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 an approved drug.
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 pulmonary diseases, cystic fibrosis, central nervous system (CNS) disorders, as well as autoimmune and inflammatory diseases.
To learn more about Concert’s sponsored clinical trials, please visit clinicaltrials.gov.
Proprietary Drug Candidates
- CTP-656: CTP-656 is a novel potentiator that may enable once-daily dosing and was developed by applying deuterium chemistry to modify ivacaftor. Concert is initially developing CTP-656 as a potential treatment for cystic fibrosis as monotherapy in class III (gating) mutations (e.g. G551D) of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein which regulates components of sweat, mucus clearance and digestion. 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). A US-based Phase 2 trial in patients with cystic fibrosis is underway. On March 6, 2017, Concert announced it signed a definitive asset purchase agreement with Vertex Pharmaceuticals under which Vertex will acquire CTP-656.
- 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. The Company intends to conduct a Phase 2a clinical trial trial evaluating CTP-543 for the treatment of moderate-to-severe alopecia areata.
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.