Disc Medicine Launches Expanded Access Program for Bitopertin for EPP Patients
Rhea-AI Summary
Disc Medicine (NASDAQ:IRON) launched an expanded access program (EAP) in the U.S. for investigational drug bitopertin for eligible erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) patients.
Bitopertin, an oral GlyT1 inhibitor, is in the double-blind, placebo-controlled Phase 3 APOLLO trial in patients 12+ years, with topline data expected in Q4 2026 and a potential FDA decision by mid-2027.
AI-generated analysis. Not financial advice.
Positive
- Expanded access program opens bitopertin to eligible EPP and XLP patients before approval
- Phase 3 APOLLO topline data expected in Q4 2026 provides clear development timeline
- Potential FDA decision on bitopertin anticipated by mid-2027, according to Disc Medicine
Negative
- Bitopertin remains investigational pending Phase 3 APOLLO results and FDA decision
- EAP access limited to U.S. EPP patients 12+ who meet specific eligibility criteria
News Market Reaction – IRON
On the day this news was published, IRON gained 3.45%, reflecting a moderate positive market reaction.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Market Reality Check
Peers on Argus
IRON fell 1.74% while close peers were mixed: AGIO up 0.74%, others like TARS, ARQT, TVTX, and OCUL down between roughly 0.56% and 1.64%. Moves do not point to a unified sector trend.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| May 12 | Portfolio presentations | Positive | -1.0% | Multiple hematology program updates and bitopertin EPP data at EHA 2026. |
| May 11 | Investor conferences | Neutral | +1.4% | Participation in H.C. Wainwright and Jefferies investor conferences. |
| May 05 | Earnings and update | Positive | -0.1% | Q1 2026 results, APOLLO completion update, and strong $730.2M cash runway. |
| Apr 21 | Clinical data preview | Positive | -3.3% | Upcoming ASCO oral presentation of Phase 2 RALLY-MF DISC-0974 data. |
| Mar 26 | APOLLO enrollment done | Positive | -3.1% | Completion of Phase 3 APOLLO enrollment in EPP, expanded to 183 patients. |
Recent development and clinical news for IRON often saw flat-to-negative next-day moves, even on seemingly positive milestones like Phase 3 enrollment completion and multiple conference presentations.
Over the last few months, Disc Medicine reported several hematology program milestones. It completed enrollment in the pivotal Phase 3 APOLLO bitopertin trial for EPP, expanded to 183 participants with results targeted for Q4 2026 and an FDA decision by mid-2027. The company also highlighted multiple data presentations at ASCO and EHA and maintained cash and securities of $730.2 million funding operations into 2029. Despite these updates, share reactions have often been muted or negative.
Market Pulse Summary
This announcement extends bitopertin access to eligible EPP and XLP patients aged 12+ ahead of pivotal Phase 3 APOLLO data expected in Q4 2026 and a potential FDA decision by mid-2027. It follows earlier completion of APOLLO enrollment at 183 patients and substantial investment in the hematology pipeline. Investors monitoring execution may focus on maintaining the strong $730.2M cash position and the quality and timing of forthcoming clinical data.
Key Terms
expanded access program regulatory
erythropoietic protoporphyria medical
X-linked protoporphyria medical
glycine transporter 1 (GlyT1) inhibitor medical
Phase 3 medical
double-blind medical
placebo-controlled medical
CRL response regulatory
AI-generated analysis. Not financial advice.
- 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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Deerfield Group
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Precision AQ
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