A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension

Inclusion Criteria:

• Subject meets the DSM-5 criteria for ASD for an autism diagnosis and is confirmedusing ADOS-2 criteria
• SRS-2, proxy version, total t-score >=66 at screening
• A full scale IQ score >=70 on the WASI®-II
• Subject has an appropriate study partner, in the opinion of the investigator
• For women of childbearing potential: agreement to remain abstinent or use acontraceptive method with a failure rate of <1% per year during the treatment periodand for at least 28 days after the last dose of study drug
• Treatment with permitted medications (at a stable dose for 12 weeks before screening)and behavioral therapy regimens (regimens stable for 6 weeks before screening), withthe intent that such treatments remain stable throughout the study and with noexpected changes before the Week 24 visit

Exclusion Criteria:

• Pregnancy or breastfeeding, or intention to become pregnant during the study
• Previous initiation of new or major change in psychosocial intervention within 6 weeksprior to screening
• Unstable or uncontrolled clinically significant affective or psychotic disordersand/or neurologic disorder that may interfere with the assessment of safety orefficacy endpoints
• Substance use disorders during the last 12 months
• Significant risk for suicidal behavior, in the opinion of the investigator
• Epilepsy or seizure disorder considered not well controlled within the past 6 monthsor changes in anticonvulsive therapy within the last 6 months
• Clinical diagnosis of peripheral neuropathy
• Within the last 2 years, unstable or clinically significant cardiovascular disease
• Uncontrolled hypertension
• Unexplained syncopal episode within the last 12 months
• Confirmed elevation above upper limit of normal of CK-MB, high sensitivity cardiactroponin T, cardiac troponin I, and/or N-terminal pro B-type natriuretic peptide
• Positive serology results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) 1 or 2
• History of coagulopathies, bleeding disorders, blood dyscrasias, hematologicalmalignancies, myelosuppression (including iatrogenic), or current major bleeding event
• Concomitant disease or condition that could interfere with, or treatment of whichmight interfere with, the conduct of the study, or what would, in the opinion of theinvestigator, pose an unacceptable risk to the subject in this study
• Confirmed clinically significant abnormality in parameters of hematology
• Confirmed clinically significant abnormality in parameters of clinical chemistry,coagulation, or urinalysis
• Medical history of malignancy, if not considered cured