BridgeBio Announces Clinical Collaboration with Amgen to Study BBP-398, a Potentially Best-in-class SHP2 Inhibitor, in Combination with LUMAKRAS® (sotorasib) in Advanced Solid Tumors with the KRAS G12C Mutation

– First clinical combination study of BBP-398 and LUMAKRAS set to evaluate safety and preliminary efficacy in solid tumors with the KRAS G12C mutation

PALO ALTO, CA – January 13, 2022 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced a non-exclusive clinical collaboration with Amgen Inc. (Amgen) to evaluate the combination of BBP-398, a potentially best-in-class SHP2 inhibitor with LUMAKRAS® (sotorasib), a KRASG12C inhibitor, in patients with advanced solid tumors with the KRAS G12C mutation.

The Phase 1/2 study will include a dose escalation period followed by dose expansion and optimization, and is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of BBP-398 in combination with LUMAKRAS. Under the terms of the non-exclusive collaboration, BridgeBio will sponsor the study and Amgen will provide a global supply of LUMAKRAS.

BBP-398 is a potent small-molecular inhibitor of SHP2 developed in collaboration with The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. SHP2 is a protein-tyrosine phosphatase that links growth factor, cytokine and integrin signaling with the downstream RAS/ERK MAPK pathway to regulate cellular proliferation and survival. By combining SHP2 inhibition with KRASG12C inhibition in patients with the KRAS G12C mutation, there is potential that the investigational combination could prevent oncogenesis and overactive cellular proliferation.

“Overactivity of the MAPK pathway is a significant cause of many types of difficult-to-treat cancers and by combining these two agents, we aim to reduce the oncogenic potential of tumor cells,” said Frank McCormick, Ph.D., chairman of oncology at BridgeBio. “Building on our collaborations with Bristol Myers Squibb and LianBio, we are excited to be working with Amgen on this new collaboration. By harnessing the power of BBP-398 as a potentially best-in-class SHP2 inhibitor with LUMAKRAS, we are hopeful that we will be able able to provide substantial relief for cancer patients in need. We will continue to pursue additional collaborations that we believe hold promise for patients.”

KRAS mutations occur in approximately 17% of malignant solid tumors. BBP-398, as a monotherapy or in combination with other targeted therapies, could potentially be a promising therapy for patients with the KRAS G12C mutation.

BridgeBio is currently advancing its Phase 1 clinical trial of its SHP2 inhibitor, BBP-398, in patients with solid tumors driven by mutations in the MAPK signaling pathway, including RAS and receptor tyrosine kinase genes. BBP-398 is part of BridgeBio’s growing precision oncology pipeline and is one of 14 programs in the broader portfolio that are being advanced in the clinic or commercial setting.

About BBP-398

BBP-398 is a potentially best-in-class SHP2 inhibitor. Earlier this year, BridgeBio entered a non-exclusive, co-funded clinical collaboration with Bristol Myers Squibb to evaluate the combination of BBP-398 with OPDIVO® (nivolumab) in patients with advanced solid tumors with KRAS mutations. BridgeBio previously also entered into a strategic collaboration with LianBio for clinical development and commercialization of BBP-398 in combination with various agents in solid tumors such as non-small cell lung cancer, colorectal cancer and pancreatic cancer in mainland China and other major Asian markets.

About BridgeBio Pharma, Inc.

BridgeBio Pharma (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible.  For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding the success of our non-exclusive clinical collaboration with Amgen, the timing and success of a Phase 1/2 study to evaluate the safety and preliminary efficacy of BBP-398 in combination with LUMAKRAS in patients with advanced solid tumors with the KRAS G12C mutation, the ability of combining SHP2 inhibition with KRASG12C inhibition in patients with the KRAS G12C mutation to prevent oncogenesis and overactive cellular proliferation, our ability to provide substantial relief for cancer patients in need, the promise of targeted therapies for patients with KRAS mutations, the success and status of current and future relationships with third-party collaborators and academic partners, the continuing success of our clinical collaboration with Bristol Myers Squibb to evaluate the combination of BBP-398 with OPDIVO® (nivolumab), and the potential ability of our product candidates to treat genetically driven diseases and cancers with clear genetic drivers, reflect our current views about our plans, intentions, expectations, strategies and prospects, and are based on the information currently available to us and on assumptions we have made and are not forecasts, promises nor guarantees. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by these forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of our product candidates to treat genetically driven diseases and cancers with clear genetic drivers, the continuing success of our collaboration with Amgen and other third parties, our ability to enter into future collaboration agreements, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, as well as those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K and BridgeBio’s other SEC filings. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com
(516) 554-5989

BridgeBio Pharma Reports Month 12 Topline Results from Phase 3 ATTRibute-CM Study

– ATTRibute-CM did not meet its primary endpoint at Month 12. Mean observed six-minute walk distance (6MWD) decline for the acoramidis and placebo arms were 9 meters and 7 meters, respectively. Both declines are similar to healthy elderly adults and less than prior untreated ATTR-CM cohorts

The company observed improvements at Month 12 on the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS, nominal p < 0.05), a quality-of-life measurement, N-terminal pro BNP (NT-proBNP, median +0.6% vs. +24.3%, nominal p < 0.05), a cardiac biomarker, and serum TTR concentration (mean +38.5% vs. -0.7%, nominal p < 0.01), a measure of TTR stabilization

Acoramidis was generally well-tolerated with no safety signals of clinical concern identified. 27% fewer treatment emergent adverse events (AEs) leading to death occurred in participants receiving acoramidis than in participants receiving placebo (4.5% vs. 6.2%)

The ATTRibute-CM independent data monitoring committee recommends continuing the study based on unblinded data reviews

BridgeBio is fully funded through the completion of ATTRibute-CM and expects to realize at least four other clinical-stage pipeline catalysts beyond acoramidis in 2022

– BridgeBio to host investor call on December 27, 2021 at 8:00 AM ET

PALO ALTO, CA – December 27, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced topline results from Month 12 (Part A) of ATTRibute-CM, an ongoing global Phase 3 study investigating acoramidis for the treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The mean observed decline in 6MWD at Month 12 in participants receiving acoramidis or placebo with baseline eGFR ≥ 30 mL/min/1.73m2 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.1 The declines were also substantially less than the >40 meter annual declines observed in previous untreated arms reviewed by the company.2 The decline in the ATTRibute-CM placebo group was more than 70% lower than the decline observed in the ATTR-ACT treatment group.2

The ATTRibute-CM independent data monitoring committee recommends continuing the study based on unblinded data reviews. Despite the unexpected performance of the six-minute walk test, the trial’s steering committee co-chairs and the Company agree that there is potential for acoramidis to demonstrate benefit on the Month 30 endpoint which includes all-cause mortality and cardiovascular hospitalizations. 

“This result is disappointing and baffling. I am, along with many others, searching for answers regarding the 6MWD,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. “The results do not appear to be due to a baseline imbalance. The hypotheses we are currently evaluating include context bias, training bias, and an evolution in diagnosis and standard of care. The drug does appear to be pharmacologically active and well-tolerated, and we observed improvement on quality of life with promising trends on adverse events leading to death. The drug seems to be doing what we are asking of it. If we observe enough clinical outcome events at Month 30, I am still hopeful that we will demonstrate the benefit of acoramidis treatment.”

