• Arises out of research collaboration with Novartis
• Potentially effective against a range of cancers
• Clinical proof of concept should be next development milestone

This is the first molecule arising out of an original research collaboration between Vernalis and Novartis, started in 2004, to enter clinical testing. Hsp90 inhibitors such as 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 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.

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. AUY922 has now moved into the clinical studies phase of development.

Our partner Novartis is currently evaluating 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, which triggered a $3 million milestone payment to us.

Pre-clinical studies completed. Phase II clinical studies now being undertaken by Novartis. Vernalis receives milestone payments during development and royalties on commercialisation of AUY922.