Theraly Fibrosis Granted US Orphan Drug Designation for TLY012 for Systemic Sclerosis


Theraly Fibrosis Granted US Orphan Drug Designation for TLY012 for Systemic Sclerosis


TLY012 is shown to reverse skin fibrosis in preclinical models of scleroderma



Germantown, Maryland, May 27, 2020 – Theraly Fibrosis, a preclinical-stage biotechnology company focused on development and commercialization of drugs for various fibrotic diseases today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TLY012 for the treatment of systemic sclerosis. The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. In 2019, the FDA granted ODD to TLY012 for the treatment of chronic pancreatitis.

Systemic sclerosis, also known as systemic scleroderma, is a chronic autoimmune disorder characterized by degenerative changes and scarring in the skin, joints, and internal organs and by blood vessel abnormalities. Currently, there are no drugs available to significantly influence the natural course of the disease.

TLY012 is a proprietary version of the recombinant human TRAIL protein with enhanced potency, stability and extended half-life in the bloodstream. The molecule binds upregulated death receptor 5 (DR5) on myofibroblasts (MFBs) and shuts down fibrotic pathways. A study recently published in Nature Communications showed that TLY012 can reverse established skin fibrosis to near normal skin in mice with scleroderma.

“Targeting the TRAIL pathway with TLY012 may represent a promising anti-fibrotic strategy to arrest scleroderma in humans,” said Viktor Roschke, Ph.D., CSO and head of R&D at Theraly.  “Because TLY012 targets and blocks the formation of MFBs regardless of tissue location, it has the potential to not only be a first-in-class treatment for systemic scleroderma, but also for other fibrotic diseases.  We look forward to initiating our clinical trial in systemic scleroderma potentially within the next year and advancing this important drug candidate for patients with significant unmet need.”

Treatment with TLY012 leads to reversal of established fibrosis in preclinical models of the disease in pancreas, skin and liver and has the potential to become a revolutionary therapy for fibrotic diseases, including systemic sclerosis, chronic pancreatitis, and liver fibrosis/cirrhosis.

About Theraly Fibrosis

Theraly is a preclinical-stage company whose mission is to develop innovative treatments for various forms of fibrotic disease including chronic pancreatitis, systemic sclerosis, liver fibrosis and NASH.  The company’s lead candidate, TLY012, selectively targets myofibroblasts, cells believed to be the critical originators of fibrosis, a condition that affects a variety of tissues. Theraly Fibrosis is a subsidiary of D&D Pharmatech, a clinical-stage global biotech company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by world-class, top-tier medical research faculty and biotechnology veterans. This corporate structure creates a unique opportunity to accelerate translation of cutting-edge research into lifesaving therapeutic products for patients. For more information about D&D Pharmatech and Theraly Fibrosis, please visit



Christine Quern

CBQ Communications