An Open-label, Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 in Subjects With Thrombotic Microangiopathy Secondary to Systemic Lupus Erythematosus

This study will evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KP104 in participants with systemic lupus erythematosus (SLE)-Thrombotic microangiopathy (TMA). The study consists of 2 parts: Part 1 (Dose Optimization) and Part 2 (Proof of Concept). All participants will receive KP104 in combination with standard of care (SOC) for SLE-TMA.

Start Date01 Mar 2025

Sponsor / Collaborator

Kira Pharmacenticals (US), LLC.

NCT05517980

/ Not yet recruitingPhase 2

An Open-label, Phase 2 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of KP104 in Subjects With IgA Nephropathy (IgAN) and Complement 3 Glomerulopathy (C3G)

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in participants with IgAN and C3G. The study will start with enrolling the IgAN cohort. Approximately 42 participants with IgAN will be enrolled in 2 stages: Stage 1 will be used to collect safety, immunogenicity, PK, and PD data to select the optimal biologic dose (OBD) of KP104 for IgAN, as well as to preliminarily explore the effect of KP104 on C3G. Stage 2 will be used to collect safety, immunogenicity, PK, PD, and efficacy data at the OBD dose of KP104 for IgAN and C3G. As soon as the OBD for IgAN is determined, eligible participants with C3G will be enrolled and dosed at the OBD for IgAN for a minimum of 48 weeks for weekly maintenance dosing and a minimum of 47 weeks for biweekly maintenance dosing. Approximately 10 participants with C3G will be enrolled.

Start Date01 Mar 2025

Sponsor / Collaborator

Kira Pharmacenticals (US), LLC.

NCT05476887

/ Active, not recruitingPhase 2

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 (PNH)

The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH. The study will be conducted in 2 parts. Part 1 is a dose-selection study to assess escalating doses and varying dose intervals of KP104. Part 2 is a proof-of-concept (POC) study assessing the efficacy of the optimal intravenous (IV) loading dose followed by the optimal maintenance dose and regimen of KP104. Participants who complete the Initial Treatment Period and demonstrate benefit from KP104 will be eligible for a 9-month open-label extension (OLE) treatment period.

Start Date25 Nov 2022

Sponsor / Collaborator

Kira Pharmacenticals (US), LLC.

100 Clinical Results associated with KP-104

100 Translational Medicine associated with KP-104

100 Patents (Medical) associated with KP-104

News (Medical) associated with KP-104

19 Nov 2024

·www.prnewswire.com

Systemic Lupus Erythematosus Clinical Trial Pipeline Gains Momentum as 120+ Key Companies at the Forefront | DelveInsight

