IRON — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-07-30

• Selcodebart is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. Selcodebart is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the Un…

• evidence of target engagement, iron mobilization and augmented erythropoiesis. We initiated a Phase 1b/2 clinical trial in June 2022 in patients with anemia of MF, with results from the Phase 1b portion of this clinical trial showing substantial and sustained reductions in hepcidin levels and increa…

• In addition, we are developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. DISC-3405 is part of our iron homeostasis portfolio and is designed to induce hepcidin production and reduce serum iron l…

• As of June 30, 2026, we had cash, cash equivalents and marketable securities of $717.7 million. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without taki…

• Research and development expenses were $46.9 million for the three months ended June 30, 2026, compared to $46.3 million for the three months ended June 30, 2025. The increase of $0.6 million was driven by increases of $7.7 million and $3.1 million related to selcodebart (DISC-0974) and DISC-3405, r…

Text removed vs the prior filing · source: 10-Q · 2026-05-05

• DISC-0974 is the lead product candidate in our iron homeostasis portfolio and was licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We completed a Phase 1 clinical trial in healthy volunteers in the United…

• In addition, we are developing DISC-3405, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. DISC-3405 is part of our iron homeostasis portfolio and is designed to induce hepcidin production and reduce serum iron l…

• As of March 31, 2026, we had cash, cash equivalents and marketable securities of $730.2 million. We believe that our cash, cash equivalents and marketable securities will be sufficient to fund our current operating and capital expenditure plans and our debt service obligations into 2029, without tak…

• planning and market research; director and officer insurance costs and other insurance costs; and facility related expenses including maintenance and allocated expenses for rent and other operating costs.

• Research and development expenses were $45.9 million for the three months ended March 31, 2026, compared to $27.8 million for the three months ended March 31, 2025. The increase of $18.1 million was primarily due to an increase of $7.5 million in DISC-3405 development expenses. This increase was pri…

Text added vs the prior filing · source: 10-Q · 2026-07-30

• The FDA, the EMA and regulatory authorities in other countries have each expressed interest in further regulating biotechnology products. Agencies at both the federal and state level in the United States, as well as the U.S. Congressional committees and other governments or governing agencies, have …

• levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory and policy changes. Average review times at the agency have fluctuated in recent years as a result.

• We expect that these existing laws and other healthcare reform measures that may be adopted in the future may result in additional reductions in Medicare and other healthcare funding, more rigorous coverage criteria, new payment methodologies and additional

• downward pressure on the price that we receive for any approved product. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors, which may adversely affect our future profitability.

• Ensuring that our future business arrangements with third parties comply with applicable healthcare laws and regulations could involve substantial costs. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations …

Text removed vs the prior filing · source: 10-Q · 2026-05-05

• We received a Commissioner’s National Priority Voucher for bitopertin but the accelerated timeline for review of our NDA submission for bitopertin in EPP and XLP by the FDA did not result in an approval and it is unclear what, if any, benefits this voucher may provide in the future.

• In June 2025, the FDA announced the creation of a new program, the Commissioner’s National Priority Voucher, or CNPV, program, to expedite the development and approval of drug products with potential to address a major national priority, such as addressing a large unmet medical need, reducing downst…

• The FDA expects the CNPV program to accelerate the standard application review timeline after NDA acceptance from 10 months to 1-2 months by convening a multidisciplinary team of physicians and scientists for a team-based review, interacting frequently with the sponsor to clarify questions, and comp…

• On October 16, 2025, the FDA announced that bitopertin for porphyria was one of the first nine recipients of a CNPV. As a result, the NDA for bitopertin for EPP and XLP was eligible for the benefits of this program. However, receipt of the CNPV does not guarantee that the NDA will be approved on an …

• The FDA, the EMA and regulatory authorities in other countries have each expressed interest in further regulating biotechnology products. Agencies at both the federal and state level in the United States, as well as the U.S. Congressional committees and other governments or governing agencies, have …

Text added vs the prior filing · source: 10-Q · 2026-07-30

• (1) The aggregate number of shares available for sale cannot yet be determined, as it will be reduced by the number of shares sold to cover tax withholding obligations upon the vesting of Restricted Stock Units (RSUs). For disclosure purposes, the figures in this table reflect the maximum number of …

• On July 29, 2026, we entered into amended and restated employment agreements, effective as of July 29, 2026 (collectively, the “Amended Employment Agreements”), with each of John D. Quisel, J.D., Ph.D., our President and Chief Executive Officer; Jean Franchi, our Chief Financial Officer; Pamela Step…

• The amended and restated employment agreement with Dr. Quisel (the “Quisel Amended Employment Agreement”) provides for his continued at-will employment as our President and Chief Executive Officer, an annual base salary of $735,000, and a target annual bonus opportunity equal to 60% of base salary, …

• Consistent with the terms of Dr. Quisel’s prior employment agreement, the Quisel Amended Employment Agreement continues to provide that, if Dr. Quisel is terminated by us without Cause or resigns for Good Reason, in either case outside the period beginning three months before and ending 12 months af…

• COBRA coverage for up to 12 months following the termination date, subject to his continued copayment at the active employee rate.

Text removed vs the prior filing · source: 10-Q · 2026-05-05

• Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c)