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

Palo Alto, CA, March 2, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that on March 1, 2021, the compensation committee of BridgeBio’s board of directors granted 17 new employees restricted stock units for an aggregate of 30,510 shares of the Company’s common stock. All of the above-described awards were made under BridgeBio’s 2019 Inducement Equity Plan (the Plan).

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

About BridgeBio
BridgeBio is a 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 30 development programs includes product candidates ranging from early discovery to late-stage development. For more information visit bridgebio.com.

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

BridgeBio Pharma and Affiliate Origin Biosciences Announce FDA Approval of NULIBRY™ (fosdenopterin), the First and Only Approved Therapy to Reduce the Risk of Mortality in Patients with MoCD Type A

Molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening genetic disorder that progresses rapidly, results in severe and largely irreversible neurological injury, and has a high infant mortality rate; median overall survival age is about four years

Clinical trials with NULIBRY or recombinant cPMP showed a meaningful increase in overall survival compared to a natural history study

NULIBRY is BridgeBio’s first FDA-approved therapeutic

PALO ALTO, February 28, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio) and affiliate Origin Biosciences, Inc. (Origin) today announced the U.S. Food and Drug Administration (FDA) has approved NULIBRY™ (fosdenopterin) for Injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. This is the first therapy of its kind. The novel therapy was developed based on BridgeBio’s commitment to developing a treatment for MoCD Type A in collaboration with the experts and families in the MoCD Type A community. The announcement was made on Rare Disease Day, which aims to raise awareness about the impact of rare diseases on patients.

MoCD Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years. MoCD Type A presents shortly after birth, often with severe encephalopathy and intractable seizures. NULIBRY is a first-in-class approved cPMP substrate replacement therapy.

“The FDA’s approval of NULIBRY means that patients with MoCD Type A and their families have an approved therapy for the first time. It also reflects our belief that every life matters and that no disease is too rare to address. As is often true in rare disease drug development, this was a community effort in which we were able to play a part – we’d like to thank the patients, caregivers, physicians, scientists, and advocates who played an essential role in achieving this important milestone,” said BridgeBio founder and CEO Neil Kumar, Ph.D.

The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical trials compared to data from a natural history study. In these studies, NULIBRY or recombinant cPMP (rcPMP; same active moiety and biologic activity as NULIBRY) reduced the risk of death by 82% compared to the untreated, genotype-matched, historical control group in the natural history study (HR=0.18, 95% CI 0.04, 0.72). At three years on study, the probability of survival in NULIBRY or rcPMP-treated patients (n=13) was 84% (95% CI 49%,96%) compared to 55% (95% CI 30%,74%) for untreated genotype-matched patients in the historical control group (n=18) at three years (Figure 1). In addition to the survival analysis, treatment with NULIBRY led to a reduction of urine concentrations of S-sulfocysteine (SSC), a toxic substance that leads to neurological damage, in patients with MoCD Type A, and the reduction was sustained with long-term treatment over 48 months.  

Animal studies have identified that NULIBRY has phototoxic potential. In the clinical trials, the most common adverse reactions reported in two or more Nulibry-treated patients with MoCD Type A were catheter-related complications (89%), pyrexia (fever; 78%), viral infection (56%), pneumonia (44%), otitis media (ear infection; 44%), vomiting (44%) and cough/sneezing (44%).  Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients.

“Today’s approval represents new hope for patients and families affected with MoCD Type A,” said Donald Basel, M.D., section chief and associate professor of pediatrics at Children’s Wisconsin. “I am encouraged by the clinical data showing that NULIBRY not only lowers the levels of toxic SSC, but importantly extends the lives of patients who previously had only a three- to four-year median survival.” 

NULIBRY was reviewed under Priority Review and received Orphan Drug Designation, Breakthrough Therapy Designation and Rare Pediatric Disease Designation from the FDA. With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Origin.

BridgeBio and Origin are committed to patient access and have developed a comprehensive patient support program, ForgingBridges, to help patients access NULIBRY. ForgingBridges also provides tools and resources to help patients and caregivers throughout their treatment journey with NULIBRY.

Visit NULIBRY.com for more information, including full Prescribing Information.

About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene (MOCS1) and characterized by a deficiency in molybdenum cofactor (MoCo) production, leading to a lack of molybdenum-dependent enzyme activity.1,2  The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine (SSC)) in the brain, which causes irreversible neurological damage.2

MoCD Type A is an ultra-rare disease. The incidence and prevalence of MoCD Type A in the United States are not known, but the estimated incidence is 1 per 342,000 to 411,000 live births (0.24 and 0.29 per 100,000).3 Based on these estimates, MoCD Type A is likely to be underdiagnosed, with an estimated 22 to 26 missed diagnoses per year in the United States and European Union.

The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.

About NULIBRY (fosdenopterin) for Injection

NULIBRY (fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Potential for Photosensitivity

NULIBRY can make the patient oversensitive to sunlight. NULIBRY-treated patients or their caregivers are advised to avoid or minimize patient exposure to sunlight and artificial UV light and adopt precautionary measures when exposed to the sun, including wearing protective clothing and sunglasses, and use broad-spectrum sunscreen with high SPF in patients 6 months of age and older. If photosensitivity occurs, caregivers/patients are advised to seek medical attention immediately and consider a dermatological evaluation.

ADVERSE REACTIONS

The most common adverse reactions in NULIBRY-treated patients were infusion catheter–related complications (89%), pyrexia (fever) (78%), viral infection (56%), pneumonia (44%), otitis media (ear infection) (44%), vomiting (44%), and cough/sneezing (44%). Adverse reactions for rcPMP-treated patients were similar to the NULIBRY-treated patients.

PATIENT COUNSELING INFORMATION

Please read the FDA-approved NULIBRY Prescribing Information and Instructions for Use and follow the instructions on how to prepare and administer NULIBRY.

NULIBRY has a potential for photosensitivity; see Warnings and Precautions. Seek medical attention immediately if the patient develops a rash or if they notice symptoms of photosensitivity reactions (redness, burning sensation of the skin, blisters).

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Clinical Trials4  

The efficacy of NULIBRY for the treatment of patients with MoCD Type A was established based on data from three clinical trials compared to data from a natural history study. 

Study 1: Study 1 was a prospective, open-label, single-arm, dose-escalation study in patients with MoCD Type A who were receiving treatment with rcPMP prior to treatment with NULIBRY.  Study 1 included eight patients, six of whom previously participated in Study 3. The initial NULIBRY dosage was matched to the patient’s rcPMP dosage upon entering the study. The NULIBRY dosage was then titrated over a period of five months to a maximum dosage of 0.9 mg/kg administered once daily as an intravenous infusion. 

Study 2: Study 2 was a prospective, open-label, single-arm, dose-escalation study in one patient with confirmed MoCD Type A who had not been previously treated with rcPMP. The initial dosage of NULIBRY in Study 2 was based on the gestational age of the patient (i.e., 36 weeks).  The initial dosage was then incrementally escalated up to a maximum dosage of 0.98 mg/kg administered once daily as an intravenous infusion (1.1 times the maximum approved recommended dosage).

Study 3: Study 3 was a retrospective, observational study that included 10 patients with a confirmed diagnosis of MoCD Type A who received rcPMP. Six of these 10 patients were later enrolled in Study 1 to receive treatment with NULIBRY.

Figure 1: Kaplan Meier Curve for Overall Survival in Patients with MoCD Type A Treated with NULIBRY (or rcPMP) Versus Genotype-Matched Untreated Patients in Historical Control

NULIBRY Efficacy and Safety Data4

The efficacy of NULIBRY and rcPMP were assessed in a combined analysis of the 13 patients with genetically confirmed MoCD Type A from Study 1 (n=8), Study 2 (n=1), and Study 3 (n=4), who received substrate replacement therapy with NULIBRY or rcPMP.  

Of the 13 treated patients included in the combined analysis, 54% were male, 77% were White and 23% were Asian; the median gestational age was 39 weeks (range 35 to 41 weeks). Of these 13 treated patients, the age at first dose was ≤ 14 days for 10 patients (with five patients initiating treatment at one day of age) and ≥32 days and <69 days for the remaining three patients. 

Efficacy was assessed by comparing overall survival in pediatric patients treated with NULIBRY or rcPMP (n=13) with an untreated natural history cohort of pediatric patients with genetically confirmed MoCD Type A who were genotype-matched to the treated patients (n=18). Patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group. Results were similar when comparing treated patients with all patients in the untreated natural history cohort with genetically confirmed MoCD Type A (n=37, including the 18 genotype-matched untreated patients as well as 19 additional untreated patients who were not genotype-matched).

Treatment with NULIBRY resulted in a reduction of urine concentrations of SSC in patients with MoCD Type A, and the reduction was sustained with long-term treatment over 48 months. The baseline level of urinary SSC normalized to creatinine was characterized in one patient (Study 2) with a value of 89.8 umol/mmol. Following treatment with NULIBRY in Studies 1 and 2 (n=9), the mean±SD levels of urinary SSC normalized to creatinine ranged from 11 (±8.5) to 7 (±2.4) umol/mmol from month 3 to month 48. 

The safety of NULIBRY was assessed in 37 pediatric patients and healthy adults who received at least one intravenous infusion of NULIBRY or rcPMP. Of these 37 patients/healthy adults, 13 were pediatric patients with MoCD Type A in Studies 1, 2 and 3; six were pediatric patients with presumptive MoCD Type A but who were later confirmed to not have MoCD type A; and 18 were healthy adults (without MoCD Type A) in a Phase 1 study. 

The most common adverse reactions from NULIBRY-treated patients in Studies 1 and 2 were catheter-related complications, pyrexia (fever), viral infection, pneumonia, otitis media (ear infection), vomiting and cough/sneezing. Adverse reactions for the rcPMP-treated patients from Study 3 were similar to the NULIBRY-treated patients, with the exception of the following additional adverse reactions that were reported in more than one patient: sepsis, oral candidiasis, varicella, fungal skin infection and eczema.

About Origin Biosciences, Inc.
Origin Biosciences, Inc., an affiliate of BridgeBio Pharma, Inc., is a biotechnology company that developed and commercialized NULIBRY for the treatment of patients with a diagnosis or presumptive diagnosis of MoCD Type A. Origin Biosciences, Inc. is led by a team of veteran biotechnology executives. For more information on Origin Biosciences, Inc., please visit the company’s website at www.origintx.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 30 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 development by Origin Biosciences, Inc. (Origin) of NULIBRY™ (fosdenopterin) for Injection as the first therapy approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A; the potential for NULIBRY as the first and only FDA approved therapy for MoCD Type A; the efficacy of NULIBRY for the treatment of patients with MoCD Type A; the safety profile of NULIBRY for the treatment of patients with MoCD Type A, including the most common adverse reactions; BridgeBio’s belief that every life matters and that no disease is too rare to address; the potential for NULIBRY to lower the levels of toxic S-sulfocysteine (SSC) and extend the lives of treated patients; the ability of NULIBRY to retain Orphan Drug Designation, Breakthrough Therapy Designation and Rare Pediatric Disease Designation from the FDA; the ability of ForgingBridges to be a comprehensive patient support program and help patients access NULIBRY; plans for the supply, manufacturing and distribution of NULIBRY; the incidence and prevalence of MoCD Type A; the current FDA-approved NULIBRY Prescribing Information and Instructions for Use; the planned approval of NULIBRY by foreign regulatory authorities and the necessary clinical trial results, and timing and completion of regulatory submissions related thereto; and the competitive environment and clinical and therapeutic potential of NULIBRY, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made.  Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved.  Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation: the fact that there has never been an approved therapy for MoCD Type A; the safety, tolerability and efficacy profile of NULIBRY observed to date may change adversely in ongoing analyses of trial data or subsequent to commercialization; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with Origin’s regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; Origin may encounter delays in meeting manufacturing or supply timelines or disruptions in its distribution plans for NULIBRY; whether and when any regulatory submissions may be filed in various foreign jurisdictions and ultimately approved by foreign regulatory authorities; and potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and clinical trials, supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy;  as well as those set forth in the Risk Factors section of BridgeBio Pharma, Inc.’s most recent Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent SEC filings, which are available on the SEC’s website at www.sec.gov.  Except as required by law, each of BridgeBio and Origin disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. Moreover, BridgeBio and Origin operate in a very competitive environment in which new risks emerge from time to time. These forward-looking statements are based on each of BridgeBio’s and Origin’s current expectations, and speak only as of the date hereof.

