Disc Medicine to Host Conference Call on Type C Meeting for Bitopertin in Erythropoietic Protoporphyria (EPP)
21 January 2025 - 11:00AM
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, will host a conference call to
discuss feedback received from its Type C meeting on bitopertin in
EPP with the U.S. Food and Drug Administration (FDA). The
conference call will be held on Tuesday, January 21, at 8:00 am
EST.
Conference Call Information
Please register for the event on the Events and
Presentations page of Disc’s website at
https://ir.discmedicine.com/.
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. Bitopertin has been studied in multiple
clinical trials in patients with EPP, including the Phase 2
open-label BEACON trial, the Phase 2 double-blind,
placebo-controlled AURORA trial, and an open-label extension HELIOS
trial.
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)
Erythropoietic protoporphyria (EPP), including
X-linked Protoporphyria (XLP), 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 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 Medicine
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.
Media Contact
Peg RusconiDeerfield
Grouppeg.rusconi@deerfieldgroup.com
Investor Relations Contact
Christina TartagliaPrecision
AQchristina.tartaglia@precisionaq.com
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