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.

NEWS: Presentation of CTP-543 THRIVE-AA1 Phase 3 Data in Alopecia Areata During Late Breaking Session at EADV Congress

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 up to approximately 1.5 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 area 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, there are limited treatment options for alopecia areata.

CTP-543 Clinical Program and Progress

Concert has a 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. Concert is evaluating the efficacy and safety of CTP-543 in adult patients with moderate to severe alopecia areata in its THRIVE-AA Phase 3 clinical program. Concert reported positive topline data from the THRIVE-AA1 and THRIVE-AA2 trials. The primary efficacy endpoint for both studies was the percentage of patients with an absolute Severity of Alopecia Tool (SALT) score of 20 or less after 24 weeks of treatment, which was met with statistical significance by both the 8 mg twice-daily and 12 mg twice-daily doses relative to placebo. Data from the THRIVE-AA1 and THRIVE-AA2 are intended to form the basis of a New Drug Application (NDA) planned to be submitted to the U.S. Food and Drug Administration (FDA) in the first half of 2023.

CTP-543 in the Clinic

PHASE 3: THRIVE-AA
Concert Pharmaceuticals Reports Positive CTP-543 Phase 3 Results in Alopecia Areata

THRIVE-AA2 study meets primary endpoint for scalp hair regrowth.

Concert Pharmaceuticals Reports Positive CTP-543 Phase 3 Results in Alopecia Areata

THRIVE-AA1 study meets primary endpoint for scalp hair regrowth.

Concert Pharmaceuticals Initiates THRIVE-AA2 Phase 3 Clinical Trial Evaluating CTP-543 for Alopecia Areata

Study to evaluate the safety and efficacy of CTP-543 in adults who have 50% or greater scalp hair loss.

Concert Pharmaceuticals Initiates THRIVE-AA1 Phase 3 Clinical Trial Evaluating CTP-543 for Alopecia Areata

Study to evaluate the safety and efficacy of CTP-543 in adults who have 50% or greater scalp hair loss.

PHASE 2 STUDY
OPEN-LABEL TRIAL
Long-Term, Open-Label Extension Study

Ongoing enrollment of patients from CTP-543 trials.

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

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