formed clots
  • Unique action, not seen in other thrombolytics
  • Long half life allowing persistence of effect and bolus administration

V10153 is a biological molecule, first developed by one of Vernalis’s predecessor companies. V10153 is human plasminogen that has been altered so that it is activated to plasmin by thrombin, rather than by the natural plasminogen activators. This makes V10153 potentially much more location-specific than the drugs currently available to tackle ischaemic stroke, and the blood clots that are its cause.

Activation by thrombin

To deliver localised effect

Most thrombolytics are tissue plasminogen activators, and so can activate plasminogen circulating in the body. Because V10153 is a thrombolytic that is only activated by the presence of thrombin (occurring on the surface of newly formed blood clots) its effect is potentially much more localised.

V10153 has a half-life of 3-4 hours so can persist in the blood as an inactive pro-drug that can be selectively activated at fresh/forming thrombi by the thrombin localised there. A key feature of thrombin-activatable plasminogen is that thrombin is only found at the sites of ongoing clotting. This novel activation mechanism results in localised plasmin generation to remove thrombi and prevent them forming/reforming. Persistence in the circulation may also reduce re-occlusion rates.

Stroke affects the blood vessels supplying the brain and occurs when an artery ruptures or becomes blocked by a blood clot. This deprives the brain of oxygen. Nerve cells in the affected area die within minutes. A severe stroke can be fatal or can leave the victim permanently disabled, as dead brain cells cannot be replaced.

There are two main types of stroke, ischaemic stroke, caused by blockage of a blood vessel, and hemorrhagic stroke, caused by bleeding. Ischaemic stroke accounts for over 80 percent of all strokes. People of all ages, including children, suffer from stroke, and it occurs slightly more commonly in men than women.

Current therapeutic options for ischaemic stroke sufferers are limited since the only current approved therapy, recombinant tissue plasminogen activator (rt-PA), must be administered within a few hours of a stroke occurring. Stroke is the third most common cause of death, behind heart disease and cancer. The market for effective and safe stroke therapeutics is considerable.

A range of studies confirm both the thrombolytic and also the anti-thrombotic effect of V10153. It has comparable thrombolytic activity to rt-PA in inducing reperfusion of occluded arteries. It is better than rt-PA at preventing reocclusion, and achieves thrombolysis with no accompanying increase in bleeding. Thrombolytic efficacy (a summation of the ability of the agent to induce reperfusion and prevent reocclusion) was clearly seen to be superior to rt-PA. V10153 has been shown to be markedly superior to heparin as an antithrombotic.

We’ve successfully completed a number of clinical studies with V10153. These included a Phase I study in healthy volunteers, and a dose escalation Phase IIA safety study in patients with acute ischaemic stroke. We also conducted a study of patients with acute myocardial infarction. With both proof of concept as a thrombolytic and the appropriate dose to study in acute ischaemic stroke established, V10153 is ready to move to the next stage of development.


Pre-clinical studies completed by Vernalis. We’ve also completed Phase I and a number of Phase II clinical studies, again with positive results. V10153 is now ready for partnering and Vernalis welcomes expressions of interest.

A novel intravenous Hsp90 inhibitor with the potential to target a range of cancers, discovered during a research collaboration with Novartis. Vernalis regained the rights to luminespib (AUY922) in December 2014 following Novartis' decision to cease all development work. Vernalis is reviewing the data, before deciding on next steps and how best to take the programme forward.



  • Arises out of research collaboration with Novartis
  • Potentially effective against a range of cancers

This molecule arose out of a research collaboration, originally between Vernalis and The Institute of Cancer Research and Cancer Research Technology and then with Novartis, started in 2004. Hsp90 inhibitors such as luminespib (AUY922) have the potential to be effective against a range of cancers

Vernalis is one of the world leaders in the cutting edge fragment-based drug discovery techniques which were employed in the discovery of luminespib (AUY922).

Inhibiting heat shock proteins

May inhibit tumour growth

Heat shock proteins such as Hsp90 are molecular chaperones, with their expression increased in stressed tissues. They may help tumour cells survive in hostile microenvironments and tolerate internal mutations, so driving tumour growth. Inhibiting such proteins may therefore inhibit growth in a wide range of tumour types.


Over-expressed in cancer cells, Hsp90 is essential for the stability and function of several proteins that are mutated into cancer-driving forms in tumours. Inhibition of Hsp90 has the potential to stop cancer progression at multiple levels by inhibiting cell growth and limitless proliferation, promoting cell death, reversing angiogenesis and restricting invasion and metastasis.

Pre-clinical studies

A range of pre-clinical studies confirm the theoretical effect of Hsp90 inhibition. Proof-of-concept for this target has been established in human tumour xenograft models and in the clinic with first-in-class natural product inhibitors derived from geldanamycin. Luminespib (AUY922) has now moved into the clinical studies phase of development.

Clinical studies

Novartis evaluated luminespib (AUY922) in a number of Phase I and Phase II studies in patients with solid tumours and haematological cancers. In December 2009 Novartis announced that the maximum tolerated dose had been reached. In March 2010 we announced that Novartis had commenced dosing in a phase II proof-of-concept clinical trial in a range of solid tumours.  In December 2014 Novartis informed Vernalis that it was ceasing all development work on this programme.

Project Status

Vernalis is in the process of regaining the rights from Novartis. A review of the data is being undertaken before deciding next steps and how best to take the programme forward.

Latest News

Servier and Vernalis plc announce achievement of 3 milestones in their oncology drug discovery collaboration

Servier and Vernalis plc are pleased to announce the achievement of two research milestones and one clinical milestone in their oncology drug discovery collaboration, triggering a total payment of €2 million to Vernalis.


Read more ...

At a glance

More information on Vernalis and its pipeline ...

FactsheetNov14 Company fact sheet

Comm thumbnail Explore the pipeline ...

Contact Vernalis

T: 0118 938 0000
F: 0118 938 0001

Contact information
Maps and directions
Contact form

To report SUSPECTED ADVERSE REACTIONS, contact Vernalis Therapeutics, Inc. at 1-855-705-9546 and drugsafety@propharmagroup.com or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch