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

BridgeBio Pharma Announces First Publication of Preclinical Data for its Potentially Best-in-Class SHP2 Inhibitor Designed for Treatment of Resistant Cancer, Showing Response in Established Non-small Cell Lung Cancer Models

-Preclinical findings that demonstrated efficacy in non-small cell lung cancer (NSCLC) driven by RAS or other MAPK-pathway activating mutations will be presented at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

-BridgeBio’s SHP2 inhibitor, BBP-398, is part of its growing precision oncology portfolio, which also includes KRAS inhibitors for KRAS cancers, GPX4 inhibitors for multiple tumors and a FGFR1-3 inhibitor for FGFR2 and FGFR3 positive cancers

-Learn more at BridgeBio’s R&D Day on Oct. 12 at 8:30 am ET

PALO ALTO, CA – October 7, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, today announced preclinical findings for its SHP2 inhibitor, BBP-398, in non-small cell lung cancer (NSCLC). The results are featured in a poster presentation titled ‘BBP-398, a potent, small molecule inhibitor of SHP2, enhances the response of established NSCLC xenografts to KRASG12C and EGFRmut inhibitors’ at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics taking place virtually on October 7 – 10, 2021.

“We are excited to share our promising preclinical data in non-small cell lung cancer models, which provides a critical step in understanding the potential that BBP-398 has for patients with tumors driven by RAS or other MAPK-pathway activating mutations,” said Eli Wallace, Ph.D., chief scientific officer at BridgeBio Oncology. “For patients with this type of progressive cancer, there is a serious need for more innovative medicines to be accessible as quickly as possible. Our potentially best-in-class SHP2 inhibitor could be an ideal combination agent for certain cancer patients given its promising profile of preclinical results and potential for once daily dosing.”

BBP-398, developed in collaboration with the University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division, exhibits preclinical monotherapy efficacy in RTK/KRAS-driven xenograft models as well as synergy in combination with both sotorasib and osimertinib. The predicted human steady-state plasma concentration-time profiles suggest continuous once daily oral dosing of BBP-398 may achieve the desired therapeutic index for patients.

BridgeBio is currently advancing its Phase 1 dose escalation clinical trial with 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. More than 30% of all human cancers – including 95% of pancreatic cancers and 45% of colorectal cancers — are driven by mutations of the RAS family of genes.

BridgeBio’s precision oncology programs are driven by the Company’s molecular dynamics and RAS structural biology platforms, which are enabled by our broad partnerships with the Lawrence Livermore National Laboratory and National RAS Initiative, respectively.

BridgeBio’s SHP2 inhibitor, BBP-398, 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.

Learn more about the preclinical data for BBP-398 at BridgeBio’s upcoming virtual R&D Day on Tuesday, October 12, 2021 at 8:30 am ET. The event will be webcast and registration information can be found here.

BridgeBio will unveil new programs, share new information about its pipeline and discuss how it is broadening the scope of its R&D engine. It will also cover the Company’s most significant near-term catalysts with a focus on the upcoming topline results for acoramidis, BridgeBio’s investigational therapy for transthyretin (TTR) amyloidosis (ATTR). ATTR is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.

Topline acoramidis results from Part A 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 U.S. Food and Drug Administration.

The R&D Day program importantly and additionally will highlight BridgeBio’s broader efforts in cardiorenal, progress in its KRAS portfolio, and advancements in its previously disclosed early-stage Mendelian programs. The Company will also be unveiling new investigational programs in gene therapy.

About SHP2

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. Overactivity of the SHP2 pathway, often driven by distinct genetic mutations, is a critical contributor to many forms of cancer, and is a mechanism of resistance to several targeted therapies. Estimated to affect approximately 500,000 patients in the United States and the European Union, cancers that are driven by hyperactive MAPK signaling, including certain RAS mutations such as KRASG12C, may be sensitive to SHP2 inhibition.

About BBP-398

BBP-398 is a potent, selective, orally bioavailable SHP2 inhibitor that demonstrates pathway inhibition across a panel of cell lines with active MAPK signaling. The therapy is designed to be optimized for continuous once daily dosing through its pharmacokinetic profile. The inhibitor was developed through a collaboration with the University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. BridgeBio has 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. The collaboration will also include the initiation of a Phase 1/2 study to evaluate the safety and preliminary efficacy of BBP-398 in combination with both OPDIVO as doublet therapy, and OPDIVO plus a KRASG12C inhibitor as triplet therapy in non-small cell lung cancer (NSCLC) with KRAS mutations, as first- and second-line treatment options. Additionally, BridgeBio previously 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 and pancreatic cancer, in mainland China and other major Asian markets.

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, including statements relating to expectations, plans, and prospects regarding preclinical study results for our SHP2 inhibitor, BBP-398, being indicative of future clinical trial results, the potential for BBP-398 to be a best-in-class SHP2 inhibitor as a once daily dose to treat patients with tumors driven by RAS or other MAPK-pathway activating mutations, the continuing success of our collaboration with the University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division, the timing and success of a Phase 1 dose escalation clinical trial of BBP-398 in patients with solid tumors driven by mutations in the MAPK signaling pathway, including RAS and receptor tyrosine kinase genes , the ability of our SHP2 inhibitor’s ability to enhance immuno-oncology and other targeted therapies to potentially provide options for patients with difficult-to-treat cancers as quickly and safely as possible, the incidence of KRAS mutations and the promise of targeted therapies for patients with such mutations, 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 collaboration with the University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division and preclinical study results being indicative of future clinical trial results 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.

Maurer, M., Schwartz, J., Gundapaneni, B., et al. “Tafamidis treatment for patients with transthyretin Amyloid cardiomyopathy”. New England Journal of Medicine 379.11 (2018): 1007–16.

2Enright, M., Duanel, S. “Reference equations for the six-minute walk in healthy adults”. American Journal of Respiratory and Critical Care Medicine 158.5 (1998): 1384–7.

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 to Host Virtual R&D Day on October 12, 2021

PALO ALTO, CA – October 6, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, today announced that it will host its second R&D Day on Tuesday, October 12, 2021, at 8:30 a.m. ET.

