Inozyme Pharma, Inc. announced the first self-administration of INZ-701 in the open-label Phase 2 extension portion of the ongoing Phase 1/2 clinical trial of INZ-701 in adult patients with ENPP1 Deficiency. The implementation of self-administration of INZ-701 followed Inozyme's submission of a protocol amendment to the U.S. Food and Drug Administration (FDA) and approval of a protocol amendment submitted to the German Federal Institute for Drugs and Medical Devices (BfArM). The Company has also submitted documentation to additional ex-U.S. regulatory agencies to allow self-administration.

These submissions were supported by preliminary safety data from the ongoing trial. Inozyme is facilitating self-administration in the Phase 2 extension portion of the clinical trial after patients complete the 32-day dose evaluation period in the clinic. INZ-701 is supplied as a lyophilized powder intended for reconstitution and subcutaneous injection.

In its lyophilized form, INZ-701 has been shown to be shelf stable for up to three years. Long-term shelf stability along with the ability for self-administration significantly improves convenience for patients. The ongoing Phase 1/2 open-label clinical trial is expected to enroll up to nine adult patients with ENPP1 Deficiency at sites in North America and Europe.

The trial will primarily assess the safety and tolerability of INZ-701 in adult patients with ENPP1 Deficiency, as well as characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profile of INZ-701, including evaluation of plasma pyrophosphate (PPi) and other biomarker levels. In the Phase 1 dose-escalation portion of the trial, Inozyme is assessing INZ-701 for 32-days at doses of 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg administered via subcutaneous injection twice weekly, with three patients per dose cohort. Doses were selected based on preclinical studies and PK/PD modeling.

The Phase 1 dose-escalation portion of the trial seeks to identify a safe, tolerable dose that increases PPi levels, and that can be used for further clinical development. The open-label Phase 2 extension portion of the trial is assessing long-term safety, pharmacokinetics, and pharmacodynamics of continued treatment with INZ-701 for up to 48 weeks, where patients may receive doses of INZ-701 at home depending on site-specific protocols. Exploratory endpoints will include evaluations of skeletal, vascular, physical function and patient-reported outcomes.