Systemic Lupus Erythematosus (SLE) is a chronic autoimmune disease characterized by inflammation and tissue damage across multiple organ systems, such as the skin, joints, kidneys, heart, and brain. The incidence of autoimmune diseases, including SLE, has been increasing globally, with a significant impact on demand for targeted and effective therapies. LAS VEGAS, Nov. 19, 2024 /PRNewswire/ -- DelveInsight's ' Systemic Lupus Erythematosus Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline systemic lupus erythematosus therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the systemic lupus erythematosus pipeline domain. Key Takeaways from the Systemic Lupus Erythematosus Pipeline Report DelveInsight's systemic lupus erythematosus pipeline report depicts a robust space with 120+ active players working to develop 140+ pipeline therapies for systemic lupus erythematosus treatment. Key systemic lupus erythematosus companies such as Roche, ImmuPharma, UCB, Idorsia Pharmaceuticals, Biogen, AbbVie, Bristol-Myers Squibb, Vera Therapeutics, Beijing Mabworks Biotech Co., Ltd., BeiGene, Jiangsu Renocell Biotech Company, AstraZeneca, Pfizer, Red de Terapia Celular, Kira Pharmacenticals (US), LLC., Jemincare, Galapagos NV, Alumis, argenx, Gilead Sciences, Novartis, Zenas Biopharma, Horizon Therapeutics, Provention Bio, Janssen Research & Development, Eli Lilly and Company, Medsenic, Sanofi, Merck KGaA, Resolve Therapeutics, ILTOO, Janssen, Alpine Immune Sciences, Kezar Life Sciences, Neovacs, Kangpu Biopharmaceuticals, Cartesian Therapeutics, Apellis Pharmaceuticals, Inc., Alexion Pharmaceuticals, Inc., Q32 Bio Inc., Conduit Pharmaceuticals, Miltenyi Biomedicine GmbH, Kyverna Therapeutics, ImmPACT Bio, Genrix (Shanghai) Biopharmaceutical Co., Ltd., Gracell Biotechnologies (Shanghai) Co., Ltd., Cabaletta Bio, Tenet Medicines, Ascentage Pharma Group Inc., InnoCare, Eisai, Sorrento Therapeutics, Carna Bioscience, Yake Biotechnology, Equillium, Daiichi Sankyo Company, SinoMab Bioscience Ltd, Citryll BV, Sareum, Shanghai Junshi Biosciences, Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Millennium Pharmaceuticals, Inc., Takeda, Synthekine, Hangzhou Sumgen Biotech Co., Ltd., Sana Biotechnology, Hoffmann-La Roche, Guangdong Ruishun Biotech Co., Ltd, Shanghai Ming Ju Biotechnology Co., Ltd., Autolus Limited, Nkarta, Inc., Luminary Therapeutics, Kyowa Kirin Co., Ltd., Shanghai Ming Ju Biotechnology, IGM Biosciences, Inc., GlaxoSmithKline, Fate Therapeutics, Cugene Inc., Century Therapeutics, Inc., Cullinan Therapeutics Inc., Juno Therapeutics, Inc., EdiGene Inc., Atara Biotherapeutics, Annexon, Inc., Adicet Therapeutics, Bioray Laboratories, PersonGen BioTherapeutics (Suzhou) Co., Ltd., Juventas Cell Therapy Ltd., Nanjing Bioheng Biotech Co., Ltd., Pregene ShenZhen Biotechnology, Artiva Bio, KeyMed Biosciences, IASO, JW Therapeutics, ROME Therapeutics, Ventus Therapeutics, Allosite Therapeutics, iCell Gene Therapeutics, Caribou Biosciences, LongBio Pharma, Amytrx Therapeutics, Corestem, Eliem Therapeutics, SinoMab Bioscience Ltd., and others are evaluating new systemic lupus erythematosus drugs to improve the treatment landscape. Promising systemic lupus erythematosus pipeline therapies such as Obinutuzumab, Lupuzor (IPP-201101), Dapirolizumab pegol, Cenerimod, BIIB059, Upadacitinib, Deucravacitinib, Atacicept, MIL62, Zanubrutinib, RY_SW01 cell injection|Basic treatment, Ravulizumab, Rapcabtagene autoleucel, PF-06823859, Mesenchymal stem cells (MSC), KP104, JMKX000189, Iptacopan, GLPG3667, ESK-001, Elsubrutinib, Efgartigimod