1. -- Cash and Cash Equivalents Totaled $52.9 Million as of September 30, 2020 --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a biopharmaceutical company, today reported financial results for the third quarter ended September 30, 2020 and provided a corporate update.

    As previously reported, the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, change from baseline in the North Star Ambulatory Assessment, over one year of treatment compared to placebo. The secondary endpoint timed function tests also did not show statistically significant improvements. Edasalonexent was observed to be generally safe and well-tolerated in this trial, consistent with the safety profile seen to date…

    -- Cash and Cash Equivalents Totaled $52.9 Million as of September 30, 2020 --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a biopharmaceutical company, today reported financial results for the third quarter ended September 30, 2020 and provided a corporate update.

    As previously reported, the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, change from baseline in the North Star Ambulatory Assessment, over one year of treatment compared to placebo. The secondary endpoint timed function tests also did not show statistically significant improvements. Edasalonexent was observed to be generally safe and well-tolerated in this trial, consistent with the safety profile seen to date. As previously announced, Catabasis is stopping activities related to the development of edasalonexent, including the ongoing GalaxyDMD open-label extension trial.

    Catabasis has engaged Ladenburg Thalmann & Co. Inc. to act as its strategic financial advisor for the previously announced plan to explore and evaluate strategic options. Potential strategic options that may be evaluated include a merger, business combination, in-licensing, out-licensing or other strategic transaction. There can be no assurance that this process will result in any such transaction. We do not intend to discuss or disclose further developments during this process unless and until our Board of Directors has approved a specific action or otherwise determined that further disclosure is appropriate.

    Third Quarter 2020 Financial Results

    Cash Position: As of September 30, 2020, Catabasis had cash, cash equivalents and short-term investments of $52.9 million, compared to $53.9 million as of June 30, 2020. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations for at least the next 12 months. Net cash used in operating activities for the three months ended September 30, 2020 was $10.0 million, compared to $6.5 million for the three months ended September 30, 2019.

    R&D Expenses: Research and development expenses were $7.8 million for the three months ended September 30, 2020, compared to $4.7 million for the three months ended September 30, 2019.

    G&A Expenses: General and administrative expenses were $3.1 million for the three months ended September 30, 2020, compared to $2.0 million for the three months ended September 30, 2019.

    Operating Loss: Loss from operations was $10.9 million for the three months ended September 30, 2020, compared to $6.7 million for the three months ended September 30, 2019.

    Net Loss: Net loss was $10.9 million, or $0.56 per share, for the three months ended September 30, 2020, compared to a net loss of $6.5 million, or $0.56 per share, for the three months ended September 30, 2019.

    About Catabasis

    Catabasis Pharmaceuticals is a biopharmaceutical company. Our mission is to bring hope with life-changing therapies to patients and families.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its exploration and evaluation of strategic options, the termination of activities related to the edasalonexent program, including the ongoing GalaxyDMD open-label extension trial, and cash to fund operations, and other statements containing the words "believes," "anticipates," "plans," "hopes," "expects," and similar expressions, constitute forward-looking statements within the meaning of applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties related to: the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented to address the pandemic; the availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; unexpected costs or expenses, including any that arise during the termination of activities related to the edasalonexent program; risks inherent in the Company's exploration, evaluation and implementation of its review of strategic options; and general market and economic conditions; and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

    Catabasis Pharmaceuticals, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)

    (Unaudited)

     

    Three Months Ended September 30,

     

    Nine Months Ended September 30,

    2020

     

    2019

     

    2020

     

    2019

     
    Operating expenses:
    Research and development

    $

    7,806

     

    $

    4,697

     

    $

    19,845

     

    $

    14,054

     

    General and administrative

     

    3,057

     

     

    1,985

     

     

    8,612

     

     

    6,287

     

    Total operating expenses

     

    10,863

     

     

    6,682

     

     

    28,457

     

     

    20,341

     

    Loss from operations

     

    (10,863

    )

     

    (6,682

    )

     

    (28,457

    )

     

    (20,341

    )

    Other income (expense):
    Interest and investment income

     

    4

     

     

    214

     

     

    231

     

     

    697

     

    Other expense, net

     

    (3

    )

     

    (46

    )

     

    (96

    )

     

    (39

    )

    Total other income, net

     

    1

     

     

    168

     

     

    135

     

     

    658

     

    Net loss

    $

    (10,862

    )

    $

    (6,514

    )

    $

    (28,322

    )

    $

    (19,683

    )

    Net loss per share - basic and diluted

    $

    (0.56

    )

    $

    (0.56

    )

    $

    (1.59

    )

    $

    (1.80

    )

    Weighted-average common shares outstanding used in net loss per share - basic and diluted

     

    19,424,866

     

     

    11,624,232

     

     

    17,769,738

     

     

    10,945,765

     

     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheets Data

    (In thousands)

    (Unaudited)

     

    September 30,

     

    December 31,

    2020

     

    2019

    Assets
    Cash and cash equivalents

    $

    52,856

    $

    9,899

    Short-term investments

     

    -

     

     

    26,345

     

    Right-of-use asset

     

    1,178

     

     

