FibroGen Announces Completion of Patient Enrollment in MATTERHORN, a Phase 3 Clinical Study of Roxadustat for the Treatment of Anemia in Patients with Lower Risk Transfusion-Dependent Myelodysplastic Syndromes (MDS)

Rhea-AI Summary

FibroGen has announced the successful completion of patient enrollment in the MATTERHORN, a Phase 3 clinical study evaluating roxadustat for treating anemia in 141 patients with lower risk transfusion-dependent myelodysplastic syndromes (MDS). This double-blind, placebo-controlled study aims to assess transfusion independence over 28 weeks, with top-line data expected in the first half of 2023. MDS affects over 80% of diagnosed patients, highlighting the need for effective treatment options. Roxadustat, already approved in several regions for anemia in chronic kidney disease, demonstrates potential in revising MDS treatment.

Positive

• Completion of enrollment in the Phase 3 MATTERHORN study with 141 patients.
• Roxadustat is being evaluated for a significant treatment indication in MDS.
• Top-line data from the study anticipated in 1H 2023, potentially advancing treatment options.

Negative

• None.

- 141 MDS Patients Enrolled -
- Topline Data Anticipated 1H:2023 -

SAN FRANCISCO, Aug. 26, 2022 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced completion of patient enrollment for MATTERHORN, a Phase 3 clinical study of roxadustat for treatment of anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes (MDS).

“We are very pleased to complete enrollment of this Phase 3 study of roxadustat for the treatment of anemia in patients with lower risk MDS,” said Mark Eisner, M.D, M.P.H, Chief Medical Officer, FibroGen. “Patients living with MDS have limited treatment options for their anemia, and MATTERHORN evaluates roxadustat as a potential new treatment that can reduce the burden of blood transfusions. On behalf of the entire study team, we would like to extend our gratitude to the patients, caregivers, investigators, and study staff for their commitment to this study.”

A total of one-hundred forty-one (141) subjects have been enrolled in MATTERHORN, a Phase 3, double-blind placebo-controlled study investigating the efficacy and safety 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 in the first 28 weeks of treatment. The main secondary endpoint is reduction of red blood cell transfusion. Top-line data from the MATTERHORN study is anticipated in 1H 2023. For more information about MATTERHORN please visit www.clinicaltrials.gov (NCT03263091).

About MDS 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.

About Roxadustat 
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. 

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 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^TM) is currently approved in China, Europe, Japan, and numerous other countries for the treatment of anemia in CKD patients on dialysis and patients not on dialysis. Roxadustat is in Phase 3 clinical development in the U.S. and Europe for anemia associated with myelodysplastic syndromes (MDS), and in Phase 3 clinical development in China for treatment of chemotherapy-induced anemia (CIA). FibroGen recently expanded its research and development portfolio to include product candidates in the immuno-oncology and autoimmune space. For more information, please visit www.fibrogen.com. 

Forward-Looking Statements  
This release contains forward-looking statements regarding the indications and potential markets we are pursuing our 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 our product candidates, and our clinical programs. These forward-looking statements include, but are not limited to, statements about our plans, objectives, representations and contentions and are not historical facts 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. 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 programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, 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 we undertake 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. Gattermann et al., Onkologie. 2012;35(6):350-6.
3. Bennett, Am. J. Hematol. 83:858–861, 2008.
4. Greenberg et al., Blood. 1997; 89:2079-2088.
5. Oliva et al., Am J Blood Res 2011;1(2):160-166.
6. Lewis et al., Cancer Management and Research 2021:13 645–657.

Contacts:
FibroGen, Inc.

Investors:
Michael Tung, M.D.
Corporate Strategy / Investor Relations
415.978.1434
mtung@fibrogen.com

Media:
Meichiel Keenan
Investor Relations and Corporate Communications
mkeenan@fibrogen.com

FAQ

What is the MATTERHORN study?

The MATTERHORN study is a Phase 3 clinical trial assessing the efficacy and safety of roxadustat for treating anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes (MDS).

What is the primary endpoint of the MATTERHORN study?

The primary endpoint is transfusion independence for 56 consecutive days within the first 28 weeks of treatment.

When will the results from the MATTERHORN study be available?

Top-line data from the MATTERHORN study is anticipated in the first half of 2023.

What is roxadustat and how does it work?

Roxadustat is an oral medication that promotes red blood cell production and can treat anemia in chronic kidney disease and myelodysplastic syndromes.

What implications does the MATTERHORN study have for FibroGen's market position?

Successful results from the MATTERHORN study could enhance FibroGen's position in the anemia treatment market, especially for patients with MDS.