ATA-100

– Announced positive two-year topline results of sevasemten ARCH open label trial and advanced GRAND CANYON pivotal cohort in Becker – – Positive interim topline data for DUNE trial of sevasemten in Becker – – Expanded Phase 2 LYNX trial of sevasemten in Duchenne – – Anticipated to overenroll Phase 2 FOX trial in Duchenne boys previously treated with gene therapy – – Initiated Phase 2 CIRRUS-HCM trial of EDG-7500 in patients with obstructive HCM – BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2024 and recent business highlights. “2024 is off to a great start with tremendous progress on our skeletal and cardiac muscle programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Most recently, we initiated our Phase 2 CIRRUS-HCM trial of EDG-7500 and announced positive 2-year topline data from our ARCH open label trial in Becker. We expect significant catalysts on our programs throughout this year including data from the CANYON and CIRRUS studies.” Recent Highlights Musculoskeletal Program / Sevasemten Becker Muscular Dystrophy (Becker) Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy. There are currently no approved therapies for individuals with Becker, a serious genetic, progressive neuromuscular disorder with significant unmet need. ARCH open-label trial in Becker: The Company announced positive two-year topline results from the ARCH trial, an open label, single-center trial assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten in adults with Becker. The ARCH trial evaluated sevasemten administered daily over two years in adults with Becker. Sevasemten was well-tolerated in all 12 participants with no discontinuations or dose reductions due to adverse events. During two years of sevasemten treatment, participants’ North Star Ambulatory Assessment (NSAA) scores stabilized and continued to diverge relative to declines reported across multiple Becker natural history studies.1, 2,3 In addition, significant decreases in key biomarkers of muscle damage including creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2) were observed in participants treated with sevasemten, which are consistent with prior observations. The positive results from the two-year ARCH trial further support the hypothesis that a reduction in contraction-induced muscle damage has the potential to preserve and improve muscle function and disease progression in Becker. CANYON Phase 2 placebo-controlled trial in adults with Becker: CANYON, the largest interventional Becker trial to date, includes 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. The primary endpoint of CANYON is change in CK over the treatment period with additional measures collected, including NSAA, 100-meter timed test, biomarkers of muscle damage and MRI. The Company expects to report CANYON data in the fourth quarter of 2024. GRAND CANYON, a global pivotal cohort in Becker: GRAND CANYON, an expansion of the CANYON placebo-controlled trial, is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker. The primary endpoint of GRAND CANYON is change in NSAA. In addition, other functional assessments, biomarkers of muscle damage and safety will be assessed. GRAND CANYON is an 18-month cohort anticipated to recruit approximately 120 individuals with Becker, aged between 18 and 50 years old and a NSAA score between 5 and 32, at up to 50 sites in 10 countries. Data from GRAND CANYON, if positive, could support a marketing application. To learn more, go to clinicaltrials.gov (NCT05291091) or the GRAND CANYON microsite: . DUNE Phase 2 Exercise Challenge trial in adults with Becker, LGMD2I or McArdle disease: The Company continues to advance the DUNE Phase 2 Exercise Challenge study. This 16-week randomized, double-blind, placebo-controlled study with an open label extension to 78 weeks, assesses safety, PK, and biomarker response to exercise in adults with Becker, limb-girdle muscular dystrophy type 2I (LGMD2I) or McArdle disease. Unlike Becker, LGMD2I is a muscular dystrophy caused by a dysfunctional dystroglycan complex while McArdle is caused by deficiencies in glycogen mobilization leading to metabolic crisis and injury of skeletal muscle. The study was designed with a 3-week screening period, which included a controlled exercise challenge and subsequent biomarker analysis, principally CK and TNNI2, followed by a 16-week double-blind period in which participants were randomized 2:1 to receive once-daily treatment with sevasemten (15 mg) or matching placebo. At week 12, participants underwent a second controlled exercise challenge to determine whether treatment with sevasemten conferred protection against exercise induced muscle damage. Topline interim results showed that sevasemten was well tolerated across all 21 participants (Becker (n=9), LGMD2I (n=9) and McArdle (n=3)). Biomarker reductions in the Becker cohort were consistent with results observed in the ARCH trial, including statistically significant decreases in TNNI2 and CK versus patients on placebo; sevasemten treatment significantly reduced mean CK by 45% (p<0.05 vs placebo) and TNNI2 by 89% (p<0.05 vs placebo). Over the 24 hours post-exercise, sevasemten treatment also significantly reduced biomarkers of muscle damage associated with exercise with a mean post-exercise reduction of CK of 49% (p<0.001 vs placebo) and TNNI2 of 75% (p=0.07 vs placebo). Biomarker data from the LGMD2I and McArdle cohorts are currently inconclusive and would likely require additional participants to interpret any response. The Company, in partnership with Dr. John Vissing, the lead investigator for DUNE, are continuing to compile and analyze the data which will be presented in full at a future medical meeting. DUNE represents the first placebo-controlled trial with sevasemten in individuals with Becker. The preliminary data from DUNE, combined with the 2-year observations from ARCH, continue to support the hypothesis that protecting fast muscle fibers with sevasemten limits contraction-induced muscle damage and offers the potential to halt disease progression in individuals with Becker. Duchenne Muscular Dystrophy LYNX Phase 2 trial in boys with Duchenne: LYNX is a 2-part multi-center, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, PK, and biomarkers of muscle damage in over 60 children aged 4 to 9 years with Duchenne treated with oral, once-daily sevasemten. The trial will also explore changes in functional measures such as NSAA, stride velocity 95th centile (SV95%) and self-reported/caregiver-reported outcomes. Part A of the trial will include a 12-week, randomized, double-blind, placebo-controlled dose ranging study period, followed by Part B, a 92-week open-label extension. Dosing in Part B was adjusted to a previously studied dose, based on the interim analysis of the higher dose for safety as well as assessment of biomarkers of muscle damage in that cohort. LYNX was designed to identify a Phase 3 dose of sevasemten that will reduce biomarkers of muscle damage and has the potential to provide functional benefit to individuals with Duchenne. To date, across five cohorts, sevasemten achieved exposures predicted to provide benefit based on preclinical models and was observed to be safe and well tolerated. Moreover, decreases in biomarkers of muscle damage with sevasemten treatment were observed. This observation provides confidence that exposures are achieving target levels and the Company is initiating a 6th cohort to validate observations thus far. The Company plans to report LYNX data, including safety, PK, changes in biomarkers of muscle damage and functional changes in NSAA and SV95 in the fourth quarter of 2024. The Company will rely on LYNX data, along with data from the FOX trial of Duchenne boys previously treated with gene therapy, to guide the design and powering of a Phase 3 trial in Duchenne, planned to be initiated in the first half of 2025. For more information on LYNX go to clinicaltrials.gov to learn more about this trial (NCT05540860). FOX Phase 2 trial in boys with Duchenne (previously treated with gene therapy): The Company is advancing FOX, a Phase 2 placebo-controlled trial to assess the effect of sevasemten over 12 weeks on safety, PK and biomarkers of muscle damage in children and adolescents aged 6 to 14 years with Duchenne who have been previously treated with gene therapy. The trial will also explore changes in functional measures such as NSAA, SV95% and self-reported/caregiver-reported outcomes. The Company expects to substantially overenroll the trial from the original plan of 24 participants. There has been exceptional enthusiasm from the Duchenne community for this trial, evident in the Company’s ability to complete the trial’s enrollment within two months. Go to clinicaltrials.gov to learn more about this trial (NCT06100887). Received Orphan Drug from the European Medicines Agency (EMA) and Fast Track from the U.S. Food & Drug Administration (FDA): The EMA granted Orphan Drug Designation (ODD) for sevasemten for the treatment of Becker and for the treatment of Duchenne in April 2024. The FDA granted sevasemten Fast Track designation for the treatment of Duchenne in February 2024. The FDA previously granted ODD for the treatment of Duchenne and Becker, Rare Pediatric Disease Designation for the treatment of Duchenne and Fast Track designation for sevasemten for the treatment of Becker. Cardiovascular Program / EDG-7500 EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with hypertrophic cardiomyopathy (HCM) and other diseases of diastolic dysfunction. Preclinical data in models of both obstructive and non-obstructive HCM suggest the ability to drive a broadly effective clinical response at a low risk of decreasing left ventricular ejection fraction below normal at all doses tested. Based on EDG-7500’s self-limiting effect on systolic contraction observed in preclinical models, the Company plans to investigate fixed-dose regimens of EDG-7500, thus potentially avoiding intensive safety monitoring of patients on current therapy. Phase 2 CIRRUS-HCM trial of EDG-7500: In April 2024, the first patient was dosed in CIRRUS-HCM, a multi-center, two-part, open-label trial of EDG-7500 in patients with obstructive HCM at up to 20 clinical sites in the U.S. The trial will evaluate the safety, tolerability, PK and pharmacodynamics of EDG-7500 in up to thirty adults with obstructive HCM. Participants enrolled in this trial will receive EDG-7500 as a single oral dose and will have the option of then receiving multiple oral doses for 28 days. To learn more about this trial (NCT06347159), go to clinicaltrials.gov. Phase 1 Trial of EDG-7500: The Company is continuing the randomized, placebo-controlled, single and multiple ascending dose Phase 1 trial evaluating safety, tolerability, PK and pharmacodynamics in healthy adults. To learn more about this trial (NCT06011317), go to clinicaltrials.gov. The Company expects to report data from the single dose arm of CIRRUS-HCM and the Phase 1 trial of EDG-7500 in healthy volunteers in the third quarter of 2024. Furthermore, the Company expects to initiate a 28-day trial in patients with obstructive and non-obstructive HCM in the second half of 2024. The Company also expects to begin an open-label extension trial of EDG-7500 in the fourth quarter of 2024. Strengthened Engagement with the Scientific and Patient Communities Musculoskeletal Program / sevasemten The Company continued its education and outreach on its musculoskeletal program with the medical and patient communities, including presentations at the Muscular Dystrophy Association Clinical and Scientific Conference, the American Academy of Neurology 2024 Annual Meeting and the 8th International Myology Congress. The Company continues to sponsor and participate in numerous other clinician and patient-focused events. Cardiovascular Program / EDG-7500 The Company sponsored and attended several cardiovascular-focused conferences, building relationships with the medical and patient communities. During the quarter, the Company presented preclinical data on the effects of EDG-7500 in a model of non-obstructive HCM at the American College of Cardiology's 2024 Annual Scientific Session. First Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $532.8 million as of March 31, 2024. Research and development (R&D) expenses were $27.7 million for both the first quarter of 2024 and for the immediately preceding quarter. During the first quarter of 2024 there was an additional $1.8 million in clinical trial expenses for the sevasemten and EDG-7500 clinical programs from increasing clinical trial enrollment, and $0.3 million of higher personnel-related costs. These increases were offset by a $1.1 million decrease in drug discovery and preclinical costs, a $0.8 million decrease in costs related to the formulation, manufacture and clinical supply of our drug product candidates, and a $0.2 million decrease in other allocated costs. General and Administrative (G&A) expenses were $7.1 million for the first quarter of 2024, compared to $6.2 million for the immediately preceding quarter. The increase of $0.9 million was primarily driven by $0.6 million in increased professional and consulting costs and $0.3 million in personnel-related costs. Net loss and net loss per share for the first quarter of 2024 was $28.5 million or $0.33 per share, compared to $30.1 million or $0.47 per share for the immediately preceding quarter. About Edgewise Therapeutics Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: or follow us on LinkedIn, X (formerly Twitter), Facebook, Instagram and Threads. References [1] Bello L, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439 [2] van de Velde NM, et al. Selection approach to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522. doi: 10.1212/WNL.0000000000012233. [3] De Wel B, et al. Lessons for future clinical trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding, Edgewise’s product candidates and programs, including sevasemten and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the CANYON Phase 2 trial, LYNX Phase 2 trial, the single dose arm of CIRRUS-HCM and the Phase 1 trial of EDG-7500 in healthy volunteers); statements regarding the advancement of Edgewise’s research and development programs; the timing of the initiation of a Phase 3 trial of sevasemten in Duchenne, a 28-day trial in patients with obstructive and non-obstructive HCM and an open-label extension trial of EDG-7500; the possibility of data from GRAND CANYON to support a marketing application; statements regarding Edgewise’s pipeline of product candidates and programs; statements regarding Edgewise’s anticipated milestones; statements regarding potential overenrollment of the FOX Phase 2 trial; and statements by Edgewise’s President and Chief Executive Officer. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete clinical trials for, obtain approvals for and commercialize any of its product candidates; Edgewise’s ability to take advantage of potential benefits associated with designations granted by FDA and/or to maintain qualifications for applicable designations over time; the timing, progress and results of clinical trials for sevasemten and EDG-7500; Edgewise’s ability to enroll and maintain patients in clinical trials; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; the potential that the outcome of preclinical testing and early clinical trials may not be predictive of the success of later clinical trials; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Edgewise Therapeutics, Inc. Condensed Statement of Operations (in thousands except share and per share amounts, unaudited) Three months ended March 31, 2024 December 31, 2023 Operating expenses: Research and development $ 27,694 $ 27,684 General and administrative 7,059 6,178 Total operating expenses 34,753 33,862 Loss from operations (34,753 ) (33,862 ) Interest income 6,228 3,719 Net loss $ (28,525 ) $ (30,143 ) Net loss per share - basic and diluted $ (0.33 ) $ (0.47 ) Weighted-average shares outstanding, basic and diluted 87,567,307 64,774,775 Edgewise Therapeutics, Inc. Condensed Balance Sheet Data (in thousands, unaudited) March 31, December 31, 2024 2023 Assets Cash, cash equivalents and marketable securities $ 532,759 $ 318,393 Other assets 21,047 21,642 Total assets $ 553,806 $ 340,035 Liabilities and stockholders' equity Liabilities 18,865 21,205 Stockholders' equity 534,941 318,830 Total liabilities and stockholders' equity $ 553,806 $ 340,035

- Presented cardiac magnetic resonance (CMR) imaging evidence consistent with clinical improvement observed in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) in the ATTRibute-CM Phase 3 study of acoramidis at the American College of Cardiology Annual Scientific Sessions; additional detailed results from ATTRibute-CM are planned for presentation at 2024 medical meetings, including four abstracts accepted for the European Society of Cardiology Heart Failure conference and eleven abstracts accepted for the International Symposium on Amyloidosis in May 2024 - 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 alpha dystroglycan (αDG) levels as a surrogate endpoint; the FORTIFY Phase 3 trial of BBP-418 in limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) continues to enroll, with full enrollment of the interim analysis population expected in 2024 - BridgeBio secured up to $1.5 billion in a Q1 2024 capital campaign that included a European licensing agreement for acoramidis in ATTR-CM, a Japanese licensing agreement for infigratinib in skeletal dysplasias, a financing in exchange for a 5% royalty on future acoramidis net sales, a credit facility refinancing existing senior secured credit, and primary stock sales - 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 Company’s Marketing Authorization Application (MAA) for acoramidis was accepted by review for the European Medicines Agency (EMA), with an expected approval in 2025; this is in addition to the November 29, 2024 PDUFA date set by the U.S. Food and Drug Administration (FDA) for the company’s New Drug Application (NDA) for acoramidis for the treatment of ATTR-CM - The Company ended the quarter with $520 million in cash, cash equivalents, marketable securities and short-term restricted cash PALO ALTO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today reported its financial results for the first quarter ended March 31, 2024, and provided an update on the Company’s operations. “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. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Operations (in thousands, except shares and per share amounts) Three Months Ended March 31, 2024 2023 (Unaudited) Revenue $ 211,120 $ 1,826 Operating costs and expenses: Research, development and other expenses 141,570 93,512 Selling, general and administrative 65,807 31,108 Restructuring, impairment and related charges 3,400 3,369 Total operating costs and expenses 210,777 127,989 Income (loss) from operations 343 (126,163 ) Other expense, net: Interest income 4,075 4,153 Interest expense (23,471 ) (20,121 ) Loss on extinguishment of debt (26,590 ) — Other income (expense), net 9,483 (601 ) Total other expense, net (36,503 ) (16,569 ) Net loss (36,160 ) (142,732 ) Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 944 2,576 Net loss attributable to common stockholders of BridgeBio $ (35,216 ) $ (140,156 ) Net loss per share, basic and diluted $ (0.20 ) $ (0.92 ) Weighted-average shares used in computing net loss per share, basic and diluted 178,705,310 152,645,635 Three Months Ended March 31, Stock-based Compensation 2024 2023 (Unaudited) Research, development and other expenses $ 12,779 $ 11,779 Selling, general and administrative 16,071 11,698 Total stock-based compensation $ 28,850 $ 23,477 BRIDGEBIO PHARMA, INC. Condensed Consolidated Balance Sheets (In thousands) March 31, December 31, 2024 2023 Assets (Unaudited) (1 ) Cash and cash equivalents and marketable securities $ 519,691 $ 375,935 Investments in equity securities - 58,949 Receivables from licensing and collaboration agreements 235,494 1,751 Short-term restricted cash 131 16,653 Prepaid expenses and other current assets 28,108 24,305 Property and equipment, net 11,414 11,816 Operating lease right-of-use assets 8,052 8,027 Intangible assets, net 25,721 26,319 Other assets 20,722 22,625 Total assets $ 849,333 $ 546,380 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit Accounts payable $ 4,728 $ 10,655 Accrued and other liabilities 118,248 122,965 Operating lease liabilities 12,841 13,109 Deferred revenue 33,847 9,823 2029 Notes, net 737,392 736,905 2027 Notes, net 543,823 543,379 Term loan, net 434,717 446,445 Other long-term liabilities 595 5,634 Redeemable convertible noncontrolling interests 525 478 Total BridgeBio stockholders' deficit (1,049,528 ) (1,354,257 ) Noncontrolling interests 12,145 11,244 Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $ 849,333 $ 546,380 (1 ) The condensed consolidated financial statements as of and for the year ended December 31, 2023 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC. Condensed Consolidated Statements of Cash Flows (Unaudited) (In thousands) Three Months Ended March 31, 2024 2023 Operating activities: Net loss $ (36,160 ) $ (142,732 ) Adjustments to reconcile net loss to net cash used in operating activities: Loss on extinguishment of debt 26,590 — Stock-based compensation 17,057 21,907 Accretion of debt 2,015 2,338 Depreciation and amortization 1,596 1,633 Noncash lease expense 1,069 1,032 Accrual of payment-in-kind interest on term loan — 3,339 Loss on deconsolidation of PellePharm — 1,241 Gain from investment in equity securities, net (8,136 ) (964 ) Other noncash adjustments 1,631 (314 ) Changes in operating assets and liabilities: Receivables from licensing and collaboration agreements (233,743 ) 6,318 Prepaid expenses and other current assets (3,345 ) (3,542 ) Other assets 444 (483 ) Accounts payable (5,927 ) (3,800 ) Accrued compensation and benefits (14,969 ) (18,369 ) Accrued research and development liabilities 11,168 (2,556 ) Operating lease liabilities (1,595 ) (1,250 ) Deferred revenue 24,024 (1,748 ) Accrued professional and other liabilities (1,256 ) (6,372 ) Net cash used in operating activities (219,537 ) (144,322 ) Investing activities: Purchases of marketable securities (44,395 ) — Maturities of marketable securities — 18,000 Purchases of investments in equity securities (20,271 ) (47,474 ) Proceeds from sales of investments in equity securities 63,229 42,287 Proceeds from special cash dividends received from investments in equity securities 25,682 — Payment for an intangible asset (797 ) — Purchases of property and equipment (695 ) (12 ) Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm — (503 ) Net cash provided by investing activities 22,753 12,298 Financing activities: Proceeds from term loan under Financing Agreement 450,000 — Issuance costs and discounts associated with term loan under Financing Agreement (12,254 ) — Repayment of term loan under Loan and Security Agreement (473,417 ) — Proceeds from issuance of common stock through public offerings, net 315,254 143,016 Proceeds from BridgeBio common stock issuances under ESPP 2,364 1,809 Proceeds from stock option exercises, net of repurchases 537 193 Repurchase of RSU shares to satisfy tax withholding (2,936 ) (512 ) Other financing activities — 5,743 Net cash provided by financing activities 279,548 150,249 Net increase in cash, cash equivalents and restricted cash 82,764 18,225 Cash, cash equivalents and restricted cash at beginning of period 394,732 416,884 Cash, cash equivalents and restricted cash at end of period $ 477,496 $ 435,109 Three Months Ended March 31, 2024 2023 Supplemental Disclosure of Cash Flow Information: Cash paid for interest $ 35,315 $ 22,059 Supplemental Disclosures of Noncash Investing and Financing Information: Unpaid issuance costs associated with term loan under Financing Agreement $ 3,732 $ — Unpaid public offering issuance costs $ 513 $ — Deferred and unpaid issuance costs recorded to "Accrued professional and other accrued liabilities" $ 458 $ — Unpaid property and equipment $ 70 $ 96 Transfers to noncontrolling interests $ (1,857 ) $ (2,843 ) Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $ 475,222 $ 407,368 Restricted cash 131 25,503 Restricted cash — Included in “Other assets” 2,143 2,238 Total cash, cash equivalents and restricted cash at end of period shown in the condensed consolidated statements of cash flows $ 477,496 $ 435,109 About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter. BridgeBio Pharma, Inc. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the clinical and therapeutic, market potential of the Company’s programs and product candidates, including the statements in Dr. Kumar’s and Dr. Stephenson’s quotes regarding the potential commercial launch of acoramidis (if approved), continued advancement in the Company’s pipeline, including enrollments in clinical trials and anticipated readouts, and other benefits resulting from recent financing activities; the statements related to the FDA’s planned actions regarding the Company’s NDA for acoramidis for the treatment of ATTR-CM; the potential outcomes of regulatory reviews by the FDA and the EMA; the timing and success of the Company’s clinical development programs, including the progress of the Company’s clinical development program for acoramidis for patients with ATTR-CM, and the Company’s plan for, and the expected timing of, presenting additional detailed results of ATTRibute-CM study at medical meetings; the potential payments we may receive under the recent license agreement with Bayer; the continuation of PROPEL 3, the Company’s Phase 3 study of infigratinib for achondroplasia and the expected timing for full enrollment in the study; the Company’s commitment to exploring the potential of infigratinib and the expectation and timing of the initiation of the Company’s clinical program for hypochondroplasia; the continuation and progress of FORTIFY, the Phase 3 trial of BBP-418 for LGMD2I/R9, including the ongoing enrollment in the United States, Europe and Australia, the expectation and timing of full enrollment of the interim analysis population, and the potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA; the continued enrollment in CALIBRATE, the Phase 3 clinical trial of encaleret, and the timing for sharing topline data from CALIBRATE; the expectation to accelerate the development of the Company’s novel precision oncology pipeline; and the Company’s financial performance, capitalization status, strategy, business plans and goals reflect the Company’s current views about the Company’s plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from the Company’s preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations the Company’s product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, despite having ongoing and future interactions with the FDA or other regulatory agencies to discuss potential paths to registration for the Company’s product candidates, the FDA or such other regulatory agencies not agreeing with the Company’s regulatory approval strategies, components of the Company’s filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of the Company’s collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in the Company’s share price, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. BridgeBio Contact: Vikram Bali contact@bridgebio.com (650)-789-8220