“Although these results were not what we hoped, the most important moment in this trial will be the Part B readout at 30 months, where we will see the effects of acoramidis on all-cause mortality and cardiovascular hospitalizations.  From what I’ve seen so far, I remain enthusiastic about getting to that endpoint,” said Daniel Judge, M.D., professor in the Division of Cardiology at the Medical University of South Carolina, and co-chair of the ATTRibute-CM Steering Committee.

ATTRibute-CM enrolled 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR, with New York Heart Association (NYHA) Class I-III symptoms. The study is designed as a two-part study with Part A (Month 12) comparing change from baseline in 6MWD and Part B (Month 30) utilizing a hierarchical comparison including all-cause mortality and cardiovascular hospitalizations.

ATTRibute-CM enrolled a similar patient population as ATTR-ACT, excepting a smaller proportion of U.S. participants and TTR variant carriers. Participants were randomized 2:1 between treatment (acoramidis 800 mg) and placebo twice daily. Based on data available after 12 months of treatment, the Company observed:

  • In the primary analysis, change from baseline in 6MWD was not improved in the acoramidis arm relative to the placebo arm (p = 0.76)
    • Key differences in NYHA class, geographic distribution, and TTR variant status compared to the ATTR-ACT population do not appear to have affected the primary outcome of ATTRibute-CM
    • The only participant sub-population the company has reviewed to date that exhibited substantial placebo decline in 6MWD by Month 12 was the variant population. In that population, observed decline in placebo and acoramidis was -40 meters and -2 meters, respectively
  • Acoramidis improved Kansas City Cardiomyopathy Questionnaire Overall Summary Score relative to placebo (nominal p < 0.05, mixed model repeated measures without imputation)
  • Acoramidis improved NT-proBNP relative to placebo. Median percent change from baseline at Month 12 in acoramidis-treated and placebo-treated participants were +0.6% and +24.3%, respectively (nominal p < 0.05 based on absolute changes from baseline between groups)
  • Acoramidis increased serum TTR levels relative to placebo. Mean percent change from baseline at Month 12 in acoramidis-treated and placebo-treated participants were +38.5% and -0.7%, respectively (nominal p < 0.01 based on absolute changes from baseline between groups)
  • Acoramidis was generally well-tolerated with no safety signals of clinical concern identified. To protect the integrity of Part B, the Sponsor’s access to unblinded adverse event data for Part A excludes AEs leading to a cardiovascular hospitalization (as determined by investigators) excepting events with the outcome of death
    • Adverse events occurred in 85.3% of placebo-treated participants and 91.9% of acoramidis-treated participants
    • Serious adverse events occurred in 23.2% of placebo-treated participants and 20.2% of acoramidis-treated participants
    • Adverse events with outcome of death occurred in 6.2% of placebo-treated participants and 4.5% of acoramidis-treated participants 

BridgeBio is well-capitalized through the completion of ATTRibute-CM and remains on track to deliver on additional catalysts in 2022 and 2023. “With the financing announced in November 2021, we currently have approximately $800 million in cash, cash equivalents and marketable securities with access to up to $300 million on achieving portfolio milestones through year-end 2022. We have ongoing clinical trials in multiple genetic diseases, including achondroplasia, autosomal dominant hypocalcemia type 1, limb-girdle muscle dystrophy type 2i and dystrophic epidermolysis bullosa, and we believe we are well-positioned to deliver in 2022,” said Brian Stephenson, Ph.D., chief financial officer of BridgeBio.

1Enright, P.L. et al. Chest 2003.
2Maurer, M.S. et al. NEJM 2018.; Lane, T. et al., Circulation 2019.

Webcast Information
BridgeBio will host a conference call and simultaneous webcast to share updates on the Phase 3 Part A data for acoramidis on December 27th at 8:00 AM ET. To access this call, dial (800) 379-2666 (U.S.) or (409) 937-8964 (International) with Conference ID: 2895217. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event. 

About Acoramidis

Acoramidis (AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to TTR amyloidosis, or ATTR. Acoramidis is currently being evaluated in Phase 2 and Phase 3 studies in patients with ATTR. Acoramidis was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene.

About Transthyretin Amyloidosis (ATTR)

Likely affecting more than 400,000 patients globally, ATTR is an underdiagnosed and life-threatening disease with limited treatment options that can devastate the heart and nervous system. When the transthyretin (TTR) becomes unstable due to inherited variants or aging, it can accumulate as amyloid fibrils in various organs in the body, causing ATTR. TTR amyloid deposits predominantly in the heart and/or peripheral nerves, causing cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN). ATTR often dramatically impairs the quality of life, functional independence and life expectancy of patients, as well as impacting caregivers due to the progressive nature of the disease. If left untreated life expectancy from diagnosis is approximately four years.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to our clinical trial results for Part A of the Phase 3 ATTRibute-CM Study, the prospects of success for Part B results from the Phase 3 ATTRibute-CM Study, the market opportunity for AG10, our anticipated cash runway and our being fully funded through the completion of the ATTRibute-CM study and our ability to access additional funding upon achievement of portfolio milestones, reflect our current views about our plans, intentions, expectations, strategies and prospects, and are based on the information currently available to us and on assumptions we have made and are not forecasts, promises nor guarantees. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by these forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of our product candidates to treat genetically driven diseases and cancers with clear genetic drivers as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and BridgeBio Pharma’s other SEC filings. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com
(516) 554-5989

BridgeBio Pharma Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)

Palo Alto, CA, December 10, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that on December 6, 2021, the compensation committee of BridgeBio’s board of directors granted nine new employees restricted stock units for an aggregate of 12,073 shares of the Company’s common stock. All of the above-described awards were made under BridgeBio’s 2019 Inducement Equity Plan (the Plan).

The above-described awards were each granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan. The Plan was adopted by BridgeBio’s board of directors in November 2019.

About BridgeBio
BridgeBio Pharma (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

Media Contact
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Pharma and Helsinn Group Announce Strategic Collaboration to Co-Develop and Co-Commercialize BridgeBio’s Novel GPX4 Inhibitor in Multiple Cancer Tumor Types

-The potentially first-in-class inhibitor designed to target glutathione peroxidase 4 (GPX4) has the potential to impact approximately 500,000 cancer patients with unmet therapeutic needs

-BridgeBio and Helsinn have also established a non-exclusive framework agreement to identify and potentially co-develop and co-commercialize additional small molecule targeted oncology therapies

-This framework agreement leverages BridgeBio’s drug discovery expertise and Helsinn’s clinical development and commercial capabilities

-The BridgeBio and Helsinn collaboration builds on the $2.45 billion USD global license and collaboration agreement signed in March 2021 for development of BridgeBio’s FGFR inhibitor infigratinib in oncology indications

Palo Alto, CA, and Lugano, Switzerland, November 19, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, and Helsinn Group, a fully integrated, global biopharma company with a diversified pipeline of innovative oncology assets and strong track record of commercial execution, announced they have entered into a strategic collaboration to codevelop and co-commercialize a potentially first-in-class inhibitor designed to target glutathione peroxidase 4 (GPX4) with the hope of providing an effective new therapy for patients with difficult-to-treat tumors.

The joint collaboration for BridgeBio’s GPX4 inhibitor was established as part of a new non-exclusive collaboration framework between BridgeBio and Helsinn that allows the companies to propose co-development and co-commercialization opportunities for preclinical precision oncology programs.

