Faeth Therapeutics has launched a new R&D initiative aimed at preserving neurocognitive function in children with tyrosinemia type 1 (TT1), marking the expansion of its platform beyond oncology into inherited metabolic disorders.

Faeth R&D is supported by MetabOS, a computational model that integrates gene expression and tumor microenvironment data to pinpoint dependencies invisible to conventional approaches, simulating how drug combinations and nutrients interact to propose optimized therapeutic regimens.

While current TT1 therapy prevents liver failure, patients continue to face unmet needs, including neurocognitive impairment. 

Untreated, TT1 can lead to fatal liver and kidney toxicity in infancy. Standard therapy dramatically improves survival but has on-label warnings of variable degrees of intellectual disability and developmental delay. Faeth’s new program aims to change that trajectory.

Anand Parikh, Faeth CEO and co-founder, said “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.”

Faeth expects to file an IND within a year to advance its TT1 program into the clinic. The company anticipates an initial study in older children or adults, followed by long-term cognition endpoints to measure the drug’s ability to protect IQ and neurodevelopment.

“Cross-disease expansion of our discovery platform is very exciting,” added Oliver Maddocks, PhD, Chief Scientific Officer of 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.”

Faeth Therapeutics is a clinical-stage biotechnology company integrating targeted therapies with precision nutrition interventions to exploit metabolic vulnerabilities in cancer. Founded by renowned researchers from Cornell, Columbia, Cambridge, Memorial Sloan Kettering, and the Crick Institute—including Drs. Lewis Cantley, Siddhartha Mukherjee, Karen Vousden, Greg Hannon, and Scott Lowe—Faeth leverages its AI-driven MetabOS discovery platform to identify metabolic targets. Its lead clinical program, combining serabelisib and sapanisertib, is a multi-node approach to more completely inhibit the PI3K/AKT/mTOR pathway, demonstrating promising outcomes in cancers strongly associated with obesity and metabolic disorders. For more information, visit www.faeththerapeutics.com.

MetabOS is Faeth Therapeutics’s machine learning-driven dynamic model of metabolism. The platform leverages AI and functional genomics to identify metabolic vulnerabilities as therapeutic targets. In cancer, Faeth is using MetabOS™ to uncover the precise metabolic vulnerabilities for a tumor based on genotype, organ of origin and therapy.