KP104 / Kira Pharma - LARVOL DELTA

KP104, a Bifunctional C5 Mab-Factor H Fusion Protein, Demonstrates Sustained Long-Term Efficacy and Safety in Complement Inhibitor-Naïve PNH Patients: Updated Results from a Phase 2 Study at 36/38 Weeks of Obd Treatment (ASH 2024) - P2 | "Conclusion : The long-term results from this Phase II study of 53-77 weeks of KP104 treatment, including ≥36/38 weeks at OBD, demonstrate sustained clinical benefits and a favorable safety profile, affirming the durable efficacy of KP104 in controlling both intravascular and extravascular hemolysis in complement inhibitor-naïve PNH patients. The data continue to provide compelling clinical evidence to support the development of KP104 as a novel first-line monotherapy option for PNH patients with desired efficacy and safety (Notaro & Luzzatto, NEJM 2022)."

Clinical • P2 data • Complement-mediated Rare Disorders • Dyslipidemia • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Influenza • Myeloproliferative Neoplasm • Novel Coronavirus Disease • Oncology • Pain • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Respiratory Diseases

Study to Evaluate Safety, Tolerability and Pharmacodynamics of KP104 in Participants With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Trial completion date: Oct 2024 ➔ Apr 2027 | Trial primary completion date: Oct 2024 ➔ Mar 2026

Trial completion date • Trial primary completion date • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 (clinicaltrials.gov) - P2 | N=35 | Active, not recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Recruiting ➔ Active, not recruiting

Enrollment closed • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of KP104 to Treat Glomerulonephritis (clinicaltrials.gov) - P2 | N=52 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Trial completion date: Sep 2025 ➔ Apr 2027 | Trial primary completion date: Sep 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date • Glomerulonephritis • IgA Nephropathy • Lupus Nephritis • Nephrology • Renal Disease

KP104, A BIFUNCTIONAL C5 MAB-FACTOR H FUSION PROTEIN, EFFECTIVELY CONTROLS INTRAVASCULAR AND EXTRAVASCULAR HEMOLYSIS IN COMPLEMENT INHIBITOR-NAÏVE PNH PATIENTS: LONG-TERM RESULTS FROM A PHASE 2 STUDY (EHA 2024) - P2 | "The long-term results of KP104 treatment in this Phase II study demonstrate consistent safety and improvedclinical response over those observed at week 24/25, affirming KP104's durable efficacy in controlling both IVHand EVH in complement inhibitor-naïve PNH patients. The data provide compelling clinical evidence thatKP104, a potent first-in-class bifunctional complement inhibitor, may represent a novel first-line monotherapyoption for PNH with desired efficacy and safety (Notaro, NEJM 2022)."

Clinical • P2 data • Complement-mediated Rare Disorders • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Influenza • Myeloproliferative Neoplasm • Novel Coronavirus Disease • Oncology • Pain • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Respiratory Diseases

Kira Pharmaceuticals to Present Positive Long-term Results of KP104 in Phase 2 Paroxysmal Nocturnal Hemoglobinuria (PNH) Study at the 2024 European Hematology Association (EHA) Hybrid Congress (PRNewswire) - P2 | N=35 | NCT05476887 | Sponsor: Kira Pharmacenticals (US), LLC. | "Kira Pharmaceuticals...today announced that it will present positive long-term safety and efficacy results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with PNH in an oral session at the 2024 European Hematology Association (EHA) Hybrid Congress, to be held in Madrid, Spain, from June 13-16, 2024....'We are actively preparing for global Phase 3 development and remain dedicated to bringing this innovative therapy to patients as quickly as possible'....Patients demonstrated continuous improvements in hemoglobin levels: 100% (18/18) of patients sustained an Hgb increase of ≥2 g/dL from baseline, with mean (SD) Hgb levels increasing by 7.0 (2.1) g/dL over baseline, and 88.9% (16/18) patients achieving Hgb normalization (≥12 g/dL)."

P2 data • Paroxysmal Nocturnal Hemoglobinuria

KP104, a Bifunctional C5 Antibody/Factor H Fusion Protein, Effectively Controls Both Intravascular and Extravascular Hemolysis: Interim Results from a Phase 2 Study in Complement Inhibitor-Naïve PNH Patients (ASH 2023) - P2 | "The findings provide compelling clinical evidence that a bifunctional inhibitor targeting the AP and TP can properly address the current treatment gaps associated with single complement pathway inhibitors, potentially eliminating the need for cumbersome and costly combination therapies (Notaro et al. N Engl J Med 2022; 387:160-6)."

Clinical • P2 data • Anemia • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Novel Coronavirus Disease • Pain • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

TRIAL IN PROGRESS: AN OPEN-LABEL, PHASE 2 STUDY TO EVALUATE EFFICACY, SAFETY, PK, PD OF KP104, A BIFUNCTIONAL COMPLEMENT INHIBITOR, IN IGAN AND C3G SUBJECTS (ISN-WCN 2023) - P2 | "Conclusions In summary, the effect of KP104 in IgAN and C3G subjects will be explored in this study. Data from this study will be used for future development of KP104."

Clinical • P2 data • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease • Vasculitis

Trial in Progress: An Open-Label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (ASH 2022) - P2 | "Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria...Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria...ASN abstract. Under review."

Clinical • P2 data • PK/PD data • Anemia • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 (clinicaltrials.gov) - P2 | N=35 | Recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Not yet recruiting ➔ Recruiting | Trial completion date: Sep 2024 ➔ Feb 2025 | Trial primary completion date: Mar 2024 ➔ Nov 2024

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

Study to Evaluate Safety, Tolerability and Pharmacodynamics of KP104 in Participants With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Trial completion date: Oct 2023 ➔ Oct 2024 | Initiation date: Nov 2022 ➔ Oct 2023 | Trial primary completion date: Oct 2023 ➔ Oct 2024

Trial completion date • Trial initiation date • Trial primary completion date • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of KP104 to Treat Glomerulonephritis (clinicaltrials.gov) - P2 | N=52 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC. | Initiation date: Dec 2022 ➔ Jul 2023

Trial initiation date • Glomerulonephritis • IgA Nephropathy • Lupus Nephritis • Nephrology • Renal Disease

To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 (clinicaltrials.gov) - P1 | N=80 | Completed | Sponsor: Kira Pharmacenticals (US), LLC. | Active, not recruiting ➔ Completed

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

SYNERGY-1: A Phase 1, First-in-Human, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics Study of KP104 in Escalating Single and Multiple Doses (KIDNEY WEEK 2022) - "The data supports planned future clinical trials in C-mediated GD. To our knowledge, KP104 is the first bifunctional biologic demonstrating successful inhibition of both AP and TP."

Clinical • P1 data • PK/PD data • Glomerulonephritis • IgA Nephropathy • Renal Disease

Study to Evaluate Safety, Tolerability and Pharmacodynamics of KP104 in Participants With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus (clinicaltrials.gov) - P2 | N=24 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC.

New P2 trial • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus

Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of KP104 to Treat Glomerulonephritis (clinicaltrials.gov) - P2 | N=52 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC.

New P2 trial • Glomerulonephritis • IgA Nephropathy • Lupus Nephritis • Nephrology • Renal Disease

To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 (clinicaltrials.gov) - P1 | N=80 | Active, not recruiting | Sponsor: Kira Pharmacenticals (US), LLC.

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

To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 (clinicaltrials.gov) - P2 | N=35 | Not yet recruiting | Sponsor: Kira Pharmacenticals (US), LLC.

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