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 it will host a
virtual KOL investor event on Tuesday, April 25, 2023, at 1:00 pm
ET/ 10:00 am PT to provide an overview of erythropoietic
protoporphyria (EPP), a rare, severe disease caused by mutations in
the heme biosynthesis pathway. The event will also include an
overview of Disc’s development plans of bitopertin for
erythropoietic protoporphyria.
Intended for investors and other interested audiences, the
virtual event will feature presentations from leading porphyria
experts, who will provide a disease overview and discuss
epidemiology, pathophysiology, disease burden, and unmet need
associated with EPP. Invited speakers will include:
- Bruce Wang, M.D., Professor of Gastroenterology, University of
California San Francisco, who runs the USCF Porphyria Center and is
a principal investigator in the US Porphyrias Consortium. In
addition to porphyria research, he also runs a research laboratory
studying liver biology.
- Jean-Charles Deybach, M.D., Ph.D., Professor of Medicine, Paris
Diderot University, who has been a porphyria specialist for more
than 40 years, including running the French reference center for
porphyria and acting as president of the European Porphyria Network
(EPNET).
Members of Disc’s leadership will also review the therapeutic
rationale for bitopertin in EPP and the trial designs of its
ongoing phase 2 studies in BEACON and AURORA. The company will also
review expectations and timing for data readouts in 2023.
A live webcast of the event will be available in the Events and
Presentations section of the Investor page of Disc’s website
(https://ir.discmedicine.com/). A webcast replay will be available
after the live presentation and will be accessible for 90 days.
Please register for the event on the Events and Presentations page
of Disc’s website or by clicking this link.
About EPP
EPP is a rare, debilitating and potentially life-threatening
disease caused by mutations that affect heme biosynthesis,
resulting in the accumulation of a toxic, photoactive intermediate,
PPIX. This causes severe reactions when patients are exposed to
sunlight, characterized by excruciating pain, edema, burning
sensations and potential blistering and disfigurement. PPIX also
accumulates in the hepatobiliary system and can result in
complications including gallstones, cholestasis, and liver damage
in 20-30% of patients and in extreme cases liver failure. Current
standard of care involves extreme measures to avoid sunlight,
including restricting outdoor activities to nighttime, use of
protective clothing and opaque shields, and pain management. This
has a significant impact on the psychosocial development, quality
of life, and daily activities of patients, particularly in young
children and families. There is currently no cure for EPP and only
one FDA-approved therapy, a surgically implanted synthetic hormone
designed to stimulate melanin production called Scenesse®
(afamelanotide).
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 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, 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 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 our
Annual Report on Form 10-K for the year ended December 31, 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
Disc Medicine (NASDAQ:IRON)
Gráfico Histórico do Ativo
De Mar 2024 até Abr 2024
Disc Medicine (NASDAQ:IRON)
Gráfico Histórico do Ativo
De Abr 2023 até Abr 2024