Clinical Development and Pipeline - BridgeBio Pharma has developed a late-stage clinical pipeline with the FDA approval of Attruby (acoramidis) for treating ATTR-CM, targeting a patient population of over 400,000 in the U.S. and EU[26][28]. - Attruby demonstrated a win ratio of 1.8 in the Phase 3 ATTRibute-CM study, with a highly statistically significant p-value (p<0.0001) for reducing cardiovascular-related hospitalization and mortality[30]. - Acoramidis has shown a statistically significant reduction in all-cause mortality within 36 months, confirming its clinical benefit in treating ATTR-CM[34]. - The FDA approval of Attruby was based on positive data from the Phase 3 ATTRibute-CM clinical trial, which met its primary endpoint[30]. - Acoramidis is designed to stabilize TTR and reduce amyloid deposition, addressing the underlying cause of ATTR[40][42]. - The company initiated the ACT-EARLY Phase 3 trial in 2024 to evaluate prophylactic acoramidis therapy for asymptomatic pathogenic TTR variant carriers[54]. - The Phase 3 ATTRibute-CM trial met its primary endpoint with a win ratio of 1.8 and a p-value of less than 0.0001, indicating significant clinical benefit[50]. - In the same trial, 45% of acoramidis-treated participants showed improvement in NT-proBNP levels, 40% in 6MWD, and 13% in NYHA class[51]. - A post-hoc analysis revealed a 42% reduction in composite all-cause mortality and recurrent cardiovascular hospitalization events at 30 months with acoramidis treatment compared to placebo[52]. - Acoramidis showed a 36% risk reduction in all-cause mortality at Month 36 and a 46% reduction in composite all-cause mortality and cardiovascular hospitalization at Month 36[53]. - Updated results from the Phase 2 OLE showed that at least 53% of patients with symptomatic ATTR-CM survived for a median follow-up of 4.6 years[46]. - In the Phase 2 open-label extension, the all-cause mortality rate was 8.5% and cardiovascular-related hospitalizations were 25.5% after a median follow-up of 15 months[45]. Market Potential and Commercialization - The total global addressable market for ATTR therapeutic interventions could reach as high as $20.0 billion, with diagnosed ATTR-CM patients in the U.S. increasing from fewer than 5,000 in 2019 to over 50,000 in 2024[35]. - The company anticipates Bayer will commercially launch Beyonttra in Europe during the first half of 2025 following its approval[26][29]. - Attruby was commercially launched in the U.S. following FDA approval on November 22, 2024, targeting approximately 240,000 ATTR-CM patients in the U.S. and a global patient population of around 500,000[98][99]. - The market opportunity for achondroplasia and other FGFR-driven skeletal dysplasias is estimated to exceed $5.0 billion globally[63]. - Low-dose infigratinib is the only oral direct FGFR1-3 inhibitor in clinical development for achondroplasia, facing competition from BioMarin's Voxzogo, which is approved in multiple regions[74]. - The prevalence of hypochondroplasia is estimated to be one in 15,000 to 40,000, with no approved treatments available apart from growth hormone in Japan[77]. - The market for ADH1 is estimated to have 25,000 carriers of causative variants in the EU and US, representing significant commercial potential for encaleret if approved[84]. Financial Agreements and Licensing - The Bayer License Agreement includes an upfront payment of $135.0 million and potential milestone payments of up to $150.0 million, with additional sales milestones of up to $450.0 million[31][32]. - Eidos received an upfront payment of $25.0 million from Alexion for the exclusive license to develop and commercialize Beyonttra in Japan, with potential for an additional one-time payment of $30.0 million upon achieving a regulatory milestone[122]. - Eidos incurred and paid $8.1 million in licensing fees to Stanford related to the Bayer License Agreement in March 2024, and recognized a milestone payable of $0.5 million upon receiving FDA approval for Attruby[128]. - Under the Novartis License, a one-time payment of $15.0 million was made to Novartis, with contingent milestone payments totaling $60.0 million upon achieving certain regulatory milestones[134]. - The company has an ongoing obligation to inform Novartis of its intent to seek regulatory approval for infigratinib, with potential reversionary rights to Novartis if regulatory approval is not pursued[133]. - The Amended Financing Agreement provides for a senior secured credit facility of up to $750.0 million, including an initial term loan of $450.0 million[137]. - The Funding Agreement allows for Royalty Interest Payments equal to 5% of global net sales of acoramidis, adjustable