ORYZON is a public clinical stage biopharmaceutical company and the European leader in the development of epigenetics-based therapeutics, with a strong focus on personalized medicine approaches to CNS disorders and oncology.
Our business model is to develop our proprietary drug candidates through clinical Phase II, at which point we decide on a case-by-case basis to either keep the development in-house or to partner or out-license the compound for late stage development and commercialization.
ORYZON has been listed on the Spanish Stock Exchange since December 2015 (ORY, ISIN Code: ES0167733015). Since then, the company has attracted specialized investors from US, Israel and Europe in several PIPEs led by different US investment banks.
The company has a broad and growing portfolio, with two compounds in clinical trials: iadademstat (aka ORY-1001), a highly potent and selective LSD1 inhibitor that has been granted orphan-drug status by EMA, currently in two Phase IIa trials in oncology, and vafidemstat (aka ORY-2001), a CNS optimized LSD1 inhibitor also in Phase IIa for the treatment of neurological diseases. Our pipeline also includes ORY-3001, a selective LSD1 inhibitor in preclinical development for the treatment of non-oncological diseases, and additional programs at advanced stages of preclinical development, such as HDAC-6 inhibitors.
The entire portfolio has been created internally from a concerted effort of our functional genomics, medicinal chemistry, preclinical and clinical development teams. The company has a very solid IP position and is the originator and owner of all our IP portfolio.
The company has a seasoned international executive management with vast experience in the industry.
ORYZON has offices in Spain and the United States.
Oryzon’s management team is composed of passionate biopharma executives exploring new fields of epigenetic science to transform it into innovative personalized medicines for patients
Oryzon Genomics is a clinical stage biopharmaceutical company that discovers, develops, and plans to commercialize novel epigenetic therapies to treat oncology and CNS diseases.
Our therapeutic strategy is to treat the underlying causes of these diseases by targeting lysine specific demethylase 1 (LSD1, also known as KDM1A). LSD1 is a histone modifying enzyme that is involved in the regulation of the expression of many genes important in the onset and progression of human diseases such as cancer and CNS disorders.
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.
Oryzon has two drugs in Phase II clinical development: iadademstat (also known as ORY-1001), a selective LSD1 inhibitor for oncology, and vafidemstat (also known as ORY-2001), a CNS optimized LSD1 inhibitor for CNS and psychiatric disorders.
Oryzon has two Phase IIa studies ongoing with iadademstat: the ALICE study, to evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with standard treatment with azacitidine in newly diagnosed patients with Acute Myeloid Leukemia (AML) not eligible for intensive chemotherapy, and the CLEPSIDRA study, to evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with platinum-etoposide in patients with small cell lung cancer. Preliminary clinical results have already been reported in both clinical trials.
With vafidemstat, Oryzon has performed Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders, namely Autism Spectrum Disorder, Borderline Personality Disorder and adult Attention Deficit Hyperactivity Disorder (REIMAGINE study), and in aggressive/agitated patients with moderate or severe Alzheimer’s disease (REIMAGINE-AD study), with positive preliminary clinical results reported in both clinical trials. Additional Phase IIa clinical trials with vafidemstat are ongoing: the ETHERAL and ETHERAL-US studies in patients with mild to moderate Alzheimer’s disease, and the SATEEN study in relapse-remitting and secondary progressive multiple sclerosis. An additional Phase IIa trial in severely ill COVID-19 patients has recently started (ESCAPE study).
Our pipeline also includes another LSD1 inhibitor, ORY-3001, in preclinical development for the treatment of non-oncological diseases, and additional programs at earlier stages of development.
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 CNS disorders and cancers.
While other companies are exploring a variety of epigenetic targets in oncology, ORYZON offers a truly differentiated proposition in this biotech landscape.
ORYZON is the most specialized LSD1 Company. We have been working on this fascinating target since 2009, we own the broadest IP portfolio and have the most extensive clinical development program in this epigenetic area. We have a promising and very advanced program in oncology with Phase II trials currently ongoing.
ORYZON is the leading industry player developing LSD1 inhibitors for CNS disorders. We have several Phase II trials in CNS concluded or ongoing, and additional trials in preparation.
ORYZON is exploring personalized medicine approaches to CNS and oncology. Our approach involves selecting particular subsets of patients harboring specific mutations causative of these diseases or selecting patients with specific biomarkers predictive of responsiveness to LSD1 inhibition.