FULC — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-04-27

• In February 2026, we announced updated 20 mg cohort results from the PIONEER trial as of the December 23, 2025 data cut off date showing:

• Indirect bilirubin decreased by 40% (vs. 37% at Week 12 in the 12 mg cohort)

• Lactate dehydrogenase decreased by 34% (vs. 28% at Week 12 in the 12 mg cohort)

• RBC distribution width decreased by 26% (vs. 27% at Week 12 in the 12 mg cohort)

• Reticulocyte counts decreased by 42% (vs. 31% at Week 12 in the 12 mg cohort)

Text removed vs the prior filing · source: 10-Q · 2025-10-29

• In July 2025, we announced results from the 12 mg dose cohort of our Phase 1b trial of pociredir in SCD, following conclusion of the 12-week treatment period (n=16). Results are as follows:

• Decreased red cell distribution width (mean decrease of 27%), indicating a more uniform red blood cell population

• Decreased reticulocyte counts (mean decrease of 30%), indicating healthier bone marrow function

• Mean hemoglobin concentration increased by 0.9 g/dL at 12 weeks of treatment with pociredir, from a baseline of 7.8 g/dL to 8.7 g/dL. Together with the observed decrease in reticulocyte counts, the increase in total hemoglobin indicates that pociredir decreased red blood cell destruction and showed …

• A trend of reduced VOC rates was observed during the study period (as assessed by VOCs reported as adverse events, or AEs), compared to cohort patients’ VOC frequency over the 6–12 months prior to enrollment. Eight of 16 patients (50%) reported no VOCs during the treatment period (12 weeks); three V…

Text added vs the prior filing · source: 10-Q · 2026-04-27

• pursue the discovery of drug targets for other genetically-defined rare diseases and the subsequent development of any resulting product candidates;

• we also had federal and state research and development tax credit carryforwards of $1.2 million and $0.2 million, respectively, which begin to expire in 2039. These net operating loss and tax credit carryforwards could expire unused and be unavailable to offset future income tax liabilities.

• In general, under Section 382 of the Code, or Section 382, and corresponding provisions of state law, a corporation that undergoes an “ownership change,” which is generally defined as a greater than 50% change, by value, in its equity ownership by certain stockholders over a three-year period, is su…

• We do not have any manufacturing facilities and expect to rely on third parties for the manufacture of pociredir for any future clinical trials and for the manufacture of any future product candidates for preclinical and clinical testing. We also expect to rely on third-party manufacturers or third-…

• In addition, legislative proposals are pending that, if enacted, could negatively impact U.S. funding for certain biotechnology providers having relationships with foreign adversaries or which pose a threat to national security. The potential downstream adverse

Text removed vs the prior filing · source: 10-Q · 2025-10-29

• pursue the discovery of drug targets for other genetically-defined rare diseases and the subsequent development of any resulting product candidates, including for the potential treatment of bone marrow failure syndromes, such as DBA, 5q deletion syndrome, Shwachman-Diamond syndrome, and Fanconi anem…

• make any milestone payments to CAMP4 under our license agreement with CAMP4;

• In general, under Section 382 of the Code, or Section 382, and corresponding provisions of state law, a corporation that undergoes an “ownership change,” which is generally defined as a greater than 50% change, by value, in its equity ownership by certain stockholders over a three-year period, is su…

• We do not have any manufacturing facilities. Although we believe we have obtained sufficient quantities of pociredir from a CMO for the completion of our Phase 1b clinical trial for SCD, we cannot be sure we have correctly estimated our drug product requirements, which could delay, prevent or impair…

• We expect to rely on third parties for the manufacture of pociredir for any future clinical trials and for the manufacture of any future product candidates for preclinical and clinical testing. We also expect to rely on third-party manufacturers or third-party collaborators for the manufacture of co…

Text added vs the prior filing · source: 10-Q · 2026-04-27

• On March 27, 2026, Alan Ezekowitz, a member of our board of directors, terminated a Rule 10b5-1 trading arrangement he had previously adopted on December 29, 2025 with respect to the sale of up to 124,713 shares of our common stock. The trading plan was intended to satisfy the affirmative defense co…

Text removed vs the prior filing · source: 10-Q · 2025-10-29

• On August 1, 2025, Robert J. Gould, a member of our board of directors, adopted a trading plan for the potential sale of up to 180,000 shares of our common stock. The trading plan is intended to satisfy the affirmative defense conditions of Rule 10b5-1(c) under the Exchange Act and is expected to re…

• On August 8, 2025, Curtis Oltmans, our chief legal officer, adopted a trading plan for the potential sale of up to 12,186 shares of our common stock and for the potential exercise of vested stock options and the associated sale of up to 40,000 shares of our common stock. The trading plan is intended…