DRUGapprovedsmall molecule

Haloperidol

Brand names: Haldol, Serenace

Mechanism

D2 dopamine antagonist - SMA repurposed via Menduti 2026.

schizophreniaacute psychosisTourette syndrome

O=C(CCCN1CCC(O)(c2ccc(Cl)cc2)CC1)c1ccc(F)cc1

Type	Predicate	Conf	Source
drug efficacy	Haloperidol, or a pharmaceutically acceptable salt, ester or solvate thereof, is for use in a method for preventing and/or treating spinal muscular atrophy.	53%	EP4387622B1
drug efficacy	Haloperidol may be used for treating spinal muscular atrophy (clinical trial result)	50%	ES3034601T3

T1: 5T4: 11untiered: 9

Target	Role	Tier	Boltz-2 iPTM	Chai-1 iPTM	Verdict
NCALD	SMA	T1	0.796	—	Primary SMA hit
GAP43	SMA	T1	0.765	—	Primary SMA hit
SMN2	SMA	T1	0.763	—	Primary SMA hit
ROCK2	SMA	T1	0.706	—	Primary SMA hit
CFL1	SMA	T1	0.570	—	Primary SMA hit
SMN1_YGbox	OTHER	T4	0.939	—	Low relevance
PLS3_EF4	OTHER	T4	0.888	—	Low relevance
LIMK2_aC	OTHER	T4	0.775	—	Low relevance
RAPSN_nterm	OTHER	T4	0.774	—	Low relevance
CHRNA1_ECD	OTHER	T4	0.772	—	Low relevance
HSP70_NBD	OTHER	T4	0.768	—	Low relevance
ROCK2_aC	OTHER	T4	0.729	—	Low relevance
LRP4_propeller	OTHER	T4	0.727	—	Low relevance
ROCK1_aC	OTHER	T4	0.704	—	Low relevance
DRD2	OTHER	T4	0.582	—	Low relevance
KCNA2_pore	OTHER	T4	0.554	—	Low relevance
PFN2	SMA	—	0.871	—	—
STMN1	SMA	—	0.768	—	—
CHRNG_fetal	OTHER	—	0.735	—	—
PERP_ECL1	OTHER	—	0.637	—	—
CFL2	SMA	—	0.551	—	—
SSH1_phosphatase	OTHER	—	0.475	—	—
DOK7_PH	OTHER	—	0.445	—	—
PERP	SMA	—	0.398	—	—
KCNA2	SMA	—	0.358	—	—