Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitopertin in Patients with Diamond-Blackfan Anemia (DBA)
21 March 2023 - 10:00PM
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, announced today a collaboration
with the National Heart Lung and Blood Institute (NHLBI) of the
National Institutes of Health (NIH) to evaluate bitopertin, a
therapeutic candidate designed to modulate heme biosynthesis, in a
phase 2 clinical study of patients with Diamond-Blackfan anemia
(DBA). The study will be conducted and funded by the NIH under a
Cooperative Research and Development Agreement (CRADA) and is
expected to initiate mid-year 2023.
“We’re thrilled to have the support of the NHLBI for this
clinical trial as there is growing evidence to suggest that
reducing excess levels of heme may be an effective treatment
strategy for DBA. This collaboration will enable us to benefit from
the experience of the NHLBI in designing and conducting clinical
studies of DBA.” said John Quisel, J.D., Ph.D., Chief Executive
Officer and President of Disc. “Disc now has ongoing development
programs of bitopertin in both erythropoietic porphyria and DBA. We
believe controlling heme synthesis has the potential to address a
wide range of hematologic conditions and are planning studies in
additional indications.”
Under the CRADA, the NHLBI will serve as the regulatory sponsor
and be responsible for conducting a phase 2 clinical study of
bitopertin in DBA patients. The study will be jointly funded by the
NHLBI and Disc. The study will be under the direction of Dr.
Cynthia Dunbar, M.D., the NIH Distinguished Investigator and Chief
Translational Stem Cell Biology Branch, and Head, Molecular
Hematopoiesis Section, NHLBI.
The phase 2 study will be a pilot, single-arm, dose-escalation
trial of bitopertin in DBA patients who either have
steroid-refractory and/or relapsed disease or are unable to
tolerate systemic corticosteroids. The study includes planned dose
escalation within each participant to continually assess for
hematologic response. Upon completion of the main treatment period,
patients may continue on extended treatment within the trial.
About Diamond-Blackfan Anemia
Diamond-Blackfan Anemia (DBA) is a rare, inherited blood
disorder characterized by the failure of bone marrow to produce red
blood cells. The incidence of DBA is approximately 1:100,000 to
1:200,000 live births every year. Patients are usually diagnosed
during infancy and commonly present with severe anemia, pallor,
fatigue, as well as other potential abnormalities. DBA is chronic
and requires lifelong management with corticosteroids and blood
transfusions, which are associated with serious toxicities and
long-term complications. Evidence suggests that the anemia of DBA
may be caused by the accumulation of excess heme in developing red
blood cells, which is toxic and leads to their premature death.
Preclinical studies have shown that targeting elevated heme levels
has the potential as a therapeutic strategy for DBA.
About Bitopertin
Bitopertin is a clinical-stage, orally administered inhibitor of
GlyT1 that is designed to modulate heme biosynthesis. GlyT1 is a
membrane transporter expressed on developing red blood cells and is
required to supply sufficient glycine for heme biosynthesis and
support erythropoiesis. Disc is planning to develop bitopertin as a
potential treatment for a range of hematologic diseases including
erythropoietic porphyrias, Diamond-Blackfan Anemia (DBA) and
others. There are currently two ongoing phase 2 clinical studies of
bitopertin in patients with erythropoietic porphyria, including an
open-label trial called BEACON and a randomized, double-blind
placebo controlled trial called AURORA.
Bitopertin is an investigational agent and is not approved for
use as a therapy in any jurisdiction worldwide. Disc obtained
global rights to bitopertin under a license agreement from Roche in
May 2021.
About Disc
Disc Medicine 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 collaboration
with the NIH and the Phase 2 clinical study of Bitopertin in
patients with DBA 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 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 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
and Exchange Commission. 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
Suzanne MessereStern Investor
Relationssuzanne.messere@sternir.com
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