RegulationOrphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Commissioner's National Priority Voucher (United States)

Structure/Sequence

Molecular FormulaC21H20F7N3O4S

InChIKeyYUUGYIUSCYNSQR-LBPRGKRZSA-N

CAS Registry845614-11-1

Clinical Trials associated with Bitopertin

NCT07123363

/ Not yet recruitingPhase 1IIT

Effect of Bitopertin on the Liver and on Levels of Protoporphyrin IX in Bile, Blood, Liver, and Stool in Patients With Erythropoietic Protoporphyria/X-linked Protoporphyria and Increased Liver Stiffness and/or Liver Enzymes at Baseline

The primary goal of this study is to assess safety and tolerability of bitopertin in subjects with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP) and evidence of compensated liver disease.

Start Date01 Jan 2026

Sponsor / Collaborator

Wake Forest School of Medicine

NCT06910358

/ RecruitingPhase 3

APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older. The main questions it aims to answer are:
* Whether bitopertin increases pain-free sunlight exposure after 6 months of treatment in participants with EPP or XLP.
* How PPIX concentration levels change from before bitopertin treatment to after 6 months of treatment.
Researchers will compare bitopertin to a placebo look-alike substance that contains no drug.
Participants will complete daily questionnaires and attend study visits for assessments.

Start Date04 Apr 2025

Sponsor / Collaborator

Disc Medicine, Inc.

NCT05883748

/ Enrolling by invitationPhase 2/3

An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP)

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

Start Date31 Aug 2023

Sponsor / Collaborator

Disc Medicine, Inc.

100 Clinical Results associated with Bitopertin

100 Translational Medicine associated with Bitopertin

100 Patents (Medical) associated with Bitopertin

Literatures (Medical) associated with Bitopertin

16 Sep 2025·JOURNAL OF CLINICAL INVESTIGATION

The GLYT1 inhibitor bitopertin mitigates erythroid PPIX production and liver disease in erythroid protoporphyria

Article

Author: Dickey, Amy K ; Xiang, Yi ; Ducamp, Sarah ; Heeney, Matthew M ; Fleming, Mark D ; Schmidt, Paul J ; Putra, Juan ; Leaf, Rebecca K ; Hong, Vu ; Wu, Min ; MacDonald, Brian ; Campagna, Dean R

Erythropoietic protoporphyria (EPP) is a genetic disorder typically resulting from decreased ferrochelatase (FECH) activity, the last enzyme in heme biosynthesis. Patients with X-linked protoporphyria (XLPP) have an overlapping phenotype caused by increased activity of 5-aminolevulinic acid synthase 2 (ALAS2), the first enzyme in erythroid heme synthesis. In both cases, protoporphyrin IX (PPIX) accumulates in erythrocytes and secondarily in plasma and tissues. Patients develop acute phototoxicity reactions upon brief exposure to sunlight. Some also experience chronic liver disease, and a small fraction develop acute cholestatic liver failure. Therapeutic options are limited, and none, save hematopoietic stem cell transplantation, directly targets erythroid PPIX accumulation. Bitopertin is an investigational orally available small-molecule inhibitor of the erythroid cell-surface glycine transporter GLYT1. We established the bitopertin PPIX inhibitory half-maximal effective concentration in a human erythroblast EPP model and confirmed a marked reduction of PPIX in erythroblasts derived from patients with EPP. We demonstrate that bitopertin also reduced erythrocyte and plasma PPIX accumulation in vivo in both EPP and XLPP mouse models. Finally, the reduction in erythroid PPIX ameliorated liver disease in the EPP mouse model. Altogether, these data support the development of bitopertin to treat patients with EPP or XLPP.

16 Sep 2025·JOURNAL OF CLINICAL INVESTIGATION

Blocking extracellular glycine uptake mediated by GlyT1 mitigates protoporphyria

Article

Author: Liesa, Marc

Accumulation of the light-reactive heme precursor protoporphyrin IX (PPIX) in blood causes protoporphyria, a disease characterized by severe pain resulting from sunlight exposure, as well as by the occurrence of liver failure in some patients. Thus, decreasing PPIX biosynthesis is a promising strategy to treat protoporphyria. In this issue of the JCI, Ducamp et al. report that inhibition of the glycine plasma membrane transporter GLYT1 using bitopertin decreased PPIX accumulation and ameliorated liver disease using human in vitro and mouse in vivo models. Their findings support the ongoing development of bitopertin to treat protoporphyria, while concurrently pointing to underexplored roles of glycine in erythroid cells.