BridgeBio founder and CEO Neil Kumar, Ph.D., will present and be joined by Richard Scheller, Ph.D., chairman of R&D at BridgeBio; Charles Homcy, M.D., chairman of pharmaceuticals at BridgeBio; and Uma Sinha, Ph.D., chief scientific officer at BridgeBio, along with senior scientists and physicians leading BridgeBio’s drug discovery and development programs. BridgeBio has 14 programs that are being advanced in the clinic or commercial setting with 30+ programs total in its pipeline for patients living with genetic diseases and genetically-driven cancers.

BridgeBio will unveil new programs, share new information about its pipeline and discuss how it is broadening the scope of its R&D engine. It will also cover the company’s most significant near-term catalysts with a focus on the upcoming topline results for acoramidis, BridgeBio’s investigational therapy for transthyretin (TTR) amyloidosis (ATTR). ATTR is a rare heart condition with a progressive and debilitating impact on quality of life likely affecting more than 400,000 patients worldwide.

Topline results from Part A 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 U.S. Food and Drug Administration.

The R&D Day program importantly and additionally will highlight BridgeBio’s broader efforts in cardiorenal, progress in its KRAS portfolio, and advancements in its previously disclosed early-stage Mendelian programs. The Company will also be unveiling new programs in gene therapy.

Agenda:

  • Welcome and introduction – Grace Rauh, vice president of marketing and communications, BridgeBio Pharma
  • Genetic basis of disease – Richard Scheller, Ph.D., chairman of R&D, BridgeBio Pharma
  • BridgeBio’s endless summer – Neil Kumar, Ph.D., founder and CEO, BridgeBio Pharma
  • Precision cardiorenal introduction – Cameron Turtle, D.Phil., chief strategy officer, BridgeBio Pharma
  • 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 Pharma
  • Precision oncology programs: KRAS, SHP2 – Eli Wallace, Ph.D., chief scientific officer, BridgeBio Oncology
  • BridgeBioX – Charles Homcy, M.D., chairman of pharmaceuticals, BridgeBio Pharma
  • Q&A

The event 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. To view the agenda and speaker profiles visit our R&D Day webpage. Attendees must register and watch the webcast to participate in the Q&A sessions.

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 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 Updated Phase 2B Data for Encaleret in Autosomal Dominant Hypocalcemia Type 1 (ADH1) Demonstrating Blood and Urine Calcium Normalization in Trial Participants

– Data presented at American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting

– 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 indicated specifically for the treatment of ADH1 if the development program is successful

PALO ALTO, Calif., Oct. 1, 2021 /PRNewswire/ — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced updated results from its ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1). Within five days of individualized dose titration in 13 participants, encaleret normalized mean blood calcium levels and 24 hour urine calcium excretion. The results are featured in an oral presentation titled ‘The Effects of Encaleret (CLTX-305) on Mineral Physiology in Autosomal Dominant Hypocalcemia Type 1 (ADH1) Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study’ at the American Society for Bone and Mineral Research (ASBMR) 2021 Annual Meeting, taking place in San Diego, California on October 1 – 4, 2021.

“Autosomal dominant hypocalcemia type 1 is a rare genetic form of hypoparathyroidism caused by gain-of-function variants of the calcium-sensing receptor (CASR) gene. The current standard of care consists of calcium and active vitamin D supplements, which do not address the root cause of ADH1,” said Rachel Gafni, M.D., Senior Research Physician and Head, Mineral Homeostasis Studies Group of the National Institute of Dental and Craniofacial Research, National Institutes of Health (NIH). “These updated results from the ongoing Phase 2b study of encaleret demonstrate consistent improvements in mineral homeostasis and support further study.”

In this update from the ongoing Phase 2b open-label, dose-ranging study, 13 adults with ADH1 with nine distinct CASR variants were administered encaleret. Calcitriol (active Vitamin D) and extra-dietary calcium supplementation beyond the recommended daily intake (current standard of care) were discontinued during the study.

Through the inpatient observation periods of defined dose escalation and individualized dose titration, encaleret was well-tolerated with no serious adverse events, no adverse events of severe intensity, or treatment discontinuation due to adverse events reported. Across 13 trial participants, encaleret normalized mean blood calcium levels and 24-hour urine calcium excretion during Periods 1 and 2. Parathyroid hormone levels increased in all participants and mean blood phosphate decreased into the normal range during Periods 1 and 2. The tolerability and consistent mineral responses following encaleret administration demonstrate that encaleret may become an efficacious therapy option for patients with ADH1.

“As a direct modulator of the calcium-sensing receptor’s (CaSR) sensitivity to calcium, encaleret is designed to target this genetic disease at its source, which is gain-of-function (increased sensitivity to calcium) variants in the receptor. The results we are achieving with encaleret for autosomal dominant hypocalcemia type 1 in the current clinical trial are remarkable, as both blood and urine calcium normalize within five days of dosing. Given the consistent improvements seen in mineral homeostasis, we are excited about encaleret’s potential to help patients with ADH1 who currently have no approved therapy indicated to treat this disease,” said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of the cardiorenal companies at BridgeBio. “We recognize the magnitude of the unmet need for these patients and are working collaboratively with regulators to define a path forward for registration. We look forward to sharing complete results next year from this ongoing study which is currently in an outpatient treatment phase.”

BridgeBio plans to engage with regulatory health authorities to discuss the design of a Phase 3 registrational study in patients with ADH1. If the development program is successful, encaleret could be the first approved therapy indicated specifically for the treatment of ADH1.

At ASBMR 2021, BridgeBio will also present a retrospective systematic literature review of ADH1 and clinical study designs for its PROPEL and PROPEL2 studies of low-dose infigratinib in people with achondroplasia, which is the most common form of genetic short stature 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.

BridgeBio’s investigational therapies for ADH1 and achondroplasia are two of the company’s 14 programs that are being advanced in the clinic or commercial setting for patients living with genetic diseases and genetically driven cancers.

BridgeBio’s first wave of programs are the now-approved drugs for Molybdenum Cofactor Deficiency (MoCD) Type A and previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. The second wave of programs includes the Company’s four major near-term catalysts for its product candidates for the treatment of ADH1 and achondroplasia, as well as transthyretin (TTR) amyloidosis (ATTR) and congenital adrenal hyperplasia (CAH).

