Muscular Dystrophy, Limb-Girdle, Type 2I

- Submitted New Drug Application (NDA) to US Food and Drug Administration (FDA) for acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) based on positive results of Phase 3 ATTRibute-CM trial, which were published in the New England Journal of Medicine; NDA has been accepted for review with a PDUFA date of November 29, 2024; Marketing Authorization Application (MAA) for acoramidis has also been accepted by the European Medicines Agency (EMA) - Presented additional data from ATTRibute-CM at the American Heart Association Scientific Sessions, demonstrating separation at Month 3 of the placebo and acoramidis time-to-first-event Kaplan-Meier curves for a composite of all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH); separation was sustained through Month 30 and represents the most rapid clinical benefit on the composite endpoint of ACM and CVH in ATTR-CM patients to the Company’s knowledge - Shared positive results of single-arm Phase 3 study of acoramidis in Japanese ATTR-CM patients, including no mortality reported over the 30 month acoramidis treatment period - PROPEL 3, the Company's Phase 3 study of infigratinib for achondroplasia continues to enroll with full enrollment expected in 2024; the Company has also announced a partnership granting Kyowa Kirin exclusive license on infigratinib for skeletal dysplasias in Japan in exchange for an upfront payment of $100 million, royalties up to the high twenties percent, and additional milestone-based payments - FORTIFY, the Company's Phase 3 study BBP-418 for limb-girdle muscular dystrophy type 2I (LMGD2I), continues to enroll, with full enrollment of interim analysis population expected in 2024 - CALIBRATE, the Company's Phase 3 study of encaleret for autosomal dominant hypocalcemia type 1 (ADH1) continues to enroll, with full enrollment expected in 2024 and topline data expected in 2025 - Secured up to $1.25 billion of capital from Blue Owl and CPP investments, including $500 million in cash in exchange for a 5% royalty on future global net sales of acoramidis and a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions - Ended the quarter with $393 million in cash, cash equivalents, and short-term restricted cash, and $59 million of investments in equity securities PALO ALTO, Calif., Feb. 22, 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 fourth quarter and full year ended December 31, 2023, and provided an update on the Company’s operations. “Our focus this year is executing on the launch of acoramidis for patients with ATTR cardiomyopathy,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio. “At the same time, we are also focused on fully enrolling three ongoing Phase 3 clinical trials by the end of 2024. Finally, we hope that reading out potentially exciting data from our Phase 1/2 trial in congenital adrenal hyperplasia later this year will let us take the next step in serving that patient community.” BridgeBio’s key programs: Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): The Company filed an NDA for acoramidis for the treatment of ATTR-CM with the US FDA; the NDA was accepted for review with a PDUFA date of November 29, 2024. The Company has also filed a Marketing Authorization Application for acoramidis with the EMA, which has been accepted for review.The regulatory filings were based on data from the Phase 3 ATTRibute-CM study, which met its primary endpoint (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001). Additional results from ATTRibute-CM include: An 81% survival rate on acoramidis, which approaches the survival rate in the age-matched U.S. database (~85%), and a 0.29 mean annual CVH rate on acoramidis, which approaches the annual hospitalization rate observed in the broader U.S. Medicare population (~0.26);Improvements from baseline observed for a large proportion of participants treated with acoramidis on laboratory and functional measures including n-terminal prohormone of brain natriuiretic peptide (NT-proBNP) and 6-minute walk distance;Rapid clinical benefit on the composite endpoint of ACM and CVH in participants treated with acoramidis, demonstrated by placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30 as presented at the American Heart Association Scientific Sessions in November 2023; andAcoramidis was well-tolerated with no safety signals of potential clinical concern identified. The Company also shared positive results of an open-label, single-arm Phase 3 study conducted in Japan by licensing partner Alexion, AstraZeneca Rare Disease, including that no mortality was reported over the 30 month acoramidis treatment period.Additional detailed results of ATTRibute-CM are planned for presentation at 2024 medical meetings. Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia: In December 2023, the Company announced the dosing of the first child in PROPEL 3, its global Phase 3 registrational study of infigratinib in achondroplasia.In February 2024, the Company announced a partnership with Kyowa Kirin wherein the Company grants Kyowa Kirin an exclusive license to develop and commercialize infigratinib for achondroplasia, hypochondroplasia, and other skeletal dysplasias in Japan; in exchange, the Company will receive an upfront payment of $100 million as well as royalties up to the high-twenties percent on sales of infigratinib in Japan, with the potential for additional milestone-based payments.The Company is committed to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias, and anticipates initiating its clinical program for hypochondroplasia in 2024. BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9): FORTIFY, the global Phase 3 registrational trial of BBP-418, continues to enroll in the U.S. with clinical trial sites planned for Europe and Australia. Full enrollment of the interim analysis population is expected in 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 αDG levels as a surrogate endpoint. Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): CALIBRATE, the Phase 3 clinical trial of encaleret, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025. Recent Corporate Updates: ·Secured up to $1.25 billion of capital from Blue Owl and CPP Investments: The raise includes $500 million in cash from Blue Owl and CPP Investments available upon FDA approval of acoramidis in exchange for a 5% royalty on future global net sales of acoramidis, as well as a $450 million credit facility from Blue Owl that refinanced existing senior secured credit, extending maturity from 2026 to 2029 subject to certain conditions. Fourth Quarter and Full Year 2023 Financial Results: Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash Cash, cash equivalents and short-term restricted cash, totaled $392.6 million as of December 31, 2023, compared to cash, cash equivalents, marketable securities and short-term restricted cash of $466.2 million as of December 31, 2022. The net decrease of $73.6 million in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net cash used in operating activities of $527.7 million and $6.9 million in repurchase of shares to satisfy tax withholdings, primarily offset by net proceeds received of $449.8 million from various equity financings, $6.0 million from stock option exercises, and $3.4 million from common stock issuances under our employee stock purchase plan during the year ended December 31, 2023. Revenue Revenue for the three months and year ended December 31, 2023 were $1.7 million and $9.3 million, respectively, as compared to $1.9 million and $77.6 million for the same periods in the prior year, respectively. The net decreases of $0.2 million and $68.3 million for the three months and year ended December 31, 2023, respectively, compared to the same periods in the prior year, were primarily due to license revenue recognized in 2022 upon the transfer of the license in accordance with the Navire-BMS License Agreement which was entered into in May 2022. Operating Costs and Expenses Operating costs and expenses for the three months and year ended December 31, 2023 were $179.2 million and $616.7 million, respectively, compared to $131.1 million and $589.9 million, for the same periods in the prior year, respectively. The overall increase of $48.1 million in operating costs and expenses for the three months ended December 31, 2023, compared to the same period in the prior year, was primarily due to an increase of $39.3 million in research and development and other expenses (R&D) to advance the Company's pipeline of development programs, an increase of $15.7 million in selling, general and administrative (SG&A) expenses to support commercialization readiness efforts, offset by a decrease of $6.9 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year. The overall increase of $26.8 million in operating costs and expenses for the year ended December 31, 2023 , compared to the same period in the prior year, was primarily due to an increase of $55.2 million in R&D expenses to advance the Company's pipeline of development programs, an increase of $7.4 million in SG&A expenses to support commercialization readiness efforts, offset by a decrease of $35.8 million in restructuring, impairment and related charges given that the majority of the restructuring initiatives occurred in the prior year. Restructuring, impairment and related charges for the three months and year ended December 31, 2023, amounted to $0.8 million and $7.9 million, respectively. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs. Restructuring, impairment and related charges for the same periods in the prior year were $7.7 million and $43.8 million, respectively. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. Stock-based compensation expenses included in operating costs and expenses for the three months ended December 31, 2023 were $37.1 million, of which $22.5 million is included in R&D expenses, and $14.6 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 $22.6 million, of which $8.9 million is included in R&D expenses, and $13.6 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the year ended December 31, 2023 were $115.0 million, of which $61.6 million is included in R&D expenses, and $53.4 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 $93.8 million, of which $38.0 million is included in R&D expenses, $54.7 million is included in SG&A expenses, and $1.2 million is included in restructuring, impairment and related charges. “Coming off of our recent royalty financing, we