Levo Therapeutics misses primary endpoint in PhIII trial of Prader-Willi drug — the latest setback in a disaster-prone field
Marking yet another setback in the Prader-Willi Syndrome field, Levo Therapeutics failed to hit its primary endpoint in a Phase III study of intranasal carbetocin. But the biotech is now shifting its focus to the secondary endpoints in an effort to pluck victory out of the jaws of defeat.
The disorder, characterized by a false sense of starvation, is caused by the absence or deletion of a father’s chromosome 15. Illinois-based Levo’s potential therapy involves a selective oxytocin-receptor agonist.
Two doses of Levo’s LV-101 were tested against a placebo across eight weeks. Topline results show that the 9.6 mg dose missed the mark on primary measurements, including improvement to hyperphagia and obsessive compulsive behavior. But “statistical significance” was achieved with the secondary dose of 3.2 mg, according to the company.
“Consistency in benefit/response was observed in the 3.2 mg dose arm across other key secondary endpoints, including clinical global impression of change (CGI-C; p=0.027) and anxiety and distress behaviors, as evaluated by the PWS Anxiety and Distress Behaviors Questionnaire (PADQ; p=0.027). Neither dose demonstrated a statistically significant effect on the Children’s Yale-Brown Obsessive Compulsive Scale (CY-BOCS),” the company announced.
PWS has proved difficult to treat.
Millendo Therapeutics’ lead drug livoletide failed a pivotal trial for the treatment of PWS in April, sending shares plummeting. The drug didn’t improve hyperphagia or other eating-related behaviors.
Zafgen’s ZGN-1258, its second attempt to treat rare cases of obesity brought on by PWS, hit a brick wall after the FDA put a hold on the company’s PWS program in 2018, citing CV safety concerns. Zafgen was later used as a shell for Larimar to reverse its way to Wall Street.
Levo execs, though, say they like where they are at this point.
“This is a long-awaited step towards addressing the substantial needs of individuals living with PWS,” Levo CEO said in a prepared statement. “We are excited by these important results that were achieved after decades of interest in addressing the oxytocin deficiency in PWS. We are also pleased that our efforts to develop new tools for clinical evaluation of this rare, neurodevelopmental disorder have enhanced our understanding of the real-world impact LV-101 has on anxiety and distress behaviors.”