Trethera Receives NIH Grant for Preclinical Development in the Pediatric Neurologic Disease Acute Disseminated Encephalomyelitis

Los Angeles, August 14, 2023 — Trethera Corporation, a clinical stage biopharmaceutical company committed to developing novel drugs targeting nucleotide metabolism for the treatment of cancer and autoimmune diseases, announced today that it was awarded a $600,000 Small Business Technology Transfer (STTR) grant from the National Institute of Allergy and Infectious Diseases (NIAID).   Trethera’s first-in-class small molecule, TRE-515, holds the only FDA Orphan Drug designation for Acute Disseminated Encephalomyelitis (ADEM), an acute neurologic disease principally of children. 

ADEM is an autoimmune disease that can present with fever and difficulty walking as well as loss of consciousness and coma. Viral infections, such as influenza, mumps, or COVID, frequently precede the disease.  ADEM is a rare disease, affecting 12,000 to 15,000 patients a year in the United States, with most cases occurring in 6 to 8 year-old children. The grant will advance IND enabling studies for a disease where no approved therapies currently exist.

TRE-515 has also received FDA Orphan Drug designation for the treatment of ADEM, a designation that confers substantial advantages, including FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the drug approval application fee, and eligibility for seven years of marketing exclusivity.  Furthermore, should the FDA approve TRE-515 for commercial use in ADEM, Trethera would be eligible for a pediatric priority review voucher.

“Achieving both NIH funding as well as FDA Orphan Drug designation confers external validation of our potential to treat ADEM,” said Dr. Ken Schultz, principal investigator and Trethera CEO. “Our team is highly motivated by the potential to develop the first FDA approved treatment to save children’s lives and improve outcomes in ADEM. Our strategy of combining TRE-515 commercial development for a rare disease with ongoing efforts to treat more common diseases, such as solid tumors and multiple sclerosis, provides multiple FDA approval opportunities, thereby reducing overall development risk.” 

“Approximately half of ADEM patients recover with hospitalized intensive care.  For others, ADEM can be fatal or lead to lifelong disability.  Any drug that could improve these outcomes for patients would be groundbreaking.  TRE-515 could potentially significantly benefit ADEM patients beyond the available therapeutic options,” said Trethera Scientific Advisory Board member Dr. Larry Steinman.  Dr. Steinman is a distinguished immunologist and pediatric neurologist at Stanford University. 

“The pathology of ADEM involves a severe bout of inflammation in the central nervous system that can include the brain, spinal cord, and sometimes the optic nerves. The inflammation damages myelin, the protective substance that coats nerve fibers throughout the central nervous system. No medications have been specifically approved by the FDA to treat ADEM,” said UCLA’s Dr. Peter Clark, member of the Trethera Scientific Advisory Board and grant co-investigator.

A discussion summary from the independent panel of NIAID experts that reviewed Trethera’s proposal noted the “rationale for the target is credible and preliminary data is strong… great rigor with third party validation… research methods are appropriate to enable commercial development… significance and commercial potential are high as there is a high unmet need… strong potential for broader application to progressive MS and other diseases.”

Sources: J Neurol. 2020 Oct;267(10); Int Care Med. 2008 Mar;34(3); Neuro. 2001 May 22;56(10)

About TRE-515

TRE-515 is an orally delivered, once daily, therapeutic engineered to inhibit dCK, the key enzyme in the nucleoside salvage pathway.  A common characteristic of tumor cells in solid malignancies and pathological immune cells in autoimmune diseases is the requirement for elevated nucleotide levels to support abnormal and accelerated cell division.  In contrast, dCK activity is not required in most healthy adult human cells.  Mediated by the rate limiting enzyme, dCK, the nucleoside salvage pathway may play a pivotal role in enabling the rapid cell proliferation of cancer cells and aberrant activated lymphocytes, suggesting dCK as a potential therapeutic target with expected enhanced safety.

About Trethera

Trethera is a clinical stage privately held biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera’s innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug.  TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors.  It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases, such as multiple sclerosis, might also respond to TRE-515 treatment.  Trethera is developing TRE-515 for use as a monotherapy or in combination, to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients.

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