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

PALO ALTO, Calif., July 6, 2020 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that on July 1, 2020, the compensation committee of BridgeBio’s board of directors granted eight employees options to purchase an aggregate of 7,120 shares of the Company’s common stock with a per share exercise price of $30.34 and restricted stock units for an aggregate of 15,134 shares of the Company’s common stock, including 1,610 performance-related restricted stock units. 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 team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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

Investor Contact:
John Grimaldi, Burns McClellan
jgrimaldi@burnsmc.com
212-213-0006 ext. 362

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. Appoints Biotech Trailblazers Brent Saunders and Randy Scott and Renowned Economist Andrew Lo to Board of Directors

PALO ALTO, Calif. – June 24, 2020 – BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that it has added three new independent directors to its board: former Allergan CEO and biopharma deal-maker, Brent Saunders; genomics pioneer and entrepreneur, Randy Scott, Ph.D.; and renowned economist and BridgeBio co-founder, Andrew Lo, Ph.D.

“We are privileged to welcome these world-class company builders, innovators, and thought leaders to our board where they can help us construct and advance a vast pipeline of meaningful medicines for patients with genetic disease,” said BridgeBio CEO and founder Neil Kumar, Ph.D. “Brent has a remarkable track record of leading companies to growth across many therapeutic areas. Randy is a giant in genomic medicine who has used his entrepreneurial skills to connect genetic information to patients in profound ways. Andrew’s groundbreaking financial engineering work led to the founding of BridgeBio and inspires us to find new ways to accelerate the drug development process. I look forward to working with and learning from these leaders as we seek to discover, develop, and deliver life-changing medicines at scale. It’s day one at BridgeBio and we are ready to go.”

Brent Saunders

Mr. Saunders will bring his considerable experience to bear as he advises BridgeBio on scaling its business across new product and therapeutic areas, expanding into new geographies, developing commercial expertise, and utilizing new corporate structures.

Mr. Saunders most recently served as chairman, president and chief executive officer of Allergan. In this capacity, he led the company to launch more than 15 products and achieve 9.4% revenue growth until its merger with AbbVie in 2020. He previously served as president and CEO at Actavis, where he led a $15 billion global pharmaceutical business until its merger with Allergan. He initially joined Actavis as part of the company’s acquisition of Forest Laboratories, where he served as president and CEO. Before joining Forest Laboratories, Mr. Saunders served as president and CEO of Bausch & Lomb. Mr. Saunders received his bachelor’s degree in economics and East Asian studies from the University of Pittsburgh, a Juris Doctor degree from Temple University School of Law and his Master of Business Administration from Temple University School of Business.

“I appreciate BridgeBio’s ability to accelerate the development of therapeutics for patients in need and I have been impressed with their unheard-of progress in pipeline growth in such a short time. As the company moves toward potential commercialization of its lead products, I’m eager to bring my experience leading and growing global pharmaceutical companies to help guide BridgeBio in its game-changing efforts,” said Mr. Saunders.

Randy Scott, Ph.D.

Dr. Scott will bring his deep expertise to BridgeBio and advise the company on how best to utilize the broad ecosystem of genomic medicine (beyond the pill) so that a wider universe of patients can be served. BridgeBio’s decentralized business model will also benefit from Dr. Scott’s guidance on developing and maintaining a strong culture of excellence.

Dr. Scott pioneered the introduction of genetics into everyday medical care as the founder of multiple cutting-edge biotech companies. Dr. Scott recently served as chief executive officer and executive chairman at Invitae, a company he co-founded to bring genetic information into routine medical practice. Prior to Invitae, he founded Genomic Health and served as the company’s CEO and later executive chairman – leading the company to develop and launch genomic diagnostic tests for breast, colon, and prostate cancer. Earlier in his career, Dr. Scott served as the president and chief scientific officer for Incyte, one of the first genetic information companies. He co-founded and currently serves as chairman of Genome Medical, a telegenomics-based clinical care company. Dr. Scott earned his bachelor’s degree in chemistry from Emporia State University and his doctorate in biochemistry from the University of Kansas.

“For the last 30 years, I’ve focused on building genomics companies to better understand the fundamental basis of human disease and improve the quality of treatment decisions through genomic diagnosis. It is with great excitement that I now join the BridgeBio board to take the next step in bringing multiple therapies for genetic disorders to market,” said Dr. Scott. “Genetic disorders are much more common than previously thought, and BridgeBio has the unique opportunity to efficiently bring multiple life-saving products to the clinic at an accelerating pace. With my experience in building and scaling companies, I plan to focus on helping the BridgeBio team to scale the organization and build a world-class pharmaceutical company focused on solving genetic disease.”

Andrew Lo, Ph.D.

Dr. Lo brings his vast knowledge and understanding of economics and financial engineering to BridgeBio and will advise the company on how to continue accessing financing from a range of markets to broaden its work for patients and pursue more groundbreaking scientific innovation. As a famously innovative and iconoclastic thinker, he will also challenge the company to continue to innovate in areas as diverse as R&D process, clinical trial statistics, and talent management.

Dr. Lo is the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management, director of the MIT Laboratory for Financial Engineering, a principal investigator at the MIT Computer Science and Artificial Intelligence Laboratory, and an affiliated faculty member of the MIT Department of Electrical Engineering and Computer Science. His research spans several areas of financial economics, but his most recent focus is on developing new statistical tools for predicting clinical trial outcomes, incorporating patient preferences into the drug approval process and accelerating biomedical innovation through novel financing structures. His work formed the foundation for BridgeBio’s business model, and he is one of the company’s co-founders. Dr. Lo earned his bachelor’s degree in economics from Yale University and a master’s degree and doctorate in economics from Harvard University.