BridgeBio’s ongoing third wave in development includes a variety of programs in the cancer and mendelian space already in the clinic.

Learn more about the updated data for encaleret, low-dose infigratinib and the BridgeBio pipeline at the Company’s upcoming virtual R&D Day on Tuesday, October 12, 2021, from 8:30 – 11:30 am ET. The event will be webcast and registration information can be found here.

The ASBMR presentation for the updated Phase 2b data in encaleret can be found here.

About Encaleret

Encaleret is an investigational, orally-administered small molecule that selectively antagonizes the CaSR, targeting ADH1 at its source. The current standard-of-care for ADH1 patients consists of oral calcium and/or vitamin D supplements that are typically administered to manage signs and symptoms associated with hypocalcemia. Encaleret has received Fast Track and Orphan Drug Designations from the U.S. FDA.

About Autosomal Dominant Hypocalcemia Type 1 (ADH1)
ADH1 is caused by gain-of-function variants of CASR, which are estimated to be harbored by 12,000 individuals in the United States.1 This gene encodes the calcium-sensing receptor, CaSR, which senses and regulates the level of extracellular calcium in the body as measured in the blood through its effects on the parathyroid glands, the kidney, and bone. Due to increased sensitivity of the CaSR to extracellular calcium, patients with ADH1 have low blood calcium (hypocalcemia), inappropriately low parathyroid hormone levels, and excess urinary excretion of calcium (hypercalciuria). Hypocalcemia can cause severe muscle cramping and seizures, while hypercalciuria can lead to kidney calcifications and impaired kidney function.

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, including 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 clinical trials and/or our operations or operating expenses; updated results from our ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1) not being indicative of final data from our Phase 2b study of encaleret; the potential size of the target patient population with a rare genetic form of hypoparathyroidism caused by pathogenic variants in the calcium-sensing receptor (CASR) gene; the inability of current standard of care therapies to treat ADH1; encaleret continuing to be well-tolerated with no serious adverse events and no adverse events of moderate or severe intensity reported in our ongoing Phase 2b proof-of concept, open-label study; tolerability and consistent mineral responses following encaleret administration in all 13 ADH1 trial participants continuing to demonstrate proof-of-concept that encaleret may be an efficacious therapy option for ADH1; 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 in patients with ADH1; the ability of encaleret to be the first approved therapy option indicated specifically for the treatment of ADH1, if the development program is successful; the continuing close collaboration between world-leading experts in calcium homeostasis at the National Institute of Dental and Craniofacial Research at the National Institutes of Health and BridgeBio; the clinical study designs for our Phase 2b study of encaleret in ADH1; 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: ongoing data from our ongoing Phase 2b proof-of-concept, open-label study of encaleret for the treatment of ADH1 not being indicative of final data; the potential size of the target patient population for ADH1 not being as large as anticipated; encaleret not being well-tolerated, with serious adverse events and adverse events of moderate or severe intensity being reported in the final Phase 2b study data; encaleret not continuing to demonstrate that it may be an efficacious therapy option for ADH1 based on the final Phase 2b data; encaleret not being the first approved therapy option indicated specifically for the treatment of ADH1, if the development program is not successful or if a competing therapy option is approved; 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 prior to initiation of a Phase 3 registrational study of encaleret in patients with ADH1, the FDA 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 our close collaboration between the National Institute of Dental and Craniofacial Research at the National Institutes of Health; 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.

[1] Dershem et al., Amer Jour of Hum Genetics, 2020

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 ADH1 Patient Advocacy Contact:
Jocelyn Ashford
jocelyn.ashford@bridgebio.com
(650) 452-4199

Helsinn Group and BridgeBio Pharma’s Affiliate QED Therapeutics Announce Health Canada Conditional Approval of TRUSELTIQ™ (infigratinib) for Patients with Cholangiocarcinoma

– Health Canada Issues Conditional Approval of TRUSELTIQ under Project Orbis (September 27th, 2021)

LUGANO, Switzerland, and PALO ALTO, CA, September 29, 2021 – Helsinn Group and BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., today announced that Health Canada has 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.

An NOC/c is a form of market approval granted to a product on the basis of promising evidence of clinical effectiveness following review of the submission by Health Canada. Products authorized under Health Canada’s NOC/c policy are intended for the treatment, prevention or diagnosis of a serious, life-threatening or severely debilitating illness. They have demonstrated promising benefit, are of high quality and possess an acceptable safety profile based on a benefit/risk assessment. In addition, they either respond to a serious unmet medical need in Canada or have demonstrated a significant improvement in the benefit/risk profile over existing therapies. Health Canada has provided access to this product on the condition that sponsors carry out additional clinical trials to verify the anticipated benefit within an agreed upon time frame.

“This is an important next step in growing TRUSELTIQ’s global reach. We are pleased to have achieved this milestone for patients with previously-treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or other rearrangement,” said Riccardo Braglia, Helsinn Group Vice Chairman and CEO. “The conditional approvals from the U.S. FDA and Health Canada mark the beginning of our journey delivering this medicine to patients in need. We look forward to working to enter further markets in the months and years ahead and working closely with BridgeBio as we make strides to reach patients.”

“We are grateful for our first international approval and the opportunity to reach patients outside the United States who are searching for options to treat FGFR2-fusion-driven cholangiocarcinoma. Helsinn has an impressive track record of advancing and commercializing oncology therapies around the globe and we partnered with them earlier this year in the hope of reaching as many patients with FGFR-driven cancers as possible,” said BridgeBio CEO and Founder Neil Kumar, Ph.D. “We believe infigratinib may be able to treat other FGFR-driven conditions and we will continue to evaluate its safety and efficacy in urothelial carcinoma and other areas of unmet need.”

Under Project Orbis, an initiative of the FDA, Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies, TRUSELTIQ received accelerated approval from the U.S. Food and Drug Administration (FDA) in May 2021. An additional marketing application for infigratinib is currently under review in Australia.

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.

