EWTX Edgewise Therapeutics Inc.

17.51
-2  -10%
Previous Close 19.51
Open 19.47
52 Week Low 16.9501
52 Week High 40.49
Market Cap $862,442,986
Shares 49,254,311
Float 19,489,349
Enterprise Value $651,080,607
Volume 64,482
Av. Daily Volume 229,740
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Latest News

  1. Edgewise Therapeutics, Inc. (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the company will present at the 2021 PPMD Annual Conference and the 2021 New Directions in Biology and Disease of Skeletal Muscle Conference.

    2021 PPMD Annual Conference
    Presentation:
    An Introduction to Edgewise Therapeutics
    Presenter: Alan Russell, Ph.D., Chief Scientific Officer, Edgewise Therapeutics

    Presentation: Development of a Novel Therapeutic for Duchenne, Becker and other Muscular Dystrophies
    Date: Wednesday, June 23, 2021 at 6:40 pm ET
    Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

    2021 New

    Edgewise Therapeutics, Inc. (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that the company will present at the 2021 PPMD Annual Conference and the 2021 New Directions in Biology and Disease of Skeletal Muscle Conference.

    2021 PPMD Annual Conference

    Presentation:
    An Introduction to Edgewise Therapeutics

    Presenter: Alan Russell, Ph.D., Chief Scientific Officer, Edgewise Therapeutics

    Presentation: Development of a Novel Therapeutic for Duchenne, Becker and other Muscular Dystrophies

    Date: Wednesday, June 23, 2021 at 6:40 pm ET

    Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

    2021 New Directions in Biology and Disease of Skeletal Muscle Conference

    Presentation:
    Selective Inhibition of Fast Skeletal Muscle Myosin as a Novel Therapeutic Strategy for Muscular Dystrophy

    Date: Monday, June 28, 2021 at 12:10 p.m. ET

    Presenter: Alan Russell, Ph.D., Chief Scientific Officer, Edgewise Therapeutics

    The presentation at the New Directions conference describes the potential of selective fast myosin inhibition to provide broad protection of muscle in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).

    The presentations will be available on the Edgewise website after they are presented.

    About Edgewise Therapeutics

    Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.

    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 drug discovery platform and product candidates including EDG-5506 and EDG-6289; statements about the expected timing and results from Edgewise's Phase 1 clinical trials for EDG-5506; statements regarding Edgewise's pipeline of product candidates and programs; and statements by Edgewise's 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 preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 and Edgewise's other product candidates in its EDG-6289, EDG-002 and EDG-003 programs; Edgewise's ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise's operations, including preclinical and clinical trials; the timing, scope and likelihood of regulatory filings and approvals; 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 Securities and Exchange Commission (the "SEC"). 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.

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  2. Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that members of its management team will participate in a fireside chat and one-on-one investor meetings at the virtual Goldman Sachs 42nd Annual Global Healthcare Conference on Wednesday, June 9, 2021 at 5:30 p.m. ET.

    The fireside chat will be webcast live on the Events and Presentations page under the investor relations section of Edgewise Therapeutic's website at https://investors.edgewisetx.com/events-and-presentations and accessible for 90 days following the presentation. It is recommended that users connect to the webcast several minutes prior…

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that members of its management team will participate in a fireside chat and one-on-one investor meetings at the virtual Goldman Sachs 42nd Annual Global Healthcare Conference on Wednesday, June 9, 2021 at 5:30 p.m. ET.

    The fireside chat will be webcast live on the Events and Presentations page under the investor relations section of Edgewise Therapeutic's website at https://investors.edgewisetx.com/events-and-presentations and accessible for 90 days following the presentation. It is recommended that users connect to the webcast several minutes prior to the start to ensure a timely connection.

    About Edgewise Therapeutics

    Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.

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  3.  – Completed successful $202.4 million Initial Public Offering (IPO); cash and marketable securities of $309.9 million as of March 31, 2021 –

    Initiated Phase 1 MAD study of EDG-5506, a small molecule drug candidate designed to arrest muscle fiber breakdown, the hallmark of ongoing disease in Duchenne and Becker muscular dystrophy (DMD and BMD) –

    Appointed Joanne Donovan, M.D., Ph.D., experienced rare disease physician, as Chief Medical Officer (CMO) –

    – Published first study to describe selective elevation of circulating biomarkers of fast but not slow skeletal muscle fiber injury in patients with DMD and BMD, in Muscle & Nerve, supporting EDG-5506's novel mechanism of action aimed at protecting injury-susceptible fast skeletal muscle

     – Completed successful $202.4 million Initial Public Offering (IPO); cash and marketable securities of $309.9 million as of March 31, 2021 –

    Initiated Phase 1 MAD study of EDG-5506, a small molecule drug candidate designed to arrest muscle fiber breakdown, the hallmark of ongoing disease in Duchenne and Becker muscular dystrophy (DMD and BMD) –

    Appointed Joanne Donovan, M.D., Ph.D., experienced rare disease physician, as Chief Medical Officer (CMO) –

    – Published first study to describe selective elevation of circulating biomarkers of fast but not slow skeletal muscle fiber injury in patients with DMD and BMD, in Muscle & Nerve, supporting EDG-5506's novel mechanism of action aimed at protecting injury-susceptible fast skeletal muscle fibers in DMD and BMD –

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today reported financial results for the first quarter 2021 and recent business highlights.

    "Our successful IPO was transformative for Edgewise, providing the financial stability to advance a meaningful pipeline of novel therapeutics for severe, rare muscle disorders," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "We continue to advance EDG-5506, our lead clinical candidate designed to protect injury-susceptible fast skeletal muscle fibers in DMD and BMD. We expect to have topline data from the Phase 1a MAD and Phase 1b BMD cohorts toward the latter half of 2021."

    Recent Highlights

    Completed Successful Initial Public Offering

    On March 30, 2021, Edgewise completed its IPO by selling 12,650,000 shares of its common stock, including the full exercise by the underwriters of their option to purchase 1,650,000 additional shares, at an IPO price of $16.00 per share, which was at the upper end of the offering range. Gross proceeds of the offering, before deducting the underwriting discount and commissions and other estimated offering expenses, were $202.4 million. The shares began trading on the Nasdaq Global Select Market on March 26, 2021 under the ticker symbol "EWTX."

    Continued to Advance EDG-5506 in Clinical Development

    Our Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and adults with BMD (Phase 1b). In March 2021, EDG-5506 advanced into the Multiple Ascending Dose (MAD) portion of the study, having successfully completed the Single Ascending Dose (SAD) portion of the study. The Company expects to report topline MAD data in healthy volunteers and BMD patient data later in 2021.

    Published Study in Muscle & Nerve Journal and Presented Data at MDA Clinical & Scientific Conference

    In March 2021, the first cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in the blood of patients with DMD and BMD was published in the journal Muscle & Nerve. The results build on previous reports and suggest that slow skeletal muscle fibers do not appear to leak muscle proteins associated with muscle injury and damage in DMD and BMD. The study further demonstrated that fast skeletal troponin I (TNNI2) may represent a more sensitive biomarker of muscle injury than creatine kinase (CK), particularly in the setting of BMD or older DMD patients where plasma CK is commonly lower than in young DMD patients. These data support EDG-5506's unique mechanism of action aimed at protecting injury-susceptible fast skeletal muscle fibers in DMD and BMD.

    These data were also presented in the scientific poster "Elevation of fast but not slow troponin I in the circulation of patients with Becker and Duchenne muscular dystrophy" at the MDA Clinical & Scientific Conference on March 16, 2021. The poster can be viewed on the Edgewise website at www.edgewisetx.com.

    Advanced Research Programs

    Edgewise has developed and characterized a library of selective fast skeletal myosin inhibitors exhibiting a broad range of pharmacological and PK properties. The Company is characterizing EDG-6289, a faster onset alternative to EDG-5506 for the treatment of muscle spasticity associated with a variety of disorders including multiple sclerosis, cerebral palsy and stroke.

    As part of our EDG-002 program, Edgewise is also advancing a novel mechanism cardiac modulator for inherited hypertrophic cardiomyopathy (HCM). Preliminary preclinical studies offer evidence that the EDG-002 program has the potential to yield molecules that could become a new standard of care for the treatment of HCM.

    At Edgewise, we believe that our muscle-targeted programs also offer substantial opportunities to expand into related rare diseases for which there are limited or no approved treatments.

    Strengthened Engagement with Muscular Dystrophy Community

    In February and March 2021, leadership presented an introduction to the Company and its plans during community webinars with the Muscular Dystrophy Association, Parent Project Muscular Dystrophy Italy and CureDuchenne. Replays of the webinars are available on the Edgewise website at www.edgewisetx.com. In February 2021, Edgewise also sponsored the Jett Foundation's Rare Disease Day Celebration.

    Strengthened Leadership: Appointed Chief Medical Officer and New Directors to the Board

    In May 2021, Edgewise appointed Joanne Donovan, M.D., Ph.D., as its Chief Medical Officer (CMO), following her most recent tenure as CMO and Senior Vice President, Clinical Development at Catabasis Pharmaceuticals. Further, Edgewise appointed Badreddin Edris, Ph.D., a co-founder and strategic advisor of Edgewise, and Laura Brege, an experienced healthcare executive, to its Board of Directors in February 2021 and December 2020, respectively.

    First Quarter 2021 Financial Results

    Cash, cash equivalents and marketable securities were $309.9 million as of March 31, 2021, which includes gross proceeds of $202.4 million from the Company's IPO.

    Research and development expenses were $5.4 million for the first quarter 2021, compared to $3.0 million for the first quarter of 2020. The increase of $2.4 million was primarily driven by $0.6 million of higher external research and development expenses related to our Phase 1 clinical trial of EDG-5506, an increase of $0.7 million related to increased drug discovery and preclinical development of our other programs and an increase of $1.0 million in employee-related costs, due largely to an increased employee headcount to support the growth of our research and development programs.

    General and Administrative expenses were $1.5 million for the first quarter 2021, compared to $0.3 million for the first quarter of 2020. The increase of $1.2 million was primarily related to $0.7 million in increased employee-related costs from increased headcount and $0.5 million in increased professional and consulting costs.

    Net loss and net loss per share for the first quarter of 2021 was $6.8 million, and $4.37, respectively.

    On a non-GAAP basis, adjusted net loss per share for the first quarter of 2021 was $0.14 based on 49.3 million shares of the Company's common stock outstanding as of March 31, 2021 following the closing of the Company's IPO. A reconciliation of GAAP net loss per share to non-GAAP adjusted net loss per share can be found below.

    About EDG-5506 for DMD and BMD

    EDG-5506 is an orally administered small molecule designed to address the root cause of dystrophinopathies, including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It can be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

    The Phase 1 study of EDG-5506 is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and adults with BMD (Phase 1b). In March 2021, EDG-5506 advanced into the Multiple Ascending Dose (MAD) portion of the study, having successfully completed the Single Ascending Dose (SAD) portion of the study. The Company expects to report topline MAD data in healthy volunteers and BMD patient data, later in 2021. To learn more about this study (NCT04585464), go to clinicaltrials.gov.

    About Edgewise Therapeutics

    Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.

    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 drug discovery platform and product candidates including EDG-5506 and EDG- 6289; statements about the expected timing and results from Edgewise's Phase 1 clinical trials for EDG-5506; statements regarding Edgewise's pipeline of product candidates and programs; and statements by Edgewise's 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 preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 and Edgewise's other product candidates in its EDG-6289, EDG-002 and EDG-003 programs; Edgewise's ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise's operations, including preclinical and clinical trials; the timing, scope and likelihood of regulatory filings and approvals; 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 Securities and Exchange Commission (the "SEC"). 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.

    Edgewise Therapeutics, Inc.
    Condensed Statement of Operations
    (in thousands except share and per share amounts, unaudited)
     
    Three months ended March 31,

    2021

    2020

    Operating expenses:
    Research and development $

    5,360

     

    $

    2,967

     

    General and administrative

    1,497

     

    281

     

    Total operating expenses

    6,857

     

    3,248

     

    Loss from operations

    (6,857

    )

    (3,248

    )

    Interest income

    42

     

    63

     

    Net loss $

    (6,815

    )

    $

    (3,185

    )

    Net loss per share - basic and diluted $

    (4.37

    )

    $

    (7.57

    )

    Weighted-average shares outstanding, basic and diluted

    1,559,868

     

    420,482

     

     
     
    Edgewise Therapeutics, Inc.
    Condensed Balance Sheet Data
    (in thousands, unaudited)
     

    March 31,

    December, 31

    2021

    2020

    Assets
    Cash, cash equivalents and marketable securities $

    309,871

     

    $

    129,094

     

    Other assets

    1,374

     

    2,042

     

    Total assets $

    311,245

     

    $

    131,136

     

    Liabilities, convertible preferred stock and stockholders' equity (deficit)
    Liabilities

    4,851

     

    4,342

     

    Convertible preferred stock

    -

     

    160,214

     

    Stockholders' equity (deficit)

    306,394

     

    (33,420

    )

    Total liabilities, convertible preferred stock and stockholders' equity (deficit) $

    311,245

     

    $

    131,136

     

    Edgewise Therapeutics, Inc.
    Reconciliation of GAAP net loss per share to non-GAAP adjusted net loss per share
    (unaudited)
    Net loss

    $

    (6,815

    )

    Net loss per share

    $

    (4.37

    )

    Weighted average shares outstanding for the three months ended 3/31/2021

     

    1,559,868

     

    Adjustments to the denominator of the net loss per share calculation(1):
    Options exercised during the period

     

    22,514

     

    Conversion of preferred shares

     

    35,162,485

     

    Issuance of shares in initial public offering

     

    12,509,444

     

    Shares outstanding as of 3/31/2021

     

    49,254,311

     

    Adjusted net loss per share

    $

    (0.14

    )

     
    (1) Adjustments reflect elimination of weighted average impact in GAAP net loss per share
     

    Use of Non-GAAP Financial Measures

    Edgewise Therapeutics has supplemented its GAAP financial measures of net loss per share with its non-GAAP measures of adjusted net loss per share. Edgewise Therapeutics believes that this non-GAAP financial measure provides useful supplemental information to management and investors regarding the performance of Edgewise Therapeutics and facilitates a potentially meaningful comparison of results for the current period with future operating results.

    There are limitations in using non-GAAP financial measures because they are not prepared in accordance with GAAP and may be different from non-GAAP financial measures used by other companies. This non-GAAP financial measure should not be considered in isolation or as a substitute for GAAP financial measures. Investors and potential investors should consider non-GAAP financial measures only in conjunction with Edgewise Therapeutics' consolidated financial statements prepared in accordance with GAAP.

    Non-GAAP financial measures used by Edgewise Therapeutics may be calculated differently from, and therefore may not be comparable to, non-GAAP measures used by other companies.

    ###

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  4. – First cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in BMD and DMD patient samples, published in Muscle & Nerve –

    – Data supports Edgewise strategy to advance EDG-5506, a small molecule drug candidate designed to protect injury-susceptible fast skeletal muscle fibers, in clinical development for BMD and DMD –

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the publication of the first cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers…

    – First cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in BMD and DMD patient samples, published in Muscle & Nerve –

    – Data supports Edgewise strategy to advance EDG-5506, a small molecule drug candidate designed to protect injury-susceptible fast skeletal muscle fibers, in clinical development for BMD and DMD –

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the publication of the first cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in the blood of patients with Becker and Duchenne muscular dystrophy (BMD, DMD) in the journal, Muscle & Nerve.

    Human skeletal muscle is composed of fast and slow fibers in roughly equal proportion. Previous studies have demonstrated that DMD patient muscle is more prone to fast fiber injury compared to slow fiber injury. This study extends these findings to examine fiber-type specific biomarkers of muscle injury in patient blood. The results build on previous reports and suggest that slow skeletal muscle fibers do not appear to leak muscle proteins associated with muscle injury and damage in BMD and DMD. Furthermore, the study demonstrated that fast skeletal troponin I (TNNI2) may represent a more sensitive biomarker of muscle injury than creatine kinase (CK), particularly in the setting of BMD or older DMD patients where plasma CK is commonly lower than in young DMD patients. These data further support EDG-5506's unique mechanism of action aimed at protecting injury-susceptible fast skeletal muscle fibers in BMD and DMD.

    "We are pleased to share our findings that elevation of fast skeletal muscle fiber injury biomarkers may be more informative, compared to slow skeletal muscle injury biomarkers, of muscle injury in patients with BMD and DMD," said Alan Russell, Ph.D., Chief Scientific Officer of Edgewise and senior author of the study. "These data guided the design of our lead clinical candidate, EDG-5506, which selectively protects injury-susceptible fast skeletal muscle fibers for BMD and DMD. Indeed, EDG-5506, in preclinical models, acts as a muscle stabilizer by protecting muscle through limiting excessive contraction. This protection reduces injury and the leak of muscle proteins that in the long term protects against fibrosis and functional declines associated with prolonged inflammation and degradation of the muscle."

    Kevin Koch, Ph.D., President and Chief Executive Officer added, "These data are invaluable as we continue to advance EDG-5506 in clinical development. We are excited about the potential of EDG-5506 for patients affected by BMD and DMD, who are currently underserved with limited treatment options."

    About the Skeletal Muscle Study

    One of the hallmarks of injured skeletal muscle is the appearance of elevated skeletal muscle proteins in circulation. Human skeletal muscle generally consists of a mosaic of slow (type I) and fast (type IIa, IIx/d) fibers, defined by their myosin isoform expression. Recently, measurement of circulating fiber‐type specific isoforms of troponin I has been used as a biomarker to suggest that muscle injury in healthy volunteers results in the appearance of muscle proteins from fast but not slow fibers. Data from this study demonstrates that this is also the case in severe myopathy patients with Becker and Duchenne muscular dystrophy (BMD, DMD).

    An ELISA test that selectively measures fast and slow skeletal troponin I (TNNI2 and TNNI1) was used to measure a cross‐section of patient plasma samples from healthy volunteers (N=50), BMD (N=49) and DMD (N=132) patients. CK activity, which is a type of protein found in skeletal and heart muscles, was also measured from the same samples for comparison.

    The fast skeletal troponin, TNNI2, was elevated in BMD and DMD and correlated with the injury biomarker, CK. In contrast, the slow skeletal troponin, TNNI1, levels were indistinguishable from levels in healthy volunteers. There was an inverse relationship between CK and TNNI2 levels and age but no relationship for TNNI1. Of note, a surprising discrepancy between TNNI1 and TNNI2 in patient plasma may have implications for the interpretation of elevated muscle protein levels in BMD, DMD and other dystrophinopathies.

    This is the first cross-sectional, retrospective study to describe differences between fast and slow skeletal muscle fiber biomarkers in BMD and DMD patient plasma. Findings of differential troponin levels is consistent with previous studies of muscle injury after eccentric exercise in healthy volunteers. This appears to be distinct from muscle injury via sepsis or trauma where both fast and slow TNNI are elevated. Previous studies have demonstrated preferential fast fiber injury in DMD patients. Our data extend these findings to suggest that slow fibers do no not appear to leak muscle proteins in the context of BMD/DMD. The majority of healthy volunteers (83%) had TNNI2 levels below the lower level of detection of the ELISA (<0.1 ng/ml), while only 4% of BMD and 6% of DMD patients had non-measurable levels of TNNI2. This is in marked contrast to CK where large overlap exists, particularly between healthy volunteers and patients with BMD. As a result, TNNI2 may represent a more sensitive biomarker of muscle injury than CK, particularly in the setting of BMD or older DMD patients where plasma CK is commonly lower than in young DMD patients.

    About EDG-5506 for BMD and DMD

    EDG-5506, is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It can be used as a single agent therapy but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

    Our Phase 1 study of EDG-5506 is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and adults with BMD (Phase 1b). In March 2021, EDG-5506 advanced into the Multiple Ascending Dose (MAD) cohort, having successfully completed the Single Ascending Dose (SAD) portion of the study. The company expects to report topline MAD and BMD patient data, later in 2021. To learn more about this study (NCT04585464), go to clinicaltrials.gov.

    About Muscular Dystrophy

    Muscular dystrophies are a group of genetic disorders associated with defects in the critical muscle-associated structural protein dystrophin or the sarcomere complex and are characterized by progressive muscle degeneration and weakness. In individuals with neuromuscular conditions such as Duchenne muscular dystrophy, normal muscle use leads to continued rounds of muscle breakdown that the body struggles to repair. Over time, fibrosis and fatty tissue accumulate in the muscle portending a steep decline and permanent loss of physical function that ends with mortality. There remains an unmet need for treatments that reduce muscle breakdown in patients with neuromuscular conditions. Arresting this amplified muscle response will have a dramatic effect on disease progression.

    About Edgewise Therapeutics

    Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.

    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 advantages of Edgewise's product candidates and programs, including plans underlying EDG-5506 or any other programs; and statements by the company's 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 preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 and Edgewise's other product candidates in its EDG-6289, EDG-002 and EDG-003 programs; Edgewise's ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise's operations, including preclinical and clinical trials; the timing, scope and likelihood of regulatory filings and approvals; 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 Edgewise's Registration Statement on Form S-1 filed with the Securities and Exchange Commission (the "SEC") on March 26, 2021, and Edgewise's future reports to be filed with the SEC. 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.

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  5. Rare disease expert joins as company continues to advance lead muscular dystrophy product candidate in clinical development –

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the appointment of Joanne M. Donovan, M.D., Ph.D., to the position of Chief Medical Officer. Dr. Donovan will be responsible for leading clinical, medical and regulatory strategy and operations for the company's pipeline, including EDG-5506, the company's lead product candidate that is advancing in clinical development for Duchenne and Becker muscular dystrophy (DMD and BMD).

    "Dr. Donovan brings a wealth of industry and…

    Rare disease expert joins as company continues to advance lead muscular dystrophy product candidate in clinical development –

    Edgewise Therapeutics, Inc., (NASDAQ:EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the appointment of Joanne M. Donovan, M.D., Ph.D., to the position of Chief Medical Officer. Dr. Donovan will be responsible for leading clinical, medical and regulatory strategy and operations for the company's pipeline, including EDG-5506, the company's lead product candidate that is advancing in clinical development for Duchenne and Becker muscular dystrophy (DMD and BMD).

    "Dr. Donovan brings a wealth of industry and clinical leadership that will be invaluable to our team as we advance EDG-5506 in clinical development," said Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics. "Having led multiple rare disease clinical programs through all phases of development, from discovery to approvals, Dr. Donovan brings strong leadership capabilities and a broad skill set to Edgewise."

    "I'm impressed with Edgewise's precision medicine drug discovery platform and novel scientific approach to treating severe, progressive and rare muscle disorders," said Dr. Donovan. "EDG-5506 has the potential to benefit a broad population of muscular dystrophy patients and represents a new strategy for treating these devastating conditions by selectively limiting contraction in susceptible fast muscle fibers to preserve muscle function. I'm delighted to lead the clinical development of EDG-5506, which provides new hope for patients with DMD and BMD who are in desperate need for alternative therapies."

    Dr. Donovan has deep experience in the biotechnology industry. Prior to joining Edgewise, Dr. Donovan served as Chief Medical Officer and Senior Vice President, Clinical Development at Catabasis, a biopharmaceutical company focused on rare diseases. In this role, Dr. Donovan led clinical development in DMD from first-in-human through Phase 3 studies and New Drug Application preparation. She led and built all clinical and regulatory functional areas, overseeing regulatory and quality affairs, pharmacovigilance, medical affairs and clinical operations. Since 1989, she has been a staff physician at the VA Boston Healthcare System, where she was formerly Chief of Gastroenterology. Dr. Donovan has held an appointment at Harvard Medical School since 1990, most recently as Associate Clinical Professor of Medicine. From 1998 to 2011, Dr. Donovan served in positions of increasing responsibility, ultimately as Vice President of Clinical Development, at Genzyme, a biotechnology company focused on rare diseases, which she joined through its acquisition of GelTex Pharmaceuticals. Dr. Donovan holds a Ph.D. in medical engineering and medical physics from the Massachusetts Institute of Technology, an M.D. from Harvard Medical School and an S.B. from the Massachusetts Institute of Technology. She completed residency training in internal medicine and a fellowship in gastroenterology at the Brigham and Women's Hospital.

    About EDG-5506 for DMD and BMD

    EDG-5506, is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It can be used as a single agent therapy but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

    The Phase 1 study of EDG-5506 is designed to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and adults with BMD (Phase 1b). In March 2021, EDG-5506 advanced into the Multiple Ascending Dose (MAD) cohort, having successfully completed the Single Ascending Dose (SAD) portion of the study. The company expects to report topline MAD and BMD patient data, later in 2021. To learn more about this study (NCT04585464), go to clinicaltrials.gov.

    About Edgewise Therapeutics

    Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare muscle disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle focused drug discovery platform. Edgewise's platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.

    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 advantages of Edgewise's product candidates and programs, including plans underlying EDG-5506 or any other programs; and statements by the company's 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 preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 and Edgewise's other product candidates in its EDG-6289, EDG-002 and EDG-003 programs; Edgewise's ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise's operations, including preclinical and clinical trials; the timing, scope and likelihood of regulatory filings and approvals; 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 Edgewise's Registration Statement on Form S-1 filed with the Securities and Exchange Commission (the "SEC") on March 26, 2021, and Edgewise's future reports to be filed with the SEC. 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.

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