Trethera Gets $2M NIH Grant for Preclinical Development in Pediatric Neurologic Disease ADEM

Trethera Corporation, a biopharmaceutical company in the clinical stage, has been awarded a $2 million grant by the NIH Small Business Innovation Research (SBIR) program. This funding aims to support preclinical studies for treating acute disseminated encephalomyelitis (ADEM), a rare neurological condition primarily affecting children. Trethera's drug, TRE-515, has the distinction of being the only medication to receive FDA Orphan Drug designation for ADEM.

ADEM is an autoimmune disease that often follows viral infections like influenza, mumps, or COVID-19. Symptoms can include fever, difficulty walking, loss of consciousness, and in severe cases, coma. In the United States, the disease affects approximately 12,000 to 15,000 children yearly, with most cases occurring in those aged six to eight. Currently, no approved therapies exist for ADEM, making the development of TRE-515 particularly significant.

The FDA Orphan Drug designation for TRE-515 provides several benefits, including assistance in designing clinical trials, access to grants, exemption from a $4 million drug approval fee, and seven years of marketing exclusivity upon approval. Furthermore, if TRE-515 is approved for commercial use, Trethera would qualify for a pediatric priority review voucher. Trethera intends to use data from the NIH-funded studies and the Orphan Drug designation to work with the FDA on the fastest clinical trial path to bring the drug to market.

Dr. Ken Schultz, the principal investigator and CEO of Trethera, expressed confidence in the potential of TRE-515. He noted that the company’s strategy combines developing treatments for rare diseases like ADEM with efforts to address more common conditions, such as solid tumors and Crohn's disease. This dual approach aims to increase the chances of receiving FDA approval while reducing overall development risks.

Approximately half of ADEM patients recover with intensive care, while the rest may face fatal outcomes or lifelong disabilities. Dr. Larry Steinman, a member of Trethera’s Scientific Advisory Board and a distinguished immunologist and pediatric neurologist at Stanford University, emphasized the potential of TRE-515 to improve patient outcomes significantly. He highlighted that a drug capable of enhancing recovery rates would be groundbreaking.

Dr. Peter Clark from UCLA, another member of Trethera’s Scientific Advisory Board, explained the pathology of ADEM. The disease involves severe inflammation in the central nervous system, damaging myelin, which protects nerve fibers. Without FDA-approved medications specifically for ADEM, the development of TRE-515 is particularly promising.

The NIH review panel praised Trethera’s proposal, noting its strong scientific basis, established safety in Phase I clinical trials for solid tumors, and high commercialization potential. Dr. Michael Levy from Harvard, also a member of Trethera’s Scientific Advisory Board, acknowledged the complexities involved in treating ADEM patients. He looked forward to the day when TRE-515 could be FDA-approved, providing much-needed assurance to affected children and their families.

Trethera Corporation, founded by UCLA scientists, focuses on developing new treatments for autoimmune diseases and cancers. Their flagship drug, TRE-515, is an orally administered capsule that inhibits deoxycytidine kinase (dCK), a key enzyme in a pathway that generates DNA precursors. This approach aims to target metabolic vulnerabilities in cancer and autoimmune diseases, offering new hopes for patient outcomes.