Previous clinical observations of potential benefit from Roxadustat in this complex patient population prompted this investigation Therefore, the investigators designed this retrospective, observational study to thoroughly investigate the effects of Roxadustat on heart failure treatment and ventricular remodelling in this specific patient population, aiming to provide new insights for patients management.

Start Date01 Dec 2025

Sponsor / Collaborator

Southeast University Affiliated Zhonda Hospital

NCT07162090

/ Not yet recruitingPhase 4IIT

Observation of the Impact of Hypoxia - Inducible Factor Prolyl Hydroxylase Inhibitors on Sarcopenia in Hemodialysis Patients

Sarcopenia, abbreviated as muscle loss, is a prevalent complication among patients with chronic kidney disease (CKD), particularly those with end - stage renal disease (ESRD). It significantly impacts patients' quality of life. The prevalence of sarcopenia in patients receiving maintenance hemodialysis (MHD) ranges from 32.7% to 73.5%, which is substantially higher than that in the general population (5% - 13%). Sarcopenia significantly elevates the mortality risk in MHD patients. Specifically, sarcopenia patients experience an increased all - cause mortality rate, a heightened risk of cardiovascular events, a decline in quality of life, and an augmented risk of falls and fractures. A close pathophysiological relationship exists between the hypoxia - inducible factor - 1 (HIF - 1) pathway and sarcopenia. HIF - 1α serves as a key transcription factor for cells to respond to hypoxic conditions. Under normoxic conditions, HIF - 1α is hydroxylated by prolyl hydroxylase (PHD) and subsequently undergoes ubiquitination - mediated degradation. Conversely, under hypoxic circumstances, HIF - 1α is stably expressed, translocates into the nucleus, and activates downstream target genes. HIF - 1α promotes the expression of genes associated with glycolysis, such as GLUT1 and LDHA, while inhibiting mitochondrial oxidative phosphorylation. This results in a shift of skeletal muscle energy metabolism from aerobic to anaerobic pathways. Research has revealed that the protein level of HIF - 1α is significantly decreased in sarcopenia patients. Roxadustat capsules, an oral medication, represent the world's first small - molecule hypoxia - inducible factor prolyl hydroxylase inhibitor (HIF - PHI) developed for the treatment of renal anemia. The physiological function of HIF - 1α not only enhances the expression of erythropoietin but also upregulates the expression of erythropoietin receptors and proteins involved in promoting iron absorption and circulation. Theoretically, roxadustat has the potential to improve sarcopenia. However, due to its prominent effect on anemia correction, it is currently only clinically applicable to anemic patients. This study aims to use ESA as a control to investigate the effect of roxadustat on sarcopenia in hemodialysis patients during the treatment of renal anemia.

Start Date10 Sep 2025

Sponsor / Collaborator

Tianjin Medical University General Hospital

ChiCTR2500106730

/ Not yet recruitingPhase 4IIT

Observation of the Impact of Hypoxia - Inducible Factor Prolyl Hydroxylase Inhibitors on Sarcopenia in Hemodialysis Patients

Start Date01 Aug 2025

Sponsor / Collaborator

Tianjin Medical University Cancer Institute and Hospital

100 Clinical Results associated with Roxadustat

100 Translational Medicine associated with Roxadustat

100 Patents (Medical) associated with Roxadustat

578

Literatures (Medical) associated with Roxadustat

31 Dec 2026·RENAL FAILURE

Efficacy and safety of prolyl hydroxylase inhibitors for anemia in chronic kidney disease: a network meta-analysis

Review

Author: Li, Yongmei ; Zhan, Yaoxuan ; Niu, Yuanchen ; Fang, Yi ; Xu, Haimei ; Hu, Zhiqin ; Wang, Tenghua ; Li, Chen ; He, Jin