alfa, Edecesertib, Ianalumab, Obexelimab (XmAb5871), Daxdilimab (VIB7734), PRV-3279, Nipocalimab, ABBV 599, NKTR-358 (LY3471851), Arscimed, SAR441344, Enpatoran, RSLV-132, Aldesleukin, Daratumumab, Branebrutinib, BMS-986256, ALPN-101, KZR-616, IFNα kinoid, KPG 818, Descartes-08, CFZ533, APL-2, ALXN2050, ADX-097, AZD1656, MB-CART19.1, KYV-101, IMPT-514, GR1603, GC012F Injection, CABA-201, Budoprutug, APG-2575, ICP-022, E6742, ALPN-303, AC0058, Mosunetuzumab, AS-0871, CD19/BCMA CAR T-cells, PF-06835375, EQ001 (Itolizumab), DS-7011a, SM03, CIT-013, SDC-1801, UBP1213, TQB3702, TAK-079, TAK-007, SYNCAR-001, SG301, SC291, RO7507062, RJMty19 (CD19-CAR-DNT cells), Relma-cel, PIT565, Obecabtagene autoleucel (obe-cel), NKX019, LMY-920, KK4277, JWCAR201, Imvotamab, GSK4527363, GSK4347859, FT819, CUG252, CNTY-101, CLN-978, CC-97540, Belantamab, ATHENA CAR-T ATA3219, ANX009, ADI-001, BRL-301, T-cell injection targeting CD19 chimeric antigen receptor, CNCT19, RD06-04, PRG-2311, PRG-1801, AlloNK, CM313, CT103A, JWCAR 029, Research program: endogenous reverse transcriptase inhibitors, VENT 03, ONT01, BCMA-CD19 cCAR T cells, CB-010, LP-005, AMTX 100, CE211AT15, TN-119, SN1011, QX002N, RSLV-145, Recombinant human plasma gelsolin, LN-008, TST 008, IBL 100s, and others are under different phases of systemic lupus erythematosus clinical trials. In October 2024, the FDA approved the investigational new drug (IND) application for Cullinan Therapeutics' CLN-978 to treat systemic lupus erythematosus (SLE). In September 2024, Cartesian Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). In September 2024, Caribou Biosciences announced that the U.S. Food and Drug Administration (FDA) had granted fast-track designation to CAR T-cell therapy CB-010 for systemic lupus erythematosus (SLE). In September 2024, UCB and Biogen reported successful results from their Phase III clinical trial of the PHOENYCS GO study, which tested dapirolizumab pegol as a treatment for moderate-to-severe systemic lupus erythematosus. In August 2024, Conduit Pharmaceuticals announced they will be conducting a Phase IIa clinical trial to evaluate AZD1656 for the treatment of multiple autoimmune diseases, including systemic lupus erythematosus (SLE). In July 2024, the first individual received the therapy Descartes-08 to treat systemic lupus erythematosus. This therapy has been previously administered to patients with myasthenia gravis, a long-term autoimmune disorder characterized by muscle weakness. In February 2024, Idorsia Pharmaceuticals formed a major global research and development partnership with Viatris to advance and commercialize two Phase III assets, selatogrel and cenerimod, on a global scale. Request a sample and discover the recent advances in systemic lupus erythematosus treatment drugs @ Systemic Lupus Erythematosus Pipeline Report The systemic lupus erythematosus pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage systemic lupus erythematosus drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the systemic lupus erythematosus clinical trial landscape. Systemic Lupus Erythematosus Overview Systemic lupus erythematosus (SLE) is a chronic autoimmune disease in which the immune system mistakenly attacks healthy tissues in various parts of the body. The exact cause of SLE is unknown, but it is believed to result from a combination of genetic, environmental, and hormonal factors. Common triggers include infections, sunlight exposure, and certain medications. Women, particularly of childbearing age, are more commonly affected. Symptoms of SLE vary widely and can affect many organ systems, leading to a wide range of clinical presentations. Common symptoms include fatigue, joint pain, skin rashes, photosensitivity, and fever. More severe cases can affect the kidneys, heart, lungs, and central nervous system, potentially leading to life-threatening complications. Diagnosis of SLE is based on a combination of clinical features and laboratory tests. The antinuclear antibody (ANA) test is a common initial screening tool, but additional tests like anti-double stranded DNA (anti-dsDNA) and anti-Smith antibodies, as well as assessments of kidney and liver function, are often necessary. Diagnosis can be challenging because SLE symptoms often mimic those of other diseases. Treatment for SLE focuses on managing symptoms and preventing flares. Mild cases may be treated with nonsteroidal anti-inflammatory drugs (NSAIDs) and antimalarial drugs like hydroxychloroquine. More severe cases may require corticosteroids, immunosuppressive drugs such as azathioprine, methotrexate, or newer biologics like belimumab. Lifestyle modifications, such as avoiding excessive sun exposure and managing stress, are also essential in controlling the disease. Find out more about systemic lupus erythematosus treatment drugs @ Drugs for Systemic Lupus Erythematosus Treatment A snapshot of the Systemic Lupus Erythematosus Pipeline Drugs mentioned in the report: Learn more about the emerging systemic lupus erythematosus pipeline therapies @ Systemic Lupus Erythematosus Clinical Trials Systemic Lupus Erythematosus Therapeutics Assessment The systemic lupus erythematosus pipeline report proffers an integral view of the systemic lupus erythematosus emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Systemic Lupus Erythematosus Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: Antibody-dependent cell cytotoxicity, Cell death inhibitors, Cell death stimulants, Immunomodulators, CD40 ligand inhibitors, Sphingosine 1 phosphate receptor modulators, Calcineurin inhibitors, Immunosuppressants, Janus kinase 1 inhibitors, TYK2 kinase inhibitors, B cell activating factor inhibitors, Tumour necrosis factor ligand superfamily member 13 inhibitors Key Systemic Lupus Erythematosus Companies: Roche, ImmuPharma, UCB, Idorsia Pharmaceuticals, Biogen, AbbVie, Bristol-Myers Squibb, Vera Therapeutics, Beijing Mabworks Biotech Co., Ltd., BeiGene, Jiangsu Renocell Biotech Company, AstraZeneca, Pfizer, Red de Terapia Celular, Kira Pharmacenticals (US), LLC., Jemincare, Galapagos NV, Alumis, argenx, Gilead Sciences, Novartis, Zenas Biopharma, Horizon Therapeutics, Provention Bio, Janssen Research & Development, Eli Lilly and Company, Medsenic, Sanofi, Merck KGaA, Resolve Therapeutics, ILTOO, Janssen, Alpine Immune Sciences, Kezar Life Sciences, Neovacs, Kangpu Biopharmaceuticals, Cartesian Therapeutics, Apellis Pharmaceuticals, Inc., Alexion Pharmaceuticals, Inc., Q32 Bio Inc., Conduit Pharmaceuticals, Miltenyi Biomedicine GmbH, Kyverna Therapeutics, ImmPACT Bio, Genrix (Shanghai) Biopharmaceutical Co., Ltd., Gracell Biotechnologies (Shanghai) Co., Ltd., Cabaletta Bio, Tenet Medicines, Ascentage Pharma Group Inc., InnoCare, Eisai, Sorrento Therapeutics, Carna Bioscience, Yake Biotechnology, Equillium, Daiichi Sankyo Company, SinoMab Bioscience Ltd, Citryll BV, Sareum, Shanghai Junshi Biosciences, Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Millennium Pharmaceuticals, Inc., Takeda, Synthekine, Hangzhou Sumgen Biotech Co., Ltd., Sana Biotechnology, Hoffmann-La Roche, Guangdong Ruishun Biotech Co., Ltd, Shanghai Ming Ju Biotechnology Co., Ltd., Autolus