BBIO BridgeBio Pharma Inc.

39.55
-1.19  -3%
Previous Close 40.74
Open 39.84
52 Week Low 39.335
52 Week High 73.5
Market Cap $5,821,169,083
Shares 147,185,059
Float 79,918,173
Enterprise Value $6,795,704,303
Volume 488,526
Av. Daily Volume 812,537
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Upcoming Catalysts

Drug Stage Catalyst Date
Acoramidis (AG10) - ATTRibute-CM
ATTR-CM
Phase 3
Phase 3
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Low-dose infigratinib (PROPEL2)
Achondroplasia
Phase 2
Phase 2
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BBP-589
Epidermolysis bullosa
Phase 1/2
Phase 1/2
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BBP-631
Congenital adrenal hyperplasia (CAH)
Phase 1/2
Phase 1/2
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BBP-711
Hyperoxaluria
Phase 1
Phase 1
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BBP-812 (CANaspire)
Canavan Disease
Phase 1/2
Phase 1/2
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BBP-418
Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
TRUSELTIQ (Infigratinib)
Cholangiocarcinoma
Approved
Approved
FDA approval announced June 1, 2021.
TRUSELTIQ (Infigratinib)
Solid tumors
Phase 2
Phase 2
Phase 2 trial initiation announced March 12, 2020.
Acoramidis (AG10) / BBP-265
ATTR-PN
Phase 3
Phase 3
Phase 3 trial has been initiated.
BBP-398
Solid tumors
Phase 1
Phase 1
Phase 1 initiation of dosing announced November 13, 2020.
Patidegib (BBP-009)
Gorlin syndrome
Phase 3
Phase 3
Phase 3 completion of enrollment announced December 2, 2019.
Patidegib (BBP-009)
High-Frequency Basal Cell Carcinoma
Phase 2
Phase 2
Phase 2 trial initiation announced January 8, 2020.
TRUSELTIQ (infigratinib)
Gastric Cancer / Gastroesophageal junction adenocarcinoma
Phase 2a
Phase 2a
Phase 2a dosing commencement announced August 25, 2021.
BBP-681 (VT30)
Venous, Lymphatic, and Venolymphatic Malformations
Phase 1/2
Phase 1/2
Phase 1/2 initiation of dosing announced February 2, 2021.
Encaleret
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Phase 2b
Phase 2b
Phase 2b data presented at ENDO March 20, 2021 well-tolerated, no adverse events. Phase 2b data presented at ASBMR noted all 13 trial participants normalized mean blood calcium levels and 24-hour urine calcium excretion during Periods 1 and 2. Parathyroid hormone levels increased in all participants and mean blood phosphate decreased into the normal range during Periods 1 and 2, noted October 1, 2021. Phase 3 trial planned to initiate 2022.
BBP-671
Healthy Volunteers
Phase 1
Phase 1
Phase 1 dosing has commenced.
TRUSELTIQ (infigratinib)(PROOF 302)
Urothelial Carcinoma
Phase 3
Phase 3
Phase 3 trial initiation announced March 12, 2020.
TRUSELTIQ (infigratinib)
Recurrent Glioblastoma
Phase 2
Phase 2
Phase 0/2 trial initiation announced July 28, 2020.
Fosdenopterin (BBP-870/ORGN001)
Molybdenum cofactor deficiency type A
Approved
Approved
FDA approved February 26, 2021.

Latest News

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

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

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

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

    PALO…

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

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

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

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

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

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

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

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

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

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

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

    About BridgeBio Pharma, Inc.

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

    About the Helsinn Group

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

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

    Helsinn Group plays an active and central role in promoting social transformation in favor of people and the environment. Corporate social responsibility is at the heart of everything we do, which is reinforced in the company's strategic plan by a commitment to sustainable growth. To learn more about Helsinn Group please visit www.helsinn.com

    BridgeBio Pharma, Inc. Forward-Looking Statements

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

    BridgeBio Media Contact:

    Grace Rauh



    (917) 232-5478

    BridgeBio Investor Contact:

    Katherine Yau



    (516) 554-5989

    Helsinn Group Media Contact:

    Paola Bonvicini

    Group Head of Communication



    +41 (0) 91 985 21 21

     



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

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

    This facility, combined with the Company's existing cash balance as of September 30, 2021, provides access to over $1.2 billion to advance the Company's pipeline programs, support commercialization…

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

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

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

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

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

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

    Key features of the credit facility include:

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

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

    Latham & Watkins LLP acted as counsel to BridgeBio.

    About BridgeBio Pharma, Inc.



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

    BridgeBio Pharma, Inc. Forward-Looking Statements

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

    BridgeBio Media Contact:

    Grace Rauh



    (917) 232-5478

    BridgeBio Investor Contact:

    Katherine Yau

     

    (516) 554-5989

     



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

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

    BridgeBio's four core value drivers:

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

    Recent pipeline progress and corporate updates:

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

    Third Quarter 2021 Financial Results:

    Cash, Cash Equivalents and Marketable Securities

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

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

    Operating Costs and Expenses

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

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

     

     

     

    BRIDGEBIO PHARMA, INC.

    Condensed Consolidated Statements of Operations

    (in thousands, except shares and per share amounts)

     





    Three Months Ended September 30,





    Nine Months Ended September 30,





    2021





    2020





    2021





    2020





    (Unaudited)





    (Unaudited)



    Revenue

    $

    2,344





    $

    8,127





    $

    56,830





    $

    8,127



    Operating costs and expenses:























    Research, development and others



    105,759







    92,050







    330,387







    246,873



    Selling, general and administrative



    46,084







    36,016







    137,461







    108,247



    Total operating costs and expenses



    151,843







    128,066







    467,848







    355,120



    Loss from operations



    (149,499)







    (119,939)







    (411,018)







    (346,993)



    Other income (expense), net:























    Interest income



    234







    692







    951







    3,567



    Interest expense



    (11,067)







    (10,929)







    (31,644)







    (25,693)



    Other income



    (684)







    9







    7,539







    (1,344)



    Total other income (expense), net



    (11,517)







    (10,228)







    (23,154)







    (23,470)



    Net loss



    (161,016)







    (130,167)







    (434,172)







    (370,463)



    Net loss attributable to redeemable convertible

       noncontrolling interests and noncontrolling

       interests



    5,081







    14,308







    18,810







    41,720



    Net loss attributable to common stockholders

       of BridgeBio

    $

    (155,935)





    $

    (115,859)





    $

    (415,362)





    $

    (328,743)



    Net loss per share, basic and diluted

    $

    (1.06)





    $

    (0.98)





    $

    (2.88)





    $

    (2.79)



    Weighted-average shares used in computing net

       loss per share, basic and diluted



    146,662,756







    118,168,063







    144,044,360







    117,663,038



     

     

    BRIDGEBIO PHARMA, INC.

    Condensed Consolidated Balance Sheets

    (In thousands)

     







    September 30,





    December 31,









    2021





    2020



    Assets





    (Unaudited)





    (1)



    Cash and cash equivalents and marketable securities





    $

    599,611





    $

    607,093



    Receivable from licensing and collaboration agreements







    7,710









    Receivable from a related party







    462









    Prepaid expenses and other current assets







    31,927







    35,731



    Investment in equity securities







    17,707









    Property and equipment, net







    27,741







    20,325



    Operating lease right-of-use assets







    17,108







    16,508



    Intangible assets, net







    46,204









    Other assets







    33,054







    23,931



    Total assets





    $

    781,524





    $

    703,588



    Liabilities, Redeemable Convertible

       Noncontrolling Interests and

       Stockholders' Equity (Deficit)















    Accounts payable





    $

    12,035





    $

    8,945



    Accrued liabilities







    78,789







    75,900



    LEO call option liability













    5,550



    Operating lease liabilities







    23,682







    18,472



    Term loans, current portion













    1,458



    2029 Notes







    732,659









    2027 Notes







    539,517







    383,436



    Term loans, net of current portion







    103,138







    92,421



    Other long-term liabilities







    27,578







    9,520



    Redeemable convertible noncontrolling interests







    2,967







    1,630



    Total BridgeBio stockholders' equity (deficit)







    (743,397)







    57,906



    Noncontrolling interests







    4,556







    48,350



    Total liabilities, redeemable convertible

       noncontrolling interests and stockholders'

       equity (deficit)





    $

    781,524





    $

    703,588



























    (1)

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

     

    About BridgeBio Pharma, Inc.

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

    BridgeBio Pharma, Inc. Forward-Looking Statements

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

    BridgeBio Media Contact:

    Grace Rauh



    (917) 232-5478

    BridgeBio Investor Contact:

    Katherine Yau

     

    (516) 554-5989

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/bridgebio-pharma-reports-third-quarter-2021-financial-results-and-business-update-301415953.html

    SOURCE BridgeBio

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

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

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

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

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

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

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

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

    About BBP-812

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

    About Canavan Disease

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

    About BridgeBio Pharma, Inc.

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

    BridgeBio Pharma, Inc. Forward-Looking Statements

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

    BridgeBio Media Contact:

    Grace Rauh



    (917) 232-5478

    BridgeBio Investor Contact:

    Katherine Yau



    (516) 554-5989

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/bridgebio-pharma-announces-dosing-of-first-patient-in-phase-12-trial-of-investigational-gene-therapy-for-canavan-disease-301414761.html

    SOURCE BridgeBio

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

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

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

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

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

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

    Columbia University 

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

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

    Mount Sinai Health System 

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

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

    About BridgeBio Pharma, Inc. 

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

    BridgeBio Pharma, Inc. Forward-Looking Statements

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

    BridgeBio Media Contact:

    Grace Rauh



    (917) 232-5478

    BridgeBio Investor Contact: 

    Katherine Yau 

     

    (516) 554-5989

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/bridgebio-pharma-announces-collaborations-with-columbia-university-and-mount-sinai-to-develop-potential-therapies-for-genetic-diseases-and-cancers-301411543.html

    SOURCE BridgeBio

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