Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Osivelotor
PF-07940367 · 3 trials · 3 indications
Number of adult participants with change from baseline in hemoglobin (Hb) through week 12 as measured by change in osivelotor concentrations from baseline or percentage change from baseline of clinical measures of anemia Hb and hemolysis (including indirect bilirubin, reticulocytes and lactate dehydrogenase).
Co-primary endpoints: Hb response (increase from baseline of \>1 g/dL) at Week 48 (based on average of Hb levels at Week 40 and Week 48) and the Annualized rate of VOC through end of Week 48. A VOC is defined as an acute episode of pain that: * Has no medically determined cause other than a vaso-occlusive event, and * Results in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), and * Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, priapism, and dactylitis that meet the requirements listed above will be included in this co-primary endpoint.
Incidence of Treatment Emergent Adverse Events: * Incidence of SAEs * Incidence of AEs leading to discontinuation * Change from baseline in laboratory parameters.
| Arm | Type | Description |
|---|---|---|
| Part B | PLACEBO_COMPARATOR | Study drug arm: Adult participants will receive osivelotor at 300 mg QD loading dose for 7 days followed by 150 mg QD through Week 48. Adolescent participant dose will be defined in a future protocol amendment. Placebo arm: Participants will receive placebo tablets for 48 weeks. |
| OLE | EXPERIMENTAL | Adult Participants will receive 150 mg open-label osivelotor up to 2 years after the last participant's visit in Part B or when the drug is commercially available in that region. The appropriate doses for adolescents will be defined in a future protocol amendment. |
| Part A | ACTIVE_COMPARATOR | Initially, participants will be randomized 1:1 to 100 mg and 150 mg daily. Upon review of the 150 mg safety data from at least 6 participants, there will be 1:1:1 randomization: 100 mg, 150 mg, and up to 200 mg. Participants will then receive maintenance once daily doses through Week 12. |
| Group 1 | EXPERIMENTAL | Participants with moderate hepatic impairment will receive a single dose of osivelotor, administered orally under fasted conditions. |
| Group 2 | EXPERIMENTAL | Participants with mild hepatic impairment will receive a single dose of osivelotor, administered orally under fasted conditions. |
| Treatment A: PF-07940367 Product II | EXPERIMENTAL | tablet by mouth |
| Treatment B: PF-07940367 Product I | EXPERIMENTAL | tablet by mouth |
| Name | Type | Description |
|---|---|---|
| Osivelotor | DRUG | Tablets which contain drug substance |
| PF-07940367 | DRUG | Participants to receive a single dose of PF-07940367 product II 150 mg by mouth |
Inclusion Criteria: Part A and Part B: * Male or female with SCD, HbSS and HbSB-zero * Participants with Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL during Screening and considered stable by the Investigator. * For participants taking hydroxyurea and/or L-glutamine, the dose must be stable for at least 90 da...
PF-07940367 is an investigational small molecule developed by Pfizer, Inc. It is in Phase 1 clinical development, and the specific diseases or conditions it is intended to treat have not been disclosed. It has been studied in healthy adult volunteers rather than in patients with a named condition.
PF-07940367 is being developed by Pfizer, Inc., which trades on the New York Stock Exchange under the ticker PFE. Pfizer sponsored the Phase 1 study of the compound conducted in the United States.
PF-07940367 is in Phase 1 clinical development. It is an investigational small molecule and is not an approved medicine. One Phase 1 trial has been completed, and no trials of the compound are currently active.
PF-07940367 has been evaluated in one Phase 1 trial, NCT06190561, titled a study to learn how different products of the study medicine called PF-07940367 are taken up into the blood in healthy adults. That trial enrolled 40 healthy adult volunteers in the United States and is completed.
The Phase 1 trial NCT06190561 was a controlled study with randomized assignment of 40 healthy adult participants, both male and female, aged 18 years and older. It was conducted in the United States and did not use a double-blind design. Participants were healthy volunteers rather than patients.