On February 20, 2025 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 fourth quarter and full year ended December 31, 2024, and provided an update on Attruby’s commercial progress (Press release, BridgeBio, FEB 20, 2025, View Source [SID1234650410]).
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Commercial Progress:
As of February 17, 2025, 1,028 unique patient prescriptions for Attruby have been written by 516 unique healthcare providers since FDA approval.
"I am very encouraged by the strength of the Attruby launch, with prescriptions being successfully filled across all patient types," said Matt Outten, Chief Commercial Officer of BridgeBio. "In conversations with healthcare providers and patients, we have repeatedly heard that Attruby’s category-leading results – time to separation of just three months, along with a 42% reduction in all-cause mortality and recurrent hospitalizations and a 50% reduction in cardiovascular hospitalizations at 30 months – set it apart as a clinically meaningful advancement for ATTR-CM. Combined with our industry-leading patient support programs, we believe Attruby is delivering a much-needed change in the treatment landscape."
Pipeline Overview:
Program Status Next expected milestone
Acoramidis for ATTR-CM Approved in U.S. and EU Japan approval in 1H 2025
BBP-418 for LGMD2I/R9 FORTIFY, Phase 3 study enrollment completed Last Participant – Last Visit and Topline results in 2H 2025
Encaleret for ADH1 CALIBRATE, Phase 3 study enrollment completed Last Participant – Last Visit and Topline results in 2H 2025
Infigratinib for achondroplasia PROPEL 3, Phase 3 study enrollment completed Last Participant – Last Visit in 2H 2025
Infigratinib for hypochondroplasia ACCEL, run-in for Phase 2 study ongoing Enrollment completion date to be announced
BBP-812 for Canavan disease CANaspire Phase 1/2 study ongoing Enrollment completion date to be announced
Key Program Updates:
"It is exciting to see patients, physicians, and payers resonate with our message that the greater levels of TTR stabilization that Attruby delivers can be of benefit to the patients we serve and that the TTR protein is clinically important, not toxic." said Neil Kumar, Ph.D., Founder and CEO of BridgeBio. "We look forward to continuing to partner with the community to ensure that we find all patients that can be helped and ease their path to getting on therapy, when appropriate, as much as possible."
Attruby (acoramidis) – the first approved, near-complete (≥90%) TTR stabilizer for treatment of transthyretin amyloid cardiomyopathy (ATTR-CM):
On November 22, 2024, the U.S. Food and Drug Administration (FDA) approved Attruby (acoramidis), a near-complete TTR stabilizer (≥90%), to reduce cardiovascular death and cardiovascular-related hospitalization (CVH) in adult patients with ATTR-CM.
On February 10, 2025, the European Commission approved BEYONTTRA (acoramidis) for use in adult patients with ATTR-CM in the EU.
Preliminary results from the ongoing ATTRibute-CM open-label extension (OLE) study of Attruby in ATTR-CM were simultaneously published in Circulation and presented at the American Heart Association Scientific Sessions, showing that Attruby demonstrated statistically significant risk reduction of 36% on All-Cause Mortality (ACM) alone at month 36 within the OLE, and 46% (p<0.0001) and 48% (p<0.0001) reductions in the composite endpoint of ACM and recurrent CVH at months 36 and 42, respectively.
Attruby is supported by industry-leading access programs designed to ensure seamless treatment initiation and continuity for all patients with ATTR-CM.
BBP-418 – Glycosylation substrate in development for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
FORTIFY, the Phase 3 clinical trial of BBP-418 in LGMD2I/R9, a rare genetic disorder caused by variants in the fukutin‑related protein (FKRP) gene, is fully enrolled with 112 participants. The trial is the largest prospective interventional study to ever be conducted in LGMD2I.
The Company expects to achieve last participant – last visit and report topline results of the interim analysis cohort in the second half of 2025.
If successful, we expect BBP-418 would be the first approved therapy for individuals living with LGMD2I/R9.
Encaleret – Calcium-sensing receptor (CaSR) antagonist in development for autosomal dominant hypocalcemia type 1 (ADH1) and postsurgical hypoparathyroidism (PSH):
CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, a genetic form of hypoparathyroidism, is fully enrolled with 71 participants. The trial is the largest prospective interventional study to ever be conducted in ADH1.
The Company expects to achieve last participant – last visit and report topline results in the second half of 2025.
If successful, we expect encaleret would be the first approved therapy indicated for individuals living with ADH1.
