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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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Pociredir is an investigational small molecule being developed for the treatment of Sickle Cell Disease. It is currently in Phase 2 clinical development and has received Fast Track and Orphan Drug designations from the FDA. The drug is being studied in adult patients with the condition.
Pociredir is being developed by Fulcrum Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FULC. The company is conducting clinical trials of Pociredir in patients with Sickle Cell Disease.
Pociredir is currently in Phase 2 clinical development for Sickle Cell Disease. It is an investigational drug and has not been approved by the FDA. The drug has received Fast Track and Orphan Drug designations from the FDA, reflecting its potential to address an unmet medical need.
Pociredir has been studied in two clinical trials. A Phase 1 trial (NCT05169580) evaluated safety, tolerability, pharmacokinetics, and pharmacodynamics in 45 patients with Sickle Cell Disease and has been completed. A Phase 2 open-label extension study (NCT07401823) is currently enrolling by invitation with an estimated enrollment of 50 patients.
Pociredir is a small molecule therapeutic being developed for Sickle Cell Disease. The specific molecular target of Pociredir has not been disclosed in available information. The drug is being evaluated for its safety, tolerability, pharmacokinetics, and pharmacodynamics in clinical trials.