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
65th 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 betterǂ
than placebo in the subset of patients having higher transfusion
burdenǂǂ (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=0.047ǂ), 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.
% (95% CI) |
Roxadustat (n=36) |
Placebo (n=26) |
Roxadustat vs Placebo |
TI for ≥56 days within 28 weeks |
36.1%(20.8–53.8) |
11.5%(2.4-30.2) |
OR: 3.823 (0.96–15.20), p=0.047ǂ |
TI for ≥56 days by EOT |
44.4% (27.9–61.9) |
19.2%(6.6–39.4) |
OR: 3.369 (1.01–11.19)p=0.048ǂ |
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
Presenter: |
Moshe
Mittelman M.D. |
Session: |
637. Myelodysplastic Syndromes – Clinical and Epidemiological:
Treatment Options and Decision Making in Low Risk MDS |
Time: |
Saturday, December 9, 2023: 2:00 PM-3:30 PM |
Location: |
Pacific Ballroom Salons 18-19 (Marriott Marquis San Diego
Marina) |
Abstract: |
Efficacy and Safety of Roxadustat for Treatment of Anemia in
Patients with Lower-Risk Myelodysplastic Syndrome (LR-MDS) with Low
Red Blood Cell (RBC) Transfusion Burden: Results of Phase III
Matterhorn Study |
|
|
About MATTERHORNA 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 RoxadustatRoxadustat, 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 FibroGenFibroGen, 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®, EVRENZOTM) 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 StatementsThis
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:
- Cogle et al., Curr Hematol Malig
Rep. 2015 Sep;10(3):272-81.
- Greenberg et al., Blood. 1997;
89:2079-2088.
- Gattermann et al., Onkologie.
2012;35(6):350-6.
- Bennett, Am. J. Hematol.
83:858–861, 2008.
- Oliva et al., Am J Blood Res
2011;1(2):160-166.
- Heptinstall K. Leuk Res.
2007;31(suppl 1):107
- Lewis et al., Cancer Management and
Research 2021:13 645–657.
- Platzbecker U, Blood. 2019 Mar
7;133(10):1020-1030.
- Carraway and Saygin, Hem Am Soc
Hematol Educ Prog. 2020 (1): 426–433.
Contacts:FibroGen,
Inc.
Investors:David DeLucia,
CFAVice President of Corporate FP&A / Investor
RelationsInvestorRelations@fibrogen.com
Media:Meichiel KeenanDirector,
Investor Relations and Corporate
Communicationsmedia@fibrogen.com
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