Inozyme Pharma (INZY) FDA Events

INZ-701 - FDA Regulatory Timeline and Events

INZ-701 is a drug developed by Inozyme Pharma for the following indication: ABCC6 Deficiency. This drug is under review by the U.S. Food and Drug Administration (FDA). Below is a timeline of key regulatory milestones for this therapy.

Data Presentation - February 21,2025

• Drug: INZ-701
• Announced Date: February 21, 2025
• Indication: ABCC6 Deficiency

Inozyme Pharma, Inc. announced that Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer, will present recently announced data from the company's Expanded Access Program (EAP) evaluating INZ-701 in infants and children with ENPP1 Deficiency at the CHOP Cardiology Annual Meeting, held February 19-23, 2025, in Orlando, Florida.

Inozyme Pharma, Inc. announced that Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer, will share new data on the enzyme replacement therapy INZ-701 during the CHOP Cardiology Annual Meeting in Orlando, Florida this February. The data, gathered from the company's Expanded Access Program (EAP), focuses on the use of INZ-701 in infants and children affected by ENPP1 Deficiency—a rare and severe condition that impacts blood vessels, soft tissues, and bones.

Dr. Gunter’s presentation, titled "Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program," is scheduled for February 22, 2025, from 10:00 to 11:00 am. This session will provide insight into the potential of INZ-701 to mitigate the symptoms and improve outcomes for a vulnerable patient group, highlighting the therapy's promise for addressing this challenging condition.

Positive Data - January 10,2025

• Drug: INZ-701
• Announced Date: January 10, 2025
• Indication: ABCC6 Deficiency

Inozyme Pharma, Inc. announced positive interim data from its ENERGY 1 trial and Expanded Access Program (EAP) evaluating INZ-701 in infants and young children with ENPP1 Deficiency, completion of enrollment in the ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency and regulatory guidance for the ASPIRE pivotal trial in children with ABCC6 Deficiency.

Inozyme Pharma, Inc. announced encouraging interim results from its ENERGY 1 trial and Expanded Access Program, which evaluated INZ-701 in infants and young children with ENPP1 Deficiency. The trial data showed improvements in survival, heart function, and the stabilization or reduction of ectopic calcification and hypophosphatemia, with no signs of rickets in later evaluations. Additionally, the ENERGY 3 pivotal trial in pediatric patients has completed enrollment, with dosing expected to finish by January 2026 and topline results projected for early 2026.

The company also received regulatory guidance from the FDA and EMA for its planned ASPIRE pivotal trial in children with ABCC6 Deficiency. This trial aims to address severe complications caused by the condition while refining its study design to meet the needs of pediatric patients. These key developments highlight INZ-701’s potential to treat rare pediatric disorders effectively.

Data Publication - July 25,2024

• Drug: INZ-701
• Announced Date: July 25, 2024
• Indication: ABCC6 Deficiency

Inozyme Pharma, Inc. announced the publication of preclinical data supporting the potential of INZ-701, the Company's lead ENPP1 enzyme replacement therapy (ERT) development candidate, to treat a broad range of diseases mediated by the PPi-Adenosine Pathway, which regulates mineralization and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels).

Inozyme Pharma recently announced promising preclinical data for INZ-701, its lead ENPP1 enzyme replacement therapy candidate. The study shows that INZ-701 can prevent the overgrowth of smooth muscle cells inside blood vessels, known as intimal proliferation, in both ENPP1-deficient and normal mice. This finding supports the idea that the therapy may benefit a wide range of diseases involving the PPi-Adenosine Pathway—a key regulator of bone mineralization and blood vessel function.

The research demonstrated that INZ-701 helps generate inorganic pyrophosphate (PPi) and adenosine, which are crucial for regulating mineral buildup and controlling vascular proliferation. These results suggest that INZ-701 might offer a broader therapeutic application beyond traditional enzyme replacement therapy, giving hope for treating various rare diseases linked to disruptions in this pathway.