to a maximum of 10% under certain conditions[138]. - The company is required to use commercially reasonable efforts to develop, manufacture, and commercialize at least one licensed product under the agreement with Stanford[129]. Regulatory and Compliance - The FDA must make a decision on accepting an NDA or BLA for filing within 60 days of receipt, with a target of ten months for initial review of a new molecular entity NDA[157]. - Orphan drug designation is granted for products intended to treat rare diseases affecting fewer than 200,000 individuals in the U.S., and provides seven years of exclusivity upon first FDA approval for that indication[159][162]. - The FDA conducts pre-approval inspections of manufacturing facilities to ensure compliance with cGMP requirements before approving an NDA or BLA[158]. - User fees are required for NDA or BLA submissions under the Prescription Drug User Fee Act, with waivers available for small businesses and orphan drugs[155]. - The FDA may request additional information before accepting an NDA or BLA for filing, which can extend the review process[157]. - The FDA requires post-marketing monitoring and reporting of adverse experiences for approved products[176]. - Manufacturers must comply with cGMP regulations and are subject to inspections to ensure product quality and safety[180]. - The Drug Supply Chain Security Act (DSCSA) mandates obligations for pharmaceutical manufacturers, wholesale distributors, and dispensers over a 10-year period culminating in November 2023, with a stabilization period extending to November 2024[181]. - The FDA has allowed an exemption period for eligible trading partners until May 27, 2025, for those who have made documented efforts to complete data connections but still face challenges[181]. - The FDA's review of a premarket approval (PMA) application typically takes between 6 to 10 months, but often extends to several years[187]. - The FDA has required premarket approval for nearly all companion diagnostics for cancer therapies, with intentions to initiate a reclassification process for most in vitro diagnostics in January 2024[189]. - The FDA's guidance indicates that a drug cannot be approved without the approval of the corresponding companion diagnostic if the diagnostic is essential for safe and effective use[188]. Legal and Compliance Risks - The company may face additional pricing pressures due to ongoing legislative and regulatory developments aimed at increasing transparency in drug pricing[216]. - The company is subject to data privacy and security regulations, including HIPAA and state laws like the California Consumer Privacy Act, which impose significant compliance costs[207][208]. - Legislative changes, such as the Affordable Care Act (ACA), have increased pharmaceutical manufacturers' rebate liabilities and imposed new requirements for Medicaid and Medicare[212]. - The ACA requires a 70% point-of-sale discount for branded drugs dispensed to Medicare Part D beneficiaries in the coverage gap[212]. - The company faces potential legal risks and increased compliance costs due to evolving state privacy laws and proposed federal privacy legislation[210][211]. - The company must ensure compliance with various healthcare laws to avoid significant civil, criminal, and administrative penalties, which could adversely affect financial results[202]. - Drug manufacturers can be held liable under federal civil and criminal false claims laws, with penalties including three times the actual damages sustained by the government for violations[204]. - The federal Physician Payments Sunshine Act requires manufacturers to report certain payments or transfers of value to healthcare providers annually[197]. - Companies must comply with various federal and state fraud and abuse laws, which may include marketing restrictions and reporting requirements related to healthcare items or services[199]. - Federal and state enforcement bodies have increased scrutiny of healthcare company interactions, leading to investigations and potential penalties for non-compliance with healthcare laws[202]. Intellectual Property and Manufacturing - The company has over 100 issued patents and more than 400 patent applications, providing a robust intellectual property portfolio to support its product candidates[109]. - The company relies on third-party contract manufacturing organizations (CMOs) for production and has long-term agreements in place to support the commercialization of its products[97]. - The company maintains workers' compensation insurance but does not have coverage for environmental liability or toxic tort claims, which may expose it to significant risks[222].

