CTP-543
An investigational JAK 1/2 inhibitor for alopecia areata
Concert Pharmaceuticals is developing CTP-543, an oral inhibitor of Janus kinases JAK1 and JAK2, as a potential treatment for alopecia areata.
Janus kinases (JAKs) are a group of enzymes that play an important role in the immune system. Research has shown that inhibiting JAKs can be beneficial in treating certain immune-mediated diseases. Concert was one of the first drug developers to leverage recent research about JAK inhibitors to develop CTP 543 as a potential new treatment to address the dysregulated autoimmune function in alopecia areata. CTP-543 is a deuterium-modified form of the JAK 1/2 inhibitor ruxolitinib.
Alopecia areata is a serious autoimmune disease with no approved treatment
Alopecia areata is an autoimmune disease in which the body’s immune system mistakenly attacks a person’s hair follicles, causing patchy or complete loss of hair on the scalp and body as well as other significant physical and psychological symptoms. Alopecia areata affects approximately 1 million Americans at any given time and can be debilitating to their physical and psychological health.
A publication in the Journal of Investigative Dermatology highlights the impacts of alopecia areata. The journal article, entitled “Burden of Illness in Alopecia Areata: A Cross-Sectional Online Survey Study,” is available online.
The FDA selected alopecia areata as a disease areas that it focused on under its Patient-Focused Drug Development Initiative (PFDDI) in 2016-2017, with the goal to bring patient perspectives into early-stage drug development. Currently, no drugs are approved by the FDA for the treatment of alopecia areata.
CTP-543 Clinical Program and Progress
Concert has an ongoing robust clinical program evaluating CTP-543 as an oral medicine for the treatment of alopecia areata. The FDA has granted Breakthrough Therapy and Fast Track designations for CTP-543. In November 2020, Concert began enrolling patients in the THRIVE-AA1 trial, a Phase 3 trial to evaluate the efficacy and safety of CTP-543 in adult patients with moderate to severe alopecia areata. Concert also completed a Phase 2 study evaluating a range of doses in different cohorts of patients with alopecia areata, which was designed to identify the optimal doses for efficacy and safety for further evaluation in the THRIVE-AA1 trial.
