List of investigational autism and pervasive developmental disorder drugs

This is a dynamic list and may never be able to satisfy particular standards for completeness. You can help by editing the page to add missing items, with references to reliable sources.

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 It is likely to become outdated with time.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 ^[1]

Phase 3

• Brexpiprazole (Rexulti) – dopamine D₂ and D₃ receptor partial agonist, serotonin 5-HT_1A receptor partial agonist, serotonin 5-HT_2A 5-HT_2B, and 5-HT₇ receptor antagonist, α₁- and α₂-adrenergic receptor antagonist, and atypical antipsychotic ^[2]
• Cariprazine (Reagila, Symvenu, Vraylar; MP-214, RGH-188, WID-RGC20) – dopamine D₂ and D₃ receptor partial agonist, serotonin 5-HT_1A receptor partial agonist, serotonin 5-HT_2B receptor antagonist, and atypical antipsychotic ^[3]
• Lumateperone (Caplyta) – dopamine D₁ and D₂ receptor antagonist, serotonin 5-HT_2A receptor antagonist, α₁-adrenergic receptor antagonist, serotonin reuptake inhibitor, and atypical antipsychotic ^[4]
• Lurasidone (Latuda) – dopamine D₂ and D₃ receptor antagonist, serotonin 5-HT_1A receptor partial agonist, serotonin 5-HT_2A and 5-HT₇ receptor antagonist, α_2C-adrenergic receptor antagonist, and atypical antipsychotic ^[5]
• Tasimelteon (Hetlioz) – melatonin MT₁ and MT₂ receptor agonist ^[6]

Phase 2

• AB-2004 – microbiome modulator ^[7]
• Acamprosate (AOP-2020; Campral; SF-679/SF-775; SF-999) – unknown / GABA_A receptor positive allosteric modulator and ionotropic glutamate NMDA receptor antagonist ^[8]
• Alogabat (RG-7816, RG7816, RO-7017773; GABA-A-α₅ PAM) – GABA_A α₅ subunit-containing receptor positive allosteric modulator ^[9]
• Arbaclofen ((R)-baclofen; STX-209) – GABA_B receptor agonist ^[10]
• Cannabidiol (CBD; Epidiolex) – cannabinoid receptor modulator, other actions ^[11]
• Cannabidiol transdermal patch/gel (Zygel; ZYN-002) – cannabinoid receptor modulator, other actions ^[12]
• Cannabidivarin (CBDV; GWP-42006) – non-intoxicating cannabinoid receptor modulator, other actions ^[13]
• CP-101 – bacteria relacement and gastrointestinal microbiome modulator ^[14]
• JNJ-42165279 (JNJ-5279) – fatty acid amide hydrolase (FAAH) inhibitor ^[15]
• Oxytocin (intranasal potentiated oxytocin; TI-001, TI-114, TNX-1900, TNX-2900) – oxytocin receptor agonist ^[16]
• Pitolisant (Wakix; tiprolisant) – histamine H₃ receptor inverse agonist ^[17]
• Racemetirosine (DL-α-methyltyrosine; L1-79) – tyrosine hydroxylase inhibitor ^[18]
• Suramin (IV suramin; PAX-101/PAX-102) – DNA synthesis inhibitor and anti-purinergic agent ^[19]
• Zolmitriptan modified-release (ML-004, ML004) – serotonin 5-HT_1B and 5-HT_1D receptor agonist ^[20]

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) ^[21]
• STP-1 (STALICLA Therapeutic Package 1) – phosphoric diester hydrolase inhibitor and sodium–potassium–chloride symporter inhibitor ^[22]
• Sulforafan alfadex (sulforaphane alfadex; sulforafan α-cyclodextrin; SFX-01, STP-2; broccoli sprout extract) – various actions ^[23]

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) ^[24] to prevent toddlers from becoming non-verbal (developmental language disorder, DLD)

