BridgeBio Pharma ( BBIO Quick Quote BBIO - Free Report) announced dismal top-line data from part A of a two-part ongoing phase III study — ATTRibute-CM — evaluating its lead pipeline candidate, acoramidis, for the treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). Data from the study demonstrated that after 12 months of treatment with acoramidis, the candidate failed to meet the study’s primary endpoint of improvement in the six-minute walk distance (6MWD) test relative to placebo.
The treatment with acoramidis achieved a mean decline of 9 meters in the 6MWD test versus 7 meters for placebo in symptomatic ATTR-CM patients.
BridgeBio had planned to file a regulatory application seeking approval for acoramidis as a potential treatment for symptomatic ATTR-CM in 2022, if the candidate achieved a highly statistically significant improvement in 6MWD scores versus placebo in part A of the ATTRibute-CM study. However, failure to achieve relative improvement in the 6MWD score over placebo will cause a significant delay in any regulatory submission for the candidate.
However, the candidate improved quality of life and also improved NT-proBNP and stabilized serum transthyretin (TTR) levels — biomarkers for increased risk of heart failure.
The independent data monitoring committee for the ATTRibute-CM study recommended the company to continue the study despite the unexpected performance of the six-minute walk test. The company and the committee believe that acoramidis holds potential to demonstrate improvement in all-cause mortality and cardiovascular hospitalizations after 30 months of treatment.
The company is now waiting for data from part B of the ATTRibute-CM study that will continue to evaluate the candidate in study participants for 30 months, including the 12 months of part A of the study. The primary endpoint of part B of the ATTRibute-CM study is to demonstrate benefit in change from baseline in hierarchical comparison including all-cause mortality and cardiovascular hospitalizations after 30 months.
The failure hurt investor sentiments badly as shares plunged almost 72% on Dec 27. In fact shares of BridgeBio have declined 84% so far this year compared with the
industry’s decrease of 15.7%. Image Source: Zacks Investment Research
Apart from acoramidis, BridgeBio has three other promising candidates — encaleret, infigratinib and BBP-631 — in its pipeline in early to mid-stage clinical studies.
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. Updated data from the study demonstrated that treatment with encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion within five days. The company plans to initiate a phase III study to evaluate the candidate for ADH1 next year.
A mid-stage study is evaluating infigratinib in patients with achondroplasia, a form of dwarfism. Initial data from the study is expected in the first half of 2022. In November, BridgeBio initiated a phase I/II study to evaluate its gene therapy candidate, BBP-631, as a potential treatment for congenital adrenal hyperplasia, a group of genetic disorders that affect the adrenal glands.
Data from the ongoing studies will be catalysts for BridgeBio’s stock in 2022.
Zacks Rank & Stocks to Consider
BridgeBio currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the biotech sector include
Vascular Biogenics ( VBLT Quick Quote VBLT - Free Report) , Aptose Biosciences ( APTO Quick Quote APTO - Free Report) and IVERIC bio ( ISEE Quick Quote ISEE - Free Report) , all carrying a Zacks Rank #2 (Buy). You can see . the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
Loss per share estimates for Vascular Biogenics have narrowed from 44 cents to 42 cents for 2021 and from 40 cents to 37 cents for 2022 in the past 60 days.
Shares of Vascular Biogenics have risen 3.7% so far this year.
Loss per share estimates for Aptose have improved from 68 cents to 66 cents for 2021 and from 92 cents to 78 cents for 2022 in the past 60 days.
Aptose delivered an earnings surprise of 7.02%, on average, in the last four quarters.
Loss per share estimates for IVERIC bio have improved from $1.18 to $1.09 for 2021 and from $1.17 to $1.03 for 2022 in the past 60 days.
IVERIC bio’s stock has surged 137.9% so far this year.