A First in Human Study of ORT247 in Healthy Volunteers

Exclusion Criteria

• •Contraindication to undergo LP including international normalized ratio (INR) \>1.4 or other coagulopathy, platelet cell count of \<120,000/μL, infection at the desired LP site, current use of anti-coagulant medication except for low dose aspirin, degenerative arthritis, spinal scoliosis, back surgery, suspected increased intracranial pressure on history or neurologic exam, non-communicating hydrocephalus or intracranial mass, or prior history of spinal mass or trauma
• •Any significant acute or chronic medical illness
• •Any history of cancer within 5 years of enrollment with the exception of resected skin basal cell carcinoma
• •Any major surgery within 4 weeks of study drug administration
• •Donation of blood or serum \>500 mL to a blood bank or in a clinical study (except screening visit) within 3 months of study drug administration
• •Inability to undergo venipuncture or tolerate venous access
• •Has smoked or used tobacco products within 3 months before study drug administration
• •Positive drug screen for alcohol, drugs of abuse, or tobacco
• •Recent (within 6 months of study drug administration) drug or alcohol abuse as defined in Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-V), Diagnostic Criteria for Drug and Alcohol Abuse
• •Evidence of any clinically significant neurological or psychiatric disorder that could interfere with study assessments as determined by investigator and sponsor
• •History of or currently has schizophrenia, schizoaffective disorder or bipolar disorder, untreated major depression (DSM-V or International Statistical Classification of Diseases and Related Health Problems, 10th edition \[ICD-10\] criteria)
• •Significant illness or infection requiring intervention within the prior 30 days as determined by investigator and sponsor (must test negative for active coronavirus disease 2019 \[COVID-19\])
• •Indication of potential suicidality risk
• •Any of the following abnormalities at screening: serum creatinine \> upper limit of normal (ULN), hepatic transaminases (aspartate aminotransferase or alanine aminotransferase) \> ULN, abnormal blood pressure based on the clinical judgment of the investigator, QTcF \>470 msec
• •Known history of hypersensitivity to any component of the ORT247 drug product or placebo
• •Currently taking, or planning to take, any medication (prescription or over-the-counter) that would potentially affect the assessment of pharmacokinetics (PK), pharmacodynamics (PD), or immunogenicity of ORT247.