뇌질환의 혁신, 뉴로벤티(NeuroVenti)

SCIENCE & TECHNOLOGY

Vision for the Future

We aim to be a world-class frontrunner company
in developing treatments for intractable brain diseases.

Autism spectrum disorder (ASD)

Overview

  • Spectrum disorder
    • Social interaction problem and restricted and repetitive behaviors
    • Comorbidity (seizure, anxiety, depression, aggression and intellectual disability)
  • Diagnosis: DSM-5 (ADOS, ADIR)
  • Pathological mechanism and therapeutic target: Not determined
  • Includes monogenic syndrome (FXS etc.)
  • World Autism Awareness Day: April 2nd (UN , 2007)
  • Medical unmet needs : The major CNS disease with NO cure
  • Current drug only controls comorbid symptoms (seizure, OCD, depression and irritability)

Market status

  • High prevalence rate : USA 3.49%, Korea 2.64%
  • Huge social cost : 3.6 Billion $/person (lifetime cost), 7 Trillion$ (USA)
  • Average direct annual cost per patient : 6 Thousand $ (USA)
  • ABA market size: 4 Billion$/Year (USA)
Global ASD therapeutic market size (2022) Global ASD therapeutic market size (2022)
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Since there’s no cure with a large market size for ASD, the possibility of new market as a first-in-disease is very high.

Fragile X syndrome (FXS)

Overview

  • Monogenic neurological disease
    • Physical (macroorchidism, macrocephaly)
    • Seizure, anxiety, depression, aggression, cognitive impairment, social impairment
  • Cause: Loss of FMRP, genetic product of FMR1 gene
  • US FDA, EMA, KFDA orphan drug designations
  • High medical unmet needs : NO cure
  • FXS awareness day: July 22nd (2014)
NATIONAL FRAGILE X FOUNDATION

Market status

  • Number of patients and annual medical and social costs
  • Prevalence rate : 0.03% (USA, Korea, UK, Germany, China, Japan)
  • US Annualized health care costs: $14,677
  • EU Mean Annual total direct cost of FXS: €25,800/patient
Country Number of Patients
South Korea 16,000
USA 100,000
Germany 27,494
UK 20,090
China 432,949
Japan 42,444

Innovation in NeuroVenti’s development strategy

Conventional ASD therapy : Limitation and solution

Limitation Solution
Non-biological care
  • Behavioral correction
  • Medicine with clearer clinical evidence
Non-biological care
  • Pharmacologically unclear mechanism
  • Scientific and evidence-based mechanism of new treatment
  • Side effect and low efficacy related to single target control
  • Increase of efficacy and reduction of adverse effects with multimodal treatment strategy
  • Low efficacy of cannabinoids with legal and drug abuse issues
  • Take advantage of the mechanism inferred by drug of addiction / hallucinogens
  • Adverse effect-free

Development strategy

  • Precision multi-target action
  • Superior efficacy and side-effect profiles
  • First-in-disease