FibroGen advances roxadustat for anemia in myelodysplastic syndromes

Published 07/08/2025, 12:06
FibroGen advances roxadustat for anemia in myelodysplastic syndromes

SAN FRANCISCO - FibroGen Inc. (NASDAQ:FGEN), currently trading at $7.11 with a market capitalization of $28.7 million, has reached an agreement with the FDA on key design elements for a pivotal Phase 3 clinical trial of roxadustat for treating anemia in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high red blood cell transfusion burden. According to InvestingPro analysis, the company faces significant financial challenges with weak overall health scores.

The company plans to file the Phase 3 protocol in the fourth quarter of 2025, following positive feedback from a Type C meeting with the FDA. The decision was based on post-hoc analysis of data from the MATTERHORN Phase 3 trial, which showed that 36% of patients with high transfusion burden achieved transfusion independence for at least 56 days on roxadustat compared to 7% on placebo. InvestingPro data reveals the company is quickly burning through cash with a debt burden of $91.2 million, which could impact trial funding capabilities.

The planned Phase 3 trial will evaluate roxadustat in approximately 200 patients with LR-MDS in a randomized, double-blind, placebo-controlled study. The patient population will include those requiring at least 4 packed red blood cell units in two consecutive 8-week periods who are refractory to, intolerant to, or ineligible for prior erythropoiesis-stimulating agents therapy.

"This indication, despite recent approvals, still represents a patient population with significant unmet need," said Thane Wettig, Chief Executive Officer of FibroGen, in the press release.

Roxadustat is an oral medication that promotes red blood cell production through increased endogenous production of erythropoietin. It is already approved in China, Europe, Japan, and other countries for treating anemia in chronic kidney disease patients.

Anemia affects approximately 80% of patients with MDS at diagnosis, with around 60% experiencing severe anemia during their disease course. About 50% of MDS patients require regular red blood cell transfusions, which can lead to complications including iron overload, cardiac events, and infections. While the market opportunity appears significant, FibroGen’s financial metrics show concerning trends, with revenue declining 80.6% in the last twelve months. Get deeper insights into FibroGen’s financial health and 10+ additional ProTips with a subscription to InvestingPro.

The company is considering either 8-week or 16-week red blood cell transfusion independence as the primary endpoint for the study.

In other recent news, FibroGen Inc. reported a notable decrease in revenue for the first quarter of 2025, with earnings falling to $2.7 million compared to $25.4 million in the same period the previous year. Despite this revenue drop, the company managed to reduce its net loss, which was positively received by the market. Additionally, FibroGen has made strategic divestment plans and is seeing promising developments in its product pipeline. The company has also amended its financing agreement with Morgan Stanley Tactical Value and Wilmington Trust National Association, lowering the minimum cash balance requirement from $22.5 million to $18.75 million. This amendment is part of an ongoing effort to adjust financial covenants related to its cash holdings. Furthermore, FibroGen has expanded its Board of Directors by appointing Dr. Michael Kauffman, a biotechnology veteran with extensive experience in oncology therapeutics. Dr. Kauffman’s appointment is expected to bolster the company’s efforts in advancing its drug development pipeline. These developments are part of FibroGen’s strategy to navigate the current financial landscape and enhance its focus on oncology.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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