Limited, Nkarta, Inc., Luminary Therapeutics, Kyowa Kirin Co., Ltd., Shanghai Ming Ju Biotechnology, IGM Biosciences, Inc., GlaxoSmithKline, Fate Therapeutics, Cugene Inc., Century Therapeutics, Inc., Cullinan Therapeutics Inc., Juno Therapeutics, Inc., EdiGene Inc., Atara Biotherapeutics, Annexon, Inc., Adicet Therapeutics, Bioray Laboratories, PersonGen BioTherapeutics (Suzhou) Co., Ltd., Juventas Cell Therapy Ltd., Nanjing Bioheng Biotech Co., Ltd., Pregene ShenZhen Biotechnology, Artiva Bio, KeyMed Biosciences, IASO, JW Therapeutics, ROME Therapeutics, Ventus Therapeutics, Allosite Therapeutics, iCell Gene Therapeutics, Caribou Biosciences, LongBio Pharma, Amytrx Therapeutics, Corestem, Eliem Therapeutics, SinoMab Bioscience Ltd., and others. Key Systemic Lupus Erythematosus Pipeline Therapies: Obinutuzumab, Lupuzor (IPP-201101), Dapirolizumab pegol, Cenerimod, BIIB059, Upadacitinib, Deucravacitinib, Atacicept, MIL62, Zanubrutinib, RY_SW01 cell injection|Basic treatment, Ravulizumab, Rapcabtagene autoleucel, PF-06823859, Mesenchymal stem cells (MSC), KP104, JMKX000189, Iptacopan, GLPG3667|Placebo, ESK-001, Elsubrutinib, Efgartigimod alfa, Edecesertib, Ianalumab, Obexelimab (XmAb5871), Daxdilimab (VIB7734), PRV-3279, Nipocalimab, ABBV 599, NKTR-358 (LY3471851), Arscimed, SAR441344, Enpatoran, RSLV-132, Aldesleukin, Daratumumab, Branebrutinib, BMS-986256, ALPN-101, KZR-616, IFNα kinoid, KPG 818, Descartes-08, CFZ533, APL-2, ALXN2050, ADX-097, AZD1656, MB-CART19.1, KYV-101, IMPT-514, GR1603, GC012F Injection, CABA-201, Budoprutug, APG-2575, ICP-022, E6742, ALPN-303, AC0058, Mosunetuzumab, AS-0871, CD19/BCMA CAR T-cells, PF-06835375, EQ001 (Itolizumab), DS-7011a, SM03, CIT-013, SDC-1801, UBP1213, TQB3702, TAK-079, TAK-007, SYNCAR-001, SG301, SC291, RO7507062, RJMty19 (CD19-CAR-DNT cells), Relma-cel, PIT565, Obecabtagene autoleucel (obe-cel), NKX019, LMY-920, KK4277, JWCAR201, Imvotamab, GSK4527363, GSK4347859, FT819, CUG252, CNTY-101, CLN-978, CC-97540, Belantamab, ATHENA CAR-T ATA3219, ANX009, ADI-001, BRL-301, T-cell injection targeting CD19 chimeric antigen receptor, CNCT19, RD06-04, PRG-2311, PRG-1801, AlloNK, CM313, CT103A, JWCAR 029, Research program: endogenous reverse transcriptase inhibitors, VENT 03, ONT01, BCMA-CD19 cCAR T cells, CB-010, LP-005, AMTX 100, CE211AT15, TN-119, SN1011, QX002N, RSLV-145, Recombinant human plasma gelsolin, LN-008, TST 008, IBL 100s, and others. Dive deep into rich insights for new drugs for systemic lupus erythematosus treatment, visit @ Systemic Lupus Erythematosus Drugs Table of Contents For further information on the systemic lupus erythematosus pipeline therapeutics, reach out @ Systemic Lupus Erythematosus Treatment Drugs Related Reports Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key SLE companies including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Epidemiology Systemic Lupus Erythematosus Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical, and forecasted SLE epidemiology in the 7MM. Lupus Nephritis Market Lupus Nephritis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key lupus nephritis companies, including NOVARTIS, MORPHOSYS, ASTRAZENECA, ROCHE, KEZAR LIFE SCIENCES, ALEXION PHARMACEUTICALS, NOVARTIS, CABALETTA BIO, among others. Lupus Nephritis Pipeline Lupus Nephritis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key lupus nephritis companies, including Inflection Biosciences, Equillium, Roche, Horizon Therapeutics, BeiGene, Janssen Research & Development, ImmPACT Bio, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Phase 2Fast TrackCell Therapy

