drugset
Drugs / INZ-701
last change Aug 2026 re-read 9 days ago

INZ-701

also known as rhENPP1-Fc

Protein / enzyme biologic targets ENPP1 via restoration

Developed for
pseudoxanthoma elasticum (inherited or acquired) · calciphylaxis
Investigated by
Inozyme Pharma · BioMarin Pharmaceutical · IQVIA Biotech

Trials 8

today NCT07473973 · Ph 3 NCT06739980 · Ph 2 NCT06462547 · Ph 2 NCT06283589 · Ph 1 NCT06046820 · Ph 3 NCT05734196 · Ph 1 NCT05030831 · Ph 1/2 NCT04686175 · Ph 1/2
2022202320242025202620272028202920302031
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 12 trials
Phase 1 NCT06283589 Feb → Jul 2024 calciphylaxis Inozyme Pharma Completed No outcome recorded
Phase 1 NCT05734196 Jun 2023 → Oct 2026 expected arterial calcification of infancy, autosomal recessive hypophosphatemic rickets, pseudoxanthoma elasticum (inherited or acquired) Inozyme Pharma Active not recruiting No outcome recorded
Phase 1/22 trials
Phase 1/2 NCT05030831 Apr 2022 → Jul 2024 arterial calcification of infancy, pseudoxanthoma elasticum (inherited or acquired) Inozyme Pharma Completed No outcome recorded
Phase 1/2 NCT04686175 Nov 2021 → Oct 2024 arterial calcification of infancy, autosomal recessive hypophosphatemic rickets Inozyme Pharma Completed No outcome recorded
Phase 22 trials
Phase 2 NCT06739980 Jan 2025 → Mar 2027 arterial calcification, generalized, of infancy, 1, autosomal recessive hypophosphatemic rickets Inozyme Pharma Withdrawn No outcome recorded
Phase 2 NCT06462547 Jun 2024 → Nov 2030 expected calcinosis, hypophosphatemic rickets, autosomal recessive, 2, pseudoxanthoma elasticum (inherited or acquired) Inozyme Pharma Recruiting No outcome recorded
Phase 32 trials
Phase 3 NCT07473973 Mar 2025 → Aug 2026 arterial calcification, generalized, of infancy, 1, autosomal recessive hypophosphatemic rickets Inozyme Pharma Terminated No outcome recorded
Phase 3 NCT06046820 Nov 2023 → Jan 2026 arterial calcification of infancy, autosomal recessive hypophosphatemic rickets Inozyme Pharma Terminated No outcome recorded

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2026-05-18 BioMarin Pharmaceutical Results BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency biomarin.com ↗
The study met one co-primary endpoint, showing treatment with BMN 401 resulted in statistically significant increases in plasma PPi through week 52 compared to the conventional therapy control arm in children with ENPP1 deficiency.

All press releases naming this drug 1 release

DateIssuerRelease

Evidence & citations 6 cited values

Every value below carries the sentence it was read from. 8 sources stand behind the page.

FieldValueCited text
Known as INZ-701 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06462547 ↗
7

NCT06739980 ↗

NCT06283589 ↗

NCT04686175 ↗

NCT05030831 ↗

NCT05734196 ↗

NCT07473973 ↗

NCT06046820 ↗
Known as rhENPP1-Fc “INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).” NCT06462547 ↗
1

“INZ-701 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody (rhENPP1-Fc).” NCT05030831 ↗
Action Restore “INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1...” NCT05734196 ↗
Modality Protein / enzyme biologic “INZ701-101 is a recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.” NCT04686175 ↗
Route Subcutaneous “administered a 2.4 mg/kg once weekly dose by subcutaneous (SC) injection” NCT06046820 ↗
Target ENPP1 “contains the extracellular domains of human ENPP1” NCT04686175 ↗