Autistic Disorder- Pipeline Insight, 2025

This “Autistic Disorder - Pipeline Insight, 2025” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in Autistic Disorder pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Autistic Disorder: Understanding

Autistic Disorder: Overview

Autism Spectrum Disorder (ASD) encompasses a range of neurodevelopmental disorders characterized by repetitive behaviors, restricted interests, and challenges in social interactions. It is a complex condition that manifests in early childhood and significantly impacts daily functioning. Children with ASD often experience distress when changes occur in their environment due to limited adaptability. In addition to behavioral symptoms, ASD is frequently associated with co-occurring issues such as language impairments, intellectual disabilities, and epilepsy. Childhood Disintegrative Disorder (CDD), also known as disintegrative psychosis or Heller syndrome, is a rare and severe form of ASD, marked by a significant regression in social, language, and motor skills after a period of normal development, typically after the age of three. Though the exact cause of CDD is unknown, it often presents with rapid skill loss that may lead to confusion or distress for the affected child. This disorder can be devastating for both the individual and their family, as it disrupts previously acquired developmental milestones.

The exact cause of ASD remains unknown, and its onset is often insidious, with no clearly defined trigger. While children with ASD have a higher risk of developing epilepsy, no direct causal relationship has been established. Childhood Disintegrative Disorder (CDD), a subtype of ASD, has been associated with several diseases, especially when it manifests with a late onset. These include subacute sclerosing panencephalitis, a chronic brain infection caused by a form of the measles virus leading to brain inflammation and nerve cell death; tuberous sclerosis, a genetic disorder that causes benign tumors in the brain and other organs; leukodystrophy, a condition involving the degeneration of white matter in the brain due to improper myelin sheath development; and lipid storage diseases, which involve the toxic accumulation of fats in the brain and nervous system.

The pathophysiology of Autism Spectrum Disorder (ASD) remains unclear, though various subtypes of the disorder may have distinct underlying mechanisms. For example, Childhood Disintegrative Disorder (CDD) occurs in children who initially achieve normal developmental milestones, but experience a rapid regression of skills, typically around the fourth year of life, though onset can vary. This sudden loss of skills has led some to consider CDD a form of childhood dementia, with the possibility that amyloid deposition in the brain may play a role in the disease’s progression. However, this hypothesis remains unproven and requires further research to establish a definitive link.

The approach for treatment is behavior-based and highly structured, aiming to help children re-learn language, self-care, and social skills. Family counseling is essential, ensuring that parents are equipped to support their child’s treatment at home. Various therapies, including speech, occupational, social skills development, and sensory integration, are tailored to each child's needs. As children with CDD experience profound skill regression, they often require long-term care. Treatment includes behavior therapy, environmental therapy, and medications. Behavior therapy often uses Applied Behavioral Analysis (ABA) with a reward system to reinforce positive behaviors and reduce problematic ones. Environmental therapy enhances sensory experiences to manage sensory processing difficulties. Medications are prescribed to manage symptoms, such as antipsychotics for repetitive behaviors and aggression, while SSRIs, stimulants, and other antipsychotics help with behavioral control. If seizures occur, anticonvulsants are used. However, there is a risk of neuroleptic malignant syndrome with neuroleptic medications, and no direct cure for CDD exists.

'Autistic Disorder - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Autistic Disorder pipeline landscape is provided which includes the disease overview and Autistic Disorder treatment guidelines. The assessment part of the report embraces, in depth Autistic Disorder commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Autistic Disorder collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Autistic Disorder R&D. The therapies under development are focused on novel approaches to treat/improve Autistic Disorder.

Autistic Disorder Emerging Drugs Chapters

This segment of the Autistic Disorder report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Autistic Disorder Emerging Drugs

AST-001: Astrogen, Inc.

AST-001 is an innovative therapeutic agent developed by Astrogen, Inc. for the treatment of Autism Spectrum Disorder (ASD). This formulation is based on L-serine, a naturally occurring amino acid that plays a crucial role in various metabolic processes, including neurotransmitter synthesis. AST-001 aims to address the core symptoms of ASD, which include deficits in social communication and interaction, as well as restricted and repetitive behaviors. By enhancing the availability of L-serine in the brain, AST-001 is thought to improve cognitive function and social skills in pediatric patients with ASD. The unique mechanism of action of this compound focuses on modulating dopaminergic pathways, which are often implicated in the behavioral challenges associated with autism. Currently, the drug is in Phase III stage of its development for the treatment of Autistic Disorder.

L1-79: Yamo Pharmaceuticals LLC

L1-79 is an innovative treatment designed to address core social-interaction deficits in individuals with Autism Spectrum Disorder (ASD). L1-79 has shown a favorable safety profile, being well-tolerated among both healthy volunteers and patients with ASD. Its efficacy is grounded in a unique mechanism of action, supported by extensive research in autism. This includes the ability to modulate catecholamine pathways, which influence critical brain functions such as mood, attention, motor skills, and social behavior. Additionally, L1-79 addresses the hyperactive CNS state often observed in autism by engaging systems that regulate brain function and gut mechanisms implicated in some forms of the disorder. By modulating catecholaminergic signaling, L1-79 offers a promising approach to improving social communication and reducing social impairments in those with ASD. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Autistic Disorder.

AB-2004: Axial Therapeutics, Inc.

AB-2004 is an investigational therapy that targets the microbiome gut-brain axis and its role in co-occurring conditions associated with Autism Spectrum Disorder (ASD). AB-2004 is a gut-targeted, molecular therapeutic, which means it is designed to work in the gut only and does not enter other bodily tissues. The active ingredient in AB-2004 is a highly engineered form of spherical carbon designed with human safety and biological selectivity in mind, making it very different from activated charcoal. Each sphere of AB-2004 consists of a network of pores that allows it to selectively adsorb metabolites that may contribute to characteristics associated with ASD like irritability and anxiety. AB-2004 is a tasteless and odorless powder formulation that can be mixed with soft food which makes taking the medication very easy. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Autistic Disorder.

Autistic Disorder: Therapeutic Assessment

This segment of the report provides insights about the different Autistic Disorder drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Autistic Disorder

• There are approx. 18+ key companies which are developing the therapies for Autistic Disorder. The companies which have their Autistic Disorder drug candidates in the most advanced stage, i.e. Phase III include, Astrogen, Inc.

Phases

The report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Autistic Disorder pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Autistic Disorder: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Autistic Disorder therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Autistic Disorder drugs.

Autistic Disorder Report Insights

• Autistic Disorder Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Autistic Disorder Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Autistic Disorder drugs?
• How many Autistic Disorder drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Autistic Disorder?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Autistic Disorder therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Autistic Disorder and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Astrogen, Inc.

Yamo Pharmaceuticals LLC

• Axial Therapeutics, Inc.

NeuroNOS

• Shanghai Auzone Biological Technology Co., Ltd.

Vanda Pharmaceuticals

• Paxmedica
• Stalicla SA
• ACADIA Pharmaceuticals Inc.

MapLight Therapeutics

• Intra-Cellular Therapies, Inc.

Aardvark Therapeutics, Inc.

Astrogen, Inc.

IAMA Therapeutics S.r.l.

GEXVal

Key Products

• AST-001
• L1-79
• AB-2004
• BA102
• TTYP01
• Tasimelteon
• Suramin Sodium
• Pimavanserin
• ML-004
• Lumateperone
• ARD-501
• Cannabidivarin
• AST-001
• AB-2004
• GXV 001

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