References
1Mechler K et al. Genet Med. 2015;17(12):965-970.
2Schwarz G. Cur Op in Che Bio. 2016;31:179-187.
3Mayr SJ, et al. Forecasting the incidence of rare diseases: an iterative computational and biochemical approach in molybdenum cofactor deficiency type A. Presented at the 2019 SSIEM meeting; September 3-6, 2019; Rotterdam, The Netherlands.
4NULIBRY (fosdenopterin) Origin Biosciences, Palo Alto, CA, USA; February 2021.

NOTE: NULIBRY is a trademark of Origin Biosciences, Inc. Origin Biosciences, Inc. is a member of the BridgeBio family. ForgingBridges is a trademark of BridgeBio.

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

BridgeBio Pharma, Inc. Reports Fourth Quarter And Full Year 2020 Financial Results And Business Update

Completed acquisition of Eidos Therapeutics, allowing BridgeBio to deploy its full clinical and commercial infrastructure to develop and plan for potential global commercialization upon regulatory approval of Eidos’ product candidate, acoramidis, a potential best-in-class therapy for patients with amyloidosis cardiomyopathy (ATTR-CM)

New Drug Application (NDA) for infigratinib for the treatment of cholangiocarcinoma accepted by the U.S. Food and Drug Administration (FDA) under Priority Review designation and Real-Time Oncology Review (RTOR) pilot program, designed to expedite the delivery of safe and effective cancer treatments to patients

Initiated two new clinical trials since last quarterly update and progressed additional 17 ongoing clinical trials

Ended quarter with $607.1 million in cash, cash equivalents and marketable securities before issuance of 2.25% Convertible Senior Notes in February 2021, which raised nearly $750 million in gross proceeds

PALO ALTO, Calif., FEBRUARY 25, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today reported its financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on the Company’s operations.

Since its last quarterly update, BridgeBio completed its acquisition of Eidos Therapeutics, Inc. (formerly Nasdaq: EIDX) (Eidos) in January 2021, acquiring all of the outstanding shares of Eidos common stock that BridgeBio did not already own. The merger returns BridgeBio and Eidos to a single unified company and is intended to allow BridgeBio to unlock the full potential of acoramidis, a potential best-in-class therapy for patients with transthyretin (TTR) amyloidosis (ATTR). 

Acoramidis for ATTR is one of BridgeBio’s four core value driver programs along with encaleret (CaSR inhibitor) for autosomal dominant hypocalcemia type 1 (ADH1), low-dose infigratinib (FGFR inhibitor) for achondroplasia, and BBP-631, an AAV5 gene therapy for congenital adrenal hyperplasia (CAH). Pivotal or potential proof-of-concept data is anticipated for each of the programs by the end of 2021 or early 2022.

BridgeBio had its second NDA accepted by the FDA and is preparing for its first two commercial launches this year, pending FDA approval, for infigratinib for the treatment of cholangiocarcinoma (CCA), or bile duct cancer, as a second-line or later therapy in patients with advanced and/or metastatic CCA with FGFR2 fusions or translocations, and for fosdenopterin for the treatment of MoCD Type A, an ultra-rare, life-threatening genetic disorder that results in severe and largely irreversible neurological injury for infants and children. Additionally, the Company initiated two new clinical trials and progressed 17 additional ongoing trials. BridgeBio established a joint venture with Maze Therapeutics to advance precision medicine to treat cardiovascular disease and entered into a partnership agreement with the University of California, San Francisco to drive the advancement of academic innovations in genetically driven diseases into potential therapeutics for patients.

“We are hopeful that the scientific innovation we are pursuing at scale begins to translate into meaningful gains for patients this year. We are on track to deliver near-term pivotal or potential proof-of-concept data in our four core value driver programs. The successful completion of our acquisition of Eidos allows us to further focus BridgeBio’s clinical and commercialization engine on acoramidis for patients suffering from ATTR,” said BridgeBio CEO and founder, Neil Kumar, Ph.D. “We are also starting the year in a strong financial position following our recent debt financing, which enables us to progress the 19 ongoing clinical trials and over 30 programs in our pipeline, as well as to prepare for the anticipated launch of our first two drugs, if approved.”

