Ivy Brain Tumor Center and BridgeBio Pharma’s QED Therapeutics Announce Dosing of First Patient in Investigator-Initiated Phase 0/2 Clinical Trial of Infigratinib in Recurrent Glioblastoma

  • Precision medicine study designed to confirm drug’s ability to cross blood-brain barrier and hit molecular targets in high-grade glioma patients with FGFR genetic alterations
  • Patients with a positive PK response will continue on infigratinib after surgery

PHOENIX, AZ and SAN FRANCISCO – July 28, 2020 – Ivy Brain Tumor Center at Barrow Neurological Institute announced today that the first patient has been dosed in an investigator-initiated Phase 0/2 clinical trial of infigratinib in recurrent high-grade glioma driven by FGFR genetic alterations. Infigratinib is an investigational, orally administered, FGFR1-3 selective tyrosine kinase inhibitor being developed by BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate company QED Therapeutics, Inc.

The investigator-initiated Phase 0/2 trial is designed to confirm drug effects within days of exposure, and only to continue dosing when the drug is active in a patient’s own tumor. The primary objective of the Phase 0 arm is to assess how effectively infigratinib can cross the blood-brain barrier – the most significant obstacle to developing new, effective therapies for aggressive brain tumors like glioblastoma. Patients with successful tumor penetration will receive infigratinib long-term in a Phase 2 expansion arm of the trial. The primary endpoint of the expansion phase is progression-free survival rate at six months. The study will also measure how well infigratinib is impacting its molecular target in each patient’s tumor.

FGFR (fibroblast growth factor receptor) genetic alterations have been shown to spur growth in malignant tumors. Five to seven percent of glioblastoma patients’ tumors are driven by FGFR signaling. During the trial screening process, the patient’s tumor tissue from prior surgery will be tested for the FGFR-TACC3 fusion gene or mutations in FGFR1 and FGFR3 genes. Patients with tumors that have these fusions or mutations are eligible for this study.

“In the preclinical studies, our pharmacokinetics program at the Ivy Brain Tumor Center tested seven FGFR inhibitors for their ability to cross the blood-brain barrier. Infigratinib was one of the most promising agents,” said Shwetal Mehta, Ph.D., deputy director of the Ivy Brain Tumor Center.

“Infigratinib was previously tested in an uncontrolled Phase 2 study for recurrent high-grade gliomas,” said Nader Sanai, M.D., director of the Ivy Brain Tumor Center. “The results were intriguing, but inconclusive. This Ivy Phase 0/2 trial seeks to provide direct biological evidence of drug effects in individual patients, allowing us to understand which glioblastoma patients may benefit from infigratinib.”

“The launch of this investigator-initiated trial is an exciting step in the study of infigratinib for patients with recurrent, high-grade glioma,” said Susan Moran, M.D., M.S.C.E., chief medical officer of QED Therapeutics. “We anticipate this study being conducted by the Ivy Center will generate valuable information on the ability of infigratinib to reach brain tumors, which is a critical first step in evaluating whether infigratinib, alone or in combination, could potentially provide a therapeutic option for patients with this dire disease.”

For additional information on this Phase 0/2 trial in recurrent high-grade glioma, including eligibility criteria, visit www.clinicaltrials.gov/ct2/show/NCT04424966.

About Ivy Brain Tumor Center

Ivy Brain Tumor Center at the Barrow Neurological Institute in Phoenix, AZ is a non-profit translational research program that employs a bold, early-phase clinical trials strategy to identify new treatments for aggressive brain tumors, including glioblastoma. The Ivy Center’s Phase 0 clinical trials program is the largest of its kind in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. Unlike conventional clinical trials focusing on single drugs, its accelerated trials program tests therapeutic combinations matched to individual patients. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, Twitter and LinkedIn.

About QED Therapeutics, Inc.

QED Therapeutics, an affiliate of BridgeBio Pharma, Inc. 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 clinical 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. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements relating to expectations, plans, and prospects regarding QED Therapeutics’ clinical development plans, clinical trial results, timing, completion and outcomes of clinical trials, including this investigator-initiated trial, the competitive environment, the success of QED Therapeutics’ collaboration with the Ivy Brain Tumor Center and its impact on QED Therapeutics’ clinical development strategy, and the clinical and therapeutic potential of infigratinib. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, QED Therapeutics’ ability to initiate and continue its planned clinical trials of infigratinib, its ability to advance infigratinib in clinical development, the timing and success of any such continued clinical development, and the Ivy Brain Tumor Center’s ability to initiate and enroll its investigator-initiated clinical trial of infigratinib and the nature of QED’s interactions with regulatory authorities. Moreover, QED Therapeutics 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 QED Therapeutics’ 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 QED Therapeutics as of the date hereof, and QED Therapeutics disclaims any obligation to update these forward-looking statements.

