뇌질환의 혁신, 뉴로벤티(NeuroVenti)

SCIENCE & TECHNOLOGY

Vision for the Future

We aim to be a global frontrunner in therapeutics for intractable brain disease

Autism spectrum disorder (ASD)

Overview

  • Spectrum disorder
    • Characterized by impaired social communication and interaction, and restricted, repetitive patterns of behavior
    • Common comorbidities: seizures, anxiety, depression, aggression, intellectual disability
  • Diagnosis: DSM-5 criteria (ADOS, ADI-R)
  • Pathophysiology and therapeutic target: not yet established
  • Includes monogenic syndromes (e.g., FXS)
  • World Autism Awareness Day: April 2 (UN, 2007)
  • High unmet medical need: a major CNS disorder with no approved cure
  • Existing drugs address only comorbid symptoms (seizures, OCD, depression, irritability)

Market status

  • High prevalence rate : USA 3.2%, Korea 2.64%
  • Huge social cost : ~$3.6 million per individual (lifetime); ~$7 trillion cumulative to date (USA)
  • Average direct medical cost: ~$6,000 per patient/year (USA)
  • ABA (Applied Behavior Analysis) therapy market: ~$4 billion/year (USA)
Global ASD therapeutic market size (2022) Global ASD therapeutic market size (2022)
Light it up blue

"With no approved cure and a large addressable market, ASD represents a substantial first-in-disease opportunity."

Fragile X syndrome (FXS)

Overview

  • A monogenic neurological disorder
    • Physical features: macroorchidism, macrocephaly
    • Associated features: seizures, anxiety, depression, aggression, cognitive and social impairment
  • Cause: loss of FMRP, the protein product of the FMR1 gene
  • Orphan drug designations: US FDA, EMA, MFDS (Korea)
  • High unmet medical need: no approved cure
  • Fragile X Awareness Day: July 22 (2014)
National Fragile X Foundation

Market status

  • Patient Population and Annual Economic Burden
  • Prevalence: 0.03% (US, Korea, UK, Germany, China, and Japan)
  • Annual healthcare cost per patient (US): $14,677
  • Average annual total direct cost per patient (EU): €25,800
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: limitations and our solutions

Limitation Solution
Non-biological care
  • Behavioral intervention
  • Medicines supported by clearer clinical evidence
Biological care
  • Poorly defined pharmacological mechanism
  • A scientifically defined, evidence-based mechanism of action
  • Side effects and limited efficacy from single-target modulation
  • Improved efficacy and reduced adverse effects through a multimodal strategy
  • Limited efficacy of cannabinoids, with legal and abuse-related concerns
  • Leverages mechanisms inferred from addictive drugs and hallucinogens
  • Devoid of hallucinogenic side effects and addictive potential

Development strategy

  • Precision multi-target action
  • Enhanced therapeutic efficacy with reduced side effects
  • First-in-disease