Concert Pharmaceuticals

Product Pipeline

Our innovative approach to drug development has resulted in multiple drug candidates that are progressing in clinical development.  For each drug candidate, application of our DCE Platform® resulted in a novel, differentiated, and potentially superior product, building on a product concept that starts with previously studied compounds, including approved drugs.

Through our wholly owned development candidates and our strategic collaborators, our DCE Platform-derived pipeline of product candidates includes novel drugs to treat a range of disease areas including autoimmune and inflammatory diseases and central nervous system (CNS) disorders

To learn more about Concert’s sponsored clinical trials, please visit clinicaltrials.gov.

Concert Pharmaceuticals Pipeline

Proprietary Drug Candidates

  • CTP-543: CTP-543 is an oral selective inhibitor of certain Janus kinases, known as JAK1 and JAK2, that we are developing for the treatment of alopecia areata, an autoimmune disease that results in partial or complete loss of hair on the scalp and/or body. CTP-543 was discovered by applying our deuterium chemistry technology to modify ruxolitinib, which is commercially available under the name Jakafi® in the U.S. for the treatment of myelofibrosis and polycythemia vera. Ruxolitinib has been used in an academic investigator-sponsored clinical trial and has been shown to promote hair growth in individuals with alopecia areata.  We conducted preclinical studies demonstrating that CTP-543 retains ruxolitinib’s JAK1and JAK2 inhibition profile and showing improved metabolic stability relative to ruxolitinib.  A Phase 2a clinical trial evaluating CTP-543 for the treatment of moderate-to-severe alopecia areata is underway.
  • CTP-692: CTP-692 is a novel drug for adjunctive treatment of schizophrenia, a devastating, chronic illness with significant unmet need. CTP-692 is a deuterated form of D-serine, an endogenous, required co-agonist of the N-methyl-D-aspartate (NMDA) receptor. NMDA receptor hypofunction is believed to contribute to the pathophysiology of schizophrenia and enhancement of D-serine levels is believed to benefit individuals with schizophrenia. CTP-692 is expected to have similar pharmacology to D-serine with the potential for an improved safety profile and improved clinical outcomes in the treatment of schizophrenia and other neuropsychiatric diseases. CTP-692 will be developed as an adjunctive therapy administered in addition to standard antipsychotic medicines to improve both positive and negative symptoms as well as cognitive function in patients with schizophrenia. The Company intends to complete preclinical evaluation and advance CTP-692 into clinical development by year-end 2018.

Partnered Drug Candidates

  • AVP-786: AVP-786 is a combination of a deuterium-substituted dextromethorphan analog and a low dose of quinidine being investigated for the treatment of neurologic and psychiatric disorders. Avanir is conducting several Phase 2 and Phase 3 clinical trials to evaluate AVP-786 for the treatment of neurologic and psychiatric disorders, the most advanced of which are Phase 3 clinical trials for the treatment of agitation associated with Alzheimer’s disease.
  • JZP-386: JZP-386 is a product candidate containing deuterated sodium oxybate for potential use in patients with narcolepsy, which is in development in collaboration with Jazz Pharmaceuticals. Sodium oxybate is the active ingredient in the marketed drug Xyrem. Jazz Pharmaceuticals is exploring formulation options designed to leverage the positive effects observed with JZP-386 in the Phase 1 trials.
  • CTP-730: CTP-730 is a deuterated apremilast analog for the potential treatment of inflammatory disease, which is in development under a multi-product collaboration with Celgene. Phase 1 testing of CTP-730 is complete.

Asset Purchase Agreement

  • CTP-656: CTP-656 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination regimens of CFTR modulators that treat the underlying cause of cystic fibrosis. In a Phase 1 cross-over comparison of CTP-656 and Kalydeco, CTP-656 demonstrated a superior pharmacokinetic profile compared to Kalydeco including a reduced rate of clearance, longer half-life, substantially increased exposure and greater plasma levels at 24 hours. CTP-656 has been granted orphan drug designation by the US Food and Drug Administration (FDA).  In July 2017, Vertex acquired worldwide development and commercialization rights to CTP-656, now known as VX-561, and other assets related to the treatment of cystic fibrosis.