Full Press Release Details
Disc Presents Positive Updated Results from Phase 2 BEACON Study of Bitopertin and Other Programs at the
65th American Society of Hematology (ASH) Annual Meeting
WATERTOWN, Mass. (December 11,
2023) 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.
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.
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 Information
Management 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/).
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 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
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.
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