# List of investigational autism and pervasive developmental disorder drugs

This is a list of investigational autism and pervasive developmental disorder drugs, or drugs that are currently under development for clinical use in the treatment of autistic spectrum disorders and/or other pervasive developmental disorders but are not yet approved.
Chemical/generic names are listed first, with developmental code names, synonyms, and brand names in parentheses.
This list was last comprehensively updated in October 2024. It is likely to become outdated with time.

## Under development
### Preregistration

* CM-4612 (CM-ADHD; CM-AT; CM-PK) – enzyme modulator/replacement for gastrointestinal or pancreatic secretory deficiencies

### Phase 3

* Brexpiprazole (Rexulti) – dopamine D$_2$ and D$_3$ receptor partial agonist, serotonin 5-HT$_{1A}$ receptor partial agonist, serotonin 5-HT$_{2A}$ 5-HT$_{2B}$, and 5-HT$_7$ receptor antagonist, α$_1$- and α$_2$-adrenergic receptor antagonist, and atypical antipsychotic
* Cariprazine (Reagila, Symvenu, Vraylar; MP-214, RGH-188, WID-RGC20) – dopamine D$_2$ and D$_3$ receptor partial agonist, serotonin 5-HT$_{1A}$ receptor partial agonist, serotonin 5-HT$_{2B}$ receptor antagonist, and atypical antipsychotic
* Lumateperone (Caplyta) – dopamine D$_1$ and D$_2$ receptor antagonist, serotonin 5-HT$_{2A}$ receptor antagonist, α$_1$-adrenergic receptor antagonist, serotonin reuptake inhibitor, and atypical antipsychotic
* Lurasidone (Latuda) – dopamine D$_2$ and D$_3$ receptor antagonist, serotonin 5-HT$_{1A}$ receptor partial agonist, serotonin 5-HT$_{2A}$ and 5-HT$_7$ receptor antagonist, α$_{2C}$-adrenergic receptor antagonist, and atypical antipsychotic
* Tasimelteon (Hetlioz) – melatonin MT$_1$ and MT$_2$ receptor agonist

### Phase 2

* AB-2004 – microbiome modulator
* Acamprosate (AOP-2020; Campral; SF-679/SF-775; SF-999) – unknown / GABA$_A$ receptor positive allosteric modulator and ionotropic glutamate NMDA receptor antagonist
* Alogabat (RG-7816, RG7816, RO-7017773; GABA-A-α$_5$ PAM) – GABA$_A$ α$_5$ subunit-containing receptor positive allosteric modulator
* Arbaclofen ((R)-baclofen; STX-209) – GABA$_B$ receptor agonist
* Cannabidiol (CBD; Epidiolex) – cannabinoid receptor modulator, other actions
* Cannabidiol transdermal patch/gel (Zygel; ZYN-002) – cannabinoid receptor modulator, other actions
* Cannabidivarin (CBDV; GWP-42006) – non-intoxicating cannabinoid receptor modulator, other actions
* CP-101 – bacteria relacement and gastrointestinal microbiome modulator
* JNJ-42165279 (JNJ-5279) – fatty acid amide hydrolase (FAAH) inhibitor
* Oxytocin (intranasal potentiated oxytocin; TI-001, TI-114, TNX-1900, TNX-2900) – oxytocin receptor agonist
* Pitolisant (Wakix; tiprolisant) – histamine H$_3$ receptor inverse agonist
* Racemetirosine (DL-α-methyltyrosine; L1-79) – tyrosine hydroxylase inhibitor
* Suramin (IV suramin; PAX-101/PAX-102) – DNA synthesis inhibitor and anti-purinergic agent
* Zolmitriptan modified-release (ML-004, ML004) – serotonin 5-HT$_{1B}$ and 5-HT$_{1D}$ receptor agonist

