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1. 02 March 2021 BridgeBio Pharma Reports Inducement Grants under Nasdaq Listing Rule 5635(c)(4)

   PALO ALTO, Calif., March 02, 2021 (GLOBE NEWSWIRE) -- 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…

   PALO ALTO, Calif., March 02, 2021 (GLOBE NEWSWIRE) -- 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
   
   
   
   (917) 232-5478

   Source: BridgeBio Pharma, Inc.

   
   
   

   

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2. 28 February 2021 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, Calif., Feb. 28, 2021 (GLOBE NEWSWIRE) -- 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…

   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, Calif., Feb. 28, 2021 (GLOBE NEWSWIRE) -- 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.

   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
   
   
   
   (917) 232-5478

   Source: BridgeBio Pharma, Inc.

   A Media Snippet accompanying this announcement is available by clicking on the image or link below:

   
   
   

   

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3. 25 February 2021 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 …

   –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., Feb. 25, 2021 (GLOBE NEWSWIRE) -- 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. (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 20^th. 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.

   
   
   

   BRIDGEBIO PHARMA, INC.
   Condensed Consolidated Statements of Operations
   (in thousands, except shares and per share amounts)
   	Three Months Ended December 31,				Year Ended December 31,
   	2020		2019		2020		2019
   	(Unaudited)				(Unaudited)		(1)
   License revenue	$122		$13,819		$8,249		$40,560
   Operating expenses:
   Cost of license revenue	—		—		—		2,500
   Research and development	90,174		57,485		337,047		209,947
   General and administrative	37,437		34,972		145,684		94,353
   Total operating expenses	127,611		92,457		482,731		306,800
   Loss from operations	(127,489)		(78,638)		(474,482)		(266,240)
   Other income (expense), net:
   Interest income	448		2,410		4,015		8,915
   Interest expense	(10,962)		(3,040)		(36,655)		(8,765)
   Share in net loss of equity method investments	—		(4,725)		—		(20,869)
   Other income (expense)	2,978		(158)		1,634		(1,626)
   Total other income (expense), net	(7,536)		(5,513)		(31,006)		(22,345)
   Net loss	(135,025)		(84,151)		(505,488)		(288,585)
   Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests	15,044		10,693		56,764		27,998
   Net loss attributable to common Stockholders of BridgeBio	$(119,981)		$(73,458)		$(448,724)		$(260,587)
   Net loss per share, basic and diluted	$(1.01)		$(0.62)		$(3.80)		$(2.48)
   Weighted-average shares used in computing net loss per share, basic and diluted	118,985,489		117,691,534		117,995,457		105,099,089

   
   
   
   
   
   
   
   
   
   
   

   BRIDGEBIO PHARMA, INC.
   Condensed Consolidated Balance Sheets
   (In thousands)
   	December 31,
   	2020		2019
   Assets	(Unaudited)		(1)
   Cash and cash equivalents and marketable securities (2)	$607,093		$577,137
   Receivable from a related party	—		2,845
   Prepaid expenses and other current assets	35,731		19,784
   Property and equipment, net	20,325		5,625
   Operating lease right-of-use assets	16,508		—
   Other assets	23,931		26,288
   Total assets	$703,588		$631,679
   Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders' Equity
   Accounts payable	$8,945		$8,852
   Accrued liabilities	75,900		39,455
   LEO call option liability	5,550		4,078
   Operating lease liabilities	18,472		—
   Term loans, current portion	1,458		—
   Build-to-suit lease obligation	—		8,000
   Term loans, net of current portion	92,421		91,791
   2027 Notes	383,436		—
   Other liabilities	9,520		3,527
   Redeemable convertible noncontrolling interests	1,630		2,243
   Total BridgeBio stockholders' equity	57,906		408,454
   Noncontrolling interests	48,350		65,279
   Total liabilities, redeemable convertible noncontrolling interests and stockholders' equity	$703,588		$631,679

   
   
   

   (1)	The condensed consolidated financial statements as of and for the year ended December 31, 2019 are derived from the audited consolidated financial statements as of that date. Certain reclassifications have been made to the condensed consolidated balance sheet as of December 31, 2019.
   (2)	December 31, 2019 amounts include long-term marketable securities of $31.1 million.

   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
   
   BridgeBio Pharma, Inc.
   
   
   
   (917) 232-5478

   Source: BridgeBio Pharma, Inc. 

   
   
   

   

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4. 24 February 2021 BridgeBio Pharma To Announce Proof-Of-Concept Data from ADH1 at the Endocrine Society's 2021 Annual Meeting

   SAN FRANCISCO, Feb. 24, 2021 (GLOBE NEWSWIRE) -- 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…

   SAN FRANCISCO, Feb. 24, 2021 (GLOBE NEWSWIRE) -- 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
   
   BridgeBio Pharma, Inc.
   
   
   
   (917) 232-5478

   Source: BridgeBio Pharma, Inc.

   
   
   

   

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5. 19 February 2021 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, Feb. 19, 2021 (GLOBE NEWSWIRE) -- 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…

   SAN FRANCISCO, Feb. 19, 2021 (GLOBE NEWSWIRE) -- 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, BridgeBio Pharma 
   
    
   
   917-232-5478 

   Source: BridgeBio Pharma  

   
   
   

   

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