Latest news with #pMN
Associated Press
6 hours ago
- Business
- Associated Press
Ono Pharmaceutical and Vertex Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea
OSAKA, Japan & BOSTON--(BUSINESS WIRE)--Jun 23, 2025-- Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; 'Ono') and Vertex Pharmaceuticals Incorporated (Headquarters: Boston, MA, U.S.; CEO: Reshma Kewalramani, M.D.; 'Vertex') today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other serious B cell-mediated diseases. Under the terms of the agreement, Ono will pay Vertex an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. 'Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,' said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. 'We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.' 'Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure, and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.' The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. View source version on CONTACT: Ono Pharmaceutical Co., Ltd. Corporate Communications [email protected] more information: Vertex Pharmaceuticals Incorporated Investors: [email protected]: [email protected] or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 KEYWORD: NORTH AMERICA UNITED STATES SOUTH KOREA ASIA PACIFIC JAPAN MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Ono Pharmaceutical Co., Ltd. Copyright Business Wire 2025. PUB: 06/23/2025 04:00 AM/DISC: 06/23/2025 04:02 AM
Associated Press
6 hours ago
- Business
- Associated Press
Vertex and Ono Pharmaceutical Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea
BOSTON & OSAKA, Japan--(BUSINESS WIRE)--Jun 23, 2025-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Ono Pharmaceutical Co., Ltd. (OTCMKTS: OPHLY) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other B cell-mediated diseases. Under the terms of the agreement, Vertex will receive an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. 'Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.' 'Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,' said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. 'We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.' About Povetacicept Povetacicept is a recombinant fusion protein therapeutic and a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation and/or survival of B cells, T cells and innate immune cells. Based upon an engineered TACI (transmembrane activator and calcium modulator ligand interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious B cell-mediated diseases including other autoimmune kidney diseases and autoimmune cytopenias. About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan. IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years. There are no approved therapies that specifically target the underlying cause of IgAN. About Primary Membranous Nephropathy (pMN) Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 150,000 people diagnosed in the U.S. and Europe. It is estimated that there are approximately 6,000 diagnosed patients with pMN in Japan. pMN is a rare glomerular disease that occurs when the body generates an abnormal immune response, including autoantibodies, against proteins that are part of the kidney. Autoantibodies trigger damage and inflammation, especially within the glomeruli (the parts of the kidney that filter blood), impairing the kidneys' ability to properly filter waste and fluid, eventually causing progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN. About RAINIER RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating urine protein to creatinine ratio (UPCR) for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104. The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. About RUBY-3 RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, primary membranous nephropathy, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. About Ono Pharmaceutical Co., Ltd Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of 'Dedicated to the Fight against Disease and Pain,' Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at Vertex Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani M.D., and Toichi Takino, in this press release, and statements about the terms of and expectations for Vertex's collaboration with Ono, statements about potential benefits and results that may be achieved through the collaboration, statements regarding the future activities of the parties pursuant to the collaboration, including Ono's help to advance clinical trials and Ono's responsibility to obtain marketing authorizations in Japan and South Korea and to commercialize povetacicept in the regions, statements regarding upfront and milestone payments, and potential royalties on future products, and statements about Vertex's plans and expectations for the RAINIER and RUBY-3 clinical trials and potential plans to seek accelerated approval in the U.S. based on interim analysis from the RAINIER trial. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of the collaboration between Vertex and Ono may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at and on the SEC's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Ono Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice. View source version on CONTACT: Vertex Pharmaceuticals Incorporated Investors: [email protected] Media: [email protected] or International: +44 20 3204 5275 or U.S.: 617-341-6992 or Heather Nichols: +1 617-839-3607 Ono Pharmaceutical Co., Ltd. Corporate Communications [email protected] KEYWORD: NORTH AMERICA UNITED STATES SOUTH KOREA ASIA PACIFIC JAPAN MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY HEALTH GENETICS PHARMACEUTICAL CLINICAL TRIALS SOURCE: Vertex Pharmaceuticals Incorporated Copyright Business Wire 2025. PUB: 06/23/2025 04:00 AM/DISC: 06/23/2025 04:02 AM
Yahoo
8 hours ago
- Business
- Yahoo
Vertex and Ono Pharmaceutical Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea
- Exclusive collaboration and license agreement for povetacicept in these countries - - Ono will leverage its deep expertise in clinical development and commercialization for chronic kidney disease in Japan and South Korea to accelerate advancement of povetacicept for patients with IgA nephropathy and primary membranous nephropathy - BOSTON & OSAKA, Japan, June 23, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Ono Pharmaceutical Co., Ltd. (OTCMKTS: OPHLY) today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other B cell-mediated diseases. Under the terms of the agreement, Vertex will receive an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. "Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases." "Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono," said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. "We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment." About Povetacicept Povetacicept is a recombinant fusion protein therapeutic and a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation and/or survival of B cells, T cells and innate immune cells. Based upon an engineered TACI (transmembrane activator and calcium modulator ligand interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious B cell-mediated diseases including other autoimmune kidney diseases and autoimmune cytopenias. About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan. IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years. There are no approved therapies that specifically target the underlying cause of IgAN. About Primary Membranous Nephropathy (pMN) Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 150,000 people diagnosed in the U.S. and Europe. It is estimated that there are approximately 6,000 diagnosed patients with pMN in Japan. pMN is a rare glomerular disease that occurs when the body generates an abnormal immune response, including autoantibodies, against proteins that are part of the kidney. Autoantibodies trigger damage and inflammation, especially within the glomeruli (the parts of the kidney that filter blood), impairing the kidneys' ability to properly filter waste and fluid, eventually causing progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN. About RAINIER RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating urine protein to creatinine ratio (UPCR) for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104. The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. About RUBY-3 RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, primary membranous nephropathy, lupus nephritis and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. About Ono Pharmaceutical Co., Ltd Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of "Dedicated to the Fight against Disease and Pain," Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at Vertex Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani M.D., and Toichi Takino, in this press release, and statements about the terms of and expectations for Vertex's collaboration with Ono, statements about potential benefits and results that may be achieved through the collaboration, statements regarding the future activities of the parties pursuant to the collaboration, including Ono's help to advance clinical trials and Ono's responsibility to obtain marketing authorizations in Japan and South Korea and to commercialize povetacicept in the regions, statements regarding upfront and milestone payments, and potential royalties on future products, and statements about Vertex's plans and expectations for the RAINIER and RUBY-3 clinical trials and potential plans to seek accelerated approval in the U.S. based on interim analysis from the RAINIER trial. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of the collaboration between Vertex and Ono may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at and on the SEC's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Ono Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice. View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Media: mediainfo@ orInternational: +44 20 3204 5275orU.S.: 617-341-6992orHeather Nichols: +1 617-839-3607Ono Pharmaceutical Co., Ltd. Corporate Communications public_relations@
Business Wire
8 hours ago
- Business
- Business Wire
Ono Pharmaceutical and Vertex Announce Strategic Agreement to Develop and Commercialize Povetacicept in Japan and South Korea
OSAKA, Japan & BOSTON--(BUSINESS WIRE)--Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; 'Ono') and Vertex Pharmaceuticals Incorporated (Headquarters: Boston, MA, U.S.; CEO: Reshma Kewalramani, M.D.; 'Vertex') today announced an exclusive collaboration and license agreement for the development and commercialization of Vertex's povetacicept in Japan and South Korea. Povetacicept is a recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential being studied for the treatment of immunoglobulin A nephropathy (IgAN), primary membranous nephropathy (pMN) and other serious B cell-mediated diseases. Under the terms of the agreement, Ono will pay Vertex an upfront payment, as well as certain regulatory and commercial milestone payments and tiered royalties. Ono will utilize its extensive development expertise to help advance Vertex's clinical trials for povetacicept and will be responsible for obtaining marketing authorizations in Japan and South Korea. Following approval, Ono will be solely responsible for commercializing povetacicept in these regions. The agreement includes povetacicept for both IgAN and pMN, with the potential to add other indications. 'Vertex has a strong track record of developing innovative therapies for serious diseases. Through this strategic partnership, we can strengthen our late-stage pipeline in the immunology field, which is a key focus area for Ono,' said Toichi Takino, Representative Director, President and Chief Operating Officer of Ono. 'We look forward to collaborating with Vertex to provide this new therapeutic option for patients with IgAN and other autoimmune diseases in Japan and South Korea, and to maximize the value of this treatment.' 