On May 2, 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 first quarter ended March 31, 2024, and provided an update on the Company’s operations (Press release, BridgeBio, MAY 2, 2024, View Source [SID1234642580]).

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"In addition to the important scientific work we have done to advance our patient mission this quarter, we have also taken a first step towards streamlining our firm and thereby unlocking value for patients and investors alike," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "We believe the launch of BBOT better aligns investors with an opportunity that was being hidden in our pipeline, and our partnership with these new investors enables us to prosecute these oncology programs more quickly and with higher fidelity. In tandem with our progress in corporate simplification and balance sheet strengthening, we continue to prepare for what we believe will be a world class launch this year in ATTR-CM and we continue to enroll our achondroplasia, ADH1 and LGMD2I/R9 clinical trials, all of which are expected to complete enrollment this year."

BridgeBio’s late stage programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):

The EMA accepted the Company’s MAA for acoramidis for review, with an expected approval in 2025. This is in addition to the Prescription Drug User Fee Act (PDUFA) date of November 29, 2024 set by the U.S. FDA for the Company’s NDA for acoramidis for the treatment of ATTR-CM.

In March 2024, the Company entered into a licensing agreement granting Bayer exclusive rights to commercialize acoramidis for ATTR-CM in Europe; in exchange, BridgeBio is entitled to receive among other consideration:

Tiered royalties beginning in the low-thirties percent, and

Up to $310 million in upfront and near-term milestone payments, as well as additional sales milestones.

The Company presented cardiac magnetic resonance imaging evidence from an exploratory substudy where treatment with acoramidis was associated with possible cardiac structural and functional improvement compared with placebo, with potential cardiac amyloid regression. This evidence is consistent with clinical improvement observed in the Company’s ATTRibute-CM Phase 3 study of acoramidis in patients with ATTR-CM.

Additional detailed results of the Company’s ATTRibute-CM study are planned for presentation at 2024 medical meetings, including four abstracts accepted for presentation at the European Society of Cardiology Heart Failure conference in May 2024, and eleven abstracts accepted for presentation at the International Symposium on Amyloidosis in May 2024.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:

The PROPEL 3 global Phase 3 registrational study of infigratinib in achondroplasia continues to enroll; study completion continues to be anticipated for 2025.

The Company anticipates initiating its clinical program for hypochondroplasia in 2024, as part of its commitment to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):

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.

FORTIFY, the global Phase 3 registrational trial of BBP-418 in LGMD2I/R9, continues to enroll in the U.S., Europe and Australia. Full enrollment of the interim analysis population is expected in 2024.

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.
Recent Corporate Updates:

The Company announced the launch of BridgeBio Oncology Therapeutics (BBOT) with a $200 million private financing to accelerate the development of its novel precision oncology pipeline.
The $200 million financing was led by Cormorant Asset Management and co-led by Omega Funds, with participation from affiliates of Deerfield Management, GV (Google Ventures), EcoR1 Capital, Wellington Management, Enavate Sciences, Surveyor Capital (a Citadel company), Aisling Capital, Casdin Capital, and Longwood Fund.
BBOT will be led by Eli Wallace, Ph.D. as CEO and Pedro Beltran, Ph.D. as CSO; the Board of Directors for BBOT will be chaired by Frank McCormick, Ph.D., David A. Wood Distinguished Professor of Tumor Biology and Cancer Research at UCSF and advisor to the National Cancer Institute’s RAS Initiative at Frederick National Laboratory for Cancer Research. Raymond Kelleher, M.D., Ph.D., Managing Director Cormorant Asset Management, Michelle Doig, Partner, Omega Funds, and Neil Kumar, Ph.D., CEO of BridgeBio will serve as directors.
First 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 $519.8 million as of March 31, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $127.2 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 $437.7 million, net proceeds received of $315.3 million from various equity financings, 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 and inclusive of prepayment fees and exit-related costs in aggregate of $473.4 million, net cash used in operating activities of $219.5 million and purchases of equity securities of $20.3 million during the three months ended March 31, 2024.

Revenue

Revenue for the three months ended March 31, 2024, were $211.1 million, as compared to $1.8 million for the same period in the prior year. The $209.3 million net increase for the three months ended March 31, 2024, compared to the same period in the prior year, was primarily due to the recognition of non-refundable upfront payments under the Bayer and the Kyowa Kirin exclusive license agreements. The remaining license and services revenue was recognized in connection with the Navire-BMS License Agreement, which was entered into in May 2022 and for which BMS provided notice of termination in March 2024 with a termination effective in June 2024.

Operating Costs and Expenses

Operating costs and expenses for the three months ended March 31, 2024 were $210.8 million compared to $128.0 million for the same period in the prior year.

The overall increase of $82.8 million in operating costs and expenses for the three months ended March 31, 2024, compared to the same period in the prior year, was primarily due to an increase of $48.1 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, and an increase of $34.7 million in selling, general and administrative (SG&A) expenses mainly to support commercialization readiness efforts. Operating costs and expenses for the three months ended March 31, 2024, include $22.5 million of nonrecurring deal-related costs for transactions that were closed during the period.

Restructuring, impairment and related charges for the three months ended March 31, 2024 amounted to $3.4 million. 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 period in the prior year was $3.4 million. 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 March 31, 2024 were $28.9 million, of which $12.8 million is included in R&D expenses, and $16.1 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 $23.5 million, of which $11.8 million is included in R&D expenses, and $11.7 million is included in SG&A expenses.

"With our recent equity financing activities, our licensing deal with Bayer for European commercial rights to acoramidis, our royalty funding agreement for $500 million upon FDA approval of acoramidis, and now the private financing of BBOT to advance our oncology pipeline into the clinic, we are in a strong financial position to launch acoramidis in the U.S. at the end of this year and deliver three Phase 3 readouts in 2025," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio.