Improving people’s lives
We seek to discover, develop, and eventually commercialize medicines with novel modes of action, addressing disease areas of high unmet medical need. Our main mission is to improve lives with medicines which offer new treatment options to patients. Our pipeline comprises programs ranging from discovery to Phase 3 clinical trials in inflammation, fibrosis, osteoarthritis (OA), and other indications.
There is a real need for medicines with novel mechanisms of action. There are many diseases for which there is no approved therapy today and many more diseases for which current therapies leave room for improvement in clinical outcomes. New mechanism of action medicines offer opportunity for new clinical options for caregivers and patients, and could possibly decrease the burden for society, including lowered healthcare costs.
Our highly flexible target and drug discovery platform has been applied across many therapeutic areas, and our pipeline today ranges from inflammation to fibrosis candidate drugs.
Almost all of these programs are based on inhibiting targets which were identified using our proprietary target discovery platform. Using human primary cells, we discover which proteins (‘targets’) play a key role in causing diseases. We then discover and develop small molecules that inhibit these targets, restore the balance, and thereby positively influence the course of the disease. This approach addresses the disease itself rather than just treating the symptoms. In this way, we aim to make a lasting positive contribution to society through discovery of breakthrough therapies.
Our target discovery platform provides a significant and substantial competitive advantage as it:
- closely mimics the in vivo situation through the use of primary human cells with relevant trigger and readout for a specific disease phenotype
- identifies possible points to intervene in a disease pathway by knocking down an individual protein in these assays; and
- enables us to analyze rapidly all of the drugable genome and select pharmaceutically tractable protein targets directly by their ability to regulate key disease biology
We aim to initiate a Phase 3 trial every other year, while conducting three proof-of-concept trials, delivering three preclinical product candidates and six new validated targets every year following the determination of more stringent, general target validation criteria in 2018. We aim to select promising programs for internal development and commercialization and establish ourselves as a fully integrated biopharmaceutical company.
Improving people’s lives - 2018 actions
- We delivered 2 new validated targets, compared to our goal of 6
- We nominated 4 new preclinical candidates, all with a novel mechanism of action, compared to our goal of 3
- We started 4 proof-of-concept trials, compared to our goal of 3
- We initiated the ISABELA 1 & 2 Phase 3 program, meeting our goal of 1
- These successes brought us to 41 preclinical candidates since 2009, most of which have novel modes of action. Of these 19 have entered the clinic, 12 with novel modes of action
- We dedicated 50 FTEs to discovery efforts exploring the Toledo class of targets in inflammation
- Expand capabilities to support more than 40 planned clinical trials in 2019
- Continue to deliver on our annual research & development ambition targets
- Invest in our target discovery capabilities to maintain our competitive edge in novel targets