• Bryostatin-1 (MW-904) – protein kinase C stimulant ^[25]
• CJRB-303 (CJRB-901, MRx-0006) – bacteria replacement ^[26]
• Debamestrocel (growth factor-producing stem cell therapy) – dopaminergic cell replacement therapy ^[27]
• FIN-211 – bacteria replacement and gastrointestinal microbiome modulator ^[28]
• Levophacetoperane (NLS-3; (R,R)-phacetoperone; methylphenidate reverse ester) – norepinephrine and dopamine reuptake inhibitor and psychostimulant ^{[29] [30]}
• LIT-001 – small-molecule oxytocin receptor agonist ^{[31] [32]}
• MBDB – serotonin and norepinephrine releasing agent and weak serotonin 5-HT₁ and 5-HT₂ receptor ligand (entactogen) ^{[33] [34] [35] [36]}
• Psilocin (PLZ-1015) – non-selective serotonin receptor agonist and psychedelic hallucinogen ^[37]
• QBM-001 – undefined mechanism of action (allosteric channel modulator; putative neuroprotective and anti-inflammatory agent) ^{[38] [39]}

Research

• Baeocystin (PLZ-1019) – non-hallucinogenic serotonin 5-HT_2A receptor agonist, other actions ^{[40] [41]}
• Norpsilocin (PLZ-1017) – non-hallucinogenic serotonin 5-HT_2A receptor agonist, other actions ^{[42] [41]}
• Roluperidone (CYR-101, MIN-101, MT-210) – serotonin 5-HT_2A, sigma σ₂, and α_1A-adrenergic receptor antagonist ^[43]
• Squalamine (ENT-01; Enterin-01; kenterin) – various actions ^[44]

Phase unknown

• Psilocybin (COMP360; COMP-360) – non-selective serotonin receptor agonist and psychedelic hallucinogen ^[45]

Not under development

Development suspended

• KM-391 – serotonin reuptake inhibitor ^[46]

No development reported

• AB-1224 – microbiome modulator ^[47]
• AGX-201 (histamine dihydrochloride salt) – histamine H₁ receptor antagonist and histamine H₃ receptor agonist ^{[48] [49] [50]}
• Aminolevulinic acid/sodium ferrous citrate (5-ALA-SFC, 5-ALA/SFX; sodium ferrous citrate/aminolevulinic acid; SPP-003) – erythropoiesis stimulant and photosensitizer ^[51]
• Aripiprazole transdermal (AQS-1301; transdermal aripiprazole) – dopamine D₂ and D₃ receptor partial agonist, serotonin 5-HT_1A and 5-HT₇ receptor partial agonist, serotonin 5-HT_2A and 5-HT_2B receptor antagonist or inverse agonist, and atypical antipsychotic ^[52]
• BAER-101 (AZ-7325; AZD-7325) – selective GABA_A α₂ and α₃ subunit-containing receptor positive allosteric modulator ^[53]
• BBP-472 – phosphatidylinositol 3 kinase β (PI3Kβ) inhibitor ^[54]
• Fasoracetam co-crystallised (co-crystallised fasoracetam; AEVI-004) – various actions and racetam ^[55]
• Guanfacine once-daily (Guanfacine Carrier Wave; SPD-547) – α₂-adrenergic receptor agonist ^[56]
• KBLP-010 – bacteria replacement and microbiome modulator ^[57]
• NBTX-001 – ionotropic glutamate NMDA receptor antagonist ^[58]
• Oxytocin intranasal (OPN-300; OptiNose oxytocin) – oxytocin receptor agonist ^[59]
• Research programme: allosteric modulators - Addex Therapeutics (various) – various actions ^[60]
• Research programme: antisense oligonucleotide therapeutics - RogCon U.R (RCUR-313, RCUR-SMP) – voltage-gated sodium channel Na_v1.2 expression stimulants ^[61]
• Research programme: autism and obesity therapeutics - Berand Neuropharmacology – histone deacetylase inhibitors ^[62]
• Research programme: brain development disorder therapeutics - Seaside Therapeutics (STX-110) – metabotropic glutamate mGlu₅ receptor antagonists and muscarinic acetylcholine M₁ receptor antagonists ^[63]
• Research programme: cannabinoid receptor modulators - GW Pharmaceuticals (cannabigerol; CBG) – cannabinoid receptor modulators ^[64]
• Research programme: cannabis extract therapeutics - Cannabis Science (CBIS compounds) – cannabinoid receptor modulators ^[65]
• Research programme: central nervous system therapeutics - AbbVie/Rugen – undefined mechanism of action ^[66]
• Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals – antioxidants, glutamate receptor modulators, SLC7A11 modulators ^[67]
• 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 ^[68]
• Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation – G protein-coupled receptor modulator and neuromedin U receptor modulator ^[69]
• Research programme: GPCR modulators - Nxera Pharma – various actions ^[70]
• Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma – bacteria replacements ^[71]
• Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics – metabotropic glutamate mGlu₅ receptor antagonists ^[72]
• Research programme: oxytocin intranasal - Pastorus Pharma – neurotransmitter modulators/oxytocin receptor agonists ^[73]
• Research programme: therapeutic autoantibodies - Sengenics – undefined mechanism of action ^[74]
• RG-7713 (RG7713) – vasopressin V_1A receptor antagonist ^[75]
• Tideglusib (AMO-02, NP-031112, NP-12; Nypta, Zentylor) – glycogen synthase kinase 3β (GSK-3β) inhibitor ^[76]

