Over 25 drugs being developed in Autistic Disorder pipeline landscape

Autism Spectrum Disorder puzzle illustrationAutistic 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.

Treatment depends on severity and comorbidities, which can include behavioral training, pharmacological use, and dietary supplement. 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 Emerging Drugs

Cariprazine: 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. Currently, the drug is in the Phase III stage of its development for the treatment of Autistic Disorder.

L1-79: 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: 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. 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.

Excerpted from Autistic Disorder – Pipeline Insight, 2024 at www.ResearchAndMarkets.com; Used by permission of www.Globenewswire.com

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