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AV078

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Selective mTORC1 inhibitor entering Phase 2 development for refractory epilepsy caused by tuberous sclerosis complex.

Modality
oral CNS-penetrant selective small-molecule mTORC1 inhibitor
Availability (US)
Phase 2 TSC refractory-epilepsy study registered as Not yet recruiting
Biomarkers
seizure frequency; Clinical Global Impression of Change; treatment-emergent adverse events; pharmacokinetics
Hallmark targeted
deregulated nutrient sensing, autophagy, proteostasis
Kind
investigational drug
Clinical trials
NCT07680322 — Phase 2, Not yet recruiting on July 2, 2026 registry update