On February 23, 2023 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, 2022 and provided an update on the Company’s operations (Press release, BridgeBio, FEB 23, 2023, View Source [SID1234627599]).

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"We are excited to enter 2023 back on the doorstep of potentially meaningful advances for the patients we serve," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "Amidst our six ongoing Phase 2 or 3 clinical trials, we anticipate important upcoming readouts from our achondroplasia Phase 2 trial in March, and our ATTR-CM Phase 3 trial mid-year."

BridgeBio’s key programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
The Phase 3 ATTRibute-CM study continues to have high operating fidelity.
The Company expects to announce topline registrational data for the month 30 primary endpoint, a hierarchical composite including all-cause mortality and cardiovascular-related hospitalizations, in mid-2023.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
In July 2022, we reported initial data from the fourth dosing cohort of the Phase 2 dose-escalation trial PROPEL 2, demonstrating a mean change from baseline in annualized height velocity (AHV) of +1.52 cm/year and a responder rate of 64% in children five years of age and older.
Through the fifth dosing cohort to date, infigratinib has been well-tolerated with no serious adverse events reported, no adverse events that required discontinuation reported, and with no dose-dependent phosphate elevation reported.
The Company expects to share preliminary data from the fifth dosing cohort in March 2023, and to initiate a registrational Phase 3 trial in 2023.
In Cohort 5, the Company hopes to observe a tolerability profile and efficacy at least in-line with Cohort 4. If successful, the Company believes infigratinib, if approved, has the potential to capture a significant share of the market based on blinded market research.

Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
In December 2022, the Company initiated the CALIBRATE Phase 3 trial, a pivotal trial comparing the effects of encaleret to standard of care on blood calcium concentration and 24-hour urine calcium excretion over a 24-week treatment period in patients with ADH1.
In a Phase 2b safety and efficacy trial of encaleret for ADH1, 69% of the participants achieved concurrent values of both blood calcium concentration and 24-hour urine calcium excretion within the reference range after 24 weeks of outpatient encaleret treatment; none of these individuals attained this dual therapeutic goal while on standard of care.
Population genetics analyses estimate approximately 25,000 carriers of gain-of-function variants of the CaSR, the underlying cause of ADH1, in the US and EU.
The Company anticipates sharing topline data from CALIBRATE in late 2023 or the first half of 2024.
If approved, encaleret could be the first therapy specifically indicated for the treatment of ADH1.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2I):
The Company reported positive top line data from the ongoing Phase 2 clinical trial in October 2022 and anticipates initiating a global Phase 3 registrational trial of BBP-418 for LGMD2I in 2023.
To that end, the Company has engaged with regulatory authorities to align on a Phase 3 trial design.
BBP-418 has a potentially-addressable population of 7,000 LGMD2I patients in the US and EU.
There are currently no disease-modifying treatments available for LGMD2I.

BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH):
The Phase 1/2 gene therapy trial of BBP-631 for CAH continued to progress; as of February 1, 2023, BBP-631 has been generally well-tolerated in four patients treated at the first two dose levels.
The Company plans to provide an update from patients treated at the third dose level by the end of 2023.
CAH is one of the most prevalent genetic diseases potentially addressable with adeno-associated virus (AAV) gene therapy, with more than 75,000 cases estimated in the United States and European Union.

RAS cancer portfolio:
BridgeBio is continuing to progress the three main programs of its RAS franchise:
BBO-8520, an investigational, next-generation small molecule KRAS G12C dual inhibitor candidate that is designed to directly bind and inhibit KRAS G12C in both its active (GTP bound) and inactive (GDP bound) conformations, which remains on track to file an IND and enter the clinic in the second half of 2023.
A PI3Kα:RAS breaker program, investigational small molecules that are designed to block Ras-driven PI3Kα activation with a novel and potentially broad mechanism of action to target not only PI3Kα mutant tumors and RAS mutant tumors, but potentially other tumors driven by RTK activation of RAS signaling. The Company remains on track to select a development candidate in 2023, with IND filing to follow in 2024.
The Company’s pan-KRAS program, which targets multiple KRAS mutants including KRASG12D and KRASG12V, which are present in a large percentage of colorectal, pancreatic, and non-small cell lung cancer tumors. Development candidate selection for this program is planned for late 2023 or early 2024.
Corporate Updates:

Board of Directors: Appointed Frank McCormick, Ph.D., FS, DSc (Hon) to the BridgeBio Board of Directors. Dr. McCormick is a co-founder of BridgeBio and serves as the Chairman of Oncology. He is also a professor at the UCSF Helen Diller Family Comprehensive Cancer Center, where he holds the David A. Wood Chair of Tumor Biology and Cancer Research, and he has led the National Cancer Institute’s Ras Initiative since its inception in 2013. Dr. McCormick rotates in as Dr. Richard Scheller leaves the Board of Directors; Dr. Scheller will continue to serve as BridgeBio’s Chairman of Research and Development. In addition, Brent Saunders will resign from the board of directors in early March in order to focus on his recent appointment as Chief Executive Officer and Chairman of the Board of Bausch + Lomb.
Fourth Quarter and Full Year 2022 Financial Results:
Cash, Cash Equivalents, Marketable Securities and Restricted Cash (Current)

Cash, cash equivalents, marketable securities and restricted cash (current), totaled $466.2 million as of December 31, 2022, compared to $787.7 million as of December 31, 2021. The net decrease of $321.5 million in cash, cash equivalents, marketable securities and restricted cash (current) is primarily attributable to net cash used in operating activities of $419.5 million. Net cash used in operating activities during the fiscal year 2022 was partially offset by a $90.0 million upfront payment received under the License, Development and Commercialization Agreement by and among the Company, its affiliate, Navire Pharma, Inc., and Bristol-Myers Squibb Company, or BMS (the "Navire-BMS License Agreement").

During fiscal year 2022, the Company also received $110.0 million from the sale of its priority review voucher, $10.0 million upon closing of an asset purchase agreement between its affiliate, Origin Biosciences, Inc., and Sentynl Therapeutics, Inc. and $4.9 million of net proceeds from the sale of common stock through an "at-the-market" offering. The Company made a $20.5 million mandatory prepayment of a portion of its term loan obligations under its Loan and Security Agreement, as amended, in connection with the upfront payment received from BMS.

As of December 31, 2022, the Company’s restricted cash (current) balance of $37.9 million primarily represents funds in a controlled account that was established in connection with the Second Amendment of the Company’s Amended Loan and Security Agreement. The use of such non-interest-bearing cash is restricted per the terms of the underlying amended loan agreement and is to be used solely for certain research and development expenses directly attributable to the performance of obligations associated with the Navire-BMS License Agreement.

Cash, cash equivalents, marketable securities and restricted cash (current), decreased by $92.3 million when compared to the balance as of September 30, 2022 of $558.5 million. Net cash used in operating activities was $93.2 million for the three months ended December 31, 2022 which primarily contributed to the decline in the cash, cash equivalents, marketable securities and restricted cash (current) during the fourth quarter of fiscal year 2022.

Operating Costs and Expenses
Operating costs and expenses for the three months and year ended December 31, 2022 were $131.1 million and $589.9 million, respectively, as compared to $178.5 million and $646.3 million for the same periods in the prior year. The overall decrease in operating costs and expenses for the three months and year ended December 31, 2022 compared to the comparative periods was mainly due to overall decreases in research, development and other (R&D) expenses and selling, general and administrative expenses resulting from the Company’s reprioritization of its R&D programs and company-wide streamlining of costs. The effects of the Company’s restructuring initiative that began in the first quarter of fiscal year 2022 are now being realized due to reductions of operating costs and expenses. Restructuring, impairment and related charges for the three months and year ended December 31, 2022 of $7.7 million and $43.8 million, respectively, were primarily comprised of winding down costs, exit and other related costs, impairments and write-offs of long-lived assets, and severance and employee-related costs. The Company continues to evaluate its restructuring alternatives to drive operational changes in business processes, efficiencies, and cost savings.

"We head into 2023 with cash on hand providing us with runway into 2024, and as we read out our key upcoming catalysts we expect to continue to allocate our capital carefully in order to preserve that runway and our optionality," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We will also continue to look for ways to extend our runway by considering potential royalty monetizations and partnerships."

The Company’s research and development and other expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While BridgeBio experienced some delays in certain of its clinical enrollment and trial commencement activities, it continues to adapt with alternative site, telehealth and home visits, and at-home drug delivery, as well as mitigation strategies with its contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on BridgeBio’s operating costs and 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,

2022 2021 2022 2021
(Unaudited) (Unaudited) (1)
Revenue $ 1,870 $ 12,886 $ 77,648 $ 69,716
Operating costs and expenses:
Research, development and others 91,549 123,751 402,896 454,138
Selling, general and administrative 31,862 54,749 143,189 192,210
Restructuring, impairment and related charges 7,691 — 43,765 —
Total operating costs and expenses 131,102 178,500 589,850 646,348
Loss from operations (129,232 ) (165,614 ) (512,202 ) (576,632 )
Other income (expense), net:
Interest income 4,092 182 7,542 1,133
Interest expense (19,990 ) (15,134 ) (80,438 ) (46,778 )
Gain from sale of priority review voucher, net — — 107,946 —
Other income (expense), net 4,560 28,284 (7,500 ) 35,823
Total other income (expense), net (11,338 ) 13,332 27,550 (9,822 )
Net loss (140,570 ) (152,282 ) (484,652 ) (586,454 )
Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,979 5,105 3,469 23,915
Net loss attributable to common stockholders of BridgeBio $ (137,591 ) $ (147,177 ) $ (481,183 ) $ (562,539 )
Net loss per share, basic and diluted $ (0.92 ) $ (1.01 ) $ (3.26 ) $ (3.90 )
Weighted-average shares used in computing net loss per share, basic and diluted 149,344,380 145,283,213 147,473,076 144,356,619


Three Months Ended December 31, Year Ended December 31,

Stock-based compensation 2022 2021 2022 2021
(Unaudited) (Unaudited) (1)
Research, development and others $ 8,941 $ 9,654 $ 37,987 $ 56,195
Selling, general and administrative 13,643 12,859 54,669 49,379
Restructuring, impairment and related charges — — 1,172 —
Total stock-based compensation $ 22,584 $ 22,513 93,828 $ 105,574
(1) The condensed consolidated financial statements as of and for the year ended December 31, 2021 are derived from the audited consolidated financial statements as of that date.

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)

December 31 December 31,

2022 2021
(Unaudited) (1)
Assets
Cash and cash equivalents and marketable securities $ 428,269 $ 787,515
Investment in equity securities 43,653 49,148
Receivable from licensing and collaboration agreements 17,079 19,749
Restricted cash, current 37,930 177
Prepaid expenses and other current assets 21,922 32,269
Property and equipment, net 14,569 30,066
Operating lease right-of-use assets 10,678 15,907
Intangible assets, net 28,712 44,934
Other assets 20,224 33,027
Total assets $ 623,036 $ 1,012,792
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable $ 11,558 $ 11,884
Accrued and other liabilities 106,195 118,247
Operating lease liabilities 15,949 22,366
2029 Notes 734,988 733,119
2027 Notes 541,634 539,934
Term loan 430,993 430,752
Other long-term liabilities 26,643 22,069
Redeemable convertible noncontrolling interests (1,589 ) 1,423
Total BridgeBio stockholders’ deficit (1,254,617 ) (870,414 )
Noncontrolling interests 11,282 3,412
Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $ 623,036 $ 1,012,792
(1) The condensed consolidated financial statements as of and for the year ended December 31, 2021 are derived from the audited consolidated financial statements as of that date.

BRIDGEBIO PHARMA, INC.
Consolidated Statements of Cash Flows
(In thousands)

Year Ended December 31,

2022 2021
Unaudited (1)
Operating activities
Net loss $ (484,652 ) $ (586,454 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation 91,559 99,505
Depreciation and amortization 6,771 5,843
Noncash lease expense 5,172 5,611
Net loss (gain) from investment in equity securities 8,222 (29,914 )
Gain from sale of priority review voucher, excluding transaction costs (110,000 ) —
Accrual of payment-in-kind interest on term loan 13,562 —
Gain from recognition of receivable from licensing and collaboration agreement (12,500 ) —
Fair value of shares issued under a license agreement 4,567 —
Accretion of debt 8,570 5,795
Fair value adjustment of warrants 1,571 1,197
Loss on sale of certain assets 6,261 —
Impairment of long-lived assets 12,720 —
LEO call option expense (income) — (5,550 )
Loss on early extinguishment of debt — 3,337
Other noncash adjustments 604 7,092
Changes in operating assets and liabilities: —
Receivable from licensing and collaboration agreements 15,169 (19,749 )
Prepaid expenses and other current assets 7,671 (4,262 )
Other assets 10,971 (9,816 )
Accounts payable (349 ) 2,833
Accrued compensation and benefits (2,362 ) 7,378
Accrued research and development liabilities (4,309 ) 11,178
Accrued professional services (4,996 ) 2,157
Operating lease liabilities (6,245 ) (6,122 )
Deferred revenue 15,262 —
Other accrued and other long-term liabilities (2,733 ) 12,007
Net cash used in operating activities (419,494 ) (497,934 )
Investing activities
Purchases of marketable securities (137,493 ) (589,892 )
Maturities of marketable securities 479,688 380,200
Sales of marketable securities — 62,691
Purchases of investment in equity securities (55,562 ) (53,383 )
Sales of investment in equity securities 52,835 34,150
Increase in cash and cash equivalents from consolidation of PellePharm — 13,654
Payment for intangible assets (1,500 ) (35,000 )
Proceeds from sale of priority review voucher, excluding transaction costs 110,000 —
Proceeds from sale of certain assets 10,000 —
Purchases of property and equipment (4,821 ) (13,246 )
Net cash provided by (used in) investing activities 453,147 (200,826 )
Financing activities
Proceeds from issuance of 2029 Notes — 747,500
Issuance costs and discounts associated with issuance of 2029 Notes — (16,064 )
Purchase of capped calls — (61,295 )
Repurchase of common stock — (200,000 )
Proceeds from issuance of noncontrolling interests — 3,500
Repurchase of Eidos noncontrolling interest, including direct transaction costs — (85,090 )
Proceeds from term loan, net of issuance costs — 456,296
Repayment of term loan (20,486 ) (124,119 )
Proceeds from common stock issuances under ESPP 2,558 3,821
Repurchase of common stock to satisfy tax withholding (1,561 ) (4,746 )
Proceeds from stock option exercises, net of repurchases 666 16,643
Proceeds from issuance of common stock through at-the-market offering, net 4,852 —
Other financing activities 837 —
Net cash (used in) provided by financing activities (13,134 ) 736,446
Net increase in cash, cash equivalents and restricted cash 20,519 37,686
Cash, cash equivalents and restricted cash at beginning of year 396,365 358,679
Cash, cash equivalents and restricted cash at end of year $ 416,884 $ 396,365

Year Ended December 31,

2022 2021
Unaudited (1)
Supplemental Disclosures of Cash Flow Information:
Cash paid for interest $ 54,443 $ 29,774
Supplemental Disclosures of Non-Cash Investing and Financing Information:
Payment-in-kind interest accrued in prior year added to principal of term loan $ 1,763 $ —
Leasehold improvements paid by landlord $ — $ 2,449
Transfers to noncontrolling interests $ (3,512 ) $ (2,124 )
Noncash contribution by a noncontrolling interest $ — $ 21,600
Unpaid property and equipment $ 47 $ 563
Recognized intangible asset recorded in other accrued and other long-term liabilities $ 11,000 $ 12,500
Unpaid debt issuance costs $ — $ 1,120
Net noncash portion of repurchase of Eidos noncontrolling interests $ — $ 38,168
Direct transaction costs in the repurchase of Eidos recorded in additional paid-in capital previously classified in prepaid expenses and other current assets $ — $ 8,749
Reconciliation of Cash, Cash Equivalents and Restricted Cash
Cash and cash equivalents $ 376,689 $ 393,772
Restricted cash (current) 37,930 177
Restricted cash – included in other assets 2,265 2,416
Total cash, cash equivalents and restricted cash at end of year $ 416,884 $ 396,365
(1) The condensed consolidated financial statements as of and for the year ended December 31, 2021 are derived from the audited consolidated financial statements as of that date.