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 upcoming
clinical data to be presented at the European Hematology
Association (EHA) 2023 Congress in Frankfurt, Germany. The
presentation will report initial results from BEACON, the Phase 2,
randomized, open‐label, parallel arm trial of bitopertin in
Erythropoietic Protoporphyria (EPP) that is ongoing in Australia.
This study was designed to assess changes in levels of
protoporphyrin IX (PPIX), the toxic metabolite that causes disease
pathology in EPP, as well as measures of photosensitivity, quality
of life, and safety and tolerability.
Additionally, an abstract outlining the study design of AURORA,
a Phase 2, randomized double-blind, placebo-controlled trial of
bitopertin in EPP will be published in the EHA Abstract Book.
Both abstracts are now available on the EHA conference
website.
Management will host a call to review the presented data on
Friday, June 9th at 7:30 am ET. Please register for the event on
the Events and Presentations page of Disc’s website
(https://ir.discmedicine.com/).Presentation Details:
Abstract Number: P1498Title:
Initial Data from the BEACON Trial: A Phase 2, Randomized,
Open‐label Trial of Bitopertin in Erythropoietic
ProtoporphyriaDate / Time: Friday, June 9th,
18:00-19:00 CEST
Abstract Number: PB2562Title:
Study Design of the AURORA Trial: A Phase 2, Randomized,
Double-Blind, Placebo-Controlled Trial of Bitopertin in
Erythropoietic Protoporphyria
About BitopertinBitopertin 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 Erythropoietic Protoporphyria (EPP) and X-linked
Protoporphyria (XLP)Erythropoietic protoporphyria (EPP)
and X-linked Protoporphyria (XLP) are rare, debilitating and
potentially life-threatening diseases caused by mutations that
affect heme biosynthesis, resulting in the accumulation of a toxic,
photoactive intermediate called protoporphyrin IX (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 Disc MedicineDisc 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 Medicine 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 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
Suzanne Messere Stern Investor
RelationsSuzanne.messere@sternir.com
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