Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Pociredir oral capsule(s), FTX-6058
Pociredir · 2 trials · 2 indications
Laboratory assessments including hematology, coagulation, serum chemistry and electrolytes, lipid panel, SCD characterization, serology, urinalysis and pregnancy tests will be performed.
To evaluate the safety and tolerability of Pociredir in adult participants with sickle cell disease based on the frequency of adverse events (AEs) and changes in clinically significant laboratory test results, vital signs and electrocardiograms (ECGs) parameters.
Blood samples will be collected to measure the plasma concentration of Pociredir at specified timepoints.
| Arm | Type | Description |
|---|---|---|
| Pociredir | EXPERIMENTAL | Participants will receive Pociredir orally once daily (QD) |
| Pociredir oral capsule(s) in Sickle Cell participants | EXPERIMENTAL | Cohort 1 will receive 6 mg of Pociredir by mouth once daily. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. A total of seven cohorts may be included. Following the first cohort, doses for all subsequent cohorts will be determined following DMC review of the safety and pharmacokinetic data observed in participants from the prior and ongoing cohorts. Alternate dosing schedules may be evaluated in some of the cohorts. |
| Name | Type | Description |
|---|---|---|
| Pociredir | DRUG | Pociredir Oral Capsules will be administered |
| Pociredir oral capsule(s) | DRUG | Participants will receive Pociredir |
Inclusion Criteria: * Participants aged ≥18 years and older must have previously participated in and successfully completed Study 6058-SCD-101. * Participant has signed and dated the informed consent form (ICF) before any study-specific procedures are performed and is willing and able to comply wit...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Pociredir is an investigational oral small molecule being developed for the treatment of sickle cell disease. It is currently in Phase 2 clinical development and has not been approved by the FDA. Fulcrum Therapeutics is evaluating it in adults 18 years and older with sickle cell disease.
Pociredir targets EED, the embryonic ectoderm development protein, which is a component of the polycomb repressive complex 2. By binding EED, the small molecule is designed to modulate PRC2 activity and induce fetal hemoglobin production, a mechanism relevant to sickle cell disease.
Pociredir is being developed by Fulcrum Therapeutics, Inc., which trades on the Nasdaq under the ticker FULC. Fulcrum is a clinical-stage biopharmaceutical company focused on small molecule therapies, and Pociredir is its oral EED-targeting candidate for sickle cell disease.
Pociredir is in Phase 2 clinical development for sickle cell disease. It is investigational and has not received FDA approval. The FDA has granted Pociredir Fast Track and Orphan Drug designations, which are intended to support and expedite development of therapies for serious conditions.
Pociredir has been studied in two clinical trials. NCT05169580 was a completed Phase 1 study of safety, tolerability, pharmacokinetics and pharmacodynamics in sickle cell disease, enrolling 45 adults across the United States, Nigeria and South Africa. NCT07401823 is an ongoing Phase 2 open-label extension study enrolling about 50 adults in the United States.
Yes. Pociredir oral capsule(s) refers to the same investigational drug as Pociredir, the oral small molecule EED-targeting candidate developed by Fulcrum Therapeutics for sickle cell disease. The capsule description simply identifies the oral dosage form used in its clinical trials.