Major milestones anticipated in 2021 or early 2022 for BridgeBio’s four core value drivers:

  • Acoramidis (AG10) – TTR stabilizer for ATTR-CM: Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 or early 2022 and from Part B in 2023. If Part A is successful, BridgeBio expects to submit an application for regulatory approval of acoramidis in 2022. ATTR is a form of amyloidosis caused by the accumulation of misfolded TTR protein. It is estimated to affect more than 400,000 people in the United States and the European Union and is largely undiagnosed today.
  • Encaleret – calcium-sensing receptor (CaSR) inhibitor for ADH1: Early results from an ongoing Phase 2 proof-of-concept study will be shared at the Endocrine Society’s 2021 Annual Meeting (ENDO) on March 20th. If the development program is successful, encaleret could be the first approved therapy for ADH1, a condition caused by gain of function variants in the CaSR gene estimated to be carried by 12,000 individuals in the United States alone.
  • Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the second half of 2021. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known product candidate in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical-stage development.
  • BBP-631 – AAV5 gene therapy candidate for CAH: Initiation of a first-in-human Phase 1/2 study is expected in the second half of 2021, with initial data anticipated in late 2021 or early 2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases 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:

  • Completed acquisition of Eidos Therapeutics in January 2021, acquiring of all of the outstanding shares of Eidos common stock that BridgeBio did not already own. The merger returns Eidos to BridgeBio’s vibrant ecosystem of innovation and is intended to allow BridgeBio to deploy its full clinical and commercial infrastructure to support the development and global commercialization plans underway for Eidos’ product candidate, acoramidis, a potential best-in-class therapy for patients with ATTR-CM.

  • Raised nearly $750 million in gross proceeds in February 2021 through issuance of 2.25% Convertible Senior Notes due in 2029. The Company expects current cash, cash equivalents and marketable securities to support its planned operations into 2023.

  • NDA accepted by the FDA for infigratinib, an oral FGFR1-3 selective inhibitor, for individuals with CCA as a second-line or later therapy in patients with advanced and/or metastatic CCA with FGFR2 fusions or translocations. The NDA has been granted Priority Review designation and is being reviewed under the Real-Time Oncology Review (RTOR) pilot program. BridgeBio has also submitted for regulatory review in Australia and Canada under Project Orbis, an initiative of the FDA’s Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies.
  • BBP-681 – Topical PI3Ka inhibitor for venous malformations (VMs), lymphatic malformations (LMs), and venolymphatic malformations (VLMs) associated with PIK3CA or TEK mutations: Dosed first patient in Phase 1/2 clinical trial.

  • BBP-398 – SHP2 inhibitor for tumors driven by RAS and receptor tyrosine kinase mutations: Dosed first patient in Phase 1 clinical trial.

  • Established Contour Therapeutics, a joint venture between BridgeBio and Maze Therapeutics, focused on transforming and advancing breakthrough precision medicine approaches designed to treat cardiovascular disease, the leading cause of death worldwide.
  • Established collaboration agreement with the University of California, San Francisco to advance the discovery of therapies for genetically driven diseases.

Fourth Quarter and Full-Year 2020 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $607.1 million as of December 31, 2020, compared to $577.1 million as of December 31, 2019. The net increase in cash balance of $30.0 million reflects $537.0 million in net proceeds received from the issuance of our 2.50% Convertible Senior Notes due 2027 (2027 Notes), $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares in capped call transactions in connection with the issuance of our 2027 Notes, $49.3 million payment related to capped call option, $15.3 million payments of interest on our debts, and $391.5 million primarily related to operating expenses. 

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $710.7 million as of September 30, 2020, resulting in a decrease of $103.6 million as compared to December 31, 2020. The decrease in cash reflects $2.0 million payments of interests on our debts and $109.6 million primarily relating to operating expenses, partially offset by a receipt of $8.0 million in upfront payment under the license agreement with LianBio.

Operating Expenses

Operating expenses for the three months and year ended December 31, 2020 were $127.6 million and $482.7 million, respectively, as compared to $92.5 million and $306.8 million, respectively, for the same periods in the prior year. The increases in operating expenses of $35.1 million and $175.9 million during the respective periods were attributable to the increase in external-related costs, including manufacturing validation activities for our late-stage programs, and increase in personnel costs resulting from an increase in the number of employees to support the progression in our research and development programs, including our increasing research pipelines, and overall growth of our operations.

Operating expenses for the three months ended December 31, 2020 decreased by $0.5 million when compared to the operating expenses for the three months ended September 30, 2020 of $128.1 million.

Our research and development expenses have not been significantly impacted by the global outbreak of COVID-19 for the periods presented. While we experienced some delays in certain of our clinical enrollment and trial commencement activities, we continue to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with our contract manufacturing organizations. The longer-term impact of COVID-19 on our operating expenses is currently unknown.

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.

BridgeBio Pharma Forward-Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of our product candidates, including, but not limited to: the unknown future impact of the COVID-19 pandemic delay on certain clinical trial milestones and/or our operations or operating expenses; uncertainty of the expected financial performance of each of us and Eidos following completion of the merger, including the possibility that the expected synergies and value creation from the merger will not be realized or will not be realized within the expected time period; the timing and success of our planned preclinical and clinical development of our development programs, and the timing and success of any such continued preclinical and clinical development and planned regulatory submissions, including for each of acoramidis, infigratinib, BBP-631 and encaleret; the potential therapeutic and clinical benefits of each of acoramidis, infigratinib, BBP-631 and encaleret; the potential size of the target patient populations for each of acoramidis, infigratinib, BBP-631 and encaleret; the timing and approval for commercialization this year for each of infigratinib for the treatment of cholangiocarcinoma and fosdenopterin for the treatment of MoCD Type A; the number of potential medicines in our portfolio; the success of our joint venture with Maze Therapeutics through Contour Therapeutics, our partnership with the University of California, San Francisco, and our other collaboration agreements with various academic institutions; our ability to produce meaningful medicines; the potential for encaleret to be the first approved therapy for ADH1; our expected runway for cash, cash equivalents and marketable securities; our ability to advance infigratinib through the Real-Time Oncology Review pilot program and under Project Orbis; the timing and success of our Phase 1/2 clinical trial of BBP-681; the timing and success of our Phase 1 clinical trial of BBP-398; 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 success of clinical trials, regulatory filings, approvals and/or sales; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those risks set forth in the Risk Factors section of our most recent quarterly or annual periodic report filed with the SEC and our other SEC filings. In addition, the information contained in this release and the condensed consolidated balance sheet information is unaudited and does not present all information necessary for an understanding of the Company’s financial condition as of December 31, 2020 and its results of operations for the three months and year ended December 31, 2020. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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


