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 700,000 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.
