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Epi-743

Leigh Syndrome

PTC TherapeuticsPhase 2Dormant
B
62/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
47/ 100
Competitive Position
89/ 100

Competitive context

4 Active assets in the Leigh Syndrome cohort.

Asset / SponsorPhaseRating
Sonlicromanol
Khondrion BV
Phase 2B
Abi-009
Aadi Bioscience, Inc.
Phase 2CCC
MANNITOL
Thiogenesis Therapeutics, Inc.
Phase 2CCC
Rapamycin
Matthew Demczko
Phase 2CCC
Tti-0102: Cysteamine-Pantetheine Disulfide
Thiogenesis Therapeutics, Inc.
Phase 2NR
Vatiquinone
PTC Therapeutics
Phase 2/3NR

Same molecule, other indications

Epi-743 is also rated in:

Friedreich ataxiaDormant
B
Indicative ·
62 / 100
View asset →
Parkinson diseaseDormant
CCC
Indicative ·
54 / 100
View asset →
Pearson syndromeDormant
CCC
Indicative ·
53 / 100
View asset →

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