This Bayesian network meta-analysis evaluated the efficacy and safety of six prolyl hydroxylase inhibitors (PHIs) versus erythropoiesis-stimulating agents (ESAs) or placebo for chronic kidney disease (CKD) anemia. Electronic databases were searched through 1 May 2025. The prespecified primary outcome was change in hemoglobin, while secondary outcomes included iron metabolism indices (serum iron, transferrin saturation [TSAT], ferritin, hepcidin) and overall composite adverse events (AEs). Surface under the cumulative ranking curve (SUCRA) was used for ranking. Forty-six randomized controlled trials with 32,305 patients were included. In non-dialysis CKD (ND-CKD), roxadustat yielded the greatest hemoglobin rise (MD = 1.21 g/dL, 95% CI: 0.72-1.71; SUCRA = 88.6%) versus placebo and ESA. In dialysis CKD (D-CKD), roxadustat again ranked the highest (MD = 1.78 g/dL, 95% CI: 1.32-2.24; SUCRA = 86.2%) versus ESA and other PHIs. For TSAT, ESA ranked best in ND-CKD (MD = 5.24%, 95% CI: 1.16-9.32), while daprodustat led in D-CKD (MD = 3.07%, 95% CI: 0.22-5.91). Roxadustat improved serum iron in D-CKD (MD = 6.19 μg/dL, 95% CI: 2.81-9.58), and vadadustat and roxadustat reduced hepcidin in ND-CKD. Consistency assessment revealed no statistically significant heterogeneity/inconsistency between direct/indirect comparisons, supporting the robustness of the results. Regarding safety, ESA had the lowest AE risk in ND-CKD (OR = 0.85, 95% CI: 0.74-0.98), while roxadustat ranked lowest (SUCRA = 18.4%). Roxadustat demonstrated the strongest efficacy in hemoglobin improvement but higher AE incidence in ND-CKD, whereas ESA and daprodustat showed safety and iron metabolism benefits, supporting individualized therapy for renal anemia.Registration number: PROSPERO (CRD420251066181).

01 Mar 2026·CELLULAR SIGNALLING

Roxadustat induces erythroid differentiation of erythroleukemia cells through the hypoxia inducible factor-α/GATA binding protein 1 axis

Article

Author: Chen, Yuanzhong ; Lin, Jia ; Luo, Zhehan ; Wu, Yong ; Yang, Liqing ; Wang, Shuang

BACKGROUND:

Acute erythroleukemia (AEL) is a rare type of acute myeloid leukemia characterized by severe anemia. Roxadustat is a marketed first-generation hypoxia inducible factor-proline hydroxylase inhibitor (HIF-PHI), currently widely used in anemia associated with chronic kidney disease. Studies on roxadustat in anemia related to myeloid neoplasms remains limited.

METHODS:

Erythroid differentiation levels of HEL and K562 cells after roxadustat treatment were assessed via flow cytometry and benzidine staining. Real timequantitative PCR and western blot were performed to detect mRNA and protein expression levels of erythroid differentiation-related genes. Small interfering RNA (siRNA) was used to knock down HIF-1α or HIF-2α to elucidate the primary target of roxadustat-induced erythropoiesis. To study the role of GATA binding protein 1 (GATA1) in roxadustat-induced erythropoiesis, CRISPR/Cas9-mediated GATA1 knockout cell lines were established. Chromatin immunoprecipitation-PCR and dualluciferase reporter assay were conducted to determine the interaction between HIF-α and GATA1.

RESULTS:

Roxadustat significantly promoted erythroid differentiation in HEL and K562 cells. Knockdown of HIF-2α rather than HIF-1α, and knockout of GATA1 both effectively inhibited this effect. Both HIF-1α and HIF-2α are responsible for the direct upregulation of GATA1 expression under hypoxia, with HIF-1α as the dominant transcription factor. Despite potentially different roles of HIF-1α and HIF-2α in leukemogenesis, roxadustat combined with ruxolitinib or imatinib exhibited synergistic anti-leukemia effect in HEL and K562 cells, respectively.

CONCLUSIONS:

Roxadustat induces erythropoiesis in erythroleukemia cells primarily by targeting HIF-2α and was partially GATA1-dependent. Besides upregulating erythropoietin, our study revealed the HIF-α/GATA1 axis as another critical regulatory mechanism for hypoxia-driven erythropoiesis.

