Since my August article on Edgewise Therapeutics (NASDAQ: EWTX ), the stock nearly doubled at one point after producing strong midstage data from a program in which it has an ongoing late stage study. This program is targeting BeckerAbout the TPT serviceThanks for reading. At the Total Pharma Tracker, we offer the following:-Our Android app and website features a set of tools for DIY investors, including a work-in-progress software where you can enter any ticker and get extensive curated research material.  ...

Financial Performance - Net losses for Edgewise Therapeutics were $133.8 million in 2024, up from $100.2 million in 2023, reflecting an increase of $33.6 million [655]. - The net loss for 2024 was $133.8 million, compared to a net loss of $100.2 million in 2023, indicating a 33% increase in losses [734]. - The total comprehensive loss for 2024 was $133.5 million, compared to $98.7 million in 2023, representing a 35% increase [734]. - The Company reported a net loss of $133.8 million for the year ended December 31, 2024, compared to a net loss of $100.2 million for 2023, representing a 33.5% increase in net loss year-over-year [816]. Research and Development Expenses - Research and development expenses rose to $127.0 million in 2024 from $90.9 million in 2023, an increase of $36.1 million [672]. - Research and development expenses for the year ended December 31, 2024, were $127.0 million, up 40% from $90.9 million in 2023 [734]. - The sevasemten clinical program incurred $51.7 million in expenses in 2024, up from $34.8 million in 2023, a rise of $16.8 million [673]. - EDG-7500 clinical program expenses increased by $8.9 million to $17.9 million in 2024, primarily due to the activation of the Phase 2 CIRRUS-HCM trial [673]. - Research and development expenses increased to $80.2 million in 2024 from $57.5 million in 2023, reflecting a 39.4% increase [824]. - Accrued research and development costs were $6.5 million as of December 31, 2024, compared to $5.7 million in 2023, indicating an increase of 14.4% [820]. Cash Flow and Liquidity - As of December 31, 2024, Edgewise Therapeutics had cash, cash equivalents, and marketable securities totaling $470.2 million [679]. - Cash used in investing activities in 2024 amounted to $184.7 million, primarily due to $477.1 million in purchases of marketable securities, offset by $277.9 million in maturities of marketable securities [685]. - Cash provided by financing activities in 2024 was $249.3 million, significantly higher than $53.2 million in 2023, driven by $239.1 million in net proceeds from the issuance of common stock [687][688]. - Cash flows from operating activities resulted in a net cash used of $109.0 million in 2024, up from $91.9 million in 2023, indicating an 18.5% increase in cash outflow [739]. - Cash and cash equivalents decreased to $41.7 million in 2024 from $86.1 million in 2023, a decline of 52% [733]. - The company believes its cash and marketable securities will be sufficient to fund operations for at least the next 12 months [749]. Capital and Financing - The company expects significant additional spending to progress its product candidates through clinical trials and regulatory approval [660]. - The company expects to require substantial additional capital to develop product candidates and fund operations, with a shelf registration statement filed for equity and debt offerings of up to $175 million [689]. - The company raised $240.0 million in gross proceeds from an underwritten registered direct offering of 21,818,182 shares at $11.00 per share in January 2024 [745]. - The company has entered into a sales agreement allowing for the potential sale of up to $175.0 million in common stock through an at-the-market offering program [746]. Operational Expenses - General and administrative expenses increased to $31.9 million in 2024 from $23.5 million in 2023, an increase of $8.4 million [677]. - The company reported total operating expenses of $158.8 million for 2024, a 39% increase from $114.4 million in 2023 [734]. - Non-cash charges in operating activities for 2024 included stock-based compensation expense of $24.7 million and depreciation of $2.1 million [683]. Shareholder Equity and Stock Options - The weighted-average shares outstanding increased to 92.4 million in 2024 from 63.7 million in 2023, reflecting a 45% increase [734]. - Additional paid-in capital rose to $837.4 million in 2024 from $563.5 million in 2023, marking a 48% increase [733]. - The company has 15,416,761 stock options outstanding with a weighted average exercise price of $9.40 and total intrinsic value of $267.4 million [787]. - The total stock-based compensation expense for the year ended December 31, 2024 was $24.7 million, an increase from $17.6 million in 2023 [800]. - The company granted 2,817,497 stock options in 2024 at a weighted average exercise price of $17.67 [787]. Compliance and Regulatory - The company ceased to be an "emerging growth company" and "smaller reporting company" as of December 31, 2024, which is expected to increase public company compliance costs [711][712]. - The company maintained effective internal control over financial reporting as of December 31, 2024, according to the auditor's opinion [719]. Marketable Securities - As of December 31, 2024, the total fair value of marketable securities was $469,978,000, with unrealized gains of $530,000 and unrealized losses of $110,000 [768]. - The remaining contractual maturities of $394.4 million of marketable securities were less than one year, and $33.7 million were between 1 and 2 years as of December 31, 2024 [769]. - The company has not recognized any impairment charges for long-lived assets during the years ended December 31, 2024, and 2023 [762].