As part of this agreement, BridgeBio and Helsinn Group’s affiliate, Helsinn Therapeutics U.S., Inc., are jointly responsible for commercialization activities for TRUSELTIQ in the U.S. and will share U.S. profits and losses on an equal basis. Helsinn Group will have exclusive commercialization rights on infigratinib outside of the U.S., excluding China, Hong Kong and Macau. BridgeBio will be eligible for tiered royalties as a percentage of adjusted net sales, and payments totaling up to approximately $2.45 billion USD in the aggregate. Helsinn Group will fund the majority of ongoing and future research and development related to infigratinib in oncology. BridgeBio previously entered a strategic collaboration with LianBio for development and commercialization of infigratinib in oncology indications in China, Hong Kong and Macau.

About TRUSELTIQ™ (infigratinib)

TRUSELTIQ (infigratinib) is a small molecule kinase inhibitor that targets FGFR, which obtained accelerated approval by FDA and was conditionally approved by Health Canada for the treatment of adults with previously treated, unresectable locally advanced or metastatic CCA with a FGFR2 fusion or other rearrangement.

Prior to initiation of TRUSELTIQ therapy, FGFR2 fusion or rearrangement should be established using a validated test.

Clinical effectiveness of TRUSELTIQ is based on overall response rate (ORR) and duration of response (DoR) from a single-arm Phase 2 trial in patients with specific FGFR2 fusion or other rearrangements.

Infigratinib is not FDA- or Health Canada-approved for any other indication in the United States and Canada, and is not approved for use by any other health authority.

About Cholangiocarcinoma (CCA)

CCA represents an aggressive group of malignancies that form in the bile ducts. The incidence of this serious and fatal disease varies considerably worldwide. As the disease is usually asymptomatic at early-stages, CCA typically presents at diagnosis as locally advanced or metastatic disease with a poor prognosis. In this respect, the five-year survival rate for patients affected by metastatic CCA is 2%.Depending on the anatomical site of origin, CCAs are classified into two subtypes: intrahepatic (iCCA – 10% of total) and extrahepatic (eCCA – 90% of total) CCA. Approximately 10% to 16% of iCCA harbor FGFR2 genetic alterations.1, 2, 3

About Helsinn Group

Helsinn is a Swiss Biopharmaceutical Group with an innovative R&D pipeline in cancer supportive care and oncology therapeutics, strategically investing in a fully integrated targeted therapy structure to develop, manufacture and commercialize small molecules in precision medicine with higher market potential, thanks to a consolidated track record, a solid revenue stream in B2B and strong cash flow and cash position.

Helsinn is building market differentiation in B2C in the U.S. and China and is owned by a third-generation healthcare entrepreneurial family.

Since 1976, Helsinn has been improving the lives of patients, guided by core family values of respect, integrity and quality, through a unique integrated licensing business model, and by collaborating with success in about 190 countries with long-standing partners who share our values.

The Group’s pharmaceutical business (Helsinn Healthcare S.A.) is headquartered in Lugano, Switzerland with operating subsidiaries in the U.S. (Helsinn Therapeutics (U.S.) Inc.) and China (Helsinn Pharmaceuticals (Beijing) Co., Ltd.) which market products directly in these countries. The company has additional operating subsidiaries in Switzerland (Helsinn Advanced Synthesis S.A., an active pharmaceutical ingredient manufacturer) and Ireland (Helsinn Birex Pharmaceuticals Ltd., a drug product manufacturer).

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.

For more information, please visit helsinn.com and follow us on Twitter, LinkedIn and Vimeo.

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 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 co-commercialization by QED Therapeutics, Inc. (QED) and partner Helsinn Group (Helsinn) of TRUSELTIQ™ (infigratinib) for the treatment of patients with previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement in Canada; Helsinn’s exclusive commercialization rights outside of the United States and in Canada, excluding China, Hong Kong and Macau; the potential for infigratinib to treat a range of FGFR-driven conditions, including other cancers; the safety profile of TRUSELTIQ for the treatment of patients with FGFR2 fusion driven CCA, including the most common adverse reactions and drug interactions; plans for the supply, manufacturing and distribution of TRUSELTIQ; the incidence and survival rate of CCA; the current -approved TRUSELTIQ dosage and administration; the planned approval of TRUSELTIQ by foreign regulatory authorities and the necessary clinical trial results, and timing and completion of regulatory submissions related thereto; and the competitive environment and clinical and therapeutic potential of TRUSELTIQ; 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 variety of risks and factors that are beyond our control including, without limitation: the safety, tolerability and efficacy profile of TRUSELTIQ observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; despite having ongoing interactions with the FDA, Health Canada or other regulatory agencies, the FDA, Health Canada or such other regulatory agencies may not agree with QED’s regulatory approval strategies, components of QED’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data; the fact that accelerated approval of TRUSELTIQ was granted by Health Canada based on overall response rate and duration of response, and continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s); QED and/or Helsinn may encounter delays in meeting manufacturing or supply timelines or disruptions in their distribution plans for TRUSELTIQ; whether and when any regulatory submissions may be filed in various foreign jurisdictions and ultimately approved by foreign regulatory authorities; the continuing success of the BridgeBio and Helsinn global collaboration and licensing agreement and the co-commercialization efforts thereunder; Helsinn’s ability to commercialize TRUSELTIQ outside of the United States and in Canada, excluding China, Hong Kong and Macau; 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 Pharma, Inc.’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.  Except as required by law, each of BridgeBio and QED disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. Moreover, BridgeBio and QED operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on each of BridgeBio’s and QED’s current expectations, and speak only as of the date hereof.

References

1 Dhanasekaran, R., Hemming, A. W., Zendejas, I., George, T., Nelson, D. R., Soldevila-Pico, C., Firpi, R. J., Morelli, G., Clark, V., Cabrera, R. “Treatment outcomes and prognostic factors of intrahepatic cholangiocarcinoma”. Oncology Reports 29.4 (2013): 1259-1267.

2 Patel N, Benipal B. Incidence of cholangiocarcinoma in the USA from 2001 to 2015: a US cancer statistics analysis of 50 states. Cureus 2019; 11: e3962.

