New PNH drug VSA012 enters early human testing

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion and Exclusion Criteria for Groups 1-4 * Participants voluntarily participate in this clinical study, and voluntarily sign the ICF; * BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age; * Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%; * Presence of one or more of PNH-related signs or symptoms within 3 months prior to screening; * Hb \< 100 g/L; * LDH value \> 1.5 × ULN; * One of the following criteria for prior drug therapy for PNH must be met: 1. Having never received any complement inhibitor therapy; 2. Having received C5, C3, or CFB complement inhibitors and having discontinued the complement inhibitor for more than 5 half-lives or 3 months prior to screening; * Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing. Inclusion and Exclusion Criteria for Groups 5 * Participants voluntarily participate in this clinical study, and voluntarily sign the ICF; * BMI ≥ 18.0 kg/m2; male or female; 18 to 75 years of age; * Confirmed diagnosis of PNH by clinical manifestation and flow cytometry; granulocyte clone size ≥ 10%; * Participants who have been on a stable dose and interval of a C5 complement inhibitor (approved locally) for at least 3 months prior to the first dose of VSA012; * Within the 3 months prior to screening, have a documented Hb level of \< 105 g/L while on C5 complement inhibitor therapy; * Hb \< 105 g/L; * Participants are willing to receive meningococcal vaccine and pneumococcal vaccine at least 14 days prior to dosing. Exclusion Criteria for Groups 1-4 * History of hypersensitivity to VSA012 or its excipients; * Use of any complement inhibitors within 3 months prior to screening * Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening; * Supportive care for PNH does not meet the stable-dose requirements: * Participants have infections; * Laboratory tests meet the following criteria: 1. Reticulocyte count \< 100 × 109/L; 2. Platelet count \< 30 × 109/L; 3. Neutrophil count \< 0.5 × 109/L; 4. Creatinine clearance \< 30 mL/min (calculated by the Cockcroft-Gault formula); Exclusion Criteria for Groups 5 * History of hypersensitivity to VSA012 or its excipients; * Use of any targeted small interfering RNA (siRNA) within 18 months prior to screening, or any antisense oligonucleotide molecule within 6 months prior to screening; * Supportive care for PNH does not meet the stable-dose requirements: * Participants have infections; * History of splenectomy; * Previous suspected/confirmed hereditary complement deficiency; * History of recurrent invasive infections with capsular bacteria, e.g., meningococcus or pneumococcus; * Laboratory tests meet the following criteria: 1. Reticulocyte count \< 100 × 109/L; 2. Platelet count \< 30 × 109/L; 3. Neutrophil count \< 0.5 × 109/L; 4. Creatinine clearance \< 30 mL/min (calculated by the Cockcroft-Gault formula);