01 Aug 2025·JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS

A semi-mechanistic population pharmacokinetic-pharmacodynamic model to assess downstream drug-target effects on erythropoiesis

Article

Author: Marchesi, Maddalena ; Silber Baumann, Hanna E ; Abrantes, João A ; Schaedeli Stark, Franziska ; Rognås, S Viktor

Abstract:

Erythropoiesis is a complex process that results in the production of erythrocytes from hematopoietic stem cells in the bone marrow. This work aimed to develop a population pharmacokinetic-pharmacodynamic (PKPD) model describing erythropoiesis and hemoglobin synthesis following bitopertin, an inhibitor of glycine transporter 1 (GlyT1), administration. Data from a Phase 1 clinical trial in 67 healthy subjects administered bitopertin (10, 30, or 60 mg) or placebo for 120 days were analyzed. Hematological assessments included erythrocyte and reticulocyte counts, immature reticulocyte fraction, hemoglobin concentration, and mean corpuscular hemoglobin. The proposed semi-mechanistic model, which leverages data and physiological knowledge, was found to adequately simultaneously describe the dose- and time-dependent changes in the biomarkers. The framework was used to illustrate the potential outcome of hypothetical drug-target interactions at distinct stages of erythropoiesis and hemoglobin synthesis, exemplifying its usefulness in a clinical setting.

News (Medical) associated with Bitopertin

17 Nov 2025

·www.biospace.com

Pressure On, Purpose Questioned as Second Round of FDA Priority Review Vouchers Revealed

