Glossary

AE Adverse events.

Alkylator A broad spectrum cytotoxic chemotherapy.

Aminopeptidases Enzymes that hydrolyze peptides. Are overexpressed in cancer cells.

CBR Clinical benefit rate, measures the number of patients with multiple myeloma who have lost 25 percent or more of their tumor mass.

CDMO Contract development and manufacturing organization.

Chemotherapy Drug administered to kill cancer cells.

Clinical studies Studies to define doses and evaluate safety and efficacy on healthy volunteers and patients.

CR Complete tumor response.

CRO Contract research organization.

Dexamethasone A potent steroid used in cancer treatment.

DOR Duration of response refers to the period from an initial tumor reduction until it begins to grow.

Double-refractory Refractory to two drugs.

EMA European Medicines Agency.

Entrapped Term that describes how a hydrophilic alkylator payload stay inside a cell.

FDA US Food and Drug Administration.

Hematology The science of blood, blood-forming organs and blood diseases. It includes the treatment of blood disorders and malignancies, including hemophilia, leukemia, lymphoma and sickle-cell anemia.

Heterogeneous disease A disease comprising different but similar sub-diseases.

IMiDs Immunomodulatory imide drugs, used in the treatment of multiple myeloma.

Interim results Partial results in ongoing trials.

IND Investigational New Drug.

IND-submission Application to enable clinical development of a drug candidate.

INN International non-proprietary name.

Late-stage RRMM Late-stage relapsed refractory multiple myeloma.

Lines of therapy After a cancer diagnosis and decision to treat the patient, the first treatment attempt is known as the first line of therapy, followed by a second line of therapy, etc.

Lipophilicity is a key parameter that determines?cell uptake?of small molecules.

MAA Marketing Authorization Application.

Melflufen A first in class peptide-drug conjugate that targets aminopeptidases and releases alkylating agents into tumor cells.

Melphalan flufenamide INN name of melflufen.

MM Multiple myeloma, a rare blood cancer that forms in plasma cells. Cancerous plasma cells accumulate in the bone marrow and crowd out healthy blood cells.

Monoclonal antibodies Laboratory-produced molecules engineered to serve as substitute?antibodies?that can restore, enhance or mimic the immune system’s attack on?cancer?cells.

MR Minimal response refers to a 25–50 percent tumor reduction.

Multi-refractory Refractory to several different drugs.

Multiple myeloma A rare blood-based cancer.

NDA New Drug Application.

OPD5 The second drug candidate coming out of the peptide drug conjugate platform.

Orphan drug A drug used to treat a rare disease.

Orphan designation A status assigned to a medicine for a rare condition. It must fulfil certain criteria to benefit from incentives such as protection from competition once it is on the market.

ORR Overall response rate, the number of patients who have lost 50 percent or more of their tumor mass.

OS Overall survival, the length of time a patient survives from the start of the treatment.

Payload Highly active molecules that are too toxic to be administered in untargeted forms at therapeutic doses.

PD Progressive disease, where the tumor mass has grown by at least 25 percent.

PDC Peptide-drug conjugate. The class of agents that includes melflufen and OPD5.

Peptidases Enzymes that break down peptides.

Peptide A molecule comprising a chain of amino acids. Key attribute to melflufen.

PFS Progression-free survival, measures the length of time from the start of the patient’s treatment until the tumor has grown by at least 25 percent.

Pharmacokinetics Data that describe how a drug is distributed and metabolized in the body.

Phase 1, 2, 3 study Various phases of clinical development.

Phase 1 A study to identify appropriate dose and evaluate safety in healthy volunteers

Phase 2 A study to evaluate efficacy and safety in patients ahead of phase 3, which repeats this process in larger patient groups and in comparison to other treatments.

PI Proteasome inhibitor (PI) used in multiple myeloma treatment.

Pivotal study A clinical study to demonstrate the safety and efficacy of a new drug in order to obtain its marketing approval by regulatory authorities).

PK Pharmacokinetics, how the drug is distributed and metabolized in the body.

PR Partial response refers to a tumor reduction of 50 to 90 percent.

Preclinical studies Early research during which feasibility, iterative testing and drug safety data are collected. Determine a safe starting dose for?first-in-human study?and assess potential?toxicity.

Progression-free No tumor growth.

Proteasome inhibitor Substance used in multiple myeloma treatment.

Quad- and penta-refractory A patient who´s disease is refractory to four or five different treatments.

Randomized clinical trial A study in which patients are randomly divided into different groups.

Refractory Resistant to treatment.

Relapse Recurrence of e.g. a tumor.

Resistance development Tumor development causing worse or no response to treatment.

RRMM Relapsed refractory multiple myeloma.

SD Stable disease where the tumor has neither grown nor shrunk by 25 percent.

Single-arm study Clinical study in which patients receive the same treatment.

Target protein Protein to which a drug binds in order to release a pharmaceutical effect.

TCR Triple class refractory, patients are refractory to or intolerant of at least one immunomodulating drug, at least one proteasome inhibitor, and at least one anti-CD38 monoclonal antibody.

Triple class refractory, patients are refractory to at least one proteasome inhibitor, one immunomodulatory drug and one anti-CD38 monoclonal antibody.

Tumor response rate Percentage of patients whose tumors respond to treatment.

VGPR Very good partial response.