BridgeBio Pharma was founded in 2015 to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers.
BridgeBio's first R&D Day is a virtual event that will feature presentations from BridgeBio founder and CEO, Neil Kumar, Ph.D., senior scientists and physicians leading BridgeBio's drug discovery and development programs, as well as top academic physicians and scientists from outside the company.
The event will focus on BridgeBio’s drug engineering platform, its targeted oncology portfolio, and four highlighted programs with clinical data anticipated within the next 12-24 months – acoramidis (AG10, TTR stabilizer) for transthyretin amyloidosis, low-dose infigratinib (FGFR inhibitor) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia, and encaleret (calcium sensing receptor antagonist) for autosomal dominant hypocalcemia type 1.Â
BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.
Presenter: Dr. Neil Kumar, Ph.D., BridgeBio CEO and Founder
BridgeBio Pharma CEO and founder Neil Kumar, Ph.D., opens R&D Day with insights on the company's corporate progress and outlook for the future, as well as BridgeBio’s drug engineering platform and four highlighted programs: achondroplasia, ATTR, CAH and ADH1.
Introduction: Dr. Susan Moran, M.D., M.S.C.E.
Presenter: Dr. Ravi Savarirayan, M.D., Ph.D.
Low-dose infigratinib (FGFRi) is a potentially best-in-class approach to treating achondroplasia at its genetic source. Achondroplasia is the most common cause of disproportionate short stature and affects 55K people in the US and EU. Achondroplasia is caused by an alteration in the FGFR3 gene, which causes the FGFR3 protein to be overly active. This interferes with skeletal development and can lead to disturbances in bone growth. Infigratinib is an oral investigational medicine that is designed to reduce the activity of FGFR3.
Introduction: Dr. Jonathan Fox, M.D., Ph.D.
Presenter: Professor Julian D. Gillmore, M.D., Ph.D.
Acoramidis (formerly AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, to halt at its outset the series of molecular events that give rise to TTR amyloidosis, or ATTR. Data from pre-clinical and clinical studies completed to date suggest acoramidis, which is in a Phase 3 clinical trial, has a potential best-in-class clinical profile. There are an estimated 400K+ patients with ATTR in the US and Europe.
Introduction: Dr. Eric David, M.D., J.D.
Presenter: Dr. Kyriakie Sarafoglou, M.D.
BridgeBio is developing BBP-631, an AAV5 gene therapy candidate for congenital adrenal hyperplasia, one of the largest potential gene therapy markets with more than 75K patients in the US and Europe. Designed to deliver functional copies of CYP21A2 gene, BridgeBio's approach is the only one aimed at restoring native physiology to drive endogenous cortisol production. IND-enabling studies are proceeding and the company is on track to initiate a first in human Phase 1/2 study and report initial data in 2021.
Introduction: Dr. Jonathan Fox, M.D., Ph.D.
Presenter: Dr. Michael Collins, M.D.
Encaleret is an investigational orally-administered CaSR antagonist for Autosomal Dominant Hypocalcemia Type 1 (ADH1), a condition caused by gain of function variants in the CaSR gene estimated to be carried by 12k individuals in the US. Encaleret has been studied in >1,200 healthy volunteers and osteoporosis patients and has shown clear ability to modify ADH1 disease drivers, which encouraged BridgeBio's investigation of the compound in ADH1 patients. BridgeBio is on track to initiate a planned Phase 2 study in 2020, with potential proof-of-concept data in 2021. If approved, encaleret would be the first approved therapy for ADH1.
Introduction: Dr. Eli Wallace, Ph.D.
Presenter: Frank McCormick, Ph.D.
BridgeBio’s world class oncology research team will introduce the collaborations and industry leading targeted oncology research capabilities that enable BridgeBio to prosecute multiple agents with first in class and/or best in class potential. They will share a deeper dive into the holy grail of targeted oncology: KRAS.
Dr. Neil Kumar, Ph.D., is the CEO and founder of BridgeBio Pharma, Inc., a company focused on the development of novel therapies for genetic diseases. Before founding BridgeBio in 2015, Dr. Kumar was a principal at Third Rock Ventures and an associate principal at McKinsey & Company. Dr. Kumar holds B.S. and M.S. degrees in chemical engineering from Stanford University. He received his Ph.D. in chemical engineering from the Massachusetts Institute of Technology.
Dr. Susan Moran, M.D., M.S.C.E., is the chief medical officer at BridgeBio Pharma affiliate company QED Therapeutics. Dr. Moran has over 20 years of industry and academic experience, successfully leading clinical trials from Phase 1 to Phase 3, as well as marketing applications for various investigational products including the successful approval of Nerlynx. Before joining QED, she was VP and Head of Clinical Development for Puma Biotechnology and previously held senior positions at Millennium and Genzyme.
Professor, Group Leader of Skeletal Biology and Disease at Murdoch Children’s Research Institute in Australia and Head of Clinical Genetics Services at the Victorian Clinical Genetic Services
Dr. Ravi Savarirayan is a clinical geneticist with a primary clinical and research focus on the inherited disorders of the skeleton causing short stature, arthritis and osteoporosis.
