Tonix Says AFFIRM Data Didn't Achieve Primary Efficacy Endpoint; Stock Down

(RTTNews) - Tonix Pharmaceuticals Holding Corp. (TNXP) announced preliminary results from its Phase 3 clinical study, AFFIRM, designed to evaluate the safety and efficacy of TNX-102 SL (cyclobenzaprine HCl sublingual tablets), 2.8 mg, in patients with fibromyalgia.

In the pre-Market trade, TNXP is currently trading at $1.83, down $0.35 or 16.06 percent.

AFFIRM was a 12-week randomized, double-blind, placebo-controlled trial of TNX-102 SL taken daily at bedtime, in which 519 participants were enrolled at 35 centers in the U.S. Fibromyalgia is a multi-symptom disorder that originates in the central nervous system and is characterized by widespread pain, non-restorative sleep, fatigue, and disability.

The AFFIRM data did not achieve statistical significance in the primary efficacy endpoint: the proportion of patients who reported a 30 percent or greater reduction in pain from baseline to the end of the 12-week treatment period based on the pre-specified primary analysis (p=0.095, Table 1).

However, TNX-102 SL did show statistically significant effects on pain when analyzed by other standard statistical approaches. TNX-102 SL activity in fibromyalgia was cross-validated by two additional endpoints, Patient Global Impression of Change (PGIC) and Fibromyalgia Impact Questionnaire-Revised (FIQ-R) (Table 2). These endpoints assess global improvement and a range of fibromyalgia symptoms and function.

TNX-102 SL showed strong effects on improving sleep quality by the daily diary and the PROMIS sleep disturbance scale. The internal consistency of these results provides clear evidence of beneficial effect of TNX-102 SL for the treatment of fibromyalgia.

An unexpected imbalance in patient discontinuations for reasons unrelated to efficacy or tolerability , created a negative bias in the primary responder analysis because any patient who left the study, for any reason prior to completion, was labeled a non-responder despite their results up to that point. Another standard statistical method for assessing the 30 percent responder analysis that considers the reason for discontinuation showed statistical significance in the primary pain data (Table 1, P=0.012).

Overall, TNX-102 SL was well-tolerated in the AFFIRM study and the adverse events reported were similar to those seen in other TNX-102 SL clinical studies (Table 4). There were seven serious adverse events (SAEs) reported during the study: four in the placebo group and three in the active group. No new safety signals were observed; multiple causal factors were involved in each SAE, and all were resolved quickly and without sequelae.