GC Pharma signs agreement with Austin's Speragen

GC Pharma's headquarters in Yongin, Gyeonggi [GC PHARMA]

GC Pharma signed a partnership agreement with Austin, Texas-based Speragen for joint development of a treatment for succinic semialdehyde dehydrogenase deficiency (Ssadhd), the company said Tuesday.

Ssadhd is a rare autosomal recessive disorder that affects the degradation of gamma-aminobutyric acid, major inhibitory neurotransmitter of the brain. Ssadhd is estimated to occur in one person per million — mainly young babies — according to GC Pharma. Symptoms include epilepsy and intellectual disabilities.

Currently, there is no treatment available for the disease.

GC Pharma said the two companies agreed to develop an enzyme replacement treatment using Ssadh proteins. The Korean company will be entitled to plasmid-related patents for Ssadhd protein production.

Speragen will be responsible for patient-focused drug development with the U.S. Food and Drug Administration and neonatal screening to secure new patients, while GC Pharma will be responsible for conducting clinical trials and biomarker research based on its enzyme treatment technology. It aims to start Phase 1 and 2 clinical trials in the second half of 2023.

"This cooperation will strengthen GC Pharma’s pipeline for rare diseases," GC Pharma CEO Huh Eun-chul said in Tuesday’s statement.

BY SARAH CHEA [chea.sarah@joongang.co.kr]