Disc Medicine Receives FDA Orphan Drug Designation for DISC-3405 for the Treatment of Polycythemia Vera
2024年2月9日 - 11:00PM
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 Orphan Drug
Designation to DISC-3405 for the treatment of patients with
polycythemia vera (PV).
“Orphan drug designation is an important milestone that
highlights the potential of DISC-3405 in PV, a rare disease with
few treatment options,” said John Quisel, J.D., Ph.D., President
and Chief Executive Officer of Disc. “We look forward to sharing
initial data from our ongoing Phase 1 trial of DISC-3405 in healthy
volunteers in the first half of 2024.”
FDA Orphan Drug Designation is granted to investigational
therapies addressing rare medical diseases or conditions that
affect fewer than 200,000 people in the United States. Orphan Drug
status provides benefits to drug developers, including assistance
in the drug development process, tax credits for clinical costs,
exemptions from certain FDA fees and seven years of post-approval
marketing exclusivity.
About DISC-3405
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.
Disc in-licensed DISC-3405 from Mabwell Therapeutics in January
2023. Preclinical studies of DISC-3405 have demonstrated an
increase in hepcidin production and suppression of serum iron
levels in animal models of beta-thalassemia and polycythemia vera.
Disc initiated a Phase 1 study of DISC-3405 in healthy volunteers
in October 2023 and plans to develop DISC-3405 initially as a
treatment for polycythemia vera as well as other hematologic
conditions.
DISC-3405 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 DISC-3405 and its
clinical development. 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: Disc’s
expectations regarding the clinical development and
commercialization of DISC-3405; Disc’s expectations regarding
future growth and innovation; 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 Securities and Exchange Commission (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
Disc Medicine (NASDAQ:IRON)
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から 4 2023 まで 4 2024