He is the Foundation Director of the Southern Cross Bone Dysplasia Centre and immediate past-President of the International Skeletal Dysplasia Society (ISDS) and was the first Australian to hold this post. He has won numerous academic awards including the Fulbright Professional Scholarship for Australia.
Dr. Jonathan Fox, M.D., Ph.D., FACC, has served as President and Chief Medical Officer of Eidos Therapeutics and as the therapeutic area lead for cardiovascular and renal diseases at BridgeBio since 2016. Previously, Dr. Fox was the chief medical officer of MyoKardia, Inc. and held senior positions at SmithKline Beecham, Merck Research Laboratories and AstraZeneca LP. He was on the faculty of the University of Pennsylvania School of Medicine from 1993 to 2013.
Professor Julian Gillmore undertook M.D. and Ph.D. degrees in the field of amyloidosis. His research interests include pathogenesis, diagnosis and treatment of amyloidosis. He was appointed Centre Head at University College London’s Centre for Amyloidosis & Acute Phase Proteins in 2019 and is research lead at the UK National Amyloidosis Centre. He has published more than 250 peer-reviewed articles, has supervised a number of students through their post-doctoral research degrees, is CI/PI on a range of amyloid-related clinical trials, and has received extensive grant funding.
Dr. Eric David, M.D., J.D., is CEO of BridgeBio Gene Therapy. A co-founder of Organovo, he most recently served as the company’s chief strategy officer and executive vice president of preclinical development. Prior to Organovo, Dr. David was an associate partner at McKinsey & Co., an assistant professor at The Rogosin Institute, adjunct faculty at The Rockefeller University and a lecturer in Medicine at Weill Cornell Medical College. Dr. David received his clinical training in internal medicine at New York-Presbyterian / Weill Cornell.
Associate Professor, University of Minnesota Medical School and University of Minnesota College of Pharmacy.
Dr. Kyriakie Sarafoglou, M.D., is an Associate Professor who holds appointments in the Department of Pediatrics, Divisions of Endocrinology and Genetics & Metabolism, University of Minnesota Medical School; and the Department of Experimental & Clinical Pharmacology, University of Minnesota College of Pharmacy. She specializes in rare endocrine and metabolic disorders holding multidisciplinary clinics that care for patients with congenital adrenal hyperplasia and disorders of sex development, hypophosphatasia, Turner syndrome and inborn errors of metabolism.
Chief of the Skeletal Disorders and Mineral Homeostasis Section at the National Institutes of Health in Bethesda, Maryland.
Dr. Michael Collins, M.D., is a physician scientist, trained in Endocrinology and Metabolism, whose research is broadly focused on the roles of PTH and FGF23 in bone biology and mineral homeostasis. Disease under investigation include fibrous dysplasia of bone/McCune-Albright syndrome, hypoparathyroidism, and disorders of FGF23 excess and deficiency such as x-linked hypophosphatemic rickets, tumor-induced osteomalacia, and familial tumoral calcinosis. Through detailed physiology studies and early phase clinical trials, he has made major contributions to understanding the pathophysiology of these conditions and significantly defined the standard of care of patients with these disorders.
Dr. Eli Wallace, Ph.D., is BridgeBio Pharma’s Chief Scientific Officer in Residence for Oncology. Prior to joining BridgeBio, Dr. Wallace was Chief Scientific Officer at Peloton Therapeutics, where he and his team discovered and developed a first-in-class HIF2a inhibitor for renal cell carcinoma through clinical proof-of-concept. Dr. Wallace also worked as Director of Medicinal Chemistry at Array BioPharma, leading multiple research projects which produced eight new chemical entities that have entered human clinical trials for the treatment of cancer, including Mektovi®, which was approved to treat melanoma in 2018, Tukya®, which was approved to treat breast cancer in 2020, Koselugo®, which was approved to treat NF1 in 2020, and Akt inhibitor ipatasertib, which is currently in 2 phase 3 studies evaluating the treatment of prostate and breast cancers.
Dr. Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon) is BridgeBio’s Chairman of Oncology and a professor at the UCSF Helen Diller Family Comprehensive Cancer Center. Since 2013, Dr. McCormick has led the National Cancer Institute’s Ras Initiative at the Frederick National Laboratories for Cancer Research overseeing the national effort to develop therapies against Ras-driven cancers. Dr. McCormick has previously worked at several biotechnology firms, including Cetus Corporation and Chiron Corporation. Dr. McCormick founded Onyx Pharmaceuticals in 1992.
Christine Siu is the Chief Operating Officer in Residence of BridgeBio. She formerly served as the Chief Financial Officer of Eidos Therapeutics, a BridgeBio company. Prior to that, Ms. Siu served as the chief business officer of the Bluefield Project to Cure Frontotemporal Dementia and was a venture principal at Third Rock Ventures, where she helped start Global Blood Therapeutics (GBT). Previously, she held roles of increasing responsibility at private equity and venture capital firms, Warburg Pincus and Thomas, McNerney & Partners, where she invested in life sciences companies.
