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 data from
initial dose cohorts of its ongoing phase 1b/2 study of DISC-0974,
a monoclonal antibody designed to suppress hepcidin by inhibiting
the hemojuvelin (HJV) co-receptor, in MF patients with anemia. The
initial data demonstrated that treatment with DISC-0974
substantially decreased serum hepcidin, increased serum iron and
resulted in improvements in hemoglobin or reduced transfusion
burden across a broad range of MF patients.
“We are thrilled to see this level of hematologic activity so
early during dose escalation and in a range of patient types.
Elevated hepcidin is an important driver of anemia in patients with
MF and the initial data suggest that DISC-0974 has leading activity
in suppressing hepcidin,” said John Quisel, J.D., Ph.D., President
and Chief Executive Officer of Disc. “This is the second program in
the last six months where Disc has shown proof-of-concept data in
patients, and we look forward to advancing DISC-0974 deeper into
development and presenting updated data from both this MF and the
ongoing CKD anemia study next year.”
The phase 1b/2a multi-center, open-label, ascending-dose study
(NCT05320198) is enrolling patients with MF and severe anemia,
including both transfusion and non-transfusion dependent patients.
The trial also includes patients who may or may not be receiving
concomitant janus kinase (JAK) inhibitor therapy. Study assessments
include safety and tolerability of DISC-0974, as well as markers of
iron regulation, such as hepcidin and iron, and hematologic
parameters. In the phase 1b dose-escalation phase, DISC-0974 is
administered subcutaneously every 4 weeks for up to 6 treatments.
Dose escalation is ongoing and the data presented reflect 11
evaluable subjects from the initial three dose levels (14 mg, 28 mg
and 50 mg) as of the October 20, 2023 data cutoff.
Key initial data presented:
- DISC-0974 dosing resulted in meaningful, dose-dependent
decreases in hepcidin across all treated patients
- These reductions in hepcidin corresponded to dose-dependent
increases in serum iron
- Patients dosed with 28 mg of DISC-0974 had a >75% reduction
in serum hepcidin and >75% increase in serum iron
- Four of seven (57%) evaluable non-transfusion-dependent (NTD)
patients at the 28 mg and 50 mg dose levels had a >1.5 g/dL
hemoglobin increase from baseline after starting DISC-0974
- One of two transfusion-dependent (TD) patients achieved
transfusion independence by the Gale Criteria
- Hematologic activity was observed in MF patients, regardless of
concomitant JAK inhibitor use
- To date, DISC-0974 has been generally well-tolerated. The
adverse events (AEs) seen in two or more subjects were fatigue,
anemia, diarrhea, and nausea. The majority of AEs were deemed not
related to DISC-0974.
These data were presented at the 65th American Society of
Hematology (ASH) Annual Meeting in San Diego, California and the
poster is available on the ASH platform. Later today, the company
will be presenting updated results from the phase 2 BEACON study of
bitopertin in EPP as an oral presentation, December 11th at 5:30 pm
PT / 8:30 pm ET.
Management will host a call to review the presented data on
Monday, December 11th at 6:30 pm PT / 9:30 pm ET. Please register
for the event on the Events and Presentations page of Disc’s
website (https://ir.discmedicine.com/).
About DISC-0974
DISC-0974 is an investigational monoclonal antibody (mAb)
targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and
is designed to suppress hepcidin production and increase serum iron
levels in patients suffering from anemia of inflammation. DISC-0974
was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation
arises from abnormally elevated hepcidin and is the second most
common form of anemia, affecting millions of patients in the US
across numerous diseases such as chronic kidney disease,
myelofibrosis, cancer, autoimmune diseases, and other conditions
with an inflammatory component. Disc has established clinical
proof-of-mechanism of DISC-0974 in a Phase 1 trial of healthy
volunteers and initiated a Phase 1b/2a clinical trial of DISC-0974
in patients with myelofibrosis and anemia, as well as a Phase 1b/2a
clinical trial of DISC-0974 in patients with chronic kidney disease
and anemia who are not receiving dialysis.
DISC-0974 is an investigational agent and is not approved for
use as a therapy in any jurisdiction worldwide.
About Anemia of MyelofibrosisMyelofibrosis (MF)
is a rare, chronic blood cancer that currently affects an estimated
16,000 to 18,500 patients in the United States alone. Severe,
progressive, and treatment resistant anemia is the primary clinical
manifestation of MF. At diagnosis, over 80% of MF patients have
anemia, which progressively worsens and ultimately renders the
majority of patients dependent on chronic red blood cell
transfusions. Recent studies have shown hepcidin to be a key
molecular driver of anemia in myelofibrosis. Hepcidin is elevated
by approximately 12-fold in MF patients, and is correlated with
disease severity, anemia, and the need for red blood cell
transfusions.
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 Phase 1b/2
clinical studies of DISC-0974 in patients with MF and NDD-CKD
patients with anemia, projected timelines for the initiation and
completion of its clinical trials, anticipated timing of release of
data, and other clinical activities; and Disc’s business plans and
objectives. 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 nature,
strategy and focus of Disc; Disc’s plans to research, develop and
commercialize its current and future product candidates; the timing
of the availability of data from Disc’s clinical trials; the timing
and anticipated results of Disc’s preclinical studies and clinical
trials and the risk that the results of Disc’s 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, Quarterly Reports on Form 10-Q
for the quarters ended March 31, 2023, June 30, 2023, and September
30, 2023, 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
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
Christina TartagliaStern Investor
Relationschristina.tartaglia@sternir.com
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