Drugs / Pociredir
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold sickle cell S-Lepore disease 1 milestone | |||||
| Clinical hold | US (FDA) | — | sickle cell S-Lepore disease | 2023-02-23 | “On February 23, 2023, the FDA placed the investigational new drug (IND) application for FTX-6058 for the potential treatment of SCD on full clinical hold.” fulcrumtx.com ↗ “full clinical hold regarding the Investigational New Drug (IND) application for FTX-6058 for the potential treatment of sickle-cell disease” fulcrumtx.com ↗ |
| Filed Indication not stated 1 milestone | |||||
| Filed | US (FDA) | — | other hemoglobinopathies, beta-thalassemia | 2022-01-10 | “Submitted Investigational New Drug (IND) application to initiate clinical development of FTX-6058 in select hemoglobinopathies, including beta-thalassemia.” fulcrumtx.com ↗ “Submitted IND application to initiate clinical development of FTX-6058 in non-SCD hemoglobinopathies, including beta-thalassemia.” fulcrumtx.com ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials · 2 met primary | ||||||
| Phase 1 | NCT07431398 | Dec 2025 → Jun 2026 | sickle cell disease | Fulcrum Therapeutics | Terminated | No outcome recorded Stop: Business |
| Phase 1 | NCT05169580 | Dec 2021 → Jan 2026 | sickle cell disease | Fulcrum Therapeutics | Completed | Met primary |
| Phase 1 | NCT04586985 | Oct 2020 → Nov 2022 | sickle cell disease | Fulcrum Therapeutics | Completed | Met primary |
| Phase 21 trial | ||||||
| Phase 2 | NCT07401823 | Mar 2026 → Feb 2030 expected | sickle cell disease | Fulcrum Therapeutics | Enrolling by invitation | No outcome recorded |
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2026-02-24 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Fourth Quarter and Full Year 2025 fulcrumtx.com ↗ |
| 2026-02-24 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Positive 12-Week Results from the 20 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease fulcrumtx.com ↗
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). |
| 2025-12-06 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Positive Initial Results from the 20 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease at the 67th American Society of Hematology Annual Meeting fulcrumtx.com ↗
At just six weeks of treatment, we have observed robust and clinically meaningful increases in fetal hemoglobin with the majority of patients achieving absolute HbF levels ≥20%. |
| 2025-07-29 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Results from the 12 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease fulcrumtx.com ↗
We are highly encouraged by these data from the 12 mg cohort of the PIONEER trial and believe they represent an important milestone in our mission to transform the treatment of sickle cell disease. |
| 2025-07-29 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Second Quarter 2025 fulcrumtx.com ↗
Announced results from the 12 mg dose cohort (n=16) of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD); pociredir was generally well-tolerated with no treatment-related serious adverse events (SAEs) |
| 2023-08-22 | Fulcrum Therapeutics | Regulatory FDA Lifts Clinical Hold on Fulcrum Therapeutics’ FTX-6058 for Sickle Cell Disease fulcrumtx.com ↗
Fulcrum Therapeutics, Inc. ® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Investigational New Drug (IND) application for FTX-6058 for the potential treatment of sickle-cell disease (SCD). |
| 2023-02-24 | Fulcrum Therapeutics | Regulatory Fulcrum Therapeutics Announces Clinical Hold on FTX-6058 in Sickle Cell Disease fulcrumtx.com ↗ |
| 2022-08-11 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Second Quarter 2022 Financial Results and Business Update fulcrumtx.com ↗
Announced data from initial patients in first cohort of Phase 1b trial of FTX-6058 in SCD; achieved HbF increases of up to 6.3% over baseline, showing proof-of-concept for FTX-6058 as an oral HbF inducer. |
| 2022-06-10 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial fulcrumtx.com ↗
Initial data from the first subjects to receive a 6 mg dose of FTX-6058, an oral HbF inducer, showed a rapid and robust induction of HbF, and subjects achieved increases of up to 6.3% over baseline. |
| 2021-12-06 | Fulcrum Therapeutics | Results Fulcrum Therapeutics® Announces Additional HBG mRNA Induction from Higher Dose Cohorts in Phase 1 Healthy Adult Volunteer Trial of FTX-6058 for Sickle Cell Disease and New Preclinical Mechanism Data fulcrumtx.com ↗
Data from the 20mg and 30mg dose cohorts demonstrated a mean 5.6-fold induction and a mean 6.2-fold induction in HBG mRNA, respectively, at day 14. |
| 2021-08-10 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Reports Recent Business Highlights and Second Quarter 2021 Financial Results fulcrumtx.com ↗
Reported positive interim results from ongoing Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease demonstrating proof of mechanism and proof of biology |
| 2021-08-10 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Positive Interim Results from Phase 1 Healthy Adult Volunteer Trial of FTX-6058 for Sickle Cell Disease fulcrumtx.com ↗
Results from the MAD portion of the trial demonstrated proof of biology as evidenced by a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes). |
| 2021-04-09 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the American Chemical Society (ACS) Spring 2021 Virtual Conference fulcrumtx.com ↗
We are also excited to report initial PK results from the SAD cohort and that our Phase 1 trial in healthy volunteers continues to progress with initiation of the multiple ascending dose cohorts. |
| 2020-12-05 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Presents Updated Data on Sickle Cell Disease Program at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition fulcrumtx.com ↗
Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. |
| 2020-10-05 | Fulcrum Therapeutics | Regulatory Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease fulcrumtx.com ↗
Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that the Investigational New Drug application (IND) is now in effect for its Phase 1 trial in healthy adult volunteers with FTX-6058 for sickle cell disease. |
| 2020-09-25 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Preclinical Proof-of-Concept Data for FTX-6058 at the Virtual 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting fulcrumtx.com ↗
FTX-6058, a small molecule designed to increase expression of fetal hemoglobin, demonstrated target engagement and good tolerability in multiple preclinical rodent models with once-a-day oral dosing. |
| 2019-10-01 | Fulcrum Therapeutics | Results Fulcrum Therapeutics Announces Multiple Presentations of Losmapimod Data at World Muscle Society Meeting fulcrumtx.com ↗
FTX-6058 showed an increase in HbF levels to ~30% total hemoglobin |
All press releases naming this drug 25 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Pociredir | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07431398 ↗ |
| Known as | FTX-6058 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04586985 ↗ |
| Modality | Small molecule | — CHEMBL6067682 ↗ |
| Route | Oral | “On Days 3-12, subjects will receive FTX-6058 by mouth once daily.” NCT04586985 ↗ |