Vafidemstat is a Small Molecule owned by Oryzon Genomics, and is involved in 15 clinical trials, of which 9 were completed, 5 are ongoing, and 1 is planned.

Vafidemstat (ORY-2001) acts by inhibiting monoamine oxidase type B (MOA-B) and lysine specific demethylase 1 (LSD1, KDM1A) inhibitor. LSD1 is an epigenetic modulator that regulates gene expression by demethylating histones. It forms a part of protein complexes involved in transcriptional regulation, and mis-regulation of these transcriptional complexes may result in disease. The  inhibition of LSD-1 by the drug candidate results in check in LSD-1 mediated complications. The  inhibition of Monoamine Oxidase results in inhibition of striatal damage in mitochondrial toxin.

The revenue for Vafidemstat is expected to reach a total of $202m through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Vafidemstat NPV Report.

Vafidemstat is originated and owned by Oryzon Genomics.

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.

The operating loss of the company was EUR19.7 million in FY2021, compared to an operating loss of EUR4.3 million in FY2020. The net loss of the company was EUR5.2 million in FY2021, compared to a net loss of EUR3.4 million in FY2020.

Quick View – Vafidemstat

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

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

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.