Market Size and Opportunity - The global ATTR market is estimated to be $15B-$20B, with significant growth driven by increased disease awareness and adoption of non-invasive diagnostic tools[17][18] - Infigratinib targets a $4B+ market opportunity with 55,000+ people in the US/EU with achondroplasia and hypochondroplasia[42] - BBP-418 targets a $1B+ market opportunity with 7,000+ patients in the US/EU with LGMD2I/R9[48] - Encaleret targets a $2B+ market opportunity with 25,000+ patients in the US/EU with ADH1 and 200,000+ patients with PSH[52][53] Regulatory Approvals and Designations - Attruby received regulatory approval for the treatment of adult patients with ATTR-CM to reduce cardiovascular death and hospitalization[8] - Infigratinib received Breakthrough Therapy Designation (BTD) for Achondroplasia, BBP-418 received Rare Pediatric Disease Designation (RPDD) for Limb-Girdle Muscular Dystrophy, and BBP-812 received Regenerative Medicine Advanced Therapy Designation (RMAT) for Canavan Disease[6] Clinical Trials and Enrollment - Three fully enrolled Phase 3 trials: FORTIFY (112 patients), CALIBRATE (70 participants), and PROPEL3 (114 participants) evaluating BBP-418, Encaleret, and Infigratinib respectively[5] - Phase 3 study (PROPEL 3) of Infigratinib for achondroplasia is fully enrolled with LPLV expected in 2H 2025[43] - Phase 3 study (FORTIFY) of BBP-418 in LGMD2I/R9 is fully enrolled with topline interim analysis data expected in 2H 2025[49] - Phase 3 study (CALIBRATE) of Encaleret for ADH1 is fully enrolled with topline results expected in 2H 2025[55] Market Access and Distribution - Attruby's market access strategy includes a 28-day free trial, independent specialty pharmacy distribution, and dedicated patient assistance programs[26] Pipeline and Portfolio - The company's pipeline includes Attruby (500,000+ patients, $2B+ market), Infigratinib (55,000+ patients), BBP-418 (7,000+ patients), Encaleret (25,000+ patients), and BBP-812 (1,000 patients)[15] - BridgeBio Oncology Therapeutics has progressed two potentially first-in-class molecules into the clinic with a third expected in 1H 2025[65] Clinical Data and Efficacy - Attruby demonstrated a 42% reduction in cardiovascular-related hospitalization events and a 50% reduction in all-cause mortality and recurrent cardiovascular hospitalization events at Month 30[21] - Attruby's clinical data showed near-complete stabilization of TTR, with acoramidis being 4 times more potent than tafamidis at a fixed plasma concentration[22] - V122I ATTRv-CM has a median survival of 40 months compared to 60 months for ATTRwt-CM[34] Company Achievements and Milestones - Attruby team has successfully led $5B+ portfolio with 12 FDA approvals spanning 6 disease states and 7 indications[31] - BridgeBio expects to hit numerous milestones in 1H and 2H 2025, including EU and Japan approvals for Acoramidis and LPLV for Infigratinib and BBP-418[68] Ecosystem and Innovation - The company's ecosystem leverages a diversified portfolio, operational nimbleness, and world-class decentralized R&D to drive innovation[13][14] Market Penetration and Prescription - Attruby achieved 430 scripts to date, with 77% of Medicare lives in formulary position and 248 unique prescribing HCPs[28]

- As of February 17, 2025, 1,028 unique patient prescriptions for Attruby™ have been written by 516 unique prescribers since FDA approval - Attruby (acoramidis), the first and only near-complete TTR stabilizer (≥90%) was approved by the FDA to reduce cardiovascular death and cardiovascular-related hospitalization in ATTR-CM patients on November 22, 2024 - Acoramidis was approved as BEYONTTRA™ in the EU on February 10, 2025, achieving a $75 million milestone payment and ongoing royalties in a tiered structur ...

PALO ALTO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced that it will report financial results for the fourth quarter and full year ended December 31, 2024, on February 20, 2025. As part of the announcement, the Company will also share updates on Attruby’s commercialization progress and its late-stage clinical pipeline. The Company will begin hosting earnings ...

The approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placeboA 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30A 50% reduction in the cumulative frequency of CVH events relative to placebo at Mo ...

Company Overview - BridgeBio Pharma Inc (NASDAQ: BBIO) develops treatments for genetic conditions [2] - The company's stock has increased by 46.5% since the previous article, primarily due to receiving approval for a treatment [2] Key Developments - BridgeBio Pharma Inc has experienced significant developments since the last analysis, contributing to its stock performance [2] Analyst Background - The author, Myriam Hernandez Alvarez, holds multiple degrees including a Ph.D. in computer applications and has a professional collaboration with Edgar Torres H [2]

Stock Performance - BridgeBio Pharma (BBIO) shares rallied 16% in the last trading session to close at $33 73, driven by notable trading volume [1] - The stock has gained 5 8% over the past four weeks [1] Financial Expectations - The company is expected to post a quarterly loss of $1 09 per share, representing a year-over-year change of -13 5% [2] - Revenues are expected to be $5 3 million, up 202 9% from the year-ago quarter [2] - The consensus EPS estimate for the quarter has been revised 4 1% lower over the last 30 days [3] Industry Comparison - BridgeBio Pharma is part of the Zacks Medical - Generic Drugs industry [3] - Bausch Health (BHC), another stock in the same industry, closed the last trading session 6 1% lower at $7 40 and has returned 0 9% in the past month [3] - For Bausch, the consensus EPS estimate for the upcoming report has changed +0 4% over the past month to $1 12, representing a change of -2 6% from the year-ago quarter [4] Drug Development and Commercial Progress - The stock price rise was attributed to encouraging commercial progress for its recently approved drug, Attruby (acoramidis), pipeline progress, and expected 2025 milestones [5] - Attruby received FDA approval in November 2024 as a near-complete TTR stabilizer to reduce cardiovascular death and cardiovascular-related hospitalization in adult patients with transthyretin amyloid cardiomyopathy [5] - The company has witnessed remarkable momentum for the drug with 430 patient prescriptions written by 248 physicians [5] - Approvals in other geographies for the same indication are anticipated in 2025 [5]

Commercial Progress and FDA Approval - Attruby (acoramidis) received FDA approval on November 22, 2024, marking a significant milestone for the company and the ATTR-CM patient community [1][8] - Since approval, 430 prescriptions have been written by 248 unique physicians, indicating strong initial commercial momentum [1][7][8] - Attruby is indicated to reduce cardiovascular death and cardiovascular-related hospitalization in adult patients with ATTR-CM, a progressive fatal disease [8][10] Pipeline Updates and Clinical Trials - The company has completed enrollment for all three major Phase 3 clinical trials: FORTIFY (BBP-418 for LGMD2I/R9), CALIBRATE (encaleret for ADH1), and PROPEL 3 (infigratinib for achondroplasia) [2][7][9] - FORTIFY, a Phase 3 trial for BBP-418, is fully enrolled with 112 patients, with Last Patient – Last Visit (LPLV) and topline readout expected in the second half of 2025 [9] - CALIBRATE, a Phase 3 trial for encaleret, is fully enrolled with 70 patients, with LPLV and topline readout expected in the second half of 2025 [9] - PROPEL 3, a Phase 3 trial for infigratinib, is fully enrolled with 114 participants, with Last Participant – Last Visit expected in the second half of 2025 [9] Financial and Regulatory Milestones - The company is well-financed with $406 million in cash as of the last quarter and received $500 million upon FDA approval of acoramidis from a royalty facility [7] - Anticipated regulatory milestones include $105 million in the first half of 2025 from acoramidis approvals in Europe and Japan [7] - EU and Japan approvals for acoramidis are expected in the first half of 2024 [3] Product Potential and Market Impact - If successful, BBP-418 would be the first approved therapy for LGMD2I/R9, a rare genetic disorder causing progressive muscle degeneration [9] - Encaleret could become the first approved therapy for ADH1, a rare genetic form of hypoparathyroidism [9] - Infigratinib has the potential to be the first approved oral therapy for children with achondroplasia, the most common form of disproportionate short stature [9] Company Overview - BridgeBio Pharma is a biopharmaceutical company focused on discovering and delivering transformative medicines for genetic diseases [11] - Founded in 2015, the company has a pipeline ranging from early science to advanced clinical trials [11]

PALO ALTO, Calif., Jan. 08, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced that co-founder and CEO, Neil Kumar, Ph.D., will present at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA on Monday, January 13 at 7:30 am PT. To access the live webcast of BridgeBio’s presentation, please visit the “Events & Presentations” page within the Investors section of t ...

The Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of acoramidis in the EU based on positive results from the Phase 3 ATTRibute-CM study; final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission within the coming monthsAcoramidis, a near-complete (≥90%) stabilizer of transthyretin (TTR), was approved on November 22, 2024, by the U.S. Food and Drug Administration (FDA) as Attruby™ to reduce cardiovascular death an ...

SANTA CRUZ, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Unnatural Products, Inc. (UNP), a biotech developing orally-delivered macrocyclic peptides to address previously undruggable targets, announced today that BridgeBio Pharma, Inc. (Nasdaq: BBIO), has exercised its option to license macrocyclic peptide candidates discovered using UNP’s AI-enabled massively parallel chemistry platform. This milestone signifies a significant milestone in the collaboration between the two companies, and reinforces the potentia ...