iStock, beast01 Priority voucher awardees and regulators could feel pressure to “meet the moment” as FDA watchers question the intent and feasibility of the Commissioner’s National Priority Voucher program. For the first 15 awardees of the FDA’s new Commissioner’s National Priority Voucher program, the pressure to succeed is palpable. “It’s super exciting,” John Quisel, CEO of Disc Medicine, told BioSpace . “It is kind of a jolt as well—a feeling of responsibility, of okay, we’re a national priority now to get this drug to these patients. We can’t fail, right?” Launched in June, the Commissioner’s National Priority Voucher (CNPV) program is intended to shorten the timeline for drug review from 10–12 months to one-to-two months. The vouchers are granted to companies that are “aligned with U.S. national priorities,” according to the announcement, including those addressing a health crisis, delivering more innovative cures, addressing an unmet public health need or increasing domestic drug manufacturing as a national security issue. The first nine vouchers were issued on October 16, followed by six more on November 6. So far, indications have included type 1 diabetes, infertility, congenital deafness, obesity and sickle cell disease. The receiving companies are as big as Johnson & Johnson and as small as Disc, which sports a market cap of a modest $3.24 billion, compared to J&J’s more than $470 billion. When the FDA put out a call for proposals for CNPVs this summer, “We felt like we have a rare disease, high unmet medical need, a potential therapy that’s addressing an underlying cause of disease . . . so we put in an application,” Quisel said, describing the company’s drug candidate for erythropoietic porphyrias (EPP). He added that Disc also had “support and positivity” in interactions with the dermatology review division at the agency. “I think that was an important part of feeling like there was a rationale for a successful bid for one of these national priority vouchers.” The first nine awardees—five of which stemmed from company applications, while four were nominated by agency reviewers, according to STAT News —were all investigational therapies. Some, such as Disc’s bitopertin , now have regulatory filings awaiting FDA review, while others are still in clinical trials. “One thing that struck me is how disparate they are, particularly across stage of development,” Eva Temkin, partner at the law firm Arnold & Porter, said of the first nine awardees. “You have some that are very close to the end, and you have some that are still in Phase III.” Then, five of the six vouchers announced in round two went to therapies already on the market. Notably, these were announced immediately following a White House press conference to unveil a drug pricing agreement for GLP-1 medicines, including Novo Nordisk’s blockbuster weight loss drug Wegovy as well as Eli Lilly’s investigational obesity pill orforglipron, each of which received a CNPV. “This was not, as I understand it, a prioritization exclusively based on public health imperative, which historically has been the purview of FDA in the way that these have been made,” Temkin, who previously served as acting policy staff director at the FDA’s Office of Therapeutic Biologics and Biosimilars, told BioSpace . “You have criteria that have not been well defined but arguably have expanded beyond that purview.” Regulatory Wegovy, Casgevy Among Latest FDA Priority Review Voucher Recipients The FDA awards a second round of Commissioner’s National Priority vouchers to six larger biopharma companies. And this time, with the exception of Eli Lilly’s orforglipron, the vouchers are for drugs that are already on the market. November 6, 2025 · 3 min read · Dan Samorodnitsky Courting Controversy Modeled after FDA Commissioner Marty Makary’s experience as a surgical oncologist, the review process for CNPV recipients will involve experts from across the agency gathering to review the chosen candidates in a one-day “tumor board style” meeting, according to the FDA’s June press announcement . The priority vouchers have reportedly been a source of conflict among Center for Biologics Evaluation and Research Chief Vinay Prasad and his former counterpart at the Center for Drug Evaluation and Research, George Tidmarsh, who resigned earlier this month amid a probe into his “personal conduct” at the agency. Tidmarsh raised questions about the legality of the program, particularly the one-day meeting format, according to reporting by STAT News . The FDA in its press release announcing the second round of vouchers appeared to address this issue, writing: “Under the Federal Food, Drug, and Cosmetic Act; the 21st Century Cures Act; and the Food and Drug Administration Safety and Innovation Act, the FDA is authorized to test innovative regulatory approaches to accelerate review, foster public health preparedness, and improve access to safe, effective, and affordable therapies.” Steven Grossman, policy and regulatory consultant and author of the FDA Matters blog, has also questioned the program. “I was initially very unhappy about it,” Grossman said during a recent BioSpace webinar , “because the whole idea is that priority at FDA should be built around unmet medical need, and by traditional standards that meant serious and life-threatening diseases, and it was clear that’s not what they were focused on.” He came around, however, when he broadened his definition of unmet medical need to include things like shortages. “I’ve looked at, for instance, what was the rationale for all nine of the choices and in all nine cases I could make a case that was unmet medical need,” he said of the initial awardees. Responding to BioSpace ’s request for comment on the second tranche of awardees, Grossman said in an email: “So far, we have less information about the new set and their intended use. Hopefully, they will meet the criteria I applied to the first batch: they address unmet medical needs that are legitimately on FDA’s priority list and can be justified without reference to separate dialogues about pricing and other national (but not necessarily FDA) priorities.”   A High Unmet Need For Disc and the EEP community, the unmet need is about as traditional as it gets. Bitopertin is a glycine transporter 1 inhibitor in confirmatory studies for EPP—a family of rare, debilitating and potentially life-threatening diseases caused by mutations that affect the heme synthesis pathway. In EEP, the partial defect occurs in the last enzyme, leading to the buildup of a chemical called protoporphyrin IX (PP9), which absorbs sunlight and generates dangerous free radicals, Quisel explained. Ultimately, patients spend their lives avoiding the sun. This build-up of PP9 can also lead to gallstones, gallbladder removal and liver damage. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! Clinical trial results, research news, the latest in cancer, cell and gene therapy hbspt.forms.create({ region: "na1", portalId: "4413123", formId: "674f4dc0-7371-4190-86cc-e1afd00b08ea", onFormSubmitted: function($form, data) { window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'event': 'GTMevent', 'event_name': 'newsletter_sign_up' }); } }); Quisel noted that Disc was “right on the cusp of submitting our [new drug application] anyway, so we knew that we would be providing the FDA with what we think is a complete file.” Disc filed an NDA seeking accelerated approval of bitopertin in EEP in September. Assuming accelerated review, Disc had anticipated launching bitopertin “sometime in the second half” of 2026, Quisel said. Now with a commissioner’s voucher in hand, the biotech is looking at potentially having a drug on the market late this year or early in 2026. “We were already building a lot of the executive functions around commercialization, selling a drug,” the CEO said. “But everything now has slipped into high gear to meet the moment.” However, according to Temkin, Disc—and the EEP community’s gain—could be a sticking point for other companies and patients, as the priority vouchers could potentially slow the path to market for other investigational drugs. “It is extremely resource intensive to do the kind of expedited review that is being imagined here, to the extent that we understand the contours of it,” she said. “I think the biggest con is probably that all of that attention by review teams means they’re not engaging on other applications. The pie doesn’t just get bigger.” The CNPV awardees are likely not the only ones who could feel they must deliver under this new program; so too could FDA reviewers. “There is a huge amount of pressure to approve or take action on something quickly, that can result in a less creative approach,” Temkin said. She suggested there could be less “back and forth” on the drug’s final label, and less opportunity to correct small problems along the way. During a typical six-month priority review period, there are usually a “series of meetings,” Temkin explained, during which the FDA has an opportunity to resolve questions. “If you have an extremely condensed review period, I suspect there’s less opportunity for that kind of engagement.”

