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Dec 31, 2020
Inozyme Q4 2020 Earnings Report
Reported full year financial results for 2020 and provided business highlights.
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.