Tonix Pharmaceuticals is getting into the addiction business, licensing a phase 2 treatment for cocaine intoxication from Columbia University. The company is planning to start a pivotal trial for the enzyme, which breaks down cocaine, in 2020.
Unlike opioid dependence or overdose, which can be treated with drugs like naloxone, cocaine intoxication has no specific treatment. Instead, cocaine abusers receive “supportive care,” that is, drugs that treat symptoms of intoxication, which can include agitation, irregular heart rhythms and high blood pressure. These problems may lead to more serious ones such as heart attack, stroke or respiratory failure rhabdomyolysis—”where the muscles kind of eat themselves,” said Tonix CEO Seth Lederman, M.D.
Naloxone works by binding to the same receptors opioids do. This blocks opioid molecules from doing the same and can reverse overdose symptoms. There hasn’t been a similar solution for cocaine abuse because of differences between the opioid receptor and the dopamine transporter, the membrane protein that cocaine binds to.
“After decades of work, nobody has been able to come up with that kind of chemical for the dopamine transporter. So anything that touches the dopamine transporter so far has acted like cocaine,” Lederman said.
Tonix’s new program, TNX-1300, was developed at Columbia University, the University of Kentucky and the University of Michigan and had been licensed to Reckitt Benckiser before the company returned the rights to Columbia. In a 29-patient phase 2 study, the drug, previously called RBP8000, cut plasma cocaine levels in cocaine abusers by 90% within two minutes. It also beat placebo in lowering blood pressure and pulse rate.
Tonix is planning a pivotal phase 2 study to determine whether TNX-1300 can achieve in humans what it has done in animals.
“In animals, what is really intriguing is you can induce seizures in rats, administer the drug and the rat stops seizing. That’s the big promise—the effect of degrading cocaine in the peripheral circulation is so significant that it actually draws the cocaine out of the brain … It has not been done in humans and that is what we hope to find out in phase 2,” Lederman said.
TNX-1300 is Tonix’s first in-licensed drug, and it joins programs aimed at the brain or central nervous system spanning psychiatry, pain and neurology. The company’s lead program, TNX-102 SL, is in the works for post-traumatic stress disorder (PTSD), fibromyalgia and Alzheimer’s disease.
Last month, the U.S. Army Medical Materiel Development Activity (USAMMDA) announced it would end an R&D pact inked with Tonix in 2015 to expand efforts to assess a sublingual form of the long-approved muscle relaxant cyclobenzaprine in patients with PTSD linked to military service. The news came shortly after the FDA said it would rescind the breakthrough designation it had granted TNX-102 SL, also called Tonmya.
After some back and forth with the FDA, Tonmya regained its breakthrough status, at least until Tonix’s meeting with the FDA this August. As for the U.S. Army deal, a key goal was to recruit active-duty personnel from military treatment facilities to participate in phase 2 and phase 3 studies for Tonmya, but USAMMDA did not follow through.
“In the two studies we did, they recruited zero patients. We tried, and it was a good idea, but they were not able to for internal reasons … [The end of the agreement] was not that big a deal to us, but investors had very different opinion,” Lederman said.