Breakthrough in Endocrine Disease:
Pionneers of Novel Therapy in CAH and PCOS
Pionneers of Novel Therapy in CAH and PCOS
HBM Alpha Therapeutics (HBMAT), Inc., an innovative biotechnology company incubated by Harbour BioMed (HKEX: 02142), announced that it completed seed financing to advance its leading programs, novel antibody therapies to treat congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS), with the lead candidate currently in IND-enabling stage.
HBMAT was founded on the expertise of Harbour BioMed’s antibody discovery and development capabilities, and expertise in the molecular pathogenesis and treatment of endocrine disorders of HBMAT’s Scientific Founder Dr. Joseph Majzoub, Professor of Pediatrics and Medicine at Boston Children’s Hospital. HBMAT has licensed relevant technologies from Boston Children’s Hospital. Dr. Majzoub chairs the Scientific Advisory Board for HBMAT and is a shareholder of the company. At present, HBMAT’s Scientific Advisory Board comprises Dr. Majzoub, Dr. Frank Grosveld, Co-founder and CSO of Harbour Antibodies and the inventor of Harbour Mice®, and Dr. Ieuan Hughes, a paediatric endocrinologist and an emeritus professor of paediatrics at the University of Cambridge.
HBMAT’s programs aim to deliver precision therapies for endocrine disorders. The leading programs target a pathway for CAH and PCOS. The financing will be used to advance these programs into the clinical stage.
CAH is a rare-autosomal recessive genetic disease with a defective CYP21A2 gene. CAH patients have life-threatening deficiencies of glucocorticoid and mineralocorticoid, with limited treatment options and no new therapeutic modalities in over 50 years. While the current standard of care is associated with inevitable treatment-related side effects, specifically Cushing Syndrome and hyperandrogenism, HBMAT is developing HAT001 which focuses on treating hyperandrogenism without causing Cushing Syndrome. HAT001 would effectively cause reversible pharmacologic adrenalectomy, converting CAH patient treatment into that for primary adrenal insufficiency, which can be easily treated with much lower, physiological doses of glucocorticoid.
As a common and chronic endocrine disorder, PCOS affects over 10% women of reproductive age around the world and is the most common cause of anovulatory female infertility. HBMAT is also developing HAT002 for PCOS, targeting a pathway shared with CAH.
"I am very excited about this unique opportunity of developing antibody therapeutics for CAH, a rare genetic disease and PCOS impacting women’s health," said Jingsong Wang, Founder, Chairman & CEO of Harbour BioMed, and Chairman of BOD of HBMAT. "The company is thrilled to take these exciting programs to the next level and translate this therapeutic antibody with very impressive biological functions into clinical benefits for patients with significant unmet medical needs."
"I’ve worked closely with patient groups and tried to help those suffering from rare diseases over the past decades. HBMAT has already identified a clinical development candidate with excellent characteristics in preclinical functional activities including in vivo efficacy in various relevant animal models. I look forward to moving the development candidate into clinical trials and beyond.” said Dr. Philip Reilly, a renowned clinical geneticist and biotech entrepreneur, and board member of HBMAT.
CAH is a group of autosomal recessive diseases characterized by cortisol deficiency and androgen excess. It is one of the most common autosomal recessive disorders in humans. Current standard of care treatment of CAH patients with supraphysiologic glucocorticoids leads to Cushing syndrome, a collection of signs and symptoms due to prolonged exposure to glucocorticoids. The CAH market had sales of $46 million in 2016, with new therapeutics is expected to generate around $5 billion in 2035.
PCOS is one of the most common endocrine and metabolic disorders in premenopausal women. PCOS is frequently associated with abdominal adiposity, virilization, insulin resistance, obesity, metabolic disorders and cardiovascular risk factors. The economic burden of PCOS has been estimated at approximately $3.7 billion annually in 2020 when considering only the costs of the initial diagnosis and of reproductive endocrine morbidities, without considering the costs of pregnancy-related and long-term morbidities.
Harbour BioMed (HKEX: 02142) is a global biopharmaceutical company committed to the discovery, development, and commercialization of novel antibody therapeutics focusing on immunology and oncology. Harbour BioMed is building its robust portfolio and differentiated pipeline through internal R&D capability, collaborations with co-discovery and co-development partners, and select acquisitions.
Harbour BioMed’s proprietary antibody technology platforms Harbour Mice® generate fully human monoclonal antibodies in two heavy and two light chain (H2L2) format, as well as heavy chain only (HCAb) format. Building upon the HCAb antibodies, the HCAb-based immune cell engagers (HBICE®) can deliver tumor-killing effects unachievable by traditional combination therapies. Integrating Harbour Mice®, HBICE® with single B cell cloning platform, the antibody discovery engine is unique and efficient for the development of next-generation therapeutic antibodies. For more information, please visit www.harbourbiomed.com.
HBM Alpha Therapeutics is a company presently developing antibody therapeutics aimed at the rare genetic disease congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS). Its therapeutics could also be indicated for other endocrinological diseases related to the dysregulation and function of the hypothalamus-pituitary-adrenal (HPA) axis.
HBMAT was founded and incorporated in Delaware, USA in 2019. It is a joint venture between Harbour BioMed and Boston Children’s Hospital, an affiliate of Harvard Medical School. For more information, please visit www.hbmalphatx.com or contact BD@harbourbiomed.com.
Leadership & Advisory Team
Jingsong Wang, M.D., Ph.D., Chairman of BOD
Founder, Chairman & CEO of Harbour BioMed
Former Head of China R&D & Head of Translational Medicine, Asia Pacific, Sanofi
Joseph Majzoub, M.D., Scientific Founder
Professor of Pediatrics, Professor of Medicine, Harvard Medical School
Chief Emeritus, Division of Endocrinology, Vice Chair for Research, Department of Pediatrics, Boston Children's Hospital
Philip Reilly, M.D., J.D., Board Member
Advisory Board to Boston University School of Public Health
Former Overseer Weill Cornell Medical College
Author of the book “Orphan: The Quest to Save Children with Rare Genetic Disorders”
Michael Rosenblatt, M.D., Board Member
Member of the Harvard Medical School Board of Fellows
Member of the research advisory committees of the Massachusetts General Hospital,
Brigham and Women's Hospital and Boston Children's Hospital
Former Chief Medical Officer at Flagship Pioneering
Former Chief Medical Officer at Merck
Robert Kamen, Ph.D., Board Member
Advisory Partner at Third Rock Ventures
Co-founder and former Chairman of BioAssets
Former Director of Neon Therapeutics and Harbour Antibodies
Former President and Unit Head of Abbott Bioresearch Centre, Former SVP at Genetics Institute, Inc.