Ygalo®, an alkylating peptide, belongs to a novel class of peptidase-enhanced compounds (PEnCs) and targets the multiple myeloma (MM) tumor transformation process with a unique mechanism of action. Aminopeptidases are heavily over-expressed in MM and are key to the transformational process of the tumor cells. Ygalo® selectively targets MM through aminopeptidase-driven accumulation; in vitro experiments show a 50-fold enrichment of alkylating metabolites in MM cells. The enrichment results in selective cytotoxicity (increased on-target potency and decreased off-target toxicity), and that resistance pathways of existing myeloma treatments (including alkylators) is overcome. Ygalo® also demonstrates strong anti-angiogenic properties.
Ygalo® is intended to be the first choice for the treatment of patients with late-stage relapsed and refractory multiple myeloma. Multiple myeloma is a haematological cancer with no cure and a median survival period of approximately 5 years from diagnosis. Patients in the late stages of multiple myeloma suffer from symptoms including skeletal pain, bone fractures and infections associated with a weakened immune system as well as from the side effects of the treatments available today.
Comparing clinical data between Ygalo® and the current standard of care in late-stage multiple myeloma indicates that treatment with Ygalo® increases overall survival, progression free survival and the number of patients with significant tumour burden reduction as well as being better tolerated by patients.
The pivotal Phase 3 clinical study (OCEAN, OP-103) is currently enrolling and has been approved under the US Food and Drug Administration’s Special Protocol Assessment. In the OCEAN study, Ygalo® is compared directly against the current standard of care in patients with relapsed or refractory multiple myeloma.
As a rare condition, multiple myeloma is classified as an orphan disease in the US and Europe and Ygalo® has been granted orphan drug designation by the relevant authorities in both these jurisdictions.