A Phase 2 study of encaleret in PSH is ongoing, with preliminary evidence suggestive of a differentiated profile for encaleret in PSH.
Infigratinib – FGFR1-3 inhibitor in development for achondroplasia and hypochondroplasia:
PROPEL 3, the Phase 3 clinical trial of infigratinib in achondroplasia, the most common form of disproportionate short stature, is fully enrolled with 114 participants randomized.
The Company expects to achieve last participant – last visit in the second half of 2025.
In November 2024, the Phase 2 PROPEL 2 study of infigratinib in children with achondroplasia was published in the New England Journal of Medicine.
If successful, we expect infigratinib would be the first approved oral therapy option for children living with achondroplasia.
The Company is currently enrolling the ACCEL run-in for a Phase 2 study of infigratinib in hypochondroplasia.
Financial Updates:
Cash, Cash Equivalents, and Short-term Restricted Cash
Cash, cash equivalents and short-term restricted cash, totaled $681.2 million as of December 31, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $288.6 million net increase in cash, cash equivalents and short-term restricted cash was primarily attributable to net proceeds received from the Funding Agreement of $488.8 million, net proceeds received from the term loan under the credit facility 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 net cash used in operating activities of $520.7 million, refinancing the Company’s previous senior secured credit term loan, inclusive of prepayment fees and exit-related costs in aggregate of $473.4 million, purchases of equity securities of $20.3 million, Funding Agreement transaction related costs of $16.3 million, and the repurchase of shares to satisfy tax withholdings of $7.5 million during the year ended December 31, 2024.
Revenue
Revenue for the three months and year ended December 31, 2024, was $5.9 million and $221.9 million, respectively, as compared to $1.7 million and $9.3 million for the same periods in the prior year.
The increase of $4.2 million in revenue for the three months ended December 31, 2024, compared to the same period in the prior year, was primarily due to the recognition of $2.9 million in net product revenue from the first commercial sales of Attruby in the U.S. following the FDA approval on November 22, 2024, and services revenue received under the exclusive license and collaboration agreements with Bayer and Kyowa Kirin. Revenue for the three months ended December 31, 2023, primarily consisted of the recognition of services revenue under the Navire-BMS License Agreement, which terminated in June 2024.
The increase of $212.6 million in revenue for the year ended December 31, 2024, compared to the same period in the prior year, was primarily due to $207.7 million from recognition of the upfront payments and service revenue under the Bayer and the Kyowa Kirin exclusive license and collaboration agreements, and $2.9 million in net product revenue from the first commercial sales of Attruby following the FDA approval on November 22, 2024.
Operating Costs and Expenses
Operating costs and expenses for the three months and year ended December 31, 2024, were $231.9 million and $814.9 million, respectively, compared to $179.2 million and $616.7 million for the same periods in the prior year.
The overall increase of $52.7 million, in operating costs and expenses for the three months ended December 31, 2024, compared to the same period in the prior year, was primarily due to an increase of $47.2 million in selling, general and administrative (SG&A) expenses mainly to support commercialization of Attruby, which included costs incurred for marketing, advertising and hiring of a sales force in the U.S., an increase of $3.9 million in restructuring, impairment and related charges, and an increase of $1.6 million in research and development (R&D) expenses to advance the Company’s pipeline of R&D programs.
The overall increase of $198.2 million, in operating costs and expenses for the year ended December 31, 2024, compared to the same period in the prior year, was primarily due to an increase of $138.3 million in SG&A expenses related to costs primarily to support the commercial launch of Attruby which included costs incurred for marketing, advertising and hiring of a sales force in the U.S., an increase of $52.2 million in R&D expenses to advance the Company’s pipeline of R&D programs, and an increase of $7.7 million in restructuring, impairment and related charges. Operating costs and expenses for the year ended December 31, 2024, include $25.0 million of nonrecurring deal-related costs for transactions that were completed during the year ended December 31, 2024.
Restructuring, impairment and related charges for the three months and year ended December 31, 2024, amounted to $4.7 million and $15.6 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 $0.8 million and $7.9 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 December 31, 2024, were $36.4 million, of which $20.0 million is included in R&D expenses, $16.3 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 $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 million is included in SG&A expenses.
Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2024, were $113.9 million, of which $63.9 million is included in SG&A expenses, $49.8 million is included in R&D expenses, and $0.2 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 $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 million is included in SG&A expenses.
