Core Insights - Edgewise Therapeutics, Inc. announced positive topline results from the Phase 2 CANYON trial of sevasemten for Becker muscular dystrophy (BMD), marking it as the largest interventional trial in this area and the first to meet its primary endpoint [1][2] Group 1: Trial Results - The trial met its primary endpoint with a significant 28% average decrease in creatine kinase (CK) levels in the sevasemten-treated group compared to placebo over months 6 through 12 (p=0.02) [2] - Secondary endpoint, North Star Ambulatory Assessment (NSAA), indicated a trend towards improvement in motor function in the sevasemten-treated group [2] - Plasma fast skeletal muscle troponin I (TNNI2) showed a significant reduction in the treatment group compared to placebo, indicating muscle damage reduction [3] Group 2: Functional Measures - Additional functional measures, including the 10-meter walk/run, 4-stair climb, and 100-meter timed test, also showed trends towards improvement in the sevasemten group compared to placebo [3] Group 3: Patient Population and Safety - The treatment population had more advanced disease compared to the placebo group, suggesting a challenging patient demographic [4] - Sevasemten was well-tolerated with no new safety concerns reported during the trial [5] Group 4: Future Plans and Market Reaction - The company is on track to complete recruitment for the GRAND CANYON cohort by the first quarter of 2025 [6] - Following the announcement, EWTX stock rose by 8.62% to $30 [7]

Financial Performance - Net losses for the three months ended September 30, 2024, were $34.1 million, compared to $25.7 million for the same period in 2023, representing an increase of $8.4 million[90]. - Total operating expenses for the nine months ended September 30, 2024, were $113.3 million, compared to $80.5 million for the same period in 2023, an increase of $32.8 million[96]. - Net cash used in operating activities was $82.1 million for the nine months ended September 30, 2024, compared to $65.1 million in 2023[105]. - Cash used in investing activities amounted to $205.6 million in 2024, primarily due to $413.9 million in purchases of marketable securities[108]. - Cash provided by financing activities was $246.4 million in 2024, significantly higher than $0.2 million in 2023[110]. Research and Development - Research and development expenses for the three months ended September 30, 2024, were $32.2 million, up from $23.8 million in 2023, reflecting an increase of $8.4 million driven by higher clinical trial activity[93]. - Research and development expenses increased to $90.6 million for the nine months ended September 30, 2024, up from $63.2 million in 2023, representing a $27.4 million increase[99]. - The increase in R&D expenses was primarily driven by a $14.7 million rise in sevasemten clinical program expenses and a $9.2 million increase in EDG-7500 clinical program expenses[99]. - Significant additional spending is expected to progress product candidates through clinical development phases, particularly for sevasemten and EDG-7500[84]. - The company is advancing two clinical-stage programs: Sevasemten (EDG-5506) for Becker muscular dystrophy and EDG-7500 for hypertrophic cardiomyopathy, with multiple Phase 2 trials ongoing[75]. Cash and Liquidity - The company had cash and cash equivalents of $492.5 million as of September 30, 2024, which is expected to fund operations for at least the next 12 months[79]. - As of September 30, 2024, the company had cash, cash equivalents, and marketable securities totaling $492.5 million[103]. - The company expects to require substantial additional capital to develop product candidates and fund operations in the foreseeable future[112]. Expenses - General and administrative expenses increased to $8.2 million for the three months ended September 30, 2024, from $5.7 million in 2023, an increase of $2.5 million primarily due to higher personnel-related costs[94]. - General and administrative expenses rose to $22.7 million in 2024 from $17.3 million in 2023, an increase of $5.4 million[100]. Interest Income - Interest income rose to $6.3 million for the three months ended September 30, 2024, compared to $3.7 million in 2023, an increase of $2.6 million attributed to higher balances of marketable securities[95]. - Interest income grew to $19.1 million for the nine months ended September 30, 2024, compared to $10.5 million in 2023, marking an increase of $8.7 million[101]. Market and Economic Conditions - The company is monitoring macroeconomic and geopolitical developments that could impact operations, including inflation and credit market conditions[80]. Company Status and Future Outlook - The accumulated deficit as of September 30, 2024, was $338.9 million, with total gross proceeds from private placements and public offerings amounting to $767.3 million since inception[79]. - The total operating lease liability balance as of September 30, 2024, was $4.4 million, with $1.0 million classified as a current liability[122]. - The company will remain an emerging growth company until December 31, 2024[127]. - As of June 30, 2024, the market value of the company's common stock held by non-affiliates exceeds $700.0 million[127]. - The company will transition to a "large accelerated filer" status effective January 1, 2025[127]. - The company has elected to take advantage of an extended transition period for adopting new or revised accounting standards[126].

