Despite FDA Breakthrough Designation, Ulotaront Shows No Significant Efficacy Over Placebo
In a setback for the treatment of schizophrenia and acute psychosis, ulotaront did not meet its primary endpoint in two recent phase 3 clinical trials. Developed by Sumitomo Pharma Co. and Otsuka Pharmaceutical Co., the drug had previously received Breakthrough Therapy Designation from the FDA. The trials aimed to evaluate the drug’s efficacy, safety, and tolerability over a six-week period.
HCN Medical Memo
The failure of ulotaront to meet its primary endpoint underscores the ongoing challenges in developing effective treatments for schizophrenia and acute psychosis. This outcome serves as a reminder that even drugs with Breakthrough Designation from the FDA are not guaranteed to succeed in later-stage trials. The need for innovative approaches remains urgent in this therapeutic area.
- The DIAMOND 1 and DIAMOND 2 trials involved a total of 899 adults with schizophrenia and acute psychosis.
- Neither trial showed ulotaront to be superior to placebo in reducing the Positive and Negative Syndrome Scale (PANSS) total score at week 6.
- Hiroshi Nomura, president and CEO of Sumitomo Pharma, noted that a large placebo effect may have masked the drug’s therapeutic impact.
- Healthcare professionals express concern over the lack of effective new treatments for schizophrenia, emphasizing the need for further research.
“We continue to work closely with Otsuka and analyze the data to determine our next steps and plan to discuss with the FDA how to proceed based on these results.”
– Hiroshi Nomura, President and CEO of Sumitomo Pharma
- Ulotaront is a trace amine-associated receptor 1 (TAAR1) agonist with 5-HT1A agonist activity.
- The drug was generally safe and well-tolerated in both trials.
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