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AUT00206

Fragile X Syndrome

ClinicalActive

Key Facts

Indication

Fragile X Syndrome

Phase

Clinical

Status

Active

Company

Autifony Therapeutics

About Autifony Therapeutics

Autifony Therapeutics is a private, clinical-stage biotech founded in 2011 as a spin-out from GlaxoSmithKline. The company's core expertise lies in translating ion channel biology into therapeutics, with two clinical-stage Kv3 modulators for Fragile X syndrome (AUT00206, with FDA Orphan Drug Designation) and rare myoclonic epilepsies (AUT00201). Beyond these lead rare disease programs, Autifony has built a broader preclinical and early-stage pipeline targeting other CNS disorders, positioning it as a specialist in neuronal excitability and hearing disorders.

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Therapeutic Areas

Rare CNS Disorders Epilepsy Neurodevelopmental Disorders Hearing Loss Schizophrenia

Other Fragile X Syndrome Drugs

Drug	Company	Phase	Status
KER-0193	Kaerus Bioscience	Preclinical	Active
p70 S6 Kinase Inhibitor	Epigen Biosciences	Preclinical	Active
HLX-0206	Healx	AI Discovery	Active
ENT-3379	Enthorin Therapeutics	Phase 1	Active
Fragile X Program	Sentinel Oncology	Preclinical	Active
Fragile X Syndrome Program	Purposeful	Discovery/Pre-clinical	Active
Fragile X Syndrome Trial	Science 37	Phase 3	Completed/Highlighted
DAYBUE® (trofinetide)	Neuren Pharmaceuticals	Phase 2	Completed
Fragile X Syndrome Test	Genomics BioSci & Tech	Clinical	Active
Xanamem (emestedastat)	Actinogen Medical	Phase 2	Active