Vafidemstat is under clinical development by Oryzon Genomics and currently in the Phase I and Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Vafidemstat’s likelihood of approval (LoA) and phase transition for Phelan-McDermid Syndrome (22q13.3 Deletion Syndrome) took place on 18 May 2021, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Vafidemstat Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Vafidemstat overview

Vafidemstat (ORY-2001) is under development for the treatment of Phelan-McDermid Syndrome, Alzheimer's disease, Huntington’s disease, Parkinson's disease, other dementias, relapsing-remitting multiple sclerosis and secondary progressive multiple sclerosis, aggressiveness (psychosis) associated with psychiatric and neuro-degenerative diseases such as lewy body dementia (DLB), Alzheimer's disease, autism spectrum syndrome (ASD), borderline personality disorder (BPD) and adult attention deficit hyperactivity disorder (ADHD), Kabuki syndrome, Schizophrenia other autoimmune and orphan CNS diseases, pneumonia/acute respiratory distress syndrome in seriously ill COVID-19 patients. The drug candidate is administered orally, formulated as capsule. It targets monoamine oxidase type B (MOA-B) and lysine specific histone demethylase 1A (LSD1). The drug candidate is developed based on the epigenetic platform.

Oryzon Genomics overview

Oryzon Genomics (Oryzon) is a clinical-stage biopharmaceutical company that offers discovers and develops epigenetic therapies to treat oncology and neurodegenerative diseases. The company provides epigenetics, a regulatory system that controls gene expression without affecting the makeup of the genes themselves and provides regulation of gene transcription that has emerged as a key biological determinant of protein production and cellular differentiation. Its clinical-stage product include ORY-1001, an oncology product for treating acute leukemia disease; ORY-2001 for treating Alzheimer disease, Parkinson disease and others; and ORY-3001 for treating other indications of cancer. Oryzon provides therapeutic programs and collaborative research services. The company serves patients with cancers or neurodegenerative disorders. It operates office in Spain and the US. Oryzon is headquartered in Cornella de Llobregat, Barcelona, Spain.

Quick View Vafidemstat LOA Data

Report Segments
  • Innovator
Drug Name
  • Vafidemstat
Administration Pathway
  • Oral
Therapeutic Areas
  • Central Nervous System
  • Genetic Disorders
  • Immunology
  • Infectious Disease
Key Developers
Highest Development Stage
  • Phase II

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.