01 Feb 2026·CANCER LETTERS

Lactic acid promotes an MDSC-like phenotype via HIF1α stabilization with impact on prognosis in renal cell carcinoma

Article

Author: Strieder, Nicholas ; Sala-Hojman, Ada ; Kreutz, Marina ; Ferretti, Roberta ; Afonso, Julieta ; Heinrich, Timo ; Herr, Wolfgang ; Hofbauer, Joshua ; Hau, Peter ; Althammer, Michael ; Martowicz, Agnieszka ; Babl, Nathalie ; Maddaloni, Marianna ; Herhaus, Christian ; Rehli, Michael ; Schmidl, Christian ; Friedl, Sarah ; Simon, Malte ; Brummer, Christina ; Mayr, Roman ; Laufs, Johanna ; Bruss, Christina ; Matos, Carina ; Siska, Peter J ; Loipfinger, Stefan ; Renner, Kathrin ; Singh, Sukh M ; Decking, Sonja-Maria ; Gebhard, Claudia ; Oefner, Peter J ; Baltazar, Fatima ; Voll, Florian ; Hoffmann, Petra ; Schnell, Annette ; Vollmann-Zwerenz, Arabel ; Evert, Katja ; Dettmer, Katja

Paradoxically, immune cell infiltration correlates with worse prognosis in renal cell carcinoma (RCC) patients, with tumor-associated myeloid cells playing a key role in tumor progression. However, little is known about factors driving their polarization. Here, we investigated the link between RCC-related glycolysis, hypoxia-inducible factor (HIF)1α-associated myeloid inflammation, and patient prognosis. TCGA data revealed a strong correlation between the expression of monocarboxylate transporter 4 (MCT4), the myeloid marker CD14 and patient survival in ccRCC patients. scRNAseq data confirmed high MCT4 expression in both tumor and myeloid cells, suggesting lactate transport. In vitro analyses proved lactate uptake by CD14⁺ monocytes, which stabilized HIF1α and induced an MDSC-like, HLA-DR low phenotype. In line, the HIF1α-stabilizing drug Roxadustat increased the number of CD14⁺ HLA-DR low cells. Lactate uptake also increased protein lactylation. To further investigate the interplay between RCC tumor and myeloid cells, we established a 3D spheroid co-culture model and analyzed the effects of MCT inhibitors. This 3D model reflected tumor-myeloid cell interactions, as spheroid-infiltrating myeloid cells exhibited spontaneous IL-6 secretion comparable to patient-derived RCC cultures. Inhibition of lactate secretion reduced lactate and IL-6 secretion while increasing CD14⁺ HLA-DR⁺ cells. These findings were validated in patient-derived RCC cultures treated with anti-glycolytic drugs. Our data dissect the intratumoral network of RCC and show that tumor-derived lactate promotes a pro-tumorigenic myeloid phenotype with low MHC-II but high immune-checkpoint, LOX-1 and S100A8/9 expression. Blocking MCT disrupts this interplay, offering a promising strategy to re-educate tumor-associated myeloid cells and enhance tumor immune surveillance.

197

News (Medical) associated with Roxadustat

03 Mar 2026

·www.fiercepharma.com

FDA’s CRLs reveal critical errors in AstraZeneca’s Saphnelo data, efficacy doubts for GSK’s Exdensur

While AZ has undergone an audit of its Saphnelo datasets to regain the agency’s trust, GSK faces a more daunting challenge: proving that Exdensur actually works in CRSwNP. The FDA is providing more insight into the rejections it recently handed two pharma giants, publishing the complete response letters (CRLs) sent to AstraZeneca and GSK that highlight shortfalls in the data included in their application packages.In a scathing rebuke (PDF) to AstraZeneca, the FDA cited “critical data quality issues that affect key analyses, including the primary endpoint” within a dataset for Saphnelo (anifrolumab) for the drug’s proposed use in systemic lupus erythematosus (SLE). Separately, the agency rejected (PDF) GSK’s Exdensur (depemokimab) for chronic rhinosinusitis with nasal polyps (CRSwNP), ruling that the clinical data failed to provide “adequate evidence” of efficacy, casting doubts on the drug’s potential expansion in that use.The publication of the rejection letters underscores the FDA’s promise of “radical transparency” regarding its drug review process. While AZ has undergone an audit of its Saphnelo datasets to regain the agency’s trust, GSK faces a more daunting challenge: proving that Exdensur actually works in CRSwNP.For AZ, the CRL was issued on Oct. 10, 2025, according to the FDA’s database. But the company didn’t disclose it until Feb. 3, 2026. At that time, the company said it had submitted the information requested by the FDA in the CRL, and that a decision on the updated application was expected in the first half of this year. AZ’s initial submission was based on an interim analysis of the phase 3 Tulip-SC trial, which showed a reduction in disease activity for subcutaneous Saphnelo versus placebo as measured using the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) at week 52. However, in the CRL, the FDA flagged that AZ had submitted a “corrected” dataset for the study late in the review cycle after initially making “errors” in the application.As a result, the FDA asked AZ to provide, among other things, a “corrected interim clinical study report” and corrected raw datasets for both the interim and final analyses, presented in formats standard for regulatory reviews. Additionally, the FDA asked the company to “provide a detailed discussion of what led to the errors in the locked database being found,” as well as “a subject-level listing of all the changes in all the datasets along with the reason for each change.”Two months following the FDA’s rejection, European regulators authorized subcutaneous Saphnelo in SLE. This is not the first time that AZ has found itself dealing with data quality issues in a drug application for a kidney disease treatment. Back in 2021, AZ’s then-partner FibroGen filed for an FDA approval but then admitted to manipulating the safety data of roxadustat in chronic kidney disease-related anemia, data that the two firms unveiled to much fanfare in 2019.Roxadustat has since been rejected by the FDA, and AZ exited the collaboration in the U.S. in 2024. Efficacy questions for GSK Meanwhile, GSK faces a different hurdle for Exdensur, as the FDA has asked for “new evidence” that can demonstrate a treatment effect of greater magnitude to prove that the antibody drug provides a “statistically persuasive and clinically meaningful” benefit to CRSwNP patients. In its CRL to GSK, the FDA acknowledged that the co-primary endpoints of the Anchor-1 and -2 trials were met, showing statistically significant improvements for Exdensur versus placebo on nasal polyp scores and nasal obstruction scores. However, on the nasal obstruction score measure, the FDA found the outcomes were “not robust to sensitivity analyses for missing data and handling of intercurrent events.” The FDA views results from the patient-reported endpoint “especially important for assessing benefit-risk because they are a direct reflection of the clinically meaningful effects for patients.”The magnitude of the effects for both primary endpoints was “numerically small,” so the FDA questioned their clinical meaningfulness.The agency’s review team also looked at secondary endpoints, including additional patient-reported symptom scores, the need for nasal sinus or polyp surgery, and oral corticosteroid use. However, because of the statistical design of the studies, none of those endpoints met statistical significance, despite trends favoring Exdensur.The FDA went on further to suggest that a higher dose than the 100 mg selected by GSK “may provide a greater treatment effect,” faulting GSK for not running phase 2 dose-ranging trials specifically in CRSwNP.To tip the review balance further against GSK, the FDA said the every-half-year dosing—a long-acting feature that GSK has touted—means that non-responders would either have to wait a long time to switch to another drug, or risk any issues from taking two biologic therapies while Exdensur is still present in their bodies. “Given the small effect size for depemokimab with lack of robustness on tipping point analysis, the benefit-risk in the setting of a six-month duration of action is not favorable,” the FDA concluded. While the FDA declined to approve Exdensur in CRSwNP, the agency approved the long-acting IL-5 agent as an add-on maintenance treatment for severe asthma with an eosinophilic phenotype. The drug is approved in the EU for both indications.“We remain confident in the benefit Exdensur could bring for patients with CRSwNP and are continuing discussions with the FDA,” a GSK spokesperson told Fierce Pharma. GSK’s focus in the U.S. is on severe asthma, which affects about two million Americans with more than half uncontrolled on current therapy, as well as chronic obstructive pulmonary disease (COPD), the spokesperson added.Exdensur is a key element of GSK's plan to reach more than 40 billion pounds sterling in annual revenue by 2031, a task that has recently fallen on the shoulders of new CEO Luke Miels. In a more important indication than CRSwNP, GSK is testing Exdensur in three phase 3 trials in COPD.

Phase 3Drug ApprovalClinical ResultPhase 2

24 Feb 2026

·www.biospace.com

Kyntra Bio Announces Positive Data from the Investigator-Sponsored Phase 1b/2 Study of FG-3246 in Combination with Enzalutamide in Patients with Metastatic Castration Resistant Prostate Cancer to Be Presented at ASCO GU 2026

