BridgeBio Pharma (NASDAQ:BBIO) used its presentation at the 44th annual J.P. Morgan Healthcare Conference to highlight commercial momentum for its transthyretin amyloid cardiomyopathy (ATTR-CM) product Attruby, outline multiple late-stage regulatory and clinical milestones, and provide an update on earlier-stage research programs the company believes can expand its genetic disease platform.
Attruby launch metrics and revenue preannouncement
Chief Executive Officer Neil Kumar said the company was “preannouncing” fourth-quarter revenue of $146 million. Kumar characterized that figure as a 35% increase versus the prior quarter and said it implied roughly a 60% compound annual growth rate. He added that BridgeBio generated $362 million in total revenue across its first four quarters following launch.
Looking ahead, Kumar said BridgeBio, together with partner Bayer in Europe, believes the Attruby franchise could generate more than $1 billion of revenue in 2026.
Clinical positioning and planned publications in ATTR-CM
Kumar emphasized what he described as the molecule’s biochemical and clinical profile, calling Attruby the “first and only near complete stabilizer” as labeled by the FDA. He cited results he summarized as “3-42-50,” including statistically significant separation from placebo at three months, a 42% relative risk reduction in all-cause mortality and cardiovascular hospitalization at 30 months, and a 50% reduction in hospitalization at 30 months.
He also discussed an observation from data presented at HFSA, saying BridgeBio saw numeric separation in cumulative morbidity as early as one month. Kumar said this led the team to analyze hemodynamic and cardiorenal properties, and he previewed a “series of publications” over the next 12 months exploring a hypothesis that Attruby may have renal-protective attributes “analogous to the SGLT2 and ARB class,” which he said would be unique to the compound.
ATTR amyloid “depleter” antibody program
BridgeBio also announced progress on an antibody program intended to promote clearance of deposited amyloid plaque, which Kumar framed as complementary to stabilizers and knockdowns. He said the company has been working with R&D Chairman Richard Scheller and colleague Christine Zhang to optimize an antibody across four dimensions: binding more target (including a cryptic fibril binding site), clearing more target via improved macrophage recruitment, improving half-life through pH sensitivity optimization, and leveraging FcRn-binding literature to extend half-life.
Kumar said BridgeBio has created an antibody “very close to development candidate” and anticipates moving the program into the clinic in the next 18 months.
Late-stage programs: LGMD2I, ADH1/CHP, achondroplasia, and Canavan
In limb-girdle muscular dystrophy type 2I (LGMD2I), Kumar reviewed interim phase III results previously disclosed, describing a 1.8x increase in alpha-dystroglycan (ADG) glycosylation and an 82% decrease in creatine kinase after one year. He said the trial also showed statistically significant improvements in measures of ambulation and breathing, with placebo decline contrasted against improvement on treatment. Kumar said the FDA asked the company to pursue an NDA for “traditional and full approval versus accelerated approval,” and BridgeBio anticipates filing the NDA mid-year.
For autosomal dominant hypocalcemia type 1 (ADH1), Kumar said BridgeBio’s phase III results with encaleret showed a 76% responder rate defined as normalization of urine and serum calcium, versus 4% with standard of care (calcium supplementation) in the trial. He also said more than 90% of patients showed response on measures including PTH moving in the expected direction. On patient finding, Kumar described three initiatives—genetic testing partnerships, creation of an ICD-10 code, and guideline updates recommending genetic testing for nonsurgical hypoparathyroidism—and said these efforts have helped identify 1,700 unique patients to date.
Kumar also discussed encaleret in chronic hypoparathyroidism, citing a small trial of about 10 patients with 80% normalization of urine and serum calcium. He said BridgeBio aligned with the FDA on a six-month study design called RECLAIM-HP, which the company intends to begin mid-year.
In achondroplasia and hypochondroplasia, Kumar said BridgeBio has reached last patient/last visit in its phase III achondroplasia trial and expects to report data in Q1. He also said the company has enrolled its first patient in a pediatric/toddler study and completed phase II enrollment in its hypochondroplasia study. Kumar described market research for infigratinib that, based on a tested target product profile, suggested a “stubbornly consistent” 52% market share, while noting the company plans to update expectations as phase III data become available.
For Canavan disease gene therapy, Kumar reiterated biomarker reductions (urine NAA mirrored by CSF) at a high dose and described dose-responsive functional improvements such as sitting, head control, reaching and grasping, and in some cases ambulation. He said BridgeBio anticipates filing a BLA in 2027.
Early-stage example: EPP program Port-77
Kumar closed with a research example in erythropoietic protoporphyria (EPP), describing Port-77 as an orally bioavailable inhibitor of the PP9 transporter ABCG2. He said BridgeBio’s phase II-A trial showed a 75% reduction in plasma PP9, with action beginning within hours and reaching steady state within days. Kumar said all 12 patients dosed showed PP9 reduction, and that high-dose Port-77 produced reductions ranging from 57% to the 75% mean. He also said the safety profile appeared clean in the study, with a numeric imbalance favoring treatment over placebo across multiple adverse event categories.
Kumar concluded by saying BridgeBio is “well financed” to pursue its commercial and development priorities and expects a steady cadence of updates across its portfolio.
About BridgeBio Pharma (NASDAQ:BBIO)
BridgeBio Pharma, Inc is a clinical-stage biopharmaceutical company headquartered in Palo Alto, California. Founded in 2015 by Neil Kumar, the company is dedicated to discovering, developing and delivering transformative medicines for patients with genetic diseases and cancers. BridgeBio operates an integrated model that spans target identification, preclinical research, clinical development and commercialization, aiming to streamline the process from bench to bedside.
BridgeBio’s pipeline comprises multiple therapeutic modalities, including small molecules, biologics and genetic therapies.
