INZY - Inozyme adds 36% on Phase 1/2 data for lead asset and cash runway

Inozyme Pharma ( NASDAQ: INZY ) shares surged ~36% on Thursday after the rare-disease-focused biotech announced topline data from its Phase 1/2 studies for lead candidate INZ-701 and raised its outlook for cash runway.

The global trials were designed to evaluate INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE). The studies assessed the safety and tolerability of the enzyme therapy and its impact on human biomarkers, including plasma pyrophosphate ((PPi)).

According to the company, each trial of nine patients indicated an increase in PPi levels comparable to those in a study of healthy subjects (n=10). The PPi increase in the trial for PXE was sustained in the highest dose cohort.

In the ENPP1 Deficiency trial, three patients experienced mild adverse events related to the study drug, while one dropped out after week 12 due to travel reasons.

In the other study, seven patients experienced adverse events related to INZ-701, while one from the highest dose group was left out on day 18 due to a moderate adverse event.

Inozyme ( INZY ) expects to evaluate INZ-701 in a pivotal study in ENPP1 Deficiency and a Phase 2/3 clinical trial in ABCC6 Deficiency in Q3 2023 and 2024, subject to regulatory clearance, respectively.

Citing preliminary financials, the company also updated its cash runway guidance, indicating ~$127.9M in cash, cash equivalents, and short-term investments as of 2022 year-end.

With $20M borrowing in mid-February, Inozyme ( INZY ) expects its cash runway will be adequate to fund operations through Q4 2024, an increase of two quarters from its previous guidance.

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