Bitopertin

Completed Type A meeting with the FDA on bitopertin and aligned that the Phase 3 APOLLO study, if successful, can serve as the basis for CRL response; topline data expected in Q4 2026 Launched Expanded Access Program (EAP) for bitopertin for eligible erythropoietic protoporphyria (EPP) patients Presented positive clinical data updates from the RALLY-MF Phase 2 trial of selcodebart (DISC-0974) in anemia of myelofibrosis and the HELIOS open-label extension trial of bitopertin in EPP Completed enrollment for RESTORE-PV Phase 2 study of DISC-3405 in polycythemia vera (PV) with initial data expected in Q3 2026 and progressing Phase 1b study of DISC-3405 in sickle cell disease (SCD) with initial data expected in Q4 2026 Strong financial position ending Q2 with approximately $718 million in cash, cash equivalents, and marketable securities, providing runway into 2029 WATERTOWN, Mass., July 30, 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 reported financial results for the second quarter ended June 30, 2026, and provided a review of recent program and corporate developments. “The anticipated readout of the Phase 3 APOLLO trial of bitopertin in EPP in Q4 marks a significant milestone. We have continued driving forward on this program over the past quarter with the launch of our EAP,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. “We also look forward to significant advancement across the rest of our pipeline in the second half of 2026. We saw strong demand for the Phase 2 RESTORE-PV trial of DISC-3405 in polycythemia vera, which is now fully enrolled with initial data coming ahead of schedule in Q3. Along with an expected end of Phase 2 FDA interaction on selcodebart in MF anemia in Q4, we will potentially be positioned to advance two more programs into pivotal-stage development in 2027.” Recent Highlights and Anticipated Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Completed Type A meeting with the FDA to discuss the CRL for bitopertin in erythropoietic protoporphyria (EPP) and aligned that the Phase 3 APOLLO study, if successful, can serve as the basis for CRL response and could potentially support a traditional approval Presented updated data from the HELIOS open-label extension trial of bitopertin in EPP at the European Hematology Association (EHA) Annual Meeting, demonstrating sustained reductions in protoporphyrin IX (PPIX), significant improvement in light tolerance measures, and favorable longer-term safety Launched an Expanded Access Program (EAP) for bitopertin in the US and select other geographies, providing eligible patients with EPP and XLP access to treatment prior to a regulatory decision On track to report APOLLO topline results in Q4 2026 and expect to submit CRL response and receive an FDA decision by mid-2027 APOLLO completed enrollment in March 2026 and enrolled ahead of schedule with a final N of 183 Selcodebart (DISC-0974): Anti-Hemojuvelin Antibody (Hepcidin Suppression) Shared updated data from RALLY-MF trial of selcodebart in anemia of myelofibrosis (MF) in oral presentations at the American Society of Clinical Oncology (ASCO) and European Hematology Association (EHA) annual meetings, solidifying selcodebart’s differentiated emerging pro MF. Data showed: Major anemia response rate of 56% and overall anemia response rate of 72% across evaluable patients Similar, strong response rates across transfusion cohorts and with or without background JAK inhibitor therapy Additional data from RALLY-MF anticipated in Q4 2026, with an end-of-Phase 2 meeting with the FDA expected to occur by year-end Received EU Orphan Drug Designation for selcodebart for the treatment of myelofibrosis Progressing Phase 2 study in patients with inflammatory bowel disease (IBD) with initial data expected in 2027 DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Completed enrollment for RESTORE-PV Phase 2 study in patients with polycythemia vera with initial data expected in Q3 2026 Progressing Phase 1b study in patients with sickle cell disease with initial data expected in Q4 2026 Second Quarter 2026 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $717.7 million as of June 30, 2026, which are expected to fund operational plans into 2029. Research and Development Expenses: R&D expenses were $46.9 million for the three months ended June 30, 2026, as compared to $46.3 million for the three months ended June 30, 2025. The increase in R&D expenses was primarily driven by the progression of Disc's portfolio, including the advancement of the selcodebart (DISC-0974) and DISC-3405 clinical studies and drug manufacturing, as well as increased headcount. These increases were partially offset by a decrease in manufacturing costs related to bitopertin and a $10.0 million milestone payment incurred upon initiation of the APOLLO study during the comparative period. Selling, General and Administrative Expenses: SG&A expenses were $18.1 million for the three months ended June 30, 2026, as compared to $15.1 million for the three months ended June 30, 2025. The increase in SG&A expenses was primarily due to increased headcount. Net Loss: Net loss was $59.5 million for the three months ended June 30, 2026, as compared to $55.2 million for the three months ended June 30, 2025. 