ALS partnership to give 6 patients expanded access to TRE-515
Mass General, Trethera to evaluate treatment using FDA program
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- Six ALS patients will receive experimental TRE-515 via FDA expanded access.
- TRE-515 aims to reduce inflammation by blocking an enzyme.
- The therapy will be evaluated for safety, tolerability, and disease progression.
Up to six people with advanced amyotrophic lateral sclerosis (ALS) will be treated with Trethera’s experimental therapy TRE-515 under a collaboration with Massachusetts General Hospital (MGH).
The patients will receive the oral treatment through a U.S. Food and Drug Administration (FDA) expanded access program (EAP), which allows people with serious conditions to try investigational therapies outside of clinical trials.
One ALS patient received TRE-515 under the FDA program last year, and early signs suggest the therapy may help stabilize disease progression and improve some measures of strength and quality of life, according to Trethera. The new collaboration will build on the data from that patient.
Researchers at MGH will assess TRE-515’s safety, tolerability, and biological activity. The trial will also track changes in disease progression using clinical measures and biomarkers.
“Therapeutic options for ALS remain limited, and there is a continuing need to explore novel approaches for patients facing this serious disease,” Merit Cudkowicz, MD, executive director of the Massachusetts General Brigham Neuroscience Institute and principal investigator for the trial, said in a press release from Trethera. “We are pleased to collaborate with Trethera to further evaluate this investigational therapy.”
Treatment blocks enzyme, aiming to quell abnormal immune responses
ALS is caused by the progressive loss of motor neurons, the nerve cells that control voluntary muscle movement, leading to worsening muscle weakness. While the exact causes of ALS are not fully understood, inflammation in the nervous system is believed to play a role in driving disease progression.
TRE-515 is a small molecule designed to block deoxycytidine kinase, an enzyme needed for immune cell activity and inflammation. By inhibiting this enzyme, the therapy aims to dampen abnormal immune responses that may contribute to nerve cell damage in ALS.
The new partnership builds on data from the ALS patient who was treated with TRE-515 last year in a separate collaboration.
The patient, who had advanced disease and had received 11 therapies over 3.5 years without meaningful clinical benefit, experienced a slowing in lung function decline after starting treatment, along with reported improvements in certain selected quality-of-life measures, including strength and vocalization.
While the person later died from pneumonia consistent with the natural course of ALS, no treatment-related safety issues were reported, supporting further investigation of the therapy.
“This collaboration with MGH follows our encouraging ALS clinical findings last year and is an important step in expanding our clinical efforts to understand and treat this devastating disease,” said Ken Schultz, MD, Trethera chairman and CEO. “MGH was among the first academic centers to engage with Trethera, and we hope to expand participation to other leading institutions following the successful conclusion of this trial.”
Patients in the MGH trial will undergo regular evaluations using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard measure of functional ability, and slow vital capacity (SVC), which assesses lung function. Researchers will also track biomarkers, such as neurofilament light chain (NfL), a protein linked to nerve cell damage, to better understand how the therapy affects disease activity.
“We are particularly encouraged by the potential of treating ALS with TRE-515 given its ability to selectively modulate inflammation and its favorable safety profile observed to date,” said Lawrence Steinman, MD, a neurology professor at Stanford University and Trethera scientific advisory board member. “While this initial pilot study is small, it builds on promising early clinical observations. A favorable readout could support expansion into larger, more definitive trials and further advance the clinical development of TRE-515 in ALS.”
TRE-515 is also being developed for other autoimmune diseases, including multiple sclerosis, lupus, inflammatory bowel disease, and certain types of cancer.
