Which autism spectrum disorder drugs are in Phase 3?

Phase 3 (or later) programs for autism spectrum disorder include Lumateperone (ITI-007) and NTI164.

What are the next autism spectrum disorder trial readouts?

Upcoming catalysts include DT402 (2026), Lumateperone (ITI-007) (1H 2027), Lumateperone (ITI-007) (1H 2027) and NTI164 (July 2029).

How many clinical trials are recruiting for autism spectrum disorder?

56 drug-interventional autism spectrum disorder trials are currently recruiting participants, out of 420 registered interventional trials.

Neurodevelopmental Disorders

Autism spectrum disorder drug pipeline

ICD-11 6A02
MeSH D000067877

4 compounds from 4 companies are in active clinical development for autism spectrum disorder, including 2 at Phase 3 or beyond.

4Compounds

4Companies

2Phase 3+

4Upcoming readouts

70Trials recruiting

Phase 21

DT402Definium Therapeutics, Inc.SERT Releaser

Phase 32

Discontinued1

BalovaptanF. Hoffmann-La Roche LtdV1A receptor Antagonist

Mechanisms: Small molecule (2) · Natural product (1) · Psychedelic (1)

Pipeline data compiled October 2026 · every fact sourced.

Upcoming readouts & recent results

2026AnticipatedDT402Topline dataNCT07303907
Initial Phase 2a data for DT402 in autism spectrum disorder anticipated in 2026 ↗
1H 2027AnticipatedLumateperone (ITI-007)Topline dataNCT06706674
Anticipated topline of Study 602, the second pivotal Phase 3 of lumateperone for irritability associated with autism spectrum disorder (pediatric); primary endpoint ABC-Irritability at Week 6. ↗
1H 2027AnticipatedLumateperone (ITI-007)Topline dataNCT06690398
Anticipated topline of Study 601, the first pivotal Phase 3 of lumateperone for irritability associated with autism spectrum disorder (pediatric); primary endpoint ABC-Irritability at Week 6. ↗
July 2029AnticipatedNTI164Registry resultsNCT07257939
ClinicalTrials.gov estimated primary completion of the Phase 3 Beyond Harmony study of NTI164 in autism spectrum disorder: July 2029. ↗
2026-08-06ReportedNTI164Full resultsmetNCT05626959
HARMONY Phase II/III (54 children with Level II/III ASD, NTI164 up to 20 mg/kg/day vs placebo for 8 weeks) published: statistically significant improvements over placebo in overall clinical severity, adaptive functioning, social responsiveness and affective symptoms, with a favourable safety profile. ↗
2022-08-01ReportedBalovaptanFull resultsmissedNCT02901431
aV1ation Phase 2 (339 children and adolescents 5-17 with ASD, 24 weeks): no difference vs placebo on Vineland-II 2DC (LS-mean difference -0.16; 90% CI -2.56 to 2.23; P=.91) or any secondary endpoint. ↗
2022-02-10ReportedBalovaptanFull resultsmissedNCT03504917
V1aduct Phase 3 (322 autistic adults, balovaptan 10 mg vs placebo, 24 weeks): terminated for futility; no improvement on Vineland-II 2DC (interim LS-mean change 2.91 vs 4.75 placebo; difference -1.84, 95% CI -5.15 to 1.48). ↗
2019-05-08ReportedBalovaptanFull resultsmissedNCT01793441
VANILLA Phase 2 (223 adult men with ASD, 12 weeks): no separation from placebo on the primary endpoint (SRS-2); dose-dependent improvements on the secondary Vineland-II composite at 4 mg and 10 mg. ↗

See the full catalyst calendar →

Compounds in development (4)

Compound	Company	Phase	Status	Mechanism
Lumateperone (ITI-007)	Johnson & Johnson	Phase 3	Recruiting	5-HT2A Antagonist
NTI164	Neurotech International Limited	Phase 3	Recruiting	Natural product
DT402	Definium Therapeutics, Inc.	Phase 2	Recruiting	SERT Releaser
BalovaptanFDA Breakthrough Therapy (Autism Spectrum Disorder; Granted 2018-01-29)	F. Hoffmann-La Roche Ltd	Discontinued	Discontinued	V1A receptor Antagonist

Trials recruiting now (56)

56 drug-interventional autism spectrum disorder trials recruiting now, of 420 registered interventional trials. Browse all on ClinicalTrials.gov →

