Disc Medicine Receives FDA Fast Track Designation for DISC-0974 for the Treatment of Anemia in Non-Dialysis Dependent Chronic Kidney Disease
20 Fevereiro 2024 - 10:30AM
Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage
biopharmaceutical company focused on the discovery, development,
and commercialization of novel treatments for patients suffering
from serious hematologic diseases, today announced that the United
States Food and Drug Administration (FDA) has granted Fast Track
Designation to DISC-0974 for the treatment of patients with
non-dialysis dependent chronic kidney disease (NDD-CKD) and anemia.
“Receiving Fast Track designation highlights the
unmet need for the millions of NDD-CKD patients with anemia, as
well as the potential of DISC-0974 to address this need,” said John
Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc.
“We believe DISC-0974 could be a transformative therapy for these
patients and are excited to share additional results from our
ongoing Phase 1b/2 study in NDD-CKD patients with anemia this
year.”
Fast Track is a process designed by the FDA to
facilitate the development and expedite the review of
investigational drugs intended to treat serious conditions and for
which nonclinical or clinical data demonstrate the potential to
address unmet medical need. A therapeutic candidate that receives
Fast Track designation may be eligible for more frequent
interactions with the FDA to discuss the candidate’s development
plan. Therapeutic candidates with Fast Track designation may also
be eligible for priority review and accelerated approval if
supported by clinical data.
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 affects 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 has ongoing
clinical studies of DISC-0974 in patients with myelofibrosis and
anemia and also in patients with NDD-CKD and anemia.
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 result 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 transfusions require
significant healthcare utilization, incur safety risks and increase
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, potentially 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, projected timelines for the initiation and completion of
its clinical trials, anticipated timing of release of data, and
other clinical activities; Disc’s business plans and objectives;
and Disc’s beliefs about operating expenses and that it will have
capital to fund Disc well into 2026. 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
are intended to identify forward-looking statements.
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 our
Annual Report on Form 10-K for the year ended December 31, 2022,
Quarterly Reports on Form 10-Q for the quarters ended March 31,
2023, June 30, 2023, and September 30, 2023, 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 SEC. Any
forward-looking statement speaks only as of the date on which it
was made. None of Disc, nor its affiliates, advisors or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as result of new
information, future events or otherwise, except as required by
law.
Media Contact
Peg RusconiVerge Scientific
Communicationsprusconi@vergescientific.com
Investor Relations Contact
Christina TartagliaStern Investor
Relationschristina.tartaglia@sternir.com
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