This section highlights FDA-related milestones and regulatory updates for drugs developed by Fulcrum Therapeutics (FULC).
Over the past two years, Fulcrum Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
pociredir, FTX-6274, and Losmapimod. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Pociredir FDA Regulatory Timeline and Events
Pociredir is a drug developed by Fulcrum Therapeutics for the following indication: In sickle cell disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- pociredir
- Announced Date:
- February 24, 2026
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics, today reported positive 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD).
AI Summary
Fulcrum Therapeutics reported positive 12‑week results from the 20 mg cohort of its Phase 1b PIONEER trial of pociredir in sickle cell disease. The cohort showed rapid, robust fetal hemoglobin (HbF) induction: mean absolute HbF rose 12.2 percentage points from a 7.1% baseline to 19.3% at Week 12. The data also showed improvements in markers of hemolysis and anemia, movement toward pan‑cellular HbF distribution, and encouraging trends toward fewer vaso‑occlusive crises (VOCs).
Based on these results, Fulcrum plans to start a potential registration‑enabling trial in the second half of 2026. The company ended 2025 with $352.3 million in cash, cash equivalents, and marketable securities, which it says provides a cash runway into 2029 to support further development of pociredir and its benign hematology programs.
Read Announcement- Drug:
- pociredir
- Announced Date:
- February 17, 2026
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, will host a conference call and webcast on Tuesday, February 24, 2026 beginning at 8:00 a.m. ET to present 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease.
AI Summary
Fulcrum Therapeutics, a clinical-stage biopharmaceutical company developing small molecules for genetically defined rare diseases, will host a conference call and webcast on Tuesday, February 24, 2026, at 8:00 a.m. ET. The company will present 12-week results from the 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease, reviewing safety, tolerability and early signs of activity at this dose and implications for further development.
Members of Fulcrum management will be joined by Dr. Martin Steinberg, Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine at Boston University Chobanian and Avedisian School of Medicine. To register, click here or visit the "Events and Presentations" section of Fulcrum’s website. A replay will be available on the company website after the event. Media contact: Kevin Gardner, LifeSci Advisors, LLC, [email protected], 617-283-2856.
Read Announcement- Drug:
- pociredir
- Announced Date:
- December 7, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics, Inc. reported positive initial results from the ongoing 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD).
AI Summary
Fulcrum Therapeutics reported positive initial results from the ongoing 20 mg dose cohort of the Phase 1b PIONEER trial of pociredir in adults with severe sickle cell disease. At Week 6 (n=12), mean absolute fetal hemoglobin (HbF) rose from 7.1% to 16.9% (a 9.9% increase), and 7 of 12 patients (58%) reached absolute HbF ≥20%. Among the six patients who reached Week 12 by the data cutoff, mean HbF induction exceeded 3.75-fold versus a 2.4-fold induction seen in the earlier 12 mg cohort, showing a clear dose response.
Early signs pointed to pan‑cellular HbF induction: F‑cells rose from 31% at baseline to 58% at Week 6 (n=9). Markers of hemolysis and anemia improved (indirect bilirubin and LDH down 37%, reticulocytes down 33%, RDW down 22%), and mean hemoglobin increased by 0.8 g/dL. Eight of 12 patients (67%) reported no vaso‑occlusive crises during treatment.
Pociredir was generally well tolerated in the 20 mg cohort with no treatment‑related serious adverse events reported. All 12 patients are expected to complete 12 weeks of treatment, with updated results planned in Q1 2026.
Read Announcement- Drug:
- pociredir
- Announced Date:
- December 2, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics, to review new clinical data from the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD).
AI Summary
Fulcrum Therapeutics will host an investor event on Sunday, December 7, 2025 at 7:00 a.m. ET to review new clinical data from the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD). The data will also be presented at the 67th American Society of Hematology (ASH) Annual Meeting. Fulcrum management will be joined by Dr. Sheinei Alan and Dr. Martin Steinberg for the presentation and discussion.
The company will share initial clinical results from the 20 mg dose cohort and full data from the 12 mg cohort. A live question-and-answer session will follow the formal presentation. Investors and other interested parties can register on Fulcrum’s website, and a replay will be posted after the event.
Pociredir is an oral small-molecule inhibitor of EED designed to raise fetal hemoglobin (HbF) by downregulating repressors like BCL11A. Early PIONEER results showed proof-of-concept with HbF increases and, through the 12 mg cohort, generally good tolerability up to three months with no treatment-related serious adverse events reported.
Read Announcement- Drug:
- pociredir
- Announced Date:
- July 29, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics, today reported positive results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD).
AI Summary
Fulcrum Therapeutics reported positive results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease. Pociredir was generally well-tolerated. There were no drug-related serious adverse events or discontinuations, and all treatment-related side effects were mild (Grade 1).
Patients experienced a rapid and pan-cellular mean increase of 8.6% in fetal hemoglobin (HbF), with 67% of red blood cells converting to F-cells. Key markers of hemolysis and anemia improved, and total hemoglobin rose by 0.9 g/dL. Early data also showed encouraging trends in vaso-occlusive crises.
Fulcrum plans to share results from the ongoing 20 mg dose cohort by the end of 2025. At the close of Q2 2025, the company held $214.1 million in cash and marketable securities. This runway supports operations into 2028. The company aims to advance pociredir into later-stage trials.
Read Announcement- Drug:
- pociredir
- Announced Date:
- July 28, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics to present topline results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease. Members of Fulcrum management will be joined by Dr. Sheinei Alan, Director of the Inova Fairfax Adult Sickle Cell Program, and Assistant Professor at UVA School of Medicine Inova Campus, and Dr. Wally Smith, Director at the VCU Adult Sickle Cell Program and Florence Neal Cooper Smith Professor of Sickle Cell Disease at Virginia Commonwealth University.
AI Summary
Fulcrum Therapeutics will host a conference call and webcast on Tuesday, July 29, 2025 at 8:00 a.m. ET to share topline results from the 12 mg dose cohort of its Phase 1b PIONEER trial of pociredir in sickle cell disease. Fulcrum management will discuss safety, tolerability and early measures of fetal hemoglobin response.
Joining the call are Dr. Sheinei Alan, Director of the Inova Fairfax Adult Sickle Cell Program and Assistant Professor at the UVA School of Medicine Inova Campus, and Dr. Wally Smith, Director of the VCU Adult Sickle Cell Program and Florence Neal Cooper Smith Professor of Sickle Cell Disease at Virginia Commonwealth University. Both experts will offer insights on how pociredir’s mechanism may benefit patients by boosting fetal hemoglobin and reducing sickling.
Investors and researchers can register via Fulcrum’s website in the “Events and Presentations” section. A replay will be available for on-demand listening after the event.
Read Announcement- Drug:
- pociredir
- Announced Date:
- May 29, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics announced upcoming presentations at the 5th Global Congress on Sickle Cell Disease and the 2025 European Hematology Association Congress.
AI Summary
Fulcrum Therapeutics, a clinical-stage biopharmaceutical company focused on rare genetic diseases, announced upcoming presentations at two major hematology conferences. At the 5th Global Congress on Sickle Cell Disease in Abuja, Nigeria (June 3-6, 2025), the company will share preclinical and early clinical data on pociredir, an oral drug aimed at increasing fetal hemoglobin for treating sickle cell disease. Two poster presentations will focus on both the drug's early results and the design of a Phase 1b, open-label study involving multiple doses in sickle cell patients.
Additionally, at the 2025 European Hematology Association Congress in Milan, Italy (June 12-15, 2025), Fulcrum will present findings from a Phase 1 study examining the pharmacokinetics, pharmacodynamics, and safety of pociredir in healthy adults and in animal models. Copies of these presentations will later be available on the company website.
Read Announcement- Drug:
- pociredir
- Announced Date:
- February 25, 2025
- Indication:
- In sickle cell disease
Announcement
Fulcrum Therapeutics, announced full year of 2024 as well as a business update.
AI Summary
Fulcrum Therapeutics reported its full year 2024 financial results along with an important business update. For 2024, the company ended the year with $241 million in cash, cash equivalents, and marketable securities, supporting a cash runway that extends at least into 2027. The business update highlighted progress on its lead program, pociredir, for treating sickle cell disease. The Phase 1b PIONEER trial is moving forward with strong momentum, having enrolled ten patients in the 12 mg dose cohort so far. Key clinical data is expected to be shared in mid-2025 for the 12 mg cohort and by the end of 2025 for the 20 mg cohort. This advancement signals potential for pociredir to increase fetal hemoglobin levels, which may offer a new oral treatment option and transform the management of sickle cell disease.
Read Announcement
FTX-6274 FDA Regulatory Events
FTX-6274 is a drug developed by Fulcrum Therapeutics for the following indication: EED, inhibitor, in castration-resistant prostate cancer.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- FTX-6274
- Announced Date:
- October 14, 2025
- Indication:
- EED, inhibitor, in castration-resistant prostate cancer.
Announcement
Fulcrum Therapeutics, nnounced that it will present preclinical data for FTX-6274, an oral embryonic ectoderm development, or EED, inhibitor, in castration-resistant prostate cancer.
AI Summary
Fulcrum Therapeutics announced it will present new preclinical data showing that FTX-6274, an oral embryonic ectoderm development (EED) inhibitor, has strong activity against castration-resistant prostate cancer. The data will be shared at the ESMO Congress 2025 in Berlin on October 18, where researchers will detail how FTX-6274 blocks cancer cell growth in laboratory models.
Jeff Jacobs, Fulcrum’s Chief Scientific Officer, said the findings highlight EED inhibition’s promise beyond their current blood disease programs. He noted that a strategic partnership could help advance FTX-6274 into clinical testing for oncology patients. The poster, led by Senior Scientist Suchi Raghunathan, will remain available on Fulcrum’s website after the congress.
Fulcrum is a clinical-stage company focused on small molecules that adjust gene expression to treat rare diseases. Its lead program, pociredir, targets sickle cell disease by boosting fetal hemoglobin production.
Read Announcement
Losmapimod (ReDUX4) FDA Regulatory Events
Losmapimod (ReDUX4) is a drug developed by Fulcrum Therapeutics for the following indication: Facioscapulohumeral muscular dystrophy (FSHD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Losmapimod (ReDUX4)
- Announced Date:
- September 12, 2024
- Indication:
- Facioscapulohumeral muscular dystrophy (FSHD)
Announcement
Fulcrum Therapeutics, announced that its Phase 3 REACH trial evaluating losmapimod in patients with FSHD, did not achieve its primary endpoint of change from baseline in RSA with losmapimod compared to placebo.
AI Summary
Fulcrum Therapeutics announced that its Phase 3 REACH trial for losmapimod in patients with facioscapulohumeral muscular dystrophy (FSHD) did not meet its primary endpoint. The study measured change from baseline in relative surface area (RSA), which reflects the reachable workspace, and the results showed no significant improvement for those treated with losmapimod compared to placebo. Secondary endpoints, like muscle fat infiltration and shoulder strength, also did not show meaningful differences between the groups.
Due to these disappointing results, Fulcrum has decided to suspend its losmapimod program for FSHD. The company expressed its deep disappointment over the trial outcomes and plans to complete a full evaluation of the data. Despite this setback, Fulcrum’s robust cash resources will support its ongoing efforts to develop treatments for other diseases with high unmet needs.
Read Announcement- Drug:
- Losmapimod (ReDUX4)
- Announced Date:
- September 9, 2024
- Indication:
- Facioscapulohumeral muscular dystrophy (FSHD)
Announcement
Fulcrum Therapeutics ahead of the company’s phase 3 losmapimod readout, expected by the end of October for Facioscapulohumeral muscular dystrophy (FSHD).
Read Announcement