“It’s remarkable to see the significant progress that BridgeBio has made in the five years since its founding to develop new medicines for genetically driven diseases,” said Dr. Lo. “I’m excited and honored to join the BridgeBio board and look forward to contributing in whatever ways I can to help them bring new therapies to patients who have no other alternatives.”

About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit bridgebio.com.

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

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma, Inc. and University of Florida Establish Collaboration to Advance Therapies for Genetically Driven Diseases

Partnership to Leverage University of Florida’s Research in Gene Therapy and BridgeBio’s Translational Expertise in Rare and Genetic Disease

PALO ALTO, Calif. – June 18, 2020 – BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, has entered into a strategic collaboration with the University of Florida to translate research in genetically driven disease towards clinical development and potential commercialization. The partnership combines University of Florida’s prowess in studying genetically driven disease, including its capabilities in gene therapy, with BridgeBio’s expertise in efficiently advancing therapeutics from the academic laboratory through preclinical studies and into human testing.

BridgeBio believes that, too often, promising research in academia sits on the shelf without partners to move it forward. The company’s mission is to bring as much of that research forward as possible, by focusing on establishing partnerships with leading institutions in the hopes of translating research into life-saving therapies.

“The scientists at University of Florida are recognized as leaders in research dedicated to genetically driven diseases, especially in the area of gene therapy. We are proud and eager to collaborate with them to push potential therapies forward for patients in need,” said BridgeBio CEO and founder Neil Kumar, Ph.D.

BridgeBio will provide sponsorship to select research programs around diseases with a genetic basis, including gene therapies and large and small molecules. The company will provide guidance for sponsored programs around medicinal chemistry for small-molecule hit optimization, strategies to modify or formulate a potential biologic therapy or approaches for testing non-optimized viral vectors. BridgeBio may conduct proof-of-concept studies for lead therapeutic compounds in relevant mammalian models.

“Great academic research scientists at the University of Florida have produced groundbreaking research, and through a partnership with BridgeBio we hope to turn more of that research into approved medicines for patients,” said Jim O’Connell, assistant vice president for commercialization at University of Florida. “BridgeBio has shown it understands the complexities in advancing gene therapies toward clinical development for patients in need – regardless of the size of an indication. We are encouraged by the company’s ability to advance multiple programs in parallel and commitment to patient communities. It is gratifying to see a drug development company take inspiration from the work that we are doing.”

BridgeBio seeks to revolutionize partnerships between drug development companies and biomedical research institutions by moving away from one-off interactions and building long-term partnerships based on trust, engagement, science and respect. The company is committed to acting responsibly towards the academic investigators who are on the front lines of understanding the mechanisms of genetically driven diseases and have great insights into how these diseases may be treated.

About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit bridgebio.com.

BridgeBio Pharma Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding the success of our strategic collaboration with the University of Florida to translate research in genetically driven disease towards clinical development and potential commercialization, our financial ability to provide sponsorship to select research programs around diseases with a genetic basis and the potential success of our product candidates to treat genetically driven diseases, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of our collaboration with the University of Florida to advance therapies for genetically driven diseases, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s most recent Quarterly Report on Form 10-Q and BridgeBio Pharma’s other SEC filings. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact:
Ian Stone
Canale Communications
ian@canalecomm.com
(619) 849-5388

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

BridgeBio Pharma, Inc. Enters Into Collaboration Agreement With Johns Hopkins University to Accelerate Development of New Medicines in Genetically Driven Diseases

PALO ALTO, Calif. – June 18, 2020 – BridgeBio Pharma, Inc. (NASDAQ: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that it has entered into a collaboration agreement with Johns Hopkins University to support the translation of academic innovations in genetically driven diseases with a goal of clinical development and potential commercialization to reach patients in need.

“We are privileged to establish a framework for collaboration with Johns Hopkins University, an institution that has pioneered our understanding of Mendelian disease. Our hope is that this relationship will allow us to build on the significant advances being made in Johns Hopkins’ labs and support work to translate them as quickly and as safely as possible into meaningful medicines for patients in need,” said BridgeBio CEO and founder Neil Kumar, Ph.D.

BridgeBio has a deep interest in understanding where great science is occurring and determining how it can support translating academic findings into treatments for patients, including by helping advance the important discoveries in genetic drivers of specific diseases made at Johns Hopkins. The company is focused on building relationships with academic partners to smooth and speed up the transition from the lab to the patient.

BridgeBio will focus on opportunities to support the incredible, early-discovery research around genetically validated targets underway at Johns Hopkins. Where possible, BridgeBio will invest heavily in programs to accelerate promising genetic-disease therapies to the clinic, from gene therapy to small molecule. It will lend its expertise to science designed to help develop a promising molecule or approach into a viable preclinical program, including medicinal chemistry for small-molecule hit optimization, strategies to modify or formulate a potential biologic therapy or approaches for testing non-optimized viral vectors. Additionally, BridgeBio may conduct proof-of-concept studies for lead therapeutic compounds in relevant mammalian models.

