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WUXI, China, Feb. 25, 2024 /PRNewswire/ -- BioCity Biopharma is pleased to announce the completion of enrollment of all 120 participants in the IgA nephropathy (IgAN) cohort in a randomized, double-blind, placebo-controlled Phase 2 clinical study of the novel, oral endothelin A (ETA)-receptor selective antagonist SC0062, currently under development for chronic kidney disease (CKD), including IgAN and diabetic kidney disease (DKD). The enrollment of DKD cohort is ongoing with expected completion by the end of Q2 2024. "We appreciate the dedication of our colleagues and collaborators. The IgAN cohort enrollment was initiated in June 2023, and we enrolled all 120 subjects in less than 9 months. Thanks to the investigators, their teams, patients, and related BioCity colleagues", Dr. Ivy Wang, co-founder and executive vice president of BioCity, stated. This accomplishment highlights BioCity's ability to coordinate resources and operate efficiently in clinical trials, and further solidifies its leading position in the field of CKD. Dr. Wang emphasized that this achievement aligns well with the company's strategic objectives and establishes a solid foundation for further corporate development. Dr. Yong jiang Hei, CEO of BioCity, noted the clear trend of drug development focus on chronic diseases globally, with CKD gaining significant attention. The early entry in this field by BioCity reflects its thoughtful vision. There are approximately 700 million potential CKD patients worldwide with 82 million in China. The vast CKD market is growing rapidly, and the unmet medical needs are significant. Proteinuria is a key prognostic indicator of disease progression in CKD and can serve as an end point to evaluate the effectiveness of interventions intended to treat CKD. Of note, the US FDA granted accelerated approval of the first treatment for IgAN using proteinuria as a surrogate clinical endpoint. "CKD (including DKD and IgAN) with proteinuria is the primary indication under development for SC0062 which has demonstrated significant safety advantages in preclinical and Phase 1 studies. Leveraging our efficient clinical operation capabilities, we plan to accelerate the phase 2 study and initiate Phase 3 trials in 2024. Our goal is to bring this innovative medicine to patients and improve their quality of life at the earliest possible time." Dr. Hei says. About SC0062 SC0062 is one of the most unique ETA receptor small molecule antagonists globally to enter clinical development for CKD, which designed with high selectivity for ETA receptors with the objective of ensuring efficacy while avoiding the potential safety risks associated with other molecules in the same class. Preclinical studies have shown that SC0062 improved pathological scores in models of acute kidney injury and CKD. In the completed Phase I study, SC0062 demonstrated favorable safety profile, good tolerability, and predictable pharmacokinetic characteristics. Fluid retention, an adverse event presumable associated with ETB blockade, was not observed in the phase 1 trial in healthy volunteers. SC0062 is potentially a best-in-class ETA receptor-selective antagonist. About the Phase 2 Clinical Study of SC0062 (CTR20230689) The ongoing Phase 2 clinical study is designed to evaluate the efficacy and safety of SC0062 in patients with CKD with proteinuria. It is a multi-center, randomized, double-blind, placebo-controlled study with two parallel cohorts (IgAN and DKD). The trial is led by professor Jianghua Chen, director of the Kidney Disease Center at the First Affiliated Hospital of Zhejiang University School of Medicine and former Chairman of the Chinese Medical Association Nephrology Branch. The study is being conducted simultaneously at over 40 study sites nationwide. Currently, the IgAN cohort enrollment is completed, and the DKD cohort continues to enroll patients. About BioCity Founded in December 2017, BioCity is a clinical-stage biopharmaceutical company committed to developing novel and highly differentiated, modality-independent therapeutics for cancer and autoimmune disorders including chronic kidney diseases (CKD). The company has established a pipeline of more than 10 innovative drug candidates including small molecules, monoclonal and bispecific antibodies as well as antibody-drug conjugates (ADCs). Currently, BioCity Biopharma has 6 oncology assets in Phase 1 development, including the first-in-class CDH3-targeting ADC, WEE1 and ATR inhibitors targeting the DNA damage response (DDR) pathway, and agents targeting the immune system including a T cell engager (CD3/EGFR BsAb), an immune checkpoint inhibitor (TIM-3 mAb), and a T cell activator (4-1BB mAb). In addition, an endothelin A (ETA)-receptor selective antagonist for CKD is in phase 2 clinical development. For more information, please visit . Contact: [email protected] [email protected] SOURCE BioCity Biopharma

Committee for Medicinal Products for Human Use (CHMP) recommends approval of the conditional marketing authorization (CMA) for sparsentan for the treatment of IgA nephropathy (IgAN) in Europe Positive CHMP opinion is based on pivotal phase-III PROTECT study results European Commission decision is expected in Q2 2024 ST. GALLEN, Switzerland, Feb. 23, 2024 /PRNewswire/ -- CSL Vifor and Travere Therapeutics, Inc., (NASDAQ: TVTX) today announced that the European Medicines Agency's (EMA) CHMP has recommended approval of sparsentan for the treatment of adults with primary IgAN with a urine protein excretion >1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). IgAN is a rare kidney disorder and a leading cause of kidney failure. The CHMP opinion provides the basis for the European Commission's final decision regarding CMA for sparsentan. If approved in Europe, sparsentan will be the first non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist for the treatment of IgAN. "The positive CHMP opinion for sparsentan is one step closer to bringing this treatment option to patients in Europe with IgAN, a rare and serious condition that can cause kidney disease," said Emmanuelle Lecomte Brisset, Senior Vice President and Head of Global Regulatory Affairs at CSL. "We look forward to the European Commission decision and to continuing to advance CSL's promise to provide innovative treatments for patients with kidney disease." "The positive recommendation from the CHMP represents a significant advancement towards the delivery of new treatment options for people living with IgAN, who face the risk of progression to kidney failure and who currently have no approved non-immunosuppressive treatment options. The PROTECT Study is the only head-to-head study in IgAN against a maximally labeled dose of irbesartan, a current standard of care. The study demonstrated treatment with sparsentan resulted in a rapid and sustained reduction in proteinuria and has the potential to preserve kidney function and significantly delay time to kidney failure compared to an active comparator, suggesting long-term benefits in IgAN," said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. "Together with CSL Vifor, we look forward to the European Commission's decision in the second quarter of 2024." The positive CHMP opinion is based on results from the pivotal phase-III PROTECT study of sparsentan in IgAN, In August 2022, CSL Vifor and Travere Therapeutics announced they had submitted a Marketing Authorization Application (MAA) for CMA to the EMA. The European Commission previously granted Orphan Medicinal Product Designation to sparsentan for the treatment of IgAN. If approved, sparsentan would receive a CMA in all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. Sparsentan is currently marketed in the U.S. and granted accelerated approval by the U.S. Food and Drug Administration under the brand name FILSPARI® based on reduction in proteinuria. In 2021, Travere Therapeutics granted CSL Vifor exclusive commercialization rights for sparsentan in Europe, Australia and New Zealand. About CSL Vifor CSL Vifor is a global partner of choice for pharmaceuticals and innovative, leading therapies in iron deficiency and nephrology. We specialize in strategic global partnering, in-licensing and developing, manufacturing and marketing pharmaceutical products for precision healthcare, aiming to help patients around the world lead better, healthier lives. Headquartered in St. Gallen, Switzerland, CSL Vifor also includes the joint company Vifor Fresenius Medical Care Renal Pharma (with Fresenius Medical Care). The parent company, CSL (ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs 32,000 people and delivers its lifesaving therapies to people in more than 100 countries. For more information about CSL Vifor visit, . About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com. About IgA Nephropathy (IgAN) IgAN, also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function. Other symptoms of IgAN may include swelling (edema) and high blood pressure. IgAN is the most common type of primary glomerular disease worldwide and a leading cause of kidney failure. IgAN is estimated to affect up to 250,000 people in the licensed territories. About the PROTECT study The PROTECT Study is one of the largest interventional studies to date in IgA nephropathy (IgAN) and the only head-to-head trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of sparsentan, compared to 300 mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite receiving maximum tolerated dose and at least ­50% of maximum labelled dose of ACE or ARB therapy. About sparsentan Sparsentan is a novel, non-immunosuppressive, single-molecule, dual endothelin angiotensin receptor antagonist with high selectivity for the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). Pre-clinical data have shown that blockade of both endothelin type A and angiotensin II type 1 pathways in forms of rare chronic kidney disease, protects podocytes, prevents glomerulosclerosis and mesangial cell proliferation, and reduces proteinuria. Sparsentan is currently available in the US and was granted accelerated approval by the US Food and Drug Administration in February 2023 under the brand name FILSPARI® based on reduction in proteinuria. In September 2023, Travere Therapeutics reported two-year confirmatory results from the Phase 3 PROTECT Study of FILSPARI in IgAN. View original content to download multimedia: SOURCE Vifor International AG (CSL Vifor) Company Codes: NASDAQ-NMS:TVTX

Pictured: AstraZeneca building/iStock, Sundry Photography AstraZeneca announced in its recent quarterly update that it discontinued a Phase IIb trial for its investigational drug tozorakimab for the treatment of diabetic kidney disease due to efficacy. However, a new report from analytics firm GlobalData is bullish on the potential prospects of the pharma’s pipeline drug zibotentan, if it gets approval. Despite removing tozorakimab from its Phase II pipeline for diabetic kidney disease (DKD), zibotentan could be “an optimal treatment of choice” for patients and “of high value” for physicians trying to find treatments for the disease. “According to key opinion leaders (KOLs) interviewed by GlobalData, unmet needs in the kidney disease space include next-generation therapies with alternate mechanisms of action and enhanced clinical pro ” Kajal Jaddoo, senior pharma analyst at GlobalData, said in a statement. Tozorakimab, which acts by blocking endothelin receptors and binds to the ET-A receptor, made it to a Phase IIb DKD study in 2019, with GlobalData stating that there was evidence that increased inflammation is associated with deterioration in renal function. The trial’s hypothesis was that the drug may be “beneficial” for DKD patients on standard of care (SOC) who have inflammation as well. But that promise was not realized. “AstraZeneca has a strong commercial position and is well known in both the metabolic and cardiovascular markets. KOLs further emphasized that until further clinical data solidifies the superior or non-inferior safety and efficacy of monoclonal antibodies in combination with SOC, physicians will not substantially change their prescribing patterns,” Jaddoo said. Zibotentan acts by blocking growth receptors called endothelin receptors, binding selectively to the ET-A receptor and inhibiting endothelin-mediated mechanisms that promote cell proliferation, according to GlobalData. However, zibotentan has not seen success in other verticals, such as cancer. In 2010, zibotentan failed to improve survival in a late-phase trial in prostate cancer, while a year later a Phase III study in prostate cancer was stopped as it was unlikely to reach the endpoints of overall and progression-free survival. Still, in 2023, a combination of zibotentan and dapagliflozin showed statistically significant results in the urinary albumin-to-creatinine ratio (UACR) to asses albuminuria, with the UACR difference being down over 33% and earning a p-value of p<0.001. AstraZeneca saw a 90% plummet in its COVID-19 business in 2023 but still managed to secure 6% in total growth for the year and an 8% growth rate in the fourth quarter of last year. Areas such as cardiovascular, rare disease, oncology, and metabolism were significant growth factors for the pharma and executives are confident the company is poised for more growth in 2024. Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.