Concert Pharmaceuticals Switches Focus After Schizophrenia Drug Falls Flat
02 Feb 2021
Breakthrough Therapy
Lexington, Mass.-based Concert Pharmaceuticals said its Phase II clinical trial for its adjunctive schizophrenia treatment, CTP-692, did not meet the primary endpoint or other secondary endpoints, causing the company to halt development of the drug. Concert is now switching focus to a Phase III trial evaluating a treatment for an autoimmune disorder that causes hair loss.
CTP-692 is a deuterated form of D-serine, which is an endogenous amino acid that also acts as a co-agonist of the NMDA receptor. The adjunctive candidate was developed to improve renal safety and brain exposure related to the amino acid.
“The body of evidence in the field supporting D-serine as an adjunctive treatment for schizophrenia was compelling and led us to advance CTP-692 into a Phase 2 proof of concept study,” said Roger Tung, Ph.D., president and chief executive officer of Concert Pharmaceuticals. “Unfortunately, we didn’t see the results we hoped for to support continuation of this program. Going forward, we will focus our internal resources on the advancement of CTP-543, which is currently in Phase 3 evaluation for the treatment of alopecia areata, and evaluation of additional pipeline candidates.”
Dr. Tung added that the company believes its Phase II study of CTP-692 was “well-designed” and “well-controlled,” and hopes the lack of benefit observed in the trial “will support future research to address the important need to improve the symptom domains of schizophrenia.”
The primary endpoint of the company’s Phase II trial was the change in the Positive and Negative Syndrome Scale (PANSS) total score at 12 weeks versus baseline. The study included 325 patients with schizophrenia, all of whom were taking a stable course of an antipsychotic treatment.
Patients were randomly assigned to receive either 1, 2 or 4-gram doses of CTP-692 or placebo, all administered once per day. Treatment with CTP-692 was not associated with a significant improvement versus placebo at any of dose levels for the primary or secondary endpoints. There were also no significant improvements with any treatment doses in terms of the negative or positive symptoms subscales of the PANSS scale.
Despite the lack of benefit with CTP-692, the treatment was well tolerated by most participants. Predominant adverse events were generally mild in severity and equally dispersed across all groups.
In the second half of September, Concert was hopeful that CTP-692 would prove effective for improving treatment of patients with schizophrenia. “CTP-692 offers a potential new mechanism to treat schizophrenia that, when combined with existing antipsychotic medications, may more broadly address disease symptoms than is currently possible,” according to a statement made by James V. Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals.
The company’s response to the disappointing findings included removing the candidate from its website and switching focus to its hair loss candidate, CTP-543. In November, the company announced it had launched the Phase III THRIVE-AA1 clinical trial to evaluate this treatment therapy in patients with alopecia areata, an autoimmune disease that causes hair loss. The randomized, double-blind trial will be conducted at sites across the U.S., Canada and Europe and will evaluate the therapy’s efficacy against placebo over a 24-week dosing period.
“Based on the results from our Phase II program, we believe CTP-543 has the potential to offer patients a best-in-class treatment for moderate to severe alopecia areata,” said Dr. Cassella. “We are fully committed to advancing CTP-543 with its Breakthrough Therapy designation in order to make a meaningful difference in the lives of individuals impacted by alopecia areata.”
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