Data: 80% of INZ-701-treated infants with ENPP1 Deficiency survived past first year
Positive interim data was reported from the ENERGY 1 trial and Expanded Access Program that featured a total of 5 infants and 1 child.
Data: 80% of INZ-701-treated infants with ENPP1 Deficiency survived past first year | Image Credit: © Bostan Natalia - © Bostan Natalia - stock.adobe.com.
Positive interim data from Inozyme Pharma has been reported for INZ-701, an investigational ENPP1 enzyme replacement therapy, in young children with ENPP1 Deficiency, including improved survival, according to an announcement from the company.1
Data was reported from the ENERGY 1 trial, a phase 1b, open-label study to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701. Data was also reported from an Expanded Access Program (EAP). ENERGY 1 featured 3 infants while EAP featured 2 infants and 1 child aged 2.5 years.1,2
The trial evaluated patients with generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 Deficiency. Those treated with INZ-701 were treated for periods of 3 weeks to 22 months.1
Results demonstrated that 80% of infants treated with INZ-701 survived beyond their first year compared to a historical survival rate of approximately 50%. Results revealed reductions or stabilization of arterial calcifications in all surviving patients, including complete resolution in some instances and there was no evidence of progression of arterial calcification in any patient, stated Inozyme.1
“We believe these highly encouraging outcomes in infants and young children, combined with previously reported data from adult studies, provide strong support for the potential impact of INZ-701 on rickets, a key clinical endpoint in the ongoing pivotal ENERGY 3 trial, and underscore its potential to address the significant needs of pediatric patients,” said Douglas A. Treco, PhD, CEO and Chairman of Inozyme Pharma, in a statement.1
In all surviving patients, stabilization or improvement in left ventricular ejection fraction was noted, and there was no radiographic evidence of rickets observed in patients evaluated after 1 year of age and at-risk of rickets development (n = 3), supported by stabilization or increases in serum phosphate levels.1
The investigational enzyme replacement therapy was well-tolerated with no serious treatment-related adverse events in infants and young children. Mild injection site reactions were the observed treatment-related events.1
In the ENERGY 1 trial and EAP, higher anti-drug antibody levels in some infants significantly affected PK and PD.1
"In infants with high ADA levels, data collected pre- and post-dosing demonstrated substantial transient increases in PPi and drug exposure following INZ-701 administration, consistent with the clinical effects observed," stated Inozyme Pharma. The company also noted that ADAs were not associated with adverse events in any patient.1
References:
1. Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates. Inozyme Pharma. Press release. January 10, 2025. Accessed January 10, 2025. https://investors.inozyme.com/news-releases/news-release-details/inozyme-pharma-announces-positive-interim-data-inz-701-infants
2. ENPP1 Deficiency Trial in Infants: The ENERGY-1 Study. Inozyme Pharma. Press release. January 10, 2025. Accessed January 10, 2025. https://www.inozyme.com/scientific-focus/clinical-trials/enpp1-deficiency-infant-trial/
