FibroGen (FGEN) to Present Safety and Efficacy Analyses from Roxadustat Global Phase 3 Program at ASH

FibroGen, Inc. (Nasdaq: FGEN) and its partner, AstraZeneca (LSE/STO/Nasdaq: AZN), will present additional analyses of roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndromes (MDS) and results from multiple roxadustat phase 3 studies. Roxadustat is a first-in-class oral small molecule hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) in development for the treatment of patients with anemia of CKD and anemia secondary to lower-risk MDS. FibroGen and its partner will present eight abstracts at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually December 5-8, 2020.

“We are pleased to share additional 52-week data from the open-label portion of our Phase 3 anemia of MDS trial with the medical community at ASH,” said Enrique Conterno, Chief Executive Officer, FibroGen. “In addition, we are presenting Phase 3 cardiovascular safety and efficacy results of roxadustat, which highlight its potential in a broad range of CKD patients.”

MDS is a group of blood disorders characterized by poorly formed or dysfunctional blood cells, resulting in chronic anemia.1 Exploratory analyses to be presented at ASH show the efficacy of roxadustat in transfusion-dependent lower-risk MDS patients regardless of ring sideroblast and baseline erythropoietin status - characteristics used to predict response to treatment.2 Patients with ring sideroblasts (RS+), without ring sideroblasts (RS-), baseline erythropoietin (BL EPO) ≤ 200 mIU/ml, and BL EPO > 200 mIU/ml achieved the primary endpoint of transfusion independence for ≥ 8 weeks during the first 28 treatment weeks (23% of MDS-RS+, 55% of MDS-RS-, 39% of BL EPO ≤ 200 mIU/ml, and 33% of BL EPO > 200 mIU/ml patients). Detailed results will be presented at the meeting.

Enrollment in the double-blind placebo-controlled portion of the Phase 3 roxadustat study of patients with anemia secondary to lower-risk MDS is ongoing (NCT03263091).

Roxadustat presentations during the 62nd ASH Annual Meeting and Exposition:

About Anemia of CKDChronic kidney disease (CKD) is generally a progressive disease characterized by gradual loss of kidney function that may eventually lead to kidney failure or end stage renal disease, requiring dialysis or kidney transplant. CKD is estimated to occur in approximately 10-12% of adults worldwide and is predicted to become the fifth most common cause of premature death globally by 2040.

Anemia, a serious medical condition in which patients have insufficient red blood cells and low levels of hemoglobin, is a common early complication of CKD, affecting approximately 20% of CKD patients. Anemia of CKD is associated with an increased risk of hospitalization, cardiovascular complications, and death, and can also cause significant fatigue, cognitive dysfunction and reduced quality of life. Blood transfusions are used for treating severe anemia, however, they may reduce a patient’s opportunity for kidney transplant and can increase the risk of infection and/or complications such as heart failure and allergic reactions.

About MDSMDS develops because the bone marrow cells do not develop into mature blood cells. Instead, these blood cells stay within the bone marrow in an immature state. There are many subtypes of MDS. Some cases are mild, while others are more severe, and carry a high risk of becoming acute myelogenous leukemia (AML). It is estimated that more than 10,000 patients are diagnosed with MDS each year in the U.S.,3 and overall prevalence is estimated to be between 60,000 – 170,000 in the country.4

About RoxadustatRoxadustat is a first-in-class orally administered inhibitor of HIF-PH, which increases hemoglobin levels through a mechanism of action that is different from that of traditional erythropoiesis-stimulating agents. As a HIF-PH inhibitor, roxadustat activates a response that occurs naturally when the body responds to reduced oxygen levels in the blood. Roxadustat promotes red blood cell production through increased endogenous production of erythropoietin; improved iron absorption, transport, and mobilization; and downregulation of hepcidin, which helps to overcome the negative impact of inflammation on hemoglobin synthesis and red blood cell production.

Roxadustat is approved and launched for the treatment of anemia of CKD in Japan and China in adult patients on dialysis (DD) and not on dialysis (NDD). A New Drug Application for the treatment of anemia of CKD in patients both DD and NDD is under review by the U.S. Food and Drug Administration with a decision expected in December 2020. The marketing authorization application for roxadustat for the treatment of anemia of CKD in patients both DD and NDD was accepted by the European Medicines Agency for review on May 21, 2020. Several other licensing applications for roxadustat have been submitted by Astellas and AstraZeneca to regulatory authorities across the globe, which are currently in 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 in the Americas and in Australia/New Zealand as well as Southeast Asia.

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