Disc Medicine Initiates a Phase 1b/2 Clinical Study of DISC-0974 in Adults with Non-Dialysis Dependent Chronic Kidney Disease (NDD-CKD) and Anemia
February 16 2023 - 7:00AM
Disc Medicine, a clinical-stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
treatments for patients suffering from serious hematologic
diseases, announced today the initiation of a Phase 1b/2 clinical
study of DISC-0974 in non-dialysis dependent chronic kidney disease
(NDD-CKD) patients with anemia. DISC-0974 is a monoclonal antibody
designed to suppress hepcidin by inhibiting the hemojuvelin (HJV)
co-receptor, and thereby address anemia by enhancing the
availability of iron for erythropoiesis. DISC-0974 is also
currently being studied in a Phase 1b/2 clinical study in patients
with myelofibrosis and anemia.
“We are excited to initiate this clinical trial of DISC-0974 in
chronic kidney disease, where hepcidin plays a key role in the
pathophysiology of anemia. There is a tremendous need for
innovative therapies that work through mechanisms outside of the
erythropoietin pathway,” said John Quisel, JD, PhD, Chief Executive
Officer at Disc Medicine. “We believe DISC-0974 has potential
across a broad range of chronic and inflammatory diseases. With the
start of this study, we now have ongoing clinical trials for
DISC-0974 in both chronic kidney disease and myelofibrosis, and
plan to explore its use in other indications as well.”
The study will be a multi-center, Phase 1b/2 trial and will
evaluate the safety, tolerability, and efficacy of DISC-0974 in
NDD-CKD patients with anemia. The study endpoints will include
hepcidin levels, serum iron and markers of iron mobilization and
changes in hemoglobin. The study enrollment criteria will include
patients with Stage II-V CKD not receiving dialysis, baseline Hb
< 11.0 g/dL for males and Hb < 10.5 g/dL for females, and
those not receiving concurrent treatment with
erythropoietin-stimulating agents (ESAs). The study will be
conducted in two parts:
- Phase 1b (Dose-Escalation):
Randomized, double-blind, placebo-controlled study design; single,
ascending doses of DISC-0974 will be administered subcutaneously at
the following planned dose levels: 28 mg, 40 mg, 60 mg, 90 mg;
safety, PK and hematologic effects will be assessed at each dose
level; a dose for the expansion phase will be selected based on
optimal increases in hemoglobin;
- Phase 2 (Expansion Stage):
Open-label, single-arm study design; multiple doses of DISC-0974
administered subcutaneously, once-monthly at the dose level
selected from the phase 1b portion of the study for three
months.
About DISC-0974
DISC-0974 is an investigational monoclonal antibody (mAb)
targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and
is designed to suppress hepcidin production and increase serum iron
levels in patients suffering from anemia of inflammation. DISC-0974
was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation
arises from abnormally elevated hepcidin and is the most common
form of anemia, affecting millions of patients in the US across
numerous diseases such as chronic kidney disease, myelofibrosis,
cancer, autoimmune diseases, and other conditions with an
inflammatory component. Disc has established clinical
proof-of-mechanism of DISC-0974 in a Phase 1 trial of healthy
volunteers and initiated a Phase 1b/2a clinical trial of DISC-0974
in patients with myelofibrosis and anemia, as well as a Phase 1b/2a
clinical trial of DISC-0974 in patients with chronic kidney disease
and anemia who are not receiving dialysis.
DISC-0974 is an investigational agent and is not approved for
use as a therapy in any jurisdiction worldwide.
About Anemia of Chronic Kidney Disease
(CKD)
Chronic kidney disease (CKD) is a global, widespread disease
characterized by progressive loss of kidney function and may lead
to end-stage renal disease (ESRD) or kidney failure. CKD affects
over 37 million patients in the United States and an estimated 700
million patients worldwide. Anemia is a serious and frequent
complication of CKD, as patients are unable to produce sufficient
red blood cells and hemoglobin. It affects approximately 5-6
million patients in the U.S. alone, may results in fatigue,
shortness of breath, and reduced physical and cognitive function,
and can be associated with a higher risk of mortality,
hospitalization and other complications. Elevated hepcidin is a
primary cause of anemia in CKD patients and prevents erythropoiesis
by depriving developing red blood cells of iron. Hepcidin
accumulates at high levels in CKD patients because its production
is stimulated by inflammation and its clearance is reduced as a
consequence of impaired renal function. The majority of CKD
patients do not receive any treatment for their anemia due to the
complexity of outpatient administration and potential safety
concerns related to the current treatments. In severe cases,
patients may receive blood transfusions, but use may lead to
increased administrative burden, safety risks and the potential for
immune sensitization which precludes eligibility for kidney
transplantation.
About Disc Medicine
Disc Medicine (NASDAQ:IRON) is a clinical-stage
biopharmaceutical company committed to discovering, developing, and
commercializing novel treatments for patients who suffer from
serious hematologic diseases. We are building a portfolio of
innovative, first-in-class therapeutic candidates that aim to
address a wide spectrum of hematologic diseases by targeting
fundamental biological pathways of red blood cell biology,
specifically heme biosynthesis and iron homeostasis. For more
information, please visit www.discmedicine.com.
Disc Cautionary Statement Regarding Forward-Looking
Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to, express or implied statements
regarding Disc’s expectations with respect to its Phase 1b/2
clinical study of DISC-0974 in NDD-CKD patients with anemia and
projected timelines for the initiation and completion of its
clinical trials and other activities. The use of words such as, but
not limited to, “believe,” “expect,” “estimate,” “project,”
“intend,” “future,” “potential,” “continue,” “may,” “might,”
“plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the
negative of these terms and other similar words or expressions that
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Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on Disc’s
current beliefs, expectations and assumptions regarding the future
of Disc’s business, future plans and strategies, clinical results
and other future conditions. New risks and uncertainties may emerge
from time to time, and it is not possible to predict all risks and
uncertainties. No representations or warranties (expressed or
implied) are made about the accuracy of any such forward-looking
statements.
Disc may not actually achieve the plans, intentions or
expectations disclosed in these forward-looking statements, and
investors should not place undue reliance on these forward-looking
statements. Actual results or events could differ materially from
the plans, intentions and expectations disclosed in the
forward-looking statements as a result of a number of material
risks and uncertainties including but not limited to: the adequacy
of Disc’s capital to support its future operations and its ability
to successfully initiate and complete clinical trials; the nature,
strategy and focus of Disc; the difficulty in predicting the time
and cost of development of Disc’s product candidates; Disc’s plans
to research, develop and commercialize its current and future
product candidates; the timing of initiation of Disc’s planned
preclinical studies and clinical trials; the timing of the
availability of data from Disc’s clinical trials; Disc’s ability to
identify additional product candidates with significant commercial
potential and to expand its pipeline in hematological diseases; the
timing and anticipated results of Disc’s preclinical studies and
clinical trials and the risk that the results of Disc’s preclinical
studies and clinical trials may not be predictive of future results
in connection with future studies or clinical trials and may not
support further development and marketing approval; the other risks
and uncertainties described in the “Risk Factors” section of the
Current Report on Form 8-K filed with the SEC on December 29, 2022
and other documents filed by Disc from time to time with the SEC,
as well as discussions of potential risks, uncertainties, and other
important factors in Disc’s subsequent filings with the Securities
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as of the date on which it was made. None of Disc, nor its
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Media Contact
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Communicationsprusconi@vergescientific.com
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Relationssuzanne.messere@sternir.com
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