Autism Spectrum Disorder Pipeline Insight 2025: Comprehensive Development 

DelveInsight's "Autism Spectrum Disorder Pipeline Insight 2025" delivers extensive market intelligence encompassing 18+ pharmaceutical and biotechnology companies alongside 20+ investigational drugs within the Autism Spectrum Disorder development ecosystem. This comprehensive analysis covers ASD Pipeline drug candidate profiles spanning both clinical and nonclinical development stages. The report presents a detailed Autism Spectrum Disorder Pipeline Therapeutics evaluation organized by product category, developmental phase, delivery route, and molecular structure. Furthermore, it highlights discontinued and dormant pipeline assets within this therapeutic domain.

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Essential Highlights from the Autism Spectrum Disorder Pipeline Report

Recent Clinical Trial Developments

21 November 2025 - Vertero Therapeutics initiated a clinical investigation designed to assess the potential therapeutic benefits, safety profile, and tolerability of AB-2004 in participants experiencing irritability associated with autism spectrum disorder.

19 November 2025 - Bazelet Nehushtan Ltd announced a clinical trial aimed at evaluating whether cannabidiol (CBD, 28%) combined with terpenes and a minimal THC concentration (1%) can effectively reduce symptoms of autism, while assessing the safety characteristics of this treatment approach.

Pipeline Development Intelligence

DelveInsight's Autism Spectrum Disorder Pipeline analysis reveals a dynamic therapeutic landscape featuring 18+ active industry participants pursuing development of 20+ investigational therapies for Autism Spectrum Disorder treatment.

Leading Autism Spectrum Disorder Companies: Astrogen, MapLight Therapeutics, Axial Therapeutics, Aardvark Therapeutics, Ajna BioSciences, Intra-Cellular Therapies, Finch Therapeutics Group, PharmAla Biotech, among additional key players.

High-Potential Autism Spectrum Disorder Therapies: ML-004 (IR)/(ER) tablet, Arbaclofen, Terpenes-Enriched CBD-Predominant Oil, Terpenes-Enriched CBD Oil (THC-Free), L1-79, Cannabidivarin, Cariprazine, plus other promising candidates.

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Report Features and Value Proposition

The Autism Spectrum Disorder Pipeline Report delivers disease background information, pipeline scenario analysis, and therapeutic assessment of critical pipeline therapies within this domain. The Autism Spectrum Disorder Pipeline Report additionally emphasizes unmet medical needs in relation to Autism Spectrum Disorder treatment.

Autism Spectrum Disorder Disease Background

Autism spectrum disorder (ASD) represents a developmental disability resulting from neurological differences in brain structure and function. Certain individuals with ASD exhibit identifiable variations, such as genetic conditions. Additional causative factors remain under investigation. Researchers hypothesize that multiple Autism Spectrum Disorder causes interact synergistically to alter typical developmental pathways. Individuals with ASD may demonstrate behavioral patterns, communication styles, social interactions, and learning approaches that differ from neurotypical populations. Frequently, no distinguishing physical characteristics differentiate them from others. The capabilities of individuals with ASD demonstrate considerable variability. Autism Spectrum Disorder (ASD) serves as the umbrella terminology for autism, encompassing three distinct subtypes within the ASD classification. The 3 types of autism comprise: Autistic Disorder, Asperger's Syndrome, and Pervasive Development Disorder.

Investigational Autism Spectrum Disorder Drug Candidates

AST-001: Astrogen

AST-001 represents a small-molecule therapeutic developed by Astrogen for treating Autism Spectrum Disorder. AST-001 operates as an L-isomer of serine, demonstrating essential involvement in diverse neurological mechanisms. Preclinical ASD models have shown improvements in social deficits and restoration of dopamine neuron functionality. The therapeutic enhances dopamine neuron firing through normalization of particular ion channel activities, indicating potential as a treatment agent for enhancing social interaction and cognitive capabilities in patients with ASD. It addresses neurological pathways to ameliorate core symptoms in ASD patients and has progressed to Phase III clinical trials subsequent to regulatory authorization in South Korea.

ML004: MapLight Therapeutics

ML-004 serves as MapLight's primary clinical compound. Its selective pharmacological characteristics position it as a highly targeted therapy with constrained adverse effects. The organization is advancing ML-004 for multiple therapeutic applications, encompassing sociability and irritability in ASD alongside agitation and aggression in Alzheimer's disease. Presently, this therapeutic agent progresses through Phase II clinical development for Autism Spectrum Disorder treatment.

