Tonix Pharmaceuticals is nearing the finish line with a non-opioid drug for fibromyalgia — potentially the first new treatment for the illness in over a decade.
Fibromyalgia is thought to be caused by a central nervous system malfunction that heightens normal sensations, triggering chronic widespread pain, sleep problems and mood symptoms such as anxiety and depression. People with the condition, primarily women, had few treatment options until the FDA approved Pfizer’s Lyrica, Ely Lilly’s Cymbalta and Forest Laboratories’ Savella between 2007 and 2009. But these drugs don’t work for everyone, and people sometimes abandon treatment due to side effects such as weight gain, sexual dysfunction and high blood pressure.
To breathe new life into the space, Tonix turned to cyclobenzaprine, a muscle relaxer that’s been approved since the 1970s.
“For years there's been this holy grail of a non-addictive pain drug,” said Dr. Seth Lederman, president and CEO of Tonix.
However, oral formulations of cyclobenzaprine can bring their own host of unwanted effects. For their own investigational therapy, TNX-102 SL, Tonix uses a patented formulation that “has been designed to optimize the delivery and absorption of cyclobenzaprine for the therapeutic benefit of improving sleep quality,” which in turn, can improve pain.
In December, the company announced clinically meaningful and statistically significant results from a phase 3 trial — its second positive late-stage readout. The company now has a pre-NDA meeting with the FDA in the first quarter of this year and is hoping to deliver a new tool in efforts to ease symptoms for the estimated 4 million American adults with fibromyalgia, while exploring its potential reach in other areas.
The drug’s broader potential
Axsome is also nearing the finish line with its investigational norepinephrine reuptake inhibitor for fibromyalgia, which it licensed from Pfizer. It also saw positive results in phase 3.
The fibromyalgia drug market is estimated to reach an estimated $4.6 billion by 2032, and Lederman said applications for TNX-102 SL could eventually expand. The company is exploring the drug as a treatment for post-traumatic stress disorder, alcohol use disorder, Alzheimer’s agitation and long COVID.
"I think it’s a step closer to the solution."
Dr. Seth Lederman
CEO, president, Tonix Pharmaceuticals
Data from the second TNX-102 SL phase 3 trial, which included 457 patients at 33 sites, found that the drug, helped relieve fibromyalgia symptoms without a harsh side effect profile, making it a viable long-term therapy — a critical goal for a condition that people often live with for two or three decades.
Importantly, the drug doesn’t target fibromyalgia pain directly, but instead relieves symptoms through a better night’s sleep.
“We call [TNX-102 SL] broad spectrum because it worked not only in pain, but it also improved fatigue and improved sleep,” Lederman said. “Our tolerability was phenomenal.”
It didn’t cause weight gain or high blood pressure, and women taking the drug said their sexual function improved, likely because they were feeling better overall, he said. Other impacts like headaches, sleepiness and tongue numbness were uncommon and temporary.
“Our concept is that disturbed sleep is an obstacle to recovery,” he said.
The results show the drug may have promise not only for fibromyalgia but other conditions caused by an altered pain response, known as nociplastic pain, which includes rheumatoid arthritis, lupus, chronic primary bladder pain syndrome, irritable bowel syndrome and migraines.
“The suffering involved in nociplastic syndromes is enormous,” Lederman said, adding that disturbed sleep may perpetuate these conditions, make them chronic and lead to functional impairment. Lederman said he’s hopeful that the path to approval for TNX-102 SL will be smooth.
“I think it’s a step closer to the solution,” Lederman said.
