Etavopivat Low dose (Ph 3) for Sickle Cell Disease | Novo Nordisk A/S | BiopharmaWatch

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Etavopivat Low dose

Phase 3

Sickle Cell Disease | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Jun 17, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMCBiomarker

Total Trials6

Total Enrollment1,045

FDA Designations

No designations recorded

Clinical trial landscape

Etavopivat Low dose · 6 trials · 2 indications

Phase 3 2Phase 2 4

Indication

NCT06612268A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell DiseaseSickle Cell Disease

RECRUITING408 Analytics

NCT04624659A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)Sickle Cell Disease

ACTIVE NOT_RECRUITING450 Analytics

PHASE3RECRUITING

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Sickle Cell Disease

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PHASE3ACTIVE NOT_RECRUITING

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

Sickle Cell Disease

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Study Endpoints

Primary Endpoints

Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact

Baseline (week 0) to week 52

Measured as Count of events.

Hemoglobin response rate

24 Weeks

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

52 Weeks

Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review

Effect of etavopivat on cerebral blood flow (CBF)

24 weeks

Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Effect of etavopivat on oxygen ejection fraction (OEF)

24 weeks

Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)

24 weeks

Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Change in the highest TAMMV in any of the L/R internal carotid artery [ICA] and L/R middle cerebral artery [MCA], whichever is highest, as measured by TCD

Baseline and Week 12

Single-dose: maximum plasma concentration (Cmax)

During the 24-week primary treatment period

Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)

During the 24-week primary treatment period

Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)

During the 24-week primary treatment period

Steady-state maximum plasma concentration (Cmax,ss)

During the 24-week primary treatment period

Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)

During the 24-week primary treatment period

Steady-state average plasma concentration (Cavg,ss)

During the 24-week primary treatment period

Steady-state minimum plasma concentration (Cmin,ss)

During the 24-week primary treatment period

Incidence of adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat

During the 24-week primary treatment period

Number of premature discontinuations

During the 24-week primary treatment period

Number of dose interruptions

During the 24-week primary treatment period

Number of dose reductions

During the 24-week primary treatment period

Cohorts A: 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

12 weeks

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

Cohorts B: 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

12 weeks

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

Cohort C: Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)

12 weeks

Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)

Secondary Endpoints

Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL)

Baseline (week 0) to week 24

Time to onset of first adjudicated Vaso-occlusive crisis (VOC)

Baseline (week 0) to week 52

Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale

Baseline (week 0) to week 52

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Study Design & Arms

AllocationRANDOMIZED

MaskingQUADRUPLE

ModelPARALLEL

PurposeTREATMENT

Treatment Arms

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.
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

Interventions

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.

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Eligibility Criteria

Age Range12 Years to N/A

SexALL

Healthy VolunteersNo

Study Sites174

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...

Countries:United StatesAustraliaBelgiumBrazilCanadaColombiaFranceGhanaGreeceIndiaItalyKenyaLebanonNetherlandsNigeriaOmanSaudi ArabiaSpainTurkey (Türkiye)UgandaUnited KingdomEgyptGermany

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Competitive Landscape -Sickle Cell Disease 27 trials

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

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Recent Changes (Last 90 Days)

LOWJun 18, 2026NCT06612268lastUpdatePostDate: changed

LOWJun 18, 2026NCT06612268lastUpdatePostDate: changed

LOWJun 18, 2026NCT06612268lastUpdatePostDate: changed

LOWJun 18, 2026NCT06612268lastUpdatePostDate: changed

MEDIUMMay 26, 2026NCT05725902TRIAL_REMOVED: changed

LOWMay 26, 2026NCT06198712Enrollment: 50 → 95

LOWMay 26, 2026NCT06612268primaryCompletionDate: changed

LOWMay 26, 2026NCT04624659primaryCompletionDate: changed

LOWMay 24, 2026NCT06198712studyFirstPostDate: changed

LOWMay 24, 2026NCT06612268studyFirstPostDate: changed

LOWMay 24, 2026NCT04624659studyFirstPostDate: changed

LOWMay 24, 2026NCT05725902studyFirstPostDate: changed