• A post hoc analysis of patients with higher transfusion burden at baseline showed a larger percentage of patients achieved transfusion independence ≥ 56 days with roxadustat vs. placebo (36.1% vs 11.5%; p=0.047^ǂ).
• Dr. Moshe Mittelman’s presentation was selected for the “2024 Highlights of ASH”

SAN FRANCISCO, Dec. 09, 2023 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) announced today the presentation of efficacy and safety data from MATTERHORN, a Phase 3 clinical study of roxadustat for the treatment of anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes (MDS) at the 65^th American Society of Hematology (ASH) Annual Meeting and Exposition being held in San Diego, California December 9 - 12, 2023. The oral presentation was selected for the “2024 Highlights of ASH”.

“We are excited to have our data selected for presentation as part of the 2024 Highlights of ASH. We believe that roxadustat could represent an important new therapy for patients with higher transfusion burden low-risk-MDS,” said Thane Wettig, Chief Executive Officer, FibroGen. “There continues to be a significant unmet need in this patient population, and the novel mechanism of roxadustat delivered orally three times a week could represent an important new and convenient treatment alternative.”

As previously disclosed, the initial analysis with all the patients who participated in the trial, showed that more patients receiving roxadustat achieved transfusion independence vs. placebo (48% vs. 33%; p=0.22). While the primary endpoint of transfusion independence (TI) for ≥ 56 consecutive days within the first 28 weeks of the study was not met, a post-hoc analysis, showed that roxadustat performed significantly better^ǂ than placebo in the subset of patients having higher transfusion burden^ǂǂ (see table). The TI for ≥56 days within the first 28 weeks of the study was 36.1% for the roxadustat group and 11.5% for the placebo group (p=0.047^ǂ), and 44.4% vs 19.2% at the end of trial, respectively. Additionally, in TI responders, more patients in the roxadustat arm vs. placebo had hemoglobin concentration increases of ≥1.5 g/dL: 45.5% vs 17.4% (p=0.004)^ǂ.

^ǂNominal statistical significance

^ǂǂHigher transfusion burden defined as ≥2 pRBC units Q4W

“The results seen from roxadustat in the subset of patients having higher transfusion burden are intriguing,” said Moshe Mittelman M.D., Professor of Medicine and Hematology at Tel Aviv Sourasky Medical Center and MATTERHORN principal investigator. “Based on these results, roxadustat may be an option in anemia of low risk-MDS which can further be explored in a confirmatory study focused on higher burden transfusion-dependent patients. There is a significant opportunity in this patient population for an effective oral treatment such as roxadustat, and if approved it would be an important advancement for the field.”

Roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, was generally well tolerated, with treatment emergent adverse events of any grade similar across treatment groups.  Overall, the adverse event profile that was observed during the double-blind portion of the study was generally consistent with previous findings in MDS patients.

Presentation Details

About MATTERHORN

A total of one-hundred forty-one (141) subjects were enrolled in MATTERHORN (NCT03263091) a Phase 3, double-blind placebo-controlled study investigating the efficacy and safety of roxadustat for the treatment of anemia in patients with lower-risk transfusion-dependent myelodysplastic syndromes. The primary endpoint of the study is transfusion independence for ≥ 56 consecutive days in the first 28 weeks of treatment. The main secondary endpoint is the reduction of red blood cell transfusion.

About Myelodysplastic Syndromes Anemia

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.S¹, thereof 77% are considered lower-risk MDS². Approximately 80% of MDS patients have anemia at the time of diagnosis³ and around 60% of MDS patients will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease⁴. Anemia in MDS patients is associated with increased risk of cardiovascular complications and the need for blood transfusion⁵. Approximately 50% of patients with MDS require regular red blood cell transfusions⁶. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections, iron overload with the related complications, and transformation to acute leukemia, associated with a decreased overall survival rate when compared with non-transfused patients with MDS, and decreased survival compared to an age-matched elderly population⁷. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. There remains high unmet need for the treatment of anemia associated with MDS. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease⁸. Current anti-anemic agents are effective in about half of patients, with approximately 2 years duration of response⁹.

About Roxadustat

Roxadustat, an oral HIF-PH inhibitor that promotes erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improving 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 chronic kidney disease (CKD) in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca, to regulatory authorities across the globe. Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in markets not licensed to Astellas.

About FibroGen

FibroGen, Inc. is a biopharmaceutical company committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics. The Company applies its pioneering expertise in connective tissue growth factor (CTGF) biology and hypoxia-inducible factor (HIF) to advance innovative medicines for the treatment of unmet needs. Pamrevlumab, an anti-CTGF fully-human monoclonal antibody, is in clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC) and metastatic pancreatic cancer. Roxadustat (爱瑞卓^®, EVRENZO^TM) is currently approved in China, Europe, Japan, and numerous other countries for the treatment of anemia in 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 by the China Health Authority. FibroGen recently expanded its research and development portfolio to include novel product candidates in oncology. 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 the development and commercialization of the company’s product candidates, the potential safety and efficacy profile of its product candidates, and its clinical programs. These forward-looking statements include, but are not limited to, statements about FibroGen’s plans and objectives and typically are 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, 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.

References:

1. Cogle et al., Curr Hematol Malig Rep. 2015 Sep;10(3):272-81.
2. Greenberg et al., Blood. 1997; 89:2079-2088.
3. Gattermann et al., Onkologie. 2012;35(6):350-6.
4. Bennett, Am. J. Hematol. 83:858–861, 2008.
5. Oliva et al., Am J Blood Res 2011;1(2):160-166.
6. Heptinstall K. Leuk Res. 2007;31(suppl 1):107
7. Lewis et al., Cancer Management and Research 2021:13 645–657.
8. Platzbecker U, Blood. 2019 Mar 7;133(10):1020-1030.
9. Carraway and Saygin, Hem Am Soc Hematol Educ Prog. 2020 (1): 426–433.

Contacts:
FibroGen, Inc.

Investors:
David DeLucia, CFA
Vice President of Corporate FP&A / Investor Relations
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Media:
Meichiel Keenan
Director, Investor Relations and Corporate Communications
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