Under the terms of the non-exclusive agreement, BridgeBio and Helsinn will have the option to collaborate on preclinical oncology programs that are identified from time to time by either party. The agreement is designed to magnify the ability of both companies to identify small oncology interventions that may have greater potential to help patients in combination with larger investigational therapies. For each program that the parties agree to pursue, they will share global development responsibilities under an agreed cost split. Helsinn will have exclusive manufacturing and commercial rights to the programs under the agreement, with BridgeBio receiving a profit share on U.S. sales and tiered royalties on ex-U.S. sales.

The first program under the framework collaboration agreement that the parties will pursue is GPX4, a potentially first-in-class inhibitor that may be an effective new therapy for certain cancer patients. GPX4 is an enzyme that is often elevated in cancer tissue and associated with a worse prognosis for patients. GPX4 neutralizes toxic free radicals at the lipid membrane, protecting cells from death by ferroptosis. The GPX4 inhibitor is being developed to induce ferroptosis in cancer cells with the potential to impact approximately 500,000 patients in need of a therapeutic option. The safety and efficacy of GPX4 has not yet been established by any health authority world-wide.

“We are excited to expand our collaboration with Helsinn to develop and potentially commercialize our GPX4 program. Our hope is that together we can move even more swiftly to advance this potential precision oncology therapy for cancer patients living with severe unmet medical needs,” said BridgeBio’s chairman of oncology, Frank McCormick, Ph.D., F.R.S., D.Sc. (Hon).

Riccardo Braglia, vice chairman and CEO at Helsinn Group, commented: “This non-exclusive pipeline agreement with BridgeBio has the potential to be transformational for Helsinn because BridgeBio’s world class drug discovery platform can augment our innovative oncology pipeline. It also affords Helsinn the opportunity to identify and offer potential programs on which the parties could collaborate. BridgeBio’s deep expertise in drug hunting and early preclinical development combined with Helsinn’s drug development and global commercial platform can facilitate an ongoing cadence of moving novel therapies into clinical development with the potential to meaningfully improve the lives of patients with cancer. We’re delighted to get started with our first program, GPX4, and look forward to updating the market on this and additional programs in due course.”

The non-exclusive framework agreement builds on an earlier global collaboration and licensing agreement that BridgeBio and Helsinn Group’s affiliates, Helsinn Healthcare S.A. and Helsinn Therapeutics (U.S.), Inc., entered into in March 2021. Under that agreement, Helsinn Therapeutics is jointly responsible for further development and commercialization activities for infigratinib, a small molecule kinase inhibitor of FGFR, in oncology and all other indications except for skeletal dysplasias (including achondroplasia) in the United States and other regions (excluding China, Hong Kong, and Macau), sharing profits and losses on an equal basis. This includes exclusive commercialization rights for infigratinib in Canada, where Health Canada recently approved TRUSELTIQ™ (infigratinib) under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement. Helsinn will fund the majority of ongoing and future research and development related to infigratinib in oncology in the foregoing territory. BridgeBio will be eligible for tiered royalties as a percentage of adjusted net sales, and potential payments totaling up to $2.45 billion USD in the aggregate. BridgeBio previously entered a strategic collaboration with LianBio for development and commercialization of infigratinib in oncology indications in China, Hong Kong and Macau.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

About the Helsinn Group

Helsinn is a fully integrated, global biopharma company headquartered in Lugano, Switzerland. It is focused on improving the lives of cancer patients all over the world with a leading position in cancer supportive care and innovative pipeline of cancer therapeutics.

Helsinn is third-generation family-owned company, that since 1976 has been focused on improving the lives of patients, guided by core values of respect, integrity and quality. It operates a unique licensing business model with integrated drug development and manufacturing capabilities. Helsinn has a commercial presence in 190 countries either directly, with operating subsidiaries in the U.S. and China, or via its network of long-standing trusted partners.

Helsinn Group plays an active and central role in promoting social transformation in favor of people and the environment. Corporate social responsibility is at the heart of everything we do, which is reinforced in the company’s strategic plan by a commitment to sustainable growth.

To learn more about Helsinn Group please visit www.helsinn.com

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with such safe harbor provisions. These forward-looking statements include statements relating to expectations, plans and prospects regarding clinical development plans, clinical and therapeutic potential, regulatory status and commercial strategy for BridgeBio’s novel glutathione peroxidase 4 (GPX4) inhibitor, including, but not limited to: the ability of BridgeBio and Helsinn to jointly develop and commercialize a potentially first-in-class inhibitor designed to target glutathione peroxidase 4 (GPX4) to provide an effective new therapy for patients with difficult-to-treat tumors, the ability of BridgeBio’s GPX4 inhibitor to be a first-class inhibitor and to induce ferroptosis in cancer cells, the size of the patient population BridgeBio’s GPX4 inhibitor may be able to impact, the success of the non-exclusive framework between BridgeBio and Helsinn to allow the companies to propose additional co-development and co-commercialization collaborations for other preclinical precision oncology programs, the ability of the agreement to magnify the ability of both companies to identify small oncology interventions that may have greater potential to help patients in combination with larger investigational therapies, the belief that the combination of BridgeBio’s early preclinical development and Helsinn’s global commercial platform will help to accelerate the identification and development of this therapeutic option for patients, the timing and cadence of updating the market on GPX4 and additional programs, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales; the fact that the U.S. Food and Drug Administration or such other regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of the various collaborations between BridgeBio and Helsinn, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, and those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and our other SEC filings. Moreover, BridgeBio and Helsinn operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com
(516) 554-5989

Helsinn Group Media Contact:
Paola Bonvicini
Group Head of Communication
Info-hhc@helsinn.com
+41 (0) 91 985 21 21

BridgeBio Pharma, Inc. Secures Up to $750 Million in Non-Dilutive Debt Financing

-Innovative financing facility and existing cash balance gives BridgeBio access to over $1.2 billion, which is expected to fully fund the Company’s 30+ genetic disease and cancer pipeline programs into 2024

PALO ALTO, Calif., NOVEMBER 18, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that it has executed a definitive credit facility agreement with a syndicate of lenders for up to $750.0 million in financing.

This facility, combined with the Company’s existing cash balance as of September 30, 2021, provides access to over $1.2 billion to advance the Company’s pipeline programs, support commercialization efforts, and enable the Company to pursue strategic business development opportunities. As structured, this financing is expected to fully fund BridgeBio’s portfolio of more than 30 drug development and discovery programs into 2024, independent of near-term milestone readouts. It is a significant achievement in BridgeBio’s broader efforts to attract diverse sources of capital to fund life science innovation and is aligned with its long-term strategy of creating non-dilutive financing pathways that leverage portfolio readouts – in addition to cash balance on hand – to extend runway.

This financing announcement follows BridgeBio’s repurchase of approximately $150.0 million in its own common stock under its 2021 Share Repurchase Program, completing about $385.6 million of equity and capped call purchases in the aggregate since its initial public offering in 2019. In addition, today’s financing replaces the Company’s $100.0 million debt facility with Hercules. Collectively, these transactions represent a strategic recapitalization of the Company ahead of upcoming clinical data readouts. 