FG-3246 and enzalutamide combination therapy, in biomarker unselected patients with androgen receptor pathway inhibitor (ARPI)-treated, taxane-naïve metastatic castration-resistant prostate cancer (mCRPC), led to a median radiographic progression free survival (rPFS) of 7.0 months in the overall study cohort, with median rPFS of 10.1 months observed in patients who progressed on only one prior ARPI Higher tumor uptake of FG-3180, a CD46 directed PET imaging agent, demonstrated a trend towards higher probability of PSA50 response, highlighting its potential for patient selection Combination therapy had a similar safety and exposure pro the previous FG-3246 Phase 1 monotherapy trial Results further validate key FG-3246 Phase 2 monotherapy design elements, most importantly the inclusion of patients who have progressed on only one prior ARPI and integration of baseline FG-3180 PET for all enrolled patients FG-3246 Phase 2 monotherapy trial on track for interim analysis in 2H 2026 SAN FRANCISCO, Feb. 23, 2026 (GLOBE NEWSWIRE) -- Kyntra Bio (Nasdaq: KYNB), formerly FibroGen (Nasdaq: FGEN), today announced that the data on anti-tumor activity of FG-3246 in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) from the investigator-sponsored Phase 1b/2 study will be presented at the 2026 American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU), taking place February 26-28, 2026 in San Francisco, CA. “The results from this Phase 1b/2 investigator-initiated study demonstrate encouraging preliminary anti-tumor activity of FG-3246 in combination with enzalutamide in patients with mCRPC. Notably, the 10.1 months of rPFS in patients with only one prior ARPI underscores the potential of FG-3246 in earlier lines of therapy,” commented Dr. Rahul Aggarwal, Professor of Medicine at the University of California San Francisco, and Principal Investigator of the study. “The positive trend observed in the association between tumor uptake of FG-3180 (CD46-targeting PET) and PSA50 response, though from a small number of patients, is especially intriguing and I’m excited to see the potential utility of this biomarker further explored in the Phase 2 monotherapy study.” “These data from the investigator sponsored trial expand on the clinically meaningful results previously observed with FG-3246,” said Thane Wettig, Chief Executive Officer of Kyntra Bio. “The 10.1 months of median rPFS observed in patients progressing on only one prior ARPI, and the mitigation of neutropenia related adverse events with prophylactic G-CSF are especially encouraging as they further validate our Phase 2 monotherapy trial design. We look forward to sharing the interim analysis of the Phase 2 monotherapy trial in the second half of 2026 as well as further characterizing the potential utility of FG-3180 as a patient selection biomarker.” The presentation includes data from 44 biomarker unselected patients with progressive metastatic castration-resistant prostate cancer, 17 of which were enrolled in the Phase 1b dose escalation portion of the study. Eligibility criteria for the trial included patients who progressed on at least one prior ARPI while patients who were treated with prior chemotherapy in the castration-resistant setting were excluded. Over 60% of the patients progressed on two or more prior ARPIs, which included prior enzalutamide treatment. The primary endpoint of the escalation phase was assessment of dose-limiting toxicities (DLT) and determination of the maximum tolerated dose and recommended dose for the Phase 2 portion of the study – which was determined to be 2.1 mg/kg of FG-3246 and 160 mg/day of enzalutamide. The primary endpoint of the Phase 2 expansion portion of the study was composite response rate (PSA50 response and/or objective response per RECIST v1.1). Secondary endpoints were PSA50 response rate, objective response rate, radiographic progression free survival (rPFS), overall survival, and treatment-related adverse events (TRAEs). FG-3246 combined with enzalutamide demonstrated anti-tumor activity with a composite response rate of 21% in the overall cohort and 40% in patients who had progressed on only one prior ARPI. Median rPFS of 7.0 months was observed in the overall cohort. Notably, median rPFS of 10.1 months was observed in patients who had progressed on only one prior ARPI, a result