• Vafidemstat (ORY-2001) – dual lysine specific demethylase 1 (LSD1) inhibitor and monoamine oxidase B (MAO-B) inhibitor ^[77]
• 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 ^[78]
• Blarcamesine (AE-37, ANA001, ANAVEX 2-73) – sigma σ₁, muscarinic acetylcholine M₁, and ionotropic glutamate NMDA receptor agonist ^[79]
• Brilaroxazine (RP-5063, RP-5000) – dopamine D₂, D₃, D₄ receptor partial agonist, serotonin 5-HT_1A receptor agonist, serotonin 5-HT_2A, 5-HT_2B, 5-HT₇ receptor antagonist, and atypical antipsychotic ^[80]
• Bumetanide oral liquid (S-95008) – sodium–potassium–chloride symporter/cotransporter inhibitor and indirect GABAergic inhibitor ^{[81] [82]} discontinued after failed phase 3 trial ^[83]
• Carbetocin (CYP-2001) – oxytocin receptor agonist ^[84]
• CX-516 (1-BCP; BDP-12, SPD-420; AMPAlex) – AMPA receptor modulator ^[85]
• EM-036 (memantine analogue) – ionotropic glutamate NMDA receptor antagonist, other actions ^[86]
• Fasoracetam (AEVI-001, LAM-105, MDGN-001, NFC-1, NS-105) – various actions and racetam ^[87]
• Fluoxetine rapid-dissolve (AT-001; AT001; NPL-2008; Serelsa; Zydis™ ODT fluoxetine) – serotonin reuptake inhibitor ^[88]
• GTS-21 (DMXB-A, DMXB-A sustained release, DMXB-A-SR) – α₇ nicotinic acetylcholine receptor partial agonist ^[89]
• Ketamine intranasal (RVT-701) – ionotropic glutamate NMDA receptor antagonist ^[90]
• Memantine (Namenda) – ionotropic glutamate NMDA receptor antagonist, other actions ^[91]
• Oxytocin intranasal (Syntocinon Nasal Spray; TUR 001) – oxytocin receptor agonist ^[92]
• Research programme: AMPA receptor agonists (ampakines, AMPAkines; CX compounds) - RespireRx – ionotropic glutamate AMPA receptor agonists ^[93]
• 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 ^[94]
• Risperidone extended-release (Risperisphere) – dopamine D₂ and D₃ receptor antagonist, serotonin 5-HT_1B, 5-HT_2A, 5-HT_2C, and 5-HT₇ receptor antagonist or inverse agonist, α₁- and α₂-adrenergic receptor antagonist, histamine H₁ receptor inverse agonist, and atypical antipsychotic ^[95]
• Secretin (INN-329, RG-1068; SecreFlo) – medical imaging enhancer (diagnosis) ^[96]
• Suramin (Antrypol) – DNA-directed DNA polymerase inhibitor and intercellular signaling peptide/protein inhibitor ^[97]
• Trichuris suis ova (CNDO-201, TSO, TSO-2500, TSO-7500) – immunomodulator ^[98]
• Trofinetide (Daybue; G-2Me-PE; Glycyl-2-methyl-L-prolyl-L-glutamic acid; NNZ-2566; IGF-1 (1–3) analogue) – unknown / various actions ^[99]

