Kira Pharmaceuticals (Suzhou) Co., Ltd.

CAMBRIDGE, Mass., Dec. 20, 2022 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, announced today that the Chinese National Medical Products Administration (NMPA) and the Australian Therapeutic Goods Administration (TGA) have approved initiation of Phase 2 studies for evaluation of KP104, a first-in-class bifunctional biologic that selectively targets the alternative and terminal complement pathways, in a renal basket study including IgA nephropathy (IgAN) and complement 3 glomerulopathy (C3G). "These clearances add to the multiple INDs Kira has secured this year for KP104 and mark our first in IgAN and C3G, serious immune-mediated conditions that cause kidney damage and often result in kidney failure," said Frederick Beddingfield, M.D., Ph.D., CEO of Kira. "We believe that KP104's ability to simultaneously and synergistically block two key complement targets makes it a unique therapeutic option with the potential to make a profound impact on the lives of patients living with these kidney diseases around the world." IgAN is an autoimmune disease that damages the kidneys, impacting organ function and often resulting in end-stage kidney disease. Though the exact pathogenesis of IgAN remains unknown, immune complex deposition in the kidneys is characteristic of the disease, causing inflammation and glomerular damage. Recent studies have implicated complement activation across multiple complement pathways as a major contributor to kidney injury and disease progression in IgAN. Drugs that selectively inhibit either the alternative or terminal pathway have shown partial efficacy in recent human studies. KP104 is a potent inhibitor of both the alternative and terminal complement pathways. Thus KP104 may have the unique potential to address IgAN more effectively, where multiple pathways are involved. In C3G, a hyperactivated alternative complement pathway causes excessive cleavage and activation of complement protein 3 (C3) and complement protein 5 (C5), resulting in harmful C3 fragments getting lodged in the kidney and C5 split products contributing to further glomerular inflammation and damage. There are currently no approved therapies for the condition, making KP104 a novel therapeutic option with the potential for transformative patient impact. The Phase 2 studies will evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of KP104 in participants with IgAN and C3G in China and Australia. Phase 1 data from the SYNERGY-1 first-in-human (FIH) study of KP104 demonstrated clinical proof-of-mechanism (PoM) for the biologic, which received Orphan Drug Designation for treatment of paroxysmal nocturnal hemoglobinuria (PNH) from the US Food and Drug Administration (FDA) earlier this year. KP104 is currently undergoing Phase 2 evaluation in PNH and will additionally be evaluated in other hematology and nephrology indications in upcoming clinical trials. About IgA Nephropathy IgA Nephropathy (IgAN) is an autoimmune disease that damages the kidneys, impacting organ function and often resulting in end-stage kidney disease. IgAN is the most common form of glomerulonephritis, with a global population incidence of roughly 2.5 per 100,000 per year, though the exact pathogenesis of the disease remains unknown. In IgAN, the body produces abnormal immunoglobulin A (IgA) antibodies that stimulate an immune response and result in formation of immune complexes that become trapped in the kidney. The immune complexes in the kidney cause inflammation and tissue damage, potentially leading to renal failure. There remains significant unmet need for therapeutics that effectively address IgAN, with current treatment approaches for serious cases largely reliant on corticosteroids to manage inflammation. Recent studies have implicated aberrant complement activity as a major contributor to glomerular inflammation and disease progression of IgAN. KP104 is a potent inhibitor of the alternative and terminal complement pathways that offers unique potential to address IgAN through selective modulation of complement activity. About Complement 3 Glomerulopathy Complement 3 Glomerulopathy (C3G) encompasses dense deposit disease (DDD) and C3 glomerulonephritis (C3GN), two conditions that result in inflammation and damage of the glomeruli in the kidney. In C3G, abnormal complement system activation results in breakdown of C3, a complement protein. The products of this breakdown become lodged in the kidney, triggering inflammation