BridgeBio Pharma, Inc. (BBIO) recently announced that it obtained FDA approval for its pipeline candidate acoramidis for the treatment of adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization.The candidate has been approved under the brand name Attruby.BBIO stock surged 16.1% on the FDA approval of the drug.BridgeBio’s shares have lost 32.6% in the year-to-date period against the industry's growth of 10.9%.Image Source: Zacks ...

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Core Insights - BridgeBio Pharma has received FDA approval for Attruby™, the first and only product with a label indicating near-complete stabilization of Transthyretin (TTR) for treating adults with ATTR-CM, aimed at reducing cardiovascular death and related hospitalizations [1][9] Group 1: Product Efficacy and Study Results - Attruby demonstrated a 42% reduction in composite all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) at Month 30 compared to placebo [1] - The treatment showed a 50% reduction in cumulative frequency of CVH events at Month 30 relative to placebo [1] - In the ATTRibute-CM Phase 3 study, Attruby met its primary endpoint with a Win Ratio of 1.8 (p<0.0001) and showed significant improvements in quality of life metrics [4] Group 2: Patient Support and Access Programs - BridgeBio will provide Attruby free for life to U.S. clinical trial participants as a gesture of appreciation [1] - The company offers a patient support services program, ForgingBridges™, to assist patients and families in accessing Attruby, including insurance resources and financial assistance [5] Group 3: Financial and Market Implications - Following the approval, BridgeBio will receive a $500 million payment under its royalty funding agreement [1] - The company plans to pursue global approvals for Attruby, with a Marketing Authorization Application submitted to the European Medicines Agency, expecting a decision in 2025 [7]

Core Insights - BridgeBio Pharma announced positive results from the PROPEL 2 Phase 2 trial of infigratinib for children with achondroplasia, showing a significant increase in annualized height velocity and an encouraging safety profile [1][2][3] Group 1: Clinical Trial Results - In Cohort 5 of PROPEL 2, a daily oral treatment of infigratinib at 0.25 mg/kg resulted in a mean change in annualized height velocity of +2.50 cm/year at Month 18 (P=0.001) [1][3] - The mean change from baseline in height Z-score was +0.54 (P<0.001) compared to an untreated achondroplasia population at Month 18 [3] - Statistically significant improvement in body proportionality was observed with a mean upper to lower body segment ratio change of -0.12 (P=0.001) at Month 18 [3] Group 2: Safety and Tolerability - Oral infigratinib was well tolerated at Month 18, with no serious adverse events or treatment-emergent adverse events leading to treatment discontinuation [3] - No accelerated progression of bone age or negative changes in bone mineral density were observed, indicating a favorable safety profile [3] Group 3: Regulatory Designations - Infigratinib has received Breakthrough Therapy Designation, Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the U.S. FDA [1][4] - These designations highlight the potential of infigratinib to demonstrate substantial improvement in efficacy over existing therapies [2][4] Group 4: Future Developments - PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, is on schedule for enrollment completion by the end of 2024 [1][5] - The company is committed to exploring the broader medical and functional impacts of infigratinib on skeletal dysplasias [5] Group 5: Company Overview - BridgeBio Pharma focuses on discovering and delivering transformative medicines for genetic diseases, with a pipeline that spans from early science to advanced clinical trials [7] - Founded in 2015, the company aims to apply advances in genetic medicine to benefit patients as quickly as possible [7]

Group 1 - The marketplace channel Haggerston BioHealth offers exclusive stock tips focused on Pharma, Biotech, and Healthcare, providing access to investment bank-grade financial models and research [1] - The group caters to both novice and experienced biotech investors, offering insights on catalysts, buy and sell ratings, product sales forecasts, and integrated financial statements [2] - Edmund Ingham, a biotech consultant with over 5 years of experience, leads the Haggerston BioHealth investing group and has compiled detailed reports on over 1,000 companies [2]

BridgeBio Pharma (BBIO) came out with a quarterly loss of $0.86 per share versus the Zacks Consensus Estimate of a loss of $1.03. This compares to loss of $1.08 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 16.50%. A quarter ago, it was expected that this rare disease drug developer would post a loss of $1.09 per share when it actually produced a loss of $1.02, delivering a surprise of 6.42%.Over the last four quarters, the ...

