Full Press Release Details
Disc Medicine Presents Positive Clinical Data Across Portfolio at the European Hematology Association
(June 14, 2024) 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 positive additional data for bitopertin in erythropoietic protoporphyria (EPP), including additional analyses of AURORA data that further demonstrated the clinical activity of bitopertin and highlighted meaningful improvements in light
tolerance, phototoxic reactions, and quality of life. Additionally, Disc shared updated data from a Phase 1b trial of DISC-0974 in MF anemia which demonstrated durable increases in hemoglobin and reduced transfusion burden in a majority of evaluable
patients. Disc also shared initial SAD data from a Phase 1 healthy volunteer trial of DISC-3405 that provided proof of mechanism, showing that the drug has the potential to provide deep and sustained increases in hepcidin and reductions in iron. The
data were presented at the European Hematology Association (EHA) 2024 Congress.
This has been a tremendously exciting EHA meeting for Disc, where
we had the opportunity to present data from ongoing clinical studies across our entire portfolio, showcasing the activity and therapeutic potential of each of our programs, said John Quisel, J.D., Ph.D., President and Chief Executive Officer
of Disc. At this meeting, we shared a deeper analysis of data from AURORA that underscores our confidence in bitopertin s potential in EPP and supports a development path forward. We also provided an update from our study of DISC-0974 in
anemia of myelofibrosis, which continues to demonstrate promising hematologic activity similar to what we showed at ASH. And, finally, we re excited to share for the first time, data from our third program, DISC-3405, in healthy volunteers,
demonstrating clinical proof-of-mechanism as an iron restriction agent with the potential for a differentiated profile.
The AURORA study is a
randomized, double-blind, placebo-controlled Phase 2 clinical trial that enrolled 75 adult subjects with EPP. Subjects were randomized 1:1:1 to receive 20 mg of bitopertin (n=26), 60 mg of bitopertin (n=25), or placebo (n=24) once daily for 17
weeks. Key AURORA data presented include:
The Phase 1b/2a multi-center, open-label, ascending-dose clinical trial of DISC-0974 is enrolling patients with MF and severe anemia, including both
transfusion and non-transfusion dependent patients. In the phase 1b dose-escalation phase, DISC-0974 is administered subcutaneously every 4 weeks for up to 6 treatments. Updated data from 34 patients with an
April 29, 2024 cutoff include:
Initial data were also presented from
the SAD portion of the Phase 1 clinical trial of DISC-3405 in healthy volunteers. In this trial, healthy males and females ages 18 to 65 were given a single dose of placebo (n=10) or DISC-3405 at 75 mg intravenously (IV) (n=6), 37.5 mg
subcutaneously (SC) (n=6), 75 mg SC (n=6), 150 mg SC (n=6), or 300 mg SC (n=6). This initial data showed:
Management will host a call to review the presented data on Friday, June 14th at 8:00 am ET. Please register for the event on the Events and Presentations page of Disc s website (https://ir.discmedicine.com/).
Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.
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,
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 trials of bitopertin and the results thereof, its Phase 1b/2 clinical trial of DISC-0974
in patients with MF and NDD-CKD patients with anemia, its initial SAD data in its Phase 1 clinical trial of DISC-3405 in healthy volunteers; and projected timelines for the initiation and completion of its
clinical trials, anticipated timing of release of data, and other clinical 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 Quarterly Report for the quarter ended March 31, 2024, 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.
Verge Scientific Communications
Investor Relations Contact
Stern Investor Relations