    2,349

     

    Other current and long-term assets

     

    2,816

     

     

    3,187

     

    Total assets

     

    56,850

     

     

    41,780

     

    Liabilities and stockholders' equity
    Current portion of operating lease liabilities

     

    648

     

     

    1,225

     

    Long-term portion of operating lease liabilities

     

    559

     

     

    1,028

     

    Other current and long-term liabilities

     

    6,332

     

     

    3,807

     

    Total liabilities

     

    7,539

     

     

    6,060

     

    Total stockholders' equity

    $

    49,311

     

    $

    35,720

     

     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Statements of Cash Flows Data

    (In thousands)

    (Unaudited)

     

    Nine Months Ended September 30,

    2020

     

    2019

    Net cash used in operating activities

    $

    (24,424

    )

    $

    (18,799

    )

    Net cash provided by (used) in investing activities

     

    26,310

     

     

    (578

    )

    Net cash provided by financing activities

     

    40,829

     

     

    21,848

     

    Net increase in cash, cash equivalents and restricted cash

    $

    42,715

     

    $

    2,471

     

     

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  2. -- PolarisDMD Trial Did Not Achieve Primary or Secondary Endpoints --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, which was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements. Edasalonexent was observed to be generally safe and well-tolerated in this trial. Catabasis is stopping activities related to the development of edasalonexent including…

    -- PolarisDMD Trial Did Not Achieve Primary or Secondary Endpoints --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, which was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements. Edasalonexent was observed to be generally safe and well-tolerated in this trial. Catabasis is stopping activities related to the development of edasalonexent including the ongoing GalaxyDMD open-label extension trial. The Company plans to work with external advisors to explore and evaluate strategic options going forward.

    "We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "I want to sincerely thank all of the boys, their families and caregivers, investigators and the trial sites that participated in and enabled this program. The entire Catabasis team has worked tirelessly to find a treatment for this progressive disease. We hope that our data and work to date can be used to benefit ongoing and future research in DMD."

    The Phase 3 trial was a one-year placebo-controlled trial designed to evaluate the safety and efficacy of edasalonexent in boys ages 4-7 (up to 8th birthday) with DMD. The global trial enrolled 131 boys across eight countries, with any mutation type, who were not on steroids. Edasalonexent was well-tolerated, consistent with the safety profile seen to date. The majority of adverse events were mild in nature and the most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash. There were no treatment-related serious adverse events and no dose reductions. The global COVID-19 pandemic had no meaningful impact on the trial or its results. Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published.

    "These results are disheartening for the Duchenne community, and specifically for the boys who participated in this trial and their families. However, the results contribute to the natural history data of Duchenne and add to the knowledge base that will one day produce a foundational, long-term therapy for this disease," said Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD). "The continued advancement of research and the development of possible treatment options will remain of critical importance to our community. We appreciate Catabasis' efforts and commitment to every family that is or has ever been affected by Duchenne."

    The Company expects to report Q3 2020 financials in November of 2020. As of September 30, 2020, Catabasis had cash and cash equivalents of approximately $52.9 million.

    About Catabasis

    Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical company. Our mission is to bring hope and life-changing therapies to patients and their families.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its exploration and evaluation of strategic options, the termination of activities related to the edasalonexent program, including the ongoing GalaxyDMD open-label extension trial, and plans to publish data from the Phase 3 PolarisDMD trial, and other statements containing the words "believes," "anticipates," "plans," "hopes," "expects," and similar expressions, constitute forward-looking statements within the meaning of applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties related to: the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented to address the pandemic; the availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; unexpected costs or expenses that arise during the termination of activities related to the edasalonexent program; risks inherent in the Company's exploration, evaluation and implementation of its review of strategic options; and general market and economic conditions; and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  3. -- New Preclinical Research Indicates that Edasalonexent Could Have Positive Effects on Cardiac Function and Preserve Bone Health --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for Duchenne muscular dystrophy (DMD) in poster presentations at the Virtual 25th International Congress of the World Muscle Society. The posters include new preclinical research findings supporting potential positive effects on cardiac function and preservation of bone health with edasalonexent in mouse models of DMD. Information from clinical trials with edasalonexent was also presented with baseline assessments from the Phase 3 PolarisDMD trial…

    -- New Preclinical Research Indicates that Edasalonexent Could Have Positive Effects on Cardiac Function and Preserve Bone Health --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for Duchenne muscular dystrophy (DMD) in poster presentations at the Virtual 25th International Congress of the World Muscle Society. The posters include new preclinical research findings supporting potential positive effects on cardiac function and preservation of bone health with edasalonexent in mouse models of DMD. Information from clinical trials with edasalonexent was also presented with baseline assessments from the Phase 3 PolarisDMD trial and long-term safety results from the Phase 2 MoveDMD trial and open-label extension.

    "As we approach top-line Phase 3 PolarisDMD results, which we expect to report in the fourth quarter of this year, we are pleased to share new findings from preclinical studies with edasalonexent that could support its potential as a foundational therapy for those affected by Duchenne," said Andrew Nichols, Ph.D., Chief Scientific Officer of Catabasis. "Cardiac function and bone health are critical components of Duchenne care and we are excited to learn more about these key areas that are important to patients, their caregivers and physicians."

    Persistent activation of NF-kB in DMD can drive cardiac dysfunction, which is the leading cause of mortality in DMD. In a preclinical study performed in the laboratory of Pradeep Mammen, M.D., Medical Director of the Neuromuscular Cardiomyopathy Clinic and Director of Translational Research for the Advanced Heart Failure and Transplant Cardiology Program at UT Southwestern, edasalonexent prevented the development of DMD-associated cardiomyopathy in the mdx:Utrn+/- mouse model of DMD. Edasalonexent reduced the cardiac hypertrophy apparent in these mice, reduced myocardial fibrosis and prevented the development of DMD-associated cardiomyopathy.

    In DMD, NF-kB activation also drives inflammation and fibrosis, leading to loss of skeletal muscle function and disease progression. Reduced skeletal muscle function results in reduced bone strength. Steroids, which activate the glucocorticoid receptor (GR), can further negatively impact bone health. In a preclinical study led by Frank Rauch, M.D., of Shriners Hospitals for Children - Canada, edasalonexent was seen to maintain bone density and bone strength in mdx mice. Consistent with these results, edasalonexent treatment in cells inhibited NF-kB and as expected did not impact the GR, while the steroid prednisolone strongly activated the GR.

    Information from the edasalonexent clinical program was also presented at the Congress of the World Muscle Society. The primary endpoint of the Phase 3 PolarisDMD trial for edasalonexent is the North Star Ambulatory Assessment (NSAA), a validated measure of physical function designed for use in ambulatory boys affected by DMD. In an analysis of baseline characteristics of the patients enrolled in the Phase 3 trial (boys affected by DMD, ages 4 to 7, up to 8th birthday, not on steroids), Catabasis saw reliable and consistent NSAA scores between the two pre-treatment measures at screening and baseline, which indicate no significant learning effect between visits.

    An additional evaluation of data from patients at baseline (prior to any treatment) in the Phase 3 PolarisDMD trial was shared by Leanne Ward, M.D., FRCPC, Scientific Director of Ottawa Pediatric Bone Health Research Group. This analysis showed that lean body mass index (a marker for muscle mass) was reduced in young boys affected by DMD and correlated with reduced muscle function. An analysis of the same patients was presented earlier this month at the American Society for Bone and Mineral Research 2020 Annual Conference and showed that DMD has a negative impact on bone strength, including low bone density and mild vertebral fractures at an early stage of disease course prior to any treatment.

    As Catabasis has previously presented, the Phase 2 MoveDMD trial and open-label extension was also shared showing that edasalonexent was well-tolerated for up to 150 weeks and associated with favorable growth patterns. The most common related adverse effect was diarrhea, generally mild and transient.

    The posters are available to registered attendees during the conference and for three months afterwards. Select posters are available in the "Our Science" section of www.catabasis.com.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the clinical program of edasalonexent, including the expected timing of release of Phase 3 data, edasalonexent potentially being a foundational therapy for DMD patients, and edasalonexent potentially having positive effects on cardiac function and preservation of bone health, along with other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of applicable securities regulations and laws. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties: inherent in the initiation and completion of clinical trials and clinical development; related to whether the results of pre-clinical studies will be predictive of results of clinical trials; related to whether the results of earlier stage clinical trials will be predictive of the results of later stage trials; related to the regulatory review and approval process; inherent in the commercialization of marketed products; related to competitive products, including those already approved and those in development; related to other matters that could affect the clinical development, regulatory status, availability or commercial potential of edasalonexent, as well as the risks and uncertainties discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  4. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program, in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD), during the 25th International Congress of the World Muscle Society which begins September 28, 2020.

    During the Virtual Poster Session on October 1, 2020 from 12:30 – 2:30pm ET, Catabasis and collaborators will present five posters entitled:

    • Inhibition of NF-kB Signaling Prevents the Development of DMD-Associated Cardiomyopathy in mdx:Utrn+/- Mice
    • Edasalonexent Maintains Bone Density and Bone Strength in the mdx Mouse Model of Duchenne Muscular Dystrophy
    • Edasalonexent Treatment in Young Boys

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program, in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD), during the 25th International Congress of the World Muscle Society which begins September 28, 2020.

    During the Virtual Poster Session on October 1, 2020 from 12:30 – 2:30pm ET, Catabasis and collaborators will present five posters entitled:

    • Inhibition of NF-kB Signaling Prevents the Development of DMD-Associated Cardiomyopathy in mdx:Utrn+/- Mice
    • Edasalonexent Maintains Bone Density and Bone Strength in the mdx Mouse Model of Duchenne Muscular Dystrophy
    • Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy is Associated with Age-Normative Growth and Normal Adrenal Function
    • In the Global Phase 3 PolarisDMD Trial for Edasalonexent, Standardized Outcome Measure Training Produces Excellent Test-Retest Variability in the North Star Ambulatory Assessment
    • Lean Body Mass is Associated with Whole Body Mineral Density and Muscle Strength in Treatment-Naïve, Ambulatory Boys with Duchenne Muscular Dystrophy

    The poster session will be available to registered conference attendees during the conference and for 3 months afterwards. Select posters will be made available in the "Our Science" section of www.catabasis.com.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  5. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has promoted Noah Clauser to Chief Financial Officer. Mr. Clauser brings close to 20 years of financial experience to this role and has been with Catabasis for 9 years, most recently as Vice President, Finance.

    "Noah has strategically built our financial and operations functions and has been a valuable team member through a critical stage for Catabasis," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are looking forward to his expanded role as we approach our potential future transition to a commercial organization."

    "I am excited for this new opportunity as we approach the next stage of our company, with…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has promoted Noah Clauser to Chief Financial Officer. Mr. Clauser brings close to 20 years of financial experience to this role and has been with Catabasis for 9 years, most recently as Vice President, Finance.

    "Noah has strategically built our financial and operations functions and has been a valuable team member through a critical stage for Catabasis," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are looking forward to his expanded role as we approach our potential future transition to a commercial organization."

    "I am excited for this new opportunity as we approach the next stage of our company, with top-line results from the Phase 3 PolarisDMD trial for edasalonexent expected in the fourth quarter of this year," said Mr. Clauser. "I am looking forward to continuing our work to make an impact in the lives of patients and families affected by Duchenne muscular dystrophy."

    Mr. Clauser most recently served as Vice President, Finance at Catabasis, leading the Company's finance and operations functions since August 2017. Previously, he served as Senior Director, Finance and Controller of the Company from January 2016 to August 2017, and Controller from April 2011 to December 2015. Prior to joining Catabasis, Mr. Clauser worked at Impress Software, where he served as Accounting Manager. Mr. Clauser is a licensed CPA in Massachusetts and holds an M.S. in Accounting and a B.S. in Management from the University of Massachusetts at Boston.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the Company's global Phase 3 PolarisDMD trial in DMD, the anticipated timing for top-line results and the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties: related to the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented to address the pandemic, including the use of telehealth visits; inherent in the completion of clinical trials and clinical development; related to whether the results of earlier stage clinical trials will be predictive of the results of later stage trials; related to the regulatory review and approval process; inherent in the commercialization of marketed products; related to successfully managing the Company's potential transformation into a fully integrated company; related to competitive products, including those already approved and those in development; inherent in transitioning from a clinical to commercial supply chain, including the ability to enter into long-term agreements with key contract manufacturers, overseeing such manufacturers, and managing inventory, particularly where the Company expects to use sole source manufacturers for the foreseeable future; related to the availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; related to other matters that could affect the clinical development, regulatory status, availability or commercial potential of the Company's product candidates; and related to general market and economic conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  6. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, plans to present a corporate overview and provide information about edasalonexent, in Phase 3 development for the treatment of Duchenne muscular dystrophy, at the following virtual investor conferences this month:

    • HC Wainwright 22nd Annual Global Investment Conference on Monday, September 14th, at 4:00pm EDT
    • Cantor Virtual Healthcare Conference on Wednesday, September 16th, at 3:20pm EDT

    Catabasis will also be participating in the Oppenheimer Virtual Fall Healthcare, Life Sciences, & MedTech Summit, which will take place from September 21st – 23rd, 2020.

    Live webcasts of the events…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, plans to present a corporate overview and provide information about edasalonexent, in Phase 3 development for the treatment of Duchenne muscular dystrophy, at the following virtual investor conferences this month:

    • HC Wainwright 22nd Annual Global Investment Conference on Monday, September 14th, at 4:00pm EDT
    • Cantor Virtual Healthcare Conference on Wednesday, September 16th, at 3:20pm EDT

    Catabasis will also be participating in the Oppenheimer Virtual Fall Healthcare, Life Sciences, & MedTech Summit, which will take place from September 21st – 23rd, 2020.

    Live webcasts of the events can be accessed from the investors section of www.catabasis.com. Archived replays will also be available for 30 days following the event.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  7. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will be holding a virtual key opinion leader (KOL) event to discuss edasalonexent, currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD), on Friday, September 11,.2020 at 9:00am ET.

    The event will feature a discussion with Craig McDonald, M.D., Director, Neuromuscular Disease Clinics at UC Davis; Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis; Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis; Andrew A. Komjathy, Chief Commercial Officer of Catabasis; and Mindy Cameron, parent of a son affected by DMD.

    The live webcast of the event can be accessed by using the following link…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will be holding a virtual key opinion leader (KOL) event to discuss edasalonexent, currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD), on Friday, September 11,.2020 at 9:00am ET.

    The event will feature a discussion with Craig McDonald, M.D., Director, Neuromuscular Disease Clinics at UC Davis; Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis; Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis; Andrew A. Komjathy, Chief Commercial Officer of Catabasis; and Mindy Cameron, parent of a son affected by DMD.

    The live webcast of the event can be accessed by using the following link: https://www.webcaster4.com/Webcast/Page/2476/36576 or from the investors section of www.catabasis.com. An archived replay will also be available for 30 days following the event.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about edasalonexent potentially being a foundational therapy for DMD patients, along with other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of applicable securities regulations and laws. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties: inherent in the initiation and completion of clinical trials and clinical development; related to whether the results of earlier stage clinical trials will be predictive of the results of later stage trials; related to the regulatory review and approval process; inherent in the commercialization of marketed products; related to successfully managing the Company's potential transformation into a fully integrated company; related to competitive products, including those already approved and those in development; related to other matters that could affect the clinical development, regulatory status, availability or commercial potential of edasalonexent, as well as the risks and uncertainties discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  8. -- Top-Line Results Expected in Q4 2020 from Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the second quarter ended June 30, 2020 and reviewed recent business progress.

    "We remain on track for top-line results from our Phase 3 PolarisDMD trial for edasalonexent in Duchenne and we are looking forward to reporting the results later this year," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "In anticipation of data, we are continuing to advance the program and are working on preparations for a New Drug Application as well as our strategy for commercialization…

    -- Top-Line Results Expected in Q4 2020 from Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the second quarter ended June 30, 2020 and reviewed recent business progress.

    "We remain on track for top-line results from our Phase 3 PolarisDMD trial for edasalonexent in Duchenne and we are looking forward to reporting the results later this year," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "In anticipation of data, we are continuing to advance the program and are working on preparations for a New Drug Application as well as our strategy for commercialization and establishment of our supply chain. We see edasalonexent as a potential foundational therapy for all patients with DMD, regardless of underlying mutation."

    Phase 3 PolarisDMD Trial and GalaxyDMD Open-Label Extension

    • Top-line results from the fully enrolled Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) are expected in Q4 2020.
      • The Phase 3 trial enrolled 131 boys enrolled across 8 countries and is intended to support a new drug application (NDA) in 2021 for commercial registration of edasalonexent.
      • Due to the COVID-19 pandemic, Catabasis has implemented steps designed to enable continued safe conduct for patients and maintain study integrity, including the option to deliver study drug to patients' homes, increased flexibility in the timing of patient visits, and utilization of telehealth.
    • The open-label extension GalaxyDMD trial is enrolling boys who have completed the Phase 3 PolarisDMD trial as well as their interested eligible siblings. The trial is progressing as planned, with visits every six months.

    Edasalonexent Highlights

    • Catabasis presented information on the edasalonexent program at the 2020 Muscular Dystrophy Association Virtual Poster Session and the Parent Project Muscular Dystrophy Virtual Annual Conference.
      • North Star Ambulatory Assessment (NSAA), the primary endpoint in the Phase 3 PolarisDMD trial, was shown to be a consistent and reproducible measure of function in young boys with DMD in an analysis of the screening and baseline values from the Phase 3 PolarisDMD trial before treatment with edasalonexent.
      • The Phase 3 PolarisDMD trial was found to have enrolled a similar patient population as the Phase 2 MoveDMD trial. An analysis found overall similar baseline characteristics in the patient populations in the two trials. There were no significant differences between the two trials in baseline age, North Star Ambulatory Assessment (NSAA) score and all three timed function test values. These findings are believed to support the assumptions on which the Phase 3 trial was powered.
      • In the Phase 2 MoveDMD trial and open-label extension, clinical findings support edasalonexent being associated with age-normative growth without negative impact on bone health with no evidence of adrenal insufficiency or clinically significant changes in adrenal function.
    • Plans are progressing for the edasalonexent non-ambulatory trial in partnership with Duchenne UK and the trial is expected to initiate in 2021.

    Corporate Highlights

    • Catabasis entered into an agreement with the Bill & Melinda Gates Medical Research Institute to study CAT-5571 in drug-sensitive and drug-resistant tuberculosis.
    • Catabasis named Ben Harshbarger as Senior Vice President, adding extensive senior legal and compliance experience to the executive team.
    • The Company plans to host a virtual investor and analyst event with a leading key opinion leader on Friday, September 11, 2020, to discuss edasalonexent and its potential in patients with DMD.

    Second Quarter 2020 Financial Results

    Cash Position: As of June 30, 2020, Catabasis had cash, cash equivalents and short-term investments of $53.9 million, compared to $55.1 million as of March 31, 2020. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations through a potential NDA filing and through the third quarter of 2021. Net cash used in operating activities for the three months ended June 30, 2020 was $7.5 million, compared to $5.7 million for the three months ended June 30, 2019.

    R&D Expenses: Research and development expenses were $6.8 million for the three months ended June 30, 2020, compared to $5.2 million for the three months ended June 30, 2019.

    G&A Expenses: General and administrative expenses were $2.8 million for the three months ended June 30, 2020, compared to $2.2 million for the three months ended June 30, 2019.

    Operating Loss: Loss from operations was $9.6 million for the three months ended June 30, 2020, compared to $7.3 million for the three months ended June 30, 2019.

    Net Loss: Net loss was $9.5 million, or $0.53 per share, for the three months ended June 30, 2020, compared to a net loss of $7.1 million, or $0.62 per share, for the three months ended June 30, 2019.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about: the potential impact of the COVID-19 pandemic on the Company's business and operations; future clinical trial plans and potential regulatory activities including, among other things, statements about the potential commencement of the Company's planned trial in non-ambulatory patients, the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes and related statements, including the anticipated timing for top-line results, steps that have been implemented to address the Covid-19 pandemic and the potential timing for the filing of an NDA; commercialization and supply chain preparations for edasalonexent; edasalonexent potentially being a foundational therapy for DMD patients; plans for an analyst and investor event; and having sufficient cash to fund operations through a potential NDA filing and through the third quarter of 2021, along with other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of applicable securities regulations and laws. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties: related to the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented to address the pandemic, including the use of telehealth visits; inherent in the initiation and completion of clinical trials and clinical development; related to whether the results of earlier stage clinical trials will be predictive of the results of later stage trials; related to the regulatory review and approval process; inherent in the commercialization of marketed products; related to successfully managing the Company's potential transformation into a fully integrated company; related to competitive products, including those already approved and those in development; inherent in transitioning from a clinical to commercial supply chain, including the ability to enter into long-term agreements with key contract manufacturers, overseeing such manufacturers, and managing inventory, particularly where the Company expects to use sole source manufacturers for the foreseeable future; related to the availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; related to other matters that could affect the clinical development, regulatory status, availability or commercial potential of the Company's product candidates; and related to general market and economic conditions, as well as the risks and uncertainties discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended June 30, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

    Catabasis Pharmaceuticals, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)

    (Unaudited)

     
    Three Months Ended June 30, Six Months Ended June 30,

    2020

    2019

    2020

    2019

     
    Operating expenses:
    Research and development

    $

    6,750

     

    $

    5,160

     

    $

    12,039

     

    $

    9,357

     

    General and administrative

     

    2,803

     

     

    2,165

     

     

    5,555

     

     

    4,302

     

    Total operating expenses

     

    9,553

     

     

    7,325

     

     

    17,594

     

     

    13,659

     

    Loss from operations

     

    (9,553

    )

     

    (7,325

    )

     

    (17,594

    )

     

    (13,659

    )

    Other income (expense):
    Interest and investment income

     

    60

     

     

    257

     

     

    227

     

     

    483

     

    Other (expense) income, net

     

    (15

    )

     

    (63

    )

     

    (93

    )

     

    7

     

    Total other income, net

     

    45

     

     

    194

     

     

    134

     

     

    490

     

    Net loss

    $

    (9,508

    )

    $

    (7,131

    )

    $

    (17,460

    )

    $

    (13,169

    )

    Net loss per share - basic and diluted

    $

    (0.53

    )

    $

    (0.62

    )

    $

    (1.03

    )

    $

    (1.24

    )

    Weighted-average common shares outstanding used in net loss per share - basic and diluted

     

    17,967,495

     

     

    11,505,542

     

     

    16,933,079

     

     

    10,600,909

     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheets Data

    (In thousands)

    (Unaudited)

     

    June 30,

     

    December 31,

    2020

    2019

    Assets        
    Cash and cash equivalents

     $

                                51,885

     $

                                  9,899

    Short-term investments  

     

                                     2,001

     

     

                                   26,345

    Right-of-use asset

     

                                     1,326

     

                                     2,349

    Other current and long-term assets  

     

                                     1,875

     

     

                                     3,187

    Total assets

     

                                   57,087

     

                                   41,780

    Liabilities and stockholders' equity        
    Current portion of operating lease liabilities

     

                                        645

     

                                     1,225

    Long-term portion of operating lease liabilities  

     

                                        718

     

     

                                     1,028

    Other current and long-term liabilities

     

                                     4,864

     

                                     3,807

    Total liabilities  

     

                                     6,227

     

     

                                     6,060

    Total stockholders' equity

     $

                                50,860

     $

                                35,720

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Statements of Cash Flows Data

    (In thousands)

    (Unaudited)

     
    Six Months Ended June 30,

    2020

    2019

    Net cash used in operating activities

    $

    (14,455

    )

    $

    (12,325

    )

    Net cash provided by (used) in investing activities

     

    24,310

     

     

    (14,229

    )

    Net cash provided by financing activities

     

    31,889

     

     

    20,875

     

    Net increase (decrease) in cash, cash equivalents and restricted cash

    $

    41,744

     

    $

    (5,679

    )

     

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  9. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that Jill C. Milne, Ph.D., Chief Executive Officer, will present a corporate overview at the virtual 2020 Wedbush PacGrow Healthcare Conference on Wednesday, August 12, 2020 at 8:00am ET.

    A webcast of the presentation will be available on the investors section of the Company's website, www.catabasis.com, and will be archived for 30 days following the presentation.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that Jill C. Milne, Ph.D., Chief Executive Officer, will present a corporate overview at the virtual 2020 Wedbush PacGrow Healthcare Conference on Wednesday, August 12, 2020 at 8:00am ET.

    A webcast of the presentation will be available on the investors section of the Company's website, www.catabasis.com, and will be archived for 30 days following the presentation.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  10. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today it entered into an agreement with the Bill & Melinda Gates Medical Research Institute (Gates MRI), a non-profit biotechnology organization, to assess CAT-5571 as a potential oral therapy to promote autophagy and clear persistent lung infections in patients with both drug-sensitive and drug-resistant tuberculosis (TB). Catabasis granted Gates MRI a non-exclusive license to CAT-5571, and will furnish samples of CAT-5571 to conduct this preclinical collaboration research program.

    Mycobacterium tuberculosis (Mtb) is known to cause TB. CAT-5571 will be studied alone and in combination with the standard of care regimen in cell and animal models…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today it entered into an agreement with the Bill & Melinda Gates Medical Research Institute (Gates MRI), a non-profit biotechnology organization, to assess CAT-5571 as a potential oral therapy to promote autophagy and clear persistent lung infections in patients with both drug-sensitive and drug-resistant tuberculosis (TB). Catabasis granted Gates MRI a non-exclusive license to CAT-5571, and will furnish samples of CAT-5571 to conduct this preclinical collaboration research program.

    Mycobacterium tuberculosis (Mtb) is known to cause TB. CAT-5571 will be studied alone and in combination with the standard of care regimen in cell and animal models of Mtb infection to determine the potential for CAT-5571 to induce autophagy and encourage Mtb elimination as well as to assess its contribution to TB drug regimens and durations.

    In previous preclinical studies, CAT-5571 was shown to restore autophagy in mouse and human cells with the delta508-CFTR mutation and promote the clearance of bacteria, including Mycobacterium abscessus, from these cells. Evidence suggests that autophagy plays a crucial role in antimycobacterial resistance in TB by acting as an alternative mechanism to control Mtb infection in macrophages, as well as to defend and counteract Mtb evasion strategies.

    "We are thrilled to work with Gates MRI to expand our understanding of CAT-5571," said Andrew Nichols, Ph.D., Chief Scientific Officer of Catabasis. "We look forward to learning more about how autophagy could potentially benefit communities with tuberculosis. We hope that a new treatment option may someday benefit those affected, especially those with TB resistant to current treatments."

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the anticipated results from the research program and CTA-5571 as a potential treatment for TB and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements under applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties related to the impact of the COVID-19 pandemic, uncertainties inherent in the initiation and completion of research programs and studying a potential therapy for a potential new indication, and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  11. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report second quarter 2020 financial results after the Nasdaq Global Market close on Monday, August 10, 2020.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report second quarter 2020 financial results after the Nasdaq Global Market close on Monday, August 10, 2020.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  12. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the Parent Project Muscular Dystrophy (PPMD) Virtual Annual Conference, which begins Wednesday, July 22, 2020.

    Joanne Donovan, M.D., Ph.D, Chief Medical Officer of Catabasis, will present "Edasalonexent (CAT-1004) Program: Oral NF-kB Inhibitor in Development for the Treatment of Duchenne Muscular Dystrophy" in a pre-recorded webinar, which will be available in the conference's On-Demand Library.

    Dr. Donovan will also provide an overview of edasalonexent during the "In the Pipeline: Reducing…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the Parent Project Muscular Dystrophy (PPMD) Virtual Annual Conference, which begins Wednesday, July 22, 2020.

    Joanne Donovan, M.D., Ph.D, Chief Medical Officer of Catabasis, will present "Edasalonexent (CAT-1004) Program: Oral NF-kB Inhibitor in Development for the Treatment of Duchenne Muscular Dystrophy" in a pre-recorded webinar, which will be available in the conference's On-Demand Library.

    Dr. Donovan will also provide an overview of edasalonexent during the "In the Pipeline: Reducing Inflammation" session on Wednesday, July 22nd at 7:00pm ET during the live stream.

    Additionally, Catabasis will share three posters during the Virtual Poster Session, which will also be available in the On-Demand Library.

    • "Characteristics of Patients Who Enrolled in PolarisDMD, a Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy"
    • "Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy is Associated with Age-Normative Growth and Normal Adrenal Function"
    • "Experience with Edasalonexent Demonstrates Ability of 4 to 7 Year Old Boys with Duchenne Muscular Dystrophy to Take Soft-gel Capsules"

    The On-Demand Library and the live stream will be made available to registered conference attendees.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the lack of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Top-line results from the Phase 3 PolarisDMD trial are expected in Q4 of this year. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the anticipated timing for top-line results from the Phase 3 PolarisDMD trial and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements under applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties related to the impact of the COVID-19 pandemic, uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  13. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has joined the broad-market Russell 3000® Index at the conclusion of the 2020 Russell Indexes annual reconstitution, effective after the US market opened today.

    Annual Russell indexes reconstitution captures the 4,000 largest US stocks as of May 8, 2020, ranking them by total market capitalization. FTSE Russell determines membership for its Russell indexes primarily by objective market-capitalization rankings and style attributes.

    Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has joined the broad-market Russell 3000® Index at the conclusion of the 2020 Russell Indexes annual reconstitution, effective after the US market opened today.

    Annual Russell indexes reconstitution captures the 4,000 largest US stocks as of May 8, 2020, ranking them by total market capitalization. FTSE Russell determines membership for its Russell indexes primarily by objective market-capitalization rankings and style attributes.

    Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russell's US indexes. Russell indexes are part of FTSE Russell, a leading global index provider.

