Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PF-07940367 or GBT021601
Osivelotor · 2 trials · 2 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. |
| Name | Type | Description |
|---|---|---|
| Osivelotor | DRUG | Tablets which contain drug substance |
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...
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 |
Osivelotor is an investigational small molecule medicine being developed by Pfizer, Inc. (ticker PFE). It is being studied in liver diseases and sickle cell disease. It has not been approved by the FDA and remains in clinical development, with studies conducted in the United States and several other countries.
Osivelotor is being studied as a treatment for sickle cell disease in adults and adolescents 12 years of age and older. A Phase 2/3 trial, NCT05431088, is recruiting participants with sickle cell disease across the United States, Brazil, India, Kenya, Nigeria, Saudi Arabia, and the United Kingdom.
Osivelotor is being developed by Pfizer, Inc., which trades under the ticker PFE. Pfizer is the sponsor of the clinical program evaluating the small molecule in liver diseases and sickle cell disease.
Osivelotor is in Phase 1 development for liver diseases and in a Phase 2/3 study for sickle cell disease. It is investigational and has not been approved by the FDA. The Phase 1 study in people with loss of liver function has been completed, while the sickle cell disease trial is recruiting.
Osivelotor has been studied in NCT06340347, a completed Phase 1 trial that examined how the body processes the medicine in people with loss of liver function, enrolling 8 participants in the United States. It is also in NCT05431088, a recruiting Phase 2/3 trial in sickle cell disease with planned enrollment of 389 participants.
Yes, Osivelotor is also known as PF-07940367. The completed Phase 1 trial in people with loss of liver function refers to the study medicine as Osivelotor (PF-07940367), confirming that both names identify the same Pfizer small molecule.