Disc Medicine Launches Expanded Access Program for Bitopertin for EPP Patients
- The expanded access program (EAP) is available to eligible patients with erythropoietic protoporphyria (EPP) and provides access to bitopertin prior to regulatory decision
- Bitopertin, an oral glycine transporter 1 (GlyT1) inhibitor, is being evaluated in the Phase 3 APOLLO clinical trial, with topline data anticipated in Q4 2026
WATERTOWN, Mass., June 01, 2026 (GLOBE NEWSWIRE) -- 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 the launch of an expanded access program (EAP) for bitopertin for eligible patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) in the U.S.
"Recognizing the urgent need for new treatment options for patients and families affected by EPP, we are proud to launch this bitopertin expanded access program," said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. "As we work to complete the Phase 3 APOLLO study, we believe providing earlier access where appropriate is an important step in supporting the EPP community. We look forward to continuing to advance the development of bitopertin, with a potential FDA decision expected by mid-2027."
Bitopertin is being evaluated for the treatment of EPP and XLP in patients age 12 and above in the ongoing double-blind, placebo-controlled Phase 3 APOLLO study. Data from APOLLO is expected in Q4 2026, after which a CRL response will be submitted with an FDA decision expected by mid-2027.
EAPs are intended to serve as a potential pathway for patients with serious conditions to gain access to an investigational medical treatment outside of clinical trials. The EAP for bitopertin may be available for patients with EPP ages 12 and above who lack satisfactory therapeutic alternatives and meet additional eligibility criteria. Additional information on Disc's EAP can be found at https://clinicaltrials.gov/study/NCT07603401, https://www.discmedicine.com/clinical-trials#eap or by contacting bitopertinEAP@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 developing 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, an open-label extension HELIOS trial, and the Phase 3 double-blind, placebo-controlled APOLLO 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 Disc Medicine
Disc Medicine (NASDAQ:IRON) 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 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 the next stages of its development program for bitopertin, including the projected timelines for the completion of the APOLLO clinical trial and release of data; and the projected timelines for Disc's response to the FDA's Complete Response Letter (CRL) and any potential approval decision by the FDA. 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 content and timing of decisions made by the FDA and other regulatory authorities; and the other risks and uncertainties described in Disc's filings with the Securities and Exchange Commission, including in the "Risk Factors" section of Disc's Annual Report on Form 10-K for the year ended December 31, 2025, and in subsequent Quarterly Reports on Form 10-Q. 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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