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     147  0 Kommentare BridgeBio Pharma Announces Proof-of-Concept Data of Encaleret in ADH1 at the Endocrine Society’s 2021 Annual Meeting

    – Normalization of Blood Calcium and Urine Calcium in 6 of 6 (100%) Autosomal Dominant Hypocalcemia Type 1 (ADH1) Participants Initially Evaluated Over 5 Days Demonstrates Proof-of-Concept

    – At Doses up to 180 mg Twice Daily, Encaleret was Well-Tolerated with No Serious Adverse Events and No Adverse Events of Moderate or Severe Intensity Reported After 5 Days

    – Company to Host Webcast to Discuss Data on March 22 at 8:00 a.m. ET

    PALO ALTO, Calif., March 20, 2021 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced early results from an ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1). The data are featured in an ePoster presentation titled ‘The Effects of Encaleret (CLTX-305) on Mineral Physiology in ADH1 Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study’ at the Endocrine Society’s 2021 Annual Meeting (ENDO 2021) taking place virtually from March 20th – 23rd.

    “ADH1 is a rare genetic form of hypoparathyroidism caused by pathogenic variants in the calcium-sensing receptor (CASR) gene that are estimated to be harbored by 12,000 individuals in the United States1. Patients with ADH1 experience a range of symptoms associated with low blood calcium and high urine calcium, which cannot be adequately addressed with current standard of care therapies,” said Rachel Gafni, M.D., Senior Research Physician and Head, Mineral Homeostasis Studies Group of the National Institute of Dental and Craniofacial Research, National Institutes of Health (NIH). “In these initial results from the Phase 2b study of encaleret in ADH1, blood and urine calcium levels were normalized in all trial participants within five days of treatment.”

    In these data from the ongoing Phase 2b open-label, dose-ranging study, six adults with ADH1 with four distinct CASR genotypes were administered encaleret. Calcitriol (active Vitamin D) therapy (current standard of care) was discontinued prior to and throughout the study. Non-dietary calcium supplements were withheld in five participants with adequate dietary calcium intake. Participants received sequential, increasing daily doses of encaleret starting at 30 mg while undergoing intensive safety monitoring and frequent blood and urine sampling for biochemical measures. Following five days of dosing with encaleret, blood calcium, parathyroid hormone, phosphorus and magnesium were within the normal range on average. Urinary calcium excretion was normal or undetectable in all participants.

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    BridgeBio Pharma Announces Proof-of-Concept Data of Encaleret in ADH1 at the Endocrine Society’s 2021 Annual Meeting – Normalization of Blood Calcium and Urine Calcium in 6 of 6 (100%) Autosomal Dominant Hypocalcemia Type 1 (ADH1) Participants Initially Evaluated Over 5 Days Demonstrates Proof-of-Concept – At Doses up to 180 mg Twice Daily, Encaleret was …

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