and immune activity that injure the glomeruli. C3 breakdown also leads to C5 and terminal complement activation, causing further glomerular inflammation and damage. C3G has a global prevalence of 2-3 people out of every million, and roughly half of those living with C3G develop end-stage renal disease within 10 years of diagnosis. There are currently no approved therapies for the condition, making KP104 a novel therapeutic option with potential for transformative patient impact. About KP104 KP104 is a first-in-class bifunctional biologic designed to simultaneously and selectively 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 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 (PNH). 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 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. SOURCE Kira Pharmaceuticals

CAMBRIDGE, Mass. and SUZHOU, China, Aug. 26, 2022 /PRNewswire/ -- Kira Pharmaceuticals, a global biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, will present preclinical data supporting its bifunctional approach to complement inhibition at the 2022 European Meeting on Complement in Human Disease (EMCHD) Conference. The research, conducted in collaboration with the Perelman School of Medicine at the University of Pennsylvania, further supports the clinical advancement of Kira's lead asset, KP104, a first-in-class biologic with a unique dual-approach mechanism of action. Designed to selectively block the alternative and terminal pathways, KP104 provides a powerful and synergistic method of targeting validated drivers of disease in the complement system. Taking place August 26-29, the conference will be held in Bern, Switzerland. Presentation abstracts are now available for viewing at the conference website. An intricate constellation of protein pathways, the complement system is a key component of innate immunity. Aberrant activity within this system can be a driver of diseases such as autoimmune and inflammatory conditions. Due to the complexity of complement biology, there remains a significant unmet medical need for next-generation and anti-complement drugs with better efficacy and convenience of administration than current therapies. The research presented at EMCHD provides evidence that using a bifunctional approach to target alternative and terminal pathways of the complement system could result in therapies with greater efficacy, longer lasting effects, and improved safety. "Based on intellectual property licensed from and developed at the University of Pennsylvania, Kira's leading product KP104 is designed to overcome the inherent challenges of complement drug discovery and in preclinical models, exhibits superior efficacy and longer-lasting inhibition than current complement-focused treatment options," said Kira President & Head of R&D and Co-founder Wenru Song, M.D., Ph.D. "We are highly encouraged that these data underscore the potential of KP104 in treating a range of debilitating diseases and we are looking forward to initiating Phase 2 trials." Kira has completed a Phase 1 first-in-human (FIH) study evaluating KP104, in which clinical proof-of-mechanism (POM) was achieved, and is initiating Phase 2 proof-of-concept (POC) trials across multiple indications. The company plans to present safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) data from the Phase 1 study at a medical conference later this year. Details for the oral presentation are as follows: Title: Pharmacokinetics/pharmacodynamics of KP104, a bi-functional antibody fusion protein inhibitor of complement, in C5/FcRn-humanized transgenic mice and Cynomolgus monkeys Authors: Jay Ma1, Xiang Gao1, Takashi Miwa2, Damodar Gullipalli2, Sayaka Sato2, Wen-Chao Song2, Xihua Zhu3, Jianjun Zhang3, Chaomei He1, Helen Fu1, Richard Lee1, Frederick Beddingfield1,4, Wenru Song1, Ping Tsui*,1 Abstract Number: 126 Session Title: Scientific Session IV: Therapeutics Session Date and Time: August 28, 2022, 9:15 – 9:30 CEST Presentation Summary and Background: The data demonstrate an optimal pharmacological profile for KP104 in human C5 transgenic mice and non-human primates (NHPs), supporting its further development as a potent bifunctional complement inhibitor suitable for both intravenous and subcutaneous administration. 