Theraly Fibrosis Granted US Orphan Drug Designation for TLY012 for Chronic Pancreatitis

GERMANTOWN, Md.--(BUSINESS WIRE)-- 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 has granted Orphan Drug Designation (ODD) to TLY012 for the treatment of chronic pancreatitis. The company plans to initiate a phase 1/2a clinical trial in 2020. 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.

Chronic pancreatitis is a progressive disorder that is characterized by pancreatic inflammation that causes fibrosis, which damages the pancreas and results in the loss of endocrine and exocrine function. Clinical manifestations of the disease include disabling abdominal pain and diabetes mellitus. 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. TLY012 binds upregulated death receptor 5 (DR5) on myofibroblasts (MFBs) and shuts down fibrotic pathways.

“MFBs, or activated fibroblasts, are believed to be the critical originators of fibrosis, a condition that affects a variety of tissues and leads to excessive scar tissue accumulation, organ damage and poor medical outcomes,” said Viktor Roschke, Ph.D., CEO 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 be a first-in-class treatment for a range of fibrotic diseases in addition to chronic pancreatitis. We look forward to initiating our Phase 1/2a trial in chronic pancreatitis 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 potentially may become a revolutionary therapy for fibrotic diseases, including chronic pancreatitis, systemic sclerosis and liver fibrosis/cirrhosis. Clinical trials evaluating TLY012 for chronic pancreatitis and other fibrotic diseases are expected to begin in 2020.

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 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 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


Source: Theraly Fibrosis

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