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MSD-001

Phase 1

Mental Health Disorders | Small molecule | Other |Design Therapeutics, Inc.|Last Updated: Sep 16, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment47
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06702332Single Ascending Dose Study of MSD-001 in Healthy ParticipantsPHASE1 COMPLETED 47Nov 11, 2024Jul 11, 2025Sep 16, 20251 Netherlands
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Study Endpoints
Primary Endpoints
Assess the safety and tolerability of MSD-001 in healthy adult participants by assessing the number, duration, severity, drug relatedness and type of adverse events
up to 30 days

Treatment emergent adverse events/ treatment emergent serious adverse events - their frequency, duration and severity, clinically significant changes in lab parameters, ECG, and vital signs

Secondary Endpoints
Plasma Pharmacokinetics (PK) of a single dose of MSD-001: Maximum Plasma Concentration (Cmax)
Pre-dose and up to 24 hours post dose
Plasma Pharmacokinetics (PK) of a single dose of MSD-001: time to attain Cmax (Tmax)
Pre-dose and up to 24 hours post dose
Plasma Pharmacokinetics (PK) of a single dose of MSD-001: Area under plasma Concentration (AUC)
Pre-dose and up to 24 hours post dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Active Treatment: MSD-001ACTIVE_COMPARATORPlanned doses of MSD-001; N = 42
Placebo ComparatorPLACEBO_COMPARATORNon-active study drug N = 10
Interventions
NameTypeDescription
MSD-001DRUGMSD-001 is being developed as part of future two-agent combination treatment approaches for the management of mental health indications
Placebo ComparatorDRUGMatching Placebo
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Eligibility Criteria
Age Range18 Years — 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Key Inclusion Criteria: * Male or female healthy consented participants 18 to 55 years of age * CYP2D6 phenotype established based on pharmacogenetic testing. * Free from psychoactive drug use from 4 weeks before dosing and until the last follow up visit. Key Exclusion Criteria: * Any current cli...

Countries:Netherlands
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Competitive Landscape -Mental Health Disorders 11 trials
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