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BridgeBio Pharma Announces Addition Of Four New Assets To Pipeline; New Drugs Target Endocrine Disorder, Inherited Eye Disease, Genetically Driven Deafness And Autism Spectrum Disorder

BridgeBio Pharma, Inc. (NASDAQ:BBIO) today disclosed four additional assets in its pipeline, expanding the number of disclosed programs to over 20 potential medicines. BridgeBio’s world-class team of experienced

Benzinga · 01/13/2020 12:36

BridgeBio Pharma, Inc. (NASDAQ:BBIO) today disclosed four additional assets in its pipeline, expanding the number of disclosed programs to over 20 potential medicines. BridgeBio’s world-class team of experienced drug developers are advancing these therapies with the aim of delivering life-changing medicines to patients. BridgeBio founder and CEO Neil Kumar, Ph.D., will discuss these new assets and BridgeBio’s pipeline during a presentation at the 38th Annual J.P. Morgan Healthcare Conference today at 3 PM (PST) in San Francisco.

The presentation will be webcast live and can be accessed at on the For Investors page under News & Events.

“The range of disorders driven by genetic mutations is vast, and our increasing understanding of the biology of such conditions is allowing us to better engineer potential therapies designed to alleviate genetically driven diseases,” said Dr. Kumar, CEO of BridgeBio. “We are excited to unveil these four programs we’ve had the privilege of working on, taking advantage of research done by companies and academic investigators on the leading edge of scientific discovery. Our teams of experts in cardiovascular and renal disease, gene therapy, and ophthalmology identified these opportunities and have been working to advance them, in some cases for more than a year.”

Encaleret is a small molecule antagonist of the calcium sensing receptor targeting conditions related to hypoparathyroidism including Autosomal Dominant Hypocalcemia Type 1 (ADH1). Individuals with ADH1 typically have low serum calcium and high urine calcium due to gain-of-function mutations in the calcium sensing receptor. BridgeBio completed the submission of an Investigational New Drug (IND) application to the US Food and Drug Administration in late 2019 to support initial development of encaleret. A Phase 2b study of encaleret in ADH1 is planned to initiate in early 2020 at the National Institutes of Health (NIH) and expected to provide proof-of-concept data in 2021. Encaleret is housed in BridgeBio subsidiary Calcilytix Therapeutics. 

BBP-551 is a novel treatment for the genetically determined retinal diseases phenotypically classified as Retinitis Pigmentosa and Leber’s Congenital Amaurosis and genotypically caused by mutations of Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT). Each of these inborn errors of metabolism disrupts key steps within the visual cycle in regenerating 11-cis-retinal, leading to a deficiency in the molecule necessary for rhodopsin formation and subsequent visual signal transduction. Both lead to progressive visual loss and retinal degeneration and, eventually, blindness. The therapy originated in the lab of Dr. Krzysztof Palczewski, Ph.D. and was advanced by David Saperstein, M.D., both at the University of Washington. BBP-551 has been granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States. BBP-551 is housed in BridgeBio subsidiary Retinagenix Therapeutics.

BBP-815 is an AAV gene therapy for the treatment for nonsyndromic hearing loss caused by recessive mutations in the TMC1 gene. TMC1 encodes the mechanosensory ion channel that converts sound vibrations in the inner ear into electrical signals in sensory hair cells. Mutations in the TMC1 gene prevent sound from eliciting the appropriate electrical response in the hair cells, resulting in moderate to severe hearing loss, often present early in life. By replacing the dysfunctional TMC1 protein using AAV gene therapy, BBP-815 is aiming to repair the deficient inner ear sensory cells at the source of this disease. The initial work for this treatment was performed by Jeffrey Holt, Ph.D, Professor of Otolaryngology and Neurology at Boston Children’s Hospital and Harvard Medical School. Dr. Holt is a world expert in hearing loss gene therapy and elucidated TMC1’s role in hearing. BBP-815 is housed in BridgeBio subsidiary Audition Therapeutics.

BBP-472 is a novel treatment designed to balance kinase signaling in the brain for the treatment of children with autism-spectrum disorders (ASD) characterized by loss of the PTEN protein. This program, currently in the lead-finding phase, is focused on advancing a brain-permeable inhibitor of PI3KB, a kinase shown to signal unabatedly in the absence of PTEN. Although PI3KB inhibitors designed to stay out of the brain are being tested experimentally for cancer indications, a PI3KB inhibitor engineered for brain penetrance has not been advanced to date, representing a major unmet need for this pediatric population.