BridgeBio Pharma To Announce Proof-Of-Concept Data from ADH1 at the Endocrine Society’s 2021 Annual Meeting

– Company to Host Webcast to Discuss Proof-of-Concept Data for Encaleret in Autosomal Dominant Hypocalcemia Type 1 on March 22 at 8:00 a.m. ET

SAN FRANCISCO, CA – February 24, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that early results from an ongoing Phase 2 proof-of-concept study of encaleret for the treatment of Autosomal Dominant Hypocalcemia Type 1 (ADH1) will be shared at the upcoming Endocrine Society’s 2021 Annual Meeting (ENDO 2021) taking place virtually from March 20 – 23.

The data are featured in an ePoster presentation titled ‘The Effects of Encaleret (CLTX-305) on Mineral Physiology in ADH1 Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study.’ If the development program is successful, encaleret could be the first approved therapy for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor gene estimated to be carried by 12,000 individuals in the United States.

Full ePoster presentation details are listed below, and the full preliminary program is available online at the ENDO 2021 website. The presentations will be on display in ENDO 2021’s virtual poster hall starting on March 20 at 11:00 a.m. ET.

BridgeBio will host an investor webcast on March 22 at 8:00 a.m. ET to discuss the proof-of-concept data for encaleret in ADH1.

Additionally at ENDO 2021, BridgeBio will present clinical study designs for its study of low-dose infigratinib, an FGFR1-3 inhibitor, for children with achondroplasia, the most common form of genetic short stature, and for its investigational AAV5 gene therapy candidate for Congenital Adrenal Hyperplasia (CAH). CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy.

BridgeBio ePoster Presentation Details:

The Effects of Encaleret (CLTX-305) on Mineral Physiology in ADH1 Demonstrate Proof-of-Concept: Early Results from an Ongoing Phase 2B, Open-Label, Dose-Ranging Study

Presenter: Rachel Gafni, M.D., Senior Physician of Skeletal Disorders and Mineral Homeostasis of the National Institute of Dental and Craniofacial Research, part of the National Institutes of Health

Poster Session & Number: P08. Parathyroid and Rare Bone Disorders, Abstract #8545

A Phase 2B, Open-Label, Dose-Ranging Study of Encaleret (CLTX-305) in ADH1

Presenter: Rachel Gafni, M.D., Senior Physician of Skeletal Disorders and Mineral Homeostasis of the National Institute of Dental and Craniofacial Research, part of the National Institutes of Health

Poster Session & Number: P08. Parathyroid and Rare Bone Disorders, Abstract #7288

Infigratinib in Children with Achondroplasia (ACH): Design of PROPEL2 – A Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study

Presenter: Ravi Savarirayan, M.D., Ph.D., Clinical Geneticist and Group Leader of Skeletal Biology and Disease at Murdoch Children’s Research Institute

Poster Session & Number: P34. Disorders of Puberty, Abstract #6897

Design of a Phase 1/2 Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy in Adults with Classic Congenital Adrenal Hyperplasia (CAH) Due to 21-hydroxylase Deficiency Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene

Presenter: Deborah Merke, M.D., Senior Investigator, Chief of Pediatric Service and Head of Congenital Disorders at the NIH Clinical Center

Poster Session & Number: P05. Adrenal – Clinical Research Studies, Abstract #8640

Webcast Information
BridgeBio will host a conference call and simultaneous webcast to share updates on the encaleret proof-of-concept data in ADH1 on March 22 at 8:00 a.m. ET. To access this call, dial (800) 379-2666 (U.S.) or (409) 937-8964 (International). Conference ID: 7371879. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event. 

About 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, please visit www.bridgebio.com.

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans and prospects regarding the preclinical and clinical development plans, clinical trial designs, clinical and therapeutic potential, and strategy of our product candidates, including, but not limited to: the unknown future impact of the COVID-19 pandemic delay on certain clinical trial milestones and/or our operations or operating expenses;; the timing and success of our planned preclinical and clinical development of our development programs, including each of infigratinib, BBP-631 and encaleret; the timing and success of any such continued preclinical and clinical development and planned regulatory submissions, including for each of infigratinib, BBP-631 and encaleret; the potential therapeutic and clinical benefits of each of infigratinib, BBP-631 and encaleret; the potential size of the target patient populations for each of infigratinib, BBP-631 and encaleret; the potential for encaleret to be the first approved therapy for ADH1; our expected runway for cash, cash equivalents and marketable securities; and the timing of these events, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by  a number of risks, uncertainties and assumptions, including, but not limited to: the success of clinical trials, regulatory filings, approvals and/or sales; despite having ongoing interactions with the FDA or other regulatory agencies, the FDA or such other regulatory agencies may not agree with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted; potential adverse impacts due to the global COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy; and those risks set forth in the Risk Factors section of our most recent quarterly or annual periodic report filed with the SEC and our other SEC filings. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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

BridgeBio Pharma and Affiliate ML Bio Solutions Announce Dosing of First Patient in Phase 2 Trial of BBP-418 in Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)

SAN FRANCISCO, CA – February 19, 2020 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, and affiliate ML Bio Solutions today announced that the first patient has been dosed in a Phase 2 trial of BBP-418 in patients with LGMD2i. BridgeBio and ML Bio’s BBP-418 is the first-ever oral disease-modifying investigational treatment for LGMD2i. BBP-418 was granted Orphan Drug Designation for LGMD2i by the US Food and Drug Administration (FDA) in 2019, and for LGMD by the European Medicines Agency (EMA) in 2020.