Financial Performance - The net loss for Q3 2024 was $34.1 million, or $0.36 per share, compared to a net loss of $31.5 million, or $0.34 per share, in the previous quarter[17]. - Research and development (R&D) expenses for Q3 2024 were $32.2 million, an increase from $30.7 million in the previous quarter, primarily due to higher manufacturing expenses[16]. - General and administrative (G&A) expenses rose to $8.2 million in Q3 2024, compared to $7.4 million in the preceding quarter, driven by increased personnel-related costs[17]. Cash and Assets - As of September 30, 2024, cash, cash equivalents, and marketable securities totaled approximately $492.5 million[15]. - Cash, cash equivalents, and marketable securities rose to $492,536 thousand, an increase of 54.6% from $318,393 thousand at the end of 2023[22]. - Total assets increased to $511,282 thousand as of September 30, 2024, up from $340,035 thousand on December 31, 2023, representing a growth of 50.4%[22]. - Stockholders' equity increased to $488,877 thousand, up 53.3% from $318,830 thousand at the end of 2023[22]. - Total liabilities increased slightly to $22,405 thousand from $21,205 thousand, reflecting a growth of 5.6%[22]. Clinical Trials - The CANYON Phase 2 trial for sevasemten in adults with Becker is set to report data in December 2024, involving 40 adults and 29 adolescents over a 12-month treatment period[4]. - The GRAND CANYON trial, an expansion of the CANYON trial, will evaluate the safety and efficacy of sevasemten in 120 adults over 18 months, with data potentially supporting a marketing application[5]. - The LYNX Phase 2 trial in boys with Duchenne is planned to report data in Q4 2024, guiding the design of a Phase 3 trial expected to start in 2025[8]. - In the Phase 1 trial of EDG-7500, no clinically meaningful changes in vital signs or LVEF were observed across a range of doses, indicating good tolerability[11]. - The CIRRUS-HCM trial reported a 67% mean reduction in resting left ventricular outflow tract pressure gradient with a single dose of EDG-7500, without significant changes in LVEF[12]. Community Engagement - The company continues to engage with the scientific and patient communities, participating in major events for Becker and muscular dystrophy awareness[14].

Company Overview - Edgewise Therapeutics (EWTX) is a small-cap pharmaceutical company with a market cap of $2.4 billion, experiencing a 55% increase in shares over the last three months and a 332% increase over the past 52 weeks [1] Drug Pipeline - The company is focusing on two main drugs: Sevasemten for muscular dystrophy (MD) and EDG-7500 for hypertrophic cardiomyopathy (HCM) [2][6] - Sevasemten is in Phase 3 trials for Becker MD and Phase 2 trials for Duchenne MD, differentiating itself by targeting multiple types of MD rather than just one [3] - EDG-7500 has shown promising results, with a 64% decrease in a biomarker linked to heart stress and maintaining normal left ventricular ejection fraction (LVEF) levels in patients [7][11] Market Potential - The potential market for Sevasemten includes approximately 47,000 patients with Becker and Duchenne MD in the US, EU, and Japan combined [5] - The market for EDG-7500 is significantly larger, affecting up to 630,000 people in the United States alone [7] Competitive Landscape - EDG-7500 competes with Bristol Myers Squibb's Camzyos, which has shown a 67% reduction in the same biomarker but has had some patients with decreased LVEF levels [8] - Analysts note that both drugs appear to have similar safety and efficacy profiles, with Edgewise's EDG-7500 showing comparable results to existing treatments [9][11] Analyst Sentiment - Several Wall Street analysts have increased their price targets for Edgewise, with an average implied upside of 55% based on recent Phase 2 results for EDG-7500 [10] - The most optimistic analyst projects a potential rise of 70% for Edgewise shares [10]

Core Viewpoint - Edgewise Therapeutics has seen significant stock price increases, with shares up 55% in the last three months and 332% over the past year, raising questions about potential investment opportunities given its current market cap of $2.4 billion [1][2]. Drug Pipeline - The company is focusing on two main drugs: Sevasemten for muscular dystrophy (MD) and EDG-7500 for hypertrophic cardiomyopathy (HCM) [1][4]. - Sevasemten is in Phase 3 trials for Becker MD and Phase 2 trials for Duchenne MD, differentiating itself by targeting multiple forms of MD [2][3]. - EDG-7500 has shown promising results, with a 64% reduction in a biomarker linked to heart stress and maintaining normal left ventricular ejection fraction (LVEF) levels in patients [5][6]. Market Potential - The market for Sevasemten is significant, with an estimated 47,000 people affected by Becker and Duchenne MD in the US, EU, and Japan combined [4]. - The HCM market is even larger, potentially affecting up to 630,000 people in the United States alone [5]. Competitive Landscape - EDG-7500 competes with Bristol Myers Squibb's Camzyos, which has shown a 67% reduction in the same biomarker but has had some patients experience decreased LVEF levels [6][7]. - Analysts note that the results of EDG-7500 and Camzyos appear similar, indicating a competitive market [7]. Analyst Sentiment - Several analysts have raised their price targets for Edgewise, with an average implied upside of 55% based on recent closing prices, and the most optimistic analyst projecting a 70% increase [8][9]. - Despite the positive outlook, the probability of drug approval remains low, averaging around 12% [9][10].

