Abcuro Granted Orphan Drug Designation for ABC008 for Inclusion Body Myositis

  • Inclusion body myositis (IBM)is an autoimmune condition in which cytotoxic T cells chronically attack muscle cells, leading to progressive weakness and severe disability
  • Abcuro’s lead program, ABC008, is an anti-KLRG1 antibody capable of depleting highly differentiated cytotoxic T cells that are pathogenic in IBM

Newton, MA, June 9, 2020 – Abcuro, Inc., a clinical-stage biotechnology company developing therapies for autoimmune diseases and cancer through precise modulation of subpopulations of T and NK cells, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABC008 for the treatment of inclusion body myositis (IBM). ABC008 is an anti-KLRG1 antibody capable of selectively depleting cytotoxic T cells present in muscle tissue that are pathogenic in IBM, thus addressing the source of immune attack.

“Receiving Orphan Drug Designation for ABC008 in IBM is an important milestone for Abcuro and adds momentum to the development of this potentially first-in-class therapeutic,” said Dick Polisson, M.D., Chief Medical Officer of Abcuro. “We are now advancing ABC008 towards a first-in-human proof of mechanism and safety trial. We believe that ABC008 will have applicability across a number of autoimmune diseases with high unmet need.”

IBM is the most prevalent acquired myopathy in adults older than age fifty, impacting approximately 20,000 patients in the United States with an estimated prevalence in the US of 71 cases per million adults. The disease becomes apparent during adulthood and is characterized by progressive weakness and atrophy of the muscles especially those of the arms and the legs. IBM can progress to cause severe disability.

IBM is an autoimmune condition in which cytotoxic T cells attack muscle, leading to degeneration of the tissue.[i]Muscle tissue from patients with IBM show the presence of highly differentiated cytotoxic T cells that express KLRG1. The extent to which KLRG1 may be an important target in other autoimmune diseases is being actively explored by Abcuro.

“Patients suffering from IBM have no effective drug treatments currently available to them,” said Steven A. Greenberg, M.D., M.S., co-founder of Abcuro and Chair of the Medical Advisory Board who also serves as a neuromuscular disease specialist at Brigham and Women’s Hospital and Professor of Neurology at Harvard Medical School. “Recognition of IBM as an autoimmune disease has been an essential step towards development of an effective treatment. By specifically targeting highly differentiated cytotoxic T cells that express KLRG1, Abcuro aims to improve outcomes for patients with IBM.”

The FDA grants orphan status to drugs intended to treat rare disorders that affect fewer than 200,000 people in the U.S. The designation provides certain benefits to the drug developer, including seven years of market exclusivity upon FDA approval, prescription drug user fee waivers and tax credits for qualified clinical trials.

About Abcuro

Abcuro is a clinical stage biotechnology company that is developing first-in-class immunotherapies for autoimmune diseases and cancer through precise modulation of T and NK cells that express KLRG1 (Killer Cell Lectin Like Receptor G1). In certain autoimmune diseases, KLRG1-expressing T cells are the source of chronic tissue damage. In oncology, tumor cells that express E- or N- cadherin inhibit anti-tumor activity of T and NK cells via their KLRG1 receptor, therefore acting as an immune checkpoint inhibitory receptor. KLRG1 was identified as a compelling target with relevance to disease biology through Abcuro’s powerful use of patient clinical data and patient tissue transcriptome analyses derived from discrete, pathological immune cell subpopulations. For more information, visit


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