MEDIA:
Ivy Brain Tumor Center
Melinda Langdon
Director, Marketing and Communications
(623) 297-1317
melinda.langdon@ivybraintumorcenter.org

QED Therapeutics
Ian Stone
Canale Communications
619-849-5388
ian@canalecomm.com

BridgeBio Pharma’s QED Therapeutics Doses First Child in Phase 2 Clinical Trial of the Investigational Medicine Infigratinib in Achondroplasia

SAN FRANCISCO – July 15, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today that the first child with achondroplasia has been dosed with the investigational medicine infigratinib, an orally available small molecule, that targets the overactivity of fibroblast growth factor receptor 3 (FGFR3) in the PROPEL 2 Phase 2 clinical trial. Achondroplasia is the most common cause of disproportionate short stature.1  

“With preclinical evidence showing increased growth in the long bones, spine and cranial bones, including the base of the skull, there is the potential for infigratinib to help children with achondroplasia by decreasing the impact of serious medical complications,” said Professor Ravi Savarirayan, M.B., B.S., M.D., Ph.D., clinical geneticist and group leader of skeletal biology and disease at Murdoch Children’s Research Institute in Australia and the lead investigator for the PROPEL 2 trial. “Additionally, infigratinib is being studied as a once-daily dose taken orally, which is an important factor for administration of therapies to children with achondroplasia.”

Achondroplasia is caused by an alteration in the FGFR3 gene, which causes the FGFR3 protein to be overly active.1 This interferes with skeletal development and can lead to disturbances in bone growth.1 Infigratinib is an oral investigational medicine that is designed to reduce the activity of FGFR3.

“The start of this clinical trial is the culmination of more than two years of work – first to secure rights to develop infigratinib, which we pursued following the publication of data relating to its potential in achondroplasia, and then to establish preclinical data showing the efficacy and safety of very low doses of the molecule,” said Michael Henderson, M.D., CEO of QED Therapeutics. “Infigratinib illustrates the heart of what BridgeBio set out to do: leverage the highest quality science to identify and develop potential therapies that target genetically driven conditions at their source. Our hope is that a daily, oral dose of infigratinib, which directly targets FGFR3, can provide health benefits for children with achondroplasia.”

The PROPEL 2 trial is a Phase 2 dose escalation and dose expansion trial and the first clinical trial to study infigratinib at low doses in children with achondroplasia. The goal of the study is to assess safety and measure changes from baseline in annualized height velocity and changes in other health factors. To be eligible for the trial, children must first complete a six-month period of assessment in the PROPEL prospective clinical assessment study.

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 we are evaluating in clinical studies for the treatment of achondroplasia. We plan to conduct further clinical trials to evaluate the potential for infigratinib to treat patients with FGFR-driven tumor types and rare disorders. At much higher doses, infigratinib 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.

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 timing, expectations, plans, and potential success of QED Therapeutics’ Phase 2 PROPEL 2 clinical trial, the regulatory approval process, clinical trial designs, clinical development plans, clinical trial results, timing and completion of the PROPEL 2 and other clinical trials, clinical and therapeutic potential of infigratinib for the treatment of achondroplasia or other FGFR-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, QED Therapeutics’ ability to continue or complete its Phase 2 PROPEL 2 clinical trial, ongoing and planned clinical trials of infigratinib for the potential treatment of achondroplasia and other FGFR-driven, the availability of data from these trials, past data from preclinical and earlier clinical studies not being indicative of future data from clinical 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 Quarterly Report on Form 10-Q 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.

  1. National Institutes of Health. https://ghr.nlm.nih.gov/condition/achondroplasia. Accessed June 23, 2020.

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

BridgeBio Pharma’s Phoenix Tissue Repair to Highlight Interim Phase 1/2 Study Data in a Presentation at the Society for Pediatric Dermatology’s 45th Annual Meeting

BOSTON – July 10, 2020 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate Phoenix Tissue Repair (PTR) today announced an upcoming presentation of interim data from an ongoing Phase 1/2 study of PTR-01 (BBP-589), an intravenously-administered recombinant collagen 7 protein replacement therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The presentation will be made during the Society for Pediatric Dermatology’s (SPD) 45th Annual Meeting, to be held virtually July 10-12, 2020.

The poster presentation, which includes safety and tolerability data observed so far in patients enrolled in cohorts 1-3, will be delivered by Anna L. Bruckner, MD, associate professor of dermatology and pediatrics at University of Colorado School of Medicine. The pre-recorded presentation will be available online to meeting registrants until December 31, 2020. The poster will also be available on the Phoenix Tissue Repair website.

Details for the presentation are below:

Title: Interim update from a Phase 1/2 trial examining the safety and tolerability of PTR-01, a collagen 7 protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa

Presenter: Anna L. Bruckner, MD

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 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