which was consistent across the different prior ARPIs administered. Additionally, higher tumor uptake of FG-3180 demonstrated a trend towards higher probability of PSA50 response (p=0.053), highlighting the potential of FG-3180 as a biomarker for patient selection. Combination therapy of FG-3246 and enzalutamide demonstrated a similar safety pro was observed in the previous Phase 1 monotherapy trial of FG-3246. Neutropenia risk was successfully mitigated with use of G-CSF prophylaxis. The most frequent TRAEs with the combination therapy included fatigue, peripheral neuropathy, anorexia, and dysgeusia. Cumulative toxicities, including peripheral neuropathy, led to treatment discontinuation for some patients. The poster presentation, titled “A phase 1b/2 study of FOR46 (FG-3246) in combination with enzalutamide (enza) in patients with metastatic castration resistant prostate cancer (mCRPC)”, is scheduled for the poster session taking place on February 26, 2026 from 11:30 AM to 12:45 PM PT. FG-3246 is currently being evaluated in a Phase 2 monotherapy trial with interim data expected in the second half of 2026. The trial also includes treatment with FG-3180, a CD46-directed PET imaging agent, which will measure expression levels of CD46 positive lesions. This will enable further assessment of the correlation between CD46 expression and response to FG-3246 and the potential of FG-3180 to serve as a biomarker to aid in patient selection in future trials of FG-3246. About FG-3246 and FG-3180 FG-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 Kyntra Bio 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-3180 is a companion diagnostic PET imaging agent, using the same CD46-targeting antibody together with an 89 Zr tracker. To date, FG-3180 demonstrated specific uptake in CD46 positive tumors and is currently being evaluated as a biomarker for its potential to inform patient selection. About the Phase 1b/2 Study of FG-3246 in Combination with Enzalutamide This Phase 1b/2 study is an investigator-sponsored trial being conducted at the University of California San Francisco to evaluate FG-3246 (FOR46) in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) after prior progression on at least one androgen receptor pathway inhibitor. The primary objective for the Phase 1b portion of the study is to determine the maximally tolerated dose (MTD) and recommended Phase 2 dose of FG-3246 in combination with enzalutamide in patients with mCRPC. The objectives of the Phase 2 portion of the study are to determine the composite response rate (CRR), proportion of participants with a greater than or equal to 50% change in prostate specific antigen (PSA50), objective response rate (ORR), median duration of response, median radiographic progression free survival (rPFS), and median overall survival (OS) of patients treated with FG-3246 in combination with enzalutamide. For more information about this study, which is currently enrolling, please visit ( NCT05011188 ). About Kyntra Bio Kyntra Bio is a biopharmaceutical company focused on development of novel therapies in oncology and rare disease. Roxadustat (爱瑞卓 ® , EVRENZO™) is currently approved in 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 the development plan for the Phase 3 trial of roxadustat in anemia associated with lower-risk myelodysplastic syndromes (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase 2 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 . Forward-Looking Statements This release contains forward-looking statements regarding Kyntra Bio’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of its partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, and potential clinical or commercial success of Kyntra Bio products and product candidates, statements regarding the potential effectiveness of FG-3180 as a biomarker or predictor of response rate, and any forward looking statements regarding the design, efficacy or safety results of our ongoing Phase 2 monotherapy trial of FG-3246. 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. Kyntra Bio’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 Kyntra Bio’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 Kyntra Bio undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia, CFA Senior Vice President and Chief Financial Officer ir@kyntrabio.com

