Inozyme Pharma's INZ-701 Shows Positive Results

Inozyne Pharma, Inc. has just released positive topline data from the ongoing phase 1/2 clinical trials of INZ-701 in adults with ABCC6 deficiency (PXE) and ENPP1 deficiency. The company announced that the drug demonstrated favorable safety and immunogenicity profiles, as well as promising clinical improvements in vascular pathology, visual function, and patient reported outcomes in adults with ABCC6 deficiency.

In the ABCC6 deficiency trial, 10 adults with heavy disease burden were assigned to three dose cohorts of INZ-701. The trial revealed notable changes, including stabilization and decrease in carotid intima-media thickness, increased choroidal thickness, and preservation and improvement of visual function over 48 weeks. The drug also exhibited a favorable safety profile, with no serious adverse events reported.

Moreover, the company's natural history studies highlighted the high incidence of major clinical events, notably stroke, in pediatric patients with ABCC6 deficiency. As a result, Inozyne Pharma plans to work expeditiously with regulatory agencies on a pivotal trial design for pediatric patients, expecting to initiate the trial in Q1 2025.

In the ENPP1 deficiency trial, 13 adults were enrolled across different dose cohorts. Significant reduction of fibroblast growth factor-23 (FGF-23) and favorable responses on patient-reported outcome measurement information scales (PROMIS) of pain intensity, fatigue, and pain interference were observed. The drug also exhibited a favorable safety profile, with no serious or severe adverse events attributed to INZ-701.

As a result of these announcements, the company's shares have moved 0.5% on the market, and are now trading at a price of $6.44. Check out the company's full 8-K submission here.

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