“We are grateful to have the support of debt investors who are committed to helping us build the next great genetic medicine company and deliver meaningful therapies for patients in need. Since our founding, we have believed in the power and importance of innovative financing approaches to advance critical biomedical research and drug development, and we are grateful that our broad diversified pipeline enables us to do this. By bringing on this additional capital, we have the potential to help more people living with genetic diseases and cancers as quickly as possible,” said Brian Stephenson, Ph.D., CFA, BridgeBio’s chief financial officer.

“Potential breakthrough medicines should never languish on the shelf because of a lack of funding. By seizing inventive financing tools to fund its growing R&D pipeline, BridgeBio is working to ensure that promising medicines in development can advance into the clinic and toward potential commercial approval. The Company’s groundbreaking approach to science and finance has made it a leader within the biotech industry and we are hopeful there are new methods we can explore to more fully unlock the power of investors to help patients,” said Andrew Lo, Ph.D., a BridgeBio co-founder and a member of the Company’s board of directors.

Key features of the credit facility include:

  • $450.0 million funded on November 17, 2021
  • An additional $300.0 million to be funded at the Company’s option following either 1) positive topline results from Part A of its Phase 3 ATTRibute-CM trial of TTR stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM), which is expected by the end of the year, OR 2) positive proof-of-concept data for various pipeline programs by year end 2022, with $100.0 million available upon each proof-of-concept, for up to three pipeline programs
  • Fixed interest rate of 9%, with 3% eligible at the Company’s discretion to be paid in kind and added to principal
  • Maturity date of November 17, 2026
  • Interest-only period for three years, which may be extended to four years upon success of the Part A readout
  • Substantial flexibility for future business development, M&A, share repurchases, and royalty transactions
  • No financial covenants

Morgan Stanley & Co. LLC acted as the sole placement agent to BridgeBio on this transaction.

Latham & Watkins LLP acted as counsel to BridgeBio.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. The Company intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include statements relating to the Company’s anticipated cash runway and its ability to advance the Company’s pipeline programs, support commercialization efforts, and enable the Company to pursue strategic business development opportunities, the continuing ability of BridgeBio to attract diverse sources of capital to fund life science innovation, the Company’s long-term strategy of creating non-dilutive financing pathways that leverage portfolio readouts to extend runway, the success of the Company’s 2021 Share Repurchase Program, the ability of the Company to build the next great genetic medicine company and deliver meaningful therapies for patients in need, the timing and success of the Company’s topline results from Part A of its Phase 3 ATTRibute-CM trial of TTR stabilizer for transthyretin amyloid cardiomyopathy, the timing and success of the Company’s proof-of-concept data for various pipeline programs, as well as the Company’s ability to unlock additional funding under the credit facility, and reflect the Company’s current views about its plans, intentions, expectations and strategies, which are based on the information currently available to the Company and on assumptions the Company has made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, it can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the Company’s anticipated cash runway and its ability to advance the Company’s pipeline programs, the success of the Company’s long-term strategy of creating non-dilutive financing pathways, the success of portfolio readouts in unlocking additional capital under the credit facility, potential volatility in the Company’s share price and its impact on the 2021 Share Repurchase Program, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, as well as those risks set forth in the Risk Factors section of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, and its other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com 
(516) 554-5989

BridgeBio Pharma Reports Third Quarter 2021 Financial Results and Business Update

Topline results from Part A of the Phase 3 ATTRibute-CM trial of TTR stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM) expected by end of 2021

Meaningful progress in RAS cancer portfolio with discovery of next-generation KRAS G12C dual inhibitors and novel PI3ka:RAS breakers

-Advancements in gene therapy pipeline with first patient dosed in Canavan disease trial and newly announced program targeting classic galactosemia (severe GALT deficiency)

Company repurchased $148.4 million in BridgeBio common stock under 2021 Share Repurchase Program, demonstrating the Company’s confidence in the long-term prospects of its pipeline

Ended quarter with $599.6 million in cash, cash equivalents and marketable securities

PALO ALTO, Calif., NOVEMBER 4, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today reported its financial results for the third quarter ended September 30, 2021 and provided an update on the Company’s operations.

BridgeBio has more than 30 programs in its pipeline for patients living with genetic diseases and cancers and 20 ongoing clinical trials underway across more than 450 sites around the world. Earlier this year BridgeBio received its first two U.S. Food and Drug Administration (FDA) drug approvals and has successfully filed 15 Investigational New Drug (IND) applications since the Company’s founding in 2015.

BridgeBio’s four core value drivers:

  • Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): Topline results from Part A of the Phase 3 ATTRibute-CM trial are expected in late 2021 and from Part B in 2023. The primary endpoint at Part A is the change from baseline in a 6-minute walk distance (6MWD) in trial participants receiving acoramidis or placebo after 12 months. If the change from baseline in 6MWD in Part A is highly statistically significant, BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA. The study enrolled more than 600 subjects with either wild-type or variant TTR across more than 80 sites in 18 countries. ATTR-CM is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.
  • Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): BridgeBio presented updated Phase 2b data for encaleret in an oral presentation at the American Society of Bone and Mineral Research (ASBMR) 2021 Annual Meeting in October. Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion. Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of current standard-of-care. If approved, encaleret could be the first therapy on the market for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor (CASR) gene estimated to be carried by 12,000 individuals in the United States alone. BridgeBio plans to initiate a Phase 3 registrational trial of encaleret in patients with ADH1 in 2022.
  • Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the first half of 2022. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with a prevalence of greater than 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known product candidate in clinical development for achondroplasia that is designed to target the disease at its genetic source and the only orally administered product candidate in clinical-stage development.
  • BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): Received Fast Track designation from the FDA in May 2021. IND cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing, with initial data anticipated in mid-2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases estimated in the United States and European Union. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to lack of endogenous cortisol production. BridgeBio’s AAV5 gene therapy candidate is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.

Recent pipeline progress and corporate updates:

  • Stock repurchases: BridgeBio repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program, demonstrating the Company’s confidence in the long-term prospects of its pipeline.
  • LianBio IPO and partnership: China-based partner LianBio raised $325 million in its initial public offering on November 1. BridgeBio is estimated to own approximately 4.7% post-IPO. BridgeBio and LianBio announced in August that the first patient was treated in a Phase 2a trial of infigratinib in patients with gastric cancer and other advanced solid tumors. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.
  • RAS cancer portfolio: BridgeBio announced the discovery of its next-generation KRAS G12C dual inhibitors, the first-known compounds that directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations, and PI3ka:RAS breakers, small molecules that block RAS driven PI3Ka activation – a novel approach with the potential to inhibit oncogenic PI3Ka signaling without adverse effects on glucose metabolism. RAS is one of the most well-known oncogenic drivers with approximately 30% of all cancers being driven by RAS mutations, including large proportions of lung, colorectal and pancreatic tumors. BridgeBio expects to select a RAS development candidate in 2022.
  • BBP-812 – AAV9 gene therapy candidate for Canavan disease: BridgeBio announced that the first patient was dosed in its Phase 1/2 trial of BBP-812 for Canavan disease. If successful, BridgeBio’s gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and life-threatening condition. An initial Phase 1/2 data readout is expected in 2022.
  • BBP-818 – AAV gene therapy candidate for classic galactosemia (severe GALT deficiency): BridgeBio announced a new gene therapy program for classic galactosemia, which is caused by a severe deficiency of the enzyme galactose-1-phosphate uridylyltransferase (GALT), affecting approximately 7,000 patients in the United States and the European Union. Preclinical studies in a mouse model of classic galactosemia have shown that BridgeBio’s BBP-818 therapy restored up to 72% of wild-type levels of GALT enzyme in the brain following a single dose.
  • BBP-398 – SHP2 inhibitor: First publication of preclinical data for BridgeBio’s potentially best-in-class SHP2 inhibitor designed for the treatment of resistant cancer. Data demonstrated activity in non-small cell lung cancer (NSCLC) driven by RAS or other MAPK-pathway activating mutations. The results were featured in a poster presentation shared at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in October.
  • BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i): Fast Track designation granted by the FDA. The Phase 2 trial was initiated in patients with LGMD2i in the first quarter of 2021. If successful, BBP-418 could be the first approved therapy for patients with LGMD2i. With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein. A Phase 2 data readout is expected in 2022.
  • BBP-711 – Glycolate oxidase (GO) inhibitor for hyperoxaluria: BridgeBio announced preliminary Phase 1 data in which BBP-711 was well-tolerated and resulted in maximal increases in plasma glycolate exceeding those achieved by any GO-targeting agents reported in healthy adult volunteers. BBP-711 is being developed for the treatment of primary hyperoxaluria type 1 (PH1) and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers. A full readout of Phase 1 data in healthy adult volunteers is expected in 2022, to be followed by initiation of a Phase 2/3 trial in PH1 and a Phase 2 proof-of-concept trial in recurrent kidney stone formers.
  • TRUSELTIQ™ (infigratinib): Health Canada approved TRUSELTIQ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement. BridgeBio’s partner Helsinn Group has exclusive commercial rights for TRUSELTIQ in Canada with BridgeBio eligible for tiered royalties as a percentage of net sales as part of the global collaboration and license agreement entered into between the two companies in March 2021.
  • BridgeBio Pharma R&D Day: Held a virtual R&D Day on October 12, 2021. Presentation replay can be found on BridgeBio’s investor website here.
  • Four new independent directors added to BridgeBio’s board:
    • Hannah Valantine, M.D., a leader in organ transplant genomics and workforce diversity who is a professor of medicine at Stanford University School of Medicine
    • Fred Hassan, a pharmaceutical and financial industry leader who is the former CEO of Schering-Plough and former chairman of Bausch & Lomb
    • Andrea Ellis, a consumer technology innovator and the chief financial officer of Lime
    • Douglas Dachille, an investment management veteran and the former chief investment officer of American International Group, Inc. (AIG)

Third Quarter 2021 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $599.6 million as of September 30, 2021, compared to $607.1 million as of December 31, 2020. Over the past three quarters, the Company repurchased $148.4 million in BridgeBio common stock under its 2021 Share Repurchase Program and $50.0 million in BridgeBio common stock in conjunction with its issuance of the 2029 convertible notes, paid $61.3 million for capped call options related to the issuance of its 2029 convertible notes and $35.0 million of regulatory-related milestone payments in connection with its approved products. Earlier during the year, BridgeBio paid $21.3 million to Eidos shareholders who elected for cash settlement in exchange for their Eidos shares and $63.8 million of direct transaction costs arising from the merger with Eidos. These were offset by cash receipts of $731.4 million in net proceeds from the issuance of BridgeBio’s 2029 convertible notes, $65.1 million from collaboration partner, Helsinn Group, and $25.0 million in net proceeds from Hercules Capital, Inc. under an amended loan agreement. The remaining change primarily related to payments of interest and operating costs and expenses.

Cash, cash equivalents and marketable securities, excluding restricted cash decreased by $298.8 million when compared to balance as of June 30, 2021, which was $898.4 million. During the quarter, the Company repurchased $143.1 million of BridgeBio common stock and paid $35.0 million of regulatory-related milestone payments in connection with its approved products.  

Operating Costs and Expenses

Operating costs and expenses for the quarter increased by $23.7 million to $151.8 million in the current quarter as compared to $128.1 million for the same period in the prior year. The increase in operating costs and expenses was due to an increase in personnel and external costs to support the progression in BridgeBio’s research and development programs and staged buildout of its commercial organization as part of commercial launch readiness activities. This increase in personnel and external costs was offset by $12.2 million in reimbursement of expenses from the cost sharing arrangement recognized under BridgeBio’s License and Collaboration Agreement with Helsinn Group. Stock-based compensation for the quarter was $16.1 million as compared to $17.7 million for the same period in the prior year.

The Company’s research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While BridgeBio experienced some delays in certain of its clinical enrollment and trial commencement activities, it continues to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with its contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on BridgeBio’s operating costs and expenses is currently unknown.


(1) The condensed consolidated financial statements as of and for the year ended December 31, 2020 are derived from the audited consolidated financial statements as of that date.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic potential of our programs and product candidates, including our four core value drivers, the availability and success of topline results from Part A and Part B of our Phase 3 ATTRibute-CM trial of acoramidis, our plans to submit an application for regulatory approval of acoramidis, the availability and success of additional data from our ongoing study of encaleret for ADH1, the timing and success of additional trials of encaleret for ADH1, the availability and success of initial data from our ongoing Phase 2 study of low-dose infigratinib for achondroplasia and our ongoing Phase 1/2 study of BBP-631 for CAH, our faith in the long-term prospects of our pipeline, the success of our continuing partnership with LianBio, the timing of our selection of a RAS development candidate, the timing and success of our Phase 1/2 trial of BBP-812 for Canavan disease, the ability of BBP-812 to be the first approved therapeutic option for children born with Canavan disease, the ability of BBP-418 to be the first approved therapy for patients with LGMD2i, the timing and success of the Phase 2 trial of BBP-418 in patients with LGMD2i, the availability and success of final data from our ongoing Phase 1 study of BBP-711 for the treatment of PH1, the timing and success of additional clinical trials of BBP-711 in PH1 and in recurrent kidney stone formers, our eligibility to receive future royalty payments under our strategic collaboration with the Helsinn Group and the timing of these events, as well as our anticipated cash runway, reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, potential volatility in our share price and its impact on our 2021 Share Repurchase Program, potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, as well as those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2020, and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com 
(516) 554-5989

BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Canavan Disease

-If successful, BridgeBio’s gene therapy could be the first approved therapeutic option for children born with Canavan disease, a devastating and life-threatening condition

-The Canavan disease program is part of BridgeBio’s growing gene therapy portfolio, which includes clinical candidates for congenital adrenal hyperplasia (CAH) and classic galactosemia (severe GALT deficiency), and preclinical programs for TMC1 hearing loss, tuberous sclerosis, cystinuria, and a genetic dilated cardiomyopathy

PALO ALTO, CA – November 3, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational adeno-associated virus (AAV) 9 gene therapy for the treatment of Canavan disease. Canavan disease is an ultra-rare and fatal disease that affects approximately 1,000 children in the United States and European Union. There are currently no approved therapies for the condition.

“Dosing the first patient in our Canavan disease trial is a significant achievement for our gene therapy team and we would not be here without the support of our collaborative partners in the patient, medical and scientific communities,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. “We are committed to the Canavan community and will work to advance this trial and the other programs in our gene therapy portfolio because we believe deeply in the potential of gene therapy to improve and save lives.”  

“Based on the efficacy and safety data we’ve observed in our preclinical studies, we are hopeful that our investigational gene therapy can become a meaningful treatment option for children living with Canavan. Right now, these children only have access to supportive care because there are currently no approved therapies to treat this devastating disease,” added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.

The Phase 1/2 open-label study is designed to evaluate the safety, tolerability, and pharmacodynamic activity of the company’s AAV9 gene therapy, BBP-812, in pediatric patients with Canavan disease. In the initial dose-finding phase of the study, each patient will receive a single intravenous (IV) infusion of BBP-812. The primary outcomes of the study are safety, as well as change from baseline of urine and central nervous system N-acetylaspartate (NAA) levels. Motor function and development will also be assessed. Preclinical proof-of-concept data have shown the approach restores survival and normal motor function.

“Gene therapy is designed to treat diseases at their source, which for Canavan disease would be an extremely beneficial treatment option. Through this trial, we hope to provide evidence that this investigational therapy could represent a promising treatment option for Canavan patients and their families. We are grateful to the first family for participating,” said Florian Eichler, M.D., director of the Leukodystrophy Service and principal investigator at Massachusetts General Hospital, the first clinical site open in the CANaspire trial.

BridgeBio’s gene therapy was originally developed by Guangping Gao, Ph.D., and Dominic J. Gessler, M.D., Ph.D., at the University of Massachusetts Medical School. Dr. Gao, a pioneer in AAV gene therapy, was also the first person to clone the ASPA gene, which in its mutated form causes Canavan disease. Dr. Gao has been working on developing a cure for Canavan disease for more than 25 years.

“Throughout my career, I’ve been passionate about connecting with families in the Canavan community to understand how to help them through their journey. After decades of research and work, I’m thrilled that we have dosed the first patient in this trial. I’m hopeful that this represents a meaningful turning point for the Canavan community,” said Dr. Gao, co-director of the Li Weibo Institute for Rare Diseases Research, director of the Horae Gene Therapy Center and Viral Vector Core, and professor at University of Massachusetts Medical School.

BridgeBio’s investigational AAV9 gene therapy for Canavan disease is one of the Company’s 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and genetically-driven cancers. An initial Phase 1/2 data readout for Canavan disease is expected in 2022.  For more information about the CANaspire trial, visit TreatCanavan.com or ClinicalTrials.gov (NCT04998396).

About BBP-812

BBP-812 is an investigational AAV9 gene therapy for Canavan disease. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, potentially correcting the disease at its source. Preclinical proof-of-concept results in Canavan disease models have shown the approach restores survival and normal motor function.  BBP-812 was granted Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation by the U.S. Food and Drug Administration. BBP-812 was also granted Orphan Drug Designation by the European Medicines Agency.

About Canavan Disease

Affecting approximately 1,000 children in the United States and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and pass away at a young age. The disease is caused by an inherited mutation of the ASPA gene, which codes for aspartoacylase, a protein that breaks down a compound called N-acetyl-L-aspartic acid (NAA). Deficiency of aspartoacylase activity results in accumulation of NAA, and ultimately results in toxicity to myelin in ways that are not well understood. Myelin insulates the nerves, and without it, neurons are unable to send and receive messages as they should. The current standard of care is limited to supportive therapy.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials, and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to the timing and success of BridgeBio’s Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, expectations, plans and prospects regarding BridgeBio’s regulatory approval process for BBP-812, the ability of BBP-812 to treat Canavan disease in humans, and the timing and success of initial top-line Phase 1/2 date of BBP-812, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio’s ability to continue and complete its Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, past data from preclinical studies not being indicative of future data from clinical trials, BridgeBio’s ability to advance BBP-812 in clinical development according to its plans, the ability of BBP-812 to treat Canavan disease, the ability of BBP-812 to retain Fast Track Designation, Rare Pediatric Drug Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; as well as those set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SEC’s website at www.sec.gov. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com
(516) 554-5989

BridgeBio Pharma Announces Collaborations with Columbia University and Mount Sinai to Develop Potential Therapies for Genetic Diseases and Cancers

– The new agreements mean BridgeBio has collaborations with 25 leading academic institutions focused on genetic disease and precision oncology

PALO ALTO, CA – October 29, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced two new academic collaborations with Columbia University and Mount Sinai (Icahn Mount Sinai) to translate cutting-edge research discoveries into potential therapies for patients with genetic diseases and genetically driven cancers.

“Columbia University and Mount Sinai are known for bringing together some of the most talented scientists to develop breakthroughs for patients. By partnering with these world-class research institutions, we are hopeful that together we will be able to help patients in need,” said BridgeBio founder and CEO Neil Kumar, Ph.D.

BridgeBio has initiated 25 collaborations with leading institutions around the world that are focused on providing therapeutic options to patients with unmet need as quickly and safely as possible. To learn more about some of the institutions BridgeBio is proud to partner with, please visit Our Partners page.

Collaborating with academic institutions to identify early discoveries is a core pillar of BridgeBio’s efforts to reach patients more quickly. The goal of these collaborations is to revolutionize the relationships between drug development companies and biomedical research institutions by moving away from one-off interactions in favor of engaging and creative partnerships.

More than two-thirds of BridgeBio’s 30+ pipeline programs have come from partnerships with academic institutions and research centers. For example, BridgeBio’s clinical trial of encaleret, which is being investigated for the treatment of autosomal dominant hypocalcemia type 1 (ADH1), has been enabled by a Cooperative Research and Development Agreement with the National Institute for Dental and Craniofacial Research at the National Institutes of Health. BridgeBio’s investigational medicine acoramidis, which is being developed for the treatment of transthyretin (TTR) amyloidosis (ATTR), originated in a lab at Stanford University. BridgeBio partnered with the Stanford researchers and advanced acoramidis from the lab to Phase 3 clinical development in less than three years.

With a diverse pipeline encompassing investigational therapies in Mendelian diseases, precision cardiorenal, precision oncology and gene therapy, BridgeBio provides the insights and support needed to rapidly progress therapeutic research from labs to clinical development. BridgeBio intends to develop similar long-term partnerships based on trust, engagement, science and respect to support its mission of developing potentially life-changing medicines for patients with genetic diseases and cancers as quickly and safely as possible.

Columbia University

Columbia University has built a research team of translational-focused investigators working on genetic diseases. It also has a technology transfer office that provides mentorship for researchers and funds the early stages of their translational work. BridgeBio intends to collaborate with Columbia University to identify and potentially develop promising therapies for patients with genetic diseases.

“Each year, Columbia researchers produce approximately 400 promising early-stage innovations, which lead to over 100 license agreements and 20 to 30 new startup companies based on Columbia intellectual property,” said Orin Herskowitz, executive director of Columbia Technology Ventures, senior vice president for Intellectual Property & Technology Transfer and adjunct professor of business and engineering at Columbia University. “We are excited to work closely with BridgeBio to identify which of these promising innovations have potential to fit into BridgeBio’s areas of expertise and benefit from their experience. By doing so, we hope to accelerate the invention’s path from the lab to the market and get therapies as quickly as possible into patients who are suffering from genetic diseases.”

Mount Sinai Health System

The Mount Sinai Health System is New York City’s largest academic medical system, encompassing eight hospitals, a leading medical and graduate school, and a vast network of ambulatory practices throughout the greater New York region. In particular, the Icahn School of Medicine at Mount Sinai is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report’s “Best Medical Schools,” aligned with a U.S. News & World Report “Honor Roll” Hospital, and No. 14 in the nation for National Institutes of Health funding. The partnership between BridgeBio and the Icahn School of Medicine at Mount Sinai will focus specifically on genetic disease and precision oncology to determine new potential therapies that may be meaningful options for patients in need.

“We look forward to collaborating with BridgeBio, and to the opportunity to combine their expertise in drug development with our exceptional scientific teams at Mount Sinai to advance breakthrough genetic disease therapies for patients,” said Erik Lium, Ph.D., president of Mount Sinai Innovation Partners and executive vice president and chief commercial innovation officer of Mount Sinai Health System.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s first two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding our partnering with Columbia University and Mount Sinai, the success of current and future relationships with third-party collaborators and academic partners, and the potential ability of our product candidates to treat genetically driven diseases and cancers with clear genetic drivers, reflect our current views about our plans, intentions, expectations, strategies and prospects, and are based on the information currently available to us and on assumptions we have made and are not forecasts, promises nor guarantees. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by these forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of our product candidates to treat genetically driven diseases and cancers with clear genetic drivers, the success of our academic collaborations with Columbia University and Mount Sinai, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s most recent Annual Report on Form 10-K and BridgeBio Pharma’s other SEC filings. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com
(516) 554-5989

BridgeBio Pharma, Inc. Appoints Pioneering National Leader in Genomics and Workforce Diversity Hannah Valantine to its Board of Directors

PALO ALTO, CA – October 25, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that it has added a new independent director to its board: Hannah Valantine, M.D., a national leader in organ transplant genomics who led the National Institutes of Health’s efforts to promote diversity, equity, and inclusion in biomedical research. Dr. Valantine currently serves as a professor of medicine at Stanford University School of Medicine, where she has been a faculty member since 1987. Dr. Valantine was elected to the National Academy of Medicine in 2020 for her research in organ transplantation and her work to promote workforce diversity.

“From her groundbreaking discovery of a new technology to help transplant patients, to her meaningful efforts to support and encourage diversity within biomedical sciences, Dr. Valantine’s impact on our industry is far-reaching,” said BridgeBio founder and CEO Neil Kumar, Ph.D. “We are proud to have Dr. Valantine join our board and we feel privileged to have the opportunity to learn from her as we work to build the next great genetic medicine company together.”

At the National Institutes of Health, Dr. Valantine served as the inaugural chief officer for scientific workforce diversity, and as a senior investigator in the intramural research program at the National Heart, Lung, and Blood Institute from 2014 to 2020. Prior to that, she was a professor of cardiovascular medicine and the senior associate dean for diversity and leadership at Stanford.

In collaboration with her colleagues at Stanford, Dr. Valantine co-invented the technology for organ donor derived cell-free DNA for diagnosis of transplant rejection, which is currently licensed and used to monitor patients for early detection of acute organ rejection. Dr. Valantine also serves as the principal and founder of HAV LLC, a consulting company for diversity, equity and inclusion that she founded in January 2021. Dr. Valantine serves on the board of directors of Pacific Biosciences of California, Inc. and of CareDx, Inc. Dr. Valantine received her M.B.B.S., M.R.C.P. and M.D. at St. Georges Hospital/London University.

“As someone who has been focused throughout my career on improving patient outcomes, I am impressed and inspired by BridgeBio’s commitment to helping people living with genetic diseases and cancers. I look forward to supporting the company in its efforts to develop meaningful medicines for patients around the world, and fostering an environment where diverse perspectives are heard and harnessed as we advance promising biomedical research for patients in need,” said Dr. Valantine.

About BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com 
(516) 554-5989

BridgeBio Pharma Announces Progress in its KRAS Portfolio, New Gene Therapy Programs, and Updates on Advancements Across its R&D Pipeline Targeting Genetic Diseases and Cancers

PALO ALTO, Calif., Oct. 12, 2021 /PRNewswire/ — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, is announcing meaningful progress in its KRAS cancer portfolio, new programs in gene therapy, and advancements in cardiorenal and early-stage Mendelian programs at its second annual R&D Day today. BridgeBio will also discuss how it is broadening the scope of its R&D engine with the launch of its new early-stage research institute, BridgeBioX.

BridgeBio’s R&D Day will feature presentations by Neil Kumar, Ph.D., BridgeBio founder and CEO; Richard Scheller, Ph.D., chairman of R&D at BridgeBio; Charles Homcy, M.D., chairman of pharmaceuticals at BridgeBio; Uma Sinha, Ph.D., chief scientific officer at BridgeBio; and scientists and physicians leading BridgeBio’s drug discovery and development programs.

BridgeBio has more than 30 programs in its pipeline for patients living with genetic diseases and genetically-driven cancers. Fourteen of those programs are being advanced in the clinic or commercial setting, and earlier this year BridgeBio received its first two U.S. Food and Drug Administration (FDA) drug approvals.

R&D Day pipeline news and updates:

BridgeBio Precision Oncology

  • KRAS inhibitors for KRAS cancers: BridgeBio announces its discovery of next-generation G12C dual inhibitors, the first-known compounds that directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations driven by insights from its molecular dynamics platform. This unique mechanism of action (MOA) is differentiated in preclinical models from first generation compounds, which only bind inactive KRAS. RAS is one of the most well-known oncogenic drivers with approximately 30% of all cancers being driven by RAS mutations, including large proportions of lung, colorectal and pancreatic tumors.
  • In preclinical models, BridgeBio compounds showed rapid and complete modification of active (GTP bound) KRAS, which is not observed with first generation compounds. BridgeBio compounds were shown to be >500 fold more potent in inhibiting KRAS:RAF effector binding and more potent at inhibiting downstream signaling than first generation inhibitors.
  • In cellular resistance models, BridgeBio’s dual KRAS inhibitors were shown to be >35x more potent at blocking the emergence of resistance clones than first generation inhibitors, suggesting the potential for more durable efficacy in the clinic.
  • PI3Ka:RAS breaker: The company will discuss the discovery of multiple PI3Ka:RAS breakers, a potential therapeutic approach developed to block RAS driven PI3Kα activation with the potential to avoid adverse effects on glucose metabolism that limit the potential of PI3Ka kinase inhibitors.
  • BridgeBio will also announce its novel G12D inhibitor research program.
  • BridgeBio expects to select a RAS development candidate in 2022.

BridgeBio Gene Therapy

  • BBP-818 – New adeno-associated virus (AAV) gene therapy program for classic galactosemia (severe GALT deficiency): Classic galactosemia, which is caused by a severe deficiency of the enzyme galactose-1-phosphate uridylyltransferase (GALT), affects approximately 7,000 patients in the United States and the European Union. Studies in more than 500 patients with galactosemia have shown that despite early detection and strict adherence to diet, children with ≤1% of GALT enzyme levels may experience language delay, speech defects, learning disabilities, cognitive impairment, osteopenia, and in females, primary ovarian insufficiency.
  • BBP-818 is designed to enable production of the GALT enzyme and to enable the body’s natural ability to metabolize galactose. Preclinical studies in a mouse model of classic galactosemia have shown that BridgeBio’s BBP-818 therapy restored up to 72% of wild-type levels of GALT enzyme in the brain following a single dose.
  • In addition to the GALT program, BridgeBio Gene Therapy is advancing clinical candidates for Canavan disease and congenital adrenal hyperplasia (CAH), and a preclinical program for TMC1 hearing loss. BridgeBio will also announce at R&D Day preclinical gene therapy programs targeting tuberous sclerosis, cystinuria, and a genetic dilated cardiomyopathy, as well as collaborations to identify and characterize next generation capsids with tropism for the central nervous system and kidney.

BridgeBio Cardiorenal

  • BBP-711 – Glycolate oxidase (GO) inhibitor for hyperoxaluria: BridgeBio will share preliminary Phase 1 data in which BBP-711 was well-tolerated and resulted in maximal increases in plasma glycolate exceeding those achieved by any GO-targeting agents reported in healthy adult volunteers. At steady state in multiple ascending dose cohorts, BBP-711 treatment resulted in plasma glycolate concentrations comparable to case reports of individuals with germline HAO1 knockout, the gene encoding GO, suggesting complete inhibition.
  • BBP-711 is being developed for the treatment of primary hyperoxaluria type 1 (PH1) and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers. A full readout of Phase 1 data in healthy adult volunteers is expected in 2022, to be followed by initiation of a Phase 2/3 trial in PH1 and a Phase 2 proof-of-concept trial in recurrent kidney stone formers.
  • Acoramidis (AG10) – TTR stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): BridgeBio will cover the company’s most significant near-term catalysts with a focus on upcoming topline results for acoramidis. Topline results from Part A of the Phase 3 ATTRibute-CM trial are expected in late 2021 and from Part B in 2023. The primary endpoint at Part A is the change from baseline in a 6-minute walk distance (6MWD) in trial participants receiving acoramidis or placebo after 12 months. If the change from baseline in 6MWD in Part A is highly statistically significant, BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA. ATTR is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.
  • Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): BridgeBio will review updated Phase 2b data for encaleret, which was originally shared in an oral presentation at the American Society of Bone and Mineral Research (ASBMR) 2021 Annual Meeting. Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion. Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of current standard-of-care. Encaleret could be the first approved therapy for ADH1, a condition caused by gain of function variants in the CaSR gene estimated to be carried by 12,000 individuals in the United States alone. BridgeBio plans to engage with regulatory health authorities to discuss the design of a Phase 3 registrational trial in patients with ADH1.

BridgeBioX

  • BridgeBio will announce BridgeBioX, the company’s new early research discovery engine with a dedicated lab at Stanford University. BridgeBio created BridgeBioX to test earlier scientific hypotheses in discovery research and target large, complex genetic diseases with high unmet need. BridgeBio’s creation comes with cutting edge tools in genetics and molecular biology, along with expanded capabilities across modalities with the goal of advancing therapies rapidly. The research lab was created to foster collaboration between industry and academia, and to build a culture driven by intellectual curiosity and a dedication to patient impact.

BridgeBio Mendelian

  • BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i): BridgeBio will review Phase 1 data in which BBP-418 was shown to be well-tolerated in healthy volunteers. The Phase 2 trial was initiated in patients with LGMD2i in the first quarter of 2021. With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein. A Phase 2 data readout is expected in 2022.
  • BBP-589 – Recombinant collagen for recessive dystrophic epidermolysis bullosa (RDEB): BridgeBio will discuss BBP-589, the only potential systemic treatment option being developed for patients with RDEB. BBP-589 was previously shown to be well-tolerated in Phase 1 with dose-dependent increase in C7 skin deposition. RDEB is a Mendelian disease that has devastating effects on patients due to the epidermis separating from the dermis and causes blistering, tearing and scarring of the skin, along with severe pain and itching. In the United States, RDEB has a prevalence of approximately 2,000 patients. Phase 2 data are expected in the first half of 2022.

BridgeBio’s R&D Day will be held today from 8:30 am ET – 11:00 am ET and it will be webcast, with a link available in the event calendar on BridgeBio’s investor website, https://investor.bridgebio.com/. A replay of the webcast will be available for one year following the event.

To register for BridgeBio’s R&D Day, please sign up here.

Agenda:

  • Welcome and introduction – Grace Rauh, vice president of marketing and communications, BridgeBio
  • Genetic basis of disease – Richard Scheller, Ph.D., chairman of R&D, BridgeBio
  • BridgeBio’s endless summer – Neil Kumar, Ph.D., founder and CEO, BridgeBio
  • Precision cardiorenal introduction – Cameron Turtle, D.Phil., chief strategy officer, BridgeBio
  • Acoramidis: TTR stabilizer for ATTR – Jonathan Fox, M.D., Ph.D., chief medical officer, BridgeBio Cardiorenal
  • Encaleret: Calcium sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1) – Mary Scott Roberts, M.D., senior director of clinical development, BridgeBio Cardiorenal
  • Gene therapy platform – Eric David, M.D., J.D., CEO, BridgeBio Gene Therapy
  • Mendelian programs: Primary hyperoxaluria type 1 (PH1), limb-girdle muscular dystrophy type 2i (LGMD2i), recessive dystrophic epidermolysis bullosa (rDEB) – Uma Sinha, Ph.D., chief scientific officer, BridgeBio
  • Precision oncology programs: KRAS, SHP2 – Eli Wallace, Ph.D., chief scientific officer, BridgeBio Oncology
  • BridgeBioX – Charles Homcy, M.D., chairman of pharmaceuticals, BridgeBio
  • Q&A

About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of over 30 development programs ranges from early science to advanced clinical trials and its commercial organization is focused on delivering the company’s two approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include statements relating to expectations, plans and prospects regarding the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of our product candidates, including, but not limited to: the unknown future impact of the COVID-19 pandemic delay on our ongoing development and/or our operations or operating expenses; the potential for our next-generation G12C dual inhibitors to be the first known compounds designed to directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations driven by insights from its molecular dynamics platform; the potential of our precision oncology program and the timing of our selection of a RAS development candidate; the potential of BBP-818 to enable production of the GALT enzyme and to enable the body’s natural ability to metabolize galactose in clinical trials; the potential and success of our Gene Therapy platform; the timing and success of BBP-711 for the treatment of primary hyperoxaluria type 1 and hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers; the timing and success of acoramidis; the timing and success of our regulatory strategy for acoramidis; the timing and success of our planned meetings with regulatory health authorities, including the U.S. Food and Drug Administration (FDA), to discuss potential paths to registration prior to initiation of a Phase 3 registrational study of encaleret in patients with ADH1; the ability of encaleret to be the first approved therapy option indicated specifically for the treatment of ADH1; the success of BridgeBioX to test earlier scientific hypotheses in discovery research and target large, complex genetic diseases with high unmet need; the continuing success of our partnership with Stanford University; the timing and success of our Phase 2 trial of BBP-418; the potential for BBP-589 to be the only potential systemic treatment option being developed for patients with RDEB; reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to: initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data; the potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated; the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales; despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; the continuing success of our collaborations; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those risks set forth in the Risk Factors section of our most recent annual report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and our other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Media Contact:
Grace Rauh
Grace.rauh@bridgebio.com
(917) 232-5478

BridgeBio Investor Contact:
Katherine Yau
katherine.yau@bridgebio.com 
(516) 554-5989