On August 7, 2025 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, reported financial results for the second quarter ended June 30, 2025, and provided a recap of recent program and corporate developments (Press release, Disc Medicine, AUG 7, 2025, View Source [SID1234654973]).
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"We are pleased with the continued momentum this past quarter, highlighted by clinical data presentations that support further advancement across our programs and continued progress toward our first NDA submission. Following positive feedback from our pre-NDA meeting, we are on track to submit an NDA for bitopertin in EPP under the accelerated approval pathway in October," said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc. "We have also made great progress across the rest of our pipeline, initiating a Phase 2 study of DISC-3405 in polycythemia vera, and looking ahead to data from the Phase 2 trial of DISC-0974 in MF anemia and Phase 1b trial in NDD-CKD anemia in the second half of the year. Our team’s commitment to execution, supported by a strong balance sheet that provides cash runway into 2028, has positioned Disc to prepare for the potential commercialization of bitopertin and advance pipeline development as we enter the next phase of growth."
Recent Highlights and Anticipated Milestones:
Bitopertin: GlyTI Inhibitor (Heme Synthesis Modulator)
Presented data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers
Progressing confirmatory Phase 3 APOLLO clinical trial of bitopertin in adults and adolescents with EPP
Completed positive pre-NDA meeting with FDA, confirming alignment with the agency on the expected timing, format, and content of planned NDA submission for bitopertin in EPP
Expect to submit an NDA in October 2025 under the FDA’s accelerated approval pathway based on Disc’s existing data package
Publication of the results from a preclinical study conducted in collaboration with Boston Children’s Hospital showing that, in mice, bitopertin may help prevent liver disease in EPP, in addition to ameliorating blood PPIX levels. The paper, "The GLYT1 inhibitor bitopertin mitigates erythroid PPIX production and liver disease in erythroid protoporphyria," was published in the Journal of Clinical Investigation (corresponding authors Sarah Ducamp and Paul Schmidt)
DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression)
Hosted a virtual MF Anemia KOL event on May 9, 2025, discussing DISC-0974 and its potential to play a significant role in the treatment of anemia in patients with MF
A replay of the webcast is available on the Events & Presentations page on the investor relations portion of the Company website
Presented clinical data from the continuation phase of the Phase 1b trial of DISC-0974 in MF anemia demonstrating durable hematologic response at EHA (Free EHA Whitepaper) 2025
Progressing RALLY-MF Phase 2 study of DISC-0974 in patients with anemia of MF with initial data expected in Q4 2025
Exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled, and trial protocol updated to allow patients on these therapies into the main study cohorts
Progressing Phase 1b study of DISC-0974 in patients with anemia of NDD-CKD with multiple-dose data expected in Q4 2025
DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction)
Presented updated SAD/MAD data from the Phase 1 trial of DISC-3405 in healthy volunteers providing proof of mechanism to support advancement of the program at EHA (Free EHA Whitepaper) 2025
Initiated a Phase 2 study of DISC-3405 in patients with PV with initial data expected in 2026
Corporate:
Appointed Nadim Ahmed, President and CEO of Cullinan Therapeutics, to the Company’s Board of Directors in July, bringing to the Company over 25 years of development and commercial leadership experience including multiple product launches in the hematology space
Second Quarter 2025 Financial Results:
Cash Position: Cash, cash equivalents, and marketable securities were $650.0 million as of June 30, 2025, which are expected to fund operational plans into 2028.
Research and Development Expenses: R&D expenses were $46.3 million for the three months ended June 30, 2025, as compared to $23.5 million for the three months ended June 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 million milestone upon initiation of the APOLLO study.
Selling, General and Administrative Expenses: SG&A expenses were $15.1 million for the three months ended June 30, 2025, as compared to $7.4 million for the three months ended June 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 $55.2 million for the three months ended June 30, 2025, as compared to $26.4 million for the three months ended June 30, 2024. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline.