Efficacy and Safety of HN2301 in Patients With Generalized Myasthenia Gravis (MG)

An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis

This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.

This study consists of a screening period of up to 4 weeks, a treatment period, and a follow-up period of 1 year.

Inclusion Criteria:

• Age: 18-80 years, no gender restriction;
• Confirmed diagnosis of generalized myasthenia gravis (MG) with positive AchR or MuSK antibodies, meeting at least one of the following conditions#(1) Repetitive nerve stimulation suggesting neuromuscular transmission defect; (2) Positive response to neostigmine test; (3) Clinically judged improvement of --MG symptoms after oral cholinesterase inhibitor therapy;
• Clinical classification of MG according to MGFA types IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa, IVb);
• Baseline MG-ADL score ≥6, ocular-related score <50%;
• Poor response and/or lack of efficacy under standard therapies;
• Minimum life expectancy > 12 weeks;
• Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function.

Exclusion Criteria:

• Subjects positive for hepatitis B surface antigen (HBsAg) and/or hepatitis B core antibody (HBcAb) with detectable or quantifiable HBV DNA, positive for hepatitis C antibody (HCV Ab) with detectable or quantifiable HCV RNA, positive for HIV antibody, positive CMV DNA, or CMV DNA above the lower limit of detection; positive for syphilis antigen or antibody;
• Presence of other uncontrolled active infections;
• History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow/hematopoietic stem cell transplantation;
• Pregnant or breastfeeding women;
• Receipt of any mRNA-LNP products or other LNP-based drugs within the past two years;
• History of any of the following cardiovascular conditions within 6 months prior to screening: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant cardiac disease;
• History of ≥Grade 2 bleeding events within 30 days prior to screening, or requiring long-term continuous anticoagulation therapy (e.g., warfarin, low molecular weight heparin, Xa factor inhibitors);
• History of live vaccination within 30 days prior to screening;
• Severe central nervous system diseases or pathological changes, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, seizures/convulsions, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disorders, organic brain syndromes, or psychosis;
• History of asthma or severe allergies;
• Patients combined with other malignant tumors;
• Any condition that, in the investigator's opinion, may increase the patient's risk or interfere with study assessments.