Roxadustat

FibroGen has reached agreement with the FDA on important design elements for a pivotal Phase 3 clinical trial for roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell (RBC) transfusion burden Company intends to file the Phase 3 protocol in the fourth quarter of 2025 Aug. 07, 2025 -- FibroGen, Inc. (NASDAQ: FGEN) today announced positive feedback from its Type C meeting with the FDA, supporting the advancement of roxadustat for the treatment of anemia in patients with LR-MDS and high RBC transfusion burden, based on a post-hoc subgroup analysis from the MATTERHORN Phase 3 trial. “We are very pleased with the feedback we received from the FDA regarding roxadustat in patients with LR-MDS and anemia with high transfusion burden. This indication, despite recent approvals, still represents a patient population with significant unmet need,” said Thane Wettig, Chief Executive Officer of FibroGen. “We believe roxadustat’s differentiated mechanism of action, favorable tolerability profile, and oral route of administration can potentially be an important addition to the treatment options for patients with high transfusion burden. We are starting preparations for the Phase 3 trial, while evaluating internal development and potential partnership opportunities for this late-stage program. We plan to submit the Phase 3 trial protocol to the FDA in the fourth quarter of this year.” “Anemia is a major cause of morbidity and complications in patients with LR-MDS, especially those with high transfusion burden, and is often associated with poor quality of life and shortened survival. While we have recent approvals of injectable drugs for this indication, there is a significant unmet need for novel, effective oral agents for this patient population,” added Amer Zeidan, M.B.B.S, M.H.S., Professor of Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center, and the global principal investigator of the planned Phase 3 study. “Roxadustat has already shown promising efficacy in this group of patients in the post-hoc analysis of the MATTERHORN study, and I am glad we have agreed on a pathway with the regulators to explore the full potential of roxadustat in the upcoming Phase 3 trial. I am excited by the prospect of roxadustat potentially becoming a novel, safe, convenient, and effective therapy for LR-MDS patients with high transfusion burden. FibroGen requested the Type C meeting based on the findings of a post-hoc analysis of data from the Phase 3 MATTERHORN trial of roxadustat in anemia-associated with LR-MDS. In patients with high RBC transfusion burden at baseline (≥4 units over 8 weeks1), a pronounced treatment effect was observed: 36% (8/22) of patients achieved transfusion independence (TI) for ≥ 56 days on roxadustat vs 7% (1/15) of patients on placebo within 28 weeks (nominal p-value of 0.041). The planned Phase 3 trial will assess the safety and efficacy of roxadustat in a randomized, double-blind, placebo-controlled design in approximately 200 patients with LR-MDS. Alignment was reached with the FDA on the patient population (patients requiring ≥ 4 pRBC units in two consecutive 8-week periods prior to randomization, who are refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents (ESA) therapy), dose regimen, as well as management of potential thrombotic risk through eligibility and dose modification and discontinuation criteria. As the primary endpoint for the study, the Company is considering either 8-week or 16-week RBC TI. Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, thereof 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS. Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority. Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen, Inc. is a biopharmaceutical company focused on accelerating the development of novel therapies at the frontiers of cancer biology. Roxadustat (爱瑞卓®, EVRENZO™) is currently approved in China, Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted for review by the China Health Authority. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46 is in development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of an associated CD46-targeted PET biomarker. In addition, FibroGen has expanded its research and development portfolio to include two immuno-oncology product candidates for the treatment of solid tumors. The content above comes from the network. if any infringement, please contact us to modify.

Total consideration for the sale of FibroGen China to AstraZeneca now expected to be approximately $210 million , a $50 million increase from initial guidance Net cash held in China at closing now estimated to be approximately $125 million Transaction expected to close in 3Q 2025 Net cash held in China at closing now estimated to be approximately $125 million Transaction expected to close in 3Q 2025 Upon close of sale of FibroGen China, cash runway extended into 2028 Initiation of the Phase 2 monotherapy trial of FG-3246, a potential first-in-class antibody-drug conjugate (ADC) targeting CD46 in metastatic castration-resistant prostate cancer (mCRPC), expected in 3Q 2025 Reached agreement with the U.S. Food and Drug Administration (FDA) on important design elements for a pivotal Phase 3 trial for roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell (RBC) transfusion burden Topline results from the investigator-sponsored study of FG-3246 in combination with enzalutamide in patients with mCRPC expected in 4Q 2025 FibroGen to host conference call and webcast presentation today at 5:00 PM ET SAN FRANCISCO , Aug. 11, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today reported financial results for the second quarter 2025 and provided an update on the company’s recent developments. “In the second quarter, we continued to make steadfast progress in advancing our clinical pipeline. Trial initiation activities for the Phase 2 monotherapy trial of FG-3246 are progressing, and we expect to start the trial in the third quarter of 2025,” said Thane Wettig , Chief Executive Officer, FibroGen . “We are also excited about reaching agreement with the FDA to advance roxadustat towards a pivotal Phase 3 trial in LR-MDS in patients with high transfusion burden, an area with high unmet need. We are working diligently towards finalizing the Phase 3 trial protocol and plan to submit to the FDA in the fourth quarter of 2025. Concurrently, we will be exploring options for either internal development or partnership opportunities. With the expected close of the FibroGen