LGMD2i is an inherited recessive muscular dystrophy caused by mutation of fukutin-related protein (FKRP). FKRP is an enzyme that adds a sugar molecule to a critical muscle cell structural protein called alpha-dystroglycan (αDG). Due to defective FKRP enzyme function, muscle cells of patients affected by LGMD2i lack the structural integrity that is provided by fully glycosylated αDG protein. This leads to chronic muscle injury and loss, muscle weakness and disability. Many affected patients ultimately lose their ability to walk, and some develop a need for ventilatory support or die from heart failure. Currently, there are no disease-modifying therapies for people with LGMD2i. 

BBP-418 is designed to bypass the metabolic defect in LGMD2i by providing the FKRP enzyme with precursor sugar molecules that supplement the body’s natural sugars used by FKRP to glycosylate the αDG protein on muscle cells. BBP-418 represents a novel substrate rescue approach with the potential to improve muscle strength and function.

The Phase 2 trial is expected to enroll up to 16 patients with a genetically-confirmed diagnosis of LGMD2i. In addition to safety, key efficacy measurements include changes in muscle αDG glycosylation levels, and changes in functional measures including 10 meter walk and North Star for Dysferlinopathy (NSAD).

“The start of our Phase 2 trial is a key milestone in our effort to develop a safe and efficacious therapy for patients with LGMD2i who lack treatment options,” said Dr. Douglas Sproule, M.D. M.Sc., Chief Medical Officer of ML Bio Solutions. “This trial launch is an outgrowth of our strong partnership with the GRASP-LGMD consortium based at Virginia Commonwealth University, where we are currently enrolling a lead-in study to define LGMD2i-specific biomarkers and functional endpoints. Our hope is that the ongoing lead-in study, together with early data from our Phase 2 trial, will hasten the successful execution of a future registrational trial and ultimately advance a meaningful medicine for patients in need.” 

“We are pleased to continue our collaboration with ML Bio Solutions on the lead-in study that is currently enrolling and look forward to further extending it by launching the first-in-patient treatment study for BBP-418. Our clinical partnership is seeking to validate the biomarker assays and establish the registrational clinical outcome measures that are needed to bring BBP-418 to LGMD2i patients,” said Dr. Nicholas Johnson M.D. M.S.C.I., Associate Professor of Neurology and Chair of Research at Virginia Commonwealth University and Director of the GRASP-LGMD consortium.  

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 ML Bio Solutions 

ML Bio Solutions, an affiliate of BridgeBio Pharma based in a Charlotte, NC, 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 2 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 2 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 2 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 BioSolutions operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. 

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

BridgeBio Pharma, Inc. Announces Pricing of Secondary Offering of Common Stock

PALO ALTO, Calif. – February 12, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (the “Company,” “we” or “BridgeBio”) announced today the pricing of a secondary public offering of 3,000,000 shares of its common stock at a price per share of $62.50 by selling stockholder KKR Genetic Disorder L.P. The selling stockholder has also granted the underwriters a 30-day option to purchase up to 450,000 additional shares of common stock on the same terms and conditions. All shares are being sold by KKR Genetic Disorder L.P. The Company is not selling any shares and will not receive any of the proceeds of the offering. The offering is expected to close on February 17, 2021, subject to customary closing conditions.

Goldman Sachs & Co. LLC is acting as the sole book-running manager, and KKR Capital Markets LLC and Raymond James & Associates, Inc. are acting as co-managers for the offering.

The securities described above are being offered pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-240147) that was previously filed by the Company with the Securities and Exchange Commission (the “SEC”) and automatically became effective upon filing on July 28, 2020.

A preliminary prospectus supplement and accompanying prospectus relating to the offering are filed with the SEC and are available on the SEC’s website at http://www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus and, when available, copies of the final prospectus supplement and accompanying prospectus can be obtained by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at 1 866 471 2526, or by email at prospectusny@ny.email.gs.com.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities described above, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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.

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 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 reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. 

Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 and our other filings with the SEC. 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.

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

BridgeBio Pharma, Inc. Announces Launch of Secondary Offering of Common Stock

PALO ALTO, Calif. – February 11, 2021 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) (the “Company,” “we” or “BridgeBio”) announced today the launch of a secondary public offering of 3,000,000 shares of its common stock by selling stockholder KKR Genetic Disorder L.P. The selling stockholder has also granted the underwriters a 30-day option to purchase up to 450,000 additional shares of common stock. All shares are being sold by KKR Genetic Disorder L.P. The Company is not selling any shares and will not receive any of the proceeds of the offering.

Goldman Sachs & Co. LLC is acting as the sole book-running manager and KKR Capital Markets LLC and Raymond James & Associates, Inc. are acting as co-managers for the offering.

The securities described above are being offered pursuant to an automatic shelf registration statement on Form S-3 (File No. 333-240147) that was previously filed by the Company with the Securities and Exchange Commission (the “SEC”) and automatically became effective upon filing on July 28, 2020.

A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at http://www.sec.gov. A copy of the preliminary prospectus supplement and accompanying prospectus can be obtained by contacting Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at 1 866 471 2526, or by email at prospectusny@ny.email.gs.com.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities described above, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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.

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 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 reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. 

Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 and our other filings with the SEC. 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.