1Kira Pharmaceuticals, Cambridge MA, 2Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, 3Kira Pharmaceuticals, Suzhou China, 4 Department of Medicine, Division of Dermatology, David Geffen School of Medicine at UCLA *Correspondence authors Details for the poster presentations are as follows: Title : Design and characterization of KP104, a bi-functional anti-C5 mAb and FH1-5 fusion protein that synergistically inhibits alternative and terminal pathways of complement activation Authors: Takashi Miwa1, Damodar Gullipalli1, Sayaka Sato1, Madhu Golla1, Xihua Zhu2, Jianjun Zhang2, Dongqiong Fei2, Ping Tsui3, Fengkui Zhang4, Wen-Chao Song1 Poster Number: 132 Session Title: Poster Viewing Session II Session Date and Time: August 28, 11:45 – 13:30 CEST Poster Summary and Background: This poster describes the design and initial characterization of KP104, a novel biologic engineered to optimize PK/PD properties to address unmet medical needs with currently approved complement drugs. Pre-clinical study results show KP104 to be a potent bifunctional complement inhibitor of the alternative and terminal pathways, possessing tissue-targeting property for cells with C5b-9 deposition. In addition, the data demonstrate a synergistic effect between the two moieties in KP104, namely the anti-C5 mAb and FH SCR 1-5. 1Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 2Kira Pharmaceuticals, Suzhou, China, 3Kira Pharmaceuticals, Cambridge, MA, 4Institute of Hematology, Chinese Academy of Medical Sciences, Tianjin, China Title: Therapeutic efficacy of a bi-functional anti-C5 mAb/FH1-5 fusion protein in a mouse model of rapidly progressing lethal C3 glomerulopathy Authors: Sayaka Sato1, Takashi Miwa1, Damodar Gullipalli1, Lin Zhou1, Jianjun Zhang2, Xiaoxia Hu2, Bingbing Jiang2, Ping Tsui3, Wen-Chao Song1 Poster Number: 174 Session Title: Poster Viewing Session II Session Date and Time: August 28, 11:45 – 13:30 CEST Poster Summary and Background: C3 glomerulopathy (C3G) is rare kidney disease caused by dysregulated activity of the complement system's alternative pathway for which no approved therapy exists. Data suggests that a murine surrogate of KP104, a bifunctional anti-C5 mAb/FH1-5 fusion protein entering phase 2 clinical trials, is more efficacious than a murine anti-C5 mAb in treating established disease in a lethal mouse model of C3G. 1Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 2Kira Pharmaceuticals, Suzhou, China, 3Kira Pharmaceuticals, Cambridge, MA Disclosure: Dr. Wen-Chao Song, a Professor of Pharmacology in the Department of Systems Pharmacology and Translational Therapeutics at the Perelman School of Medicine at the University of Pennsylvania and co-founder of Kira Pharmaceuticals as well as Chair of the Kira Scientific Advisory Board, owns equity, receives consultant fees and benefits from sponsored research funding from Kira. Dr. Song is also an inventor of patents owned by the University of Pennsylvania and licensed to Kira Pharmaceuticals, and as such, both Dr. Song and Penn may further benefit financially from the successful development and commercialization of products by the company. About KP104 KP104 is a first-in-class biologic with a unique dual-approach mechanism of action designed to selectively block both the alternative and terminal complement pathways. KP104 provides a powerful and synergistic method of targeting validated drivers of disease in the complement system. KP104 has also been engineered to have an extended half-life and potency and has a formulation suitable for both intravenous and subcutaneous administrations. KP104 is entering Phase 2 POC trials across multiple indications, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), thrombotic microangiopathies secondary to systemic lupus erythematosus (SLE-TMA) and paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 trials will be conducted globally including in the U.S., China, Australia, and South Korea. KP104 is an investigational agent not yet approved for any indication by any health authority. About Kira Pharmaceuticals Kira Pharmaceuticals is a global biotechnology company pioneering complement-targeted therapies to treat immune-mediated diseases. Enabled by its LOGIC drug discovery platform, the company is committed to advancing first-in-class and best-in-class therapies to transform the lives of patients. With offices in Cambridge, Massachusetts, and facilities in Suzhou and Shanghai, China, and Australia, Kira Pharmaceuticals is committed to establishing a global footprint and advancing life-changing therapies to patients around the world. More information on Kira can be found at www.kirapharma.com as well as on LinkedIn. SOURCE Kira Pharmaceuticals