On November 12, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a new type of biopharmaceutical company focused on genetic diseases, reported its financial results for the third quarter ended September 30, 2024, and provided an update on the Company’s operations (Press release, BridgeBio, NOV 12, 2024, View Source [SID1234648160]).
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"I’m grateful for the continued progress that we have seen across our late-stage pipeline, and I’m excited for the upcoming opportunity to serve patients with ATTR-CM in the commercial marketplace," said Dr. Neil Kumar, Ph.D., CEO and Founder of BridgeBio. "Underpinning this headway is a corporate experiment we have been conducting for over 9 years now that posits a new type of biotech business model, and so I’m also proud to have released our first case study in The Journal of Portfolio Management, highlighting salient elements of that model."
Pipeline overview:
Program Status Next expected milestone
Acoramidis for ATTR-CM NDA filed with U.S. FDA November 29, 2024 PDUFA date
Encaleret for ADH1 Enrolling CALIBRATE, Phase 3 study Enrollment completion in 2024
BBP-418 (ribitol) for LGMD2I/R9 FORTIFY, Phase 3 study enrollment completed Interim analysis in 2025
Infigratinib for achondroplasia Enrolling PROPEL 3, Phase 3 study Enrollment completion in 2024
Infigratinib for hypochondroplasia Enrolling observational run-in for ACCEL 2, Phase 2 study Enrollment completion date to be announced
BBP-812 for Canavan disease Enrolling at high dose in Phase 1/2 study Enrollment completion date to be announced
Late-stage investigational programs updates:
Acoramidis – Near-complete transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
Based on the positive results from ATTRibute-CM, BridgeBio filed a new drug application (NDA) to the FDA, which has been accepted with a PDUFA action date of November 29, 2024, and the late cycle meeting with the FDA has been completed.
Outcomes data through 42 months from the ongoing long-term open-label extension (OLE) of ATTRibute-CM, the Company’s Phase 3 study of acoramidis in ATTR-CM, will be shared at the American Heart Association (AHA) Scientific Sessions on November 18th.
During the European Society of Cardiology (ESC) 2024, a new analysis was shared in an oral presentation, showing:
Increased serum TTR at Day 28 of ATTRibute-CM was correlated with reduced risk of ACM, cardiovascular mortality (CVM) and CVH in ATTR-CM.
A mean of 3.0mg/dL increase in serum transthyretin (TTR) at Month 1 of the OLE (n=21) and mean of 3.4mg/dL increase in serum TTR at Month 6 of the OLE (n=18) in participants who switched from tafamidis and placebo to acoramidis in the ATTRibute-CM study.
A post-hoc analysis of ATTRibute-CM evaluating the effect of acoramidis on the composite endpoint of ACM and recurrent CVH events was shared at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, which included the following data:
A 42% reduction in composite ACM and recurrent CVH events at 30 months observed with acoramidis treatment compared to placebo by applying a negative binomial regression model (post-hoc) (p=0.0005).
A 42% reduction in the total number of ACM and recurrent CVH events per patient observed over 30 months with acoramidis treatment compared to placebo.
A 30.5% hazard reduction in ACM and recurrent CVH events at 30 months observed with acoramidis treatment compared to placebo by applying the Andersen-Gill model (post-hoc) (p=0.0008).
BridgeBio announced the initiation of a scientific collaboration with the CarDS Lab, led by cardiologist-data scientist, Rohan Khera, M.D., M.S. at the Yale School of Medicine, for the launch of the TRACE-AI Network, a novel paradigm of large-scale federated AI screening for ATTR-CM.
Upon FDA approval of acoramidis, it is our intent to honor the courage of our U.S. clinical trial patients by providing them acoramidis free for life.
Encaleret – Calcium-sensing receptor (CaSR) antagonist for autosomal dominant hypocalcemia type 1 (ADH1):
CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, completed screening; the Company anticipates completing enrollment of the CALIBRATE study in 2024.
Proof-of-principle data of encaleret, an oral option for post-surgical hypoparathyroidism, were presented at the American Society for Bone Mineral Research meeting demonstrating a concomitant normalization of blood and urine calcium in 86% of participants within 5 days.
BBP-418 (ribitol) – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
BridgeBio completed enrollment of FORTIFY, the Company’s Phase 3 registrational study of BBP-418 in individuals with LGMD2I/R9, with topline data readout from the interim analysis expected in 2025.
BridgeBio believes there is an opportunity to pursue Accelerated Approval in the U.S. for BBP-418 in LGMD2I/R9 based on a potential biomarker surrogate endpoint of glycosylated alpha-dystroglycan (αDG) at time of the interim analysis.