Phase 1ASCOClinical ResultPhase 2ADC

07 Jan 2026

·fibrogen.gcs-web.com

FibroGen Rebrands as Kyntra Bio to Reflect a New Era of Focus and Momentum

Name change to Kyntra Bio reflects the newly sharpened focus of the Company on novel therapies with potential for outsized impact in oncology and rare disease Kyntra Bio will begin trading on Nasdaq under the trading symbol “KYNB” effective January 8th SAN FRANCISCO, Jan. 07, 2026 (GLOBE NEWSWIRE) -- FibroGen, Inc. (Nasdaq: FGEN), today announced it is rebranding the company to Kyntra Bio, representing the next step of the transformation of the Company and its focus on oncology and rare disease assets. The Company’s common stock will begin trading under the new Nasdaq symbol “KYNB” at stock market open on January 8, 2026. “2025 was a transformational year, highlighted by the sale of FibroGen China, the payoff of our senior secured term loan, and the extension of our cash runway into 2028. We begin this year as Kyntra Bio, a name that captures our company journey and evolution and reflects our purposeful move to a company obsessed with creating outsized impact for patients and shareholders,” said Thane Wettig, Chief Executive Officer of Kyntra Bio. “Today, with a sharpened direction, Kyntra Bio is laser-focused on our mid- and late-stage assets – specifically, FG-3246, our first-in-class, CD46 targeting antibody drug conjugate, and FG-3180, our companion PET imaging agent, currently in a Phase 2 monotherapy trial in prostate cancer, and roxadustat, our Phase 3 ready asset, for which we recently received Orphan Drug Designation in myelodysplastic syndromes. We are thrilled to move forward with renewed purpose into this bold era for our company and are excited for what is in front of us.” Recent Highlights and Upcoming Milestones FG-3246 (CD46 Targeting ADC) and FG-3180 (CD46 Targeting PET Imaging Agent) Topline results from the investigator-sponsored Phase 1b/2 study, conducted by UCSF, of FG-3246 in combination with enzalutamide in patients with mCRPC are expected to be presented at ASCO GU in the first quarter of 2026. Interim results from the recently commenced Phase 2 monotherapy trial are expected in the second half of 2026. The trial will also assess the diagnostic performance of FG-3180 to determine the potential correlation between CD46 expression and response to FG-3246. Roxadustat Granted Orphan Drug Designation from the FDA for the treatment of myelodysplastic syndromes (MDS). Submitted the pivotal Phase 3 clinical trial protocol for roxadustat for the treatment of anemia in patients with lower-risk MDS and high transfusion burden to the U.S. Food and Drug Administration. In addition to the new name, the Company is refreshing its corporate website to better reflect the Company’s strategy moving forward. The CUSIP number for the Company’s common stock is not affected by the name change. About Kyntra Bio Kyntra Bio is a biopharmaceutical company focused on development of novel therapies in oncology and rare disease. Roxadustat (爱瑞卓®, EVRENZO™) is currently approved in 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 the development plan for the Phase 3 trial of 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 Phase 2 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.kyntrabio.com. Forward-Looking Statements This release contains forward-looking statements regarding Kyntra Bio’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, and potential clinical or commercial success of Kyntra Bio products and product candidates, statements under the caption “Recent Highlights and Upcoming Milestones”, statements about regulatory interactions, the payoff of the Morgan Stanley Tactical Value term loan, statements regarding cash, such as the expectation that cash, cash equivalents and accounts receivable will be sufficient to fund Kyntra Bio’s operating plans into 2028, and statements about Kyntra Bio’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. Kyntra Bio’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 Kyntra Bio’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 Kyntra Bio undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia, CFA Senior Vice President and Chief Financial Officer ir@kyntrabio.com Source: FibroGen, Inc.

Orphan DrugPhase 2Drug ApprovalPhase 3ADC

100 Deals associated with Roxadustat

External Link

KEGG	Wiki	ATC	Drug Bank
D10593	Roxadustat	B03XA05	DB04847

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Anemia	United Kingdom	Astellas Pharma, Inc.	01 Dec 2021
Anemia in chronic kidney disease	South Korea	AstraZeneca PLC	09 Jul 2021
Anemia of renal disease	Japan	Astellas Pharma, Inc.	20 Sep 2019
chronic renal failure anemia	China	FibroGen (China) Medical Technology Development Co. Ltd.	17 Dec 2018

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Neoplasms	Phase 3	China	Kyntra Bio, Inc.	16 Mar 2022
Myelodysplastic Syndromes	Phase 3	Belgium	AstraZeneca PLC	04 Sep 2017
Myelodysplastic Syndromes	Phase 3	Belgium	Kyntra Bio, Inc.	04 Sep 2017
Peritoneal Diseases	Phase 3	Japan	Astellas Pharma, Inc.	22 Jun 2016
Peritoneal Diseases	Phase 3	Japan	Kyntra Bio, Inc.	22 Jun 2016
Chronic kidney disease, Stage V	Phase 3	Slovakia	AstraZeneca AB	10 Dec 2014
Autoimmune Haemolytic Anaemias	Phase 3	Estonia	Kyntra Bio, Inc.	09 Nov 2013
Kidney Failure, Chronic	Phase 3	Estonia	Kyntra Bio, Inc.	09 Nov 2013
Chronic Kidney Diseases	Phase 3	United States	Kyntra Bio, Inc.	05 Nov 2012
Chronic Kidney Diseases	Phase 3	United States	AstraZeneca PLC	05 Nov 2012

