Gliknik GL0719-01- A Phase 1, Double-blind, Placebo-controlled, Single Ascending Intravenous and Subcutaneous Injection Dose, Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and an Open-label Evaluation of Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects with Cold Agglutinin Disease

GL-0719 has been developed to bind to a protein that helps our innate (non-specific) immune system fight infection and remove damaged cells. The protein (C1q) is part of a group of proteins called the complement system. These proteins are normally useful but in some people they don’t work properly and can cause damage. GL-0719 is a recombinant human Fc fusion protein which binds complement C1q causing reductions in C2 (another protein in the complement system), which thereby inhibits the complement Classical and Lectin pathways (important protein cascades which are key to our innate immune system), leaving the Alternative pathway intact. GL-0719 has the potential to help people with conditions caused by abnormalities of the complement system. This first in human study will assess the safety, tolerability, pharmacokinetics (what the body does to the drug) and pharmacodynamics (what the drug does to the body) following single and multiple intravenous doses in healthy adults. The study will be in 2 parts, a single ascending dose (SAD) and a multiple ascending dose (MAD) part. The SAD study will have 6 groups, the first group consisting of 4 participants and groups 2-6 consisting of 8 participants. Participants will receive a single intravenous dose of GL-0719/placebo and will reside at the study site from Day -1 to Day 3 with outpatient visits to Day 31. The MAD portion of the study will take part in 2 groups of 8 participants. All participants will receive 2 doses of intravenous GL-0719/placebo on Days 1 and 8. Participants will be resident in the CRU from Day -1 until Day 3, will return for outpatient visits on Days 4 and 5, will be resident from Day 7 until Day 10 and will return for outpatient visits to Day 38. An optional subcutaneous cohort with 8 participants may also be enrolled.