Small, struggling biotech winds up with a 3X loser as another PhIII of its lead drug collapses
Little Tonix Pharmaceuticals has run into another brick wall as its lead drug — a reformulated muscle relaxant originally approved 44 years ago — has failed another Phase III study, sending shares back into penny stock territory.
Three years after going down in their first Phase III trial of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for symptoms of PTSD, the biotech — which had been encouraged by a breakthrough designation at the FDA — reported late Friday the drug also failed its second late-stage challenge for pain associated with fibromyalgia. Outside data monitors recommended the Phase III trial be halted for futility after deciding interim data made it unlikely the drug would pass muster.
Researchers will now halt enrollment and continue to follow patients already on the drug.
Tonix shares $TNXP, which earlier had to be rehabilitated to stay on Nasdaq, crumbled — dropping 35% and leaving shares at 64 cents at the start of trading Monday.
The failure follows Tonix’s initial reported success in its first Phase III fibromyalgia study.
The drug failed a late-stage trial for symptoms of PTSD in 2018, sending execs back to the drawing board with another Phase III PTSD trial, which also failed. They still have it in the pipeline for PTSD, shifting focus yet again to sleep disturbance.
The latest failure at Tonix underscores that breakthrough designations may make it easier to discuss things with regulators, but it’s no guarantee of success in a clinical trial.
“We are surprised and disappointed that the interim analysis did not support continued enrollment in this Phase 3 RALLY study, especially considering the previous Phase 3 RELIEF study, which had a similar design and achieved statistical significance on the primary endpoint. After the currently enrolled participants complete the study, we will proceed with a full analysis of the unblinded data from the study to determine the next steps in this program,” commented CEO Seth Lederman.