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 presented updated results
from the phase 2 open-label BEACON study of bitopertin, an orally
administered glycine transporter 1 (GlyT1) inhibitor, in patients
with erythropoietic protoporphyria (EPP) as an oral presentation at
the 65th ASH Annual Meeting. The updated data are consistent with
and confirm the initial positive results presented in June,
demonstrating significant decreases in PPIX, robust and consistent
improvements in sunlight tolerance across all study measures,
including the precedented pivotal endpoint, and improvements in
patient quality of life.
“This has been a tremendous ASH meeting for Disc, as we
presented data across our two most advanced programs. We are
especially proud and excited to present the updated results from
BEACON, which reflect a more robust open label data set and clearly
indicate that reducing PPIX with bitopertin has the potential to
result in dramatic benefits for patients with EPP. Importantly,
this improvement was observed across every efficacy measure of the
study, including our analysis of the precedented pivotal endpoint,
cumulative time in light over 6 months, which we debuted at this
meeting,” said John Quisel, J.D., Ph.D., President and Chief
Executive Officer. “With these results and the positive initial
efficacy data from the DISC-0974 myelofibrosis study that we
presented earlier today, Disc is preparing to enter its next stage
of growth. We look forward to next year as we advance our full
portfolio and obtain the readouts from AURORA and other
studies.”
The BEACON study (ACTRN12622000799752) is a randomized,
open-label, parallel-arm study that enrolled 22 adult subjects with
EPP or X-linked protoporphyria (XLP) in Australia, and has been
expanded to include adolescents. This trial was designed to assess
changes in levels of PPIX, as well as measures of photosensitivity,
quality of life, and safety and tolerability. Subjects are
randomized to receive either 20 mg or 60 mg of bitopertin
once-daily for 24 weeks, after which patients have the option of
continuing in an open-label extension of the trial for up to an
additional 24 weeks. The updated data presented reflects results
from all 22 adults, with a data cutoff of September 18,
2023 for PPIX data and October 20, 2023 for all other
endpoints. The data are consistent with and confirm the initial
positive results presented in June 2023.
- Protoporphyrin IX
(PPIX) levels: Significant, dose-dependent, and sustained
reductions in whole blood PPIX levels; mean reduction > 40%
(p<0.001 versus baseline)
- Demonstrated
substantial and consistent improvements in sunlight tolerance
across all study measures
- Highlights of the
data presented:
- Average time to
prodrome: Greater than 3x improvement vs. baseline
(p<0.001)
- Increased proportion
of days without symptoms: 78% vs. 33% (baseline)
- Increased proportion
of sunlight challenges without prodromes: 54% vs. 7%
(baseline)
- Phototoxic
reactions: 92% reduction in patient-reported reactions while on
treatment compared to baseline
- Nearly all
participants reported improvements in multiple quality-of-life
measures at the end of study
- Mean cumulative
total time in light on days without pain observed over the 6-month
treatment period (precedented pivotal endpoint): 222.6 + 129.3
hours
- Bitopertin-treated
participants had a >3x increase relative to historical
control
- Bitopertin was
generally well tolerated at both dose levels with no serious
adverse events, stable mean hemoglobin levels, and no anemia
adverse events (AEs) reported.
- The most common AEs
were dizziness, lightheadedness, headache, and nausea.
Earlier today, Disc also presented initial positive data from
the ongoing phase 1b study of DISC-0974 in myelofibrosis (MF)
patients with anemia. The data were presented as a poster during
the ASH meeting and demonstrated suppression of hepcidin, increased
iron levels and improvements in hematologic parameters, including
increased hemoglobin and reduction in transfusion burden. The
presentation was announced in a separate press release issued
earlier today and will be reviewed again during the management
call, as well as initial data from the first dose-escalation cohort
of the ongoing phase 1b/2 study in non-dialysis dependent chronic
kidney disease (NDD-CKD) patients with anemia.
Bitopertin and DISC-0974 are investigational agents and are not
approved for use as a therapy in any jurisdiction worldwide.
Webcast Conference Call InformationManagement
will host a call on Monday, December 11th at 9:30 pm ET / 6:30 pm
PT to review data and operational plans. Please register for
management’s webcast on the Events and Presentations page of Disc’s
website (https://ir.discmedicine.com/).
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, as amended, including, but not limited to, express or implied
statements regarding Disc’s expectations, hopes, beliefs,
intentions or strategies with respect to its AURORA Phase 2 and
BEACON Phase 2 clinical studies of bitopertin and the results
thereof, its Phase 1b/2 clinical studies of DISC-0974 in patients
with MF and NDD-CKD patients with anemia, its Phase 1 clinical
study of DISC-3405 in healthy volunteers, projected timelines for
the initiation and completion of its clinical trials, anticipated
timing of release of data and other clinical activities, Disc’s
business plans and objectives, Disc’s analysis of market potential
for patients with EPP, and Disc’s beliefs about operating expenses
and that it will have capital to fund Disc well into 2026. The use
of words such as, but not limited to, “anticipate,” “believe,”
“contemplate,” “continue,” “could,” “estimate,” “expect,” “future,”
“intends,” “may,” “might,” “plan,” “possible,” “potential,”
“predict,” “project,” “should,” “seek,” “suggest,” “will,” or
“would” 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
Quarterly Report on Form 10-Q for the quarter ended September 30,
2023, 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
Christina TartagliaStern Investor
Relationschristina.tartaglia@sternir.com
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