Summary of the impact

Innovative formulation science to create and develop the commercially successful PowderHale^® technology was undertaken within the Department of Pharmacy & Pharmacology at the University of Bath, and subsequently by Vectura. This has directly provided the basis for novel, potentially life-saving treatments for chronic obstructive pulmonary disease (COPD). Seebri^® Breezhaler^® and Ultibro^® Breezhaler^® are once-daily, maintenance bronchodilators for the relief of various symptoms due to airways obstruction caused by COPD. Seebri^® Breezhaler^® was approved in the EU and Japan at the end of 2012 and has now been launched by Novartis. Ultibro^® Breezhaler^® a first-in-class combination bronchodilator was approved in Japan and the EU in September 2013. Under the terms of the licence agreement with Novartis concerning these products, Vectura has already received $52.5M with an additional >$100M anticipated upon achievement of regulatory and commercialisation targets. These medicines are major advances to treat and manage a disease that, according to the WHO, affects an estimated 210 million people worldwide and was the third leading cause of death in the developed world in 2012.

Summary of the impact

The development of new paradigms for toxicity testing has benefitted the Scottish economy and population in Tayside through two biotechnology companies which, between them, employ up to 40 staff and have had a combined turnover of some £15M over the last five years. The benefits extend to the international pharmaceutical, cosmetic, chemical and consumer product industries, which have gained access to innovative new methods for safety testing at a time of acute need for more predictive methods to evaluate drug safety and better in vitro tests for consumer products. Patients and consumers in Europe and worldwide have benefitted indirectly from improved risk assessment of drugs, consumer products and environmental contaminants.

Summary of the impact

i2c Pharmaceutical Services is the trading name for a Cardiff University spin-out company based on Cardiff University research excellence and specialising in pharmaceutical inhaler product research and development. i2c's research in formulation technologies and clinical testing has enabled development of new inhalational medicinal products for the healthcare markets in both developed and emerging countries. Impacts arising from research are at local, national and international levels and evidenced by marketed products, the improved business performance of commercial concerns and the creation of highly skilled jobs.

Summary of the impact

The provision of effective and sustainable healthcare is a major challenge for society. In the developed world escalating costs are placing a huge burden on finite resources; in the developing world, where financial resources are often extremely limited, providing affordable healthcare is an even greater problem. One innovative route to help alleviate these problems is through drug redeployment, whereby existing drugs are employed in new ways to tackle serious diseases. Combining their knowledge of haematological disease gained from their research over the past 20 years together with a drug redeployment strategy, researchers in the School of Biosciences have developed and trialled new interventions for two blood cell cancers, Acute Myeloid Leukaemia (AML) and Burkitt's Lymphoma (BL), based on the administration of a combination of the lipid lowering drug Bezalip (Bez) and the female contraceptive Provera (MPA). As a result:

• Definitive significant outcomes have been demonstrated in terms of halting disease progression and / or diminishing disease load in patients suffering from AML and BL.
• Successful drug redeployment, on the basis of efficacy, absence of toxicity and low cost of drugs has been achieved.
• This intervention has created the means to reduce childhood mortality and improve the length and quality of life in areas of sub-Saharan Africa.

Summary of the impact

Impact: Economic. The EaStCHEM spin-out company Deliverics has commercialised biodegradable transfection reagents for both the "research tool" and the "RNAi therapeutics" markets (globally valued at £400M and £4 billion respectively). Beneficiaries are the pharmaceutical and biotechnology sectors, and clinicians. The turnover since 2010/11 is £330k and the company currently has five employees.

Significance: Deliveric's agents out-perfom existing materials in term of efficacy and reduced levels of toxicity. They are not hampered by the immunogenicity, manufacturing issues, and carcinogenicity previously seen for viral vectors used as delivery agents. This presents a wide ranging ability to deliver nucleic acids into cells and tissues for biological applications.

Research; date; attribution: EaStCHEM research (2008) led by Bradley reported a family of non-viral DNA delivery agents that offered a highly-efficient and non-toxic method of delivering siRNA/DNA into mammalian cells and tissues. Development and patenting of this technology led to the spin-out of Deliverics Ltd. in 2010.

Reach: International customer base (20 research groups and 10 companies) including specially appointed distributors in Spain (Albyn Medical), South Korea (CoreSciences), and US (Galen).

Summary of the impact

The university's Pharmacy and Pharmacology unit has developed and validated novel in silico and in vitro/ex vivo models for use by the pharma industry to select drug candidates, optimise formulations, determine the posology for clinical trials and show bioequivalence. This resulted in: the approval of two products for actinic keratosis (Picato® and Zyclara®); a generic nail formulation approved for use based on the demonstration of equivalence using the in vitro/ex vivo models described with no clinical testing (the first time this has occurred); and the translation and commercialisation of two dermal drug delivery-based patented technologies (licensing deals with Sinclair IS and major pharmaceutical companies).

Summary of the impact

The transition, at the end of the 20^th century, from ozone-depleting chlorofluorocarbons (CFCs) to hydrofluoralkane (HFA) propellants in metered dose inhalers (MDIs), for drug delivery to the upper airways in the lungs, taxed the ingenuity of formulation scientists and device design engineers. The regulatory requirement for clinical equivalence between the CFC and HFA products demanded an unchanged drug dosing regimen and identical lung deposition profiles.

Research funded by Chiesi Farmaceutici (Parma, Italy) in the Centre of Drug Formulation Studies (CDFS) at the University of Bath led to development of the Modulite^® technology which met the challenges posed and mimicked the performance of CFC MDI using HFA propellants. The proprietary technology enabled Chiesi to re-formulate and commercialise a number of products, which now represent mainstay therapies in the treatment of asthma and chronic obstructive pulmonary disease (COPD).

The Modulite^® technology has provided the greatest contribution to both the turnover and the global development of the Chiesi group, via several successful in-house developmements and collaboration agreements with leading pharmaceutical companies. Global sales of Chiesi's Atimos Modulite^®, Fostair/Foster (25% of sales) and Clenil Modulite^® (14.4% of sales) MDI products produced revenue of in excess of $450 Million in 2012.

Summary of the impact

Cancer research at the University of Salford focuses on developing new and improved treatments for cancer, particularly for children with cancer, demonstrating the following impact:

• The development of RH1, a novel anticancer drug and a second generation novel agent, Es5, arising from RH1;
• Participation in clinical studies in paediatric and adult cancers with North West, UK and international partners in the health, charitable and commercial sectors to trial and develop the technologies;
• The establishment of spin-out company, Onco-NX to develop and exploit the technologies and IP arising from the research;
• The establishment of Kidscan, a University-based registered charity to support research into new and improved treatments for children with cancer and generating dedicated support for and commitment to cancer research among North West UK communities.

Summary of the impact

Research by Professor Abdul Basit's group at the UCL School of Pharmacy is leading to improved treatments for ulcerative colitis and other conditions through increased knowledge of the complex physiology of the gastrointestinal tract. Improved understanding of in vivo drug release and uptake has allowed development of three patent-protected technologies for improved drug delivery: PHLORAL^TM, for release of drugs in the colon, and DuoCoat^TM and ProRelease^TM formulations designed to allow intact transit through the stomach followed by immediate release upon gastric emptying. These technologies are the subject of licences and ongoing development programmes, with PHLORAL^TM currently in phase III clinical trials. The impact is therefore the introduction of enabling technologies that have positively influenced the drug development programmes of pharmaceutical companies.

Summary of the impact

ProTide technology, discovered by the McGuigan team at Cardiff University, is a pro-drug strategy with proven capacity to generate new drug candidates for nucleoside-based antiviral and anti- cancer indications. In the assessment period the McGuigan team has attracted more than £2 million direct research funding through sustained collaborations on ProTide technology with global pharmaceutical companies and smaller biotech firms in the USA and Europe. In the same period, either through working directly with Cardiff or by independent adoption of McGuigan's research, eight ProTide entities have progressed to clinical trials as cancer, HIV and hepatitis C treatments. The technology is demonstrating significant commercial impact for companies with ProTide-based drug candidates.