3 Cancer.net. Bile Duct Cancer (Cholangiocarcinoma): Statistics. https://www.cancer.net/cancer-types/bile-duct-cancer-cholangiocarcinoma/statistics (accessed Aug 30, 2021).

4 Javle M, Raychowdhury S, Kelley RK et al. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. The Lancet Gastroenterology & Hepatology Published Online August 3, 2021 https://doi.org/10.1016/S2468-1253(21)00196-5

TRUSELTIQ is a trademark of Helsinn Group.

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

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

BridgeBio Pharma Receives FDA Fast Track Designation for Investigational Therapy for the Treatment of Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)

– If successful, BridgeBio’s drug could be the first approved therapy for patients with LGMD2i

-BridgeBio’s investigational therapy for LGMD2i is one of more than 30 R&D programs in the company’s diverse pipeline targeting genetic diseases and genetically-driven cancers

-12 of BridgeBio’s programs are in the clinic and two have received FDA approval

PALO ALTO, CA, September 15, 2021–BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the United States Food and Drug Administration (FDA) granted Fast Track designation for the investigation of BBP-418 as a treatment option for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i). The FDA grants development programs Fast Track designation to help drive the development and expedite its review process for drugs being investigated to treat serious conditions and fill unmet medical needs. The FDA utilizes this program to provide patients access to important new drugs as early as possible. This is the fifth Fast Track designation for an investigational therapy that BridgeBio has received this year.

BridgeBio’s LGMD2i investigational therapy 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.

BridgeBio’s first wave of programs are the now-approved drugs for Molybdenum Cofactor Deficiency (MoCD) Type A and previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. The second wave of programs includes the Company’s four major near-term catalysts for its product candidates for the treatment of transthyretin (TTR) amyloidosis (ATTR), achondroplasia, congenital adrenal hyperplasia (CAH) and autosomal dominant hypocalcemia type 1 (ADH1).

LGMD2i represents one of the leading programs in BridgeBio’s ongoing third wave in development, which includes a variety of programs in the cancer and mendelian space already in the clinic.

With approximately 7,000 patients with potentially treatable mutations, LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is a critical enzyme that adds a specific sugar molecule to a muscle cell structural protein called alpha-dystroglycan (αDG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack a robust cushioning system that is provided by fully glycosylated αDG proteins. Pediatric and adult patients with LGMD2i most commonly present with upper and lower extremity (“limb”) and thoracic (“girdle”) dysfunction (“limb-girdle” pattern of weakness), and without treatment often develop additional severe clinical manifestations, including loss of independent ambulation, severe breathing issues which can require mechanical ventilation, cardiomyopathy and premature death.

“As of now, there are no approved treatment options for people born with Limb-girdle Muscular Dystrophy Type 2i. People living with this disease can lose their ability to perform routine daily activities, and ultimately may lose the ability to walk, need ventilatory support or face the risk of heart failure,” said Douglas Sproule, M.D., M.Sc., chief medical officer of ML Bio Solutions, Inc., the BridgeBio company developing BBP-418. “We are grateful the FDA has granted our program Fast Track designation based on the potential of our investigational therapy to treat this very serious condition. We are hopeful the designation will allow us to address this unmet medical need by allowing us to potentially deliver our medicine to patients more quickly.”

BBP-418 is being investigated as a treatment for LGMD2i. The investigational therapy is designed to overcome the enzymatic limitation of the defective FKRP enzyme by supplementing endogenous sugar molecules to glycosylate αDG and to improve muscle cell integrities, resulting in improved muscle strength and function for patients. Clinical trials to verify the safety and efficacy of BBP-418 are ongoing.

BBP-418 has received Orphan Drug Designation for the treatment of LGMD2i from the FDA and for LGMD from the European Medicines Agency. BridgeBio is currently advancing its Phase 2 clinical trial in subjects with a genetically confirmed diagnosis of LGMD2i. If the development program is successful, BBP-418 could be the first approved therapy for the treatment of patients with LGMD2i.

About Limb-girdle Muscular Dystrophy Type 2i

LGMD2i is a monogenic autosomal recessive disease caused by partial loss of function mutations in the FKRP gene, and these FKRP mutations impair glycosylation of α-DG, a protein associated with stabilizing muscle cells.  LGMD2i is a disease that has pediatric symptomatic onset with most individuals developing manifestations of disease between 5 and 18 years of age.  Clinical manifestations typically present as a skeletal myopathy affecting the lower and then upper limbs, which is commonly later accompanied by respiratory muscle and cardiac muscle involvement.  Patients who harbor a homozygous genotype typically develop disease manifestations during late childhood with progression to loss of independent ambulation (25%), assisted ventilation (5%), and cardiomyopathy (10%) in adulthood. Cardiomyopathy is progressive, with an annual loss of 0.4% of left ventricular ejection fraction (LVEF). Patients with heterozygous genotypes have an earlier childhood onset with a more severe clinical course, rapid loss of mobility by 20 years of age, more frequent cardiac involvement (25%), and eventual respiratory failure by 30 years of age in nearly all cases.

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 the timing and success of ML Bio Solutions’ clinical trials of BBP-418 for the treatment of LGMD2i, expectations, plans and prospects regarding ML Bio Solutions’ regulatory approval process for BBP-418, the ability of BBP-418 to treat LGMD2i in humans, the potential for BBP-418 to be the first approved therapy for the treatment of LGMD2i and the timing and success of BridgeBio’s clinical trials and development pipeline, 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, ML Bio Solutions’ ability to continue and complete its clinical trials of BBP-418 for the treatment of LDMD2i, past data from preclinical studies not being indicative of future data from clinical trials, ML Bio Solutions’ ability to advance BBP-418 in clinical development according to its plans, the ability of BBP-418 to be the first approved therapy for the treatment of patients with LGMD2i, BridgeBio’s ability to advance its clinical trials and development pipeline, the success of BridgeBio’s approved drugs, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s Annual Report on Form 10-K for the year ended December 31, 2020, and BridgeBio Pharma’s other SEC filings. Moreover, ML BioSolutions 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 Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)

Palo Alto, CA, September 8, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that on September 2, 2021, the compensation committee of BridgeBio’s board of directors granted 29 new employees restricted stock units for an aggregate of 61,210 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 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 approved therapy. 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.

