“Adverse Events” and “Serious Adverse Events”

an adverse event is any undesirable experience associated with the use of a medical product in a patient. The event is serious and should be reported to the FDA and/or EMA as appropriate when the patient outcome is: death, life- threatening, hospitalisation (initial or prolonged), disability or permanent damage, congenital anomaly/birth defect, required intervention to prevent permanent impairment or damage (devices), other serious (important medical events, may jeopardise the patient and may require medical or surgical intervention [treatment] to prevent one of the other outcomes)

“Antisense”

a nucleotide sequence that is complementary and therefore binds to a sequence of another nucleotide and can prevent its function

“Antisense Inhibitor”

a molecule which inhibits the function of DNA or RNA by the way of the complementary binding action of antisense DNA or RNA; used, for example, to refer to alicaforsen, which binds to a specific messenger RNA molecule and inhibits production of the protein ICAM-1

“API”

active pharmaceutical ingredient

“Biologics” or “Biologicals”

medicinal preparations made from living organisms and their products, including sera, vaccines, antigens, antibodies and antitoxins

“cGMP” or “GMP”

(current) Good Manufacturing Practices, general methodologies and procedures that are to be followed in the testing and manufacture of pharmaceuticals so as to ensure a consistent quality of product, as mandated by relevant regulatory authorities

“CMO”

Contract Manufacturing Organisation, an organisation that provides contract manufacturing services to biotechnology and pharmaceutical companies

“CRO”

Clinical Research Organisation, an organisation that provides outsourced clinical trial services to biotechnology and pharmaceutical companies

“Data Exclusivity”

during a period of Market Exclusivity for a drug, Data Exclusivity applies to ensure that confidentiality of data relating to that drug is maintained, preventing development of generic versions of the originator drug

“Disease Activity Index”

a research tool used to quantify the signs (seen by the doctor) and symptoms (experienced by the patient) of a patient’s disease

​“DNA”

deoxyribonucleic acid is a molecule, made up of nucleotides, that encodes the genetic instructions in living organisms; DNA consists of two strands of a sequence of nucleotides making up genes (being regions of DNA that code for a specific proteins)

​“EMA”

European Medicines Agency, the European agency for protecting and promoting public health through the supervision of, amongst other things, prescription products

“Enema”

the means of introducing a solution into the rectum and colon via the anus

​“Fast Track”

a process designed to facilitate the development and expedite the regulatory review and approval process for drugs to treat serious diseases where there is an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious diseases

​“FDA”

Food and Drug Administration, the United States agency for protecting and promoting public health through the supervision of, amongst other things, prescription products

“First-In-Class”

a first-in-class drug is a product with a novel mechanism of action (that differentiates it from any other product on the market) being investigated in a clinical trial, or marketed, for the first time

​“Gastrointestinal (GI) Tract”

the gastrointestinal or GI tract runs from the mouth to the anus and is responsible for digestion and processing of foodstuffs. The upper part of the GI tract encompasses the oesophagus, stomach and small intestine. The lower part of the GI tract comprises the large intestine, which may be further divided into ascending, transverse and descending colon, and the rectum

​“Generic” or “Generic Drug”

a generic drug is a drug product that is comparable to a brand/reference listed drug product in dosage form, strength, route of administration, quality and performance characteristics, and intended use. A generic drug is manufactured and marketed once the originator’s period of patent protection and Market Exclusivity has lapsed

“ICAM-1”

ICAM-1 (“Intercellular Adhesion Molecule-1”) is a protein that in humans is encoded by the ICAM-1 gene. This gene encodes a cell surface glycoprotein which is typically expressed on endothelial cells and cells of the immune system. ICAM-1 may be significantly over- expressed during inflammation and contribute to the overall inflammatory response

“Ionis”

Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals, Inc.), a US biotechnology company quoted on NASDAQ/(NASDAQ:IONS) and the originator of alicaforsen

​“KOLs” or “Key Opinion Leaders”

specialist physicians recognised in their field of expertise

​“Letter of Fast Track”

the designation letter is issued by the FDA if it determines that the criteria for designation as a U.S. fast track drug development program have been met

​“Market Exclusivity”

the period of time during which a new drug is protected from direct competition by generic versions of the drug. Market exclusivity is provided by regulators through a number of mechanisms including granting of Orphan Drug designation

​“Marketing Approval”

approval by the relevant regulatory body (such as the FDA or EMA) for the marketing of a compound as an approved drug in the relevant territory

​“MHRA”

Medicines and Healthcare Products Regulatory Agency, the UK agency responsible for protecting and promoting public health through the supervision of, amongst other things, prescription products

​“Named Patient Supply” or “NPS”

often grouped under the labels of compassionate use, special access, expanded access, or named patient supply, regulatory agencies, such as the MHRA in the UK, have provisions to allow companies to provide patients with access to medicines while they are still undergoing clinical trials prior to Marketing Approval. Strict regulations governing such access define access criteria, data collection, non-promotion, and control of drug distribution

​“NCE” or “New Chemical Entity”

a New Chemical Entity or NCE is a synthetic (chemically synthesised) molecule that has not previously received Marketing Approval from the relevant regulatory authorities

​“Nucleotide”

a building block of DNA and RNA

“Orphan Drug”

a drug intended for the safe and effective treatment or prevention of rare diseases/disorders that affect small numbers of patients. (defined in the U.S. as those affecting fewer than 200,000 people and in Europe not more than 5 in 10,000 people). Orphan drug status conveys a number of incentives for companies to develop therapies. Orphan Drugs also generally command higher drug prices and can be funded through separate budgets for rare diseases

​“Phase 1”

the first phase of clinical trial testing in humans for a new drug compound or treatment to evaluate its safety, determine a safe dosage range and identify any common side effects. Phase 1 trials are normally conducted on a limited number of subjects. Subjects for Phase 1 trials are commonly healthy volunteers rather than patients

​“Phase 2”

the second phase of clinical trial testing for a new drug compound or treatment, conducted if Phase 1 trials have progressed satisfactorily. Phase 2 trials are intended to assess side effect potential and some measures of efficacy and dose response with respect to a particular indication or indications in patients with the disease or condition under study

​“Phase 3”

the third stage of clinical testing, conducted to determine whether a new drug compound or treatment can be granted regulatory approval for distribution and sale. Phase 3 trials involve a larger group of patients and take place after obtaining preliminary evidence of efficacy and are intended to help determine risk, benefit and appropriate labelling

​“Pre-Clinical Development”

the development of a drug compound prior to clinical development (Phase 1-3), including a series of effectiveness and safety tests before it is used in humans in order to be as certain as possible that there will be a positive effect and no serious adverse effects

​“RNA”

ribonuncleic acid (“RNA”) is a single strand sequence of nucleotides analogous to the “sense” strand of the DNA sequence of a gene. RNA is involved in the expression (manufacture) of proteins (such as ICAM-1)

​“Specialist-Led Hospital Sector”

the market for drugs in which a hospital- or specialist care centre-based doctor or consultant/specialist initiates a prescription

​“Specialist-Led” or “Speciality Products”

a specialist-led or speciality product is a product that targets a disease with a small-to-medium population that has significant unmet medical need and is typically prescribed by specialist practitioners (hospital- or specialist care-centre based doctors and consultants); such products may command a premium price and may be administered as part of a wider disease management programme

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