BridgeBio seeks to revolutionize partnerships between drug development companies and biomedical research institutions by moving away from one-off interactions and building long-term partnerships based on trust, engagement, science and respect. The company is committed to acting responsibly with academic investigators who are on the front lines of understanding the mechanisms of genetically driven diseases and have great insights into how these diseases may be treated.

About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, please visit bridgebio.com.

BridgeBio Pharma Forward-Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions.  We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions.  These forward-looking statements, including statements relating to expectations, plans, and prospects regarding our ability to build on the significant advances being made in Johns Hopkins’ labs and translate them into meaningful medicines for patients in need, 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, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the success of our product candidates to treat genetically driven diseases and the success of our collaboration with Johns Hopkins, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s most recent Quarterly Report on Form 10-Q and BridgeBio Pharma’s other SEC filings. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact:
Ian Stone
Canale Communications
ian@canalecomm.com
(619) 849-5388

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

BridgeBio Pharma’s ML Bio Solutions Announces Dosing of First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)

  • ML Bio Solutions has initiated a Phase 1 clinical trial of BBP-418 in healthy volunteers
  • ML Bio Solutions’ sponsored lead-in study is enrolling patients with LGMD2i across twelve sites in the U.S. and internationally, in collaboration with the LGMD-GRASP consortium

SAN FRANCISCO, CA– June 11, 2020— BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate ML Bio Solutions  today announced that the first subject has been dosed in the Phase 1 clinical trial of BBP-418, an orally-administered small molecule therapy being evaluated for the treatment of of limb girdle muscular dystrophy type 2i (LGMD2i). The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics, and food effect of BBP-418 in healthy volunteers. In collaboration with the GRASP-LGMD consortium based at Virginia Commonwealth University which, is led by Nicholas Johnson, M.D., M.S.C.I., ML Bio Solutions is also enrolling a LGMD2i lead-in study across multiple centers in the U.S. and Europe. ML Bio Solutions also announced that the U.S. Food and Drug Administration (FDA) approved its Investigational New Drug (IND) application for BBP-418 in March 2020.

“The FDA’s acceptance of the investigational new drug application for BBP-418 marks a turning point for patients with LGMD2i, who currently have no targeted treatment options. BBP-418 has demonstrated efficacy in both mild and severe animal models of LGMD2i, and the start of the Phase 1 trial brings us one step closer to translating its potential to change the course of  this incurable and debilitating disease,” said Uma Sinha, Ph.D., chief scientific officer of ML Bio Solutions and BridgeBio.

LGMD2i is an inherited muscular dystrophy that is associated with two mutant copies of a gene called  fukutin-related protein (FKRP). The FKRP enzyme enables the proper functioning of a fully glycosylated alpha-dystroglycan (α-DG) protein on muscle cells. While fully glycosylated α-DG anchors the inside and the outside of cells to act as a “shock-absorber,” hypo-glycosylated α-DG – such as occurs in LGMD2i and fukutinopathies – leads to muscle cells that cannot withstand the stress of normal cellular activities. ML Bio Solutions’ oral therapy BBP-418 is designed to enhance the function of enzymes like FKRP in order to restore glycosylation levels of α-DG protein and thereby recover its shock absorber functionality. The hope is that BBP-418 is ultimately able to change the prognosis of patients with LGMD2i – who experience progressive loss of skeletal, respiratory and cardiac muscle strength that leads to loss of walking, need for ventilatory support and even death from heart failure – and who today have only palliative treatment options. BBP-418 has previously been granted Orphan Drug Designation for LGMD2i by the FDA.

At this pivotal juncture, ML Bio Solutions has appointed a chief medical officer, Douglas Sproule, M.D. M.Sc., to lead clinical development as well as regulatory strategy and affairs for BBP-418. Dr. Sproule is a trained pediatric neuro-muscular specialist who worked most recently as vice president, clinical development and medical affairs and spinal muscular atrophy (SMA) therapeutic area head at AveXis.

“It is a privilege to join ML Bio Solutions as it rapidly advances an oral medicine with the potential to treat patients with LGMD2i as well as other alpha-dystroglycanopathies in the U.S., European Union and rest of world. Data from the ongoing lead-in and Phase 1 studies, guidance from our world-class scientific and clinical advisors, and feedback from the FDA and other regulatory authorities will inform and accelerate development of BBP-418 to address a critical unmet need for new treatment options in LGMD2i,” said Dr. Sproule.

“The potential of a disease-modifying treatment from ML Bio Solutions for LGMD2i represents a beacon of hope for our community. We stand ready to provide guidance about the needs and experiences of patients and caregivers to inform development of BBP-418 to ensure it can have the maximal potential impact and benefit for patients,” said Kathryn Bryant, CEO and founder of the Speak Foundation, a patient-led organization for people with limb girdle muscular dystrophy.

