BridgeBio Pharma, Inc. (BridgeBio) operates as a commercial-stage biopharmaceutical company.
BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. The company works across over 20 disease states at various stages of development.
The company’s focus on genetic diseases because they exist at the intersection of high unmet patient need and tractable biology. The company’s approach is to translate research pioneered at academic laboratories and leading...
BridgeBio Pharma, Inc. (BridgeBio) operates as a commercial-stage biopharmaceutical company.
BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. The company works across over 20 disease states at various stages of development.
The company’s focus on genetic diseases because they exist at the intersection of high unmet patient need and tractable biology. The company’s approach is to translate research pioneered at academic laboratories and leading medical institutions into products that it will ultimately reach patients. The company is able to realize this opportunity through a confluence of scientific advances, including identification of the genetic underpinnings of disease as more cost-efficient genome and exome sequencing becomes available; progress in molecular biology; and the development and maturation of longitudinal data and retrospective studies that enable the linkage of genes to diseases.
Commercial Product and Late-Stage Clinical Pipeline
The company’s late-stage clinical pipeline has successfully resulted in the approval of its commercial product Attruby by the FDA on November 22, 2024, for the treatment of ATTR-CM in adults to reduce cardiovascular death and cardiovascular-related hospitalization and the approval of Beyonttra (acoramidis) by the EC on February 10, 2025.
Attruby/Beyonttra (acoramidis) and the three clinical product candidates in the company’s late-stage development pipeline along with their target indications, drug mechanism, estimated patient population, and development status:
Commercial Product-Attruby (U.S.)/ Beyonttra (EU) for the Treatment of TTR Amyloidosis
The company’s product, Attruby (acoramidis), previously known as AG10, a next-generation oral small molecule near-complete TTR stabilizer, was approved by the FDA in November 2024 for the treatment of cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Acoramidis has demonstrated differentiated clinical benefit in ATTR patients in the Phase 3 ATTRibute-CM study and its open label extension. Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. In December 2024, acoramidis received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending the approval of acoramidis in the European Union (EU) based on positive results from the Phase 3 ATTRibute-CM study. On February 10, 2025, the European Commission approved acoramidis for the treatment of ATTR-CM in Europe. The company licensed commercial rights in Europe to Bayer who following approval will commercialize acoramidis under the name Beyonttra as a treatment for transthyretin amyloidosis in all EU member states, as well as all member and extension states of the European Patent Organization as of the effective date of the license agreement.
The FDA’s approval of Attruby was based on positive data from its Phase 3 ATTRibute-CM clinical trial of acoramidis for patients with ATTR-CM. In July 2023, the company announced that the primary endpoint (a hierarchical analysis inclusive of all-cause mortality and frequency of cardiovascular-related hospitalization) was met (win ratio of 1.8) with a highly statistically significant p-value (p<0.0001). This primary endpoint results consistently favored acoramidis treatment across key subgroups, including across both variant and wild-type ATTR patients, as well as across New York Heart Association (NYHA) Class I, II, and III patients. Absolute values observed across all-cause mortality (ACM), cardiovascular mortality (CVM) and cardiovascular-related hospitalization (CVH) showed that over 30 months, patients survived more and were hospitalized less than, to its knowledge, has been seen in prior controlled studies of ATTR-CM. Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified.
In March 2024, the company entered into an exclusive license agreement with Bayer Consumer Care AG (‘Bayer’), a wholly owned subsidiary of Bayer AG (the ‘Bayer License Agreement’), to commercialize acoramidis in Europe. Following approval, Bayer will commercialize acoramidis under the name Beyonttra.
In September 2019, through the company’s subsidiary Eidos Therapeutics, Inc. (‘Eidos’), it entered into a license agreement (the ‘Eidos-Alexion License Agreement’), with Alexion Pharma International Operations Unlimited Company, a subsidiary of Alexion Pharmaceuticals, Inc. (together, ‘Alexion’), to develop and commercialize Beyonttra in Japan.
The company presented results from the open label extension (OLE) of ATTRibute-CM in 2024, which confirmed sustained benefit of acoramidis on cardiovascular outcomes, including statistically significant reduction in ACM within 36 months.
Design Criteria
The company designed acoramidis to meet two primary criteria to preserve circulating native transthyretin (TTR) and to reduce amyloid deposition by minimizing toxic TTR monomer formation.
