Yishining (lanoracopan) / Createrna - LARVOL DELTA

Efficacy and Safety of Lanoracopan in the Treatment of PNH (clinicaltrials.gov) - P=N/A | N=90 | Not yet recruiting | Sponsor: Peking Union Medical College Hospital

New trial • Real-world evidence • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MY008211A, AN ORAL COMPLEMENT FACTOR B INHIBITOR, IN SINGLE- AND MULTIPLE-ASCENDING-DOSE STUDIES IN HEALTHY PARTICIPANTS (EHA 2026) - P1 | "Steady-state concentration was achieved within 1 week of dosing, and AP activity was rapidly and dose-dependently decreased, with sustained suppression observed with BID regimens. These data support continued clinical development of MY008211A for the treatment of PNH."

Clinical • First-in-human • PK/PD data • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

ORAL MY008211A DEMONSTRATES SUPERIOR EFFICACY VERSUS ECULIZUMAB IN COMPLEMENT INHIBITOR–NAÏVE CHINESE PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) (EHA 2026) - P3 | "Oral MY008211A resulted in significant improvements in Hb and superior reduction in reticulocyte counts compared with eculizumab while maintaining a favorable safety profile. These results support MY008211A as a potentially practice-changing oral therapy for patients with PNH."

Clinical • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Respiratory Diseases • CFB

Significant Proteinuria Reduction with the Oral Factor B Inhibitor MY008211A in IgA Nephropathy: 12-Week Interim Results from a Phase 2 Trial (ERA 2026) - "Conclusion Conclusion MY008211A achieved substantial and clinically meaningful reductions in proteinuria with a favorable safety profile. These findings support Factor B inhibition as a promising therapeutic strategy in IgAN and indicate the potential to achieve clinically relevant treatment targets, warranting confirmation in Phase 3 trials."

P2 data • Glomerulonephritis • IgA Nephropathy • Renal Disease

ORAL MY008211A MONOTHERAPY ACHIEVES TRANSFUSION AVOIDANCE AND HEMOGLOBIN NORMALIZATION IN CHINESE PNH PATIENTS WITH RESIDUAL ANEMIA DESPITE C5 INHIBITION (EHA 2026) - P3 | "Notably, 95.0% of patients achieved continuous transfusion avoidance, with 70% reaching complete hemoglobin normalization, accompanied by remarkable improvements in fatigue-related quality of life. The favorable safety profile and convenient twice-daily oral dosing support MY008211A as a promising alternative to address the unmet need of persistent anemia in patients with PNH who have inadequate response to C5 inhibition."

Clinical • Monotherapy • Anemia • Complement-mediated Rare Disorders • Dyslipidemia • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

A Pharmacokinetic and Safety Study of MY008211A Tablets in Subjects With Impaired or Normal Hepatic Function (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P1 trial • Hepatitis C • Liver Failure

A Phase 2 Dose-Finding Trial of MY008211A in Complement Inhibitor–Naive Patients with Paroxysmal Nocturnal Hemoglobinuria and Signs of Active Hemolysis (ASH 2024) - "Improvements in secondary EPs, including fatigue, were of similar magnitude across dosage groups. This, together with the evidenced safety profile, supports the continued development of MY008211A."

Clinical • P2 data • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Rare Diseases • Respiratory Diseases • Thrombosis • CFB

Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=40 | Not yet recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd | Trial completion date: Dec 2025 ➔ Dec 2027 | Trial primary completion date: Jul 2025 ➔ Jul 2027

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=34 | Completed | Sponsor: Wuhan Createrna Science and Technology Co., Ltd | Recruiting ➔ Completed | Trial completion date: Jul 2024 ➔ Nov 2024

Trial completion • Trial completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - P2/3 | N=120 | Recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P2/3 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (clinicaltrials.gov) - P3 | N=20 | Recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P3 trial • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P3 | N=66 | Recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P3 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study to Evaluate the Efficacy and Safety of MY008211A in Subjects with Primary Immunoglobulin a Nephropathy (IgAN) (clinicaltrials.gov) - P2 | N=72 | Not yet recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P2 trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects (clinicaltrials.gov) - P1 | N=16 | Not yet recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P1 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=40 | Not yet recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

Trial completion date • Trial initiation date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=40 | Not yet recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P2 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) (clinicaltrials.gov) - P2 | N=40 | Recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P2 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of Multiple Ascending Doses MY008211A in Healthy Adults (clinicaltrials.gov) - P1 | N=40 | Completed | Sponsor: Wuhan Createrna Science and Technology Co., Ltd | Active, not recruiting ➔ Completed | N=30 ➔ 40

Enrollment change • Trial completion • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of Single-dose MY008211A in Healthy Adults (clinicaltrials.gov) - P1 | N=63 | Completed | Sponsor: Wuhan Createrna Science and Technology Co., Ltd | Active, not recruiting ➔ Completed | N=46 ➔ 63

Enrollment change • Trial completion • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of Multiple Ascending Doses MY008211A in Healthy Adults (clinicaltrials.gov) - P1 | N=30 | Active, not recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P1 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P1 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study of Single-dose MY008211A in Healthy Adults (clinicaltrials.gov) - P1 | N=46 | Active, not recruiting | Sponsor: Wuhan Createrna Science and Technology Co., Ltd

New P1 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases