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MOD-4023

Phase 3

Growth Hormone Deficiency | Small molecule | Endocrine |Opko Health Inc.|Last Updated: Aug 12, 2021

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment86
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03874013Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone DeficiencyPHASE3 COMPLETED 44Dec 7, 2017Mar 6, 2020Aug 12, 202146 Japan
NCT02418767Pharmacokinetics and Pharmacodynamics of Long Acting Human Growth Hormone (hGH) Product (MOD-4023) in Healthy Caucasian and Japanese VolunteersPHASE1 COMPLETED 42Jan 1, 2015Jun 1, 2015Sep 30, 20191 United States
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Study Endpoints
Primary Endpoints
Annual Height Velocity (HV) After 12 Months
12 months

Annual Height Velocity in cm/year after 12 months of treatment.

Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
30 days

To assess the safety, tolerability and immunogenicity of MOD-4023 in healthy Caucasian and Japanese subjects after single subcutaneous (SC) dose.

Pharmacokinetics (PK) profile of MOD-4023 in healthy Caucasian and Japanese subjects after single SC dose
7 days

MOD-4023 serum levels (T1/2, Area-Under-the-Curve, Cmax, Tmax)

Secondary Endpoints
Height Velocity at 6 Months
6 months
Change in Height Standard Deviation Score (SDS) Compared to Baseline After 12 Months
12 months
Change in Bone Maturation (BM) After 12 Months
12 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeOTHER
Treatment Arms
ArmTypeDescription
MOD-4023 Treatment ArmEXPERIMENTALMOD-4023 (investigational treatment): weekly MOD-4023 SC injections for 12 months; initially over the first 6 weeks, MOD-4023 will be administered in 3 stepwise escalating doses (0.25 mg/kg/week, 0.48 mg/kg/week and 0.66 mg/kg/week), each for two weeks sequentially. For the remaining 46 weeks, patients will continue to receive MOD-4023 at a dose of 0.66 mg/kg/week.
Genotropin Treatment ArmACTIVE_COMPARATORGenotropin® (reference treatment): daily Genotropin® (0.025 mg/kg/day).
Low doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
Mid doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
High doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
Interventions
NameTypeDescription
MOD-4023DRUGMOD-4023 is a long-acting modified recombinant human growth hormone (r-hGH) which utilizes C-terminal peptide (CTP) technology. It will be provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a multi-dose disposable pre-filled PEN. MOD-4023 will be administered as a SC injection once weekly, using a delivery device.
GenotropinDRUGGenotropin® is dispensed in a 2-chamber cartridge. The front compartment contains recombinant somatropin, glycine, mannitol, sodium dihydrogen phosphate anhydrous and disodium phosphate anhydrous. The rear compartment contains m-Cresol and mannitol in water for injections. A delivery device (Genotropin®) will be used for daily (evening/bedtime) SC administration of Genotropin® into the region of the upper arms, buttocks, thighs or abdomen (8 locations). Injection sites should be rotated. Dose regimen for Genotropin®: 0.025 mg/kg/day (or 0.175 mg/kg/w divided equally to 7 injections over a week).
PlaceboDRUG -
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Eligibility Criteria
Age Range3 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites46

Inclusion Criteria: 1. Pre-pubertal child aged ≥ 3 years old, and not yet 10 years for girls (9 years and 364 days) or not yet 11 years for boys (10 years and 364 days), on the date of ICF signature, with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency. 2. ...

Countries:JapanUnited States
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