Clinical Data
Phase 2 Data
In November 2018, the company announced Phase 2 data for acoramidis in symptomatic patients with ATTR-CM. The randomized, placebo-controlled, dose-ranging clinical trial included 49 patients with symptomatic ATTR-CM, of which 14 had ATTR-CM.
In November 2019, the company announced data from its Phase 2 open-label extension (‘OLE’), suggesting long-term tolerability of acoramidis and stabilization of ATTR-CM disease measures.
In October 2023, the company presented updated results from its Phase 2 OLE, demonstrating continued long-term tolerability of acoramidis and stabilization of ATTR-CM disease measures.
Phase 3 Data
In February 2019, the company-initiated ATTRibute-CM, a global Phase 3 randomized, placebo-controlled clinical trial of acoramidis in ATTR-CM. ATTRibute-CM enrolled 632 subjects with symptomatic ATTR-CM, associated with either wild-type or variant TTR and NYHA Class I-III symptoms.
On December 27, 2021, the company reported topline data from Part A of the ATTRibute-CM trial, which did not meet its primary endpoint of change from baseline in 6MWD (p = 0.76). Mean observed 6MWD decline for the acoramidis and placebo arms were 9 meters and 7 meters, respectively. Decline observed in both arms of ATTRibute-CM was similar to expected functional decline in healthy elderly adults at 12 months. The company observed improvements in acoramidis-treated participants relative to placebo-treated participants at Month 12 on secondary and exploratory endpoints including NT-proBNP, serum TTR concentration and KCCQ-OS.
On July 17, 2023, the company announced positive data from its Phase 3 ATTRibute-CM clinical trial of acoramidis for patients with ATTR-CM.
On September 27, 2024, the company announced a post-hoc analysis of ATTRibute-CM demonstrating a 42% reduction in composite ACM and recurrent CVH events at 30 months observed with acoramidis treatment compared to placebo by applying a negative binomial regression model (p=0.0005).
On November 18, 2024, the company announced acoramidis demonstrated statistically significant risk reduction in the OLE of 36% on ACM alone at Month 36 (p=0.009) and 34% by Month 42 (p=0.006), as assessed by the Stratified Cox proportional hazards model.
Products in Clinical Development
The company’s three late-stage assets, the following section summarizes the products that it has in clinical development and has the greatest potential to drive significant near-term value due to a combination of factors, including their stage of development, potential availability of expedited development pathways, degree of unmet medical need and potential market size in the applicable target indication.
Low-dose Infigratinib for the treatment of FGFR-driven skeletal dysplasias
The company developed low-dose infigratinib, an oral FGFR1-3 selective tyrosine kinase inhibitor (‘TKI’) for the treatment of children with achondroplasia and hypochondroplasia. On January 13, 2025, the company announced full enrollment of PROPEL 3, a Phase 3, multicenter, double-blinded, placebo-controlled pivotal study of low-dose infigratinib at a dose level of 0.25 milligrams per kilogram per day (mg/kg/day) in children with achondroplasia. The company enrolling patients in ACCEL, a prospective natural history study in children with hypochondroplasia.
On June 4, 2024, the company announced positive sustained results from PROPEL 2, its Phase 2 clinical trial of infigratinib in children with achondroplasia. In the highest dose level (Cohort 5, 0.25mg/kg/day), an increased annualized height velocity was observed, which persisted throughout the duration of the study.
On February 7, 2024, the company’s subsidiary, QED, granted Kyowa Kirin an exclusive license to develop, manufacture, and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan for an upfront payment of $100.0 million that was received in June 2024.
Design Criteria
The company developed low-dose infigratinib based upon two key design principles –the company seek to target achondroplasia and hypochondroplasia at their source (FGFR3 gain-of-function mutations) to maximize clinical activity against all manifestations of the condition, not just height; and the company provides a tolerable oral treatment option in order to provide a reduced burden of treatment versus injection for children and their families.
Low-dose Infigratinib: Hypochondroplasia
The company also developed low-dose infigratinib as a treatment option for children living with hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and similarly driven by FGFR3 gain-of-function variants. The company also committed to exploring the potential of infigratinib on the wider medical and functional impacts of hypochondroplasia, which hold significant unmet needs for families.
Clinical Development Plan
The company currently enrolling children living with hypochondroplasia in ACCEL, an observational study for infigratinib in hypochondroplasia.
Encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 and Hypoparathyroidism
Encaleret is an oral small molecule antagonist of the calcium sensing receptor (‘CaSR’) that it develops for the treatment of Autosomal Dominant Hypocalcemia Type 1 (‘ADH1’). The company currently studying encaleret in an ongoing Phase 3 clinical trial (NCT05680818) as a potential treatment for patients with ADH1. The company’s reported results from the Phase 2b study of encaleret in ADH1 (NCT04581629) in 2022 and published the results from the same study in the New England Journal of Medicine in September 2023.
The company also currently studying encaleret in an ongoing Phase 2 clinical trial (NCT05735015) as a potential treatment for patients with post-surgical hypoparathyroidism, in collaboration with clinical researchers at the National Institutes of Health. Preliminary results from this ongoing Phase 2 study were reported at the American Society for Bone Mineral Research annual meeting in September 2024.
Clinical Data
On June 13, 2022, the company reported positive data from its Phase 2b clinical trial of encaleret in patients with ADH1. Thirteen adults with ADH1 caused by nine unique CASR variants participated in the three-period, Phase 2b, open-label, dose-ranging clinical trial. Oral calcium and activated vitamin D supplements were discontinued prior to encaleret initiation. Periods 1 and 2 each evaluated encaleret over the course of five inpatient days and Period 3 included a 24-week outpatient evaluation. Based on 24-week outpatient data, it observed:
Mean values of blood calcium, urinary calcium, and blood parathyroid hormone, key biochemical parameters of mineral homeostasis, were normalized by Period 2, Day 5 and were sustained through Period 3, Week 24 of the trial;
At Week 24 of encaleret treatment, 92% (12/13) of participants had achieved normal trough blood calcium levels in the absence of extra-dietary calcium supplements and active vitamin D, and 77% (10/13) of participants had normal urinary calcium excretion; and
Encaleret was well-tolerated with no serious adverse events reported; there were no treatment discontinuations or study withdrawals;
In September 2024, the company reported preliminary results from an ongoing Phase 2 clinical study of encaleret in participants in post-surgical hypoparathyroidism.
In December 2022, the company also announced the initiation of its Phase 3 registrational trial of encaleret in ADH1 and completed screening for this study in November 2024.
BBP-418 for the treatment of Limb Girdle Muscular Dystrophy Type 2I
BBP-418 is an investigational, orally administered, small molecule substrate supplementation therapy that the company developed for the treatment of LGMD2I, also known as LGMDR9 FKRP-related. In October 2023, the company shared positive long-term data from its Phase 2 trial in patients with LGMD2I, including that early assessment of increased glycosylated aDG may predict subsequent ambulatory improvements at later time points, supporting the use of glycosylated aDG levels as a potential surrogate endpoint in LGMD2I. The company is currently studying BBP-418 in FORTIFY, an ongoing global registrational Phase 3 clinical trial in patients with LGMD2I.
Design Criteria
The rationale for developing BBP-418 as a potential treatment for LGMD2I is based on the company’s understanding of the disease mechanism. In healthy tissue, a properly functioning Fukutin-Related Protein (‘FKRP’) glycosylates alpha-dystroglycan (‘aDG’).
Clinical Data
On October 9, 2023, the company shared positive long-term results from its ongoing Phase 2 clinical trial of BBP-418 in patients with LGMD2I. Based on the data after 21 months of treatment, the company observed:
Increased glycosylated aDG levels from baseline observed as early as three months and sustained with treatment;
Large (=80%), sustained reduction in creatine kinase observed over an extended (up to 21-months) treatment period;
Stabilization in NSAD scores and ambulatory measures observed over 21-months of BBP-418 treatment;
BBP-418 continues to be well-tolerated with longer-term treatment; and
No treatment-related serious adverse events (‘SAEs’) or dose limiting toxicities observed with 21-months of BBP-418 dosing.
Clinical Development Plan
Following the release of top-line data from the Phase 2 trial, the company engages with regulatory authorities to align on a Phase 3 trial design, including an interim analysis at 12 months on study intended to support accelerated approval. The company’s Phase 3 trial, FORTIFY, was initiated in the U.S., with the first patient enrolled in June 2023.
