Inozyme Pharma Q1 2024 Financial Results and Business Highlights

Inozyme Pharma, Inc., a clinical-stage biopharmaceutical company specializing in treatments for rare diseases related to pathologic mineralization and intimal proliferation, has released its financial results for the first quarter ending March 31, 2024. They have also highlighted significant business achievements during this period.

CEO and Chairman Douglas A. Treco, Ph.D., expressed satisfaction with the preliminary evidence showing improved vascular health in adults treated with INZ-701 for ABCC6 Deficiency, underscoring the potential for further clinical development. The company is also focused on children with ABCC6 Deficiency who are at a high risk for neurological and visual impairments, expanding the target population significantly. Treco emphasized plans to collaborate with regulators to find a path to approval for the ABCC6 Deficiency program and to present additional data from ongoing trials.

Key recent highlights include:

1. Phase 1/2 Clinical Trial of INZ-701 in Adults with ABCC6 Deficiency: In April 2024, Inozyme announced positive safety and immunogenicity data, along with clinical improvements in vascular pathology, visual function, and patient-reported outcomes.

2. Natural History Studies and Development Plans for Pediatric ABCC6 Deficiency: Initial findings from the studies indicate a significant disease burden among children, manifesting as major clinical events early in life. Subject to regulatory approval and funding, the company plans to initiate a pivotal trial in the first quarter of 2025 for pediatric patients.

3. Phase 1/2 Clinical Trial of INZ-701 in Adults with ENPP1 Deficiency: Positive topline data were announced in April 2024, showing the maintenance of favorable safety, immunogenicity, and clinical outcomes through 48 weeks in Cohorts 1-3. Data from Cohort 4 supports once-weekly dosing.

Anticipated milestones include:

1. ENPP1 Deficiency:
- Initiation of the ENERGY-2 pivotal trial in infants outside the U.S. in the second half of 2024.
- Interim data from the ENERGY-1 Phase 1b trial in infants in the second half of 2024.
- Topline data from the ENERGY-3 pivotal trial in pediatric patients by mid-2025.

2. ABCC6 Deficiency:
- Planned initiation of a pivotal clinical trial in pediatric patients in the first quarter of 2025, pending regulatory review and funding.

3. Calciphylaxis:
- Interim data from the SEAPORT-1 Phase 1 trial in patients with end-stage kidney disease receiving hemodialysis in the fourth quarter of 2024.

Financially, Inozyme reported cash, cash equivalents, and short-term investments totaling $166.2 million as of March 31, 2024. These funds are expected to support operations into the fourth quarter of 2025.

Key financial metrics for the first quarter of 2024 include:

- Research and Development (R&D) Expenses: $19.1 million, up from $11.9 million in the previous year.
- General and Administrative (G&A) Expenses: $5.2 million, down from $6.5 million in the prior year.
- Net Loss: $23.3 million or $0.38 per share, compared to $17.4 million or $0.40 per share in the previous year.

Inozyme is actively developing INZ-701, an enzyme replacement therapy (ERT), to treat rare diseases that affect the vasculature, soft tissue, and skeleton. The therapy has shown potential in preventing the pathologic mineralization and intimal proliferation associated with ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. INZ-701 has demonstrated a favorable safety profile and increased levels of pyrophosphate (PPi) in clinical trials.

Inozyme Pharma continues to advance its clinical programs, focusing on innovative treatments to address significant unmet medical needs in rare diseases.

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