On February 22, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations (Press release, BridgeBio, FEB 24, 2024, View Source [SID1234640418]).
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"Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community."
BridgeBio’s key programs:
•
Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
o
The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.
o
The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include:
▪
An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);
▪
Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;
▪
Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; and
▪
Acoramidis was well-tolerated with no safety signals of potential clinical concern identified.
o
The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.
o
Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings.
•
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
o
In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.
o
In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.
2
o
The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024.
•
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
o
FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 2024. The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint.
•
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
o
CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025.
Recent Corporate Updates:
•
Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions.
Fourth Quarter and Full Year 2023 Financial Results:
Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash
Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023.
Revenue
Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022.
3
Operating Costs and Expenses
Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively.
The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.
The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company’s pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year.
Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 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.
Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 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 same period in the prior year were $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses.
Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 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. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges.
"Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term."
4
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,
2023
2022
2023
2022
(Unaudited)
(Unaudited)
(1)
Revenue
$
1,745
$
1,870
$
9,303
$
77,648
Operating costs and expenses:
Research, development and other expenses
130,824
91,549
458,157
402,896
Selling, general and administrative
47,583
31,862
150,590
143,189
Restructuring, impairment and related charges
754
7,691
7,926
43,765
Total operating costs and expenses
179,161
131,102
616,673
589,850
Loss from operations
(177,416
)
(129,232
)
(607,370
)
(512,202
)
Other income (expense), net:
Interest income
5,578
4,092
18,038
7,542
Interest expense
(20,268
)
(19,990
)
(81,289
)
(80,438
)
Gain from sale of priority review voucher, net
—
—
—
107,946
Other income (expense), net
21,778
4,560
17,370
(7,500
)
Total other income (expense), net
7,088
(11,338
)
(45,881
)
27,550
Net loss
(170,328
)
(140,570
)
(653,251
)
(484,652
)
Net loss attributable to redeemable convertible
noncontrolling interests and noncontrolling interests
2,180
2,979
10,049
3,469
Net loss attributable to common stockholders
of BridgeBio
$
(168,148
)
$
(137,591
)
$
(643,202
)
$
(481,183
)
Net loss per share, basic and diluted
$
(0.96
)
$
(0.92
)
$
(3.95
)
$
(3.26
)
Weighted-average shares used in computing net
loss per share, basic and diluted
174,462,332
149,344,380
162,791,511
147,473,076
Three Months Ended December 31,
Year Ended December 31,
Stock-based Compensation
2023
2022
2023
2022
(Unaudited)
(Unaudited)
(1)
Research, development and others
$
22,495
$
8,941
$
61,647
$
37,987
Selling, general and administrative
14,638
13,643
53,369
54,669
Restructuring, impairment and related charges
—
—
—
1,172
Total stock-based compensation
$
37,133
$
22,584
$
115,016
$
93,828
(1)
The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.
5
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)
December 31,
December 31,
2023
2022
(Unaudited)
(1)
Assets
Cash, cash equivalents and marketable securities
$
375,935
$
428,269
Investment in equity securities
58,949
43,653
Receivable from licensing and collaboration agreements
1,751
17,079
Short-term restricted cash
16,653
37,930
Prepaid expenses and other current assets
24,305
21,922
Property and equipment, net
11,816
14,569
Operating lease right-of-use assets
8,027
10,678
Intangible assets, net
26,319
28,712
Other assets
22,625
20,224
Total assets
$
546,380
$
623,036
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable
$
10,655
$
11,558
Accrued and other liabilities
129,061
106,195
Operating lease liabilities
13,109
15,949
2029 Notes, net
736,905
734,988
2027 Notes, net
543,379
541,634
Term loan, net
446,445
430,993
Other long-term liabilities
9,361
26,643
Redeemable convertible noncontrolling interests
478
(1,589
)
Total BridgeBio stockholders’ deficit
(1,354,257
)
(1,254,617
)
Noncontrolling interests
11,244
11,282
Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit
$
546,380
$
623,036
(1)
The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.
6
BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Cash Flows
(In thousands)
Year Ended December 31,
2023
2022
(Unaudited)
(1)
Operating activities:
Net loss
$
(653,251
)
$
(484,652
)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation
108,710
91,559
Depreciation and amortization
6,494
6,771
Noncash lease expense
4,032
5,172
Accrual of payment-in-kind interest on term loan
10,207
13,562
Loss on deconsolidation of PellePharm
1,241
—
(Gain) loss from investment in equity securities, net
(18,314
)
8,222
Fair value of shares issued under a license agreement
—
4,567
Accretion of debt
8,907
8,570
Fair value adjustment of warrants
(984
)
1,571
Loss on sale of certain assets
—
6,261
Impairment of long-lived assets
—
12,720
Gain from sale of priority review voucher, excluding transaction costs
—
(110,000
)
Gain from recognition of receivable from licensing and collaboration agreement
—
(12,500
)
Other noncash adjustments
181
604
Changes in operating assets and liabilities:
Receivable from licensing and collaboration agreements
15,328
15,169
Prepaid expenses and other current assets
(2,702
)
7,671
Other assets
(1,546
)
10,971
Accounts payable
2,780
(349
)
Accrued compensation and benefits
7,802
(2,362
)
Accrued research and development liabilities
(9,855
)
(4,309
)
Operating lease liabilities
(4,829
)
(6,245
)
Deferred revenue
(5,438
)
15,262
Accrued professional and other liabilities
3,517
(7,729
)
Net cash used in operating activities
(527,720
)
(419,494
)
Investing activities:
Purchases of marketable securities
(29,726
)
(137,493
)
Maturities of marketable securities
82,550
479,688
Purchases of investment in equity securities
(107,538
)
(55,562
)
Sales of investment in equity securities
110,556
52,835
Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm
(503
)
—
Payment for intangible asset
—
(1,500
)
Proceeds from sale of priority review voucher
—
110,000
Proceeds from sale of certain assets
—
10,000
Purchases of property and equipment
(1,306
)
(4,821
)
Net cash provided by investing activities
54,033
453,147
Financing activities:
Proceeds from issuance of common stock through Private Placement offering, net
240,796
—
Proceeds from issuance of common stock through Follow-on offering, net
144,049
—
Proceeds from issuance of common stock through ATM offering, net
64,965
4,852
Transactions with noncontrolling interests
(801
)
—
Repayment of term loan
—
(20,486
)
Proceeds from BridgeBio common stock issuances under ESPP
3,398
2,558
Repurchase of RSU shares to satisfy tax withholding
(6,880
)
(1,561
)
Proceeds from stock option exercises, net of repurchases
6,008
666
Other financing activities
—
837
Net cash provided by (used in) financing activities
451,535
(13,134
)
Net increase (decrease) in cash, cash equivalents and restricted cash
(22,152
)
20,519
Cash, cash equivalents and restricted cash at beginning of period
416,884
396,365
Cash, cash equivalents and restricted cash at end of period
$
394,732
$
416,884
7
Year Ended December 31,
2023
2022
(Unaudited)
(1)
Supplemental Disclosure of Cash Flow Information:
Cash paid for interest
$
61,108
$
54,443
Supplemental Disclosures of Noncash Investing and Financing Information:
Unpaid property and equipment
$
100
$
47
Recognized intangible asset recorded in "Other accrued and other long-term liabilities"
$
—
$
11,000
Transfers (to) from noncontrolling interests
$
(10,534
)
$
(3,512
)
Payment-in-kind interest added to principal of term loan
$
—
$
1,763
Reconciliation of Cash, Cash Equivalents and Restricted Cash:
Cash and cash equivalents
$
375,935
$
376,689
Short-term restricted cash
16,653
37,930
Restricted cash — Included in "Other assets"
2,144
2,265
Total cash, cash equivalents and restricted cash at end of periods
$
394,732
$
416,884
(1)
The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date.