Total Other Income (Expense), net
Total other income (expense), net for the three months and year ended December 31, 2024, were ($40.2) million and $50.8 million, respectively, compared to $7.1 million and ($45.9) million for the same periods in the prior year.
The increase in total other expense, net of $47.3 million for the three months ended December 31, 2024, compared to the same period in the prior year, was primarily due to a decrease in other income, net of $20.1 million mainly due to market fair value adjustments from the Company’s investments in equity securities, a net loss from equity method investments of $16.7 million, an increase in interest expense, net of $9.6 million, and a decrease in interest income of $0.9 million.
The increase in total other income, net of $96.7 million for the year ended December 31, 2024 , compared to the same period in the prior year, was primarily due to gains the Company recognized on the deconsolidation of subsidiaries of $178.3 million. These gains were partially offset by recognition a net loss from equity method investments of $31.2 million, a loss on extinguishment of debt of $26.6 million, an increase in interest expense, net of $18.0 million, a decrease in other income, net of $5.0 million mainly due to market fair value adjustments from the Company’s investments in equity securities, and a decrease in interest income of $0.8 million.
Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share
For the three months and year ended December 31, 2024, the Company recorded a net loss attributable to common stockholders of BridgeBio of $265.1 million and $535.8 million, respectively, compared to $168.1 million and $643.2 million, respectively, for the three months and year ended December 31, 2023.
For the three months and year ended December 31, 2024, the Company reported a net loss per share of $1.40 and $2.88, respectively, compared to $0.96 and $3.95, respectively, for the three months and year ended December 31, 2023.
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,
2024 2023 2024 2023
(Unaudited) (1) (Unaudited) (1)
Revenue, net $ 5,882 $ 1,745 $ 221,902 $ 9,303
Operating costs and expenses:
Research, development and other expenses 132,434 130,824 510,339 458,157
Selling, general and administrative 94,782 47,583 288,931 150,590
Restructuring, impairment and related charges 4,693 754 15,605 7,926
Total operating costs and expenses 231,909 179,161 814,875 616,673
Loss from operations (226,027 ) (177,416 ) (592,973 ) (607,370 )
Other income (expense), net:
Interest income 4,683 5,578 17,249 18,038
Interest expense, net (29,821 ) (20,268 ) (99,290 ) (81,289 )
Gain on deconsolidation of subsidiaries — — 178,321 —
Loss on extinguishment of debt — — (26,590 ) —
Net loss from equity method investments (16,695 ) — (31,183 ) —
Other income (expense), net 1,624 21,778 12,272 17,370
Total other income (expense), net (40,209 ) 7,088 50,779 (45,881 )
Loss before income taxes (266,236 ) (170,328 ) (542,194 ) (653,251 )
Income tax expense 1,153 — 1,153 —
Net loss (267,389 ) (170,328 ) (543,347 ) (653,251 )
Net loss attributable to redeemable convertible
noncontrolling interests and noncontrolling interests 2,339 2,180 7,585 10,049
Net loss attributable to common stockholders
of BridgeBio $ (265,050 ) $ (168,148 ) $ (535,762 ) $ (643,202 )
Net loss per share, basic and diluted $ (1.40 ) $ (0.96 ) $ (2.88 ) $ (3.95 )
Weighted-average shares used in computing net
loss per share, basic and diluted 189,437,438 174,462,332 186,075,873 162,791,511
Three Months Ended December 31, Year Ended December 31,
Stock-based Compensation 2024 2023 2024 2023
(Unaudited) (1) (Unaudited) (1)
Research, development and other expenses $ 20,004 $ 22,495 $ 49,844 $ 61,647
Selling, general and administrative 16,351 14,638 63,862 53,369
Restructuring, impairment and related charges 79 — 160 —
Total stock-based compensation $ 36,434 $ 37,133 $ 113,866 $ 115,016
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)
December 31, December 31,
2024 2023
(Unaudited) (1)
Assets
Cash and cash equivalents $ 681,101 $ 375,935
Investments in equity securities — 58,949
Accounts receivable 4,722 1,751
Short-term restricted cash 126 16,653
Prepaid expenses and other current assets 34,743 24,305
Investment in nonconsolidated entities 143,747 —
Property and equipment, net 7,011 11,816
Operating lease right-of-use assets 5,767 8,027
Intangible assets, net 23,926 26,319
Other assets 18,195 22,625
Total assets $ 919,338 $ 546,380
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable $ 9,618 $ 10,655
Accrued and other liabilities 125,672 122,965
Operating lease liabilities 9,202 13,109
Deferred revenue 31,699 9,823
2029 Notes, net 738,872 736,905
2027 Notes, net 545,173 543,379
Term loan, net 437,337 446,445
Deferred royalty obligation, net 479,091 —
Other long-term liabilities 286 5,634
Redeemable convertible noncontrolling interests 142 478
Total BridgeBio stockholders’ deficit (1,467,904 ) (1,354,257 )
Noncontrolling interests 10,150 11,244
Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $ 919,338 $ 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
(In thousands)
Year Ended December 31,
2024 2023
(Unaudited) (1)
Operating activities:
Net loss $ (543,347 ) $ (653,251 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation 95,800 108,710
Loss on extinguishment of debt 26,590 —
Accretion of debt 15,763 8,907
Depreciation and amortization 6,075 6,494
Noncash lease expense 4,110 4,032
Accrual of payment-in-kind interest on term loan — 10,207
Net loss from equity method investments 31,183 —
Loss (gain) on deconsolidation of subsidiaries (178,321 ) 1,241
Loss (gain) from investment in equity securities, net (8,136 ) (18,314 )
Impairment of long-lived assets 271 —
Other noncash adjustments, net (2,756 ) (803 )
Changes in operating assets and liabilities:
Accounts receivable (2,971 ) 15,328
Prepaid expenses and other current assets (13,918 ) (2,702 )
Other assets 1,542 (1,546 )
Accounts payable 1,512 2,780
Accrued compensation and benefits 16,986 7,802
Accrued research and development liabilities 8,729 (9,855 )
Operating lease liabilities (5,902 ) (4,829 )
Deferred revenue 21,875 (5,438 )
Accrued professional and other liabilities 4,189 3,517
Net cash used in operating activities (520,726 ) (527,720 )
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 ) (107,538 )
Proceeds from sales of investments in equity securities 63,229 110,556
Proceeds from special cash dividends received from investments in equity securities 25,682 —
Payment for an intangible asset (7,975 ) —
Purchases of property and equipment (933 ) (1,306 )
Decrease in cash and cash equivalents resulting from deconsolidation of subsidiaries (140 ) (503 )
Net cash provided by investing activities 60,781 54,033
Financing activities:
Proceeds from royalty obligation under Funding Agreement 500,000 —
Issuance costs and discounts associated with royalty obligation
under Funding Agreement (27,513 ) —
Proceeds from term loan under Amended Financing Agreement 450,000 —
Issuance costs and discounts associated with term loan
under Amended Financing Agreement (15,986 ) —
Repayment of term loans (473,417 ) —
Proceeds from issuance of common stock through public offerings, net 314,741 449,810
Proceeds from BridgeBio common stock issuances under ESPP 4,502 3,398
Proceeds from stock option exercises, net of repurchases 3,656 6,008
Transactions with noncontrolling interests — (801 )
Repurchase of RSU shares to satisfy tax withholding (7,526 ) (6,880 )
Net cash provided by financing activities 748,457 451,535
Net increase (decrease) in cash, cash equivalents and restricted cash 288,512 (22,152 )
Cash, cash equivalents and restricted cash at beginning of year 394,732 416,884
Cash, cash equivalents and restricted cash at end of year $ 683,244 $ 394,732
Year Ended December 31,
2024 2023
(Unaudited) (1)
Supplemental Disclosure of Cash Flow Information:
Cash paid for interest $ 91,342 $ 61,108
Supplemental Disclosures of Noncash Investing and Financing Information:
Unpaid property and equipment $ 279 $ 100
Transfers to noncontrolling interests $ (5,819 ) $ (10,534 )
Reconciliation of Cash, Cash Equivalents and Restricted Cash:
Cash and cash equivalents $ 681,101 $ 375,935
Restricted cash 126 16,653
Restricted cash — Included in "Other assets" 2,017 2,144
Total cash, cash equivalents and restricted cash at end of period shown in the
consolidated statements of cash flows $ 683,244 $ 394,732
About Attruby (acoramidis)
INDICATION
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively).
About BEYONTTRA (acoramidis)
On 10 February 2025, the European Commission granted Marketing Authorization for BEYONTTRA (acoramidis) for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). For full prescribing information, please refer to the Summary of Product Characteristics (SmPC).