About GRASP-LGMD (Genetic Resolution and Assessments Solving Phenotypes in LGMD) Consortium

The GRASP-LGMD consortium assembles an international team of neuromuscular specialists, basic scientists, physical therapists, geneticists, informaticians, and patient advocates to address issues related to: diagnostics; outcome measure development, patient engagement; and therapeutic development, to advance the state of LGMD research and readiness to support translation of science into therapeutic development.  For more information visit: http://www.grasp-lgmd.org

About the ML Bio Solutions-sponsored GRASP Consortium lead in study:

A lead-in study is currently enrolling patients with a confirmed genetic diagnosis of LGMD2i  across 12 centers in the U.S. and Europe to collect data on clinical outcome assessments as well as disease specific muscle biomarkers and MR imaging biomarkers that can be used to inform future planned drug study trials in LGMD2i. For more information visit: https://clinicaltrials.gov/ct2/show/NCT04202627 OR contact info@mlbiosolutions.com

About ML Bio Solutions

ML Bio Solutions, an affiliate of BridgeBio Pharma, is a biotechnology company focused on developing a small molecule as an oral substrate supplementation therapy for LGMD2i. ML Bio Solutions is led by a team of veteran biotechnology executives, and together with patients and physicians, aims to bring safe, effective treatments to market as quickly as possible. For more information, visit mlbiosolutions.com.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

BridgeBio Pharma 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’ Phase 1 clinical trial 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, and the timing and success of initial top-line Phase 1 date of BBP-418, 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 Phase 1 clinical trial of BBP-418 for the treatment of LDMD2i, the continuing success of ML Bio Solutions’ collaboration with the GRASP-LGMD consortium based at Virginia Commonwealth University, 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 treat LGMD2i, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s most recent Quarterly Report on Form 10-Q and BridgeBio Pharma’s other SEC filings. Moreover, ML Bio Solutions 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.

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

Investor Contact:
John Grimaldi, Burns McClellan
jgrimaldi@burnsmc.com
212-213-0006 ext. 362

Source: BridgeBio Pharma

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

PALO ALTO, Calif., June 5, 2020 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that on June 1, 2020, the compensation committee of BridgeBio’s board of directors granted nine employees options to purchase an aggregate of 55,517 shares of the Company’s common stock with a per share exercise price of $28.88 and restricted stock units for an aggregate of 19,532 shares of the Company’s common stock, including 607 performance-related restricted stock units. 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 team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development.

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

Investor Contact:
John Grimaldi, Burns McClellan
jgrimaldi@burnsmc.com
212-213-0006 ext. 362

Source: BridgeBio Pharma, Inc.

BridgeBio Pharma’s QED Therapeutics Presents Data on Infigratinib in Cholangiocarcinoma and Urothelial Carcinoma at the American Society of Clinical Oncology 2020 Virtual Scientific Program

SAN FRANCISCO – May 29, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today that it will present data at the American Society of Clinical Oncology 2020 Virtual Scientific Program showing clinical advancement for infigratinib, QED’s oral FGFR1-3 inhibitor, in both urothelial carcinoma and cholangiocarcinoma (CCA).

Title: Infigratinib (BGJ398) in advanced/unresectable or metastatic urothelial carcinoma demonstrates consistent treatment response in both first-line and later-line treatment settings

Abstract: 5038

Presenter: Yung Lyou, City of Hope Comprehensive Cancer Center

An analysis of response rates in patients with advanced/unresectable or metastatic urothelial carcinoma based on the amount of prior lines of treatment showed consistent response to infigratinib. The objective response rate (ORR) for all patients (n=67) was 25% (95% CI 15.5-37.5), while the ORR for patients receiving infigratinib as first-line treatment (n=13) saw a response rate of 31% (95% CI 9.1-61.4) compared to 24% (95% CI 13.5-37.6) for patients receiving infigratinib as a second-line or later treatment (n=54). All eight patients in the study with upper tract urothelial carcinoma (UTUC) received infigratinib as second-line or later therapy. The response rates were higher for patients with UTUC, with an ORR of 50% (95% CI 15.7–84.3) and a disease control rate of 100%. In the study, treatment emergent adverse events occurring in >30% of patients were: hyperphosphatemia (46%), elevated creatinine (42%), fatigue (37%), constipation (37%), anemia (36%), decreased appetite (33%), dry mouth (31%), and alopecia (31%).

“These findings support the design of the ongoing, placebo-controlled PROOF 302 study to evaluate the efficacy of infigratinib in adjuvant urothelial carcinoma,” said author and PROOF 302 trial lead Sumanta Pal, MD, professor of medical oncology and therapeutics research at City of Hope Comprehensive Cancer Center. “The results in upper tract urothelial cancer (UTUC) build upon earlier research that the disease has a different genetic profile than urothelial carcinoma of the bladder, particularly with respect to FGFR3 alterations, and warrants further investigation in an even earlier setting.”

Title: A retrospective analysis of post second-line chemotherapy treatment outcomes for patients with advanced or metastatic cholangiocarcinoma and FGFR2 fusions

Abstract: 4591

Presenter: Milind M. Javle, MD Anderson Cancer Center

In a retrospective analysis of a subset of a single-arm Phase 2 study of infigratinib (n=37), outcomes from patients with FGFR-fusion-positive bile duct cancer receiving infigratinib as third- and later-line therapy were compared with the tumor response when those same patients received second-line therapy with chemotherapy. Treatment with infigratinib resulted in progression free survival (PFS) improvements.  The median PFS was 6.8 months (95% CI 3.9-7.8 months) for third- and later-line infigratinib treatment compared to 4.6 months (95% CI 2.7-7.2 months) for second-line chemotherapy.

“Through this retrospective analysis, we can see that infigratinib may have potential for patients whose tumors progress after second-line chemotherapy,” said Susan Moran, MD, MSCE, chief medical officer for QED. “These data support continued investigation of infigratinib in patients with FGFR-driven cholangiocarcinoma.”

About QED Therapeutics

QED Therapeutics, an affiliate of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity that we believe to be meaningful in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED intends to submit a New Drug Application (NDA) with the United States Food and Drug Administration for second and later-line cholangiocarcinoma in 2020. QED Therapeutics is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with other FGFR-driven tumor types and rare disorders.

For more information on QED Therapeutics, please visit the company’s website at www.qedtx.com.

About BridgeBio Pharma, Inc.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

BridgeBio Pharma 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 QED Therapeutics’ regulatory approval process, the timing of the submission of a New Drug Application (NDA) for second- and later-line cholangiocarcinoma, clinical trial designs, including the design of the ongoing, placebo-controlled PROOF 302 study, clinical development plans, clinical trial results, timing and completion of clinical trials, clinical and therapeutic potential of infigratinib, including for patients whose tumors progress after second-line chemotherapy, 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, QED Therapeutics’ ability to initiate and continue its ongoing and planned clinical trials of infigratinib, the availability of data from these trials, past data not being indicative of future data, its ability to advance infigratinib in clinical development according to its plans, and the timing of these events, including the planned NDA submission in 2020, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma’s most recent Quarterly Report on Form 10-Q and BridgeBio Pharma’s other SEC filings. Moreover, QED Therapeutics 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.

QED Contact:
Carolyn Hawley
Canale Communications
carolyn@canalecomm.com
858-354-3581

BridgeBio Pharma’s Phoenix Tissue Repair Provides Updates to its Recessive Dystrophic Epidermolysis Bullosa (RDEB) Program and Announces New Leadership Appointments

BOSTON — May 13, 2020 – Phoenix Tissue Repair, Inc. (PTR), an affiliate company of BridgeBio Pharma, Inc. (Nasdaq: BBIO) today announced updates to its ongoing Phase 1/2 study of PTR-01 (BridgeBio designation BBP-589), an intravenously-administered recombinant collagen 7 protein replacement therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). Cohorts 1 – 3 have completed treatment at escalating dose levels, and an additional fourth cohort to evaluate higher dosing of PTR-01 has begun enrolling patients.

Based on an interim review of data from the first three cohorts of trial participants, PTR-01 has been well tolerated in all nine patients, and there have been no treatment-related serious adverse events at any dose. In addition, as assessed by our investigators, there has been a dose-dependent increase in collagen 7 skin deposition after just three infusions over 28 days.  

RDEB is a rare genetic disorder characterized by severe blistering and scarring of the skin caused by even minor friction or trauma, as well as systemic manifestations including esophageal strictures and dysphagia, corneal abrasions, anemia and nutritional deficiencies.

“RDEB is a debilitating disease, and current treatment options are limited to palliative skin care involving daily wound maintenance, protective bandaging, pain and itch management, and treatment of secondary complications such as anemia,” said Anna L. Bruckner, M.D., MSCS, associate professor of dermatology and pediatrics at University of Colorado School of Medicine. “Intravenous collagen 7 replacement therapy with PTR-01 is the only treatment in development designed to provide broad disease-modifying effects for this multisystem disease. In addition to benefiting the skin, this treatment has the potential to improve wounds that affect the eye or internal mucosa surfaces such as the esophagus.”

The ongoing clinical trial is a randomized, saline-controlled, double-blind, repeat dose, dose-escalation, multi-center study. Patients enrolled in the fourth cohort will each be dosed over a 10-week period, with three 3.0 mg/kg doses of PTR-01 and three doses of saline control. The primary objective of the trial is to evaluate the safety and tolerability of PTR-01 in adults with RDEB. Additional endpoints include assessment of biologic activity through skin biopsy evaluation of collagen 7 deposition and clinical assessments including wound healing, pain, itch and quality of life.

After completion of this trial, the company is planning to follow it up with a six-month open-label clinical trial to inform the design of a pivotal clinical trial. To learn more about the PTR-01 Phase 1/2 clinical trial, please visit www.clinicaltrials.gov and search the identifier NCT03752905.

The company also announced that Sanuj K. Ravindran, M.D. has been appointed to lead PTR as executive chairman, and Hal Landy, M.D., and Deborah Ramsdell have joined PTR as chief medical officer and chief operating officer, respectively. Dr. Ravindran is currently CEO of PellePharm Inc., another BridgeBio company focused on rare genetic skin diseases. Both Dr. Landy and Ms. Ramsdell were involved in PTR-01’s early development at Lotus Tissue Repair, which was acquired by Shire prior to PTR securing rights to the PTR-01 program.

“With safety as a top priority, we are committed to creating the first-ever systemic treatment for RDEB, which targets the genetic root of the disease by replacing the collagen protein that normally helps keep the epidermis from separating from the dermis,” said Dr. Ravindran. “As the program advances, we look forward to working closely with the epidermolysis bullosa patient community to help raise awareness of this debilitating disease and ensure that we develop our therapy to best address the unmet needs that patients currently face.”

About Dystrophic Epidermolysis Bullosa (DEB)

DEB is a rare genetic disorder symptomatic from birth that is caused by mutations in the gene for a protein called collagen type VII (C7). The C7 protein is essential for the formation of anchoring fibrils, structures which connect the epidermis and dermis—the uppermost two layers of the skin. Patients with the recessive form of DEB (RDEB) tend to have particularly severe symptoms due to severe insufficiency of functional C7. Symptoms include extreme skin and mucosal fragility that present as recurrent, painful blistering and scarring of the skin, as well as ulcerations of the mouth, tongue and dental caries. In addition to the cutaneous and oral symptoms, severe forms are associated with erosions and scarring of mucous membranes of the eye, esophagus, genitals and anus. Joint contractures, mutilating deformities of hands and feet, malnutrition, growth retardation, recurrent infections and a significantly increased risk for squamous cell carcinoma are also common. There are currently no approved disease-modifying therapies for any form of DEB, and the standard of care focuses on wound and pain management.

About Phoenix Tissue Repair and PTR-01

Phoenix Tissue Repair is a Boston-based company that is an affiliate of BridgeBio Pharma, and is focused on advancing a novel systemic treatment for recessive dystrophic epidermolysis bullosa (RDEB). PTR-01 is an investigational protein replacement therapy which uses a recombinant collagen type VII (rC7) for the treatment of RDEB. PTR-01 is designed to be systemically available through intravenous delivery. Phoenix Tissue Repair acquired worldwide rights to PTR-01 in 2017. Preclinical studies of PTR-01 have demonstrated C7 staining in basement membranes with de novo anchoring fibril formation and improved survival in models of RDEB.

PTR-01 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration and the European Medicines Agency.

About BridgeBio Pharma, Inc.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

Forward-Looking Statements

This press release contains forward-looking statements. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements relating to expectations, plans, and prospects regarding Phoenix Tissue Repair’s clinical development plan, clinical trial results, timing and completion of clinical trials, and ability to take advantage of expedited FDA review for PTR-01. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, Phoenix Tissue Repair’s ability to advance PTR-01 in clinical development in accordance with its plans, the results from any clinical trials and nonclinical studies of PTR-01, and the nature of Phoenix Tissue Repair’s interactions with regulatory authorities. Moreover, Phoenix Tissue Repair 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 Phoenix Tissue Repair’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. All forward-looking statements in this press release are based on information available to Phoenix Tissue Repair as of the date hereof, and Phoenix Tissue Repair disclaims any obligation to update these forward-looking statements except as required by law.

Investor Relations Contact:
Mike Mangone, Ph.D.
Vice President, Business Development & Corporate Strategy
857-449-0970
info@phoenixtissuerepair.com

Media Relations Contact:
Carolyn Hawley
Canale Communications
(619) 849-5382
carolyn@canalecomm.com

BridgeBio Pharma, Inc. Reports First Quarter 2020 Financial Results and Business Update

Ended quarter with $928 million in cash, cash equivalents and marketable securities

SAN FRANCISCO, May 13, 2020 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), today reported its financial results for the first quarter ended March 31, 2020 and provided an update on the company’s operations.

The company is working with its committed partners to minimize disruption from the COVID-19 pandemic on its clinical trials and other drug development programs. BridgeBio salutes the brave physicians, nurses, first responders and medical staff who are working across the globe to care for patients with COVID-19.

BridgeBio’s fully enrolled trials and preclinical programs are moving ahead with minimal delays, and the company is on track to file its second New Drug Application (NDA) later this year, along with the anticipated filing of multiple investigational new drug applications (INDs). Despite the challenges presented by COVID-19, BridgeBio’s clinical trials of infigratinib in tumors with FGFR genetic alterations are proceeding as planned.

Certain clinical trials that were in the process of enrolling have slowed as a result of the COVID-19 outbreak. BridgeBio is working with hospitals and investigators to deliver investigational medicines to patients and to develop solutions that will allow the company to continue to measure key endpoints for these trials. BridgeBio is also prepared to continue enrolling patients as the healthcare system regains the ability to accommodate this activity.

For trials that have not yet begun enrolling, BridgeBio is prepared to begin them as soon as trial sites can accommodate enrollment. Given the severe and often life-threatening nature of many of the disease areas on which BridgeBio is focused, we anticipate many of our planned trials will be prioritized.

“Given COVID-19’s impact, we estimate that certain milestones related to clinical trials that were anticipated in 2020 are now more likely to come in 2021. Other programs are moving forward without delay, thanks to the committed efforts of our partners,” said BridgeBio founder and CEO Neil Kumar, Ph.D. “We believe we are in a position financially and operationally to move our critical work forward the moment the public health situation allows for that. Many of the patients we work with are battling devastating diseases that will not wait for this pandemic to pass.”

Just prior to substantial market dislocation driven by COVID-19, BridgeBio strengthened its balance sheet by raising $550 million in gross proceeds through the issuance of 2.50% Convertible Senior Notes due in 2027 (2027 Notes). This important financing added to approximately $700 million in gross proceeds raised from our initial public offering in June 2019 and Series D announced in January 2019, and puts BridgeBio in a strong financial position to execute on the 20+ drug development and discovery programs in its pipeline.  The company expects current cash, cash equivalents and marketable securities to carry it through critical and high-value milestones stretching into 2022.

“Our recent financings have focused on solving for two objectives at once: securing the capital required to independently advance our pipeline, while minimizing shareholder dilution,” said BridgeBio CFO Brian Stephenson, Ph.D., CFA. “One of the original goals in the founding of BridgeBio was to create a vehicle that could attract a new set of investors to fund critical biomedical research and drug development by taking a sufficiently large number of shots on goal, within an advantaged research and development space, to build conviction that multiple approved products are not just possible but statistically probable over time.  We appreciate the confidence investors have demonstrated in the critical work we are doing for patients.”

