Tonix Pharmaceuticals Enrolls First Patient in Phase 2 CATALYST Study of TNX-1300 for Cocaine Intoxication Treatment

In 2022, over 27,569 people in the United States succumbed to drug overdose deaths involving cocaine. Despite the staggering number, there currently exists no FDA-approved treatment specifically targeting cocaine intoxication. In response, Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a biopharmaceutical company, is spearheading a critical Phase 2 clinical trial to address this gap. The trial involves TNX-1300, a recombinant enzyme intended to treat acute cocaine intoxication in emergency department settings.

The trial, known as CATALYST, is a single-blind, placebo-controlled, proof-of-concept study. This phase of research is designed to evaluate the efficacy and safety of TNX-1300 in real-world emergency department conditions. The enzyme TNX-1300 has shown promise in earlier studies by rapidly degrading and metabolizing cocaine, thus potentially offering life-saving intervention for acute cocaine intoxication. This claim is supported by prior Phase 2a clinical studies, which demonstrated the enzyme's ability to swiftly reduce the physiological effects of cocaine.

In a significant development, Tonix has secured a Cooperative Agreement Grant from the National Institute on Drug Abuse (NIDA), a division of the National Institutes of Health (NIH), to aid in the development of TNX-1300. Furthermore, the FDA has granted TNX-1300 Breakthrough Therapy designation, underscoring its potential to meet an urgent medical need.

The Phase 2 trial will involve approximately 60 participants who present to the emergency department with symptoms of cocaine intoxication. These subjects will either receive a single 200 mg dose of TNX-1300 or a placebo injection, in addition to the standard of care. The primary goal is to measure the reduction in systolic blood pressure 60 minutes post-treatment. Secondary endpoints include reductions in circulating cocaine and its metabolites.

Seth Lederman, M.D., CEO of Tonix Pharmaceuticals, highlighted the critical nature of this study. He noted that the current standard of care for cocaine intoxication primarily focuses on managing symptoms rather than addressing the root cause. TNX-1300 aims to fill this void by targeting the cocaine itself, thus offering potential advantages over existing treatments.

TNX-1300 is produced using recombinant DNA technology and is derived from a non-pathogenic strain of E. coli. The enzyme, originally identified in bacteria that use cocaine as their sole source of carbon and nitrogen, catalyzes the breakdown of cocaine into less harmful metabolites. This innovative approach could revolutionize emergency treatment protocols for cocaine intoxication.

Cocaine, a potent stimulant, is known for its euphoric effects but also has a high potential for abuse and addiction. Pharmacologically, it blocks the reuptake of dopamine in the brain, leading to excessive accumulation and heightened neural stimulation. This mechanism underlies both its addictive properties and the severe health risks associated with overdose, including cardiac arrhythmias, hypertension, and, in severe cases, myocardial infarction and stroke.

The urgency for an effective treatment is underscored by the sheer number of emergency room visits related to cocaine use. Annually, around 505,000 such visits occur in the U.S., with 61,000 requiring detox services. The National Institute on Drug Abuse reported that in 2022 alone, cocaine-related overdose deaths affected over 27,569 individuals.

Tonix Pharmaceuticals is committed to addressing this public health crisis. The company's broader portfolio includes treatments for various central nervous system disorders, autoimmune diseases, and infectious diseases. Among its notable projects is TNX-102 SL for fibromyalgia, which has received Fast Track designation from the FDA.

In summary, the ongoing Phase 2 trial of TNX-1300 represents a pivotal step in addressing the urgent need for an effective treatment for cocaine intoxication. With the support of NIDA and the FDA’s Breakthrough Therapy designation, Tonix Pharmaceuticals aims to bring this potentially life-saving enzyme to the market, significantly impacting public health outcomes related to cocaine overdose. Topline results from this crucial study are anticipated in the first half of 2025.