Jazz's midphase essential tremor test fails to meet endpoint

Jazz Pharmaceuticals has faced a setback with their phase 2b essential tremor trial, which failed to meet its primary endpoint. However, the company observed some numerical improvements and is awaiting additional data from another study before making a final decision on the future of the program.

The trial tested three doses of suvecaltamide, a T-type calcium channel modulator, against a placebo in 420 patients suffering from essential tremor, the most prevalent pathological movement disorder. Despite its prevalence, treating essential tremor has proven difficult, with physicians often relying on older medications like the beta-blocker propranolol.

Jazz Pharmaceuticals ventured into this market by acquiring Cavion for $52.5 million in 2019 and initiated the phase 2b study in 2021. However, the recent results have cast doubt on the potential return on this investment. The study revealed that suvecaltamide did not show a statistically significant improvement over the placebo on the TETRAS essential tremor assessment scale, thus failing to meet the primary endpoint. Nevertheless, Jazz highlighted trends favoring the highest dose, 30 mg, over the placebo on both primary and key secondary endpoints as a positive outcome. The company also noted that the placebo group performed better than anticipated.

In 2022, Jazz started a phase 2 trial of suvecaltamide in patients with Parkinson’s disease tremor. The results of this study, expected in the first quarter of next year, could determine the future of suvecaltamide. Rob Iannone, M.D., global head of R&D at Jazz, stated that they are still assessing the essential tremor data and will decide on the next steps after reviewing the Parkinson’s trial results.

Kelvin Tan, Chief Medical Officer at Jazz, discussed their approach to evaluating the data at a recent Goldman Sachs event. Tan emphasized the importance of considering factors beyond statistical significance. He stated, “We’ll look at not just statistical significance, but we’ll also look at all of the other endpoints that we’ve collected in that trial to understand what is it that we need to determine makes a clinically relevant impact, and then we’ll take that into our decisions around what, hopefully, the phase 3 trial then looks like.”

Despite the setback, Jazz had high hopes for the phase 2b study, identifying it as potentially pivotal. The primary endpoint was chosen based on its relevance to patients’ daily living activities, such as washing, dressing, and feeding. However, the phase 2b trial results suggested that suvecaltamide did not provide better outcomes than the placebo in improving these patient-centric measures.