PART I. FINANCIAL INFORMATION This section presents BridgeBio Pharma's unaudited condensed consolidated financial statements, including balance sheets, operations, comprehensive loss, equity, and cash flows. [Item 1. Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents BridgeBio Pharma's unaudited condensed consolidated financial statements, including balance sheets, operations, comprehensive loss, equity, and cash flows. [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Balance sheets show decreased cash, increased total assets (restricted cash, investments), stable liabilities, and improved stockholders' deficit. | Metric | September 30, 2024 (in thousands) | December 31, 2023 (in thousands) | |:---|:---|:---| | Cash and cash equivalents | $266,324 | $375,935 | | Restricted cash | $139,409 | $16,653 | | Total current assets | $444,578 | $477,593 | | Investment in nonconsolidated entities | $160,443 | — | | Total assets | $664,977 | $546,380 | | Total current liabilities | $139,216 | $143,844 | | Total liabilities | $1,883,369 | $1,888,915 | | Total stockholders' deficit | $(1,219,037) | $(1,343,013) | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Reduced net loss for Q3 and YTD 2024, primarily from deconsolidation gains and increased revenue, despite higher SG&A expenses. | Metric | Three Months Ended Sep 30, 2024 (in thousands) | Three Months Ended Sep 30, 2023 (in thousands) | Nine Months Ended Sep 30, 2024 (in thousands) | Nine Months Ended Sep 30, 2023 (in thousands) | |:---|:---|:---|:---|:---| | Revenue | $2,732 | $4,091 | $216,020 | $7,558 | | Research and development | $120,444 | $125,136 | $376,111 | $325,485 | | Selling, general and administrative | $68,819 | $35,777 | $194,149 | $103,007 | | Loss from operations | $(191,750) | $(157,692) | $(366,946) | $(429,954) | | Gain on deconsolidation of subsidiaries | $52,027 | — | $178,321 | — | | Net loss | $(164,254) | $(179,488) | $(275,958) | $(482,923) | | Net loss attributable to common stockholders of BridgeBio | $(162,040) | $(176,999) | $(270,712) | $(475,054) | | Net loss per share (basic and diluted) | $(0.86) | $(1.08) | $(1.46) | $(2.99) | [Condensed Consolidated Statements of Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) Comprehensive loss improved for Q3 and YTD 2024, totaling **$(164.2) million** and **$(276.0) million** respectively, driven by reduced net loss. | Metric | Three Months Ended Sep 30, 2024 (in thousands) | Three Months Ended Sep 30, 2023 (in thousands) | Nine Months Ended Sep 30, 2024 (in thousands) | Nine Months Ended Sep 30, 2023 (in thousands) | |:---|:---|:---|:---|:---| | Net loss | $(164,254) | $(179,488) | $(275,958) | $(482,923) | | Other comprehensive loss (gains) | $9 | $(29) | $(26) | $362 | | Comprehensive loss | $(164,245) | $(179,517) | $(275,984) | $(482,561) | | Comprehensive loss attributable to common stockholders of BridgeBio | $(162,031) | $(177,028) | $(270,738) | $(474,692) | [Condensed Consolidated Statements of Redeemable Convertible Noncontrolling Interests and Stockholders' Deficit](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Redeemable%20Convertible%20Noncontrolling%20Interests%20and%20Stockholders'%20Deficit) Stockholders' deficit improved, reflecting increased common stock and additional paid-in capital from equity offerings and compensation, and a reduced accumulated deficit. | Metric | September 30, 2024 (in thousands) | December 31, 2023 (in thousands) | |:---|:---|:---|\n| Common stock | $195 | $181 | | Treasury stock | $(275,000) | $(275,000) | | Additional paid-in capital | $1,876,091 | $1,481,032 | | Accumulated deficit | $(2,831,213) | $(2,560,501) | | Total BridgeBio stockholders' deficit | $(1,229,922) | $(1,354,257) | | Total stockholders' deficit | $(1,219,037) | $(1,343,013) | - Issuance of common stock under public offerings, net, contributed **$314,741 thousand** to additional paid-in capital for the nine months ended September 30, 2024[10](index=10&type=chunk) - Stock-based compensation added **$27,125 thousand** to additional paid-in capital for the nine months ended September 30, 2024[10](index=10&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net increase in cash, cash equivalents, and restricted cash was **$13.1 million** for YTD 2024, a significant decrease from prior year, due to lower financing activities. | Metric | Nine Months Ended Sep 30, 2024 (in thousands) | Nine Months Ended Sep 30, 2023 (in thousands) | |:---|:---|:---|\n| Net cash used in operating activities | $(325,400) | $(402,901) | | Net cash provided by investing activities | $64,018 | $54,099 | | Net cash provided by financing activities | $274,526 | $456,058 | | Net increase in cash, cash equivalents and restricted cash | $13,144 | $107,256 | | Cash, cash equivalents and restricted cash at end of period | $407,876 | $524,140 | - Cash paid for interest increased to **$78,236 thousand** for the nine months ended September 30, 2024, from **$50,826 thousand** in the prior year[14](index=14&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail BridgeBio's financial position, operations, and cash flows, covering accounting policies, fair value, debt, licensing, stock compensation, and restructuring. [1. Organization and Description of Business](index=10&type=section&id=1.%20Organization%20and%20Description%20of%20Business) BridgeBio, a genetic disease biopharmaceutical company, divested subsidiaries for cost streamlining and faces going concern doubt, contingent on acoramidis FDA approval and a **$500 million** milestone. - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on genetic diseases, with a pipeline ranging from early science to advanced clinical trials[15](index=15&type=chunk) - During the nine months ended September 30, 2024, BridgeBio divested and deconsolidated Portal Therapeutics, Inc., Sub21, Inc., and TheRas, Inc. to streamline costs and explore business opportunities[15](index=15&type=chunk) - The company concluded there is substantial doubt about its ability to continue as a going concern for one year from the issuance date, contingent on receiving a **$500.0 million** milestone payment upon FDA approval of acoramidis, anticipated in late November 2024[15](index=15&type=chunk) [2. Summary of Significant Accounting Policies](index=11&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) Outlines key accounting policies: basis of presentation, consolidation, cash classification, restricted cash, equity method investments, accrued liabilities, and estimates. - The condensed consolidated financial statements are prepared in accordance with GAAP and SEC regulations for interim reporting, reflecting all normal recurring adjustments[17](index=17&type=chunk) - Cash equivalents are highly liquid investments with original maturities of 90 days or less, primarily money market instruments and U.S. treasury bills[18](index=18&type=chunk) - Restricted cash increased significantly to **$139.4 million** as of September 30, 2024, primarily due to funds deposited into escrow accounts from asset sale transactions under the Financing Agreement[21](index=21&type=chunk) [3. Fair Value Measurements](index=15&type=section&id=3.%20Fair%20Value%20Measurements) Financial assets and liabilities are measured at fair value (Level 1, 2, 3

