AUSTIN, Texas– Faeth Therapeutics, a clinical-stage company developing therapies that target tumor metabolism, has launched a new research program aimed at preserving neurocognitive function in children with tyrosinemia type 1 (TT1). The initiative marks the company’s first expansion beyond oncology into inherited metabolic disorders.
The program is supported by MetabOS, Faeth’s proprietary computational model that integrates gene expression and tumor microenvironment data to reveal hidden biological dependencies. By simulating the interplay between nutrients and drug combinations, the platform proposes optimized therapeutic regimens that are not visible through conventional drug development.
While standard therapy for TT1 prevents liver failure, patients often face lasting unmet needs, including neurocognitive impairment. Left untreated, TT1 can cause fatal liver and kidney toxicity in infancy. Although current treatment significantly improves survival, it carries on-label warnings of developmental delays and intellectual disabilities. Faeth’s new program seeks to change this trajectory.
“We have demonstrated that interrogating biology through a metabolic lens reveals insights not apparent in conventional drug development, and that these insights can be used not just in oncology, which is reflected in our lead clinical programs, but also in rare diseases,” said Anand Parikh, Chief Executive Officer and co-founder of Faeth.
The company expects to file an Investigational New Drug (IND) application within the next year to advance its TT1 program into clinical testing. Initial studies are anticipated in older children or adults, with long-term endpoints designed to measure the therapy’s ability to protect IQ and neurodevelopment.
“Cross-disease expansion of our discovery platform is very exciting,” said Oliver Maddocks, Ph.D., Chief Scientific Officer at Faeth. “The fact that our R&D has yielded programs in oncology and now rare disease demonstrates its potential to systematically open up new categories of metabolism-driven therapies.”