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

BridgeBio Pharma and LianBio Announce First Patient Treated in Phase 2a Trial of Infigratinib in Patients with Gastric Cancer and Other Advanced Solid Tumors

Palo Alto, CA and Shanghai and Princeton, NJ – August 25, 2021 – LianBio, a biotechnology company dedicated to bringing paradigm-shifting medicines to patients in China and other major Asian markets, and BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced the first patient has been treated in a Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification and other advanced solid tumors with FGFR genomic alterations.

“Infigratinib is a potent and selective FGFR inhibitor that has demonstrated compelling clinical activity across multiple tumor types with FGFR alterations,” said Yizhe Wang, Ph.D., chief executive officer of LianBio. “Given the disproportionately high prevalence rate of gastric cancer in China, LianBio is pursuing a region-specific development strategy focused on this area of great unmet need. This study marks LianBio’s first trial initiation and demonstrates our continued progress in delivering potentially transformational medicines to patients in Asia.”

TRUSELTIQ™ (infigratinib) is an oral selective inhibitor of FGFR1-3 that is approved in the United States for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test. It is also being further evaluated in clinical trials based on demonstration of clinical activity in patients with advanced urothelial carcinoma with FGFR3 genomic alterations. LianBio in-licensed rights from BridgeBio for infigratinib for development and commercialization in Mainland China, Hong Kong and Macau.

The Phase 2a trial is a multicenter, open-label, single-arm study in China designed to evaluate the safety and efficacy of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with FGFR2 gene amplification and other advanced solid tumors with FGFR alterations. The primary endpoint is objective response rate (ORR). Secondary endpoints include duration of response, safety, disease control rate, progression-free survival and overall survival.

Preclinical data have demonstrated the potential infigratinib may have for patients with gastric cancer. These results, published in Cancer Discovery, demonstrated tumor regression in multiple in vivo FGFR2 amplified gastric models.

“We believe that infigratinib could have a meaningful impact for people living with gastric cancer as well as many other cancers with FGFR alterations, and are pleased LianBio is initiating this clinical trial in China where more therapeutic options are needed to match the growing diagnosis rate,” said BridgeBio founder and chief executive officer Neil Kumar, Ph.D. “On the heels of TRUSELTIQ recently obtaining accelerated approval in the United States, we are hopeful that this trial will yield pivotal results in another subset of cancer patients as we continue to build our portfolio of oncology indications with the aim of reaching as many people in need as possible.”

About TRUSELTIQ™ (infigratinib)

TRUSELTIQ (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of fibroblast growth factor receptor (FGFR) that received accelerated approval from the FDA in the United States for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. TRUSELTIQ targets the FGFR protein, blocking downstream activity. In clinical studies, TRUSELTIQ demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response. TRUSELTIQ is not FDA-approved for any other indication in the United States and is not approved for use by any other health authority, including any Chinese or other Asian health authority. It is currently being evaluated in clinical studies for first-line cholangiocarcinoma, urothelial carcinoma (bladder cancer), locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma, and other advanced solid tumors with FGFR genomic alterations.

About 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.

About LianBio

LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and other major Asian markets, through partnerships that provide access to  innovative therapeutic discoveries with a strong scientific basis and compelling clinical data. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a broad and clinically validated pipeline with the potential to impact patients with unmet medical needs. For more information, please visit www.lianbio.com.

About the LianBio and BridgeBio Pharma, Inc. Strategic Alliance

In August 2020, LianBio entered into a strategic alliance with BridgeBio, a commercial-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, to develop and commercialize BridgeBio’s programs in China and other major Asian markets. This strategic relationship initially focuses on two of BridgeBio’s targeted oncology drug candidates: FGFR inhibitor infigratinib, for the treatment of FGFR-driven tumors, and SHP2 inhibitor BBP-398, in development for tumors driven by MAPK pathway mutations. The agreement also provides LianBio with preferential future access in China and certain other major Asian markets to more than 20 drug development candidates currently owned or controlled by BridgeBio. This collaboration is designed to advance and accelerate BridgeBio’s programs in China and other major Asian markets, allowing BridgeBio and LianBio to potentially bring innovation to large numbers of patients with high unmet need.

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 the Phase 2a clinical trial of infigratinib in patients with locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with fibroblast growth factor receptor-2 (FGFR2) gene amplification, and other advanced solid tumors with FGFR genomic alterations; the planned approval of infigratinib by foreign regulatory authorities in China and the necessary clinical trial results, and timing and completion of regulatory submissions related thereto; and the competitive environment and clinical and therapeutic potential of infigratinib; 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 variety of risks and factors that are beyond our control including, without limitation: the safety, tolerability and efficacy profile of infigratinib observed to date may change adversely in ex-U.S. clinical trials, ongoing analyses of trial data or subsequent to commercialization; foreign 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 BridgeBio and LianBio strategic alliance; 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 Pharma, Inc.’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. Except as required by law, each of BridgeBio and QED disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. Moreover, BridgeBio and QED operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on each of BridgeBio’s and QED’s current expectations, and speak only as of the date hereof.

Guagnano, V., Kauffman, A., Wörle, S., et al. “FGFR Genetic Alterations Predict for Sensitivity to NVP-BGJ398, a Selective Pan-FGFR Inhibitor.” Cancer Discovery 2 (2012): 1118-1133.

Contacts:

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

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

LianBio Investor Contact:
Elizabeth Anderson, VP Communications and Investor Relations
(646) 655-8390
elizabeth.anderson@lianbio.com

LianBio Media Contact:
Tyler Gagnon, CanaleComm
(508) 904-9446
tyler.gagnon@canalecomm.com

BridgeBio Pharma, Inc. Appoints Finance and Pharmaceutical Leaders Fred Hassan, Andrea Ellis and Douglas Dachille to its Board of Directors

PALO ALTO, CA – August 18, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that it has added three new independent directors to its board: Fred Hassan, a pharmaceutical and financial industry giant 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; and Douglas Dachille, an investment management veteran and the former chief investment officer of American International Group, Inc. (AIG).

