Jazz drops PTSD drug over phase 2 trial failure

Jazz Pharmaceuticals is planning to drop its post-traumatic stress disorder (PTSD) therapy after the small molecule selective fatty acid amide hydrolase (FAAH) inhibitor failed to beat placebo in a phase 2 study.

The biotech had been testing the drug, dubbed JZP150, in a trial of 282 patients diagnosed with PTSD aged 18 to 70 years who were randomized to receive either 0.3 mg or 4 mg doses of the therapy or placebo. At 12 weeks, JZP150 was unable to show a statistically significant decrease in the severity of PTSD symptoms over placebo, missing the trial’s primary endpoint.

No new safety signals were observed, the biotech noted. The most common treatment-emergent adverse events were headache, nausea and urinary tract infection, although the company pointed out that these also occurred in placebo-treated participants.

"We plan to fully evaluate these data; however, based on topline results we do not anticipate moving forward with additional JZP150 development in PTSD,” Rob Iannone, M.D., Jazz’s global head of R&D, said in the post-market release Thursday.

“We recognize the significant unmet need for PTSD patients and plan to share the findings from this trial with the medical community at a future date," Iannone added.

Jazz had high hopes for JZP150, having previously pointed out that it had a chance of becoming the first new drug approved in 20 years for patients with the disorder. PTSD has been in the headlines recently as the Multidisciplinary Association for Psychedelic Studies' submitted a new drug submission for MDMA to treat the condition, marking the first time ever that the agency will consider approving a psychedelic-assisted therapy.

Jazz’s latest clinical setback comes less than a month after the biotech paused a phase 1 trial of its sleep disorder med JZP441 in the wake of reports of “visual disturbances” and cardiovascular effects. Executives wouldn’t go as far as to fully terminate the orexin-2 receptor agonist, which was licensed from Sumitomo Pharma, instead saying at the time that they were looking to further investigate the side effects and how to potentially mitigate them. 

Discussing those JZP441 results on a Nov. 28 conference call, executives made sure to swiftly transition from bad news to good, announcing that Jazz planned to initiate rolling submission of its bispecific antibody zanidatamab before the end of the year. The company is seeking accelerated approval as a second-line treatment for patients with biliary tract cancer, with the application set to be completed by the first half of 2024.