### Phase 1

* (R)-MDMA ((R)-midomafetamine; MM-402) – serotonin, norepinephrine, and dopamine releasing agent and weak serotonin 5-HT$_{2A}$, 5-HT$_{2B}$, and 5-HT$_{2C}$ receptor agonist (entactogen and weak psychedelic hallucinogen)
* STP-1 (STALICLA Therapeutic Package 1) – phosphoric diester hydrolase inhibitor and sodium–potassium–chloride symporter inhibitor
* Sulforafan alfadex (sulforaphane alfadex; sulforafan α-cyclodextrin; SFX-01, STP-2; broccoli sprout extract) – various actions

### Preclinical

* ASD-002 - a child-specific (non-acidic, sustained-release) anti-hyper excitatory ester prodrug of the potassium channel modulator NSAID mefenamic acid (MFA, approved as Ponstel) to prevent toddlers from becoming non-verbal (developmental language disorder, DLD)
* Bryostatin-1 (MW-904) – protein kinase C stimulant
* CJRB-303 (CJRB-901, MRx-0006) – bacteria replacement
* Debamestrocel (growth factor-producing stem cell therapy) – dopaminergic cell replacement therapy
* FIN-211 – bacteria replacement and gastrointestinal microbiome modulator
* Levophacetoperane (NLS-3; (R,R)-phacetoperone; methylphenidate reverse ester) – norepinephrine and dopamine reuptake inhibitor and psychostimulant
* LIT-001 – small-molecule oxytocin receptor agonist
* MBDB – serotonin and norepinephrine releasing agent and weak serotonin 5-HT$_1$ and 5-HT$_2$ receptor ligand (entactogen)
* Psilocin (PLZ-1015) – non-selective serotonin receptor agonist and psychedelic hallucinogen
* QBM-001 – undefined mechanism of action (allosteric channel modulator; putative neuroprotective and anti-inflammatory agent)

### Research

* Baeocystin (PLZ-1019) – non-hallucinogenic serotonin 5-HT$_{2A}$ receptor agonist, other actions
* Norpsilocin (PLZ-1017) – non-hallucinogenic serotonin 5-HT$_{2A}$ receptor agonist, other actions
* Roluperidone (CYR-101, MIN-101, MT-210) – serotonin 5-HT$_{2A}$, sigma σ$_2$, and α$_{1A}$-adrenergic receptor antagonist
* Squalamine (ENT-01; Enterin-01; kenterin) – various actions

### Phase unknown

* Psilocybin (COMP360; COMP-360) – non-selective serotonin receptor agonist and psychedelic hallucinogen