The FDA has granted Rare Pediatric Disease Designation for BBP-418 in the treatment of LGMD2I/R9. 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 New Drug Application or can be sold and transferred to another company looking to receive priority review for one of its applications.
Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
The FDA granted Breakthrough Therapy Designation to infigratinib for demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s).
The PROPEL 3 global Phase 3 registrational study of infigratinib in achondroplasia continues to enroll; study completion anticipated by the end of the year. PROPEL, BridgeBio’s observational lead-in study in achondroplasia for PROPEL 3, has completed enrollment.
The initial phase of MyAchonJourney, a new online resource to support individuals and families living with achondroplasia, was launched.
ACCEL 2/3 will be a global Phase 2/3 multicenter, single-dose study, to evaluate the efficacy and safety of 0.25mg/kg/day of infigratinib in children living with hypochondroplasia. ACCEL, BridgeBio’s observational lead-in study for hypochondroplasia, continues to enroll.
BBP-812 – Adeno-associated virus (AAV) 9 gene therapy for Canavan disease:
The Canavan disease program received RMAT Designation based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial.
BridgeBio will leverage the benefits of RMAT designation, including early and more frequent interactions with the FDA, to establish an Accelerated Approval pathway for BBP-812.
New positive data from the high-dose cohort includes:
Progressive and continued post-dose improvement in gross motor function (measured by Gross Motor Function Measure (GMFM)-88) and achievement of motor milestones (measured by Hammersmith Infant Neurological Examination (HINE)-2).
In the low-dose cohort, these strikingly divergent trajectories resulted in statistically significant improvements in achieved motor function and milestones at 12-months after treatment with BBP-812, compared to what is observed in and predicted by the natural history of the disease seen in BridgeBio’s study, CANinform; data from the high dose cohort are not yet available.
Third Quarter 2024 Financial Results:
"We are prepared to launch acoramidis in the U.S., upon approval by the FDA, at the end of 2024 as well as to read out our three ongoing Phase 3 studies in 2025," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "As we continue to move our late-stage pipeline forward, we are excited to also take an initial step in explaining the thesis and underlying logic of our decision making with the recent release of our case study in The Journal of Portfolio Management."
Cash, Cash Equivalents, and Short-term Restricted Cash
Cash, cash equivalents and short-term restricted cash, totaled $405.7 million as of September 30, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $13.1 million net increase in cash, cash equivalents 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 $314.7 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 increases in cash, cash equivalents and short-term restricted cash were primarily offset by the impacts of refinancing 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 $325.4 million, purchases of equity securities of $20.3 million, and repurchase of shares to satisfy tax withholdings of $6.1 million during the nine months ended September 30, 2024.
Revenue
Revenue for the three and nine months ended September 30, 2024 were $2.7 million and $216.0 million, respectively, as compared to $4.1 million and $7.6 million for the same periods in the prior year.
The decrease of $1.4 million in revenue for the three months ended September 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 September 30, 2023 primarily consists of the recognition of services revenue under the Navire-BMS License Agreement, which terminated effective June 2024.
The increase of $208.4 million in revenue for the nine months ended September 30, 2024, compared to the same period in the prior year, was primarily due to $205.3 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 nine months ended September 30, 2024 were $194.5 million and $583.0 million, respectively, compared to $161.8 million and $437.5 million for the same periods in the prior year.
The overall increase of $32.7 million in operating costs and expenses for the three months ended September 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $33.0 million in selling, general and administrative (SG&A) expenses mainly to support commercialization readiness efforts which included costs incurred for marketing, advertising and buildup of salesforce, an increase of $4.3 million in restructuring, impairment and related charges, offset by a decrease of $4.6 million in research and development and other expenses (R&D) mainly due to the deconsolidation of certain subsidiaries.
The overall increase of $145.5 million in operating costs and expenses for the nine months ended September 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $91.1 million in SG&A expenses mainly to support commercialization readiness efforts which included costs incurred for marketing, advertising and buildup of salesforce, an increase of $50.6 million in R&D expenses to advance the Company’s pipeline of research and development programs, and an increase of $3.8 million in restructuring, impairment and related charges. Operating costs and expenses for the nine months ended September 30, 2024, include $25.0 million of nonrecurring deal-related costs for transactions that were completed during the nine months ended September 30, 2024.
Restructuring, impairment and related charges for the three and nine months ended September 30, 2024 amounted to $4.6 million and $10.9 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 were $0.3 million and $7.2 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 September 30, 2024 were $27.1 million, of which $12.1 million is included in R&D expenses, $15.0 million is included in SG&A expenses, and less than $0.1 million is included in restructuring, impairment and related charges. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $27.2 million, of which $14.1 million is included in R&D expenses, and $13.1 million is included in SG&A expenses.
Stock-based compensation expenses included in operating costs and expenses for the nine months ended September 30, 2024 were $77.4 million, of which $29.8 million is included in R&D expenses, $47.5 million is included in SG&A expenses, and $0.1 million is included in restructuring, impairment and related charges. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $77.9 million, of which $39.2 million is included in R&D expenses, and $38.7 million is included in SG&A expenses.
Total Other Income (Expense), net
Total other income (expense), net for the three and nine months ended September 30, 2024 were $27.5 million and $91.0 million, respectively, compared to ($21.8) million and ($53.0) million for the same periods in the prior year.
The increase in total other income (expense), net of $49.3 million for the three months ended September 30, 2024, compared to the same period in the prior year, was primarily due to the Company’s gain on deconsolidation of subsidiaries of $52.0 million and an increase in other income (expense), net of $7.1 million mainly due to mark to market fair value adjustments from the Company’s investments in equity securities. This was partially offset by a net loss from an equity method investment of $6.6 million and an increase in interest expense of $2.8 million.
The increase in total other income (expense), net of $144.0 million for the nine months ended September 30, 2024, compared to the same period in the prior year, was primarily due to the Company’s gain on deconsolidation of subsidiaries of $178.3 million and an increase in other income (expense), net of $15.1 million mainly due to mark to market fair value adjustments from the Company’s investments in equity securities. These were partially offset by recognition of a loss on extinguishment of debt of $26.6 million, a net loss from equity method investments of $14.5 million and an increase in interest expense of $8.4 million.
Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share
For the three and nine months ended September 30, 2024, the Company recorded a net loss attributable to common stockholders of BridgeBio of $162.0 million and $270.7 million, respectively, compared to $177.0 million and $475.1 million, respectively for the three and nine months ended September 30, 2023.
For the three and nine months ended September 30, 2024, the Company reported a net loss per share of $0.86 and $1.46, respectively compared to $1.08 and $2.99, respectively for the three and nine months ended September 30, 2023.
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Operations
(in thousands, except shares and per share amounts)
Three Months Ended September 30, Nine Months Ended September 30,
2024 2023 2024 2023
(Unaudited) (Unaudited)
Revenue $ 2,732 $ 4,091 $ 216,020 $ 7,558
Operating costs and expenses:
Research, development and other expenses 121,042 125,734 377,905 327,333
Selling, general and administrative 68,819 35,777 194,149 103,007
Restructuring, impairment and related charges 4,621 272 10,912 7,172
Total operating costs and expenses 194,482 161,783 582,966 437,512
Loss from operations (191,750 ) (157,692 ) (366,946 ) (429,954 )
Other income (expense), net:
Interest income 3,296 3,793 12,566 12,460
Interest expense (23,061 ) (20,306 ) (69,469 ) (61,021 )
Gain on deconsolidation of subsidiaries 52,027 — 178,321 —
Loss on extinguishment of debt — — (26,590 ) —
Net loss from equity method investments (6,563 ) — (14,488 ) —
Other income (expense), net 1,797 (5,283 ) 10,648 (4,408 )
Total other income (expense), net 27,496 (21,796 ) 90,988 (52,969 )
Net loss (164,254 ) (179,488 ) (275,958 ) (482,923 )
Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,214 2,489 5,246 7,869
Net loss attributable to common stockholders of BridgeBio $ (162,040 ) $ (176,999 ) $ (270,712 ) $ (475,054 )
Net loss per share, basic and diluted $ (0.86 ) $ (1.08 ) $ (1.46 ) $ (2.99 )
Weighted-average shares used in computing net loss per share, basic and diluted 188,510,372 163,308,632 184,947,173 158,891,152
Three Months Ended September 30, Nine Months Ended September 30,
Stock-based Compensation 2024 2023 2024 2023
(Unaudited) (Unaudited)
Research, development and other expenses $ 12,124 $ 14,144 $ 29,840 $ 39,152
Selling, general and administrative 14,969 13,086 47,511 38,731
Restructuring, impairment and related charges 38 — 81 —
Total stock-based compensation $ 27,131 $ 27,230 $ 77,432 $ 77,883
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)
September 30, December 31,
2024 2023
Assets (Unaudited) (1)
Cash and cash equivalents $ 266,324 $ 375,935
Investments in equity securities — 58,949
Receivables from licensing and collaboration agreements 478 1,751
Short-term restricted cash 139,409 16,653
Prepaid expenses and other current assets 38,367 24,305
Investment in nonconsolidated entities 160,443 —
Property and equipment, net 8,701 11,816
Operating lease right-of-use assets 6,439 8,027
Intangible assets, net 24,525 26,319
Other assets 20,291 22,625
Total assets $ 664,977 $ 546,380
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable $ 13,363 $ 10,655
Accrued and other liabilities 109,482 122,965
Operating lease liabilities 10,433 13,109
Deferred revenue 30,398 9,823
2029 Notes, net 738,376 736,905
2027 Notes, net 544,719 543,379
Term loan, net 436,221 446,445
Other long-term liabilities 377 5,634
Redeemable convertible noncontrolling interests 645 478
Total BridgeBio stockholders’ deficit (1,229,922 ) (1,354,257 )
Noncontrolling interests 10,885 11,244
Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $ 664,977 $ 546,380
(1) The condensed consolidated financial statements as of and for the year ended December 31, 2023 are derived from the audited consolidated financial statements as of that date.