Exhibit 99.1 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 th ...

- Case study co-authored by members of BridgeBio senior management and BridgeBio co-founder and MIT professor, Andrew W. Lo, Ph.D. - Case study features BridgeBio’s unique model that uses portfolio theory to enable early-stage research for genetic diseases and conditions that are not typically addressed by the pharmaceutical industry PALO ALTO, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on ge ...

Core Viewpoint - Upcoming FDA decisions for BridgeBio Pharma, PTC Therapeutics, and Vertex Pharmaceuticals could lead to significant stock price movements before the end of 2024 [1] Group 1: BridgeBio Pharma - BridgeBio Pharma's acoramidis is under FDA review for treating transthyretin amyloidosis cardiomyopathy (ATTR-CM), with a decision expected by Nov. 29 [2] - Recent evidence shows acoramidis reduced the risk of death by 42% and hospitalization due to cardiovascular events by 50% compared to a placebo [2] - BridgeBio has a market cap of $4.7 billion, and a positive FDA decision could significantly increase its stock value [3] Group 2: PTC Therapeutics - PTC Therapeutics has two approved therapies and receives royalties from Roche for Evrysdi, generating $118 million in product sales and $53 million in royalties in Q2 [4] - The FDA is reviewing Upstaza, a gene therapy for AADC deficiency, with a decision expected by Nov. 13 [4] - PTC Therapeutics has a market cap of $2.8 billion, and a positive decision on Upstaza could enhance its stock performance [5] Group 3: Vertex Pharmaceuticals - Vertex Pharmaceuticals' lead drug, Trikafta, is projected to generate over $10.5 billion in sales this year, treating over 68,000 cystic fibrosis patients [6] - The FDA is reviewing a new once-daily dosing regimen for Vertex, with a decision expected by Jan. 2, 2025 [6] - Trikafta patents are expected to maintain market exclusivity until 2037, and a new approval could further solidify Vertex's market position [7]