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

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

Palo Alto, CA, February 5, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases, today announced that on February 1, 2021, the compensation committee of BridgeBio’s board of directors granted 11 new employees restricted stock units for an aggregate of 16,772 shares of the Company’s common stock. All of the above-described awards were made under BridgeBio’s 2019 Inducement Equity Plan (the Plan).

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

About BridgeBio
BridgeBio 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

BridgeBio Pharma and Affiliate Venthera Announce Dosing of First Patient in Phase 1/2 Clinical Trial of BBP-681 for Venous, Lymphatic, and Venolymphatic Malformations

  • First-in-human Phase 1/2 trial in patients with venous malformations (VMs), lymphatic malformations (LMs), and venolymphatic malformations (VLMs) associated with PIK3CA or TEK mutations
  • Clinical trial enrolling patients across multiple sites in the United States

PALO ALTO, CA, February 2, 2021—BridgeBio Pharma, Inc. (Nasdaq: BBIO) and affiliate Venthera, which is focused on developing new treatment options for patients with rare vascular anomalies, today announced that the first subject has been dosed in its Phase 1/2 clinical trial of BBP-681 (also known as VT30 Topical Gel).

This novel investigational therapy is designed to target the genetic drivers of venous (VM), lymphatic (LM), and venolymphatic malformations (VLM) at their source by delivering a potent PI3Kα inhibitor directly to affected tissue. The majority of these cutaneous vascular malformations are driven by somatic mutations in the PIK3CA and TEK genes. These mutations result in overactivation of the PI3K pathway and have been identified as the root cause of the vast majority of VMs, LMs, and VLMs. Because BBP-681 is applied directly to skin lesions, it may minimize complications seen with systemic treatments.

Part 1 of the trial is a four-week treatment, open-label dose escalation study which will determine the dose and regimen of BBP-681 to be carried into Part 2 of the protocol. Part 2 is a 12-week treatment, randomized, placebo-controlled, double-blind, safety and exploratory efficacy study of topically administered BBP-681.

People living with VMs, LMs, and VLMs have significant unmet medical needs related to pain, functional impairment, and disfigurement. Current treatment options include laser ablation, and invasive procedures such as sclerotherapy and surgical excision. BBP-681 is the first potential therapy designed specifically to address the needs of people living with cutaneous forms of these lesions. There are 117,000 people estimated to be living with cutaneous VMs, LMs, and VLMs in the United States and Europe.

“We are dedicated to giving hope to patients by developing new treatment options and working to improve mobility and alleviate discomfort of people living with cutaneous VMs, LMs, and VLMs. The initiation of this study represents an important milestone for the patient community as we translate cutting-edge science into a clinical-stage investigational therapy designed to target these conditions at their known genetic and tissue-specific source,” said Thomas M. Rossi, Ph.D., CEO of Venthera.  


The Phase 1/2 study is currently enrolling up to 51 patients with VMs, LMs, and VLMs associated with PIK3CA or TEK gene mutations across multiple centers in the U.S. to evaluate the safety and tolerability of BBP-681. The study will also determine the dose and regimen of BBP-681 to be carried into Part 2 of the protocol. Other aims include documenting plasma drug levels of BBP-681 and its active form VT10 and, on an exploratory basis, examining pharmacologic target engagement and change in potential efficacy readouts. For more information, visit https://clinicaltrials.gov/ct2/show/NCT04409145.

About Venthera
Venthera is dedicated to improving the lives of patients living with rare vascular anomalies by suppressing the underlying pathways that drive vascular anomaly growth. By targeting the genetic root cause of disease(s), Venthera is seeking to address unmet needs and create a new option for treating VMs, LMs, and VLMs with the hope of reducing the need for invasive and systemic treatments.

As a member of the BridgeBio family, Venthera’s management team is supported by deep expertise in drug discovery, development, and innovation that advances its mission to bring safe and efficient new treatments to patients living with rare vascular anomalies.

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. For more information, visit https://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 Venthera’s Phase 1/2 clinical trial of BBP-681/VT30 for the treatment of venous malformations, lymphatic malformations, and venolymphatic malformations associated with PIK3CA or TEK mutations, expectations, plans and prospects regarding Venthera’s regulatory approval process for BBP-681/VT30, the ability of BBP-681/VT30 to treat venous malformations, lymphatic malformations, and venolymphatic malformations associated with PIK3CA or TEK mutations in humans, and the timing and success of initial top-line Phase 1/2 data of BBP-681/VT-30, 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, Venthera’s ability to continue and complete its Phase 1/2 clinical trial of BBP-681/VT30 for the treatment of venous malformations, lymphatic malformations, and venolymphatic malformations associated with PIK3CA or TEK mutations, past data from preclinical studies not being indicative of future data from clinical trials, Venthera’s ability to advance BBP-681/VT30 in clinical development according to its plans, the ability of BBP-681/VT30 to treat venous malformations, lymphatic malformations, and venolymphatic malformations associated with PIK3CA or TEK mutations, 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, Venthera 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.

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

BridgeBio Pharma, Inc. Announces Completion of Merger with Eidos Therapeutics, Inc.

PALO ALTO, CA – January 26, 2021 – BridgeBio Pharma, Inc. (“BridgeBio”) (Nasdaq: BBIO) today announced that it has completed its acquisition of all of the outstanding shares of Eidos Therapeutics, Inc. (“Eidos”) (formerly Nasdaq: EIDX) common stock that BridgeBio did not already own. The transaction was overwhelmingly approved by BridgeBio and Eidos stockholders.

The merger reunites the teams at BridgeBio and Eidos and allows BridgeBio to deploy its full clinical and commercial infrastructure to support the development and global commercialization plans underway for Eidos’ acoramidis, a potential best-in-class therapy for patients with transthyretin (TTR) amyloidosis (ATTR). BridgeBio’s mission is to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers.

