ciprocopan (NXP100) / Haisco, Nuvectis Pharma - LARVOL DELTA

Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria. (PubMed, Chin Med J (Engl)) - No abstract available

Journal • Monotherapy • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Nuvectis Expands Pipeline with Haisco Licensing Agreement for Two Clinical-Stage Drug Candidates (NVCT) (Yahoo Finance) - "Under the agreement, Nuvectis will acquire ex-China rights to NXP100, a Complement Factor B inhibitor, and NXP200, a next-generation BRAF inhibitor...The company said the addition of the two candidates advances its transition into a late-stage clinical development business while diversifying its therapeutic focus beyond oncology alone...As part of the transaction, Haisco will receive upfront and near-term payments of up to $40 million....The agreement also includes the potential for up to $1.421 billion in development, regulatory and commercial milestone payments, in addition to tiered royalties on future product sales."

Licensing / partnership • Colorectal Cancer • IgA Nephropathy • Lupus Nephritis • Melanoma • Non Small Cell Lung Cancer • Paroxysmal Nocturnal Hemoglobinuria

EFFICACY AND SAFETY OF SWITCHING TO ORAL HSK39297 MONOTHERAPY IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA PATIENTS WITH PERSISTENT ANEMIA DESPITE ANTI-C5 THERAPY: A PHASE 3 STUDY (HSK39297-302) (EHA 2026) - P3 | "Thirty-six adult PNH patients (hemoglobin [Hb] < 100 g/L) who had received stable anti-C5 therapy (eculizumab/crovalimab) for at least 6 months were switched to oral HSK39297 200 mg once daily for 24 weeks. Summary/Conclusion Switching to oral HSK39297 monotherapy resulted in significant and sustained hemoglobin improvement, near-complete elimination of transfusion need, effective control of hemolysis (including resolution of C3-mediated extravascular hemolysis), and reduced fatigue in PNH patients with persistent anemia on anti-C5 therapy, with a favorable safety profile. HSK39297 represents a promising once- daily oral treatment option for this patient population."

Clinical • Monotherapy • P3 data • Anemia • Complement-mediated Rare Disorders • Dyslipidemia • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Efficacy and safety of HSK39297, a once-daily oral CFB inhibitor, in patients with primary IgA nephropathy: a double-blind phase II trial (ERA 2026) - P2 | "These findings suggest that HSK39297 may reduce the risk of IgAN progression. The results strengthen the therapeutic rationale for selective CFB inhibitors and further support the evaluation of HSK39297 in Phase III clinical trials for the treatment of IgAN."

Clinical • P2 data • Dyslipidemia • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Lupus Nephritis • Renal Disease • Respiratory Diseases • CFB

A PHASE III STUDY OF A HEAD-TO-HEAD COMPARISON OF A FACTOR B INHIBITOR HSK39297, VERSUS ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA NAIVE TO COMPLEMENT INHIBITION (EHA 2026) - P3 | "Drug-related TEAEs occurring in >5% of patients in the HSK39297 group included headache (18.9%), lipid disorders (10.8%), and bacterial infections (5.4%). Summary/Conclusion HSK39297 (200 mg once daily) demonstrated significantly superior efficacy compared with eculizumab in the treatment of patients with PNH, along with a favorable safety profile."

Clinical • Head-to-Head • P3 data • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Dyslipidemia • Hematological Disorders • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis • CFB

A Study to Evaluate the Long-Term Safety and Efficacy of HSK39297 Tablets in Primary IgA Nephropathy (clinicaltrials.gov) - P2 | N=73 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

New P2 trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

Phase II Study of Efficacy and Safety of HSK39297 Tablet in Treatment of Patients With Primary IgAN (clinicaltrials.gov) - P2 | N=87 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd. | Recruiting ➔ Completed

Trial completion • Glomerulonephritis • IgA Nephropathy • Renal Disease

EFFICACY AND SAFETY OF HSK39297, A ONCE-DAILY ORAL CFB INHIBITOR, IN PATIENTS WITH PRIMARY IMMUNOGLOBULIN A NEPHROPATHY: A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE II TRIAL (ISN-WCN 2026) - P2 | "These findings suggest that HSK39297 may reduce the risk of IgAN progression. The results strengthen the therapeutic rationale for selective CFB inhibitors and further support the evaluation of HSK39297 in Phase III clinical trials for the treatment of IgAN."

Clinical • P2 data • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Lupus Nephritis • Renal Disease • Respiratory Diseases • CFB

Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia (clinicaltrials.gov) - P3 | N=36 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd. | Recruiting ➔ Completed

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

Phase Ⅲ Study of Efficacy and Safety of HSK39297 Tablet in Treatment of Patients With Primary IgAN (clinicaltrials.gov) - P3 | N=370 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

New P3 trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

Study to Assess Efficacy and Safety of HSK39297 Tablets in Patients With LN (clinicaltrials.gov) - P2 | N=105 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

New P2 trial • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

HSK39297-301: A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P3 | N=73 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd. | Recruiting ➔ Completed

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

Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH) (ASH 2025) - P2 | "Despite anti-C5 therapy (e.g.,eculizumab), many patients remain anemic due to residual intravascular hemolysis and C3-mediatedextravascular hemolysis (EVH). The 200 mgQD cohort showed sufficient exposure to sustained complement inhibition at the trough.ConclusionsHSK39297 monotherapy significantly improved hematologic and clinical outcomes in complementinhibitor- naïve PNH patients, demonstrating robust efficacy across all doses and a favorable safetyprofile. The 200 mg QD regimen demonstrated optimal efficacy and pharmacokinetics and istherefore proposed for further investigation in phase 3 trials."

Clinical • Monotherapy • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A phase II clinical study to evaluate the long-term safety and efficacy of HSK39297 tablets for the treatment of primary IgA nephropathy (ChiCTR) - P2 | N=65 | Not yet recruiting | Sponsor: Peking University First Hospital; Peking University First Hospital

New P2 trial • Glomerulonephritis • IgA Nephropathy • Renal Disease

Hiasco: Innovative drug HSK39297 tablets included in breakthrough therapy program [Google translation] (Sina Corp)

Breakthrough therapy • IgA Nephropathy

Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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

Announcement from Hiseco Pharmaceuticals Group Co., Ltd. on the Acceptance Notice of the IND Application for the New Indication of the Innovative Drug HSK39297 Tablets [Google translation] (Sina Corp)

New trial • Age-related Macular Degeneration

Efficacy and Safety of HSK392975 in Anti-C5 Treated PNH Patients With Anemia (clinicaltrials.gov) - P3 | N=32 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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

Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of HSK39297 in Chinese Healthy Subjects: A Phase 1 Clinical Trial. (PubMed, Clin Transl Sci) - "PD analysis indicated that oral administration of HSK39297 tablets resulted in dose-dependent inhibition of the AP activity and Bb concentration. These findings support advancing HSK39297 into later-phase trials for complement-mediated glomerular diseases."

Clinical • Journal • P1 data • PK/PD data • Renal Disease • CFB

HSK39297-201: A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH） (clinicaltrials.gov) - P2 | N=47 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd. | Recruiting ➔ Completed

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

Haisco plans to raise no more than 1.365 billion yuan through a private placement to invest in new drug research and development projects and replenish cash flow [Google translation] (STCN) - "On the evening of February 27, Haisco (002653) announced its plan to issue A-shares to specific objects in 2025. The company plans to issue no more than 70 million shares to specific objects, and the total amount of funds raised will not exceed 1.365 billion yuan....According to the plan, the research and development of innovative drugs raised by Haisco this time include HSK31679 (non-alcoholic fatty hepatitis), HSK31858 (respiratory diseases), propofol (general anesthesia), HSK39297 (paroxysmal nocturnal hemoglobinuria, primary IgA nephropathy), HSK21542 (postoperative pain, postoperative nausea and vomiting) and HL231 (respiratory diseases) and other drugs for subsequent domestic and overseas clinical research and market registration."

Financing • Anesthesia • Metabolic Dysfunction-Associated Steatohepatitis • Paroxysmal Nocturnal Hemoglobinuria • Respiratory Diseases

Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects (clinicaltrials.gov) - P1 | N=7 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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

A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P3 | N=66 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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

HSK39297-101: A Phase I Study to Assess the Safety,Tolerability, PK, PD, and Food Effect of HSK39297 in Healthy Subjects (clinicaltrials.gov) - P1 | N=96 | Completed | Sponsor: Haisco Pharmaceutical Group Co., Ltd. | Recruiting ➔ Completed

Trial completion

Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - P2 | N=47 | Recruiting | Sponsor: Haisco Pharmaceutical Group Co., Ltd.

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