Corporate strategy: A differentiated proposition in Epigenetics
ORYZON is a clinical-stage biopharmaceutical company that leverages epigenetics to discover and develop innovative, personalized therapeutics for patients with central nervous system (CNS) disorders and cancer.
While other companies are exploring a variety of epigenetic targets in oncology, ORYZON offers a truly differentiated proposition within the epigenetics landscape.
ORYZON specializes in LSD1 (Lysine-specific demethylase 1) biology. The company has been building expertise in this fascinating target since 2009, owns a strong and broad intellectual property portfolio, and has the most extensive clinical development program in this epigenetic area, with two distinct, orally active, clinical-stage LSD1 inhibitors optimized for CNS disorders and for oncology and hematology, respectively.
ORYZON has the broadest clinical program with an LSD1 inhibitor in Oncology. Its lead oncology asset, iadademstat, believed to be the most potent LSD1 inhibitor in clinical development, is in multiple ongoing clinical trials across several therapeutic indications, including first- and second-line acute myeloid leukemia, myelodysplastic syndrome, myeloproliferative neoplasms and small cell lung cancer. In addition, the asset is being expanded into non-oncological hematological disorders, with a clinical trial ongoing in sickle cell disease and a trial in essential thrombocythemia in preparation.
ORYZON is the leading industry player developing LSD1 inhibitors for CNS disorders. LSD1 is a key epigenetic enzyme involved in CNS disease biology. Oryzon is advancing vafidemstat, a CNS-optimized LSD1 inhibitor, through an advanced clinical development program that includes a Phase III clinical trial in preparation in aggression in borderline personality disorder, a Phase II clinical trial currently ongoing in schizophrenia, and an additional Phase II clinical trial in autism spectrum disorder in preparation.
ORYZON is exploring personalized medicine approaches to CNS and oncology. This approach focuses on identifying patient subpopulations most likely to benefit from LSD1 inhibition, either through selecting particular subsets of patients harboring specific genetic alterations causative of these diseases or through selecting patients with specific biomarkers predictive of responsiveness to LSD1 inhibition, with the objective of maximizing therapeutic impact.
Our business model integrates our epigenetic platform in the identification and validation of novel biomarkers and therapeutic targets, to develop first-in-class new small molecules for cancer and CNS disorders.
ORYZON typically develops its investigational medicines as far as human proof-of-concept and then partners with pharmaceutical companies that have the resources to advance the programs in late stage clinical trials and through the regulatory market approval process.
Nevertheless, in some special cases (e.g. orphan diseases or subsets of genetically defined patients), the company may make the strategic decision to develop a program in house and seek regulatory market approval directly.