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ChiCTR2100044799 (NANLING, ASN2025)	Phase 4	Anemia	195	Roxadustat	dlojfnpozz(vxnoqeahsy) = decreased from baseline (-81.2 μg/L [95%CI -112.9 to -49.6]) fccovgzjge (trbfwjaugc ) View more	Positive	07 Nov 2025
NCT04408820 (Pubmed \| Expert Opin Pharmacother)	Not Applicable	Anemia in chronic kidney disease C-reactive protein \| albumin	2,084	roxadustat (Non-dialysis-dependent group)	zgpadiulor(fwvdcvoceh) = kszrkselne bgjlxwcisb (uwphmoutve ) View more	Positive	04 Mar 2025
				roxadustat (Hemodialysis group)	zgpadiulor(fwvdcvoceh) = hwyvonmshc bgjlxwcisb (uwphmoutve ) View more
NCT05301517 (Pubmed \| J Clin Oncol)	Phase 3	Anemia	159	Roxadustat	ckhdgptugd(ksztgeacsk) = innworgrst gfyrkjqgew (jjegzjxmby, 13.58 - 20.71)	Positive	10 Jan 2025
				Recombinant human erythropoietin-α (rHuEPO-α)	ckhdgptugd(ksztgeacsk) = oaluhsygwd gfyrkjqgew (jjegzjxmby, 11.34 - 19.50)
NCT04059913 (ASN2024)	Phase 4	-	178	Roxadustat QW	ilqomynise(qpdbwbxnff) = pueigpkvuu bvyckgktca (yedhxjsfzq, 103.6 - 108.9) View more	Non-inferior	24 Oct 2024
				Roxadustat BIW	ilqomynise(qpdbwbxnff) = anmarufvth bvyckgktca (yedhxjsfzq, 104.4 - 109.7) View more
NCT04059913 (ASN2024)	Phase 4	Anemia in chronic kidney disease	178	Roxadustat QW	vbjoqburlp(iawlshewfk) = zhzyqnrhdc uvrknevusp (ipnrlfufqq )	-	24 Oct 2024
				Roxadustat BIW	vbjoqburlp(iawlshewfk) = nqxxoffuen uvrknevusp (ipnrlfufqq )
NCT03263091 (MATTERHORN, CTgov)	Phase 3	Anemia \| Myelodysplastic Syndromes	184	Roxadustat (OL Component (Cohort 1): Roxadustat 1.5 mg/kg)	donqebqgll = caueurtvfq qntbpadbhm (oclvrexthn, vdbnnylpna - ubyjvpgxjl) View more	-	01 Aug 2024
				Roxadustat (OL Component (Cohort 2): Roxadustat 2.0 mg/kg)	donqebqgll = gyepslucwo qntbpadbhm (oclvrexthn, rojdpgwmoq - dazgojzuph) View more
NCT04655027 (CTgov)	Phase 4	Anemia in chronic kidney disease	25	Roxadustat (Roxadustat)	pymediujon(uznhyrcoof) = mjotvshjrs mxfgaimwgo (nwfpklmzmv, 28.1506) View more	-	22 Apr 2024
				rHuEPO (rHuEPO)	pymediujon(uznhyrcoof) = pwtnifcykz mxfgaimwgo (nwfpklmzmv, 9.7369) View more
NCT02780141 \| NCT02952092 \| NCT02779764 \| NCT02780726 (Pubmed \| Adv Ther)	Phase 3	Chronic Kidney Diseases transferrin saturation \| transferrin level \| roxadustat dose ... View more	444	Roxadustat	vxsqeryvgf(cgjfiesrxu) = hwshivhdbc vgtbazszmf (nuielbosnr )	Negative	01 Apr 2024
NCT02174731 \| NCT02052310 \| NCT02278341 \| NCT02273726 (Pubmed)	Phase 3	Chronic Kidney Diseases hemoglobin level \| transferrin saturation	2,354	Roxadustat	wbfpagixay(mjdvyystxk) = high hemoglobin rate of rise (≥ 0.5 g/dL/week; odds ratio [OR] 2.09; 95% confidence interval [CI] 0.98-4.46) showed a trend towards increased risk of thromboembolic events before week 12 yxeorwmrxx (nnigvgfvpu ) View more	-	01 Apr 2024
NCT03263091 (MATTERHORN, GlobeNewswire) Manual	Phase 3	Myelodysplastic Syndromes	62	Roxadustat (higher transfusion burden)	ycmwuodhwh(ziqxgxcwxk) = dhzvfcldlb clbtvgsmmn (pwhxwrnvkl, 20.8 - 53.8) View more	Positive	09 Dec 2023
				placebo (higher transfusion burden)	ycmwuodhwh(ziqxgxcwxk) = hdadtklcoj clbtvgsmmn (pwhxwrnvkl, 2.4 - 30.2) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis