Following meetings with the FDA and the European Medicines Agency's Paediatric Committee, Inozyme Pharma Inc (NASDAQ:INZY) updated its global development strategy for INZ-701, a treatment for ENPP1 Deficiency.

Earlier this year, Inozyme Pharma announced topline pharmacokinetic (PK), pharmacodynamic (PD), and safety data from the ongoing Phase 1/2 trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency. 

The company plans to initiate the ENERGY-3 pivotal trial, a multicenter, randomized, open-label trial in pediatric patients with ENPP1 Deficiency, in October 2023. It is expected to enroll up to 33 patients between the ages of one and less than 13 years. 

Patients will be randomized in a 2:1 ratio to an INZ-701 arm or a control arm (conventional therapy, i.e., oral phosphate and active vitamin D) for 52 weeks, followed by an open-label extension period during which all patients may receive INZ-701. INZ-701 will be administered at a 2.4 mg/kg once weekly dose via subcutaneous injection.

The company also plans to conduct the ENERGY-2 trial for infants with ENPP1 Deficiency. This trial will start outside of the U.S. and aims to enroll up to 12 infants.

Subject to regulatory approval and funding, Inozyme intends to carry out the ENERGY-4 pivotal trial in adolescents and adults with ENPP1 Deficiency. This multicenter, randomized, controlled trial is expected to enroll up to 30 patients aged 13 years and older.

Based on feedback from FDA and EMA, positive results from these clinical trials could lead to the submission of marketing applications for INZ-701 in the U.S. and EU. 

If approved, the firm anticipates commercially launching INZ-701 for infant and pediatric patients by the second half of 2026.

The company said a cash balance of $140.2 million is expected to provide a cash runway into the first quarter of 2025. 

Price Action: INZY shares are down 11.40% at $5.47 on the last check Wednesday.