17 Jun 2024

·www.biospace.com

Kira Pharmaceuticals Presents Positive Results of KP104 Phase 2 Paroxysmal Nocturnal Hemoglobinuria (PNH) Study at European Hematology Association (EHA) Congress 2024 Demonstrating Long-term Safety and Efficacy

CAMBRIDGE, Mass., June 17, 2024 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, today announced positive long-term results from its Phase 2 study of KP104 in complement inhibitor-naïve patients with PNH. KP104 a first-in-class dual-targeting complement inhibitor (C5 & Factor H) of both alternative and terminal pathways. The findings were presented at the 2024 European Hematology Association (EHA) Hybrid Congress in Madrid, Spain, highlighting KP104's transformative potential as a novel monotherapy for PNH to address the existing unmet medical needs. Key highlights from the study include results from 18 patients treated with KP104 subcutaneously for up to 65 weeks, with 24- 26 weeks of the treatment time being on Optimal Biological Dose (OBD) for all patients, switched from 3 cohorts (n=6 per cohort) in dose escalation phase. Patients continued to show sustained improvements in hemoglobin levels after switching to OBD (Figure 1): 100% (18/18) achieved a ≥2 g/dL increase from baseline, and 89% (16/18) attained hemoglobin normalization (≥12 g/dL) with an average (SD) of 13.5 (1.4) g/dL in the absence of RBC transfusions. The remaining two patients with co-existing conditions, one with aplastic anemia (AA) and the other with myeloproliferative neoplasms (MPN), also demonstrated hemoglobin improvement approaching near-normal levels. Patients showed sustained control of LDH levels (Figure 2) to near-normal level throughout the entire treatment period, demonstrating strong IVH control. At the end of 24/26 weeks post-OBD switch, 94% (17/18) patients achieved LDH <1.5x ULN. All patients remained free from RBC transfusions between day 1 and week 65 of KP104 treatment. Significant clinical improvements were also observed in all other secondary endpoints: normalization of absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores after switching to OBD. KP104 was safe and well-tolerated, and produced no treatment-emergent adverse events (TEAEs) at or above grade 3. The affirming long-term results from the Phase 2 study further demonstrate KP104's potential as an optimal first-line monotherapy to safely and effectively control both intravascular and extravascular hemolysis of PNH. Global Phase 3 studies are being planned to potentially establish KP104 as the new standard of care for PNH. "We are very encouraged by the robust efficacy and favorable safety pro by KP104 in our Phase 2 study," said Dr. Wenru Song, Head of R&D at Kira Pharmaceuticals, "these long-term results support the advancement KP104 into Phase 3 trials. We are committed and look forward to bringing this promising therapy to patients suffering from PNH as quickly as possible." Oral Presentation Details: Title: 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 Authors: Bing Han, Fengkui Zhang, Li Zhang, Chen Yang, Changhe Yue, Chunrong Wang, Jay Ma, Chaomei He, Ping Tsui, Jingtao Wu, Qing Yu Christina Weng, Richard Lee, Helen Fu, Hui Yan, Wenru Song Session: s454 Clinical and Translational in Bone Marrow Failure and PNH Time: June 15, 2024, 16:30 - 17:45 CEST The presentation slides will be available on Kira's website at . About KP104 KP104 is a first-in-class bifunctional biologic designed to simultaneously block both the alternative (Factor H) and terminal (C5) complement pathways, providing a powerful and synergistic method of targeting the validated drivers of complement-mediated disease. This dual-target mechanism of action uniquely positions KP104 to address complement-mediated diseases and potentially provide greater benefits than single-target complement agents. Engineered to have an extended half-life and enhanced potency, KP104 has a formulation suitable for both intravenous and subcutaneous administrations. KP104 is entering Phase 2 POC trials across multiple renal disease and hematologic indications and has been granted Orphan Drug Designation by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria. Phase 2 trials will be conducted globally, including in the U.S., China, and Australia. KP104 is an investigational agent not yet approved for any indication by any health authority. About Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disease that is characterized by the destruction of red blood cells, formation of blood clots, and impairment of bone marrow function. PNH affects between 1 and 5 people per million and is almost always caused by a genetic mutation that results in production of aberrant hematopoietic stem cells. These stem cells produce irregular red blood cells that are highly susceptible to destruction via complement activation. Current therapies include C5 inhibitors, which do not address extravascular hemolysis (EVH) related to the alternative pathway or a C3 inhibitor, which may address EVH but may not adequately block C5 downstream, leading to life-threatening breakthrough hemolysis (Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition, N Engl J Med, July 14, 2022). About Kira Pharmaceuticals Kira Pharmaceuticals is a clinical-stage biotechnology company pioneering complement- targeted therapies to treat immune-mediated diseases. Enabled by its LOGIC platform, the company has developed a robust pipeline of novel assets against validated complement targets. Headquartered in Cambridge, Massachusetts and with facilities in China and Australia, Kira Pharmaceuticals has established a global team committed to advancing life-changing therapies to patients around the world. More information on Kira can be found at and on LinkedIn. CONTACT: Kiramedia@kirapharma.com

Clinical ResultPhase 2Orphan Drug

16 May 2024

·www.prnewswire.com

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

CAMBRIDGE, Mass., May 16, 2024 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, 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 excited to share these promising Phase 2 results of KP104 at the upcoming EHA 2024 Hybrid Congress, which represents a significant advancement in addressing the unmet medical needs of patients with PNH," said Dr. Wenru Song, Head of R&D at Kira Pharmaceuticals. "The encouraging long-term safety and efficacy data demonstrate the potential of KP104 as an optimal and safe first-line monotherapy for PNH patients. We are actively preparing for global Phase 3 development and remain dedicated to bringing this innovative therapy to patients as quickly as possible." Key findings from 18 patients who received 33-58 weeks of treatment of KP104, of which at least 16 weeks were under the optimal biological dose (OBD) regimen. 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). Patients showed sustained control of LDH levels: LDH <1.5xULN was achieved by 94.4% (17/18) patients, with a mean (SD) % LDH reduction of 84.3 (8.7) % from baseline. All patients remained free from RBC transfusion between Day 1 and Week 33-58 of KP104 treatment. Sustained clinical improvements in all other secondary endpoints: normalization of absolute reticulocyte counts, bilirubin levels, and FACIT-fatigue scores were continuously observed across all three cohorts KP104 was safe and well-tolerated without treatment-emergent adverse events (TEAEs) at or above Grade 3. Convenient dosing regimen with SC administration every 2 weeks. Details of the oral presentation are as follows: Title: 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 Authors: Bing Han, Fengkui Zhang, Li Zhang, Chen Yang, Changhe Yue, Chunrong Wang, Jay Ma, Chaomei He, Ping Tsui, Jingtao Wu, Qing Yu Christina Weng, Richard Lee, Helen Fu, Hui Yan, Wenru Song Session Name : s454 Clinical and Translational in Bone Marrow Failure and PNH Oral Presentation Date and Time : June 15, 2024, 16:30 - 17:45 CEST Additional information about the EHA 2024 and the abstract is available at: About KP104 KP104 is a first-in-class bifunctional biologic designed to simultaneously block both the alternative and terminal complement pathways, providing a powerful and synergistic method of targeting validated drivers of complement-mediated disease. This dual-target mechanism of action uniquely positions KP104 to address complement-mediated diseases and potentially provide greater benefits than single-target complement agents. Engineered to have an extended half-life and enhanced potency, KP104 has a formulation suitable for both intravenous and subcutaneous administrations. KP104 is entering Phase 2 POC trials across multiple renal disease and hematologic indications and has been granted Orphan Drug Designation by the FDA for the treatment of paroxysmal nocturnal hemoglobinuria. Phase 2 trials will be conducted globally, including in the U.S., China, and Australia. KP104 is an investigational agent not yet approved for any indication by any health authority. About Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disease that is characterized by the destruction of red blood cells, formation of blood clots, and impairment of bone marrow function. PNH affects between 1 and 5 people per million and is almost always caused by a genetic mutation that results in production of aberrant hematopoietic stem cells. These stem cells produce irregular red blood cells that are highly susceptible to destruction via complement activation. Current therapies include C5 inhibitors, which do not address extravascular hemolysis (EVH) related to the alternative pathway or a C3 inhibitor, which may address EVH but may not adequately block C5 downstream, leading to life-threatening breakthrough hemolysis (Breakthrough Hemolysis in PNH with Proximal or Terminal Complement Inhibition, N Engl J Med, July 14, 2022). About Kira Pharmaceuticals Kira Pharmaceuticals is a clinical-stage biotechnology company pioneering complement- targeted therapies to treat immune-mediated diseases. Enabled by its LOGIC platform, the company has developed a robust pipeline of novel assets against validated complement targets. Headquartered in Cambridge, Massachusetts and with facilities in China and Australia, Kira Pharmaceuticals has established a global team committed to advancing life-changing therapies to patients around the world. More information on Kira can be found at and on LinkedIn. CONTACT: [email protected] SOURCE Kira Pharmaceuticals

Clinical ResultPhase 2Orphan Drug

100 Deals associated with KP-104

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
C3 glomerulopathy	Phase 2	China	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Glomerulonephritis, IGA	Phase 2	China	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Hemoglobinuria, Paroxysmal	Phase 2	China	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Hemoglobinuria, Paroxysmal	Phase 2	United States	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Hemoglobinuria, Paroxysmal	Phase 2	South Korea	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Hemoglobinuria, Paroxysmal	Phase 2	Australia	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022
Thrombotic Microangiopathies	Phase 2	China	Kira Pharmaceuticals (Hong Kong) Ltd.Startup	29 Jul 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05476887 (ASH2024) Manual	Phase 2	Hemoglobinuria, Paroxysmal	18	KP104 at OBD	(mtwndbpepw) = oslutsbdxa hnilzrikgh (ppygrkjscl, 2.0) View more	Positive	08 Dec 2024
NCT05476887 (PRNewswire) Manual	Phase 2	Hemoglobinuria, Paroxysmal	18	KP104	(hmdkqefwmc) = azbirzpbgv htoohhlyju (qsuebbpvcl ) View more	Positive	16 May 2024
NCT05476887 (EHA 12024 \| EHA 22024) Manual	Phase 2	Hemoglobinuria, Paroxysmal	18	KP104	(rvjpvggdsx) = hnavxmnbuw fpbcpolpgy (onlehpcuge, 2.1) View more	Positive	14 May 2024
NCT05476887 (572 KP104, ASH2023)	Phase 2	Hemoglobinuria, Paroxysmal	15	KP104	(jxshoyovwr) = ehqejfrqby gmpmfbgysh (caokejuoqt, ±1.7) View more	-	10 Dec 2023
NCT05490017 (SYNERGY-1, ASN2022) Manual	Phase 1	Glomerular disease	56	KP-104 (SAD)	(qmfhzrclvl) = xebbpkmhpo mpbxtdhfkt (kcwbprtmqr )	Positive	05 Nov 2022
NCT05490017 (SYNERGY-1, ASN2022) Manual	Phase 1	Glomerular disease	56	KP-104 (MAD)	(qmfhzrclvl) = bnfcwezkgj mpbxtdhfkt (kcwbprtmqr )	Positive	05 Nov 2022

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