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  The Independent Research Enabler

Call for Proposals for Target Assays and Chemical Scaffolds

The European Lead Factory is looking for biology targets and, from January onwards, also for chemical scaffolds. Companies and academic institutions can submit their proposals via the European Lead Factory website.

The European Lead Factory urgently needs the best target assays from academics and SMEs. It’s your chance to take part in something big. Find out how a major pan-European project is taking open innovation to the next level.

The European Lead Factory is an international consortium of 30 partners funded by the European Innovative Medicines Initiative (IMI). It aims to provide best-in-class resources and in-kind funding to academia and SMEs working on promising biology targets or chemistry scaffolds. The goals are to promote cutting-edge research and to help transform that research quickly into high-quality drug lead molecules on an unprecedented scale. The benefits of submitting proposals are multiple for both target program owners or compound contributors. SMEs and academia can now join a highly specialized team within this large open innovation platform for drug discovery, and gain access to state-of-the-art resources. 

Today, 300,000 high-quality compounds contributed by seven European Federation of Pharmaceutical Industries and Associations (EFPIA) partners have been collected. The European Lead Factory’s world-class chemistry assets are designed to facilitate the synthesis and addition of around 200,000 novel compounds. This will ultimately be resulting in a Joint European Compound Collection (JECC) of around 500.000 compounds.

Find out how you can benefit by submitting your assays! Visit the European Lead Factory website for details.

A Portfolio of Partnerships: FES in Review

In 2007, TI Pharma started a strategic research program with funding support from the government. That program is now drawing to a close, and its portfolio of 74 scientific projects is being reviewed.

A committee of internationally leading scientists in drug discovery and development is currently looking back at our FES program, which concludes at the end of this year. The committee’s findings will be shared at our annual Spring Meeting on April 15, 2014 with our partners, the Dutch government and other stakeholders.

Join us at the Spring Meeting to find out what can be achieved when multidisciplinary partnerships work towards scientific breakthrough.

Mark your calendar!

TI Pharma Spring Meeting
Date: April 15, 2014
Location: De Rijtuigenloods in Amersfoort, the Netherlands

Paving the Way for Ultra-Rare Disease Therapies

55 million people in the EU and US suffer from a rare disease. For many, there is neither cure nor even effective treatment.

Rare diseases are a serious health issue. These some 6000-8000 diseases are genetic in origin; they are often chronic, progressive, debilitating, and even life-threatening. Many affect children at a very young age. More prevalent rare diseases, like Cystic Fibrosis and Amyotrophic lateral sclerosis, are well-known. But there are thousands of other rare diseases virtually unknown to the general public, such as Niemann-Pick type C, Darier’s disease, and alkaptonuria. 

What is the level of scientific knowledge on ultra-rare metabolic inherited diseases?
How many of these diseases currently have therapies in development?

These questions were addressed by Remco de Vrueh, program manager at TI Pharma, together with Escher PhD student Michelle Putzeist of the University of Utrecht. Their study was recently published in the Orphanet Journal of Rare Diseases.

Their study confirmed that knowledge on the pathophysiological consequences of rare disorders is available, and that this knowledge can be a driving force behind accelerating research into the development of novel therapies. The study was inspired by the observation that, although not in abundance, approved orphan medicinal products for exceptionally rare diseases (<10 patients per 1 million) certainly exist. In total, 166 rare metabolic genetic diseases were included in the analysis. For 42 (25%) of the diseases, an orphan designation application was submitted with either the FDA or EMA before January 2012. Explanatory factors included in the study ranged from prevalence to metabolic disease class, from identification of the causative gene to clinical data availability. A multivariate analysis identified prevalence of the disease and the availability of a preclinical proof of concept of a potential treatment as major determinants for the initiation of an orphan drug development program.

Senior author of the article Remco de Vrueh explains, "The key message of the study is that development for an ultra-rare metabolic disorder may be tough, but certainly not impossible. Not only do we reveal that for a quite a number of ultra-rare metabolic disorders a treatment is being developed, but that for some of these disorders, for example type I tyrosinemia, a marketed product has indeed become a reality. In several instances, patients, or their parents, seem to be pivotal in generating the necessary disease knowledge and translating this knowledge into an orphan drug development program."

Founder of Rare disease matters

Disease registries play a key role in making this knowledge available in a structural way. As part of the TI Pharma project Orphan Drugs Registry, the Dutch Diagnosis Registration of Metabolic Diseases (DDRMD) database is being built.

Life Sciences Academy: A Chance to Win €500,000

Enrollment for the first courses begins in February. Gain invaluable experience and boost your career!

The Life Sciences Academy will begin its first two courses in June 2014 in Leiden. The first course will focus on clinical development and clinical trial applications, and is being developed in close collaboration with the Center for Human Drug Research (CHDR). A second course will address intellectual property (IP) practices, and is being developed together with Leiden University Research and Innovation Services (LURIS).

On top of the educational experience, a total €500,000 in grants is available from the Netherlands Organization for Health Research and Development (ZonMw) to finance the execution of the best clinical trial applications with licensed medicinal products that are developed during the clinical development and clinical trial application course.

The courses at the Life Sciences Academy revolve around “learning by doing”, and aim to educate a new generation of leaders in life sciences and health, who can take on the complex challenges of the development process. Dedicated teaching case studies with group assignments are being developed based on real-life cases. Top experts are being recruited to provide the latest insights in the various aspects of clinical development and intellectual property practices. The two courses form a starting point towards the development of a full curriculum that will entail the complete discovery, development, and delivery process combined with entrepreneurship and project management skills.

Complement and extend your current knowledge by joining the first courses at the Life Sciences Academy. The academy’s website will go live in February, and enrollment will start. Stay up-to-date via this newsletter!

Why Animal Studies Are Not Always Necessary

In December, two PhD students, Peter van Meer and Marlous Kooijman, will defend their theses which both address the predictive value and thus the use of animal testing. The theses are published in the TI Pharma project “Predictive value of animal testing”.

These theses entail four years of research into the scientific basis for the testing of new drug candidates in animals. Such tests are mainly performed to establish the safety and quality of potential new medicines. But initial research findings, first published in EOS Magazine in an article titled “Een nutteloze dood”, already demonstrated that many animal tests are not very useful in this respect. The final theses further elaborate on this, and provide a scientific basis towards further development of animal-free testing of pharmaceuticals.

The Principal Investigator of this project, Professor Huub Schellekens, recently won the “Lef in het Lab Award” for his critical assessment of animal testing.

Peter van Meer: “The scientific value of non-clinical animal studies in drug development”

Marlous Kooijman: “Why animal studies are still being used in drug development” (available after December 20, 2013)

2014 TI Pharma Annual Spring Meeting event | Save the date!

Join us for the 7th edition of the TI Pharma Spring Meeting event on April 15th, 2014.

Photo: Rijtuigenloods

Find out what can be achieved when multidisciplinary partnerships work towards scientific breakthrough. Concrete examples and results from the concluding TI Pharma government co-funded FES portfolio will demonstrate best practices and lessons learnt.

Extend your network. Connect, interact and exchange knowledge with a large variety of participants. Meet experts from academia, SMEs, major pharma, representatives of patient organizations, relevant healthcare foundations as well as policy makers.

More details on the program, topics and speakers will follow in January.

Mark your calendar!

TI Pharma Spring Meeting
Date: April 15, 2014
Location: De Rijtuigenloods in Amersfoort