Inozyme Q4 2020 Earnings Report
Key Takeaways
Inozyme Pharma reported its full year 2020 financial results, highlighting its cash position of $159.9 million, which is expected to fund operations into the second half of 2022. The company is advancing its INZ-701 clinical development program, with Phase 1/2 trials planned for ENPP1 and ABCC6 deficiencies.
Received Orphan Drug Designation for INZ-701 for ABCC6 Deficiency from the FDA.
Cleared to proceed with Phase 1/2 Clinical Trial of INZ-701 for ENPP1 Deficiency in the U.S. and U.K.
Completed a burden of disease patient study providing an overview of the burden in patients with ENPP1 and ABCC6 deficiencies.
Completed a healthy volunteer pyrophosphate (PPi) study validating the assay for evaluating INZ-701 in clinical trials.
Inozyme
Inozyme
Forward Guidance
Inozyme anticipates several milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions.
Positive Outlook
- Initiate prospective natural history study for ENPP1 Deficiency in H1 2021.
- Initiate Phase 1/2 clinical trial for ENPP1 Deficiency in H1 2021.
- Report preliminary safety and biomarker data from Phase 1/2 clinical trial for ENPP1 Deficiency in H2 2021.
- File Clinical Trial Applications for ABCC6 Deficiency in H1 2021.
- Initiate Phase 1/2 clinical trial for ABCC6 Deficiency by mid-2021.
Challenges Ahead
- Potential delays due to COVID-19-related restrictions.
- Risks associated with the ability to initiate planned Phase 1/2 clinical trials of INZ-701 for ENPP1 deficiency and ABCC6 deficiency.
- Risks related to obtaining and maintaining necessary approvals from the FDA and other regulatory authorities.
- Uncertainties related to replicating positive results from preclinical studies and early-stage clinical trials in later clinical trials.
- Challenges in advancing the development of product candidates under anticipated timelines.