This is 2-part study. The primary objective of Part 1 is to evaluate safety and tolerability of single subcutaneous (SC) doses of PATAS in healthy subjects. The secondary objective of this study is to determine the pharmacokinetics (PK) of single SC doses of PATAS in healthy subjects. The primary objectives of Part 2 are to evaluate the safety and tolerability of 4 weekly SC doses of PATAS in subjects with T2D; and to determine the PK and pharmacodynamics (PD) of 4 weekly SC doses of PATAS in subjects with T2D. Secondary objectives of Part 2 include evaluation of the potential effect of multiple SC doses of PATAS on markers of glycemic control, as measured by glucose levels, insulin levels, and other metabolomic biomarkers and characterization of of adverse event (AE) profiles of the various dose levels of PATAS.
Part 1: Single Ascending Dose Inclusion criteria
Healthy male and female subjects, 18 to 55 years of age, inclusive, at the time of signing the Informed Consent Form (ICF);
Willing and able to give written informed consent for participation in the study prior to the initiation of any Screening or study-specific procedures;
Body mass index (BMI) within the range of 20.0 to 35.0 kg/m2, inclusive, at Screening;
Clinically significant history of asthma, eczema, or any other allergic condition or previous severe hypersensitivity; Note: Non-active hay fever is not exclusionary.
Liver function tests (alanine aminotransferase [ALT], aspartate aminotransferase [AST], alkaline phosphatase [ALP], total bilirubin) outside the following upper limits of normal (ULNs) at Screening or at Check-In (Day -1): a. For ALT and AST, measurements > ULN; b. For ALP, measurements >ULN; or c. For total bilirubin, measurements > ULN.
Estimated glomerular filtration rate </= 90 mL/min/1.73 m2 based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation at Screening or at Check-In (Day -1);
Thyroid-stimulating hormone (TSH) outside of reference range (e.g., TSH <1 × lower limit of normal [LLN] or TSH >1 × ULN) at Screening; Note: Abnormal TSH results will reflex to a free thyroxine (T4) test.
Sequential
PATAS Trifluoroacetate
DrugA drug targeting the interaction between the ALMS1 protein and alpha-PKC
Placebo
DrugExcipient only formulation, without active compound
Phase 1B: Safety
Incidence and severity (using CTCAE version 5.0.) of treatment-emergent AEs and SAEs
OGTT
Oral glucose tolerance test
Cmax
Maximum observed plasma concentration
Tmax
Time to Cmax
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