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List of investigational autism and pervasive developmental disorder drugs

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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 It is likely to become outdated with time.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

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Preregistration

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  • CM-4612 (CM-ADHD; CM-AT; CM-PK) – enzyme modulator/replacement for gastrointestinal or pancreatic secretory deficiencies [1]

Phase 3

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  • Brexpiprazole (Rexulti) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2A 5-HT2B, and 5-HT7 receptor antagonist, α1- and α2-adrenergic receptor antagonist, and atypical antipsychotic [2]
  • Bumetanide oral liquid (S-95008) – sodium–potassium–chloride symporter inhibitor [3]
  • Cariprazine (Reagila, Symvenu, Vraylar; MP-214, RGH-188, WID-RGC20) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2B receptor antagonist, and atypical antipsychotic [4]
  • Lumateperone (Caplyta) – dopamine D1 and D2 receptor antagonist, serotonin 5-HT2A receptor antagonist, α1-adrenergic receptor antagonist, serotonin reuptake inhibitor, and atypical antipsychotic [5]
  • Lurasidone (Latuda) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1A receptor partial agonist, serotonin 5-HT2A and 5-HT7 receptor antagonist, α2C-adrenergic receptor antagonist, and atypical antipsychotic [6]
  • Tasimelteon (Hetlioz) – melatonin MT1 and MT2 receptor agonist [7]

Phase 2

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  • AB-2004 – microbiome modulator [8]
  • Acamprosate (AOP-2020; Campral; SF-679/SF-775; SF-999) – unknown / GABAA receptor positive allosteric modulator and ionotropic glutamate NMDA receptor antagonist [9]
  • Alogabat (RG-7816, RG7816, RO-7017773; GABA-A-α5 PAM) – GABAA α5 subunit-containing receptor positive allosteric modulator [10]
  • Arbaclofen ((R)-baclofen; STX-209) – GABAB receptor agonist [11]
  • Cannabidiol (CBD; Epidiolex) – cannabinoid receptor modulator, other actions [12]
  • Cannabidiol transdermal patch/gel (Zygel; ZYN-002) – cannabinoid receptor modulator, other actions [13]
  • Cannabidivarin (CBDV; GWP-42006) – non-intoxicating cannabinoid receptor modulator, other actions [14]
  • CP-101 – bacteria relacement and gastrointestinal microbiome modulator [15]
  • JNJ-42165279 (JNJ-5279) – fatty acid amide hydrolase (FAAH) inhibitor [16]
  • Oxytocin (intranasal potentiated oxytocin; TI-001, TI-114, TNX-1900, TNX-2900) – oxytocin receptor agonist [17]
  • Pitolisant (Wakix; tiprolisant) – histamine H3 receptor inverse agonist [18]
  • Racemetirosine (DL-α-methyltyrosine; L1-79) – tyrosine hydroxylase inhibitor [19]
  • Suramin (IV suramin; PAX-101/PAX-102) – DNA synthesis inhibitor and anti-purinergic agent [20]
  • Zolmitriptan modified-release (ML-004, ML004) – serotonin 5-HT1B and 5-HT1D receptor agonist [21]

Phase 1

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  • (R)-MDMA ((R)-midomafetamine; MM-402) – serotonin, norepinephrine, and dopamine releasing agent and weak serotonin 5-HT2A, 5-HT2B, and 5-HT2C receptor agonist (entactogen and weak psychedelic hallucinogen) [22]
  • STP-1 (STALICLA Therapeutic Package 1) – phosphoric diester hydrolase inhibitor and sodium–potassium–chloride symporter inhibitor [23]
  • Sulforafan alfadex (sulforaphane alfadex; sulforafan α-cyclodextrin; SFX-01, STP-2; broccoli sprout extract) – various actions [24]

Preclinical

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  • 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 "Delving into the Latest Updates on LIT-001 with Synapse". Synapse. 2 August 2024. Retrieved 18 August 2024.[31]
  • Psilocin (PLZ-1015) – non-selective serotonin receptor agonist and psychedelic hallucinogen [32]
  • QBM-001 – undefined mechanism of action [33]

Research

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  • Baeocystin (PLZ-1019) – non-hallucinogenic serotonin 5-HT2A receptor agonist, other actions [34][35]
  • Norpsilocin (PLZ-1017) – non-hallucinogenic serotonin 5-HT2A receptor agonist, other actions [36][35]
  • Roluperidone (CYR-101, MIN-101, MT-210) – serotonin 5-HT2A, sigma σ2, and α1A-adrenergic receptor antagonist [37]
  • Squalamine (ENT-01; Enterin-01; kenterin) – various actions [38]

Phase unknown

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  • Psilocybin (COMP360; COMP-360) – non-selective serotonin receptor agonist and psychedelic hallucinogen [39]

Not under development

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Development suspended

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No development reported

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  • AB-1224 – microbiome modulator [41]
  • AGX-201 (histamine dihydrochloride salt) – histamine H1 receptor antagonist and histamine H3 receptor agonist [42]
  • Aminolevulinic acid/sodium ferrous citrate (5-ALA-SFC, 5-ALA/SFX; sodium ferrous citrate/aminolevulinic acid; SPP-003) – erythropoiesis stimulant and photosensitizer [43]
  • Aripiprazole transdermal (AQS-1301; transdermal aripiprazole) – dopamine D2 and D3 receptor partial agonist, serotonin 5-HT1A and 5-HT7 receptor partial agonist, serotonin 5-HT2A and 5-HT2B receptor antagonist or inverse agonist, and atypical antipsychotic [44]
  • BAER-101 (AZ-7325; AZD-7325) – selective GABAA α2 and α3 subunit-containing receptor positive allosteric modulator [45]
  • BBP-472 – phosphatidylinositol 3 kinase β (PI3Kβ) inhibitor [46]
  • Fasoracetam co-crystallised (co-crystallised fasoracetam; AEVI-004) – various actions and racetam [47]
  • Guanfacine once-daily (Guanfacine Carrier Wave; SPD-547) – α2-adrenergic receptor agonist [48]
  • KBLP-010 – bacteria replacement and microbiome modulator [49]
  • NBTX-001 – ionotropic glutamate NMDA receptor antagonist [50]
  • Oxytocin intranasal (OPN-300; OptiNose oxytocin) – oxytocin receptor agonist [51]
  • Research programme: allosteric modulators - Addex Therapeutics (various) – various actions [52]
  • Research programme: antisense oligonucleotide therapeutics - RogCon U.R (RCUR-313, RCUR-SMP) – voltage-gated sodium channel Nav1.2 expression stimulants [53]
  • Research programme: autism and obesity therapeutics - Berand Neuropharmacology – histone deacetylase inhibitors [54]
  • Research programme: brain development disorder therapeutics - Seaside Therapeutics (STX-110) – metabotropic glutamate mGlu5 receptor antagonists and muscarinic acetylcholine M1 receptor antagonists [55]
  • Research programme: cannabinoid receptor modulators - GW Pharmaceuticals (cannabigerol; CBG) – cannabinoid receptor modulators [56]
  • Research programme: cannabis extract therapeutics - Cannabis Science (CBIS compounds) – cannabinoid receptor modulators [57]
  • Research programme: central nervous system therapeutics - AbbVie/Rugen – undefined mechanism of action [58]
  • Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals – antioxidants, glutamate receptor modulators, SLC7A11 modulators [59]
  • Research programme: CNS disorders therapeutics - Sage Therapeutics (SAGE-105; SGE-202; SGE-301; SGE-516) – GABAA receptor modulators and ionotropic glutamate NMDA receptor modulators [60]
  • Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation – G protein-coupled receptor modulator and neuromedin U receptor modulator [61]
  • Research programme: GPCR modulators - Nxera Pharma – various actions [62]
  • Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma – bacteria replacements [63]
  • Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics – metabotropic glutamate mGlu5 receptor antagonists [64]
  • Research programme: oxytocin intranasal - Pastorus Pharma – neurotransmitter modulators/oxytocin receptor agonists [65]
  • Research programme: therapeutic autoantibodies - Sengenics – undefined mechanism of action [66]
  • RG-7713 (RG7713) – vasopressin V1A receptor antagonist [67]
  • Tideglusib (AMO-02, NP-031112, NP-12; Nypta, Zentylor) – glycogen synthase kinase 3β (GSK-3β) inhibitor [68]
  • Vafidemstat (ORY-2001) – dual lysine specific demethylase 1 (LSD1) inhibitor and monoamine oxidase B (MAO-B) inhibitor [69]
  • Xenon (NBTX-001) – ionotropic glutamate NMDA receptor antagonist and dissociative hallucinogen NBTX 001 - AdisInsight

Discontinued

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  • Balovaptan (RG-7314, RO-5028442, RO-5285119) – vasopressin V1A receptor antagonist [70]
  • Blarcamesine (AE-37, ANA001, ANAVEX 2-73) – sigma σ1, muscarinic acetylcholine M1, and ionotropic glutamate NMDA receptor agonist [71]
  • Brilaroxazine (RP-5063, RP-5000) – dopamine D2, D3, D4 receptor partial agonist, serotonin 5-HT1A receptor agonist, serotonin 5-HT2A, 5-HT2B, 5-HT7 receptor antagonist, and atypical antipsychotic [72]
  • Carbetocin (CYP-2001) – oxytocin receptor agonist [73]
  • CX-516 (1-BCP; BDP-12, SPD-420; AMPAlex) – AMPA receptor modulator [74]
  • EM-036 (memantine analogue) – ionotropic glutamate NMDA receptor antagonist, other actions [75]
  • Fasoracetam (AEVI-001, LAM-105, MDGN-001, NFC-1, NS-105) – various actions and racetam [76]
  • Fluoxetine rapid-dissolve (AT-001; AT001; NPL-2008; Serelsa; Zydis™ ODT fluoxetine) – serotonin reuptake inhibitor [77]
  • GTS-21 (DMXB-A, DMXB-A sustained release, DMXB-A-SR) – α7 nicotinic acetylcholine receptor partial agonist [78]
  • Ketamine intranasal (RVT-701) – ionotropic glutamate NMDA receptor antagonist [79]
  • Memantine (Namenda) – ionotropic glutamate NMDA receptor antagonist, other actions [80]
  • Oxytocin intranasal (Syntocinon Nasal Spray; TUR 001) – oxytocin receptor agonist [81]
  • Research programme: AMPA receptor agonists (ampakines, AMPAkines; CX compounds) - RespireRx – ionotropic glutamate AMPA receptor agonists [82]
  • 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 [83]
  • Risperidone extended-release (Risperisphere) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1B, 5-HT2A, 5-HT2C, and 5-HT7 receptor antagonist or inverse agonist, α1- and α2-adrenergic receptor antagonist, histamine H1 receptor inverse agonist, and atypical antipsychotic [84]
  • Secretin (INN-329, RG-1068; SecreFlo) – medical imaging enhancer (diagnosis) [85]
  • Suramin (Antrypol) – DNA-directed DNA polymerase inhibitor and intercellular signaling peptide/protein inhibitor [86]
  • Trichuris suis ova (CNDO-201, TSO, TSO-2500, TSO-7500) – immunomodulator [87]
  • Trofinetide (Daybue; G-2Me-PE; Glycyl-2-methyl-L-prolyl-L-glutamic acid; NNZ-2566; IGF-1 (1–3) analogue) – unknown / various actions [88]

Clinically used drugs

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Approved drugs

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  • Aripiprazole (Abilify) – dopamine D2 and D3 partial agonist, serotonin 5-HT1A and 5-HT7 receptor partial agonist, serotonin 5-HT2A and 5-HT2B receptor antagonist or inverse agonist, and atypical antipsychotic [89]
  • Risperidone (Risperdal) – dopamine D2 and D3 receptor antagonist, serotonin 5-HT1B, 5-HT2A, 5-HT2C, and 5-HT7 receptor antagonist or inverse agonist, α1- and α2-adrenergic receptor antagonist, histamine H1 receptor inverse agonist, and atypical antipsychotic [90]

Off-label drugs

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See also

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References

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  1. ^ "CM 4612". AdisInsight.
  2. ^ "Brexpiprazole - Lundbeck/Otsuka". AdisInsight.
  3. ^ "Bumetanide oral - Neurochlore". AdisInsight.
  4. ^ "Cariprazine - Gedeon Richter". AdisInsight.
  5. ^ "Lumateperone - Intra-Cellular Therapies". AdisInsight.
  6. ^ "Lurasidone - Sumitomo Pharma". AdisInsight.
  7. ^ "Tasimelteon - Vanda Pharmaceuticals". AdisInsight.
  8. ^ "AB 2004". AdisInsight.
  9. ^ "Acamprosate - Confluence Pharmaceuticals". AdisInsight.
  10. ^ "Alogabat - Genentech". AdisInsight.
  11. ^ "Arbaclofen - Seaside Therapeutics". AdisInsight.
  12. ^ "Cannabidiol - GW Pharmaceuticals". AdisInsight.
  13. ^ "Cannabidiol - Zynerba Pharmaceuticals". AdisInsight.
  14. ^ "Cannabidivarin - GW Pharmaceuticals". AdisInsight.
  15. ^ "CP 101 - Finch Therapeutics Group". AdisInsight.
  16. ^ "JNJ 42165279". AdisInsight.
  17. ^ "Oxytocin - Tonix Pharmaceuticals". AdisInsight.
  18. ^ "Pitolisant - Bioprojet". AdisInsight.
  19. ^ "Racemetirosine - Yamo Pharmaceuticals". AdisInsight.
  20. ^ "Suramin - PaxMedica". AdisInsight.
  21. ^ "ML 004". AdisInsight.
  22. ^ "R(-)-Methylenedioxymetamfetamine - Mindmed". AdisInsight.
  23. ^ "STP 1". AdisInsight.
  24. ^ "Sulforafan alfadex - TheraCryf". AdisInsight.
  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. ^ 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. pp. 153–174. doi:10.1007/978-1-0716-1759-5_10. ISBN 978-1-0716-1758-8. PMID 34550574.
  32. ^ "Psilocin - Pilz Bioscience". AdisInsight.
  33. ^ "QBM 001". AdisInsight.
  34. ^ "Baeocystin - Pilz Bioscience". AdisInsight.
  35. ^ a b 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.
  36. ^ "Norpsilocin - Pilz Bioscience". AdisInsight.
  37. ^ "Roluperidone - Minerva Neurosciences/Mitsubishi Tanabe Pharma Corporation". AdisInsight.
  38. ^ "Squalamine - Enterin". AdisInsight.
  39. ^ "Psilocybin - COMPASS Pathways". AdisInsight.
  40. ^ "Research programme: autism therapeutic - Innovation Pharmaceuticals". AdisInsight.
  41. ^ "AB 1224". AdisInsight.
  42. ^ "AGX 201". AdisInsight.
  43. ^ "Aminolevulinic acid/sodium ferrous citrate - SBI Pharmaceuticals". AdisInsight.
  44. ^ "Aripiprazole transdermal - Aequus Pharmaceuticals". AdisInsight.
  45. ^ "BAER 101". AdisInsight.
  46. ^ "BBP 472". AdisInsight.
  47. ^ "Co-crystallised fasoracetam - Avalo Therapeutics". AdisInsight.
  48. ^ "Guanfacine once-daily - Takeda". AdisInsight.
  49. ^ "Research programme: autism spectrum disorder therapeutics - KoBioLabs". AdisInsight.
  50. ^ "NBTX 001". AdisInsight.
  51. ^ "Oxytocin intranasal - OptiNose". AdisInsight.
  52. ^ "Research programme: allosteric modulators - Addex Therapeutics". AdisInsight.
  53. ^ "Research programme: antisense oligonucleotide therapeutics - RogCon U.R". AdisInsight.
  54. ^ "Research programme: autism and obesity therapeutics - Berand Neuropharmacology". AdisInsight.
  55. ^ "Research programme: brain development disorder therapeutics - Seaside Therapeutics". AdisInsight.
  56. ^ "Research programme: cannabinoid receptor modulators - GW Pharmaceuticals". AdisInsight.
  57. ^ "Research programme: cannabis extract therapeutics - Cannabis Science". AdisInsight.
  58. ^ "Research programme: central nervous system therapeutics - AbbVie/Rugen". AdisInsight.
  59. ^ "Research programme: CNS disorder therapeutics - Promentis Pharmaceuticals". AdisInsight.
  60. ^ "Research programme: CNS disorders therapeutics - Sage Therapeutics". AdisInsight.
  61. ^ "Research programme: G protein-coupled receptor modulating small molecules - Omeros Corporation". AdisInsight.
  62. ^ "Research programme: GPCR modulators - Nxera Pharma". AdisInsight.
  63. ^ "Research programme: immunomodulating bacteria-based therapeutics - 4D Pharma". AdisInsight.
  64. ^ "Research programme: metabotropic glutamate receptor 5 antagonists - Roche/Seaside Therapeutics". AdisInsight.
  65. ^ "Research programme: oxytocin intranasal - Pastorus Pharma". AdisInsight.
  66. ^ "Research programme: therapeutic autoantibodies - Sengenics". AdisInsight.
  67. ^ "RG 7713". AdisInsight.
  68. ^ "Tideglusib - AMO Pharma". AdisInsight.
  69. ^ "Vafidemstat - Oryzon Genomics". AdisInsight.
  70. ^ "Balovaptan - Roche". AdisInsight.
  71. ^ "Blarcamesine - Anavex Life Sciences". AdisInsight.
  72. ^ "Brilaroxazine - Reviva Pharmaceuticals". AdisInsight.
  73. ^ "Carbetocin - Travere Therapeutics". AdisInsight.
  74. ^ "CX 516". AdisInsight.
  75. ^ "EM 036". AdisInsight.
  76. ^ "Fasoracetam - Avalo Therapeutics". AdisInsight.
  77. ^ "Fluoxetine-rapid dissolve - Autism Therapeutics". AdisInsight.
  78. ^ "GTS 21". AdisInsight.
  79. ^ "Ketamine intranasal - Roivant Sciences". AdisInsight.
  80. ^ "Memantine - Children's Medical Center Corporation/Merz Pharma". AdisInsight.
  81. ^ "Oxytocin intranasal - Vyera Pharmaceuticals". AdisInsight.
  82. ^ "Research programme: AMPA receptor agonists - RespireRx". AdisInsight.
  83. ^ "Research programme: NMDA receptor modulators - AbbVie". AdisInsight.
  84. ^ "Risperidone extended release - Xbrane". AdisInsight.
  85. ^ "Secretin - 9 Meters Biopharma". AdisInsight.
  86. ^ "Suramin sodium - Bayer". AdisInsight.
  87. ^ "Trichuris suis ova - Fortress Biotech". AdisInsight.
  88. ^ "Trofinetide - ACADIA Pharmaceuticals/Neuren Pharmaceuticals". AdisInsight.
  89. ^ "Aripiprazole - Bristol-Myers Squibb/Otsuka Pharmaceutical". AdisInsight.
  90. ^ "Risperidone - Janssen". AdisInsight.
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