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
(In thousands)
Nine Months Ended September 30,
2024 2023
Operating activities:
Net loss $ (275,958 ) $ (482,923 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation 65,673 71,685
Loss on extinguishment of debt 26,590 —
Accretion of debt 5,399 6,724
Depreciation and amortization 4,708 4,909
Noncash lease expense 3,119 3,024
Accrual of payment-in-kind interest on term loan — 6,742
Net loss from equity method investments 14,488 —
Loss (gain) on deconsolidation of subsidiaries (178,321 ) 1,241
Loss (gain) from investment in equity securities, net (8,136 ) 2,951
Other noncash adjustments, net (2,059 ) (332 )
Changes in operating assets and liabilities:
Receivables from licensing and collaboration agreements 1,273 11,909
Prepaid expenses and other current assets (17,543 ) (980 )
Other assets (428 ) 1,443
Accounts payable 5,257 (3,404 )
Accrued compensation and benefits 5,580 (4,156 )
Accrued research and development liabilities 15,454 (10,544 )
Operating lease liabilities (4,459 ) (3,671 )
Deferred revenue 20,575 (4,464 )
Accrued professional and other liabilities (6,612 ) (3,055 )
Net cash used in operating activities (325,400 ) (402,901 )
Investing activities:
Purchases of marketable securities (93,811 ) (29,726 )
Maturities of marketable securities 95,000 82,550
Purchases of investments in equity securities (20,271 ) (78,314 )
Proceeds from sales of investments in equity securities 63,229 80,963
Proceeds from special cash dividends received from investments in equity securities 25,682 —
Payment for an intangible asset (4,785 ) —
Purchases of property and equipment (886 ) (871 )
Decrease in cash and cash equivalents resulting from deconsolidation of subsidiaries (140 ) (503 )
Net cash provided by investing activities 64,018 54,099
Financing activities:
Proceeds from term loan under Financing Agreement 450,000 —
Issuance costs and discounts associated with term loan under Financing Agreement (15,986 ) —
Repayment of term loan under Loan and Security Agreement (473,417 ) —
Proceeds from issuance of common stock through public offerings, net 314,741 450,264
Proceeds from BridgeBio common stock issuances under ESPP 4,502 3,397
Proceeds from stock option exercises, net of repurchases 808 5,222
Transactions with noncontrolling interests — 1,500
Repurchase of RSU shares to satisfy tax withholding (6,122 ) (4,325 )
Net cash provided by financing activities 274,526 456,058
Net increase in cash, cash equivalents and restricted cash 13,144 107,256
Cash, cash equivalents and restricted cash at beginning of period 394,732 416,884
Cash, cash equivalents and restricted cash at end of period $ 407,876 $ 524,140
Nine Months Ended September 30,
2024 2023
Supplemental Disclosure of Cash Flow Information:
Cash paid for interest $ 78,236 $ 50,826
Supplemental Disclosures of Noncash Investing and Financing Information:
Unpaid public offering issuance costs $ — $ 455
Unpaid property and equipment $ 274 $ 192
Transfers to noncontrolling interests $ (4,719 ) $ (8,313 )
Reconciliation of Cash, Cash Equivalents and Restricted Cash:
Cash and cash equivalents $ 266,324 $ 505,213
Restricted cash 139,409 16,652
Restricted cash — Included in "Other assets" 2,143 2,275
Total cash, cash equivalents and restricted cash at end of period shown in the
condensed consolidated statements of cash flows $ 407,876 $ 524,140