Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 being developed for calciphylaxis, ectonucleotide pyrophosphatase/phosphodiesterase1 (ENPP1) deficiency, gene mutations, and ATP-binding cassette subfamily C member 6 (ABCC6) deficiency. It is being studied in adults, children, and infants with these conditions.
INZ-701 targets ENPP1, an enzyme involved in regulating pyrophosphate metabolism. By targeting ENPP1, the drug aims to address deficiencies that lead to conditions such as autosomal recessive hypophosphatemic rickets and generalized arterial calcification of infancy.
INZ-701 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker BMRN. The company is conducting clinical trials to evaluate the safety, tolerability, and efficacy of the drug across multiple patient populations.
INZ-701 is in Phase 1 clinical development, with one Phase 3 trial also active. The Phase 3 ENERGY 3 study is evaluating the drug in children with ENPP1 deficiency, while Phase 1 trials are ongoing or completed in adults, infants, and patients with end-stage kidney disease.
INZ-701 is being studied in four clinical trials. NCT04686175 and NCT06283589 are completed Phase 1 studies in adults with ENPP1 deficiency and calciphylaxis, respectively. NCT05734196 is a recruiting Phase 1 trial in infants, and NCT06046820 is an active Phase 3 trial in children.
INZ-701 is a distinct investigational drug with no alternative names listed in clinical trial records. It is being studied under its own name across multiple trials for ENPP1 deficiency, ABCC6 deficiency, and calciphylaxis.