Autistic Disorder - Pipeline Insight, 2024

This “Autistic Disorder- Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 25+ 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.

Geography Covered

• Global coverage

Autistic Disorder: Understanding

Autistic Disorder: Overview

Autistic disorder, also known as Autism Spectrum Disorder (ASD), encompasses a spectrum of neurodevelopmental disabilities. This spectrum is characterized by repetitive patterns of behavior, interests, activities, and problems in social interactions. ASD is a complicated neurodevelopmental disorder that is characterized by behavioral and psychological problems in children. These children become distressed when their surrounding environment changes because their adaptive capabilities are minimal. The symptoms are present from early childhood and affect daily functioning. Children with ASD have co-occurring language problems, intellectual disabilities, and epilepsy at higher rates than the general population.

ASD is a neurobiological disorder influenced by both genetic and environmental factors affecting the developing brain. Ongoing research continues to deepen our understanding of potential etiologic mechanisms in ASD, but currently no single unifying cause has been elucidated. Genetic factors play a role in ASD susceptibility, with siblings of patients with ASD carrying an increased risk of diagnosis when compared to population norms, and a much higher, although not absolute, concordance of autism diagnosis in monozygotic twins. Ultimately, research continues to reveal factors that correlate with ASD risk, but no causal determinations have been made. This leaves much room for discovery with investigators continuing to elucidate new variants conveying genetic risk, or new environmental correlates that require further study.

The diagnosis is conducted mainly by observational screening tools that measure a child’s social and cognitive abilities. The two main tools used in the diagnosis of ASD are DSM-5 and M-CHAT, which examine persistent deficits in interaction and social communication, and analyze responses to “yes/no” items that cover different developmental domains to formulate a diagnosis. The diagnostic features historically associated with ASD are a triad of impaired social interactions, verbal and nonverbal communication deficits, and restricted, repetitive behavior patterns. These core features are observed irrespective of race, ethnicity, culture, or socioeconomic status. However, ASD individuals tend to differ from one another, so one feature may be more prevalent than another. Despite recent advancements, there are currently no reliable biomarkers for ASD.

Treatment depends on severity and comorbidities, which can include behavioral training, pharmacological use, and dietary supplement. Behavior-oriented treatments include a series of programs that aim to re-condition target behaviors, and develop vocational, social, cognitive, and living skills. However, to date, no single or combination treatments have been able to reverse ASD completely. Medications have been most effective in treating the associated behavioral symptoms of autism, though studies have examined potential benefits in some of the core symptoms of autism with certain medications, especially the repetitive behaviors often seen with this diagnosis. Risperidone and aripiprazole are the medications FDA approved for symptoms associated with autism spectrum disorders, targeting the irritability often seen with this diagnosis. Children and adolescents with autism spectrum disorder appear to be more susceptible to adverse effects with medications; therefore, initiation with low doses and titrating very slowly is recommended. Some complementary alternative treatments have been researched as possible treatments in autism, though evidence supporting many of these is very limited.

Autistic Disorder- Pipeline Insight, 2024 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 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

Cariprazine: AbbVie Cariprazine is an atypical antipsychotic, FDA-approved for the treatment of schizophrenia and the acute treatment of manic or mixed episodes associated with bipolar I disorder. It is thought to be effective due to its partial agonist activity at the dopamine D2 receptor and serotonin 5HT1A receptor and its antagonism at the serotonin 5HT2A receptor. Additionally, Cariprazine has a high affinity for dopamine D3 receptors, acting as a partial agonist at D3 receptors. Cariprazine is primarily metabolized by CYP3A4 into Desmethyl Cariprazine (DCAR), and DCAR is then further metabolized by CYP3A4 into Didesmethyl Cariprazine (DDCAR). DCAR and DDCAR both display similar pharmacological potencies as Cariprazine. Currently, the drug is in the Phase III stage of its development for the treatment of AutisticDisorder.

L1-79: Yamo Pharmaceuticals L1-79 is a novel therapy that targets the core symptoms of autism. Previous experience with open-label administration of L1-79 in patients with autism demonstrated that L1-79 is a well-tolerated oral treatment that has the potential to improve the core symptoms of autism. In a recently completed Phase II study, multiple independent efficacy measures assessed using commonly accepted and validated psychometric tests demonstrated positive trends supporting improvements in the target core symptom domains affected by autism, especially social domains, despite a short treatment period and small number of patients. This data supported the granting of a Fast Track Designation by the Food and Drug Administration (FDA) in May of 2018.

AB-2004: Axial Therapeutics AB-2004 is Axial Therapeutic’s lead 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. We believe this mechanism may result in minimal side effects due to lack of exposure across other parts of the body, although this hypothesis needs to be verified in controlled clinical trials. Currently, AB-2004 is being studied in the Phase II stage of its development as a potential new treatment for 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. 20+ 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, AbbVie.

Phases

This report covers around 25+ 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 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

• Eli Lilly and Company
• Scioto Biosciences
• AbbVie
• Yamo Pharmaceuticals
• Axial Therapeutics
• Jazz Pharmaceuticals
• Hoffmann-La Roche

Key Products

• Tasimelteon
• Cariprazine
• L1-79
• JZP541
• AB-2004
• RO6953958
• SB-121

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