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