PHILADELPHIA, PA — iECURE announced that new clinical data from its ongoing OTC-HOPE trial evaluating ECUR-506 in neonatal-onset ornithine transcarbamylase deficiency will be presented this month at two medical conferences.
The company said preliminary results from the first three dose cohorts of the OTC-HOPE study will be presented during the American Society of Gene & Cell Therapy annual meeting in Boston from May 11-15.
According to iECURE, data from seven patients showed a decreased rate of hyperammonemic crises following administration of ECUR-506. Hyperammonemic crises are considered among the most severe complications of ornithine transcarbamylase deficiency and can be life-threatening.
The ASGCT presentation, titled “Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration: preliminary update from the OTC-HOPE study,” is scheduled for Wednesday, May 13, from 4:00 p.m. to 4:15 p.m. Eastern time.
Gabriel Cohn, the company’s chief medical officer, will deliver the oral presentation.
A second presentation at the Society for Inherited Metabolic Disorders annual meeting in Puerto Rico from May 17-20 will include one-year follow-up data from the first infant treated in the study.
The company said the patient achieved a complete clinical response under the study protocol, including sustained discontinuation of standard-of-care therapies one year after treatment.
The SIMD poster presentation, titled “Sustained Clinical Response in First Clinical Trial Patient Dosed with ECUR-506 One Year Post Discontinuation of Standard of Care,” is scheduled for Monday, May 18, from 8:00 p.m. to 9:00 p.m. Eastern time.
George Diaz, vice president and therapeutic area lead for urea cycle disorders, will present the findings.
iECURE said it will also host an exhibitor booth during the SIMD conference at the Wyndham Rio Mar Beach Resort in Puerto Rico.
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