Disc Medicine Receives FDA Fast Track Designation for MWTX-003 for the Treatment of Polycythemia Vera
September 20 2023 - 4:30PM
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 MWTX-003 for the treatment of patients with
polycythemia vera (PV).
“We are delighted to have received Fast Track
designation for MWTX-003, which highlights the unmet need for PV
patients and the potential of MWTX-003 in a disease where there are
few treatment options,” said John Quisel, J.D., Ph.D., President
and Chief Executive Officer of Disc. “We believe MWTX-003 is
uniquely positioned to address the needs of PV patients and are
excited to initiate a Phase 1 trial in the coming months.”
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 MWTX-003MWTX-003, also
known as DISC-3405, is an investigational, anti-TMPRSS6
(Transmembrane Serine Protease 6, also known as Matriptase-2)
monoclonal antibody designed to increase hepcidin production and
suppress serum iron, that Disc in-licensed from Mabwell
Therapeutics in January 2023. Preclinical studies of MWTX-003 have
demonstrated an increase in hepcidin production and suppression of
serum iron levels in animal models of beta-thalassemia and
polycythemia vera. The IND was accepted in November 2022 and Disc
plans to initiate a Phase 1 study of MWTX-003 in healthy volunteers
during the second half of 2023. Disc plans to develop MWTX-003
initially as a treatment for polycythemia vera as well as other
hematologic conditions.
MWTX-003 is an investigational agent and is not
approved for use as a therapy in any jurisdiction worldwide.
About Polycythemia Vera
(PV)
Polycythemia vera (PV) is a chronic and rare myeloproliferative
neoplasm characterized by the abnormal proliferation of red blood
cells. PV affects approximately 150,000 patients in the U.S. and
has a similar prevalence in Europe. The overproduction of red blood
cells alters the viscosity of blood, causing it to thicken and
placing patients at an elevated risk of cardiovascular and
thromboembolic events, such as heart attack and stroke. Patients
also experience complications such as enlarged spleen and symptoms
of their disease such as fatigue, pruritis, difficulty
concentrating and others. Current therapy involves phlebotomy to
physically remove blood and iron to limit erythropoiesis or
treatment with cytoreductive agents, with the goal of reducing red
blood cell count and managing symptoms.
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 AURORA Phase 2 and BEACON Phase 2 clinical studies of
bitopertin, and its Phase 1b/2 study of bitopertin in
Diamond-Blackfan Anemia, its Phase 1b/2 clinical study of DISC-0974
in NDD-CKD patients with anemia, its anticipated Phase 1 study of
MWTX-003 and potential development of MWTX-003 as a treatment for
polycythemia vera and other indications, 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 and June 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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