Study Did Not Meet Primary Endpoint
SAN FRANCISCO, May 05, 2023 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that MATTERHORN, a Phase 3 clinical study of roxadustat for treatment of anemia in patients with transfusion-dependent lower risk myelodysplastic syndromes (MDS) did not meet its primary efficacy endpoint. The proportion of patients who achieved red blood cell transfusion independence in the first 28 weeks was 47.5% for the roxadustat arm compared to 33.3% for placebo (p=0.217). The adverse event profile of roxadustat that was observed in the preliminary safety analysis was generally consistent with previous findings. Safety will be further evaluated at study completion.
A total of one-hundred forty (140) patients were enrolled in MATTERHORN, a Phase 3, double-blind placebo-controlled study investigating the safety and efficacy of roxadustat for 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 during the first 28 weeks of treatment, and patients are followed for up to 52 weeks. The MATTERHORN study is sponsored and conducted by FibroGen and is part of FibroGen’s co-development collaborations with AstraZeneca and with Astellas Pharma Inc.
About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by poorly formed or dysfunctional blood 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. Approximately 80% of MDS patients have anemia at the time of diagnosis2 and around 60% of MDS patients will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease3. Lower-risk MDS patients represent approximately 77% of the total diagnosed MDS population4. Anemia in MDS patients is associated with increased risk of cardiovascular complications and the need for blood transfusion5. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections and transformation to acute leukemia, and a decreased overall survival rate when compared with non-transfused patients with MDS, and decreased survival compared to an age-matched elderly population6. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Currently, there are few options available for treating anemia in MDS. Patients with MDS typically rely on repeated blood transfusions and administration of ESAs.
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 anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA).
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). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca to regulatory authorities across the globe, and are currently under review.
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 the U.S., China, and other markets not licensed to Astellas.
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 human monoclonal antibody, is in clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), metastatic pancreatic cancer, and Duchenne muscular dystrophy (DMD). Roxadustat (爱瑞卓®, EVRENZO™) 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 anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA). FibroGen recently expanded its research and development portfolio to include product candidates in the immuno-oncology space. For more information, please visit www.fibrogen.com.
This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, the development and commercialization of the company’s product candidates, the potential safety and efficacy profile of its product candidates, and the potential impact of clinical data. 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, as filed with the Securities and Exchange Commission (SEC) on February 27, 2023, 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.
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Michael Tung, M.D.
Corporate Strategy / Investor Relations
Investor Relations and Corporate Communications
Source: FibroGen, Inc.