'Ono is a proven leader in Japan and South Korea, bringing established local relationships, infrastructure, and nephrology expertise that make them a perfect partner for Vertex as we look to deliver povetacicept to the thousands of potential patients in these countries,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'We are very pleased to partner with Ono and look forward to close collaboration as we continue to advance this potentially best-in-class treatment for IgAN, pMN and other serious B cell-mediated diseases.' About Povetacicept Povetacicept is a recombinant fusion protein therapeutic and a dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines, which play key roles in pathogenesis of multiple autoimmune diseases via their roles in the activation, differentiation, and/or survival of B cells, T cells, and innate immune cells. Based upon an engineered TACI (transmembrane activator and calcium modulator ligand interactor) domain, povetacicept has higher binding affinity and greater potency in preclinical studies versus other inhibitors of BAFF and/or APRIL alone and has demonstrated potential best-in-class efficacy in a clinical trial in patients with IgA nephropathy and primary membranous nephropathy. Povetacicept is also in development for multiple serious B cell-mediated diseases including other autoimmune kidney diseases and autoimmune cytopenias. About IgA Nephropathy (IgAN) IgAN is a serious, progressive, life-threatening, B cell-mediated chronic kidney disease that is the most common cause of primary (idiopathic) glomerulonephritis, affecting approximately 300,000 people in the United States and Europe. It is estimated that there are approximately 33,000 diagnosed patients in Japan. IgAN results from deposition of circulating immune complexes consisting of immunoglobulins and galactose-deficient immunoglobulin A (Gd-IgA1) in the renal glomerular mesangium, triggering kidney injury and fibrosis. Up to 72% of adult IgAN patients progress to end-stage renal disease within 20 years. There are no approved therapies that specifically target the underlying cause of IgAN. About Primary Membranous Nephropathy (pMN) Primary membranous nephropathy is a serious, progressive, life-threatening B cell-mediated chronic kidney disease affecting people worldwide, with approximately 150,000 people diagnosed in the U.S. and Europe. It is estimated that there are approximately 6,000 diagnosed patients with pMN in Japan. pMN is a rare glomerular disease that occurs when the body generates an abnormal immune response, including autoantibodies, against proteins that are part of the kidney. Autoantibodies trigger damage and inflammation, especially within the glomeruli (the parts of the kidney that filter blood), impairing the kidneys' ability to properly filter waste and fluid, eventually causing progressive loss of kidney function. There are no approved therapies that specifically target the underlying cause of pMN. About RAINIER RAINIER is a global Phase 3 pivotal trial of povetacicept 80 mg vs. placebo on top of standard of care in approximately 480 people with IgAN. The study is designed to have a pre-planned interim analysis evaluating urine protein to creatinine ratio (UPCR) for the povetacicept arm versus placebo after a certain number of patients reach 36 weeks of treatment. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval in the U.S. Final analysis will occur at two years of treatment, with a primary endpoint of total eGFR slope through Week 104. The Phase 3 clinical trial is underway in multiple regions, including the U.S., EU and Asia. Specifically, Japanese and South Korean regulatory authorities have approved the Clinical Trial Application (CTA) for RAINIER, where the Phase 3 trial is underway. About RUBY-3 RUBY-3 is an ongoing, multiple ascending dose, multi-cohort, open label, Phase 1/2 basket study of povetacicept in autoimmune glomerulonephritis, including IgAN, primary membranous nephropathy, lupus nephritis, and ANCA-associated vasculitis with glomerulonephritis where povetacicept is being administered subcutaneously for up to 104 weeks. About Ono Pharmaceutical Co., Ltd Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of 'Dedicated to the Fight against Disease and Pain,' Ono targets areas with unmet medical needs including oncology, immunology, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Ono Forward-Looking Statements In this press release, statements made with respect to current plans, estimates, strategies and beliefs, and other statements that are not historical facts are forward-looking statements about the future performance of the company. These statements are based on current assumptions and beliefs in light of the information currently available and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in the business environment in the pharmaceutical market and amendments to relevant laws and regulations, (ii) disruptions to product supply due to stagnation or delays in production caused by natural disasters, fires, etc., (iii) the possibility that sales activities for new and existing products may not achieve the expected results, (iv) the emergence of new side effects in post-marketing drugs, and (v) infringements of intellectual property rights by third parties. Information about pharmaceutical products included in this press release is not intended to constitute an advertisement or medical advice. Vertex Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani M.D., and Toichi Takino, in this press release, and statements about the terms of and expectations for Vertex's collaboration with Ono, statements about potential benefits and results that may be achieved through the collaboration, statements regarding the future activities of the parties pursuant to the collaboration, including Ono's help to advance clinical trials and Ono's responsibility to obtain marketing authorizations in Japan and South Korea and to commercialize povetacicept in the regions, statements regarding upfront and milestone payments, and potential royalties on future products, and statements about Vertex's plans and expectations for the RAINIER and RUBY-3 clinical trials and potential plans to seek accelerated approval in the U.S. based on interim analysis from the RAINIER trial. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of the collaboration between Vertex and Ono may not be achieved on the anticipated timeline, or at all, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy, or other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at and on the SEC's website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Yahoo
