Trethera Administers TRE 515 to Advanced-Stage ALS Patient

Trethera Corporation, a biopharmaceutical company in its clinical stage, has unveiled promising clinical outcomes from its treatment of a patient diagnosed with Amyotrophic Lateral Sclerosis (ALS), using their novel drug, TRE-515, under the FDA's Expanded Access program. ALS, also referred to as Lou Gehrig’s disease, is a severe neurodegenerative condition characterized by the gradual degeneration of nerve cells in the brain and spinal cord, leading to the loss of muscle control. The patient, who had been diagnosed in May 2022, had previously pursued over ten different treatment options, including the sole FDA-approved drug, Riluzole, without success.

TRE-515, developed by Trethera, is an innovative drug currently undergoing dose escalation trials for treating solid tumors. It targets deoxycytidine kinase (dCK), a crucial enzyme within the nucleoside salvage pathway. Prior to the trial with TRE-515, the patient's forced vital capacity—a critical indicator of lung function—had significantly declined, dropping from 60% to 37%. However, following a three-month treatment regimen with TRE-515, the patient's disease showed signs of stabilization. This stabilization was observed not only in forced vital capacity but also in the Revised ALS Functional Rating Scale (ALSFRS-R), with noted improvements in body weight, neck tone, and arm strength. Remarkably, no adverse effects were reported during the treatment, prompting a unanimous decision from the multicenter medical review group to increase the dosage and extend the trial period.

Dr. Ken Schultz, CEO of Trethera, highlighted the significance of this trial as it marked the first clinical application of TRE-515 beyond cancer treatments. He emphasized the drug's potential to improve survival rates and quality of life for ALS patients, a condition for which FDA-approved therapies are extremely limited. Dr. Frank Diaz, a neurologist at Cedars-Sinai, underscored the importance of exploring alternative treatments for ALS patients who have exhausted available therapies, marking TRE-515 as a pioneering advancement in the field. Dr. Dan Kelly from the Pacific Neuroscience Institute Foundation expressed optimism regarding the drug's selective modulation of inflammation and its promising safety profile, stating their eagerness to further develop this promising therapeutic option.

The patient enrolled in this treatment under the FDA's Expanded Access program, designed to offer investigational treatments to individuals with life-threatening conditions when no satisfactory alternatives exist. These trials are carefully structured to align with ongoing clinical development, offering benefits such as enhanced patient engagement and insightful data collection, which can guide future research. However, to establish clinical efficacy, larger randomized trials will be needed in the future.

Trethera is at the forefront of developing innovative therapies targeting nucleotide metabolism, with TRE-515 being a first-in-class drug that inhibits dCK within the nucleoside salvage pathway. This pathway is crucial for DNA precursor generation, and certain cancers as well as autoimmune diseases may depend on it for growth and maintenance. By focusing on this metabolic vulnerability, Trethera aims to revolutionize treatment outcomes for patients. TRE-515 has been recognized by the FDA as an Orphan Drug, highlighting its potential to address unmet medical needs in rare conditions.

In collaboration with Cedars-Sinai and Saint John's Health Center, Trethera continues its commitment to exploring groundbreaking therapies that could fundamentally change the landscape of ALS treatment and potentially other autoimmune conditions.