Group 1 - BridgeBio's shares (NASDAQ: BBIO) are approaching 52-week highs following strong fourth quarter results and a positive commercial update on Attruby, which has seen over 1,000 unique patient prescriptions in the United States [2] - The Growth Stock Forum focuses on identifying attractive risk/reward situations in growth stocks, particularly in the biotech sector, and maintains a model portfolio of 15-20 stocks that is updated regularly [2] Group 2 - The article reflects the author's personal opinions and does not constitute investment advice or recommendations for buying or selling stocks [3][4]

Company Performance - BridgeBio Pharma reported a quarterly loss of $1.31 per share, which was worse than the Zacks Consensus Estimate of a loss of $1.09, and compared to a loss of $0.96 per share a year ago, indicating a significant decline in performance [1] - The company posted revenues of $5.88 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 10.98%, and showing a substantial increase from year-ago revenues of $1.75 million [2] - Over the last four quarters, BridgeBio Pharma has surpassed consensus EPS estimates three times and topped consensus revenue estimates two times [2] Stock Outlook - The stock has gained approximately 32.3% since the beginning of the year, significantly outperforming the S&P 500's gain of 4.5% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.89 on revenues of $41.63 million, and for the current fiscal year, it is -$3.66 on revenues of $223.42 million [7] - The estimate revisions trend for BridgeBio Pharma is currently favorable, leading to a Zacks Rank 2 (Buy) for the stock, suggesting it is expected to outperform the market in the near future [6] Industry Context - The Medical - Generic Drugs industry, to which BridgeBio Pharma belongs, is currently ranked in the bottom 22% of over 250 Zacks industries, indicating potential challenges ahead [8] - Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions, which could impact investor sentiment and stock performance [5]

Core Insights - BridgeBio Pharma, Inc. reported strong commercial progress for Attruby™, with 1,028 unique patient prescriptions written by 516 healthcare providers since FDA approval on November 22, 2024 [1][2] - Attruby (acoramidis) is the first near-complete TTR stabilizer approved to reduce cardiovascular death and related hospitalizations in patients with ATTR-CM [1][6] - The company ended Q4 2024 with $681 million in cash and expects to receive $105 million in regulatory milestones in the first half of 2025 from acoramidis approvals in Europe and Japan [1][7] Commercial Progress - As of February 17, 2025, Attruby has achieved significant prescription volume, indicating strong market acceptance [2] - The Chief Commercial Officer highlighted the product's category-leading results, including a 42% reduction in all-cause mortality and a 50% reduction in cardiovascular hospitalizations at 30 months [3] Pipeline Overview - The company has fully enrolled three global registrational studies: FORTIFY for BBP-418, CALIBRATE for encaleret, and PROPEL 3 for infigratinib, with results expected before the end of 2025 [1][5] - Acoramidis was also approved as BEYONTTRA™ in the EU, achieving a $75 million milestone payment and ongoing royalties [1][6] Financial Updates - Revenue for Q4 2024 was $5.9 million, up from $1.7 million in Q4 2023, primarily due to the first commercial sales of Attruby [8][9] - For the full year 2024, revenue increased to $221.9 million from $9.3 million in 2023, driven by upfront payments and service revenue from collaboration agreements [10] Operating Costs and Expenses - Operating costs for Q4 2024 were $231.9 million, compared to $179.2 million in Q4 2023, largely due to increased selling, general, and administrative expenses to support Attruby's commercialization [11][12] - The company reported a net loss attributable to common stockholders of $265.1 million for Q4 2024, compared to $168.1 million in Q4 2023 [21][22] Cash Flow and Liquidity - The company experienced a net increase of $288.5 million in cash, cash equivalents, and short-term restricted cash during 2024, ending the year with $681 million [7][28] - Cash used in operating activities for the year was $520.7 million, reflecting ongoing investments in R&D and commercialization efforts [28]

Core Insights - BridgeBio Pharma, Inc. is set to report its financial results for Q4 and the full year of 2024 on February 20, 2025, along with updates on Attruby's commercialization and its late-stage clinical pipeline [1] - The company plans to start hosting earnings calls with the Q1 earnings release, anticipated in late April or early May [2] - Founded in 2015, BridgeBio focuses on discovering and delivering transformative medicines for genetic diseases, with a pipeline that spans from early science to advanced clinical trials [3]

Core Points - BridgeBio Pharma, Inc. has received marketing authorization from the European Commission for acoramidis, branded as BEYONTTRA™, for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) [1][4] - Acoramidis is a selective small molecule that stabilizes transthyretin (TTR) by ≥90% and is administered orally [1][8] - Bayer will handle all commercial activities for acoramidis in the EU, with a launch planned for the first half of 2025 [4][5] Clinical Study Results - The approval is based on the pivotal ATTRibute-CM Phase 3 study, which involved 632 participants and demonstrated significant benefits in cardiovascular outcomes [3][7] - The study showed a 42% reduction in composite all-cause mortality (ACM) and recurrent cardiovascular-related hospitalizations (CVH) events at month 30 compared to placebo [7] - Acoramidis is the first and only approved treatment for ATTR-CM in the U.S. and EU with a label specifying near-complete stabilization of TTR [7] Financial Aspects - BridgeBio will receive a $75 million milestone payment from Bayer upon European Commission approval and will earn tiered royalties starting in the low-thirties percent on sales of acoramidis in the EU [5][7] - Since its U.S. approval in November 2024, BridgeBio has reported strong commercial momentum with 430 patient prescriptions written by 248 physicians [4][5] Future Prospects - Acoramidis is currently under review for approval by the Japanese Pharmaceuticals and Medical Devices Agency and the Brazilian Health Regulatory Agency [6]

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]

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]

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]