DENVER, Colo. (247marketnews.com) -- Vistagen (NASDAQ:VTGN) has given investors a potentially important new piece of the fasedienol puzzle, but one that needs to be viewed alongside a major setback earlier this year.
The company announced preliminary data from the open-label extension of its PALISADE-4 Phase 3 study showing that 322 patients who used fasedienol as needed for up to 12 months experienced no new drug-related safety findings, while exploratory measures of social anxiety improved over the first four months. More than 95% of treatment-emergent adverse events were mild or moderate, and no serious adverse events were considered related to fasedienol.
The efficacy numbers make the update particularly interesting.
On the clinician-administered Liebowitz Social Anxiety Scale (LSAS), the average improvement from baseline increased from 20.2 points at Month 1 to 31.4 points at Month 4. The percentage of participants achieving at least a 20-point improvement rose from 44% to 65%. On the patient-reported Social Phobia Inventory (SPIN), average improvement reached 14.9 points by Month 4.
Chief Medical Officer Angel S. Angelov said the results are important because they reflect “the potential impact of repeated, as-needed use of fasedienol in everyday life settings where social anxiety symptoms occur.”
There is a giant asterisk.
In June, the randomized, placebo-controlled portion of PALISADE-4 failed to achieve its primary endpoint in the overall population. Fasedienol produced an LS mean SUDS change of -9.5 versus -11.4 for placebo, with a p-value of 0.427. A post-hoc analysis of patients with very severe social anxiety produced a nominally statistically significant result, but that was a subgroup analysis rather than the trial's primary outcome.
That failure forced Vistagen to rethink the regulatory path.
Rather than simply pursuing the original acute-treatment strategy, the company said it plans to discuss with the FDA a potential registrational pathway focused on treatment of social anxiety disorder over time, potentially using the LSAS as the primary endpoint in a future Phase 3 study, which makes the new OLE data more than another safety update.
They potentially provide Vistagen with evidence supporting the very clinical concept the company is now emphasizing: that repeated, patient-directed use of fasedienol may be associated with improvement in the broader burden of social anxiety, including fear, anxiety and avoidance.
The company has also been building a substantial safety database. Vistagen previously reported more than 1,500 people exposed to fasedienol, including more than 100 with at least 12 months of exposure.
The catch is that the OLE is open-label and uncontrolled. There is no placebo comparison in these observations, so the improving LSAS and SPIN scores cannot establish that fasedienol itself caused the improvement. Vistagen's own subsequent corporate disclosure explicitly cautions that the OLE efficacy observations should be interpreted carefully because the study was open-label and uncontrolled.
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