Clinically used drugs

Approved drugs

• Aripiprazole (Abilify) – dopamine D₂ and D₃ partial agonist, serotonin 5-HT_1A and 5-HT₇ receptor partial agonist, serotonin 5-HT_2A and 5-HT_2B receptor antagonist or inverse agonist, and atypical antipsychotic ^{[100] [101]}
• Risperidone (Risperdal) – dopamine D₂ and D₃ receptor antagonist, serotonin 5-HT_1B, 5-HT_2A, 5-HT_2C, and 5-HT₇ receptor antagonist or inverse agonist, α₁- and α₂-adrenergic receptor antagonist, histamine H₁ receptor inverse agonist, and atypical antipsychotic ^{[102] [101]}

Off-label drugs

• α₂-Adrenergic receptor agonists (e.g., clonidine, guanfacine) ^{[101] [82] [103]}
• Anticonvulsants/mood stabilizers (e.g., valproic acid, lamotrigine) ^[103]
• Antipsychotics (non-licensed) (e.g., haloperidol, olanzapine) ^{[103] [104]}
• Dietary supplements (e.g., N-acetylcysteine, omega-3 fatty acids, sulforaphane) ^{[82] [103]}
• Entactogens (serotonin releasing agents) (e.g., MDMA) ^{[105] [106] [107]}
• Melatonin receptor agonists (e.g., melatonin) ^{[101] [82]}
• NMDA receptor antagonists (e.g., memantine, amantadine) ^{[108] [109] [110] [111]}
• Norepinephrine reuptake inhibitors (NRIs) (e.g., atomoxetine) ^{[101] [82] [103]}
• Opioid receptor antagonists (e.g., naltrexone) ^[103]
• Other antidepressants (e.g., mirtazapine) ^[103]
• Oxytocin receptor agonists (e.g., oxytocin) ^{[82] [103]}
• Probiotics and prebiotics ^{[112] [113] [103]}
• Psychostimulants (norepinephrine–dopamine releasing agents and/or reuptake inhibitors) (e.g., amphetamine, methylphenidate) ^{[82] [101]}
• Selective serotonin reuptake inhibitors (SSRIs) (e.g., fluoxetine, fluvoxamine, sertraline, citalopram) ^{[101] [82] [103]}
• Serotonergic psychedelics (e.g., psilocybin, lysergic acid diethylamide (LSD)) ^[114]
• Serotonin–norepinephrine reuptake inhibitors (SNRIs) (e.g., milnacipran) ^{[82] [103]}
• Serotonin releasing agents (e.g., fenfluramine—withdrawn and no longer recommended) ^{[115] [116] [114] [101]}
• Serotonin 5-HT_1A receptor agonists (e.g., buspirone) ^{[103] [117]}
• Tricyclic antidepressants (TCAs) (e.g., clomipramine) ^{[82] [103] [101]}

See also

• List of investigational drugs
• List of investigational aggression drugs
• List of investigational agitation drugs
• List of investigational social anxiety disorder drugs

References

1. "CM 4612". AdisInsight.
2. "Brexpiprazole - Lundbeck/Otsuka". AdisInsight.
3. "Cariprazine - Gedeon Richter". AdisInsight.
4. "Lumateperone - Intra-Cellular Therapies". AdisInsight.
5. "Lurasidone - Sumitomo Pharma". AdisInsight.
6. "Tasimelteon - Vanda Pharmaceuticals". AdisInsight.
7. "AB 2004". AdisInsight.
8. "Acamprosate - Confluence Pharmaceuticals". AdisInsight.
9. "Alogabat - Genentech". AdisInsight.
10. "Arbaclofen - Seaside Therapeutics". AdisInsight.
11. "Cannabidiol - GW Pharmaceuticals". AdisInsight.
12. "Cannabidiol - Zynerba Pharmaceuticals". AdisInsight.
13. "Cannabidivarin - GW Pharmaceuticals". AdisInsight.
14. "CP 101 - Finch Therapeutics Group". AdisInsight.
15. "JNJ 42165279". AdisInsight.
16. "Oxytocin - Tonix Pharmaceuticals". AdisInsight.
17. "Pitolisant - Bioprojet". AdisInsight.
18. "Racemetirosine - Yamo Pharmaceuticals". AdisInsight.
19. "Suramin - PaxMedica". AdisInsight.
20. "ML 004". AdisInsight.
21. "R(-)-Methylenedioxymetamfetamine - Mindmed". AdisInsight.
22. "STP 1". AdisInsight.
23. "Sulforafan alfadex - TheraCryf". AdisInsight.
24. Wittkowski KM, Sonakya V, Bigio B, Tonn MK, Shic F, Ascano M, Nasca C, Gold-Von Simson G (2014). "A novel computational biostatistics approach implies impaired dephosphorylation of growth factor receptors as associated with severity of autism". Transl Psychiatry. 4 (1): e354. doi:10.1038/tp.2013.124. PMC   3905234 . PMID   24473445.
25. "Bryostatin 1 - Synaptogenix". AdisInsight.
26. "CJRB 303". AdisInsight.
27. "Debamestrocel - BrainStorm Cell Therapeutics". AdisInsight.
28. "FIN 211". AdisInsight.
29. "Phacetoperane - NLS Pharmaceutics Ltd". AdisInsight.
30. Konofal E, Lecendreux M, Bizot JC, Lormier AT, Figadère B (2024). "NLS-3 (Levophacetoperane or (R,R) Phacetoperane): A Reverse Ester of Methylphenidate: A Literature Review". Curr Med Chem. 31 (9): 1069–1081. doi:10.2174/0929867330666230120161837. PMID   36683369.
31. "Delving into the Latest Updates on LIT-001 with Synapse". Synapse. 2 August 2024. Retrieved 18 August 2024.
32. Nashar PE, Whitfield AA, Mikusek J, Reekie TA (2022). "The Current Status of Drug Discovery for the Oxytocin Receptor". Oxytocin. Methods Mol Biol. Vol. 2384. Springer. pp. 153–174. doi:10.1007/978-1-0716-1759-5_10. ISBN   978-1-0716-1758-8. PMID   34550574.
33. Kaur H, Karabulut S, Gauld JW, Fagot SA, Holloway KN, Shaw HE, Fantegrossi WE (2023). "Balancing Therapeutic Efficacy and Safety of MDMA and Novel MDXX Analogues as Novel Treatments for Autism Spectrum Disorder". Psychedelic Medicine. 1 (3): 166–185. doi:10.1089/psymed.2023.0023. PMC   11661495 . PMID   40046567. Author Disclosure Statement: H.K. is Vice President of Research at PharmAla Biotech and is listed as an inventor on patents related to the research in this review article. H.E.S. receives salary supported from a contract between PharmAla Biotech and UAMS. W.E.F. receives research funds and salary support from a contract between PharmAla Biotech and UAMS. The other authors have no conflicts to disclose.
34. PharmAla Biotech (23 February 2023). "Patent Application Published for PharmAla Biotech's MDMA Analogs". GlobeNewswire News Room (Press release). Retrieved 10 November 2024.
35. PharmAla Biotech (26 March 2024). "PharmAla Biotech Signs Sale Agreement with Numinus". GlobeNewswire News Room (Press release). Retrieved 10 November 2024.
36. Clark MR, Shaw HE, Fantegrossi WE (March 2024). Poster 21: In vivo characterization of MBDB and its enantiomers in C57BL/6 and autism-like BTBR T⁺Itpr3^tf/J mice (PDF). 16th Annual Behavior, Biology, and Chemistry: Translational Research in Substance Use Disorders, San Antonio, Texas, Embassy Landmark, 22-24 March 2024.
37. "Psilocin - Pilz Bioscience". AdisInsight.
38. "QBM 001". AdisInsight.
39. Lesko L (13 November 2018). "Biomarkers for autism". Drug Discovery News. Retrieved 10 November 2024. According to Derham, "This drug (QBM-001), acts as an allosteric regulator of faulty channels in the brain to potentially alleviate the condition and allow toddlers to actively develop language and speech and avoid life-long speech and intellectual disability of being non-verbal. QBM-001 also reduces inflammation in the brain, and by so doing, can reduce the amount of long-term nerve loss."
40. "Baeocystin - Pilz Bioscience". AdisInsight.
41. Rakoczy RJ, Runge GN, Sen AK, Sandoval O, Wells HG, Nguyen Q, Roberts BR, Sciortino JH, Gibbons WJ, Friedberg LM, Jones JA, McMurray MS (October 2024). "Pharmacological and behavioural effects of tryptamines present in psilocybin-containing mushrooms". Br J Pharmacol. 181 (19): 3627–3641. doi: 10.1111/bph.16466 . PMID   38825326.
42. "Norpsilocin - Pilz Bioscience". AdisInsight.
43. "Roluperidone - Minerva Neurosciences/Mitsubishi Tanabe Pharma Corporation". AdisInsight.
44. "Squalamine - Enterin". AdisInsight.
45. "Psilocybin - COMPASS Pathways". AdisInsight.
46. "Research programme: autism therapeutic - Innovation Pharmaceuticals". AdisInsight.
47. "AB 1224". AdisInsight.
48. "AGX 201". AdisInsight.
49. Devadoss, Thangaraj; Pingili, Ravindra Babu (2024). "Drugs Under Clinical Trials for the Treatment of Migraine". Management of Migraine Pain. Singapore: Springer Nature Singapore. p. 229–249. doi:10.1007/978-981-97-4529-6_12. ISBN   978-981--974528-9 . Retrieved 1 October 2025. The AGX-201 is chemically 2-(4-imidazolyl)ethylamine dihydrochloride, refer to Fig. 12.7 for the chemical structure. [...] AGX-201 is a dihydrochloride salt of histamine. [...]
50. Pellesi L, Do TP, Hougaard A (April 2024). "Pharmacological management of migraine: current strategies and future directions". Expert Opin Pharmacother. 25 (6): 673–683. doi:10.1080/14656566.2024.2349791. PMID   38720629. AGX-201 is a histamine receptor modulator that acts as an antagonist at H1 receptors and an agonist at H3 receptors. This dual mechanism of action offer a unique approach to mitigate migraine symptoms by reducing the release of proinflammatory neuropeptides from trigeminal nerve endings. Subcutaneous AGX-201 is currently tested in a phase II trial (NCT02021474) enrolling subjects with migraine requiring prophylactic treatment.
51. "Aminolevulinic acid/sodium ferrous citrate - SBI Pharmaceuticals". AdisInsight.
52. "Aripiprazole transdermal - Aequus Pharmaceuticals". AdisInsight.
53. "BAER 101". AdisInsight.
54. "BBP 472". AdisInsight.
55. "Co-crystallised fasoracetam - Avalo Therapeutics". AdisInsight.
56. "Guanfacine once-daily - Takeda". AdisInsight.
57. "Research programme: autism spectrum disorder therapeutics - KoBioLabs". AdisInsight.
58. "NBTX 001". AdisInsight.
59. "Oxytocin intranasal - OptiNose". AdisInsight.
60. "Research programme: allosteric modulators - Addex Therapeutics". AdisInsight.
61. "Research programme: antisense oligonucleotide therapeutics - RogCon U.R". AdisInsight.
62. "Research programme: autism and obesity therapeutics - Berand Neuropharmacology". AdisInsight.
63. "Research programme: brain development disorder therapeutics - Seaside Therapeutics". AdisInsight.
64. "Research programme: cannabinoid receptor modulators - GW Pharmaceuticals". AdisInsight.
65. "Research programme: cannabis extract therapeutics - Cannabis Science". AdisInsight.
66. "Research programme: central nervous system therapeutics - AbbVie/Rugen". AdisInsight.
67. "Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals". AdisInsight.
68. "Research programme: CNS disorders therapeutics - Sage Therapeutics". AdisInsight.
69. "Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation". AdisInsight.
70. "Research programme: GPCR modulators - Nxera Pharma". AdisInsight.
71. "Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma". AdisInsight.
72. "Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics". AdisInsight.
73. "Research programme: oxytocin intranasal - Pastorus Pharma". AdisInsight.
74. "Research programme: therapeutic autoantibodies - Sengenics". AdisInsight.
75. "RG 7713". AdisInsight.
76. "Tideglusib - AMO Pharma". AdisInsight.
77. "Vafidemstat - Oryzon Genomics". AdisInsight.
78. "Balovaptan - Roche". AdisInsight.
79. "Blarcamesine - Anavex Life Sciences". AdisInsight.
80. "Brilaroxazine - Reviva Pharmaceuticals". AdisInsight.
81. "Bumetanide oral - Neurochlore". AdisInsight.
82. Aishworiya R, Valica T, Hagerman R, Restrepo B (January 2022). "An Update on Psychopharmacological Treatment of Autism Spectrum Disorder". Neurotherapeutics. 19 (1): 248–262. doi:10.1007/s13311-022-01183-1. PMC   9130393 . PMID   35029811.
83. "Autism drug fails in late-stage trials, Servier says". biopharma.dive. 2021-01-08. Retrieved 2025-02-13.
84. "Carbetocin - Travere Therapeutics". AdisInsight.
85. "CX 516". AdisInsight.
86. "EM 036". AdisInsight.
87. "Fasoracetam - Avalo Therapeutics". AdisInsight.
88. "Fluoxetine-rapid dissolve - Autism Therapeutics". AdisInsight.
89. "GTS 21". AdisInsight.
90. "Ketamine intranasal - Roivant Sciences". AdisInsight.
91. "Memantine - Children's Medical Center Corporation/Merz Pharma". AdisInsight.
92. "Oxytocin intranasal - Vyera Pharmaceuticals". AdisInsight.
93. "Research programme: AMPA receptor agonists - RespireRx". AdisInsight.
94. "Research programme: NMDA receptor modulators - AbbVie". AdisInsight.
95. "Risperidone extended release - Xbrane". AdisInsight.
96. "Secretin - 9 Meters Biopharma". AdisInsight.
97. "Suramin sodium - Bayer". AdisInsight.
98. "Trichuris suis ova - Fortress Biotech". AdisInsight.
99. "Trofinetide - ACADIA Pharmaceuticals/Neuren Pharmaceuticals". AdisInsight.
100. "Aripiprazole - Bristol-Myers Squibb/Otsuka Pharmaceutical". AdisInsight.
101. Howes OD, Rogdaki M, Findon JL, Wichers RH, Charman T, King BH, Loth E, McAlonan GM, McCracken JT, Parr JR, Povey C, Santosh P, Wallace S, Simonoff E, Murphy DG (January 2018). "Autism spectrum disorder: Consensus guidelines on assessment, treatment and research from the British Association for Psychopharmacology". J Psychopharmacol. 32 (1): 3–29. doi:10.1177/0269881117741766. PMC   5805024 . PMID   29237331.
102. "Risperidone - Janssen". AdisInsight.
103. Siafis S, Çıray O, Wu H, Schneider-Thoma J, Bighelli I, Krause M, Rodolico A, Ceraso A, Deste G, Huhn M, Fraguas D, San José Cáceres A, Mavridis D, Charman T, Murphy DG, Parellada M, Arango C, Leucht S (March 2022). "Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis". Mol Autism. 13 (1): 10. doi: 10.1186/s13229-022-00488-4 . PMC   8896153 . PMID   35246237.
104. Siegel M, Beaulieu AA (August 2012). "Psychotropic medications in children with autism spectrum disorders: a systematic review and synthesis for evidence-based practice". J Autism Dev Disord. 42 (8): 1592–1605. doi:10.1007/s10803-011-1399-2. PMID   22068820.
105. Kisely S, Connor M, Somogyi AA, Siskind D (March 2023). "A systematic literature review and meta-analysis of the effect of psilocybin and methylenedioxymethamphetamine on mental, behavioural or developmental disorders". Aust N Z J Psychiatry. 57 (3): 362–378. doi:10.1177/00048674221083868. PMID   35285280.
106. Danforth AL, Struble CM, Yazar-Klosinski B, Grob CS (January 2016). "MDMA-assisted therapy: A new treatment model for social anxiety in autistic adults". Prog Neuropsychopharmacol Biol Psychiatry. 64: 237–249. doi: 10.1016/j.pnpbp.2015.03.011 . PMID   25818246.
107. Chaliha D, Mamo JC, Albrecht M, Lam V, Takechi R, Vaccarezza M (2021). "A Systematic Review of the MDMA Model to Address Social Impairment in Autism". Curr Neuropharmacol. 19 (7): 1101–1154. doi:10.2174/1570159X19666210101130258. PMC   8686313 . PMID   33388021.
108. Brignell A, Marraffa C, Williams K, May T (August 2022). "Memantine for autism spectrum disorder". Cochrane Database Syst Rev. 8 (8) CD013845. doi:10.1002/14651858.CD013845.pub2. PMC   9409629 . PMID   36006807.
109. Aboul-Fotouh S, Zohny SM, Elnahas EM, Habib MZ, Hassan GA (February 2025). "Can memantine treat autism? Answers from preclinical and clinical studies". Neurosci Biobehav Rev. 169 106019. doi:10.1016/j.neubiorev.2025.106019. PMID   39826825.
110. Shamabadi A, Karimi H, Arabzadeh Bahri R, Motavaselian M, Akhondzadeh S (March 2024). "Emerging drugs for the treatment of irritability associated with autism spectrum disorder". Expert Opin Emerg Drugs. 29 (1): 45–56. doi:10.1080/14728214.2024.2313650. PMID   38296815.
111. Joshi G, Gönenc A, DiSalvo M, Faraone SV, Ceranoglu TA, Yule AM, Uchida M, McDougle CJ, Wozniak J (October 2025). "Memantine to Treat Social Impairment in Youths With Autism Spectrum Disorder: A Randomized Clinical Trial". JAMA Netw Open. 8 (10): e2534927. doi:10.1001/jamanetworkopen.2025.34927. PMID   41032298.
112. Zhang S, Han F, Wang Q, Fan F (January 2024). "Probiotics and Prebiotics in the Treatment of Autism Spectrum Disorder: A Narrative Review". J Integr Neurosci. 23 (1): 20. doi: 10.31083/j.jin2301020 . PMID   38287844.
113. He X, Liu W, Tang F, Chen X, Song G (March 2023). "Effects of Probiotics on Autism Spectrum Disorder in Children: A Systematic Review and Meta-Analysis of Clinical Trials". Nutrients. 15 (6): 1415. doi: 10.3390/nu15061415 . PMC   10054498 . PMID   36986145.
114. Markopoulos A, Inserra A, De Gregorio D, Gobbi G (2021). "Evaluating the Potential Use of Serotonergic Psychedelics in Autism Spectrum Disorder". Front Pharmacol. 12 749068. doi: 10.3389/fphar.2021.749068 . PMC   8846292 . PMID   35177979.
115. Heifets BD, Salgado JS, Taylor MD, Hoerbelt P, Cardozo Pinto DF, Steinberg EE, Walsh JJ, Sze JY, Malenka RC (December 2019). "Distinct neural mechanisms for the prosocial and rewarding properties of MDMA". Sci Transl Med. 11 (522). doi:10.1126/scitranslmed.aaw6435. PMC   7123941 . PMID   31826983. FEN has been reported to improve social deficits in children with autism (34). However, like MDMA, long-term and/or heavy use of FEN is associated with cardiovascular and neurological toxicity (1, 7, 35).
116. Aman MG, Kern RA (July 1989). "Review of fenfluramine in the treatment of the developmental disabilities". J Am Acad Child Adolesc Psychiatry. 28 (4): 549–565. doi:10.1097/00004583-198907000-00014. PMID   2670881.
117. Gupta N, Gupta M, Gandhi R (May 2023). "Buspirone in Autism Spectrum Disorder: A Systematic Review". Cureus. 15 (5) e39304. doi: 10.7759/cureus.39304 . PMC   10291962 . PMID   37378184.

External links

• AdisInsight - Springer