Other Development
The company’s robust late-stage pipeline is supported by its productive early-stage research engine, which has produced 19 INDs since its inception. The company recently announced in the fourth quarter of 2024 a new trial ACT-EARLY, the first Phase 3 clinical trial to evaluate prophylactic acoramidis therapy for the prevention or delay of ATTR amyloidosis in asymptomatic pathogenic TTR variant carriers. In addition to this Phase 3 study, the company’s clinical product pipeline includes a Phase 2 study (ACCEL) for infigratinib for hypochondroplasia that is currently enrolling. The dosing of the first patient is anticipated in the middle of 2025. The company has an ongoing Phase 2 clinical trial for encaleret for postsurgical hypoparthyroidism. The company anticipate formalizing plans to advance development of encaleret in this indication by the end of 2025. The company is also conducting a Phase 1/2 study (CANaspire) for BBP-812 for Canavan disease.
Manufacturing
The company currently utilize third-party contract manufacturing organizations (‘CMOs’) for all required raw materials, drug substance, drug product and packaging for Attruby and Beyonttra and for its preclinical research and its ongoing clinical trials of its product candidates.
Commercialization and Product Support
U.S. Launch of Attruby (acoramidis)
The company has built its own commercial organization in the U.S. to support the commercialization of Attruby in the U.S. Following the receipt of FDA approval in November 2024, the company commercially launched Attruby in the U.S. with a sales force appropriately sized to call on all ATTR-CM treatment centers in the U.S. and other community cardiologists who treat ATTR-CM patients in the U.S. Attruby was approved by the FDA for the treatment of ATTR-CM in adults to reduce cardiovascular death and cardiovascular-related hospitalization. The company’s sales force is focused on promoting Attruby to healthcare providers and effectively communicating product benefits.
The company’s pre-launch activities, including claims analytics and disease state educational programs for healthcare providers, helped identify key stakeholders and ensure a smooth transition post-approval in November 2024
The company also offer Attruby for free to patients who are uninsured or underinsured through its Patient Assistance Program (PAP) and provide a free 28-day trial to patients new to Attruby.
The company currently partners with Bayer for the commercialization of Beyonttra in Europe and Alexion for commercialization of acoramidis in Japan if approved. The company’s plan to leverage a full-service distribution partnership to support commercialization of acoramidis in rest of world markets.
The company evaluate its commercialization strategy as it advances each product candidate through clinical development and to regulatory approval.
Intellectual Property
The company strives to protect the proprietary technology that is important to its business through a variety of methods, including seeking and maintaining patents and patent applications intended to cover its product candidates and compositions, their methods of use and processes for their manufacture, the company’s platform technologies and any other aspects of inventions that are commercially important to the development of its business. The company has entered into various license agreements to obtain the rights to use certain patents for the development and commercialization of its product candidates, as discussed further in the section titled, ‘Material Agreements.’
The company’s success will depend on its ability to obtain and maintain patent and other proprietary rights protecting its commercially important technology, inventions and know-how related to its business, defend and enforce its current and future issued patents, if any, preserve the confidentiality of the company’s trade secrets and operate without infringing the valid and enforceable patents and proprietary rights of third parties.
As of February 13, 2025, the company’s intellectual property portfolio is composed of over 100 issued patents and over 400 patent applications that it licenses from academic and research institutions and other third parties or that its own or co-own, including through its subsidiaries. These patents and patent applications generally provide with the rights to develop the company’s product candidates in the United States and worldwide. The company’s intellectual property portfolios for each of the programs that it considers to be the company’s core value drivers are further described below.
For the company’s subsidiary, QED Therapeutics, Inc. (‘QED’), its license rights from Novartis to two issued U.S. patents, and related pending and issued foreign patents and patent applications in Australia, Canada, China, Europe, Japan and Mexico, as well as in other countries in Asia and in South America, that are directed to compositions of matter of infigratinib.
The company also license rights from Inserm Transfert ESA and Assistance Publique-HÔpitaux de Paris to two issued U.S. patents and one pending U.S. patent application, and one granted patent in Europe, that are directed to methods of treating skeletal dysplasias using infigratinib.
For the company’s subsidiary Eidos Therapeutics, Inc., licenses rights from the Board of Trustees of the Leland Stanford Junior University, or Stanford, to ten issued U.S. patents, two pending U.S. patent applications, one issued European patent, and one issued Japanese patent with claims directed to composition of matter and methods of use relating to acoramidis.
In addition, the company owns five issued U.S. patents, six pending U.S. patent applications, and over 60 related foreign issued patents and patent applications in various jurisdictions, including Australia, Canada, Europe, China, Japan, and Mexico, with claims directed to salt and solid forms, methods of manufacturing, dosing methods, and/or formulations relating to acoramidis.
For the company’s subsidiary, Calcilytix, Inc., it licenses rights from Japan Tobacco Company to one issued U.S. patent and two foreign patents in Europe and Japan that are directed to compositions of matter of encaleret.
For the company’s subsidiary, ML Bio Solutions, Inc. (‘ML Bio’), its license rights from the Charlotte-Mecklenburg Hospital Authority d/b/a Atrium Health to seven issued U.S. patents, one pending U.S. patent application, and over thirty related foreign patent applications pending in various jurisdictions, including Australia, Canada, Europe, China, Japan, and Mexico with claims to methods of treatment, dosing methods, and compositions relating to BBP-418.
Material Agreements
Acoramidis (Attruby/Beyonttra)
License Agreement with Bayer
In March 2024, certain of the company’s subsidiaries, including Eidos Therapeutics, Inc. (‘Eidos’), BridgeBio International GmbH and BridgeBio Europe B.V. (‘BridgeBio B.V.’), entered into an exclusive license agreement (the ‘Bayer License Agreement’) with Bayer, to develop and commercialize acoramidis (Beyonttra) as a treatment for ATTR-CM in all EU member states as well as all member and extension states of the European Patent Organization as of effective date of the license agreement (the ‘Licensed Territory’).
License Agreement with Alexion
In September 2019, through the company’s subsidiary Eidos, the company entered into a license agreement (the ‘Eidos-Alexion License Agreement’), with Alexion Pharma International Operations Unlimited Company, a subsidiary of Alexion Pharmaceuticals, Inc. (together, ‘Alexion’), to develop and commercialize Beyonttra in Japan.
Under the terms of the Eidos-Alexion License Agreement, Eidos granted Alexion an exclusive license to certain of the company’s intellectual property rights to develop, manufacture and commercialize Beyonttra in Japan.
License Agreement with the Board of Trustees of the Leland Stanford Junior University
In April 2016, through Eidos, the company entered into an exclusive license agreement with Stanford for rights relating to novel transthyretin aggregation inhibitors. Under the company’s agreement, Stanford has granted it an exclusive worldwide license to make, use and sell products that are covered by the licensed patent rights.
Under the license agreement with Stanford, the company is obligated to use commercially reasonable efforts to develop, manufacture, and commercialize at least one licensed product; to develop markets for such licensed products; and to meet certain development milestones as agreed upon between the company and Stanford.
Infigratinib
License Agreement with Novartis International Pharmaceutical Ltd.
In January 2018, through the company’s subsidiary QED, it entered into a license agreement with Novartis International Pharmaceutical Ltd. (‘Novartis’), for certain intellectual property rights, including patents and know-how, related to infigratinib for the treatment of patients with FGFR-driven diseases, including CCA, UC and achondroplasia. the company refers to this agreement as the Novartis License.
The license grant to the company includes the right to sublicense through multiple tiers. The company also has certain rights to intellectual property licensed to Novartis’ affiliate under a materials transfer agreement with a third party.
The Novartis License is subject to Novartis’ existing obligations to supply a third party with infigratinib to support the third party’s clinical trials, and the company has an ongoing obligation to inform Novartis the company’s or its sublicensees’ intent to seek regulatory approval for and commercialize infigratinib for various indications, with potential reversionary rights to Novartis in the event of a subsequent decision not to seek regulatory approval and commercialization, or a determination by Novartis that it has failed to sufficiently pursue regulatory approval and commercialization, for Novartis to grant such third party limited rights to develop and commercialize infigratinib.
Under the Novartis License, the company is required to use commercially reasonable efforts to develop infigratinib, and to obtain regulatory approval for and commercialize infigratinib in the United States and the European Union.
Government Regulation
The company's product candidates must be approved by the FDA through either a New Drug Application, or NDA or a Biologics License Application, or BLA, process before they legally marketed in the United States.
History
BridgeBio Pharma, Inc., a Delaware corporation, was founded in 2015. The company was incorporated in 2019.
