Positive Results Published in Nephrology Dialysis Transplantation From Two China Phase 2 Trials of Roxadustat for Treatment of Anemia in Chronic Kidney Disease

Findings are Consistent with China Phase 3 Trials Results, and Support China NDA Submission

SAN FRANCISCO, March 30, 2017 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ:FGEN), a science-based biopharmaceutical company, today announced that clinical results from two Phase 2 studies in China of roxadustat for the treatment of anemia in patients with chronic kidney disease (CKD) were published in the journal Nephrology Dialysis Transplantation. In these studies, in dialysis-dependent, and in non-dialysis-dependent CKD patients, roxadustat corrected and maintained hemoglobin levels regardless of the patients’ baseline iron repletion status, and levels of C-reactive protein (CRP), a marker of inflammation, consistent with what was seen in previously published results from the roxadustat global program. The publication, entitled “Phase 2 studies of oral hypoxia-inducible factor prolyl hydroxylase inhibitor FG-4592 for treatment of anemia in China,” is now available online at Nephrol Dial Transplant 2017 gfx011. doi: 10.1093/ndt/gfx011.

“Chronic kidney disease is a major chronic health condition in China, and anemia is one of CKD’s most common complications. The publication of this Phase 2 clinical data, along with recently reported Phase 3 primary endpoint data from trials conducted in China, show consistent findings of roxadustat’s potential to treat for dialysis and non-dialysis CKD patients,” said lead investigator Nan Chen, M.D., Department of Nephrology, Shanghai Ruijin Hospital, Shanghai Jiao Tong University School of Medicine. “We are gratified to see this demonstration in China of the potential benefits of roxadustat, an oral anemia therapy.”

“We are pleased with the positive data that we have seen from our China clinical development program for roxadustat. The published results from our Phase 2 program continue to show that that roxadustat corrects and maintains hemoglobin levels, maintain iron stores, and decreases levels of hepcidin,” said Chris Chung, FibroGen’s Vice President of China Operations.

  • In the first study, 91 non-dialysis-dependent patients were dosed over eight weeks, according to two different weight-based dosing regimens. Hemoglobin levels were increased over baseline by more than 1.0 gm/dL in a significantly greater proportion of patients receiving roxadustat than patients receiving placebo (80% and 87.1% for the low- and high-dose roxadustat arms versus 23.3% in the placebo arm (p<0.0001, both)).
  • In the second study, a conversion study in 87 dialysis-dependent CKD patients who had previously been treated for anemia, roxadustat was dosed according to one of three weight-based dosing regimens and compared with epoetin alfa. In this study, a higher proportion of the roxadustat-treated subjects − 59.1%, 88.9% (p=0.008), and 100% (p=0.0003), randomized to low, medium, and high doses of roxadustat, respectively − maintained their hemoglobin levels after five and six weeks of treatment, as compared to 50% of the epoetin alfa-treated subjects.

Similar effects on iron metabolism were observed in each of the China Phase 2 studies, consistent with previously reported Phase 2 global trials. In particular, significant reductions in hepcidin levels were observed in roxadustat-treated subjects. Serum iron remained stable or increased in the roxadustat-treated arms, while total iron binding capacity (TIBC) and transferrin levels rose significantly.

About CKD Anemia in China
Anemia commonly develops in association with chronic kidney disease (CKD) and is linked to significant morbidity and mortality in both the dialysis and non-dialysis populations. CKD affects an estimated 119.5 million patients in China (Zhang et al. Lancet 2012; 379: 815–822). Although CKD may occur at any age, it is more common in aging populations, and its prevalence is increasing. CKD can be both a cause and a consequence of cardiovascular disease and is a critical healthcare issue. Currently, there is no treatment available that is curative, or has the ability to stop kidney deterioration in patients with CKD with the exception of kidney transplantation. 

The dialysis population in China, exceeding 400,000 patients, has been growing at a double-digit rate. The number of patients that require anemia therapy in China and other emerging markets is expected to increase steadily, as the CKD population continues to grow and the number of hemodialysis and peritoneal dialysis patients increases. We believe there is a significant opportunity for roxadustat to treat CKD anemia patients on dialysis (hemodialysis and peritoneal dialysis) and not on dialysis, as well as to address need in the large number of patients whose anemia remains undertreated or untreated in China.

About Roxadustat
Roxadustat (FG-4592) is a first-in-class, orally administered small molecule currently in global Phase 3 clinical development as a potential therapy for anemia associated with chronic kidney disease (CKD). Roxadustat is a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) that promotes erythropoiesis through increasing endogenous erythropoietin, improving iron regulation, and reducing hepcidin. Administration of roxadustat has been shown to induce coordinated erythropoiesis – increasing red blood cell count while maintaining plasma erythropoietin levels within or near normal physiologic range in multiple subpopulations of CKD patients – including in the presence of inflammation and without a need for supplemental intravenous iron. 

Roxadustat is currently advancing through Phase 3 clinical trials worldwide, supported by extensive Phase 2 clinical data demonstrating correction and maintenance of hemoglobin levels in multiple subpopulations of CKD anemia patients. To date, roxadustat has been evaluated in Phase 1 and Phase 2 studies, involving more than 1,400 subjects. Globally, a total of 15 Phase 3 studies, with target enrollment of about 10,000 patients worldwide, are currently being conducted to support independent regulatory approvals of roxadustat in both NDD-CKD and DD-CKD patients in the U.S., Europe, Japan, and China. Roxadustat is also entering a Phase 3 clinical trial in the U.S., and a Phase 2/3 clinical trial in China for the treatment of anemia in patients with myelodysplastic syndrome (MDS). For information about roxadustat trials currently recruiting patients, please visit www.clinicaltrials.gov.

About FibroGen, Inc.
FibroGen, Inc., headquartered in San Francisco with subsidiary offices in Beijing and Shanghai, is a leading science-based biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics. The company applies its pioneering expertise in fibrosis and hypoxia-inducible factor (HIF) biology and clinical development to advance innovative medicines for the treatment of anemia, fibrotic disease, and cancer. Roxadustat (FG-4592), the company’s most advanced product candidate, is an oral small molecule inhibitor of HIF prolyl hydroxylase activity in Phase 3 clinical development for the treatment of anemia in chronic kidney disease (CKD), and is entering Phase 3 development for anemia in myelodysplastic syndrome (MDS). Pamrevlumab (FG-3019), a fully-human monoclonal antibody that inhibits the activity of connective tissue growth factor (CTGF), is in Phase 2 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), pancreatic cancer, and Duchenne muscular dystrophy (DMD). FibroGen is also developing a biosynthetic cornea in China. For more information, please visit www.fibrogen.com.

Forward-Looking Statements
This release contains forward-looking statements regarding our strategy, future plans and prospects, including statements regarding the development of the Company's product candidate, roxadustat, the potential safety and efficacy profile of roxadustat, the potential for regulatory submissions, and our clinical plans. Our 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 our various non-clinical and clinical programs, including enrollment and conduct of our Phase 3 trials, the potential for Phase 2 results to be replicated or successful in larger Phase 3 trials, whether the results will support our planned NDA submission and the potential for approval in China, and our collaboration partners’ clinical trials for roxadustat in anemia associated with CKD, the continued progress of our plans and programs in China, and other matters that are described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2016, 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 we undertake no obligation to update any forward-looking statement in this press release, except as required by law.