Phase 4Buspirone for Anxiety in Autistic Youth
buspirone, Placebo
Massachusetts General Hospitaln=20Started Aug 2026NCT07439042
Phase 4The TEAM Study - Treatment Efficacy for Autism/Attention Using Mixed Amphetamine
Adderall XR (mixed salts amphetamine), Placebo
Gagan Joshin=196United States · 1 siteStarted Feb 2026NCT06853665
Phase 4AWARE: Management of ADHD in Autism Spectrum Disorder
Randomization to either Amphetamine (AMP) class of stimulant medication or Methylphenidate (MPH) class of stimulant medication, Randomization to either Alpha 2 agonist class of medication or alternate class of stimulant.
Daniel Couryn=500Canada, United States · 12 sitesStarted Oct 2023NCT05916339
Phase 3A Phase III Double-blind, Randomised, Placebo-controlled Study Investigating the Efficacy and Safety of NTI164 in Children and Young Adults With Autism Spectrum Disorder
NTI164, Placebo
Fenix Innovation Groupn=200Australia · 1 siteStarted Sep 2026Tracked: NTI164 NCT07257939
Phase 3A Study of KarXT + KarX-EC for Treatment of Irritability in Children and Adolescents With Autism Spectrum Disorder
KarXT, KarX-EC, KarXT + KarX-EC Matching Placebo
Bristol-Myers Squibb Companyn=176France, Germany +7 · 55 sitesStarted Sep 2026NCT07285798
Phase 3A Study of KarXT + KarX-EC for Treatment of Irritability in Children and Adolescents With Autism
KarXT, KarX-EC, KarXT + KarX-EC Matching Placebo
Bristol-Myers Squibb Companyn=176Argentina, Australia +3 · 45 sitesStarted Sep 2026NCT07284745
Phase 3A Study to Evaluate the Long-term Safety and Tolerability of KarXT and KarX-EC for the Treatment of Schizophrenia and Autism-Related Irritability in Adolescents, Respectively
KarXT
Karuna Therapeuticsn=400Argentina, Canada +8 · 58 sitesStarted Apr 2026NCT07424404
Phase 3Single Dose Double-blind, Placebo-controlled Cross-over (SDDBPCCO) Shiftability Study, Will be Followed by a 10-week Open-label Study With Arbaclofen (4 Weeks of Titration and Then 6 Weeks of Active/Stable Treatment). The Effects of Arbaclofen on Target EEG and ERG Metrics Will be Associated With th
Arbaclofen
Hospital General Universitario Gregorio Marañonn=103France, Spain · 5 sitesStarted Nov 2025NCT07655115
Phase 3Study 602: A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age
Lumateperone high dose, Lumateperone low dose, Placebo
Intra-Cellular Therapies, Inc.n=174United States · 39 sitesStarted Dec 2024Tracked: Lumateperone (ITI-007)NCT06706674
Phase 3Study 601: A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of Lumateperone in the Treatment of Irritability Associated With Autism Spectrum Disorder in Pediatric Patients 5 to 17 Years of Age
Lumateperone high dose, Lumateperone low dose, Placebo
Intra-Cellular Therapies, Inc.n=174United States · 37 sitesStarted Nov 2024Tracked: Lumateperone (ITI-007)NCT06690398
Phase 3An Open-label, Multicenter Trial to Assess the Safety and Tolerability of Lumateperone in the Treatment of Pediatric Patients With Schizophrenia, Bipolar Disorder or Autism Spectrum Disorder
Lumateperone
Intra-Cellular Therapies, Inc.n=500Serbia, United States · 50 sitesStarted Jan 2024Tracked: Lumateperone (ITI-007)NCT06229210
Phase 3Role of Bumetanide in Treatment of Autism
Bumetanide, Placebo
Sherief Abd-Elsalamn=80Egypt · 1 siteStarted Jan 2021NCT04766177

Showing the 12 most advanced of 56 recruiting drug trials · see all on ClinicalTrials.gov →

Frequently asked

How many drugs are in development for autism spectrum disorder?: 4 compounds from 4 companies are in active clinical development for autism spectrum disorder, including 2 at Phase 3 or beyond.
Which autism spectrum disorder drugs are in Phase 3?: Phase 3 (or later) programs for autism spectrum disorder include Lumateperone (ITI-007) and NTI164.
What are the next autism spectrum disorder trial readouts?: Upcoming catalysts include DT402 (2026), Lumateperone (ITI-007) (1H 2027), Lumateperone (ITI-007) (1H 2027) and NTI164 (July 2029).
How many clinical trials are recruiting for autism spectrum disorder?: 56 drug-interventional autism spectrum disorder trials are currently recruiting participants, out of 420 registered interventional trials.