“2021 is an important year for BridgeBio and the patients we serve,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. “With significant near-term pivotal and proof-of-concept data anticipated in our four core programs, including acoramidis, we are eager to accelerate our critical work for patients as a single unified company.”

Acoramidis for ATTR is one of BridgeBio’s four core value driver programs, along with encaleret (CaSR inhibitor) for autosomal dominant hypocalcemia type 1 (ADH1), low-dose infigratinib (FGFR inhibitor) for achondroplasia, and BBP-631, an AAV5 gene therapy for congenital adrenal hyperplasia (CAH). 2021 is poised to be a transformational year for BridgeBio with major catalysts in all four programs anticipated in 2021 or the first quarter of 2022. This year BridgeBio also expects to launch two drugs, if approved, and is building its global commercial capabilities.

  • Acoramidis (AG10) – TTR stabilizer for ATTR:  Topline results from Part A of the ATTRibute-CM trial are expected in late 2021 or early 2022 and from Part B in 2023. If Part A is successful, BridgeBio expects to file for regulatory approval of acoramidis in 2022. ATTR is a form of amyloidosis caused by the accumulation of misfolded TTR protein. It is estimated to affect more than 400,000 people worldwide and is largely undiagnosed today.
  • Encaleret – calcium-sensing receptor (CaSR) inhibitor for ADH1: Phase 2 proof-of-concept results are anticipated in the third quarter of 2021. If the development program is successful, encaleret would be the first approved therapy for ADH1, a condition caused by gain of function variants in the CASR gene estimated to be carried by 12,000-13,000 individuals in the United States alone.
  • Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Initial data from the ongoing Phase 2 dose ranging study are expected in the fourth quarter of 2021. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known therapy in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical stage development.
  • BBP-631 – AAV5 gene therapy candidate for CAH:  Initiation of a first-in-human Phase 1/2 study is expected in 2021, with initial data anticipated in the fourth quarter of 2021 or the first quarter of 2022. CAH is one of the most prevalent genetic diseases potentially addressable with AAV gene therapy, with more than 75,000 cases 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. Our 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.

As a result of the merger, former Eidos stockholders are entitled to receive, for each share of Eidos common stock issued and outstanding immediately prior to the effective time of the merger that was not owned by BridgeBio or any of its subsidiaries and that was not a restricted share award, either (i) 1.85 shares of BridgeBio common stock or (ii) if an election to receive cash was properly made prior to 5:00 P.M., New York City time, on January 21, 2021, $73.26 in cash. Eidos stockholders should contact American Stock Transfer & Trust Company, LLC, the exchange agent for the transaction, by calling toll-free at (877) 248-6417 or at (718) 921-8317, if they have any questions regarding the consideration to which they are entitled.

About BridgeBio Pharma

BridgeBio Pharma 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 www.bridgebio.com.

About Eidos Therapeutics

Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR). Eidos is developing acoramidis, a potentially disease-modifying therapy for the treatment of ATTR. For more information, visit www.eidostx.com.

Forward-Looking Statements

This press release contains forward-looking statements relating to the proposed transaction involving BridgeBio and Eidos, including financial estimates and statements as to the expected timing, completion and effects of the proposed transaction. Statements in this press release that are not statements of historical fact are considered forward-looking statements within the meaning of Section 27A of 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,” “continues”, “could”, “estimates,” “expects,” “intends,” “may,” “plans,” “potential”, “predicts”, “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 are neither forecasts, promises nor guarantees, and are based on the current beliefs of BridgeBio’s management as well as assumptions made by and information currently available to BridgeBio. Such statements reflect the current views of BridgeBio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about BridgeBio and Eidos, including, without limitation, (i) potential adverse effects or changes to relationships with customers, employees, suppliers or other parties resulting from the completion of the merger, (ii) potential litigation relating to the merger that could be instituted against BridgeBio, Eidos or their respective directors and officers, including the effects of any outcomes related thereto, (iii) possible disruptions from the merger that could harm BridgeBio’s or Eidos’ respective business, including current plans and operations, (iv) unexpected costs, charges or expenses resulting from the merger, (v) uncertainty of the expected financial performance of each of BridgeBio and Eidos following completion of the merger, including the possibility that the expected synergies and value creation from the merger will not be realized or will not be realized within the expected time period, (vi) the ability of BridgeBio and/or Eidos to implement their respective business strategies, (vii) the ability of each of BridgeBio or Eidos to continue its planned preclinical and clinical development of its respective development programs, and the timing and success of any such continued preclinical and clinical development and planned regulatory submissions, including for BridgeBio’s low-dose infigratinib (FGFR inhibitor) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia (CAH), acoramidis and encaleret for ADH1, (viii) the potential therapeutic and clinical benefits of each of acoramidis, infigratinib, BBP-631 and encaleret, (ix) the potential size of the target patient populations for each of acoramidis, infigratinib, BBP-631 and encaleret, (x) the potential for encaleret to be the first approved therapy for ADH1, (xi) inability to retain and hire key personnel and (xii) the unknown future impact of the COVID-19 pandemic delay on certain clinical trial milestones and/or BridgeBio’s or Eidos’ operations or operating expenses. Although BridgeBio believes that BridgeBio’s and Eidos’ plans, intentions, expectations, strategies and prospects as reflected in or suggested by these forward-looking statements are reasonable, BridgeBio cannot give any 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, without limitation, those risks and uncertainties described under the heading “Risk Factors” in BridgeBio’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K filed with the SEC and in subsequent filings made by BridgeBio with the SEC, 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. 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. We anticipate that subsequent events and developments will cause our views to change. Except as required by law, BridgeBio disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.

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

Source: BridgeBio Pharma, Inc.