find ourselves well capitalized to launch acoramidis this year alongside strong new partners who share our confidence in acoramidis’ potential in the ATTR-CM market,” said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. “We are excited for this launch, as well as for the continued advancement of our late stage pipeline, which we hope will allow us to serve patients with genetic diseases both directly with the advancement of those medicines towards the market as well as by diversifying our top line revenue and enabling reinvestment into the R&D and business development opportunities that will allow us to be sustainable in the long term.” BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Operations(in thousands, except shares and per share amounts) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Revenue $1,745 $1,870 $9,303 $77,648 Operating costs and expenses: Research, development and other expenses 130,824 91,549 458,157 402,896 Selling, general and administrative 47,583 31,862 150,590 143,189 Restructuring, impairment and related charges 754 7,691 7,926 43,765 Total operating costs and expenses 179,161 131,102 616,673 589,850 Loss from operations (177,416) (129,232) (607,370) (512,202)Other income (expense), net: Interest income 5,578 4,092 18,038 7,542 Interest expense (20,268) (19,990) (81,289) (80,438)Gain from sale of priority review voucher, net — — — 107,946 Other income (expense), net 21,778 4,560 17,370 (7,500)Total other income (expense), net 7,088 (11,338) (45,881) 27,550 Net loss (170,328) (140,570) (653,251) (484,652)Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,180 2,979 10,049 3,469 Net loss attributable to common stockholders of BridgeBio $(168,148) $(137,591) $(643,202) $(481,183)Net loss per share, basic and diluted $(0.96) $(0.92) $(3.95) $(3.26)Weighted-average shares used in computing net loss per share, basic and diluted 174,462,332 149,344,380 162,791,511 147,473,076 Three Months Ended December 31, Year Ended December 31, Stock-based Compensation 2023 2022 2023 2022 (Unaudited) (Unaudited) (1) Research, development and others $22,495 $8,941 $61,647 $37,987 Selling, general and administrative 14,638 13,643 53,369 54,669 Restructuring, impairment and related charges — — — 1,172 Total stock-based compensation $37,133 $22,584 $115,016 $93,828 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Balance Sheets(In thousands) December 31, December 31, 2023 2022 (Unaudited) (1) Assets Cash, cash equivalents and marketable securities $375,935 $428,269 Investment in equity securities 58,949 43,653 Receivable from licensing and collaboration agreements 1,751 17,079 Short-term restricted cash 16,653 37,930 Prepaid expenses and other current assets 24,305 21,922 Property and equipment, net 11,816 14,569 Operating lease right-of-use assets 8,027 10,678 Intangible assets, net 26,319 28,712 Other assets 22,625 20,224 Total assets $546,380 $623,036 Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit Accounts payable $10,655 $11,558 Accrued and other liabilities 129,061 106,195 Operating lease liabilities 13,109 15,949 2029 Notes, net 736,905 734,988 2027 Notes, net 543,379 541,634 Term loan, net 446,445 430,993 Other long-term liabilities 9,361 26,643 Redeemable convertible noncontrolling interests 478 (1,589)Total BridgeBio stockholders' deficit (1,354,257) (1,254,617)Noncontrolling interests 11,244 11,282 Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $546,380 $623,036 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. BRIDGEBIO PHARMA, INC.Condensed Consolidated Statements of Cash Flows(In thousands) Year Ended December 31, 2023 2022 (Unaudited) (1 ) Operating activities: Net loss $(653,251) $(484,652)Adjustments to reconcile net loss to net cash used in operating activities: Stock-based compensation 108,710 91,559 Depreciation and amortization 6,494 6,771 Noncash lease expense 4,032 5,172 Accrual of payment-in-kind interest on term loan 10,207 13,562 Loss on deconsolidation of PellePharm 1,241 — (Gain) loss from investment in equity securities, net (18,314) 8,222 Fair value of shares issued under a license agreement — 4,567 Accretion of debt 8,907 8,570 Fair value adjustment of warrants (984) 1,571 Loss on sale of certain assets — 6,261 Impairment of long-lived assets — 12,720 Gain from sale of priority review voucher, excluding transaction costs — (110,000)Gain from recognition of receivable from licensing and collaboration agreement — (12,500)Other noncash adjustments 181 604 Changes in operating assets and liabilities: Receivable from licensing and collaboration agreements 15,328 15,169 Prepaid expenses and other current assets (2,702) 7,671 Other assets (1,546) 10,971 Accounts payable 2,780 (349)Accrued compensation and benefits 7,802 (2,362)Accrued research and development liabilities (9,855) (4,309)Operating lease liabilities (4,829) (6,245)Deferred revenue (5,438) 15,262 Accrued professional and other liabilities 3,517 (7,729)Net cash used in operating activities (527,720) (419,494)Investing activities: Purchases of marketable securities (29,726) (137,493)Maturities of marketable securities 82,550 479,688 Purchases of investment in equity securities (107,538) (55,562)Sales of investment in equity securities 110,556 52,835 Decrease in cash and cash equivalents resulting from deconsolidation of PellePharm (503) — Payment for intangible asset — (1,500)Proceeds from sale of priority review voucher — 110,000 Proceeds from sale of certain assets — 10,000 Purchases of property and equipment (1,306) (4,821)Net cash provided by investing activities 54,033 453,147 Financing activities: Proceeds from issuance of common stock through Private Placement offering, net 240,796 — Proceeds from issuance of common stock through Follow-on offering, net 144,049 — Proceeds from issuance of common stock through ATM offering, net 64,965 4,852 Transactions with noncontrolling interests (801) — Repayment of term loan — (20,486)Proceeds from BridgeBio common stock issuances under ESPP 3,398 2,558 Repurchase of RSU shares to satisfy tax withholding (6,880) (1,561)Proceeds from stock option exercises, net of repurchases 6,008 666 Other financing activities — 837 Net cash provided by (used in) financing activities 451,535 (13,134)Net increase (decrease) in cash, cash equivalents and restricted cash (22,152) 20,519 Cash, cash equivalents and restricted cash at beginning of period 416,884 396,365 Cash, cash equivalents and restricted cash at end of period $394,732 $416,884 Year Ended December 31, 2023 2022 (Unaudited) (1) Supplemental Disclosure of Cash Flow Information: Cash paid for interest $61,108 $54,443 Supplemental Disclosures of Noncash Investing and Financing Information: Unpaid property and equipment $100 $47 Recognized intangible asset recorded in “Other accrued and other long-term liabilities” $— $11,000 Transfers (to) from noncontrolling interests $(10,534) $(3,512)Payment-in-kind interest added to principal of term loan $— $1,763 Reconciliation of Cash, Cash Equivalents and Restricted Cash: Cash and cash equivalents $375,935 $376,689 Short-term restricted cash 16,653 37,930 Restricted cash — Included in “Other assets” 2,144 2,265 Total cash, cash equivalents and restricted cash at end of periods $394,732 $416,884 (1)The condensed consolidated financial statements as of and for the year ended December 31, 2022 are derived from the audited consolidated financial statements as of that date. 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 our 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 our pipeline, including enrollments in clinical trials and anticipated readout, and other benefits resulting from recent financing; the statements related to the FDA's planned actions regarding our 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 our clinical development programs, including the progress of our clinical development program for acoramidis for patients with ATTR-CM, and our plan for, and the expected timing of, presenting additional detailed results of ATTRibute-CM study at medical meetings; the potential success of our partnership granting Kyowa Kirin an exclusive license on infigratinib for skeletal dysplasias in Japan and the potential payments we may receive under the license; the continuation of PROPEL 3, our Phase 3 study of infigratinib for achondroplasia and the expected timing for full enrollment in the study; our commitment to exploring the potential of infigratinib and the expectation and timing of the initiation of our clinical program for hypochondroplasia; the continuation and progress of FORTIFY, the Phase 3 trial of BBP-418 for LGMD2I, including the ongoing enrollment in the United States, the expectation to enroll in clinical trial sites planned in 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 expectation and timing of full enrollment and sharing topline data from CALIBRATE; the Company’s financial performance, capitalization status, strategy, business plans and goals reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our 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 our preclinical studies and clinical trials not being indicative of final data, the potential size of the target patient populations our 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 our product candidates, the FDA or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, the Company’s ability to obtain additional funding, including through less dilutive sources of capital than equity financings, potential volatility in our share price, uncertainty regarding any impacts due to global health emergencies such as COVID-19, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, 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 our most recent Annual Report on Form 10-K and our 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 our 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

– Fully enrolled CANYON and now enrolling the global pivotal cohort, GRAND CANYON, of EDG-5506 in Becker muscular dystrophy (Becker) – – Expanding Phase 2 LYNX trial of EDG-5506 including new cohort for boys with Duchenne not currently treated with corticosteroids – – Initiated new Phase 2 FOX trial in boys with Duchenne muscular dystrophy (Duchenne) who have been previously treated with gene therapy – – Advancing Phase 1 trial of EDG-7500, a novel sarcomere modulator for hypertrophic cardiomyopathy (HCM), and other serious diseases of cardiac diastolic dysfunction – BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2023 and recent business highlights. “I’m thrilled with the progress we’ve made in 2023 across our skeletal and cardiovascular programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “Most recently, we started enrolling individuals in two important studies: GRAND CANYON, a global pivotal study of EDG-5506 in Becker, a disorder with no approved treatments and a Phase 1 trial of EDG-7500, our novel sarcomere modulator for HCM. The team also initiated FOX, the Phase 2 trial of EDG-5506 in boys with Duchenne who have received gene therapy and continues to rapidly enroll the Phase 2 LYNX trial in Duchenne.” Recent Highlights Musculoskeletal Program / EDG-5506 Initiated GRAND CANYON, a Global Pivotal Study in Becker EDG-5506 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. The Company initiated enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion of the CANYON placebo-controlled study. CANYON, which was over-enrolled, includes 40 adults and 29 adolescents and a treatment period of 12 months. GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. Data from GRAND CANYON, if positive, could support a marketing application. The primary endpoint of GRAND CANYON is North Star Ambulatory Assessment (NSAA). In addition, other functional assessments, biomarkers of muscle damage and safety will be assessed. GRAND CANYON is anticipated to recruit approximately 120 individuals with Becker, aged between 18 and 50 years old, at up to 50 sites in 10 countries. The treatment period for participants will be 18 months. To learn more, go to clinicaltrials.gov (NCT05291091) or the GRAND CANYON microsite: . Rapidly enrolling and continuing dose escalation of Phase 2 LYNX trial Based on the safety pro to date, the Company is continuing dose escalation and expanding enrollment in their Phase 2 placebo-controlled LYNX trial of EDG-5506 in children aged 4 to 9 years with Duchenne; one of the new LYNX cohorts will include boys with Duchenne not currently treated with corticosteroids. The LYNX trial is enrolling at 14 sites across the United States, with the first three cohorts having over-enrolled. LYNX is a Phase 2 placebo-controlled trial to assess the effect of multiple doses of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage. The trial will also explore changes in functional measures, such as the North Star Ambulatory Assessment (NSAA) and self-reported/caregiver-reported outcomes. Approximately 45 children with Duchenne aged 4 to 9 years are expected to be enrolled in this trial. Participants will then continue in an open-label extension portion of the trial for a total of 24 months to gain further insights into safety and functional measures. Importantly, this trial is designed to identify a dose of EDG-5506 that will reduce biomarkers of muscle damage and has the potential to provide functional benefit to patients in a Phase 3 trial. The Company expects to report Phase 2 interim data in the first half of 2024, once the Phase 3 dose is identified. Go to clinicaltrials.gov to learn more about this trial (NCT05540860). Initiated new Phase 2 FOX trial in boys with Duchenne who have been previously treated with gene therapy FOX is a Phase 2 placebo-controlled trial to assess the effect of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage in children and adolescents with Duchenne who have been previously treated with gene therapy. Approximately 24 participants, aged 6 to 14 years, are expected to be enrolled in the trial at multiple sites across the United States, which is expected to begin dosing in early 2024. Participants will then continue in an open-label extension portion of the trial for a total of 12 months to gain further insights into safety, PK, function and biomarker measures. Go to clinicaltrials.gov to learn more about this trial (NCT06100887). Advancing Phase 2 DUNE Trial in Adults with LGMD2I/R9, Becker and McArdle Disease The Company is advancing the DUNE Phase 2 exercise challenge trial to evaluate the effect of EDG-5506 on biomarkers of muscle damage following controlled exercise in adults with LGMD2I/R9, Becker or McArdle disease at a single site in Denmark. Participants in the placebo-controlled 16-week trial then continue to an open label extension through 52 weeks. The goal of this trial is to assess safety and efficacy in individuals with myopathy distinct from Duchenne/Becker where muscle contraction is associated with exaggerated injury. LGMD2I/R9 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 Company expects to announce placebo-controlled data in the first half of 2024. Initiated Phase 2 Open-Label Extension MESA Trial in Adults and Adolescents with Becker The Company initiated MESA, an open-label treatment extension trial to assess the long-term effect of EDG-5506 on safety, biomarkers, and functional measures. MESA will provide continued access to EDG-5506 treatment to study participants who were previously enrolled in Edgewise studies. Go to clinicaltrials.gov to learn more about this trial (NCT06066580). Cardiovascular Program / EDG-7500 Advancing Phase 1 Trial of EDG-7500, a First-In-Class Sarcomere Modulator EDG-7500 is a first-in-class oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. The Company is enrolling a randomized, double-blind, placebo-controlled, single and multiple ascending dose Phase 1 trial evaluating safety, tolerability, pharmacokinetics and pharmacodynamics in healthy adults. To learn more about this trial (NCT06011317), go to clinicaltrials.gov. The Company is also planning to begin a Phase 1b trial of EDG-7500 in individuals with obstructive HCM in the first half of 2024. Strengthened Engagement with the Scientific and Patient Communities Musculoskeletal Program / EDG-5506 The Company continued its education and outreach on its Becker program with the medical and patient communities. In October 2023, the Company held an Industry Symposium at the Annual Congress of the World Muscle Society (WMS). Additionally, the Company held webinars with clinicians hosted by Muscular Dystrophy Association in August 2023 and the patient community hosted by Parent Project Muscular Dystrophy in October 2023. Replays of the webinars can be viewed here. 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. In October 2023, the Company shared information on its Phase 1 trial of EDG-7500, at the Heart Failure Society of America Annual Scientific Meeting, participated in a panel discussion on emerging pharmacologic therapies for HCM at the HCM Society Annual Scientific Sessions and attended the HCM Association Annual Patient Meeting. The Company is also presenting on EDG-7500 at the American Heart Association Scientific Sessions on November 13. The presentations are available on the Edgewise website. Third Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $290 million as of September 30, 2023. Research and development (R&D) expenses were $23.8 million for the third quarter of 2023, compared to $19.6 million for the immediately preceding quarter. The increase of $4.2 million was primarily driven by an increase of $3.3 million in clinical trial expenses for both the EDG-5506 and EDG-7500 clinical programs in addition to $0.6 million of higher personnel-related costs and $0.3 million of other R&D expenses. General and Administrative (G&A) expenses were $5.7 million for the third quarter of 2023, compared to $5.8 million for the immediately preceding quarter. Net loss and net loss per share for the third quarter of 2023 was $25.7 million or $0.41 per share, compared to $21.5 million or $0.34 per share for the immediately preceding quarter. About EDG-5506 for Becker and Duchenne Muscular Dystrophies EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Duchenne and Becker. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle damage that leads to functional impairment, EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders Its unique mechanism of action provides the potential to establish EDG-5506 as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. In August 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to EDG-5506 for the treatment of individuals with Becker. The Company has advanced EDG-5506 through the clinic including completing a Phase 1 trial evaluating safety, tolerability, PK and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with Becker (Phase 1b) (NCT04585464). In ARCH, an open-label, single-center trial (NCT05160415) assessing long-term safety and PK, decreases in biomarkers of muscle damage and trends toward improvement in NSAA have been observed following 12 months of treatment with EDG-5506. A Phase 2 cohort of EDG-5506 in Becker (CANYON) is fully enrolled and has now been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON, which is currently enrolling (NCT05291091). In Duchenne, the Company is advancing its Phase 2 studies, LYNX (NCT05540860) and FOX (NCT06100887), both assessing safety, PK and biomarkers of muscle damage. The Company is also advancing the DUNE Phase 2 exercise challenge trial, to evaluate the effect of EDG-5506 on biomarkers of muscle damage following exercise in adults with LGMD2I, Becker or McArdle disease at a single site in Denmark. About EDG-7500 for Hypertrophic Cardiomyopathy EDG-7500 is a first-in-class oral, selective, cardiac sarcomere modulator that is advancing in a Phase 1 trial (NCT06011317). The compound is designed to slow early contraction velocity and improve impaired cardiac relaxation. This novel mechanism is anticipated to have a broad therapeutic index that may facilitate fixed patient dosing and minimize monitoring during therapy. Preclinical data of EDG-7500 support beneficial activity in animal models of both obstructive HCM and cardiac diastolic dysfunction with minimal changes in left ventricular systolic performance. 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 first-in-class therapeutics. EDG-5506 is an orally administered skeletal myosin inhibitor in clinical trials in patients with Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel cardiac sarcomere modulator for the treatment of HCM and other disorders of cardiac diastolic dysfunction. 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. 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 EDG-5506 and EDG-7500; statements regarding Edgewise’s expectations relating to its clinical trials, including timing of reporting data (including the Phase 2 interim data for the LYNX Phase 2 clinical trial of EDG-5506 and Phase 2 DUNE Trial of EDG-5506) and commencement of trials (including patient enrollment for GRAND CANYON, certain milestones of the Phase 2 FOX trial in boys with Duchenne and a Phase 1b trial of EDG-7500); and statements regarding Edgewise’s pipeline of product candidates and programs. 