CAMBRIDGE, Mass. & ROTTERDAM, The Netherlands & SUZHOU, China--(BUSINESS WIRE)--Harbour BioMed (HBM) announced today that it has received IND approval from the National Medical Products Administration (NMPA) in China to begin a seamless Phase 2/3 clinical trial to evaluate the safety and efficacy of its therapeutic antibody HBM9161 to treat adult immune thrombocytopenia (ITP). The approval provides an accelerated development pathway for HBM by allowing the Phase 3 study to proceed directly following Phase 2.
HBM9161 is a fully human monoclonal antibody targeting the neonatal Fc receptor (FcRn) that accelerates the degradation of autoantibodies that drive ITP as well as other autoimmune disorders. Based on its novel mechanism and high unmet need in China, HBM plans to begin clinical trials this year in several other autoimmune diseases, including myasthenia gravis, Grave’s ophthalmopathy, and neuromyelitis optica spectrum disorder.
“This Phase 2/3 clinical trial, and our broader clinical program for HBM9161, builds on the significant potential it has demonstrated in preclinical studies to treat autoimmune diseases through a novel mechanism and initial clinical studies, including our Phase 1 trial, demonstrating it has a good safety profile and potent IgG reduction capability,” said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. “ITP presents a high disease burden for patients, not only through its clinical symptoms, but often by leading to fatigue anxiety, fear, depression and isolation. We are pleased that the NMPA supported this seamless trial design to speed its development and address this high unmet need in China.” He added that HBM expects to follow this trial with clinical studies of HBM9161 in other severe autoimmune diseases, establishing a “pipeline in a product.”
“ITP is a chronic heterogeneous disease that has significant effects on patients’ lives and for which treatment options are limited. There has been no breakthrough treatment for this condition so far. New treatments based on innovative mechanisms, like reducing IgG by blocking FcRn, I believe may offer patients who are non-responsive and/or refractory to current therapies a new and promising therapeutic solution,” said Dr. Renchi Yang, Chief Physician & Professor at the Institute of Hematology and Blood Diseases, Chinese Academy of Medical Sciences and Peking Union Medical College, and the trial’s principal investigator. Both the Phase 2 and Phase 3 studies are double-blind placebo-controlled trials.
HBM9161 is an antibody targeting the neonatal Fc receptor (FcRn). FcRn expression is upregulated by proinflammatory cytokines like TNF-α and extends the half-life of IgGs and serum albumin by reducing lysosomal degradation in endothelial cells and bone-marrow derived cells. Blocking the FcRn-IgG interaction accelerates the degradation of autoantibodies and alleviates the flare-up in wide array of pathogenic IgG-mediated autoimmune diseases, including myasthenia gravis, Grave’s ophthalmopathy, neuromyelitis optica spectrum disorder and immune thrombocytopenia amongst others.
HBM licensed HBM9161 from HanAll Biopharma and has the right to develop, manufacture and commercialize in Greater China (including Hong Kong, Macau and Taiwan).
About Adult Primary Immune Thrombocytopenia
Adult ITP is a rare autoimmune bleeding disorder usually resulting from development of an autoantibody directed against a structural platelet antigen. This results in abnormally low platelet levels leading to bruising, excessive bleeding (e.g. gums, nosebleeds, or prolonged menstrual bleeding) and spontaneous bleeding events, that exact a significant toll on patients’ quality of life. There is also an increased risk of potentially fatal internal organ bleeding events. The disease is heterogeneous, with some patients displaying no symptoms or showing symptoms when the platelet count is very low.
There is no cure for ITP; current treatment options are also limited due to inadequate efficacy and significant side effects. Current first line treatment includes corticosteroid drugs (e.g., prednisone, dexamethasone, methylprednisolone). The second line consists of thrombopoietin receptor agonists (TPO-RAs) that stimulate the body’s production of platelets by megakaryocytes in the bone marrow, which release proplatelets that mature into platelets. Another option is anti-CD20 which reduces the overall IgG antibody production. Splenectomy is an option if drug therapy fails.
The average global prevalence of ITP, including in China1, is close to 10 cases per 100,000. The incidence of ITP increases with age and is more common over the age of 60, of which there are 2.6 cases among women for every case involving a male2. The incidence of ITP among adults in the United States and China is estimated to be 3.3 and 5-10 per 100,000 adults/year, respectively.
About Harbour BioMed
Harbour BioMed is a global, clinical stage biopharmaceutical company developing innovative therapeutics in the fields of immuno-oncology and inflammatory diseases. The company is building its proprietary pipeline through internal R&D programs, collaborations with co-discovery and co-development partners and select acquisitions. The company's internal discovery programs are centered around its two patented transgenic mouse platforms (Harbour Mice®) for generating both fully human monoclonal antibodies and heavy chain only antibodies (HCAb) based next generation antibodies. Harbour BioMed also licenses the platforms to companies and academic institutions. The company has operations in Cambridge, Massachusetts; Rotterdam, The Netherlands; and Suzhou & Shanghai, China. For more information, visit: www.harbourbiomed.com.
1. Consensus of Chinese experts on diagnosis and treatment of adult primary immune thrombocytopena (Version 2016)
2. National Organization for Rare Disorders Report (accessed 1/21/2020), USA