SSc is a rare, progressive autoimmune, rheumatic disease associated with major disability due to organ failure, reduced quality of life and shorter life expectancy.
Systemic sclerosis (SSc) is a rare, progressive autoimmune rheumatic disease of idiopathic origin that is characterized by microvascular damage, dysregulation of the immune system and generalized fibrosis in multiple organs.
SSc affects the skin, lungs, heart, gastrointestinal tract and kidneys, with the impairments of the internal organs the most disabling and life threatening manifestations of the disease. There are two primary forms of SSc: limited cutaneous SSc (lcSSc), which has more limited skin lesions and delayed onset of internal organ involvement, and diffuse cutaneous SSc (dcSSc), which has more extensive skin lesions and rapid onset of internal organ involvement2.
SSc is an orphan disease, with a prevalence estimated to be 154 per million in the US and Europe.
lcSSc and dcSSc have a 10-year survival rate of 80-90% and 60-80% respectively.
Women are 5x more likely to develop SSc; diagnosis typically occurs between ages 40 and 50.
Lanifibranor is currently in a Phase IIb clinical trial called FASST.
How is SSc diagnosed?
Women are more commonly affected by SSc; diagnosis typically occurs between ages 40 and 50. Often, the first symptom is “Raynaud’s phenomenon”, a cold- and stress-induced swelling of fingers and hands along with discoloration of the skin, or digital ulcers, sores on fingers or toes caused by poor blood circulation.
Lanifibranor – Our Solution
Lanifibranor is a small molecule activating all three subtypes of peroxisome proliferator-activated receptors (PPAR). PPARs are nuclear receptors involved in regulating fibrotic processes in the body. In several preclinical studies, lanifibranor has been shown to reduce fibrosis in the organs affected by SSc: the skin, lungs and kidneys with positive effects on lung and heart function.
Lanifibranor is currently being studied in a Phase IIb clinical trial, called FASST, for the treatment of patients with dcSSc. Enrolment has been completed for this trial and results are expected in early 2019.