## Not under development
### Development suspended

* KM-391 – serotonin reuptake inhibitor

### No development reported

* AB-1224 – microbiome modulator
* AGX-201 (histamine dihydrochloride salt) – histamine H$_1$ receptor antagonist and histamine H$_3$ receptor agonist
* Aminolevulinic acid/sodium ferrous citrate (5-ALA-SFC, 5-ALA/SFX; sodium ferrous citrate/aminolevulinic acid; SPP-003) – erythropoiesis stimulant and photosensitizer
* Aripiprazole transdermal (AQS-1301; transdermal aripiprazole) – dopamine D$_2$ and D$_3$ receptor partial agonist, serotonin 5-HT$_{1A}$ and 5-HT$_7$ receptor partial agonist, serotonin 5-HT$_{2A}$ and 5-HT$_{2B}$ receptor antagonist or inverse agonist, and atypical antipsychotic
* BAER-101 (AZ-7325; AZD-7325) – selective GABA$_A$ α$_2$ and α$_3$ subunit-containing receptor positive allosteric modulator
* BBP-472 – phosphatidylinositol 3 kinase β (PI3Kβ) inhibitor
* Fasoracetam co-crystallised (co-crystallised fasoracetam; AEVI-004) – various actions and racetam
* Guanfacine once-daily (Guanfacine Carrier Wave; SPD-547) – α$_2$-adrenergic receptor agonist
* KBLP-010 – bacteria replacement and microbiome modulator
* NBTX-001 – ionotropic glutamate NMDA receptor antagonist
* Oxytocin intranasal (OPN-300; OptiNose oxytocin) – oxytocin receptor agonist
* Research programme: allosteric modulators - Addex Therapeutics (various) – various actions
* Research programme: antisense oligonucleotide therapeutics - RogCon U.R (RCUR-313, RCUR-SMP) – voltage-gated sodium channel Na$_v$1.2 expression stimulants
* Research programme: autism and obesity therapeutics - Berand Neuropharmacology – histone deacetylase inhibitors
* Research programme: brain development disorder therapeutics - Seaside Therapeutics (STX-110) – metabotropic glutamate mGlu$_5$ receptor antagonists and muscarinic acetylcholine M$_1$ receptor antagonists
* Research programme: cannabinoid receptor modulators - GW Pharmaceuticals (cannabigerol; CBG) – cannabinoid receptor modulators
* Research programme: cannabis extract therapeutics - Cannabis Science (CBIS compounds) – cannabinoid receptor modulators
* Research programme: central nervous system therapeutics - AbbVie/Rugen – undefined mechanism of action
* Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals – antioxidants, glutamate receptor modulators, SLC7A11 modulators
* Research programme: CNS disorders therapeutics - Sage Therapeutics (SAGE-105; SGE-202; SGE-301; SGE-516) – GABA$_A$ receptor modulators and ionotropic glutamate NMDA receptor modulators
* Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation – G protein-coupled receptor modulator and neuromedin U receptor modulator
* Research programme: GPCR modulators - Nxera Pharma – various actions
* Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma – bacteria replacements
* Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics – metabotropic glutamate mGlu$_5$ receptor antagonists
* Research programme: oxytocin intranasal - Pastorus Pharma – neurotransmitter modulators/oxytocin receptor agonists
* Research programme: therapeutic autoantibodies - Sengenics – undefined mechanism of action
* RG-7713 (RG7713) – vasopressin V$_{1A}$ receptor antagonist
* Tideglusib (AMO-02, NP-031112, NP-12; Nypta, Zentylor) – glycogen synthase kinase 3β (GSK-3β) inhibitor
* Vafidemstat (ORY-2001) – dual lysine specific demethylase 1 (LSD1) inhibitor and monoamine oxidase B (MAO-B) inhibitor
* Xenon (NBTX-001) – ionotropic glutamate NMDA receptor antagonist and dissociative hallucinogen NBTX 001 - AdisInsight

### Discontinued

* Balovaptan (RG-7314, RO-5028442, RO-5285119) – vasopressin V$_{1A}$ receptor antagonist
* Blarcamesine (AE-37, ANA001, ANAVEX 2-73) – sigma σ$_1$, muscarinic acetylcholine M$_1$, and ionotropic glutamate NMDA receptor agonist
* Brilaroxazine (RP-5063, RP-5000) – dopamine D$_2$, D$_3$, D$_4$ receptor partial agonist, serotonin 5-HT$_{1A}$ receptor agonist, serotonin 5-HT$_{2A}$, 5-HT$_{2B}$, 5-HT$_7$ receptor antagonist, and atypical antipsychotic
* Bumetanide oral liquid (S-95008) – sodium–potassium–chloride symporter/cotransporter inhibitor and indirect GABAergic inhibitor discontinued after failed phase 3 trial
* Carbetocin (CYP-2001) – oxytocin receptor agonist
* CX-516 (1-BCP; BDP-12, SPD-420; AMPAlex) – AMPA receptor modulator
* EM-036 (memantine analogue) – ionotropic glutamate NMDA receptor antagonist, other actions
* Fasoracetam (AEVI-001, LAM-105, MDGN-001, NFC-1, NS-105) – various actions and racetam
* Fluoxetine rapid-dissolve (AT-001; AT001; NPL-2008; Serelsa; Zydis™ ODT fluoxetine) – serotonin reuptake inhibitor
* GTS-21 (DMXB-A, DMXB-A sustained release, DMXB-A-SR) – α$_7$ nicotinic acetylcholine receptor partial agonist
* Ketamine intranasal (RVT-701) – ionotropic glutamate NMDA receptor antagonist
* Memantine (Namenda) – ionotropic glutamate NMDA receptor antagonist, other actions
* Oxytocin intranasal (Syntocinon Nasal Spray; TUR 001) – oxytocin receptor agonist
* Research programme: AMPA receptor agonists (ampakines, AMPAkines; CX compounds) - RespireRx – ionotropic glutamate AMPA receptor agonists
* Research programme: NMDA receptor modulators - AbbVie/Naurex (NRX-1050; NRX-1051; NRX-1059; NRX-105x; NRX-1060; NRX-2085; NRX-20xx) – ionotropic glutamate NMDA receptor modulators
* Risperidone extended-release (Risperisphere) – dopamine D$_2$ and D$_3$ receptor antagonist, serotonin 5-HT$_{1B}$, 5-HT$_{2A}$, 5-HT$_{2C}$, and 5-HT$_7$ receptor antagonist or inverse agonist, α$_1$- and α$_2$-adrenergic receptor antagonist, histamine H$_1$ receptor inverse agonist, and atypical antipsychotic
* Secretin (INN-329, RG-1068; SecreFlo) – medical imaging enhancer (diagnosis)
* Suramin (Antrypol) – DNA-directed DNA polymerase inhibitor and intercellular signaling peptide/protein inhibitor
* Trichuris suis ova (CNDO-201, TSO, TSO-2500, TSO-7500) – immunomodulator
* Trofinetide (Daybue; G-2Me-PE; Glycyl-2-methyl-L-prolyl-L-glutamic acid; NNZ-2566; IGF-1 (1–3) analogue) – unknown / various actions