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; the timing, progress and results of clinical trials for EDG-5506 and the 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; 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 September 30, 2023 June 30, 2023 Operating expenses: Research and development $ 23,786 $ 19,559 General and administrative 5,666 5,780 Total operating expenses 29,452 25,339 Loss from operations (29,452 ) (25,339 ) Interest income 3,739 3,870 Net loss $ (25,713 ) $ (21,469 ) Net loss per share - basic and diluted $ (0.41 ) $ (0.34 ) Weighted-average shares outstanding, basic and diluted 63,459,560 63,380,430 Edgewise Therapeutics, Inc. Condensed Balance Sheet Data (in thousands, unaudited) September 30, December 31, 2023 2022 Assets Cash, cash equivalents and marketable securities $ 289,697 $ 351,947 Other assets 21,667 15,154 Total assets $ 311,364 $ 367,101 Liabilities and stockholders' equity Liabilities 20,937 20,385 Stockholders' equity 290,427 346,716 Total liabilities and stockholders' equity $ 311,364 $ 367,101

ATA-100 is being evaluated in a multi-center phase 1b/2b “ATA-001-FKRP” study Completion of enrollment of the first dose cohort of 3 patients First results of cohort one presented at ESGCT in oral presentation Good clinical and biological safety/tolerability in the first cohort Preliminary efficacy documented with biomarkers and functional assessments DSMB clearance to proceed with enrollment of the 2nd cohort with a 3-times higher dose EVRY, France--(BUSINESS WIRE)-- Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced the presentation of the first clinical results obtained with ATA-100 in the on-going ATA-001 Phase 1b/2b clinical trial. ATA-100 is a one-time gene therapy for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy Type 2I/R9 (LGMD2I/R9). Atamyo also announced today that Data Safety Monitoring Board (DSMB) authorized the enrollment of the second dose cohort of the ATA-001 clinical trial. ATA-100 is being evaluated in a multi-center phase 1b/2b in Denmark, France, and the United Kingdom, and has received IND clearance by FDA. Preliminary results in the first cohort show: No unexpected safety signal identified Marked decline in levels of creatine kinase in all three patients Improved velocity, sustained at one year Disappearance of symptoms (cramps, myalgia) and improved quality of life Correction of centronucleation and evidence of transgene expression on the 3-month muscle biopsy Pr. John Vissing, principal investigator of the study, noted that “the preliminary results from cohort 1 with the first dose tested already show encouraging results from a safety and efficacy perspective. ATA-100 treatment has a life-changing potential in an indication where there is no approved treatment.” These first results will be presented during oral communications in two forthcoming conferences: 1) The 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) that will take place on October 24-27, 2023 in Brussels, (Belgium): “Gene therapy for LGMDR9: preliminary results of a dose-escalation study” • Oral presentation by Nicolai Preisler, MD, Rigshospitalet, Copenhagen, during the Cardiovascular and muscular diseases session, on Friday, October 27, 11h30-13h00 – Le BEL, Tour & Taxis 2) The 2023 International Limb-Girdle Muscular Dystrophy Conference, which will take place on 28-29 October 2023, Washington, DC. “Preliminary experiences with Atamyo’s FKRP gene therapy” • Oral presentation by John Vissing, MD, Rigshospitalet, Copenhagen, during the Clinical Trials Updates (Part 1) Session, on October 29, 11h00am-12h10pm – Constitution Ballroom, Grand Hyatt “These first results, in particular those related to transgene expression in muscle fibers are really exciting,” said Dr. Sophie Olivier, Atamyo’s Chief Medical Officer. “With the DSMB clearance to start the 2nd cohort, we are looking forward to enrolling new patients in Europe and in the US.” LGMD2I/R9 is a rare genetic disease caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the US and Europe. In the most common form, symptoms appear around late childhood or early adulthood. Patients suffer from progressive muscular weakness leading to loss of ambulation. They are also prone to respiratory impairment. There are currently no curative treatments for LGMD2I/R9. ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins. The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon. About Atamyo Therapeutics Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for muscular dystrophies and cardiomyopathies. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with one clinical-stage program targeting LGMD-R9. Atamyo is committed to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit . View source version on businesswire.com: Contacts U.S. Contact: Charles Craig, Opus Biotech Communications charles.s.craig@gmail.com, 404-245-0591 European contact: contact@atamyo.com Source: Atamyo Therapeutics View this news release online at: