Oncology and CNS Discovery

At Astex, we are focusing our creative energy and innovative ability on discovering novel therapies that will make a real difference to patients with cancer or diseases of the CNS.

Our open innovation approach

As well as carrying out research in-house we embrace the powerful synergies that collaborations and open innovation brings in accelerating our understanding of disease biology and in enabling the journey from the discovery laboratory to treating patients. In addition to working with a range of global partners from academic centers, disease charities and industry, we also have several joint oncology and CNS projects with scientists from our sister companies, Otsuka Pharmaceuticals, and Taiho Pharmaceutical.

Our discovery strategy – precision medicine

In both oncology and diseases of the CNS we are pursuing a strategy that aims to first understand the molecular basis of the disease and then to develop a targeted therapy which can arrest or reverse its progression; an approach generally referred to as ‘precision medicine.’

Our Pyramid™ platform plays a pivotal role in the rational design of novel small molecule targeted therapies. These therapies are based on very small, low molecular weight fragments which we then further design so that they interact optimally – either through inhibition or activation – with an oncology or CNS protein target. Additionally, as molecules are rationally designed this allows us to potentially control the physicochemical properties and molecular weight of compounds and thus increase the chances of these compounds being able to cross the blood-brain barrier and engage the molecular target within the CNS.

Oncology programs

We have successfully generated a robust proprietary clinical pipeline of multiple novel targeted oncology therapies with multiple drugs in mid-late stage clinical development worldwide, as well as a number of partnered products being developed by leading pharmaceutical companies.

Our oncology discovery programs further expand our pipeline. They combine pre-clinical drug and biomarker discovery approaches using molecular and genetic data to identify new targets in cancer cells that can then be treated with our novel targeted small molecule therapies.

CNS programs

We are developing a product portfolio of novel targeted therapeutics for defined neurodegenerative disease populations. Our research programs are focused on examining the underlying molecular processes of neurodegenerative diseases to identify targets and biomarkers for efficacy and patient selection.

Informed by the latest genetics research and advances in human disease understanding, target discovery is focusing on areas of biology that increase an individual’s vulnerability to developing a neurodegenerative disease. We are currently exploring some of the common neuropathological mechanisms that could yield new targets, these include mitochondrial quality control, proteostasis and neuroinflammation.

We are investigating a range of neurodegenerative diseases including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis and frontotemporal dementia. We will also examine orphan CNS diseases with a clear genetic association, as well as epigenetic targets.