China sale in the near term, extending our cash runway into 2028, we are strongly positioned to bring significant value for both patients and shareholders.” Recent Developments and Key Highlights of Second Quarter 2025: Sale of FibroGen China to AstraZeneca now expected to be for a total consideration of approximately $210 million , representing an enterprise value of $85 million plus estimated net cash held in China at closing of approximately $125 million . The transaction is expected to close in the third quarter of 2025. Upon closing, FibroGen will repay its term loan to Morgan Stanley Tactical Value, further simplifying the Company’s capital structure. FibroGen maintains its rights to roxadustat in the U.S. and in all markets outside of China , South Korea , and those licensed to Astellas. Appointed Michael Kauffman , M.D., Ph.D. to the Board of Directors. Had a positive Type-C meeting with the FDA in July 2025 , where the Company reached agreement with the FDA on important design elements for a pivotal Phase 3 trial for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden. Upon closing, FibroGen will repay its term loan to Morgan Stanley Tactical Value, further simplifying the Company’s capital structure. FibroGen maintains its rights to roxadustat in the U.S. and in all markets outside of China , South Korea , and those licensed to Astellas. Upcoming Milestones: FG-3246 (CD46 Targeting ADC) and FG-3180 (CD46 Targeting PET Imaging Agent) Trial initiation activities for the Phase 2 monotherapy dose optimization study of FG-3246 in mCRPC remain ongoing, with an expected trial start in the third quarter of 2025 upon the close of the sale of FibroGen China. The trial will also assess the diagnostic performance of FG-3180 to determine the potential correlation between CD46 expression and response to FG-3246. Topline results from the investigator-sponsored Phase 1b/2 study, conducted by UCSF, of FG-3246 in combination with enzalutamide in patients with mCRPC expected in the fourth quarter of 2025. The results will include data on FG-3180. Roxadustat FibroGen intends to file the pivotal Phase 3 clinical trial protocol for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden in the fourth quarter of 2025. Financial: Total revenue from continuing operations for the second quarter of 2025 was $1.3 million , as compared to $1.0 million for the second quarter of 2024. Net loss from continuing operations for the second quarter of 2025 was $13.7 million , or $3.38 net loss per basic and diluted share, compared to a net loss of $47.1 million , or $11.79 net loss per basic and diluted share, one year ago. On June 30, 2025 , FibroGen reported $23.5 million in cash, cash equivalents and accounts receivable in the U.S. and $142.1 million in total consolidated cash, cash equivalents and accounts receivable. Upon closing of the announced sale of FibroGen China, the Company expects its cash, cash equivalents and accounts receivable to be sufficient to fund our operating plans into 2028. Conference Call and Webcast PresentationThe FibroGen management team will host a conference call and webcast presentation to discuss the financial results and provide a business update. A live Q&A session will follow the brief presentation. Interested parties may access a live audio webcast of the conference call here. To access the call by phone, please register here, and you will be provided with dial in details. A replay of the webcast will also be available for a limited time on the Events & Presentations page on FibroGen’s website. About FG-3246FG-3246 (FOR46) is a potential first-in-class fully human antibody-drug conjugate (ADC), exclusively in-licensed from Fortis Therapeutics, and is being developed by FibroGen for metastatic castration-resistant prostate cancer and potentially other tumor types. FG-3246 binds to an epitope of CD46, a cell receptor target, that induces internalization upon antibody binding, is present at high levels in prostate cancer and other tumor types and demonstrates very limited expression in most normal tissues. FG-3246 is comprised of an anti-CD46 antibody, YS5, linked to the anti-mitotic agent, MMAE, which is a clinically and commercially validated ADC payload. FG-3246 has demonstrated anti-tumor activity in both preclinical and clinical studies. FG-3246 is currently in an ongoing Phase 1b/2 study being conducted at UCSF as an investigator-sponsored trial to evaluate FG-3246 in combination with enzalutamide. An additional investigator-sponsored radiopharmaceutical marker trial using a zirconium-89 positron emission tomography (PET) tracer for CD46 that utilizes the YS5 antibody is also underway at UCSF. The initiation of the Phase 2 monotherapy dose optimization trial for FG-3246 in metastatic castration-resistant prostate cancer is anticipated in the third quarter of 2025. FG-3246 is an investigational drug and not approved for marketing by any regulatory authority. About RoxadustatRoxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority . Roxadustat is approved in China , Europe , Japan , and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States , Canada , Mexico , and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan , Europe , Turkey , Russia , and the Commonwealth of Independent States, the Middle East , and South Africa . About FibroGen FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓®, EVRENZOTM) is currently approved in China , Europe , Japan , and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. The Company continues to evaluate a development plan for roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46 is in development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. For more information, please visit www.fibrogen.com. Forward-Looking Statements This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements under the caption “Upcoming Milestones”, statements about regulatory interactions, the estimated net cash portion of the purchase price and closing of the sale of FibroGen China as well as the payoff of the Morgan Stanley Tactical Value term loan, statements regarding cash, such as the expectation that, upon closing of the FibroGen China sale, cash, cash equivalents and accounts receivable will be sufficient to fund FibroGen’s operating plans into 2028, and statements about FibroGen’s plans and objectives. These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia , CFASenior Vice President and Chief Financial Officerir@fibrogen.com Source: FibroGen, Inc.