Intra-Cellular Therapies have announced inconclusive results from the second Phase 3 trial of ITI-007 (Study ‘302), a first-in-class investigational medicine for the treatment of schizophrenia.

The study included a total of 696 patients who were randomized (1:1:1:1) to receive ITI-007 60mg or 20mg, risperidone 4mg as the active control, or placebo once daily for 6 weeks.

Neither of the two doses (60mg or 20mg) met the trial’s pre-specified primary efficacy measure, which was change from baseline vs. placebo at the study endpoint, measured by the centrally-rated PANSS total score. 

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The results are contrary to two previous studies (Study ‘302 and Study ‘301) which found positive results for ITI-007 in 305 and 450 patients, respectively. The latest trial did however find statistically better safety and tolerability parameters than risperidone and exhibited a safety profile similar to placebo. 

The company believes ITI-007 did not separate from placebo in the endpoint of the study in part due to an unusually high placebo response at certain sites, disproportionately affecting the trial results. The high placebo response was –15.1 point change from baseline on PANSS score, compared to a –7.4 and –10.3 change in the two previous studies.

“It is not uncommon in the field of psychiatry for studies to be challenged by high placebo response and there has been great variability in the effects observed from one study to the next,” said Christoph Corell, MD, Professor of Psychiatry at Hofstra Northwell School of Medicine.

The company believes the treatment has the capability to advance treatment for patients with schizophrenia and remains committed to developing ITI-007 for this population.

ITI-007 combines potent serotonin 5-HT2A receptor antagonism, dopamine receptor phosphoprotein modulation (DPPM), glutamatergic modulation, and serotonin reuptake inhibition into a single drug candidate for the treatment of acute and residual schizophrenia. 

A meeting with the Food and Drug Administration’s Division of Psychiatry Products has been requested by Intra-Cellular to discuss ITI-007’s regulatory path.

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