Concert Pharmaceuticals’ CTP-692 efficacy failure is disappointing given its mild adverse effects

Following the news that Concert Pharmaceuticals is stopping development of its adjunctive treatment for schizophrenia, CTP-692 (D-serine), after it failed to meet its primary endpoint and other secondary endpoints in a Phase II trial;

Philippa Salter, Neurology Analyst at GlobalData, a leading data and analytics company, offers her view:

“A key unmet need in the schizophrenia market is for drugs with improved safety profiles. While there is evidence that D-serine can improve outcomes in patients with schizophrenia, it is associated with renal safety issues. CTP-692 was designed to improve upon the renal safety issues, so failure to demonstrate efficacy compared to placebo in the Phase II trial is especially disappointing given that the majority of adverse effects reported in the trial were mild in severity.

“Having discontinued CTP-692, Concert Pharmaceuticals will now be focused on its one remaining pipeline product, CTP-543, which is being investigated as a treatment for alopecia areata. CTP-543 is currently in Phase III development in the US, having shown promising results in Phase II trials, with a long-term extension study showing continued maintenance of hair growth in patients treated with CTP-543. GlobalData forecasts annual revenue of $228m by 2026 in the US for CTP-543.”

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