Glossary of terms
ACR
American College of Rheumatology
ADR
American Depositary Receipt; Certificate representing an American Depositary Share
ADS
American Depositary Share; One Galapagos ADS represents the right to receive, and to exercise the beneficial ownership interests in, one ordinary share in Galapagos NV on deposit with depositary Citibank, N.A.; Galapagos’ ADSs are listed on NASDAQ under the symbol “GLPG”
Bioavailability
Assessment of the amount of (candidate) drug that reaches a body’s systemic circulation after administration
Candidate drug
Substance that has satisfied the requirements of pre-clinical testing and has been selected for clinical testing for the treatment of a certain disorder in humans
CFTR
Cystic fibrosis transmembrane conductance regulator protein. CFTR is a protein involved in the transport of chloride ions across cell membranes. Cystic fibrosis patients have a genetic mutation resulting in absence of or dysfunction of CFTR
CIR
Crédit d’Impôt Recherche, or research credit. Under the CIR, the French government refunds up to 30% of the annual investment in French R&D operations, over a period of three years. Galapagos benefits from the CIR through its operations in Romainville, just outside Paris
Clinical Proof of Concept (PoC)
Point in the drug development process where the candidate drug shows efficacy in a therapeutic setting
Colitis ulcerosa/ulcerative colitis (UC)
see IBD
Compound
A chemical substance, often a small molecule with drug-like properties
Corrector drug
Drug that restores the protein forming the ion channel opening in cystic fibrosis patients. In most CF patients, a potentiator and corrector drug are needed in combination to restore the genetic defect causing CF. GLPG2222 and GLPG2665 are corrector drugs with different mechanisms of action
Crohn’s (CD)
see IBD
Cystic fibrosis (CF)
A life-threatening genetic disease that affects approximately 80,000 people worldwide. Although the disease affects the entire body, difficulty breathing is the most serious symptom as a result of frequent lung infections
Development
Process of bringing a new drug to the market. At Galapagos, this is the department which performs pre-clinical and clinical development research, clinical batch scale-up, and regulatory filings of Galapagos’ drug candidates
Discovery
Process by which new medicines are discovered and/or designed. At Galapagos, this is the department that oversees target and drug discovery research through to nomination of pre-clinical candidates
Drug development
Process of bringing a new drug to the market; includes both pre-clinical development and human clinical trials
Drug discovery
Process by which a (potential) therapeutic is either discovered or designed
Efficacy
Effectiveness for intended use
Fee-for-service
Payment system where the service provider is paid a specific amount for each procedure or service performed
FIH
First-in-human clinical trial, usually conducted in healthy volunteers with the aim to assess the safety, tolerability and bioavailability of the candidate drug
filgotinib
Also known as GLPG0634. Small molecule selective JAK1 inhibitor which showed excellent efficacy and safety in rheumatoid arthritis patients in Phase 2 trials in November 2011, November 2012, and DARWIN 1 & 2 studies reported in 2015. Currently being prepared by Galapagos to enter a Phase 3 study in rheumatoid arthritis, with a Phase 2 study topline result in Crohn’s disease expected before end 2015
FSMA
The Belgian market authority: Financial Services and Markets Authority
GLPG0634
See filgotinib
GLPG1205
Novel mode of action medicine in inflammatory bowel disease, fully owned by Galapagos, currently in a Phase 2 Proof-of-Concept study in ulcerative colitis
GLPG1690
A novel drug targeting autotaxin, with potential applications in idiopathic pulmonary fibrosis. Fully proprietary to Galapagos. Currently in preparations for the start of a Phase 2 Proof of concept study in IPF
GLPG1837
A potentiator drug which completed Phase 1 in 2015
GLPG2222
A corrector drug currently in pre-clinical candidate stage, which is expected to enter Phase 1 before end 2015
GLPG2665
A corrector drug currently in pre-clinical candidate stage
IBD
Inflammatory Bowel Disease. This is a general term for autoimmune disease affecting the bowel, including Crohn’s disease and ulcerative colitis. Crohn’s disease affects the small intestine primarily, while ulcerative colitis affects the large intestine. Both diseases involve inflammation of the intestinal wall, leading to pain, bleeding, and ultimately in some cases removal of bowel tissue
IPF
Idiopathic pulmonary fibrosis. A chronic and ultimately fatal disease characterized by a progressive decline in lung function. Pulmonary fibrosis involves scarring of lung tissue and is the cause of shortness of breath. Fibrosis is usually associated with a poor prognosis. The term “idiopathic” is used because the cause of pulmonary fibrosis is still unknown
Inflammatory diseases
A large, unrelated group of disorders associated with abnormalities in inflammation
In-/out-licensing
Receiving/granting permission from/to another company or institution to use a brand name, patent, or other proprietary right, in exchange for a fee and/or royalty
Intellectual property
Creations of the mind that have commercial value and are protected by patents, trademarks or copyrights
JAK
Janus kinases (JAK) are critical components of signaling mechanisms utilized by a number of cytokines and growth factors, including those that are elevated in rheumatoid arthritis
Milestone
Major achievement in a project or program; in Galapagos’ alliances, this is usually associated with a payment
MTX
Methotrexate
Oral dosing
Administration of medicine by the mouth, either as a solution or solid (capsule, pill) form
Orkambi®
Orkambi is a prescription medicine sold by Vertex Pharmaceuticals Incorporated, used for the treatment of cystic fibrosis (CF) in patients age 12 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene
Pharmacokinetics (PK)
Study of what a body does to a drug; the fate of a substance delivered to a body
Phase 1
First stage of clinical testing of a potential new treatment designed to assess the safety and tolerability of a drug, usually performed in a small number of healthy human volunteers
Phase 2
Second stage of clinical testing, usually performed in 20-300 patients, in order to determine efficacy, tolerability and the most effective dose to use
Phase 3
Large clinical trials, usually conducted in 300-3000 patients to gain a definitive understanding of the efficacy and tolerability of the candidate treatment by comparing it to the “gold standard” treatment; serves as the principal basis for regulatory approval
Potentiator drug
Drug that restores the ion channel opening in cystic fibrosis patients. In most CF patients, a potentiator and corrector drug are needed in combination to restore the genetic defect causing CF. GLPG1837 is a potentiator drug
Pre-clinical
Stage of drug research development, undertaken prior to the administration of the drug to humans. Consists of in vitro and in vivo screening, pharmaco-kinetics, toxicology, and chemical upscaling
Pre-clinical candidate (PCC)
A potential drug that meets chemical and biological criteria to begin the development process
Rheumatoid arthritis (RA)
A chronic, systemic inflammatory disease that causes joint inflammation, and usually leads to cartilage destruction, bone erosion and disability
R&D operations
Research and development operations; unit responsible for discovery and developing new candidate drugs for internal pipeline or as part of risk/reward sharing alliances with partners
Screening
Method usually applied at the beginning of a drug discovery campaign, where a target is tested in a biochemical assay against a series of small molecules or antibodies to obtain an initial set of “hits” that show activity against the target. These hits are then further tested or optimized
Service operations
Business unit primarily focused on delivering products and conducting fee-for-service work for clients. Galapagos’ service operations included the BioFocus and Argenta business units, which were both sold in April 2014 to Charles River Laboratories
Target
Protein that has been shown to be involved in a disease process and forms the basis of therapeutic intervention or drug discovery
Target discovery
Identification and validation of proteins that have been shown to play a role in a disease process