Designation Grant - July 2,2024

Fast Track

• Drug: INZ-701
• Announced Date: July 2, 2024
• Indication: ABCC6 Deficiency

Inozyme Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency.

Inozyme Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its experimental therapy, INZ-701, for the treatment of ABCC6 Deficiency. Fast Track is a program designed to speed up the development and review of drugs that address serious conditions and fill an unmet medical need. This designation highlights the potential of INZ-701 to become an important therapy, especially for pediatric patients who face increased risks of stroke, severe neurological, and cardiovascular events due to ABCC6 Deficiency. Inozyme plans to work closely with the FDA to streamline the development process and reach an agreement on pivotal study plans by the end of 2024. The decision follows encouraging early clinical data from the ongoing Phase 1/2 trial in adults with the condition.

Top-line data - May 23,2024

Phase 1/2

• Drug: INZ-701
• Announced Date: May 23, 2024
• Indication: ABCC6 Deficiency

Inozyme Pharma, announced that the Company will present recently announced topline data from the Company's ongoing Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (manifesting as pseudoxanthoma elasticum, or PXE), during oral presentations at two upcoming medical conferences.

Inozyme Pharma announced that it will share topline data from its ongoing Phase 1/2 clinical trials of INZ-701 during two upcoming medical conferences. The results being presented focus on adults with ENPP1 Deficiency and ABCC6 Deficiency (which manifests as pseudoxanthoma elasticum or PXE). At the European Calcified Tissue Society Congress in Marseille, France, Dr. Yves Sabbagh will present two studies: one detailing the impact of INZ-701 on bone and mineral metabolism biomarkers and clinical outcomes in ENPP1 Deficiency, and another examining safety and exploratory efficacy in ABCC6 Deficiency. Additionally, at The Endocrine Society’s Annual Meeting in Boston, Dr. Kurt Gunter will present further data on the treatment’s effects in ENPP1 Deficiency. These presentations are expected to offer valuable insights into the potential of INZ-701 as a treatment for these rare, debilitating conditions.

Positive Data - April 8,2024

Phase 1/2

• Drug: INZ-701
• Announced Date: April 8, 2024
• Indication: ABCC6 Deficiency

Inozyme Pharma, Inc. announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company's ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Inozyme Pharma, Inc. announced positive topline results from its ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency. The study showed that INZ-701 was generally well tolerated, with no serious adverse events, and demonstrated a favorable safety and immunogenicity profile. Notably, in ABCC6 Deficiency patients, the treatment led to improvements in vascular pathology and visual function, as seen with both stabilization or reduction in carotid intima-media thickness and increased choroidal thickness. The trial’s pharmacokinetic (PK) and pharmacodynamic (PD) data also revealed a rapid and sustained increase in plasma pyrophosphate levels, reaching levels similar to those in healthy subjects. These promising safety and exploratory efficacy results support further development of INZ-701 as a potential disease-modifying therapy for these rare conditions.

Inozyme Pharma FDA Events - Frequently Asked Questions

Has Inozyme Pharma received FDA approval?

As of now, Inozyme Pharma (INZY) has not received any FDA approvals for its therapy in the last two years.

What drugs has Inozyme Pharma submitted to the FDA?

In the past two years, Inozyme Pharma (INZY) has reported FDA regulatory activity for INZ-701.

What is the most recent FDA event for Inozyme Pharma?

The most recent FDA-related event for Inozyme Pharma occurred on February 21, 2025, involving INZ-701. The update was categorized as "Data Presentation," with the company reporting: "Inozyme Pharma, Inc. announced that Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer, will present recently announced data from the company's Expanded Access Program (EAP) evaluating INZ-701 in infants and children with ENPP1 Deficiency at the CHOP Cardiology Annual Meeting, held February 19-23, 2025, in Orlando, Florida."

What conditions do Inozyme Pharma's current drugs treat?

Currently, Inozyme Pharma has one therapy (INZ-701) targeting the following condition: ABCC6 Deficiency.