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 diseases and central nervous system (CNS) disorders.

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

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. Deuterium modification of ruxolitinib was found to alter its human pharmacokinetics in ways which may enhance its use as a treatment for alopecia areata.  In November 2018, we announced positive interim topline results from the first two cohorts of the CTP-543 Phase 2 trial in patients with moderate-to-severe alopecia areata. At 24 weeks, patients treated with an 8 mg twice-daily dose of CTP-543 met the primary efficacy endpoint vs. placebo. At this dose, significant differences from placebo were observed beginning at Week 12.  Regrowth of hair did not appear to plateau at Week 24.  The Phase 2 clinical trial evaluating CTP-543 for the treatment of moderate-to-severe alopecia areata is ongoing.
  • 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 safety assessments in the single- and multiple-ascending dose Phase 1 trials in healthy volunteers showed that the drug was well tolerated over the dose ranges tested, which include the doses expected to be evaluated in Phase 2 testing. Importantly, key blood and urine markers of kidney function did not indicate any signs of renal impairment. These data are consistent with preclinical findings with CTP-692 indicating an improved renal safety profile compared to D-serine which is known to produce renal toxicity in rats. The Company intends to advance the program into a Phase 2 trial in the fourth quarter of 2019.

Partnered Drug Candidates

  • Avanir/Otsuka: 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 under a collaboration with Avanir. 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.
  • Jazz Pharmaceuticals: Jazz Pharmaceuticals’ current once-nightly development efforts are focused on lower sodium compounds. The collaboration with Jazz Pharmaceutcals provides for the evaluation of deuterium as an option for a once-nightly sodium oxybate product.
  • Celgene: 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.