Steps BridgeBio has taken in response to COVID-19

  • Delivering investigational medicines to trial participants directly now that many can no longer visit the hospital
  • Implementing out-of-hospital solutions for clinical trials – including telehealth appointments and remote clinical monitoring
  • Engaging in conversations with the U.S. Food and Drug Administration (FDA) across multiple programs to understand how to preserve the fidelity of key endpoints
  • Working closely with contract manufacturing partners to anticipate potential downstream impacts to BridgeBio’s immediate supply chain
  • Working on key aspects of site activation and support so that BridgeBio can continue enrolling patients where and when possible
  • Transferring many of our non-clinical laboratory activities to contract research organizations that continue to work on them, when BridgeBio has not been able to sustain them

First quarter 2020 and recent pipeline progress:

Mendelian

  • BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa (RDEB): Due to a slowdown in enrollment related to COVID-19, BridgeBio now plans to share topline data from the ongoing Phase 1/2 study (NCT03752905) in 2021.
  • BBP-265 (AG10) – TTR stabilizer for ATTR:  Due to a slowdown in site activation and participant enrollment related to COVID-19, BridgeBio currently expects enrollment of ATTRibute-CM to be completed in the first half of 2021 (ATTRibute-CM) and plans to initiate its Phase 3 trial of AG10 in ATTR-PN (ATTRibute-PN) in the second half of 2020
  • Low-dose infigratinib for achondroplasia:The Phase 2 clinical program (PROPEL2) is on track to dose the first children in 2020 (NCT04265651). Enrollment has continued in areas where it is safe for children and their families to participate and BridgeBio continues to activate sites remotely.
  • Fosdenopterin – cPMP replacement therapy for MoCD type A: Remains on track to complete the rolling NDA submission to FDA in 2020.
  • Topical patidegib gel for Gorlin syndrome and high-frequency basal cell carcinoma[1]: Phase 3 study is fully enrolled as of the fourth quarter of 2019 and remains on track for last patient last visit in the fourth quarter of 2020 (NCT03703310).

Multiple new clinical studies are ready to initiate pending stabilization of COVID-19:

  • Encaleret (CaSR antagonist) Phase 2 study in autosomal dominant hypocalcemia type 1.
  • Ribitol (BBP-418)Phase 1 study in healthy volunteers.
  • Zuretinol (syntetic retinoid) Phase 2/3 study in inherited retinal diseases due to RPE65 or LRAT gene mutations.

[1] PellePharm, which is focused on developing patidegib topical gel, 2% entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

Targeted oncology

  • Infigratinib – FGFR1-3 inhibitor for FGFR+ cancer: Preparing NDA submission in 2020 for second-line FGFR2+ cholangiocarcinoma. Our front-line cholangiocarcinoma (NCT03773302), adjuvant urothelial carcinoma (NCT04197986), and tumor agnostic (NCT04233567) studies continue to enroll as BridgeBio activates sites remotely.

Gene therapy

  • BBP-812 – Gene therapy candidate for Canavan disease: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.
  • BBP-631 – Gene therapy candidate for congenital adrenal hyperplasia: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.

Additional updates

  • Anticipate disclosing new product candidates and filing multiple new INDs in 2020.

First quarter 2020 financial results:

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $928.4 million as of March 31, 2020 compared to $577.1 million at December 31, 2019.  The net change in cash balance of $351.3 million reflects $537.6 million in net proceeds received from the issuance of our 2027 Notes, $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares, $49.3 million payment related to capped call option and the remaining payment of $86.1 million primarily related to operating expenses. 

Operating expenses

Operating expenses for the first quarter of 2020 were $102.5 million, as compared to $63.8 million for the same period in the prior year. The increase in operating expenses of $38.7 million was attributable to the increase in external-related costs and increase in headcount to support the progression in our research and development programs, including our increasing research pipelines, and overall growth of our operations.

We have experienced some delays in our research and development activities arising from delays in enrollment of certain of our ongoing clinical trials and in the commencement of certain planned trials due to the global outbreak of COVID-19. The delays experienced occurred towards the end of the first quarter of 2020 and therefore, our research and development expenses have not been significantly impacted for the full quarter.  However, we believe that delays in our ongoing and planned clinical trials and adjustments to certain of our study procedures, such as enabling alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with our contract manufacturing organizations, may increase our expenses or draw them out over a longer period of time.  The longer term impact of COVID-19 on our operating expenses is currently unknown.

(1) The condensed consolidated balance sheet as of December 31, 2019 is derived from the audited consolidated financial statements as of that date.
(2) March 31, 2020 and December 31, 2019 amounts include long-term marketable securities of $23.2 million and $31.1 million, respectively.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com.

BridgeBio Pharma 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 BridgeBio’s product candidates, including, but not limited to, the unknown future impact of the COVID-19 pandemic’s delay to certain clinical trial milestones and effect on BridgeBio’s operations, the number of potential medicines in our portfolio, our plans to file an NDA for infigratinib in cholangiocarcinoma and multiple INDs for new product candidates in 2020, the initiation of our planned Phase 2/3 trial of Zuretinol, the availability of topline data from our Phase 1/2 study of BBP-589, the anticipated dosing of the first children in our Phase 2 dose-ranging study of infigratinib in achondroplasia, the completion of enrollment in our ongoing Phase 3 study of BBP-265 in ATTR-CM, the initiation of our Phase 3 trial of BBP-265 in ATTR-PN, the completion of our rolling NDA submission to FDA for fosdenopterin, the timing of last patient last visit for topical patidegib gel for Gorlin syndrome and high-frequency basal cell carcinoma, the availability of updated results for infigratinib and our plans for further clinical development of infigratinib in FGFR+ cancer, our expected runway for cash, cash equivalents and marketable securities, 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, 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 quarterly or annual periodic report filed with the 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.

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

Investor Contact:
John Grimaldi, Burns McClellan
jgrimaldi@burnsmc.com
212-213-0006 x362

Source: BridgeBio Pharma

BridgeBio Pharma’s QED Therapeutics Announces Preclinical Data Demonstrating Potential of Low-Dose Infigratinib in Achondroplasia

Data Accepted to ENDO 2020

SAN FRANCISCO – May 11, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today that in vitro and in vivo data from two studies support QED’s plans to evaluate a low dose of infigratinib as a treatment option for children with achondroplasia, the most common cause of disproportionate short stature. Data were accepted for presentation at the Endocrine Society’s Annual Meeting (“ENDO 2020”), which was cancelled due to COVID-19, and the studies were published in the special supplemental section of the Journal of the Endocrine Society.

The first study, entitled “Support for a new therapeutic approach of using a low-dose FGFR tyrosine kinase inhibitor (infigratinib) for achondroplasia,” showed that in a mouse model of achondroplasia, treatment with infigratinib led to dose-dependent improvement in achondroplasia-associated phenotypes. At a low dose of 0.5 mg/kg, infigratinib was associated with a statistically significant improvement in bone length of 7% to 14% in the upper limbs, 10% to 17% in the lower limbs and 12% in the foramen magnum (the opening at the base of the skull). Also presented in this study was in vitro data demonstrating that, at similar concentrations, infigratinib had greater activity over a CNP analog (vosoritide) in an achondroplasia cell line. The data suggest that inhibition of multiple key pathways downstream of FGFR3 controlling either proliferation or differentiation of the chondrocytes may lead to better efficacy compared with MAPK inhibition alone.

The second study, entitled “Low-dose infigratinib treatment does not lead to changes in phosphorus in preclinical animal studies,” showed that rats and mice treated with infigratinib at or below 5 mg/kg showed no relationship between dose and blood levels of phosphorus, a potential safety concern for infigratinib treatment.

In addition, two other posters accepted to ENDO 2020, including the design of QED’s PROPEL natural history study in achondroplasia, are available for review on the QED corporate website. Titles for the presentations are:

“The currently-enrolling PROPEL natural history study is an important step in establishing a baseline of growth trajectory for children with achondroplasia and will help evaluate whether treatment with infigratinib has a positive effect on growth,” said Melita Irving, Consultant Clinical Geneticist, Guy’s and St Thomas’ Hospital (London). “Fully enrolling the study will help us to advance infigratinib as a potential treatment for children with achondroplasia.”

QED Therapeutics is currently looking to continue enrolling children in PROPEL, a natural history study in children with achondroplasia. The study aims to learn more about the overall health, growth, and possible medical complications in children with achondroplasia. To participate in or learn more about PROPEL, please contact us at: PROPEL@QEDTx.com or visit ClincialTrials.gov.

Children enrolled in PROPEL may have the opportunity to enroll in PROPEL 2, an interventional phase 2 trial to assess the safety of daily dosing, evidence of efficacy, and dose finding of infigratinib in children with achondroplasia. QED Therapeutics anticipates submitting our IND to the FDA in 2020.

About QED Therapeutics

QED Therapeutics, an affiliate of BridgeBio Pharma, is a biotechnology company focused on precision medicine for FGFR-driven diseases. Our lead investigational candidate is infigratinib (BGJ398), an orally administered, FGFR1-3 selective tyrosine kinase inhibitor that has shown activity that we believe to be meaningful in clinical measures, such as overall response rate, in patients with chemotherapy-refractory cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED intends to submit a New Drug Application (“NDA”) with the United States Food and Drug Administration (“FDA”) for second and later-line cholangiocarcinoma in 2020. QED Therapeutics is also evaluating infigratinib for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with other FGFR-driven tumor types and rare disorders.

For more information on QED Therapeutics, please visit the company’s website at www.qedtx.com.

About BridgeBio Pharma, Inc.

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 20 development programs includes product candidates ranging from early discovery to late-stage development. For more information, visit bridgebio.com

BridgeBio Pharma 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 potential for a low dose of infigratinib as a treatment option for children with achondroplasia, expectations, plans, and prospects regarding QED Therapeutics’ regulatory approval process, clinical trial designs, clinical development plans, clinical trial results, timing and completion of clinical trials, 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 number of risks, uncertainties and assumptions, including, but not limited to, in vitro and in vivo data from prior studies being indicative of future study data, QED Therapeutics’ ability to initiate and continue its ongoing and planned clinical trials of infigratinib, QED Therapeutics’ ability to enroll children in its ongoing and planned clinical trials, the availability of data from these trials, its ability to advance infigratinib in clinical development according to its plans, and the timing of these events, as well as those risks set forth in the Risk Factors section of BridgeBio Pharma, Inc.’s most recent Annual Report on Form 10-K and our other SEC filings. Moreover, QED Therapeutics 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.

QED Contact:
Carolyn Hawley
Canale Communications
carolyn@canalecomm.com
858-354-3581