Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as (rhENPP1-Fc).
INZ-701 · 7 trials · 10 indications
For each subject, their change from baseline in Plasma Inorganic Pyrophosphate (PPi) concentration will be assessed.
For each subject, their change in overall survival based on time from date of birth to event of all-cause mortality will be assessed.
For each subject, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
Treatment-emergent AEs are defined as any AE occurring from the first dose of INZ-701 through 30 days after the last dose of INZ-701.
For each subject, the presence of ADAs will be assessed and, if present, further evaluation will determine specificity and subtypes.
For each participant, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the participant's baseline value over time to determine if there's been a change.
For each participant, an echocardiogram will be collected, and used to assess heart function. (Including measurement of left ventricular ejection fraction), and to identify any other abnormalities, for example, calcification of heart valves.
For each subject, variation of concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
For each subject, the maximum concentration of INZ-701 in the plasma will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
For each subject, clearance of INZ-701 from the body will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
For each subject, plasma PPi will be measured via a series of blood samples obtained throughout the study, comparing the subject's baseline value over time.
| Arm | Type | Description |
|---|---|---|
| INZ-701 | EXPERIMENTAL | Participants receive INZ-701 (rhENPP1-Fc) administered by subcutaneous injection once weekly at the protocol-specified dose. The visit final volume to administer (mL) is determined using protocol-defined parameters. |
| Control Arm (Conventional Therapy) | ACTIVE_COMPARATOR | Subjects randomized to the control arm will continue taking their conventional therapy as clinically indicated by their treating physician for the duration of the 52-week Randomized Treatment Period. |
| Name | Type | Description |
|---|---|---|
| INZ-701 | DRUG | Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody. |
| Control Arm (Conventional Therapy) | DRUG | Conventional therapy is defined as oral phosphate supplements and calcitriol or other active forms of vitamin D3 (or analogs). No other agents for treatment of ENPP1 Deficiency are allowed in the control arm. |
Participants must meet all of the following: Inclusion Criteria: 1. Infant aged ≤ 1 year at the time of enrollment. 2. Confirmed diagnosis of ENPP1 deficiency, based on genetic testing. 3. Clinical features consistent with generalized arterial calcification of infancy (GACI) (e.g., vascular calcif...
INZ-701 is an investigational small molecule developed by BioMarin Pharmaceutical Inc. (ticker BMRN). It is being studied for ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and conditions involving gene mutations. It is not approved and remains in clinical development.
INZ-701 targets ENPP1, short for ectonucleotide pyrophosphatase/phosphodiesterase 1. ENPP1 is a gene, and the drug is designed to address conditions linked to ENPP1 Deficiency and related gene mutations, including ABCC6 Deficiency and calciphylaxis.
BioMarin Pharmaceutical Inc., which trades under the ticker BMRN, is developing INZ-701. The company is running the clinical program across ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis, including the ENERGY and ADAPT studies.
INZ-701 is in clinical development and is not approved. Its program spans Phase 1 through Phase 3, with four trials in total: one completed Phase 1 study, one Phase 2 long-term safety study, and two Phase 3 studies in ENPP1 Deficiency.
INZ-701 trials include NCT07473973 (ENERGY 2, Phase 3, recruiting in infants), NCT06046820 (ENERGY 3, Phase 3, active but not recruiting in children), NCT06462547 (ADAPT, Phase 2 long-term safety), and NCT06283589 (SEAPORT 1, completed Phase 1 in adults on hemodialysis).
INZ-701 is being evaluated as a treatment for ENPP1 Deficiency, including autosomal recessive hypophosphatemic rickets and generalized arterial calcification of infancy. The ENERGY 2 and ENERGY 3 Phase 3 trials study it in infants and children with this condition.