Core Viewpoint - Edgewise Therapeutics has seen a significant increase in its stock price due to promising early trial results for its oral treatment for obstructive hypertrophic cardiomyopathy (HCM), a condition that is a leading cause of sudden cardiac death in individuals under 35 [1][2]. Company Overview - Edgewise Therapeutics is focused on developing a novel treatment for HCM, a genetic disease that causes thickening of the heart muscle, within a market valued at over $1.25 billion and growing [2]. - The company has a small but attractive pipeline targeting conditions such as Duchenne and Becker muscular dystrophy, with positive early-stage trial results expected to be announced in Q4 [8]. Stock Performance - The stock price of Edgewise Therapeutics has increased by 50% in a single session and 450% from recent lows, currently trading at $28.18, with a 52-week range of $5.12 to $30.00 [1][3]. - Analysts have revised price targets upward, with Wedbush raising its target by 42% to $44, indicating a potential 70% upside within the next twelve months [5]. Market Sentiment - Institutional ownership is strong, with institutions, private equity, and venture capital holding about 90% of the stock, reflecting high conviction in the company's prospects [6]. - There has been a notable increase in sell-side interest, leading to a positive shift in price targets and market sentiment [4]. Volatility and Risks - The stock has experienced a short-squeeze, with short interest reported at nearly 15%, which may lead to volatility in the near term as short-sellers adjust their positions [7]. - The company is currently not generating revenue and is operating at a loss, but it has over half a billion in cash, sufficient to sustain operations for over 15 quarters [8]. Technical Analysis - The price chart indicates a recovery from previous lows, supported by high trading volume, suggesting a high probability of continued upward movement, although resistance may be encountered near all-time highs [9].

Core Viewpoint - Edgewise Therapeutics (EWTX) shares surged 30% in pre-market trading following positive top-line data for EDG-7500 from early-stage studies in healthy subjects and mid-stage studies in patients with obstructive hypertrophic cardiomyopathy (HCM) [1] Group 1: Clinical Data and Efficacy - EDG-7500 demonstrated no significant changes in left ventricle ejection fraction (LVEF) in phase I study participants, indicating a favorable safety profile [2][3] - In Part A of the phase II CIRRUS-HCM study, EDG-7500 treatment resulted in a 67% mean reduction in resting left ventricular outflow tract (LVOT) pressure gradient and a 55% mean reduction in provokable LVOT pressure gradient, without affecting LVEF [4] - The 200 mg cohort also showed a 64% mean reduction in NT-proBNP, a key heart failure biomarker, highlighting EDG-7500's potential for treating diastolic dysfunction diseases [4] Group 2: Development Plans - Edgewise has initiated dosing for the first patients with obstructive and non-obstructive HCM in Part B of the phase II CIRRUS-HCM study, addressing a significant unmet medical need [5] - The study will assess tolerability, pharmacokinetics, and effects on LVOT pressure gradient, LVEF, biomarkers, and patient function, with initial data expected in Q1 2025 [6] Group 3: Other Candidates - In addition to EDG-7500, Edgewise is evaluating its lead candidate, sevasemten, for Becker and Duchenne muscular dystrophies, which is a first-in-class fast myosin inhibitor [7]

Core Viewpoint - Edgewise Therapeutics' heart drug EDG-7500 shows promising results in clinical trials, leading to a significant increase in the company's stock price, but the path to commercialization remains long and uncertain [1][2][5]. Group 1: Clinical Trial Results - The phase 1 testing of EDG-7500 for obstructive hypertrophic cardiomyopathy (HCM) demonstrated that the drug was well tolerated and did not lower left ventricular ejection fraction levels, indicating it does not cause inefficient blood flow [2][3]. - The phase 2 CIRRUS-HCM trial also confirmed the drug's efficacy and tolerability, suggesting that higher doses may help prevent other forms of heart failure in select patients without significant drops in left ventricular ejection fraction levels [3]. Group 2: Market and Financial Outlook - Despite the positive trial results, Edgewise Therapeutics remains unprofitable and is currently selling stock to fund operations, with no revenue generation and no products beyond phase 2 testing [5]. - The FDA approval process for new drugs requires three full phases of clinical testing, which can take years, and the company is still in the early stages of this process [5]. - Edgewise Therapeutics is positioned in underserved markets such as muscular dystrophy and hypertrophic cardiomyopathy, which could provide long-term competitive advantages, but the market may not be patient for immediate returns [6].

On Thursday, Edgewise Therapeutics Inc. EWTX released announced topline data of EDG-7500 from the Phase 1 trial in healthy subjects and the single-dose arm of the Phase 2 CIRRUS-HCM trial for obstructive hypertrophic cardiomyopathy. Obstructive hypertrophic cardiomyopathy is a heart condition that occurs when the wall between the left and right ventricles of the heart thickens, blocking blood flow from the left ventricle to the body's main artery. In the Phase 1 single ascending dose (SAD) trial (n=48), hea ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-40236 Edgewise Therapeutics, Inc. (Exact name of registrant as specified in its charter) (State or other juri ...