Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Etavopivat Tablets Low dose, Etavopivat
Etavopivat Low dose · 6 trials · 2 indications
Measured as Count of events.
Hemoglobin response rate at Week 24 (increase of \> 1 g/dL \[\> 10 g/L\] from baseline) during the blinded treatment period
Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review
Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)
| Arm | Type | Description |
|---|---|---|
| Etavopivat | EXPERIMENTAL | Participants will be randomised to receive oral dose of Etavopivat. |
| Placebo | PLACEBO_COMPARATOR | Participants will be randomised to receive oral dose of placebo. |
| Double blind etavopivat Low Dose | EXPERIMENTAL | Double blind etavopivat Low Dose |
| Double blind etavopivat High Dose | EXPERIMENTAL | Double blind etavopivat High Dose |
| Double blind placebo | EXPERIMENTAL | Double blind placebo |
| Open label etavopivat | EXPERIMENTAL | Open label etavopivat |
| Etavopivat with HU | EXPERIMENTAL | Participants will receive Etavopivat 400 mg QD orally in combination with HU. The dose of HU (mg/kg) will be stable (no more than a 20% change in dosing except for weight-based changes) during the study, in the opinion of the Investigator. |
| Cohort 1: Etavopivat (12 to less than [<] 18 years) | EXPERIMENTAL | Participants aged 12 to less than 18 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. |
| Cohort 2: Etavopivat (6 to <12 years) | EXPERIMENTAL | Participants aged 6 to less than 12 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. |
| Cohort 3: Etavopivat (2 to <6 years) | EXPERIMENTAL | Participants aged 2 to less than 6 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in younger children. |
| Cohort 4: Etavopivat (6 months to <2 years) | EXPERIMENTAL | Participants aged 6 months to less than 2 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in infants. |
| Etavopivat 400 mg daily - SCD with transfusions | EXPERIMENTAL | Patients with sickle cell disease on chronic red blood cell transfusions |
| Etavopivat 400 mg daily - Thalassemia with transfusions | EXPERIMENTAL | Patients with thalassemia on chronic red blood cell transfusions |
| Etavopivat 400 mg daily - Thalassemia | EXPERIMENTAL | Patients with thalassemia not on chronic red blood cell transfusions |
| Name | Type | Description |
|---|---|---|
| Etavopivat | DRUG | Etavopivat will be administered orally. |
| Placebo | DRUG | Placebo matching Etavopivat will be administered orally. |
| Etavopivat Tablets Low dose | DRUG | 200 mg once daily |
| Etavopivat Tablets High dose | DRUG | 400 mg once daily |
| Placebo Tablets | DRUG | Placebo once daily |
| Etavopivat Tablets | DRUG | Selected dose once daily |
| Hydroxyurea | DRUG | Participants will receive a stable dose of HU. |
Inclusion Criteria: * Male or female. * Age 12 years or above at the time of signing the informed consent. * Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of lab...
| 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 |
Etavopivat is an investigational small molecule being studied for sickle cell disease, thalassemia, and liver diseases. It is also used in clinical trials involving healthy volunteers to assess safety, food effects, and pharmacokinetics. The drug is in Phase 1 clinical development and is not yet approved by regulatory authorities.
Etavopivat targets and activates pyruvate kinase liver and red blood cell (PKLR), an enzyme involved in red blood cell metabolism. By activating PKLR, the drug aims to modify disease processes in sickle cell disease and thalassemia. This mechanism is under investigation in early-stage clinical trials.
Etavopivat is being developed by Novo Nordisk A/S, a pharmaceutical company listed on the New York Stock Exchange under the ticker symbol NVO. The company is conducting Phase 1 clinical trials to evaluate the drug's safety and pharmacological properties in various participant populations.
Etavopivat is in Phase 1 clinical development. It is an investigational drug, meaning it has not received regulatory approval and is still being studied in early-stage trials. These trials focus on safety, tolerability, and how the drug behaves in the body, including studies in healthy volunteers and patients with liver disease.
Etavopivat has been studied in several Phase 1 trials. Completed trials include NCT06336018 in participants with liver disease, NCT06433661 on food effects in healthy participants, NCT06581627 in healthy Chinese participants, and NCT07023029 on heart rhythm effects. The drug has been evaluated in a total of six trials with 1,045 participants enrolled.
Yes, Etavopivat is also known as Etavopivat Tablets Low dose and Etavopivat Low dose. These alternative names refer to the same drug substance, which is being developed by Novo Nordisk A/S for sickle cell disease, thalassemia, and liver diseases.