Acoramidis demonstrated a significant impact on mortality, hospitalizations, and quality of life Starting at Month 3, patients taking acoramidis showed meaningful and sustained improvement in time to first event (CVH or ACM) Acoramidis demonstrated a 42% reduction in composite CVH and ACM events relative to placebo by Month 30 Increased serum TTR levels by Day 28 were correlated with a reduced risk of events (ACM, CVM, and CVH) through Month 30 Acoramidis resulted in a statistically significant reduction in ACM in the intention-to-treat (ITT) population at Month 30 Shared +2.50 cm/yr annualized height velocity (AHV) at Month 18 in Cohort 5 of PROPEL 2, the Phase 2 study of infigratinib in achondroplasia, as well as the first ever reported statistically significant improvement in body proportionality The first child consented in ACCEL, the observational run-in study for infigratinib in children living with hypochondroplasia, a skeletal dysplasia closely related to achondroplasia The Company surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) The Company ended the quarter with $587 million in cash, cash equivalents, marketable securities and short-term restricted cash PALO ALTO, Calif., Aug. 01, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases, today reported its financial results for the second quarter ended June 30, 2024, and provided an update on the Company's operations. "Our team has continued its preparation for the commercial launch of acoramidis while also executing against our goal of fully enrolling our three Phase 3 clinical programs by the end of 2024. We are well positioned to launch acoramidis and achieve three readouts in 2025. Our differentiated capability to develop multiple candidates for genetic-based diseases provides a unique opportunity to create significant value for patients and shareholders," said Dr. Neil Kumar, CEO and Founder of BridgeBio. Pipeline overview: Program Status Next expected milestone Acoramidis for ATTR-CM NDA filed with FDA November 29, 2024 PDUFA date Encaleret for ADH1 Enrolling CALIBRATE, Phase 3 study Enrollment completion in 2024 BBP-418 for LGMD2I/R9 Enrolling FORTIFY, Phase 3 study Enrollment completion in 2024 Low-dose infigratinib for achondroplasia Enrolling PROPEL 3, Phase 3 study Enrollment completion in 2024 Low-dose infigratinib for hypochondroplasia Enrolling observational run-in for ACCEL 2, Phase 2 study Enrollment completion date to be announced BBP-631 for congenital adrenal hyperplasia (CAH) Dose finding / analysis in Phase 2 study Program update in August 2024 BBP-812 for Canavan disease Enrolling at high dose in Phase 1/2 study Key regulatory interactions Program updates: Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): During the 2024 International Symposium of Amyloidosis (ISA), five new analyses were disclosed through oral presentations and posters, discussing the following: Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular hospitalization (CVH) in ATTR-CM participants through Month 30. Acoramidis treatment resulted in a significant improvement in the composite endpoint of ACM and CVH in ATTR-CM participants, with benefit evident as early as Month 3. In ATTRibute-CM, participants with at least one CVH had a significantly higher risk of mortality, highlighting the need for ATTR-CM treatments that reduce the risk of CVH. BridgeBio also shared the rationale and design of ACT-EARLY, the acoramidis ATTR amyloidosis prevention trial, which it expects to initiate later this year. At this year's European Society of Cardiology Heart Failure (ESC-HF) Congress 2024, BridgeBio shared four positive analyses, which included the following data: In a pre-specified Cochran-Mantel-Haenszel sensitivity analysis applied to the entire ITT population of the study (N=632), acoramidis significantly reduced ACM (p=0.04), with no safety signals of potential clinical concern. Among ATTRibute-CM participants enrolled with Stage 4 chronic kidney disease (CKD) (N=21), acoramidis treatment was associated with proportionally fewer deaths compared with placebo, with no safety signals of potential clinical concern. At Month 30 of the ATTRibute-CM study, acoramidis treatment resulted in a statistically significant and clinically important reduction in the progressive decline in health-related quality of life as assessed by the EuroQoL Health Outcomes Assessment tool, EQ-5D-5L. Acoramidis treatment also reduced the decline in health status and quality of life as shown by statistically significant and clinically meaningful benefits in the Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score and supported by numerical and consistent benefits in individual KCCQ domains. In ATTRibute-CM, acoramidis significantly improved NT-proBNP indices that can be a signal of ATTR-CM disease progression and be predictive of subsequent mortality risk. During the 2024 American College of Cardiology (ACC), BridgeBio presented cardiac magnetic resonance (CMR) imaging evidence consistent with clinical improvement observed in the ATTRibute-CM. The data demonstrate that targeting near-complete TTR stabilization with acoramidis may enable cardiac remodeling and functional recovery in patients with ATTR-CM. Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025. BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025. Recent Type C interactions with U.S. FDA focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio's belief that there is potential to pursue Accelerated Approval for BBP-418. Rare Pediatric Disease Designation for BBP-418 highlights that LGMD2I/R9 is a rare disease with serious manifestations, which primarily impacts children. If BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher, which can be applied to another therapy in the Company's pipeline for a shorter timeline during the review process of a NDA or can be sold to another company looking to receive priority review for one of its applications. Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia: BridgeBio shared data from Month 12 and 18 for Cohort 5 of PROPEL 2 (0.25 mg/kg/day), its Phase 2 trial in achondroplasia with the oral treatment, infigratinib, with results including: A statistically significant and sustained increase in AHV, with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015). At Month 18, there was a statistically significant improvement in body proportionality (p-value of 0.001). The mean upper to lower body segment ratio was 1.88 at Month 18, as compared to 2.02 at baseline. Infigratinib continues to be well-tolerated as a single daily oral therapy with no adverse events (AEs) assessed as treatment-related in any participant in Cohort 5. Infigratinib for achondroplasia was granted Fast Track Designation and Rare Pediatric Drug Designation by the U.S. FDA. If infigratinib is approved, BridgeBio may qualify for a Priority Review Voucher. In May 2024, the first participant consented to be part of ACCEL, the observational run-in study for infigratinib in children living with hypochondroplasia. Second Quarter 2024 Financial Results: Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash Cash, cash equivalents, marketable securities and short-term restricted cash, totaled $587.2 million as of June 30, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $194.6 million net increase in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net proceeds received from the term loan under the credit facility with Blue Owl of $434.0 million, net proceeds received from various equity financings of $314.8 million, proceeds from the sale of investments in equity securities of $63.2 million, and special cash dividends received from investments in equity securities of $25.7 million. These were primarily offset by refinancing of the Company's previous senior secured credit term loan, inclusive of prepayment fees and exit-related costs in aggregate of $473.4 million, net cash used in operating activities of $144.8 million, purchases of equity securities of $20.3 million, and repurchase of shares to satisfy tax withholdings of $4.7 million during the six months ended June 30, 2024. Revenue Revenue for the three and six months ended June 30, 2024 were $2.2 million and $213.3 million, respectively, as compared to $1.6 million and $3.5 million for the same periods in the prior year. The increase of $0.6 million in revenue for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the recognition of services revenue under the exclusive license and collaboration agreements with Bayer and Kyowa Kirin. Revenue for the three months ended June 30, 2023 primarily consists of the recognition of services revenue under the Navire-BMS License Agreement, which terminated effective June 2024. The increase of $209.8 million in revenue for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to $202.9 million from recognition of non-refundable upfront payments and service revenue under the Bayer and the Kyowa Kirin exclusive license and collaboration agreements. Operating Costs and Expenses Operating costs and expenses for the three and six months ended June 30, 2024 were $177.7 million and $388.5 million, respectively, compared to $147.7 million and $275.7 million for the same periods in the prior year. The overall increase of $30.0 million in operating costs and expenses for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $23.4 million in selling, general and administrative (SG&A) expenses mainly to support commercialization readiness efforts, an increase of $7.2 million in research and development and other expenses (R&D) to advance the Company's pipeline of research and development programs, offset by a decrease of $0.6 million in restructuring, impairment and related charges. The overall increase of $112.8 million in operating costs and expenses for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $58.1 million in SG&A expenses mainly to support commercialization readiness efforts, and an increase of $55.3 million in R&D expenses to advance the Company's pipeline of research and development programs, offset by a decrease of $0.6 million in restructuring, impairment and related charges. Operating costs and expenses for the six months ended June 30, 2024, include $22.5 million of nonrecurring deal-related costs for transactions that were closed during the three months ended March 31, 2024. Restructuring, impairment and related charges for the three and six months ended June 30, 2024 amounted to $2.9 million and $6.3 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. Restructuring, impairment and related charges for the same periods in the prior year was $3.5 million and $6.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Stock-based compensation expenses included in operating costs and expenses for the three months ended June 30, 2024 were $21.5 million, of which $4.9 million is included in R&D expenses, $16.5 million is included in SG&A expenses, and $0.1 million is included in Restructuring expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $27.2 million, of which $13.2 million is included in R&D expenses, and $14.0 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the six months ended June 30, 2024 were $50.3 million, of which $17.7 million is included in R&D expenses, $32.5 million is included in SG&A expenses, and $0.1 million is included in Restructuring expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $50.7 million, of which $25.0 million is included in R&D expenses, and $25.7 million is included in SG&A expenses. Total Other Income (Expense), netTotal other income (expense), net for the three and six months ended June 30, 2024 were $100.0 million and $63.5 million, respectively, compared to ($14.6) million and ($31.2) million for the same periods in the prior year. The increase in total other income (expense), net of $114.6 million for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the Company's gain on deconsolidation of a subsidiary of $126.3 million. This was partially offset by a net loss from an equity method investment of $7.9 million and an increase in interest expense of $2.3 million. The increase in total other income (expense), net of $94.7 million for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the Company's gain on deconsolidation of a subsidiary of $126.3 million and an increase in other income (expense), net of $8.1 million mainly from income or mark to market fair value adjustments from the Company's investments in equity securities. These were partially offset by a loss on extinguishment of debt of $26.6 million, a net loss from an equity method investment of $7.9 million and an increase in interest expense of $5.7 million. Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share For the three months and six months ended June 30, 2024, the Company recorded a net loss attributable to common stockholders of BridgeBio of $73.5 million and $108.7 million, respectively, compared to $157.9 million and $298.1 million, respectively for the three months and six months ended June 30, 2023. For the three months and six months ended June 30, 2024, the Company reported a net loss per share of $0.39 and $0.59, respectively compared to $0.98 and $1.90, respectively for the three months and six months ended June 30, 2023.   BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Operations(in thousands, except shares and per share amounts)       Three Months Ended June 30,     Six Months Ended June 30,       2024     2023     2024     2023       (Unaudited)     (Unaudited)   Revenue   $ 2,168     $ 1,641     $ 213,288     $ 3,467   Operating costs and expenses:                         Research, development and other expenses     115,293       108,087       256,863       201,599   Selling, general and administrative     59,523       36,122       125,330       67,230   Restructuring, impairment and related charges     2,891       3,531       6,291       6,900   Total operating costs and expenses     177,707       147,740       388,484       275,729   Loss from operations     (175,539 )     (146,099 )     (175,196 )     (272,262 ) Other income (expense), net:                         Interest income     5,195       4,514       9,270       8,667   Interest expense     (22,937 )     (20,594 )     (46,408 )     (40,715 ) Gain on deconsolidation of a subsidiary     126,294       —       126,294       —   Loss on extinguishment of debt     —       —       (26,590 )     —