- Tonix Pharmaceuticals on Tuesday said it will end further development of an experimental drug for fibromyalgia, focusing instead on the drug's potential in treating post-traumatic stress disorder (PTSD).
- The drug, known as TNX-102, failed to hit its pre-specified efficacy endpoint in a Phase 3 study across 519 patients with fibromyalgia. While Tonix did its best to dress up the negative results, citing other statistical analyses, company CEO Seth Lederman said development in fibromyalgia would be discontinued going forward.
- Instead, Tonix hopes to prove TNX-102's efficacy as a treatment for PTSD and plans to begin two Phase 3 trials for that indication next year. Investors may not be that patient, however. Tonix stock fell by nearly 60% Tuesday on news of the trial miss and change in strategy.
"Despite achieving clinically meaningful results from AFFIRM, we have greater clarity on the regulatory path forward in our PTSD program," Lederman said in a statement. "We will therefore discontinue the fibromyalgia program in order to fully focus Tonix’s resources on advancing our potential breakthrough PTSD program to Phase 3."
TNX-102 failed to reach statistical significance as measured by the proportion of patients who reported a 30% or higher reduction in pain compared to baseline through 12 weeks — the pre-specified endpoint for the Phase 3 trial. But that didn't stop Tonix from trying to prove TNX-102 underlying efficacy.
Tonix gave results from five other statistical analyses which the company claims demonstrate "clear evidence of beneficial effect." The company also noted unexpected discontinuations from reasons unrelated to efficacy or tolerability, such as a patient moving away from a trial site, negatively influenced the primary responder analysis.
Still, the company is shutting down its development efforts in fibromyalgia and moving ahead with its PTSD plans for TNX-102.
The drug is also designed to target sleep quality and stress, both characteristics of PTSD, so salvaging some evidence of safety and efficacy from the Phase 3 in fibromyalgia could point to better results in upcoming PTSD trials.
Tongue numbness, fatigue and somnolence were the three most common adverse reactions observed in patients in the most recent trial.
Tonix recently received the go-ahead from the Food and Drug Administration to move forward with its plans for the two Phase 3 studies in PTSD.
But some new financing might be in order to fund further R&D. As of June 30, Tonix had roughly $15 million in cash and equivalents, with another $16 million in marketable securities. Operating activities over the six months through June 30 used up over $23 million.