## Clinically used drugs
### Approved drugs

* Aripiprazole (Abilify) – dopamine D$_2$ and D$_3$ partial agonist, serotonin 5-HT$_{1A}$ and 5-HT$_7$ receptor partial agonist, serotonin 5-HT$_{2A}$ and 5-HT$_{2B}$ receptor antagonist or inverse agonist, and atypical antipsychotic
* Risperidone (Risperdal) – dopamine D$_2$ and D$_3$ receptor antagonist, serotonin 5-HT$_{1B}$, 5-HT$_{2A}$, 5-HT$_{2C}$, and 5-HT$_7$ receptor antagonist or inverse agonist, α$_1$- and α$_2$-adrenergic receptor antagonist, histamine H$_1$ receptor inverse agonist, and atypical antipsychotic

### Off-label drugs

* α$_2$-Adrenergic receptor agonists (e.g., clonidine, guanfacine)
* Anticonvulsants/mood stabilizers (e.g., valproic acid, lamotrigine)
* Antipsychotics (non-licensed) (e.g., haloperidol, olanzapine)
* Dietary supplements (e.g., N-acetylcysteine, omega-3 fatty acids, sulforaphane)
* Entactogens (serotonin releasing agents) (e.g., MDMA)
* Melatonin receptor agonists (e.g., melatonin)
* Norepinephrine reuptake inhibitors (NRIs) (e.g., atomoxetine)
* Opioid receptor antagonists (e.g., naltrexone)
* Other antidepressants (e.g., mirtazapine)
* Oxytocin receptor agonists (e.g., oxytocin)
* Probiotics and prebiotics
* Psychostimulants (norepinephrine–dopamine releasing agents and/or reuptake inhibitors) (e.g., amphetamine, methylphenidate)
* Selective serotonin reuptake inhibitors (SSRIs) (e.g., fluoxetine, fluvoxamine, sertraline, citalopram)
* Serotonergic psychedelics (e.g., psilocybin, lysergic acid diethylamide (LSD))
* Serotonin–norepinephrine reuptake inhibitors (SNRIs) (e.g., milnacipran)
* Serotonin releasing agents (e.g., fenfluramine—withdrawn and no longer recommended)
* Serotonin 5-HT$_{1A}$ receptor agonists (e.g., buspirone)
* Tricyclic antidepressants (TCAs) (e.g., clomipramine)