The goal of this clinical trial is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential efficacy of IM-101 in adult participants with AChR antibody-positive gMG. Subsequently, the safety and efficacy of the selected IM-101 dose-regimen will be tested in participants with AChR antibody-negative gMG and participants with AChR antibody-positive or AChR antibody-negative oMG.
Able and willing to provide signed informed consent
Willingness to consent to screening for genetic muscular diseases
Male or female aged ≥ 18 years and < 75 years
Diagnosed with MG
Previous exposure to IM-101
Anti-MuSK antibody Positive
History of malignant thymoma, or history of cancer within the past 5 years of screening
History of N. meningitidis infection
Parallel
IM-101 Part A
DrugParticipants will receive IM-101 intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose and Day 29.
Placebo Part A
DrugParticipants will receive Placebo intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29.
IM-101 Part B
DrugParticipants will receive IM-101 intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29, D57 and D85.
Placebo Part B
DrugParticipants will receive Placebo intravenously (IV), at a loading dose on Day 1 and Day 15 followed by maintenance dose on Day 29, D57 and D85.
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Treatment groupsSee each treatment group below.
[Part A] Incidence of TEAEs, SAEs, AEs that led to premature discontinuation, and AESIs across 3 ascending dose regimens (each with 3 administrations) in participants with AChR antibody-positive gMG
An AE is any untoward medical occurrence in a clinical study participant administered with a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product and is medically important. Treatment-emergent AEs associated with a. Grade \> 2 hypersensitivity or IRR, b. Infection c. Any potential kidney failure and d. Any potential Hy's Law case (\> 3 × ULN of either ALT/AST with concurrent \> 2 × ULN of total bilirubin and lack of alternate etiology) will be considered AESIs.
[Part B] Incidence of TEAEs, SAEs, AEs that led to premature discontinuation, and AESIs of IM-101, compared with placebo, in participants with AChR antibody-positive gMG, AChR antibody-negative gMG, and oMG
An AE is any untoward medical occurrence in a clinical study participant administered with a pharmaceutical (investigational or non investigational) product. An AE does not necessarily have a causal relationship with the intervention. An SAE is any untoward medical occurrence that at any dose results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a suspected transmission of any infectious agent via a medicinal product and is medically important. Treatment-emergent AEs associated with a. Grade \> 2 hypersensitivity or IRR, b. Infection c. Any potential kidney failure and d. Any potential Hy's Law case (\> 3 × ULN of either ALT/AST with concurrent \> 2 × ULN of total bilirubin and lack of alternate etiology) will be considered AESIs.
[Part B] Change from baseline to Week 16 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score for gMG cohorts
Change from baseline in MG-ADL total score over 16 Weeks will be reported. The MG-ADL provides a rapid assessment of the participant's MG symptom severity. Eight functions (talking, chewing, swallowing, breathing, impairment of ability to brush teeth or comb hair, impairment of ability to arise from a chair, double vision, eyelid droop) are rated on a 4-point scale: 0 (no impairment) to 3 (severe impairment). The total score will be sum of eight function scores and can range from 0 to 24. A higher score indicates greater symptom severity.
[Part B]Change from baseline to Week 16 in Myasthenia Gravis Impairment Index (MGII) ocular score for oMG cohorts
Change from baseline in MGII ocular score over 16 Weeks will be reported. The MGII is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.
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