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DT-216P2

Phase 1

Friedreich Ataxia | Small molecule | Neurology |Design Therapeutics, Inc.|Last Updated: May 18, 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 Trials2
Total Enrollment56
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06874010A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's AtaxiaPHASE1 RECRUITING 20Jun 1, 2025Mar 1, 2027May 18, 20253 Australia
NCT06772870A Single Ascending Dose Study of DT-216P2 in Normal Healthy ParticipantsPHASE1 NOT YET_RECRUITING 36Feb 1, 2025Dec 1, 2025Feb 21, 20251 Australia
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Study Endpoints
Primary Endpoints
Frequency of treatment-emergent adverse events (TEAE)
Up to Week 12
Frequency of treatment emergent adverse events (TEAEs)
From first dose to end of study, Day 30 post first dose administration.

To evaluate the safety and tolerability of single ascending doses of DT-216P2 in normal healthy participants by frequency of treatment-emergent adverse events (TEAEs).

Secondary Endpoints
Area under the curve (AUClast, AUCinf)
Up to Week 12
Maximum concentration (Cmax)
Up to Week 12
Time to Cmax (Tmax)
Up to Week 12
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
DT-216P2EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
DT-216P2DRUGActive
SalineDRUGNormal saline solution will be used as placebo control.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Participants must be 18-65 years of age inclusive, at the time of signing the informed consent. * Genetically confirmed diagnosis of FA, with homozygous GAA repeat expansions in the frataxin gene. * Stage 5.5 or less on the FSA at screening. * BMI between 16 and 32 kg/m2 at sc...

Countries:Australia
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06874010primaryCompletionDate: changed
LOWMay 26, 2026NCT06772870primaryCompletionDate: changed
LOWMay 24, 2026NCT06874010studyFirstPostDate: changed
LOWMay 24, 2026NCT06772870studyFirstPostDate: changed