BridgeBio Pharma Reports Third Quarter 2024 Financial Results and Business Update

- Patients on acoramidis, a near complete (≥90%) TTR stabilizer in clinical development, lived longer and better as shown in the ATTRibute-CM study. This is the only Phase 3 study of an ATTR-CM disease-modifying treatment to demonstrate improvement in hard clinical outcomes in the combined assessment of CVH and ACM to this degree, this quickly 

- BridgeBio's late-stage pipeline continues to rapidly progress with three Phase 3 readouts expected in 2025. Program highlights from this quarter include:  

- CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, completed screening

- FORTIFY, the Phase 3 clinical trial for LGMD2I/R9, completed enrollment

- The FDA granted Breakthrough Therapy Designation to oral infigratinib for achondroplasia

- Regenerative Medicine Advanced Therapy Designation awarded to BBP-812 for Canavan disease

- Published a MIT Business School case study on BridgeBio's pioneering business model in the 50th edition of the Journal of Portfolio Management celebrating the work of Harry Markowitz, the Nobel prize-winning inventor of Modern Portfolio Theory, outlining our efforts to build a new type of biopharmaceutical company 

- The Company ended the quarter with $406 million in cash, cash equivalents, and short-term restricted cash. It anticipates receiving a $500 million milestone payment under our royalty funding agreement upon FDA approval of acoramidis, as well as $105 million in aggregate regulatory milestone payments upon approval of acoramidis in European and Japanese territories

PALO ALTO, Calif., Nov. 12, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a new type of biopharmaceutical company focused on genetic diseases, today reported its financial results for the third quarter ended September 30, 2024, and provided an update on the Company's operations.

"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:

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. 
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• 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.

"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)