“We are privileged to have the opportunity to work with and learn from Fred, Andrea and Doug, and are grateful they are committed to helping us grow and advance our pipeline of potential therapies to treat patients with genetic diseases,” said BridgeBio founder and CEO Neil Kumar, Ph.D. “We are confident that the collective guidance these new directors offer will strengthen our ability to serve patients around the world.”

Fred Hassan

Mr. Hassan brings extensive experience leading and scaling global pharmaceutical companies and advising major corporates as a director, which will benefit BridgeBio as it expands its commercial infrastructure and broadens its reach to patients.

Mr. Hassan works as Director at Warburg Pincus LLC, a global private equity institution. Previously, Mr. Hassan was the chairman and chief executive officer of Schering-Plough and chairman and chief executive officer of Pharmacia Corporation, a company formed through the merger of Monsanto Company and Pharmacia & Upjohn, Inc.  Mr. Hassan is a Director at Prometheus Biosciences, and Precigen, both public biopharma companies using advanced technologies.

His previous directorships include Time Warner Inc. until its acquisition by AT&T Inc. in 2018, and Avon Products, where he served as chairman of the board, and Bausch and Lomb, where he also served as chairman. He has also served as a director at Amgen.  Mr. Hassan earned a bachelor’s degree from Imperial College of Science and Technology, University of London and a master’s degree in business administration from Harvard Business School.

“BridgeBio is creating something unique within the industry and I have been deeply impressed by its ability to accelerate the development of therapies for patients in need,” Mr. Hassan said. “I look forward to drawing on my experience as a leader of global pharmaceutical businesses to help BridgeBio grow into a world-class company with the ability to help millions of patients around the world who are suffering from genetic diseases and genetically-driven cancers.”

Andrea Ellis

As a financial expert in the technology and food sector, Mrs. Ellis will bring a fresh outsider perspective and industry diversity to the BridgeBio board.

Mrs. Ellis is the chief financial officer of Lime, an innovative transportation technology company that offers access to shared electric scooters, bikes and mopeds that is built on sustainability. Prior to joining Lime, Mrs. Ellis held various roles in finance, operations, and product management at Restaurant Brands International for many leading food chains. She previously spent six years at Goldman Sachs in investment banking and trading. Mrs. Ellis earned her bachelor’s degree from the University of Pennsylvania and a master’s degree in business administration from Harvard Business School.

“I’m thrilled to have the chance to help BridgeBio revolutionize the biotech and pharmaceutical industry with its groundbreaking approach to drug development. I am ready and eager to bring my experience in tech and finance to the board and aid the company in its mission to develop and deliver transformative medicines for patients suffering from genetic diseases,” Mrs. Ellis said.

Douglas Dachille

Mr. Dachille is an investment veteran who brings nearly 30 years of deep financial and strategic expertise to the board. He previously served as a board observer for the Company.

Mr. Dachille is AIG’s former chief investment officer, where he had responsibility for overseeing the investment portfolios of AIG’s insurance companies and the investments of third-party clients managed by First Principles Capital Management, LLC, an AIG subsidiary. He joined the company when AIG acquired First Principles Capital Management, LLC, an institutional fixed income asset manager, which he co-founded and served as chief executive officer. Mr. Dachille previously served as president and chief operating officer of Zurich Capital Markets and began his investment career at JPMorgan Chase & Company, where he served as global head of proprietary trading as well as co-treasurer. He earned his bachelor’s degree in a special joint program through Union University and Albany Medical College, and later was a Pew Scholar at the University of Chicago, where he earned a master’s degree in business administration.

“As a former board observer, I’m excited to make the leap and become a director at BridgeBio, a company I firmly believe is transforming the biotech industry with its innovative hub-and-spoke business model and deep and diversified drug pipeline,” Mr. Dachille said. “I look forward to helping BridgeBio grow into a global commercial organization that is able to reach and help patients in need around the world.”

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.

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

BridgeBio Pharma, Inc. Reports Second Quarter 2021 Financial Results and Business Update

Received U.S. Food and Drug Administration (FDA) approval for TRUSELTIQ™ (infigratinib) under the accelerated approval program for patients with cholangiocarcinoma (CCA)

Received FDA Fast Track designation for encaleret for the treatment of autosomal dominant hypocalcemia (ADH1)

Received FDA Fast Track designation for investigational gene therapy designed for the treatment of congenital adrenal hyperplasia (CAH)

Ended quarter with $898.4 million in cash, cash equivalents and marketable securities

PALO ALTO, Calif., AUGUST 5, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today reported its financial results for the second quarter ended June 30, 2021 and provided an update on the Company’s operations.

“We measure success by the number of meaningful medicines we are able to develop and deliver to patients. By that metric, our most significant accomplishment of the quarter was the approval of TRUSELTIQ™ (infigratinib) for patients with cholangiocarcinoma – our second FDA approval as a company and our first for a cancer treatment. All drug approvals are a team effort that extends far beyond BridgeBio. We thank the patient community, physicians, scientists and advocates for their commitment and drive. They made this approval possible,” said BridgeBio founder and CEO Neil Kumar, Ph.D.

Major milestones anticipated in the next 12 months for BridgeBio’s four core value drivers:

  • Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): Topline results from Part A of the 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. In a previous Phase 3 study in ATTR-CM patients, participants receiving the current standard of care treatment demonstrated a decline in 6MWD of approximately 25 meters (m) from an average baseline of 351m at 12 months.1 From a comparable average 6MWD baseline in the ATTRibute-CM study, the Company is seeking to more potently halt the observed decline in acoramidis-treated participants. As a reminder, healthy elderly adults typically decline only 7m in 12 months.2 If the change from baseline in 6MWD in Part A is highly statistically significant (p < 0.01), BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022 to the FDA.
  • Encaleret – Calcium-sensing receptor (CaSR) inhibitor for ADH1: Received Fast Track designation from the FDA. Early results from an ongoing Phase 2 proof-of-concept study shared at the Endocrine Society’s 2021 Annual Meeting (ENDO) in March 2021 showed normalization of blood calcium and urine calcium in 6 of 6 (100%) ADH1 participants. Additional data from the ongoing study are expected in the second half of 2021. If the development program is successful, 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.
  • 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. Investigational New Drug (IND) application 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:

  • FDA approval received for TRUSELTIQ™ (infigratinib) under the accelerated approval program for the treatment of patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as detected by an FDA-approved test. TRUSELTIQ is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. In the pivotal trial of patients with advanced, unresectable CCA, an aggressive malignancy with poor prognosis, TRUSELTIQ led to cases of tumor shrinkage. BridgeBio is eligible to receive upfront, regulatory and commercial milestone payments totaling up to approximately $2.45 billion USD through its strategic collaboration with Helsinn Group to co-develop and commercialize infigratinib in certain oncology indications.
  • Fast Track designation received from the FDA for BBP-812, BridgeBio’s AAV9 gene therapy candidate for Canavan disease. IND cleared by the FDA and site activation for initiation of a first-in-human Phase 1/2 study is ongoing. Canavan disease is an extremely rare genetic condition starting in infancy with an incidence of approximately one in 100,000 births worldwide.
  • Fast Track designation received from the FDA for infigratinib, an FGFR inhibitor, for the treatment of urothelial carcinoma (urinary tract and bladder cancer).
  • New Drug Application (NDA) acceptance from the Israeli Ministry of Health for NULIBRY™ (fosdenopterin) for injection to treat patients with molybdenum cofactor deficiency (MoCD) Type A. The FDA approved NULIBRY as the first therapy to reduce the risk of mortality in patients with MoCD Type A in February 2021. MoCD Type A is an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate.
  • First-in-human Phase 1 trial of BBP-711, a glycolate oxidase (GO) inhibitor to treat patients with hyperoxaluria, initiated in May 2021.
  • Research collaborations initiated with MUSC Foundation for Research Development, Stanford University and the University of Pittsburgh to identify and advance therapies for genetic diseases and cancers for a total of 23 partnerships among BridgeBio and leading academic and research institutions to date.
  • Non-exclusive, co-funded clinical collaboration initiated with Bristol Myers Squibb to study BBP-398, a potentially best-in-class SHP2 inhibitor, in combination with OPDIVO® (nivolumab) in patients with advanced solid tumors with KRAS mutations.
  • BridgeBio Pharma R&D Day: BridgeBio will hold a virtual R&D Day on Tuesday, October 12, 2021, from 8:30 am ET – 11:30 am ET. The event will be webcast and registration information can be found here.

Second Quarter 2021 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $898.4 million as of June 30, 2021, compared to $607.1 million as of December 31, 2020. The net increase in balance of $291.3 million is attributed to $731.4 million in net proceeds received from the issuance of our 2.25% Convertible Senior Notes due 2029 (the 2029 Notes) in January 2021 and $30.2 million in upfront payment and reimbursements received in connection with our License and Collaboration Agreement with Helsinn Healthcare S.A. and Helsinn Therapeutics (U.S.), Inc. (collectively, Helsinn) , which became effective in April 2021, and $25.0 million in net proceeds from our Amended Loan and Security Agreement with Hercules Capital, Inc. in April 2021 (the Amended Hercules Term Loan). The cash receipts were partially offset by a $61.3 million payment related to capped call options and a $50.0 million payment to repurchase shares of BridgeBio common stock, both in relation to the issuance of our 2029 Notes. In connection with our acquisition of Eidos Therapeutics, Inc. (Eidos) in January 2021, we paid $63.6 million in direct transaction costs and $21.3 million to Eidos stockholders who elected cash settlement. The remaining change of $299.1 million primarily related to payments of interest and operating costs and expenses.

Cash, cash equivalents and marketable securities, excluding restricted cash, decreased by $102.9 million compared to our balance as of March 31, 2021, which was $1,001.3 million. The decrease in cash is mainly due to cash used primarily related to our operating costs and expenses and partially offset by the payments received from Helsinn and proceeds from the Amended Hercules Term Loan as also discussed above.

Revenues

Total revenues for the three and six months ended June 30, 2021 were $54.0 million and $54.5 million, respectively. Our revenues mainly include upfront and milestone payments arising from the License and Collaboration Agreement with Helsinn and the License Agreement between our affiliate Navire Pharma, Inc. and LianBio.

Operating Costs and Expenses

Operating costs and expenses for the three and six months ended June 30, 2021 were $148.0 million and $316.0 million, respectively, as compared to $124.6 million and $227.1 million for the same periods in the prior year. The increases in operating costs and expenses of $23.4 million and $88.9 million, respectively, during the periods were attributable to the increase in personnel costs resulting from an increase in the number of employees to support the progression in our research and development programs, including our increasing research pipelines, as well as an increase in stock-based compensation related to the achievement of various performance-based milestone compensation arrangements tied to regulatory and development milestones. Stock-based compensation for the three and six months ended June 30, 2021 was $32.0 million and $66.9 million, respectively, as compared to $18.4 million and $28.6 million for the same periods in the prior year. Amounts for the three and six months ended June 30, 2021 reflect the reduction in operating costs and expenses arising from cost sharing recognized under our License and Collaboration Agreement with Helsinn. Our research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While we experienced some delays in certain of our clinical enrollment and trial commencement activities, we continue to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with our contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on our operating costs and expenses is currently unknown.

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.

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, the availability and success of topline results from Part A and Part B of our ATTRibute-CM trial of acoramidis, our plans to submit an application for regulatory approval of acoramidis, the availability of additional data from our ongoing study of encaleret for ADH1, the availability of initial data from our ongoing Phase 2 study of infigratinib for achondroplasia and our ongoing Phase 1/2 study of BBP-631 for CAH, our eligibility to receive future milestone 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, 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.

1 Maurer, M., Schwartz, J., Gundapaneni, B., et al. “Tafamidis treatment for patients with transthyretin Amyloid cardiomyopathy”. New England Journal of Medicine 379.11 (2018): 1007–16.

2Enright, M., Duanel, S. “Reference equations for the six-minute walk in healthy adults”. American Journal of Respiratory and Critical Care Medicine 158.5 (1998): 1384–7. 

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

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