09-06-2025
- Business
- Yahoo
Everest Medicines Presents Positive Results in Preliminary Analysis of Phase 1b/2a Clinical Trial of Novel BTK Inhibitor EVER001 at the 62nd Congress of the European Renal Association
EVER001 is a covalent reversible BTK inhibitor with potentially best-in-class characteristics for the treatment of primary membranous nephropathy (pMN) and other autoimmune renal diseases, including IgA nephropathy (IgAN), minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and lupus nephritis (LN), offering treatment options for over 10 million patients worldwide. No drug has been approved globally for the treatment of pMN currently. There are approximately 2 million patients with pMN in China, and nearly 220,000 patients in the United States, Europe and Japan. As of December 17th, 2024, the ongoing Phase 1b/2a clinical trial of EVER001 includes longer-term data collected from some patients: 10 patients in the low-dose cohort completed 52 weeks of follow-up, and 10 patients in the high-dose cohort completed 24 weeks of treatment. Preliminary results showed that EVER001 was well-tolerated and effective in patients with pMN. These results support the potential of EVER001 as a treatment for proteinuric autoimmune glomerular diseases.- Compared to baseline, the least squares (LS) geometric mean levels of anti-PLA2R autoantibodies decreased by 62.1% in the low-dose cohort and 87.3% in the high-dose cohort at week 12. The reductions in both cohorts reached approximately 93% at week 24.- In the low-dose cohort, a 78.0% of reduction in proteinuria was observed by the end of 36 weeks of treatment. This reduction was sustained through week 52. In the high-dose cohort, a 70.1% of reduction in proteinuria at week 24 was shown.- EVER001 was generally safe and well tolerated. No clinically significant adverse events commonly associated with covalent irreversible BTK inhibitors were observed. SHANGHAI, June 9, 2025 /PRNewswire/ -- Everest Medicines (HKEX "Everest", or the "Company"), a biopharmaceutical company focused on the discovery, clinical development, manufacturing, and commercialization of innovative therapeutics, today announced that positive results in the ongoing Phase 1b/2a clinical trial for the treatment of primary membranous nephropathy (pMN) with EVER001 (previously known as XNW1011), a next-generation covalent reversible Bruton's tyrosine kinase (BTK) inhibitor will be presented in a focused oral session at the 62nd Congress of the European Renal Association (ERA 2025). Preliminary results showed that EVER001 was well-tolerated and effective in patients with pMN. These results support the potential of EVER001 as a treatment for proteinuric autoimmune glomerular diseases. This initial unveiling of the preliminary data at an international congress focuses on pMN, which is the second most common cause of primary glomerulonephritis. EVER001 is a covalent reversible BTK inhibitor with potentially best-in-class characteristics for the treatment of autoimmune renal diseases. Compared to covalent irreversible BTK inhibitors, EVER001 offers improved selectivity while maintaining high potency, thereby potentially avoiding many of the side effects associated with earlier-generation BTK inhibitors. Everest Medicines holds global rights to EVER001 for the treatment of renal diseases. This oral presentation highlights an ongoing Phase 1b/2a clinical trial of EVER001 for the treatment of pMN, which is being conducted in China. The study is designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of EVER001 in Chinese patients with glomerular diseases characterized by proteinuria. A total of 31 patients with biopsy-proven pMN who tested positive for anti-PLA2R autoantibodies were enrolled into two cohorts. The total treatment duration was 36 weeks. The presentation includes longer-term data collected from some patients (10 patients in the low-dose cohort completed 52 weeks of follow-up, and 10 patients in the high-dose cohort completed 24 weeks of treatment). Preliminary results showed that EVER001 was well-tolerated and effective in patients with pMN. These results support the potential of EVER001 as a treatment for proteinuric autoimmune glomerular diseases. The trial results, based on data analysis as of December 17th, 2024, demonstrated that EVER001 was generally safe and well tolerated, with the most common TRAEs being Grade 1-2. No clinically significant adverse events commonly associated with covalent irreversible BTK inhibitors, such as bleeding, arrhythmia, severe infections, or severe liver function impairment were observed. Compared to baseline, the least squares (LS) geometric mean levels of anti-PLA2R autoantibodies decreased by 62.1% in the low-dose cohort and 87.3% in the high-dose cohort at week 12. The reductions in both cohorts reached approximately 93% at week 24. 76.9% of patients in the low-dose cohort and 81.8% in the high-dose cohort achieved immunological complete remission at week 24. For 24-hour proteinuria, the LS geometric mean decreased by 78.0% from baseline in the low-dose cohort at week 36 and the reduction was maintained for 16 weeks after the end of treatment. At week 36, 69.2% of patients in the low-dose group achieved clinical remission. In the high-dose cohort, proteinuria had already decreased by 70.1% at week 24, with 80.0% of patients achieving clinical remission. Patients in both cohorts maintained stable renal function during the treatment period. "As a next-generation BTK inhibitor, EVER001 offers key advantages, including covalent reversibility, high selectivity, strong target-binding affinity, and reduced off-target toxicity. These attributes highlight its substantial potential in the treatment of pMN." Professor Minghui Zhao, leading principal investigator of EVER001 and an influential nephrologist at Peking University First Hospital, said: "Preliminary results from the Phase 1b/2a clinical trial of EVER001, presented at the 62nd ERA Congress, demonstrate that EVER001 induced rapid reductions in anti-PLA2R autoantibodies and proteinuria observed across both low- dose and high-dose cohorts. The treatment exhibits a favorable safety and tolerability profile. Currently, no drug has been approved globally for the treatment of pMN. Traditional immunosuppressive therapies carry a high risk of relapse following discontinuation, and many are associated with adverse effects. We therefore hope that new treatment options will become available to offer patients safer and more effective therapies." "To date, no drug has been approved globally for the treatment of pMN. As a potential best-in-class therapy, EVER001 holds promise to offer more treatment options for over 10 million patients worldwide affected by pMN, IgA nephropathy (IgAN), minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and lupus nephritis (LN)." Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines, said: "We are very pleased to see that the positive results in the preliminary analysis of our Phase 1b/2a clinical proof-of-concept trial of EVER001 were presented for the first time at an international academic conference. These preliminary results, disclosed at the 62nd ERA Congress, demonstrated that this next-generation covalent reversible BTK inhibitor was well-tolerated and effective in patients with pMN. Moving forward, we will continue to drive the global clinical development of EVER001, to meet patients' urgent clinical needs." pMN is a common pathological type of nephrotic syndrome in adults, and its prevalence in China has been increasing, ranking second only to IgA nephropathy[1]. There are approximately 2 million patients with pMN in China, and nearly 220,000 patients in the United States, Europe and Japan. There are no approved drugs for this indication worldwide. The current treatment goal is to improve remission rates, reduce high relapse rates, and minimize the risk of chronic toxicity caused by currently available treatments. More than one-third of pMN patients still progress to end-stage renal disease under current standards of care. This Phase 1b/2a clinical trial was approved by the Center for Drug Evaluation of the National Medical Products Administration in September 2022 to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of EVER001 in Chinese patients with glomerular diseases characterized by proteinuria. About EVER001 EVER001 (previously known as XNW1011) is a next-generation covalent reversible Bruton's tyrosine kinase (BTK) inhibitor in development globally for the treatment of renal diseases. BTK is an essential component of the B-cell receptor signaling pathways that regulate the survival, activation, proliferation, and differentiation of B lymphocytes. Targeting BTK with small molecule inhibitors has been demonstrated to be an effective treatment option for B-cell lymphomas and autoimmune diseases. Under an exclusive licensing agreement with Sinovent Pharmaceuticals and SinoMab BioScience, Everest owns global rights to develop, produce and commercialize EVER001 for the treatment of renal diseases. About Everest Medicines Everest Medicines is a biopharmaceutical company focused on discovering, developing, manufacturing and commercializing transformative pharmaceutical products and vaccines that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record from both leading global pharmaceutical companies and local Chinese pharmaceutical companies in high-quality discovery, clinical development, regulatory affairs, CMC, business development and operations. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules in the company's core therapeutic areas of renal diseases, infectious diseases and autoimmune disorders. For more information, please visit its website at Forward-Looking Statements: This news release may make statements that constitute forward-looking statements, including descriptions regarding the intent, belief or current expectations of the Company or its officers with respect to the business operations and financial condition of the Company, which can be identified by terminology such as "will," "expects," "anticipates," "future," "intends," "plans," "believes," "estimates," "confident" and similar statements. Such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, or other factors, some of which are beyond the control of the Company and are unforeseeable. Therefore, the actual results may differ from those in the forward-looking statements as a result of various factors and assumptions, such as future changes and developments in our business, competitive environment, political, economic, legal and social conditions. The Company or any of its affiliates, directors, officers, advisors or representatives has no obligation and does not undertake to revise forward-looking statements to reflect new information, future events or circumstances after the date of this news release, except as required by law. References: 1. Expert consensus on the application of rituximab in the treatment of membranous nephropathy, Chin J Intern Med, March 2022, Vol. 61, No. 3. View original content: SOURCE Everest Medicines Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