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 . 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, selcodebart (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 traditional approval, the potential for the APOLLO clinical trial to serve as the basis for the CRL response and any such approval, and the timing of any such approval, if granted; anticipated discussions with regulatory agencies; 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, 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. Media Contact Peg Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com DISC MEDICINE, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In thousands, except share and per share amounts) (Unaudited) ​ Three months ended June 30, Six Months Ended June 30, ​ 2026 ​ 2025 ​ 2026 ​ 2025 Operating expenses: ​ ​ ​ ​ Research and development ​ $ 46,933 $ 46,319 $ 92,837 $ 74,082 Selling, general and administrative ​ 18,142 15,091 41,762 27,274 Total operating expenses ​ 65,075 ​ 61,410 ​ 134,599 ​ 101,356 Loss from operations ​ (65,075 ) ​ (61,410 ) ​ (134,599 ) ​ (101,356 ) Other income (expense), net 5,612 ​ 6,215 ​ 11,683 ​ 12,195 Income tax expense ​ (69 ) (52 ) (120 ) (171 ) Net loss ​ $ (59,532 ) ​ $ (55,247 ) ​ $ (123,036 ) ​ $ (89,332 ) Net loss per share, basic and diluted ​ $ (1.54 ) ​ $ (1.58 ) ​ $ (3.19 ) ​ $ (2.61 ) Weighted-average common shares outstanding, basic and diluted ​ 38,685,565 35,024,592 38,589,035 34,179,364 DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) ​ June 30, 2026 December 31, 2025 (Unaudited) Assets ​ Cash, cash equivalents, and marketable securities ​ $ 717,746 $ 791,152 Other current assets ​ 15,015 12,746 Total current assets ​ 732,761 803,898 Non-current assets ​ 2,310 2,981 Total assets ​ $ 735,071 $ 806,879 Liabilities and Stockholders’ Equity ​ Current liabilities ​ $ 30,360 $ 36,641 Non-current liabilities ​ 60,547 30,412 Total liabilities ​ 90,907 67,053 Total stockholders’ equity ​ 644,164 739,826 Total liabilities and stockholders’ equity ​ $ 735,071 $ 806,879

iStock, Zerbor A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations. While the U.S. continues to be the world leader in developing treatments for rare diseases, other countries—China especially—are catching up and could ultimately cause Americans to miss out on crucial medicines, a group of Congressional advisors argues. “Developing novel rare disease treatments in the United States is critical to improving U.S. health outcomes, as well as ensuring U.S. national security,” according to a Tuesday report published by the National Security Commission on Emerging Biotechnology (NSCEB), a legislative advisory board convened by Congress to assess how advances in biotech could direct national defense initiatives. While the U.S. still currently comes ahead of other countries in rare disease innovation, the NSCEB warned that the country’s “lead is eroding” due to its slow and expensive system of drug development. China, meanwhile, has rapidly grown as a key biotech innovator on the global stage, fueled largely by its “accelerated clinical trial pathways and active recruitment” of participants, the group wrote in the report. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); Indeed, the Asian nation has hosted more clinical trials than the U.S. since 2020, up from less than 8% in 2010, according to an April report by Stanford University. If the U.S. fails to keep up with China’s progress, largely through policy changes, then “China’s competitive edge in biopharmaceuticals may soon expand into an insurmountable lead and point of geopolitical leverage,” the NSCEB cautioned. Editorial Biotech’s China problem: An issue of national security or economic value for the US? Congressional letters sent to the CEOs of Eli Lilly, Pfizer, Merck, BMS and AbbVie this week voicing concerns about the pharmas’ clinical trials in China highlight an ongoing discrepancy in how government and industry think about the rise of the Asian country’s biotech industry. July 2, 2026 · 6 min read · Jef Akst Read more The U.S. federal government has put in place some policies designed to tamp down on the industry’s Chinese dealmaking rather than bolster drug development. Last year, for instance, president Donald Trump greenlit the BIOSECURE Act , which targets certain Chinese “companies of concern,” flagged as those who could pose national security harms to the U.S., though the final text of the law didn’t name these companies. But initiatives are also underway to expedite drug development in the U.S. Last month, the FDA announced a pilot program to improve clinical trial protocols, with an eye toward minimizing the time from drug identification to a first-in-human study, as well as improving the overall quality of new drug submissions. The regulator has also recently unveiled rare disease–specific policies. In February, for instance, the agency released draft guidance for its plausible mechanism pathway for personalized genetic medicines for diseases affecting very few patients. This framework opens up the possibility of approval for drugs that can demonstrably target a condition’s underlying biological cause. FDA Busy FDA gives rare disease sector complementary pathways, unanswered questions The FDA has expanded the regulatory toolkit during President Donald Trump’s second term, adding new mechanisms for rare diseases while putting the Biden-era platform technology designation into action. June 8, 2026 · 8 min read · Nick Paul Taylor Read more Although the report doesn’t mention these specific programs, the NSCEB does offer policy recommendations that are in line with the FDA’s approach, meant to remove barriers for the industry instead of restricting pipeline development. The commission suggests, for example, that Congress urge the FDA to finalize its platform technology designation program, which allows for the carryover of validated data across programs based on a specific platform. The FDA launched this initiative in 2024 and named Sarepta as the first awardee in June 2025. The biotech’s award was revoked a month later, however, after three deaths were linked to Sarepta’s gene therapy platform. Krystal Biotech won the second platform designation last October. Congress should also find ways to allow novel trial designs for small patient populations, the NSCEB suggests—otherwise a key stumbling block of many rare disease programs. To this end, the FDA in September 2025 unveiled the Rare Disease Evidence Principles . Under this effort, the agency will work with drug sponsors, patients and experts to determine what evidence will be taken into consideration for approval. Only drugs that affect fewer than 1,000 patients in the U.S. are eligible for this program. Likewise key to the rare disease sector is FDA stability, according to Tuesday’s report, which flagged the “persistent staffing shortages and knowledge gaps” at the regulator that render it unable to properly assess emerging technologies and therapies. Notably, thousands of employees were laid off or voluntarily left the FDA last year amid Health Secretary Robert F. Kennedy Jr.’s efforts to overhaul the agency. Nearly 2,000 more staff were expected to get the boot this year, according to a fact-sheet of the regulator’s 2026 budget. The topmost ranks at the FDA have also undergone considerable churn in recent months, marked by the recent departure of Commissioner Marty Makary, as well as his controversial deputies Vinay Prasad and Tracy Beth Høeg . There is some light on this horizon, however, as the FDA is looking to hire some looking to hire some 2,200 people, Acting Chief of Staff and Deputy Commissioner Lowell Zeta said at BIO’s International Convention last month. Under the previous FDA leadership, the rare disease space suffered from contentious decisions and inconsistencies. In February, for instance, Disc Medicine failed to secure an approval for bitopertin, being proposed for a rare blood disorder, amid reports of skepticism from Prasad. In November 2025, the FDA said Phase 1/2 data were no longer enough to support a filing for its uniQure’s Huntington’s disease gene therapy—despite having previously signed off on the biotech’s protocols. The agency has since reversed that reversal , paving the way for uniQure to accelerated approval, which the company plans to do in the third quarter. Huntington’s disease UniQure plans Q3 submission for Huntington’s gene therapy after FDA reverses course—again After a regulatory odyssey that delayed a filing for what would be the first genetic medicine for Huntington’s disease, the FDA has agreed that three-year data from uniQure’s Phase 1/2 trial are sufficient to support an accelerated biologics license application. June 17, 2026 · 4 min read · Heather McKenzie Read more