Glossary

AGM

Annual General Meeting

AMR

Antibody mediated rejection. Major cause of graft loss in HLA-incompatible kidney transplants

Antibody

One type of protein produced by the body’s immune system with the ability to recognize foreign substances, bacteria or viruses. Antibodies are also called immunoglobulins.

 Anti-GBM disease (Goodpasture syndrome)

Anti-GBM disease is a rare autoimmune disorder in which circulating antibodies are directed against an antigen normally present in the glomerular basement membrane (GBM) in the kidney.

Antigen

Substance usually protein based, that stimulates production of antibodies that face the perceived threat.

Autoimmune disease

Diseases that occur when the body’s immune system reacts against the body’s own structures.

Bacteria

Bacteria are single-cell organisms that can cause infections

B-cells

B-cells, also known as B-lymphocytes, are a type of white blood cell of the lymphocyte subtype. They are an important part of the adaptive immune system and secrete antibodies.

Biopharmaceutical

A pharmaceutical drug that is manufactured using biotechnology.

Biotechnology

The use of live cells or components of cells, to produce or modify products used in health care, food, and agriculture.

CD20

B-lymphocyte antigen CD20 is a protein expressed on the surface of B-cells. Its function is to enable optimal B-cell immune response.

Clinical studies

Investigation of a new drug or treatment using healthy subjects or patients with the intention to study the efficacy and safety of a not-yet-approved treatment.

Clinical Phase 1

The first time a drug under development is administered to humans. Phase 1 studies are often conducted with a small number of healthy volunteers to assess the safety and dosing of a not yet approved form of treatment.

Clinical Phase 2

Refers to the first time a drug under development is administered to patients for the study of safety, dosage and efficacy of a not yet approved treatment regimen.

Clinical Phase 3

Trials that involve many patients and often continue for a longer time; they are intended to identify the drug’s effects and side effects during ordinary but still carefully controlled conditions.

Controlled study

A clinical study comparing the efficacy of the drug in development against an approved drug or no treatment.

Crossmatch

A test where a sample of the recipient´s blood is mixed with a potential donor´s blood prior to transplantation, to demonstrate how a recipient´s antibodies will react with the potential donor.

Desensitization

A method to lower the levels of antibodies and thus reduce the body's negative reaction to a substance or stimulus.

DSA

Donor Specific Antibodies in a transplant patient are antibodies which bind to HLA and/or non-HLA molecules on the endothelium of a transplanted organ, or a potential donor organ. The presence of pre-formed and de novo (newly formed) DSA, specific to donor/recipient mismatches are major risk factors for antibody-mediated rejection.

EBITDA

Reported Profit before tax plus net finance expense, share of after-tax losses of joint ventures and associates and charges for depreciation, amortisation and impairment.

Efficacy

The degree to which a drug produces the intended therapeutic effect.

EGM

Extraordinary General Meeting.

EMA

The European Medicines Agency (EMA) is a European Union agency for the evaluation of medicinal products.

Enzyme

A protein that accelerates or starts a chemical reaction without itself being consumed.

FDA

U.S. Food and Drug Administration.

Gene therapy

Insertion of normal or genetically altered genes into cells to obtain a therapeutic effect.

GBS

GBS, Guillian-Barré syndrome, is an acute autoimmune disease in which the peripheral nervous system is attacked by the immune system and IgG antibodies.

HBP

Heparin Binding Protein is a naturally occurring protein that is produced by certain immune cells, i.e. neutrophilic granulocytes, to direct immune cells from the bloodstream into the tissues.

Highly sensitized patients

People in critical need of an organ transplant, where very few donors can provide a match.

HLA

Human Leukocyte Antigen is a protein complex found on the surface of all cells in a human. The immune system uses HLA to distinguish between endogenous and foreign substances.

IdeS

Immunoglobulin G-degrading enzyme of Streptococcus pyogenes, a bacterial enzyme with strict specificity for IgG antibodies. The enzyme has a unique ability to cleave and thereby inactivate human IgG antibodies while leaving other Ig-isotypes intact.

IgG

IgG, Immunoglobulin G, is the most common type of antibody in the blood and tissue. It is the primary source of antibody-based immunity against invading pathogens.

Imlifidase

Imlifidase is the generic name, International Non-proprietary Name (INN), for IdeS.

Immunology

Immunology is the study of the body's mechanisms or defending it selves against foreign materials that may cause a disease or infection.

IND

Investigational New Drug, submission of request to commence clinical trials to develop a new pharmaceutical product.

INN

International Non-proprietary Name (INN) is a generic and non-proprietary name to facilitate the identification of a pharmaceutical substance or active pharmaceutical ingredient.

In vitro

Term within biomedical science to indicate that experiments or observations are made, for example in test tubes, i.e. in an artificial environment and not in a living organism.

In vivo

Term within biomedical science to indicate that experiments or observations are made in living organisms.

IVD

IVD, in vitro diagnostics, are tests that can detect diseases, conditions, or infections, usually from blood samples or urine samples. Some tests are used in laboratory or other health professional settings, and other tests are for consumers to use at home.

KAS

Kidney Allocation System

NDA

New Drug Application, submission of request for license to market a new pharmaceutical in the U.S.

MSL

A healthcare professional who works closely with key opinion leaders to share preliminary findings from ongoing clinical trials, information about planned clinical studies, and data on off-label drug use with interested prescribers, as allowed under guidelines set forth by the FDA.

Negative crossmatch

The recipient´s antibodies do not work against the donor´s and are suitable for transplant.

Operating profit

Sales, less cost of sales, less operating costs, plus operating income.

Orphan drug

A pharmaceutical agent developed for use in rare medical conditions called orphan diseases, which affect a small percentage of the population.

Orphan designation

Orphan designation or orphan status provide companies with specific incentives to facilitate development of drugs for rare diseases. This includes an extended market exclusivity beyond patent coverage.

Pivotal trial

A clinical trial intended to provide efficacy and safety data for NDA approval at e.g. FDA or EMA. In some cases, Phase 2 studies can be used as pivotal studies if the drug is intended to treat life-threatening or severely debilitating conditions.

Positive crossmatch

The recipient´s antibodies work against the donor´s and are not suitable for transplant.

PRA

Panel Reactive Antibody (PRA) is an immunological blood test routinely performed on people awaiting organ transplantation. The PRA score represents the proportion of the population to which the person being tested will react via pre-existing antibodies (0-99 %).

Pre-clinical development

Testing and documentation of a pharmaceutical candidate’s properties (e.g. safety and feasibility) before initiation of clinical trials.

Proof of Concept

The data verifying that a candidate drug provides a clinical change on a defined endpoint or surrogate in patients suffering from the disease.

Sensitized patients

People with antibodies that react towards foreign tissue, such as a transplanted organ from a living donor. Sensitization of the immune system may occur from blood transfusions, previous transplants or pregnancies.

Sepsis

Diagnosed or suspected infection in combination with the patient being in a systemic inflammatory state (SIRS). Clinical symptoms of systemic inflammation may be a combination of fever, increased heart rate and increased respiratory rate.

Severe sepsis

Sepsis is progressing into severe sepsis when the patient may suffer circulatory effects and reduced functions of vital organs such as the brain, heart, lungs, kidneys or liver.

Side effect

A clinically unwanted, unintended effect of a drug

Streptococcus pyogenes

A Gram-positive bacterium that causes a wide variety of acute infections, such as soft tissue infections and infections in the throat.

Transplant waitlist

Public listing of patients waiting to receive an organ from a deceased or living donor. Patients need to be listed to be eligible for a transplantation. There are specific lists for each organ, e.g. kidney transplant waitlist.