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BridgeBio (BBIO) Begins Dosing with Gene Therapy in CAH Study
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BridgeBio Pharma, Inc. (BBIO - Free Report) announced that it has dosed the first patient in the phase I/II ADventure study, which is evaluating its investigational adeno-associated virus (“AAV”) 5 gene therapy, BBP-631, for the treatment of patients with classic congenital adrenal hyperplasia (“CAH”), a group of genetic disorders that affect the adrenal glands.
Initial data from the study is expected in the second half of 2022.
The open-label phase I/II study will investigate the safety, tolerability and pharmacodynamic activity of BBP-631 in adults with classic CAH. The primary outcomes of the study are to check the safety, as well as change from baseline in endogenous cortisol levels.
Per the company, if successfully developed, BBP-631 will become the first treatment for CAH to restore the body’s hormone and steroid balance by helping people with the disease make their own cortisol and aldosterone.
Shares of BridgeBio have plunged 84.2% in the past year compared with the industry’s decrease of 32.6%.
Image Source: Zacks Investment Research
Apart from BBP-631, BridgeBio has an AAV9 gene therapy candidate, BBP-812, which is being developed for treating Canavan disease. In November 2021, the company dosed the first patient in a phase I/II study evaluating BBP-812 for treating Canavan disease. Initial data from the study is expected later in 2022.
Other promising pipeline candidates in BridgeBio’s portfolio include acoramidis, encaleret and infigratinib.
The company is evaluating its lead pipeline candidate, acoramidis, for the treatment of symptomatic transthyretin (“TTR”) amyloid cardiomyopathy (ATTR-CM). In December 2021, BridgeBio announced dismal top-line data from part A of a two-part ongoing phase III ATTRibute-CM study evaluating acoramidis for the given indication.
A phase IIb study is evaluating encaleret as a potential treatment for autosomal dominant hypocalcemia type 1 (ADH1), a rare form of hypoparathyroidism caused by mutations in the CASR gene. A phase II study is evaluating infigratinib in patients with achondroplasia, a form of dwarfism.
Zacks Rank & Stock to Consider
BridgeBio currently carries a Zacks Rank #4 (Sell).
Image: Bigstock
BridgeBio (BBIO) Begins Dosing with Gene Therapy in CAH Study
BridgeBio Pharma, Inc. (BBIO - Free Report) announced that it has dosed the first patient in the phase I/II ADventure study, which is evaluating its investigational adeno-associated virus (“AAV”) 5 gene therapy, BBP-631, for the treatment of patients with classic congenital adrenal hyperplasia (“CAH”), a group of genetic disorders that affect the adrenal glands.
Initial data from the study is expected in the second half of 2022.
The open-label phase I/II study will investigate the safety, tolerability and pharmacodynamic activity of BBP-631 in adults with classic CAH. The primary outcomes of the study are to check the safety, as well as change from baseline in endogenous cortisol levels.
Per the company, if successfully developed, BBP-631 will become the first treatment for CAH to restore the body’s hormone and steroid balance by helping people with the disease make their own cortisol and aldosterone.
Shares of BridgeBio have plunged 84.2% in the past year compared with the industry’s decrease of 32.6%.
Image Source: Zacks Investment Research
Apart from BBP-631, BridgeBio has an AAV9 gene therapy candidate, BBP-812, which is being developed for treating Canavan disease. In November 2021, the company dosed the first patient in a phase I/II study evaluating BBP-812 for treating Canavan disease. Initial data from the study is expected later in 2022.
Other promising pipeline candidates in BridgeBio’s portfolio include acoramidis, encaleret and infigratinib.
The company is evaluating its lead pipeline candidate, acoramidis, for the treatment of symptomatic transthyretin (“TTR”) amyloid cardiomyopathy (ATTR-CM). In December 2021, BridgeBio announced dismal top-line data from part A of a two-part ongoing phase III ATTRibute-CM study evaluating acoramidis for the given indication.
A phase IIb study is evaluating encaleret as a potential treatment for autosomal dominant hypocalcemia type 1 (ADH1), a rare form of hypoparathyroidism caused by mutations in the CASR gene. A phase II study is evaluating infigratinib in patients with achondroplasia, a form of dwarfism.
Zacks Rank & Stock to Consider
BridgeBio currently carries a Zacks Rank #4 (Sell).
Top-ranked stocks in the biotech sector include Codexis, Inc. (CDXS - Free Report) , Cara Therapeutics, Inc. (CARA - Free Report) and Alkermes plc (ALKS - Free Report) , all sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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