Financial Performance - Edgewise Therapeutics reported a net loss of $39.7 million or $0.42 per share for Q4 2024, compared to a net loss of $34.1 million or $0.36 per share in the previous quarter[18]. - Total operating expenses for Q4 2024 were $45.5 million, compared to $40.4 million in the previous quarter[23]. - Cash, cash equivalents, and marketable securities increased to approximately $470.2 million as of December 31, 2024, up from $318.4 million in the previous year[16][25]. Research and Development - Research and development expenses for Q4 2024 were $36.4 million, an increase from $32.2 million in Q3 2024, driven by higher personnel costs and clinical development activities[17]. - The GRAND CANYON study completed enrollment with 175 adults across 51 sites in 12 countries, with data expected in Q4 2026[6]. - Positive top-line results were announced from the Phase 2 CANYON trial, which evaluated 40 adults and 29 adolescents over a 12-month treatment period[5]. - The CIRRUS-HCM trial is on track to report data in Q1 2025, following positive topline results from Part A of the trial[12][13]. - The LYNX and FOX trials for Duchenne muscular dystrophy are ongoing, with data expected in the first half of 2025 to inform Phase 3 strategy[10]. - Edgewise Therapeutics aims to file an investigational new drug application for a novel heart failure candidate in 2025[14]. Regulatory Engagement - The company plans to engage the U.S. FDA in the first half of 2025 to discuss marketing authorization strategies for sevasemten in Becker muscular dystrophy[5].

Core Viewpoint - Edgewise Therapeutics (EWTX) shares surged over 18% following positive mid-stage results from the CANYON study for its drug sevasemten, aimed at treating Becker Muscular Dystrophy (BMD) [1][5]. Group 1: Study Results - The CANYON study achieved its primary endpoint, showing a significant 28% reduction in creatine kinase (CK) levels in the sevasemten-treated group compared to placebo over six to twelve months [2]. - Sevasemten treatment indicated a trend towards improvement in the North Star Ambulatory Assessment, a key secondary endpoint, and showed a significant reduction in plasma levels of fast skeletal muscle troponin I (TNNI2) [3]. - The sevasemten-treated group had patients with more advanced disease compared to the placebo group, suggesting the drug's efficacy in a challenging patient population [3]. Group 2: Future Plans and Pipeline - Edgewise is on track to complete recruitment for the pivotal GRAND CANYON cohort by the first quarter of 2025, with plans to seek FDA and EMA approval for sevasemten if results are positive [4]. - Besides BMD, sevasemten is being evaluated for other muscular disorders, including Duchenne muscular dystrophy (DMD), Limb-Girdle muscular dystrophy (LGMD), and McArdle Disease [8]. - Edgewise is also developing another candidate, EDG-7500, for obstructive hypertrophic cardiomyopathy (HCM), with initial data expected in the first quarter of 2025 [9][11]. Group 3: Stock Performance - EWTX shares have increased nearly 200% year to date, contrasting with an 11.4% decline in the industry, reflecting investor confidence in the CANYON study results and the potential of sevasemten [6].

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].