Trethera Awarded $1.6M to Develop TRE-515 as Optic Neuritis Treatment

National Eye Institute grant will advance oral therapy for inflammation

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
An eye is highlighted in a telescope lens as a person looks at the stars.

Trethera has been awarded a $1.6 million grant to further develop TRE-515 as a potential treatment for optic neuritis — inflammation of the nerves that transmit data from the eyes to the brain — which also is a common symptom of multiple sclerosis (MS).

TRE-515 is a first-in-class treatment for optic neuritis that has already won orphan drug status from the U.S. Food and Drug Administration (FDA) — which has not approved a therapy for this disease in more than 50 years.

The Small Business Technology Transfer commercialization grant for Trethera comes from the National Eye Institute (NEI), a division of the National Institutes of Health.

The grant was awarded through the “fast track” category, which is “perhaps the most competitive at the NEI, and a mark of distinction,”  Michael Levy, MD, PhD, a Trethera grant supporter at Massachusetts General Hospital, said in a company press release.

“As an [optic neuritis] treatment has not been approved by the FDA since 1952, I look forward to helping TRE-515 continue its progress to the clinic for this disease,” Levy said.

Recommended Reading

Optic neuritis is characterized by problems with vision, and some people who initially experience optic neuritis will go on to develop MS. Anti-inflammatory steroids can help resolve an attack of optic neuritis, but do not affect long-term outcomes or the risk of developing multiple sclerosis.

Developing TRE-515 as an optic neuritis treatment

“For some patients,  ON [optic neuritis] can be self-resolving, but for others ON can lead to lifelong disability. In all ON cases, the threat of future conversion to MS remains,” said Larry Steinman, MD, a member of Trethera’s scientific advisory board and supporter of the grant.

“TRE-515 could potentially provide significant benefit to ON patients beyond the available therapeutic options, especially those taking long-term steroids,” Steinman said, adding, “Any drug that could improve these outcomes for patients would be game changing.” Steinman said.

The oral medication is designed to reduce the activity of inflammatory immune cells that drive disease in optic neuritis. The experimental therapy works by blocking the activity of a protein called dCK, which is a key component of the biochemical pathways that cells use to recycle and salvage nucleotides — the building blocks of DNA.

In autoimmune diseases like optic neuritis and MS, the inflammatory immune cells that drive the disease are rapidly dividing, and that means they need a lot of nucleotides to copy their DNA and make more cells. Thus, blocking dCK is expected to reduce the amount of nucleotides available to these cells, preventing their rapid expansion.

In animal models of MS, treatment with TRE-515 significantly delayed the onset of symptoms and eased disease severity.

Blocking dCK also is being explored as a therapy for tumors, which are defined by uncontrolled cell growth. According to Trethera, although dCK is required for the growth of some immune and cancer cells, the protein’s activity is not required for most healthy adult cells, making it a highly specific therapeutic target.

Funds from the newly awarded grant will be shared by Trethera and scientists at the University of California Los Angeles (UCLA), with support from researchers at Stanford and Harvard. Scientists at the three universities will be conducting tests in mice to see how different doses of TRE-515 impacts the medicine’s effects.

The teams also will conduct studies that aim to support an investigational new drug (IND) application — a formal request to regulatory authorities in the U.S. to begin clinical testing of TRE-515 in people.

“We are excited and grateful for this NEI funding as it allows Trethera to build upon our promising preclinical work conducted to date,” said Ken Schultz, MD, Trethera’s CEO and grant principal investigator.

Added Peter M. Clark, PhD, a professor at UCLA: “The use of animal models to evaluate safety and efficacy is essential to the responsible and successful development of novel therapies.”

Clark, who is heading the parts of the project based at the university, called the preclinical data to date “very encouraging.”

“We believe that the work supported by this new grant will provide important information that will help to optimize the design of human clinical trials for ON,” he said.

Dancing Doodle

Did you know some of the news and columns on Multiple Sclerosis News Today are recorded and available for listening on SoundCloud? These audio news stories give our readers an alternative option for accessing information important for them.

Listen Here