AJA001: Ajna BioSciences

AJA001 is a comprehensive cannabinoid formulation sourced from Cannabis sativa, engineered specifically for Autism Spectrum Disorder (ASD). Developed through DEFLORIA LLC, a collaborative venture between Ajna, Charlotte's Web, and BAT, it aspires to become the inaugural FDA-approved botanical hemp extract for ASD. Utilizing Charlotte's Web's patented genetic strains, AJA001 optimizes bioavailability, demonstrating promising symptom amelioration and quality-of-life enhancements in preliminary clinical investigations.

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Comprehensive Insights from the Autism Spectrum Disorder Pipeline Report

Company and Therapeutic Development Intelligence

The report delivers granular information regarding pharmaceutical companies pursuing therapies for Autism Spectrum Disorder treatment, including cumulative therapeutic counts within each organization's development portfolio targeting this indication.

Development Phase Categorization

Systematic assessment of diverse therapeutic candidates organized into early-stage, mid-stage, and late-stage development categories for Autism Spectrum Disorder Treatment.

Project Activity Analysis

Autism Spectrum Disorder Companies engaged in targeted therapeutic development programs, encompassing both active initiatives and inactive (dormant or terminated) projects.

Therapeutic Characterization Framework

Autism Spectrum Disorder Drugs undergoing development categorized by development phase, administration route, receptor target, monotherapy versus combination therapy strategy, mechanism of action diversity, and molecular classification.

Partnership and Investment Evaluation

Comprehensive examination of strategic partnerships (company-company alliances and company-academia collaborations), licensing agreements, and financing arrangements supporting future Autism Spectrum Disorder market advancement.

Key Autism Spectrum Disorder Companies

Astrogen, MapLight Therapeutics, Axial Therapeutics, Aardvark Therapeutics, Ajna BioSciences, Intra-Cellular Therapies, Finch Therapeutics Group, PharmAla Biotech, and additional industry participants.

Therapeutic Classification Systems

Route of Administration Analysis

The Autism Spectrum Disorder Pipeline report categorizes therapeutic candidates according to Route of Administration, with products classified under various ROAs including:

• Oral

• Intravenous

• Subcutaneous

• Parenteral

• Topical

Molecular Structure Classification

Autism Spectrum Disorder Products are organized according to various Molecule types encompassing:

• Recombinant fusion proteins

• Small molecule

• Monoclonal antibody

• Peptide

• Polymer

• Gene therapy

From emerging drug candidates to competitive intelligence, the Autism Spectrum Disorder Pipeline Report encompasses comprehensive insights – explore now at Autism Spectrum Disorder Market Drivers and Barriers, and Future Perspectives

Report Scope and Comprehensive Coverage

Geographic Scope: Global

Featured Autism Spectrum Disorder Companies: Astrogen, MapLight Therapeutics, Axial Therapeutics, Aardvark Therapeutics, Ajna BioSciences, Intra-Cellular Therapies, Finch Therapeutics Group, PharmAla Biotech, and additional organizations.

Autism Spectrum Disorder Therapies: ML-004 (IR)/(ER) tablet, Arbaclofen, Terpenes-Enriched CBD-Predominant Oil, Terpenes-Enriched CBD Oil (THC-Free), L1-79, Cannabidivarin, Cariprazine, and other investigational agents.

Assessment Framework Categories

Autism Spectrum Disorder Therapeutic Assessment by Product Type: Monotherapy, Combination therapy, Mono/Combination approaches

Autism Spectrum Disorder Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

1. Introduction

2. Executive Summary

3. Autism Spectrum Disorder: Overview

4. Pipeline Therapeutics

5. Therapeutic Assessment

6. Autism Spectrum Disorder– DelveInsight’s Analytical Perspective

7. Late Stage Products (Phase III)

8. AST-001: Astrogen

9. Mid Stage Products (Phase II)

10. ML004: MapLight Therapeutics

11. Early Stage Products (Phase I)

12. AJA001: Ajna BioSciences

13. Preclinical and Discovery Stage Products

14. Drug Name: Company Name

15. Inactive Products

16. Autism Spectrum Disorder Key Companies

17. Autism Spectrum Disorder Key Products

18. Autism Spectrum Disorder- Unmet Needs

19. Autism Spectrum Disorder- Market Drivers and Barriers

20. Autism Spectrum Disorder- Future Perspectives and Conclusion

21. Autism Spectrum Disorder Analyst Views

22. Autism Spectrum Disorder Key Companies

23. Appendix

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