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evinacumab

Phase 3

Homozygous Familial Hypercholesterolemia | Small molecule | Metabolic |Regeneron Pharmaceuticals, Inc.|Last Updated: Apr 8, 2025

Target and mechanism

Molecular targetANGPTL3
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials3
Total Enrollment201

FDA Designations

No designations recorded

Clinical trial landscape

evinacumab · 6 trials · 4 indications

Phase 3 3Phase 2 2Phase 1 1
NCT04233918Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
COMPLETED20 Analytics
NCT03409744Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
COMPLETED116 Analytics
NCT03399786Efficacy and Safety of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial Hypercholesterolemia
COMPLETED65 Analytics
PHASE3COMPLETED
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics
PHASE3COMPLETED
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics
PHASE3COMPLETED
Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial HypercholesterolemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab
At day 12

Cmax was obtained directly from the plasma concentration versus time curve.

Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab
Up to Week 12

AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.

Part A: Terminal Half-Life (t1/2) of Evinacumab
Up to week 12

T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.

Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24
Baseline to Week 24

Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Up to 216 weeks

The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24 (Intent-to-Treat [ITT] Estimand)
Week 24

Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).

Percent Change From Baseline in Fasting Triglycerides (TG) Level Following 12 Weeks of Repeated IV Doses of Evinacumab in Actual Cohort 3 Participants
Participants randomized to evinacumab: Week 0 corresponds to Baseline and 12 weeks treatment corresponds to Week 12 of the DBTP; Participants randomized to placebo: Week 12 corresponds to Baseline and 12 weeks treatment corresponds to Week 24 of the SBTP

For participants randomized to evinacumab treatment group, baseline (Week 0) TG was defined as the geometric mean of all available TG results at day -28, day -14 and week 0; for participants randomized to placebo treatment group, baseline (Week 12) TG was defined as the geometric mean of all available TG results at weeks 6, 8, and 12.

Percent Change From Baseline in Calculated Low Density Lipoprotein Cholesterol (LDL-C) at Week 16 (Intent-to-Treat [ITT] Estimand)
Baseline and Week 16
Incidence of Treatment Emergent Adverse Events (TEAEs)
Baseline up to week 31
Severity of TEAEs
Baseline up to week 31

Secondary Endpoints

Part A and Part B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Part A: up to Week 24; Part B: up to Week 48
Part B: Percent Change in Apolipoprotein B (Apo B) From Baseline to Week 24
Baseline to Week 24
Part B: Percent Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Week 24
Baseline to Week 24
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
EvinacumabEXPERIMENTALPart A: Single intravenous (IV) dose Part B: IV dose every 4 weeks (Q4W) until week 20 Part C: IV dose Q4W
PlaceboEXPERIMENTAL -
Group A: dosing regimen 1EXPERIMENTALSC Evinacumab QW for 16 weeks
Group A: dosing regimen 2EXPERIMENTALSC Evinacumab Q2W for 16 weeks (alternating with matching placebo on opposite weeks)
Group A: dosing regimen 3EXPERIMENTALSC Evinacumab QW for 16 weeks
Group A: matching placeboEXPERIMENTALPlacebo SC QW for 16 weeks
Group B: dosing regimen 1EXPERIMENTALIntravenous (IV) Evinacumab Q4W for 24 weeks
Group B: dosing regimen 2EXPERIMENTALIV Evinacumab Q4W for 24 weeks
Group B: matching placeboEXPERIMENTALPlacebo IV Q4W for 24 weeks
Cohort 1EXPERIMENTALEvinacumab SC or placebo SC
Cohort 2EXPERIMENTALLow dose regimen: evinacumab IV or placebo IV
Cohort 3EXPERIMENTALHigh dose regimen: evinacumab IV or placebo IV
Cohort 4EXPERIMENTALEvinacumab or placebo SC every week (QW) x 8 doses
Cohort 5EXPERIMENTALEvinacumab or placebo SC x 1 dose

Interventions

NameTypeDescription
EvinacumabDRUGPart A: Single IV dose Part B \& C: IV dose Q4W
PlaceboDRUGIV administration of placebo
Matching placeboDRUGSC or IV administration
Background Lipid Modifying Therapy (LMT)OTHERAll participants should be on a stable, maximally tolerated statin throughout the duration of the study. The dose of statin and of PCSK9 inhibitor, such as alirocumab or evolocumab, as well as other LMT (if applicable), should remain stable throughout the study duration, from screening through the end of study (EOS) visit.
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Eligibility Criteria

Age Range5 Years to 11 Years
SexALL
Healthy VolunteersNo
Study Sites11

Key Inclusion Criteria: 1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol 2. LDL-C \>130 mg/dL at the screening visit 3. Body weight ≥15 kg 4. Receiving stable maximally tolerated therapy\*at the screening visit \*Maximally tolerated therapy could inc...

Countries:United StatesAustraliaAustriaNetherlandsTaiwanUkraineCanadaCzechiaFranceGreeceItalyJapanSouth AfricaUnited KingdomDenmarkIsraelJordanNew ZealandNorwayPolandRussiaSpainSweden
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Frequently asked questions about evinacumab

What is Evinacumab used for?

Evinacumab is an investigational drug being studied for severe hypertriglyceridemia, hypercholesterolemia, and homozygous familial hypercholesterolemia. It has also been evaluated in healthy volunteers. Clinical trials have tested it in adults and pediatric patients with these lipid disorders.

What does Evinacumab target?

Evinacumab targets ANGPTL3, a protein that regulates lipid metabolism. It acts as an inhibitor of this target, which is being studied for its potential to lower lipid levels in conditions like hypercholesterolemia and homozygous familial hypercholesterolemia.

Who makes Evinacumab?

Evinacumab is developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. The company has conducted multiple clinical trials to evaluate the drug's safety and efficacy.

What phase is Evinacumab in?

Evinacumab has completed Phase 1, Phase 2, and Phase 3 clinical trials. It is an investigational drug and has not been reported as FDA approved. All completed trials include Phase 1 in healthy volunteers, Phase 2 in hypercholesterolemia, and Phase 3 in homozygous familial hypercholesterolemia.

What clinical trials is Evinacumab in?

Evinacumab has been studied in several completed trials, including NCT03146416 in healthy volunteers, NCT03175367 in hypercholesterolemia, NCT03399786 in homozygous familial hypercholesterolemia, and NCT04233918 in pediatric patients with homozygous familial hypercholesterolemia. These trials were conducted across multiple countries.

Is Evinacumab the same as REGN1500?

Yes, Evinacumab is also known as REGN1500. Clinical trial titles refer to it as Evinacumab (REGN1500), confirming that both names refer to the same investigational drug being developed by Regeneron Pharmaceuticals.