Priority ReviewPhase 3

06 Nov 2025

·ir.discmedicine.com

Disc Medicine Reports Third Quarter 2025 Financial Results and Provides Business Update

Submitted a New Drug Application (NDA) for accelerated approval of bitopertin in erythropoietic protoporphyria (EPP) on September 29, 2025; Awarded the FDA Commissioner’s National Priority Voucher (CNPV), which is designed to shorten the expected NDA review period to 1-2 months from NDA acceptance Acceleration of ongoing efforts to support the potential earlier approval and commercialization of bitopertin for EPP in the US, expected in late 2025 or early 2026 Initial data from Phase 2 study of DISC-0974 in patients with anemia of myelofibrosis (MF) to be presented at the American Society of Hematology (ASH) conference in December Progressing ongoing Phase 2 study of DISC-3405 in polycythemia vera (PV) and initiated a Phase 1b study of DISC-3405 in sickle cell disease (SCD) in October Strong financial position ending Q3 with approximately $616 million in cash, cash equivalents, and marketable securities; completed a public offering in October 2025, with net proceeds of approximately $211M expected to extend cash runway into 2029 WATERTOWN, Mass., Nov. 06, 2025 (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 reported financial results for the third quarter ended September 30, 2025, and provided a review of recent program and corporate developments. “We are incredibly proud of the progress across our entire pipeline this quarter, most notably around our NDA submission for bitopertin in EPP at the end of September and subsequent receipt of the CNPV,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “Achieving this milestone is a strong testament to our team’s commitment to execution and dedication to bringing a potential new treatment option to the EPP patient community. We continue to work with the Agency on their review of our application, and we are furthering our commercial infrastructure in preparation for a potential launch.” “Beyond bitopertin, our broader pipeline is progressing well, as we expect new data from the Phase 2 trial of DISC-0974 in MF anemia by the end of the year and data from studies of DISC-3405 in PV and SCD next year. With a strong balance sheet providing cash runway into 2029, we are well-positioned to prepare for the anticipated launch of bitopertin and ultimately advancing towards our goal of delivering new treatment options to patients suffering from hematological diseases.” Recent Highlights and Anticipated Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Submitted NDA for bitopertin in EPP seeking priority review and accelerated approval with reduction in protoporphyrin IX (PPIX) as the surrogate endpoint for approval supported by clinical results from BEACON and AURORA Phase 2 trials Received the CNPV, which is designed to shorten the NDA review process to 1-2 months from NDA acceptance Accelerating activities to support a potential US approval and launch in late 2025 or early 2026 Progressing confirmatory Phase 3 APOLLO clinical trial of bitopertin in adults and adolescents with EPP DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Initial data from Phase 2 RALLY-MF trial of DISC-0974 in patients with anemia of myelofibrosis (MF) to be presented at ASH Annual Meeting in December, with topline data expected in 2026 Data from recently completed multiple-dose cohorts of Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD to be presented at ASN Kidney Week DISC-0974 was generally well-tolerated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin (EPO) levels Disc is assessing options for the program based on full analysis of the data Phase 2 study in patients with inflammatory bowel disease (IBD) and anemia anticipated to begin in Q1 2026 DISC-0974 was generally well-tolerated with substantial decreases in hepcidin, increases in iron, and improvements in markers of erythropoiesis Meaningful hemoglobin increases observed in only a subset of patients were in part driven by those with higher baseline erythropoietin (EPO) levels Disc is assessing options for the program based on full analysis of the data DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Progressing ongoing Phase 2 study of DISC-3405 in patients with PV with initial data expected in 2026 Initiated Phase 1b study of DISC-3405 in patients with SCD in October 2025 with initial data expected in 2026 Third Quarter 2025 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $615.9 million as of September 30, 2025. In October 2025, Disc completed a public offering with net proceeds of approximately $211 million, extending cash runway into 2029. Research and Development Expenses: R&D expenses were $50.3 million for the three months ended September 30, 2025, as compared to $24.7 million for the three months ended September 30, 2024. The increase in R&D expenses was primarily driven by the progression of Disc’s portfolio, including bitopertin’s clinical studies and drug manufacturing, the advancement of the DISC-0974 program, and increased headcount, as well as a payment of a $10.0 million milestone upon first administration to a patient in the Phase 2 trial of DISC-3405. Selling, General and Administrative Expenses: SG&A expenses were $17.4 million for the three months ended September 30, 2025, as compared to $8.2 million for the three months ended September 30, 2024. The increase in SG&A expenses was primarily due to increased headcount including establishing infrastructure to support potential commercialization. Net Loss: Net loss was $62.3 million for the three months ended September 30, 2025, as compared to $26.6 million for the three months ended September 30, 2024. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline. 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. Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at ir.discmedicine.com in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. 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 programs for bitopertin, DISC-0974 and DISC-3405, including projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the registrational pathway for bitopertin, including the potential for accelerated approval, benefits of the CNPV, expected review period and the timing of approval, if granted; anticipated discussions with regulatory agencies; ongoing preparations for the potential launch of bitopertin; and the strength of its financial position and its anticipated cash runway. 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, 2024, 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. Media Contact Peg Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com

