Inozyme Pharma Announces Q2 2024 Financial Results and Business Updates

Inozyme Pharma, Inc. recently provided a comprehensive update on its financial results for the second quarter of 2024 and significant advancements in its clinical programs. The clinical-stage rare disease biopharmaceutical company, which specializes in developing treatments for conditions affecting bone health and blood vessel function, reported notable progress in its INZ-701 clinical trials.

Douglas A. Treco, Ph.D., the Chief Executive Officer and Chairman of Inozyme, highlighted the company's dedication to advancing its clinical programs and achieving critical milestones by the end of the year. Treco emphasized the company's commitment to developing transformative treatments for rare diseases involving the PPi-Adenosine Pathway, which plays a crucial role in regulating mineralization and intimal proliferation.

A key recent development was the publication of preclinical data in July 2024, which supported the potential of INZ-701 to treat a variety of diseases linked to the PPi-Adenosine Pathway. The study, titled "Inhibition of Vascular Smooth Muscle Cell Proliferation by ENPP1: The Role of CD73 and the Adenosine Signaling Axis," appeared in the journal Cells. This evidence suggests that INZ-701 could address multiple conditions by targeting this crucial biological pathway.

In June 2024, Inozyme presented findings on ENPP1 Deficiency at the 11th International Conference on Children’s Bone Health in Salzburg, Austria. The company shared results from a radiographic study on the skeletal features of pediatric patients with ENPP1 Deficiency and hosted a symposium to raise awareness about the condition, often misdiagnosed as hypophosphatemic rickets.

In April 2024, Inozyme announced positive topline data from its Phase 1/2 Clinical Trial of INZ-701 in adults with ENPP1 Deficiency. The data demonstrated that favorable safety, immunogenicity, and clinical outcomes persisted through 48 weeks. This information was later presented at prestigious gatherings such as the European Calcified Tissue Society Congress (ECTS) 2024 in May and the Endocrine Society’s Annual Meeting (ENDO) 2024 in June.

Inozyme also made strides with its ABCC6 Deficiency program. In July 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency, which can present as pseudoxanthoma elasticum (PXE). Positive topline data from its Phase 1/2 Clinical Trial in adults showed improvements in vascular pathology, visual function, and patient-reported outcomes. Initial findings from natural history studies indicated a high disease burden among pediatric patients, who frequently experience severe clinical events early in life.

Looking ahead, Inozyme has several key milestones on the horizon. These include:

- Complete enrollment in ENERGY 3, a pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency, expected in the third quarter of 2024.
- Interim data from ENERGY 1, a Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency, anticipated in the fourth quarter of 2024.
- Interim data from SEAPORT 1, a Phase 1 trial of INZ-701 in patients with end-stage kidney disease receiving hemodialysis, expected in the fourth quarter of 2024.
- The company's cash, cash equivalents, and short-term investments as of June 30, 2024, are expected to fund operations into the fourth quarter of 2025.

Financially, Inozyme reported a cash position of $144.5 million as of June 30, 2024, which is expected to support the company's operations through late 2025. Research and development expenses for the quarter were $21.8 million, an increase from $11.7 million in the prior-year period. General administrative expenses also rose to $5.9 million from $4.7 million in the previous year. The net loss for the quarter stood at $27.0 million, or $0.44 per share, compared to $15.6 million or $0.35 per share for the same period last year.

Inozyme continues to focus on developing INZ-701 as a potential treatment for several rare diseases caused by deficiencies in the PPi-Adenosine Pathway. The company's efforts target pathological mineralization and intimal proliferation, addressing significant morbidity and mortality associated with these diseases.