Disc Medicine Announces First Patient Enrolled in Phase 1/2 Clinical Trial of Bitopertin in Diamond-Blackfan Anemia (DBA)
July 27 2023 - 9:00AM
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 that the first
patient has been enrolled in the National Institutes of
Health-sponsored Phase 1/2 clinical trial of bitopertin in
Diamond-Blackfan anemia (DBA). Bitopertin is an investigational,
orally administered glycine transporter 1 (GlyT1) inhibitor
designed to modulate heme biosynthesis.
“The start of this trial represents another step toward
demonstrating the potential of bitopertin to address a wide range
of hematologic conditions.” said Will Savage, M.D., Ph.D., Chief
Medical Officer of Disc. “We are excited to test the potential of
treating DBA by modulating heme synthesis with bitopertin,
particularly in light of our recent positive initial data from an
open label trial of bitopertin in erythropoietic protoporphyria
(EPP) at EHA. We appreciate the support and leadership of the
National Heart, Lung, and Blood Institute and look forward to
continued collaboration on this trial.”
The Phase 1/2 study will be a 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. This study will be
conducted and funded by the NIH under a Cooperative Research and
Development Agreement (CRADA), 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, with Dr. David Young, M.D., Ph.D.,
NHLBI Staff Clinician as Principal Investigator.
The content of this research is solely the responsibility of the
authors and does not necessarily represent the official views of
the National Institutes of Health.
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, both of 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 an investigational, clinical-stage,
orally-administered inhibitor of glycine transporter 1 (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, where it has potential to be the first
disease-modifying therapy. There are currently two ongoing Phase 2
clinical trials 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 (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, as amended, including, without limitation, express or implied
statements related to Disc’s expectations regarding the timing and
closing of the offering, and the anticipated use of proceeds from
the offering. The words “may,” “will,” “could,” “would,” “should,”
“expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,”
“predict,” “project,” “potential,” “continue,” “seek,” “target” and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Any forward-looking statements in this
press release are based on management’s current expectations and
beliefs and are subject to a number of risks, uncertainties and
important factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release. These risks and
uncertainties include fluctuations in Disc’s stock price, changes
in market conditions, the satisfaction of customary closing
conditions related to the public offering, and other risks
identified in our SEC filings, including our Quarterly Report on
Form 10-Q for the quarter ended March 31, 2023, and in the
preliminary prospectus supplement related to the offering filed
with the SEC on June 12, 2023. We caution you not to place undue
reliance on any forward-looking statements, which speak only as of
the date they are made. We disclaim any obligation to publicly
update or revise any such statements to reflect any change in
expectations or in events, conditions or circumstances on which any
such statements may be based, or that may affect the likelihood
that actual results will differ from those set forth in the
forward-looking statements.
Media Contact
Peg RusconiVerge Scientific
Communicationsprusconi@vergescientific.com
Investor Relations Contact
Christina TartagliaStern Investor
Relationschristina.tartaglia@sternir.com
Disc Medicine (NASDAQ:IRON)
Historical Stock Chart
From Jun 2024 to Jul 2024
Disc Medicine (NASDAQ:IRON)
Historical Stock Chart
From Jul 2023 to Jul 2024