Phase 2Financial StatementClinical ResultNDAPhase 1

21 Oct 2025

·ir.discmedicine.com

Disc Medicine Announces Pricing of $250 Million Upsized Public Offering of Common Stock and Pre-Funded Warrants

WATERTOWN, Mass., Oct. 21, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ: IRON) (Disc), 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 pricing of the upsized underwritten offering of shares of its common stock and, in lieu of common stock to certain investors that so choose, pre-funded warrants to purchase shares of its common stock. Disc is selling 2,619,049 shares of common stock and pre-funded warrants to purchase 59,523 shares of common stock and AI DMI LLC (the Selling Stockholder) is selling 297,619 shares of common stock in the offering. The shares of common stock are being sold at an offering price of $84.00 per share, and the pre-funded warrants are being sold at an offering price of $83.9999 per pre-funded warrant, which represents the per share offering price for the common stock less the $0.0001 per share exercise price for each such pre-funded warrant. The aggregate gross proceeds to Disc from this offering are expected to be approximately $225 million, before deducting underwriting discounts and commissions and other offering expenses, excluding the exercise of any pre-funded warrants. The aggregate gross proceeds to the Selling Stockholder from this offering are expected to be approximately $25 million, before deducting underwriting discounts and commissions. In addition, the Selling Stockholder has granted the underwriters a 30-day option to purchase up to an additional 446,428 shares at the public offering price, less underwriting discounts and commissions. The offering is expected to close on October 22, 2025, subject to the satisfaction of customary closing conditions. Disc intends to use the net proceeds from the offering to support the potential commercialization of bitopertin for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), to fund research and clinical development of its current or additional product candidates, and for working capital and other general corporate purposes. Disc will not receive any proceeds from the sale of shares to be offered by the Selling Stockholder. Jefferies, Leerink Partners, Morgan Stanley and Cantor are acting as joint book-running managers for the offering. Wedbush PacGrow and H.C. Wainwright & Co. are acting as co-managers for the offering. The offering is being made pursuant to an automatic shelf registration statement on Form S-3 (No. 333-281359) that was previously filed with the Securities and Exchange Commission (SEC) on August 8, 2024 and a resale registration statement on Form S-3 (No. 333-269270) that was previously filed with the SEC on January 18, 2023. This offering is being made only by means of a prospectus supplement and accompanying prospectuses that form a part of the registration statements. A final prospectus supplement and accompanying prospectuses related to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectuses relating to this offering may also be obtained, when available, by contacting: Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388, or by email at prospectus_department@jefferies.com; Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, (800) 808-7525 ext. 6105 or by email at syndicate@leerink.com; Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014; or Cantor Fitzgerald & Co., Attention: Capital Markets, 110 East 59th Street, 6th Floor, New York, New York, 10022, or by email at prospectus@cantor.com. This press release does not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that state or jurisdiction. 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. 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, without limitation, express or implied statements related to Disc’s expectations regarding the timing and closing of the offering, and the anticipated use of proceeds from the offering. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “seek,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release. These risks and uncertainties include fluctuations in Disc’s stock price, changes in market conditions, the satisfaction of customary closing conditions related to the underwritten offering, and other risks identified in our SEC filings, including our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, and in the prospectus supplement related to the offering we will file with the SEC. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. We disclaim any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Media Contact Peg Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com

Clinical Study

100 Deals associated with Bitopertin

External Link

KEGG	Wiki	ATC	Drug Bank
D10186	Bitopertin	-	DB12426

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Protoporphyria, Erythropoietic, X-Linked Dominant	NDA/BLA	United States	Disc Medicine, Inc.	30 Sep 2025
Porphyria, Erythropoietic	Phase 3	United States	Disc Medicine, Inc.	04 Apr 2025
Porphyria, Erythropoietic	Phase 3	Australia	Disc Medicine, Inc.	04 Apr 2025
Porphyria, Erythropoietic	Phase 3	Canada	Disc Medicine, Inc.	04 Apr 2025
Porphyria, Erythropoietic	Phase 3	France	Disc Medicine, Inc.	04 Apr 2025
Porphyria, Erythropoietic	Phase 3	Norway	Disc Medicine, Inc.	04 Apr 2025
Paranoid Schizophrenia	Phase 3	United States	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Argentina	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Australia	Hoffmann-La Roche, Inc.	11 Dec 2010
Paranoid Schizophrenia	Phase 3	Colombia	Hoffmann-La Roche, Inc.	11 Dec 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05308472 (AURORA, ASH2024) Manual	Phase 2	Protoporphyria, Erythropoietic	75	Bitopertin 20mg	uyebgrtetx(bljztdmxlh) = nearly all treatment-emergent adverse events were mild to moderate in intensity chriesdfji (enblklsfph ) View more	Positive	07 Dec 2024
				Bitopertin 60mg
ACTRN12622000799752 (BEACON, ASH2024) Manual	Phase 2	Protoporphyria, Erythropoietic	22	Bitopertin 20 mg	sixyqlassm(itpjjjdsgv) = mezmubjygx fywgshuxhg (csipttkjdr, 7.1) View more	Positive	07 Dec 2024
				Bitopertin 60 mg	sixyqlassm(itpjjjdsgv) = czfepofbds fywgshuxhg (csipttkjdr, 7.0) View more
ACTRN12622000799752 (BEACON, EHA2024) Manual	Phase 2	Protoporphyria, Erythropoietic PPIX	22	Bitopertin 20 mg	ovvvpavlii(xjogcrsitp) = aewsuowoex hlwbqffqdr (tnvbloalou ) View more	Positive	14 May 2024
NCT05308472 (AURORA, GlobeNewswire) Manual	Phase 2	Porphyria, Erythropoietic \| Protoporphyria, Erythropoietic	75	bitopertin 20 mg	wdiealueef(iuoydxyryq) = nmbuvafdem bmcwcexxul (zaqlxgcaqp ) Met View more	Positive	01 Apr 2024
				bitopertin 60 mg	wdiealueef(iuoydxyryq) = hbookauenx bmcwcexxul (zaqlxgcaqp ) Met View more
ACTRN12622000799752 (Beacon, ASH2023) Manual	Phase 2	Protoporphyria, Erythropoietic	17	Bitopertin	zhtrtakybd(mcetqtlvow) = xbrfvofgph pnznfzmrpl (gbzzhzetrg, 17) View more	Positive	11 Dec 2023
NCT01116830 (Pubmed \| J Clin Psychopharmacol) Manual	Phase 1	Schizophrenia	29	bitopertin	egafnlrbvy(cyatjueaea) = cpapscgaod ptbptrxnla (balztoqlgl, 7.4) View more	Negative	01 Aug 2017
				Placebo	egafnlrbvy(cyatjueaea) = pwjkdquzbp ptbptrxnla (balztoqlgl, 11.5) View more
NCT01613040 (Pubmed \| Clin Ther)	Phase 3	-	169	Bitopertin 30 mg	bhfkxjuqps(avyemichfc) = nnmtveffcx lkjlwchccy (tcojwbnuud )	-	01 Oct 2012
				Bitopertin 175 mg	bhfkxjuqps(avyemichfc) = spmvgwmjwj lkjlwchccy (tcojwbnuud )

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