    For more information on the Russell 3000® Index and the Russell indexes reconstitution, visit the "Russell Reconstitution" section on the FTSE Russell website.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    About FTSE Russell:

    FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98% of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

    FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. Approximately $16 trillion is currently benchmarked to FTSE Russell indexes. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create investment funds, ETFs, structured products and index-based derivatives. FTSE Russell indexes also provide clients with tools for asset allocation, investment strategy analysis and risk management.

    A core set of universal principles guides FTSE Russell index design and management: a transparent rules-based methodology is informed by independent committees of leading market participants. FTSE Russell is focused on index innovation and customer partnership applying the highest industry standards and embracing the IOSCO Principles. FTSE Russell is wholly owned by London Stock Exchange Group.

    For more information, visit www.ftserussell.com

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  14. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has named Ben Harshbarger as Senior Vice President, General Counsel. Mr. Harshbarger brings to Catabasis more than 20 years of experience at commercial-stage pharmaceutical and biotechnology companies.

    "Ben brings to Catabasis extensive senior legal and compliance experience in the pharmaceutical industry, including in product launches, and the transition from pre-commercial to commercial-stage. His deep expertise will be especially valuable in the coming months as we prepare for top-line results from the Phase 3 PolarisDMD trial and the potential commercialization of edasalonexent for the treatment of Duchenne muscular dystrophy…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has named Ben Harshbarger as Senior Vice President, General Counsel. Mr. Harshbarger brings to Catabasis more than 20 years of experience at commercial-stage pharmaceutical and biotechnology companies.

    "Ben brings to Catabasis extensive senior legal and compliance experience in the pharmaceutical industry, including in product launches, and the transition from pre-commercial to commercial-stage. His deep expertise will be especially valuable in the coming months as we prepare for top-line results from the Phase 3 PolarisDMD trial and the potential commercialization of edasalonexent for the treatment of Duchenne muscular dystrophy," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are delighted that Ben has joined the team and will be leveraging his operating and rare disease experience."

    "I am thrilled to be joining the Catabasis team as we work towards making a new treatment option available to the Duchenne community," said Mr. Harshbarger. "Catabasis is preparing for the potential future transition to a commercial organization, and I am excited to join at such an important time."

    Mr. Harshbarger most recently served as the Interim Chief Executive Officer and General Counsel at Novelion Therapeutics, Inc., the parent company of Aegerion Pharmaceuticals, Inc. Prior to that, he served in several legal roles at Aegerion Pharmaceuticals, Inc., including as Deputy General Counsel, VP, EMEA Legal Counsel, and Acting General Counsel and General Counsel. As Acting General Counsel and General Counsel, he played a critical role in revamping Aegerion's compliance program. Mr. Harshbarger has also held senior legal positions at Cubist Pharmaceuticals, Inc., Viacell, Inc. and Biogen, Inc. Mr. Harshbarger holds his JD from Boston College Law School, and his BA from the University of Richmond.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties related to the impact of the COVID-19 pandemic, uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

    ###

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  15. Top-Line Results Expected in Q4 2020 from Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the first quarter ended March 31, 2020 and reviewed recent business progress.

    "Our fully enrolled Phase 3 PolarisDMD trial for edasalonexent in Duchenne is progressing well and we are continuing to prepare for top-line Phase 3 results in the fourth quarter of this year and a subsequent NDA filing in 2021. We have initial commercialization and supply chain preparations underway and recently strengthened our financial position," said Jill C. Milne, Ph.D., Chief Executive Officer of…

    Top-Line Results Expected in Q4 2020 from Fully Enrolled Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the first quarter ended March 31, 2020 and reviewed recent business progress.

    "Our fully enrolled Phase 3 PolarisDMD trial for edasalonexent in Duchenne is progressing well and we are continuing to prepare for top-line Phase 3 results in the fourth quarter of this year and a subsequent NDA filing in 2021. We have initial commercialization and supply chain preparations underway and recently strengthened our financial position," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "In the current environment, our priorities are focused on the safety of patients as well as maintaining study integrity. Advantages of our Phase 3 trial include that enrollment was completed last year, clinical trial site visits are only every three months, and that patients take the oral study drug at home. Together with our clinical trial sites and consistent with recent regulatory guidance, we have developed contingency plans that we are implementing as needed to enable the continued conduct of the Phase 3 trial as well as the open-label extension GalaxyDMD trial."

    Recent and Upcoming Corporate Highlights

    • The fully enrolled Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) is progressing with top-line results expected in Q4 2020.
      • Patient enrollment was completed in 2019 with 131 boys enrolled across 8 countries.
      • The Phase 3 PolarisDMD trial is intended to support a new drug application (NDA) for commercial registration of edasalonexent in 2021.
      • Contingency plans are in place to enable the continued conduct of our Phase 3 PolarisDMD and GalaxyDMD clinical trials. These plans include the delivery of study drug to patients' homes, increased flexibility in the timing of patient visits, and use of telehealth for remote visits to monitor safety and assess patients where in-person visits are not available.
    • The open-label extension GalaxyDMD trial continues to enroll boys who have completed treatment in the Phase 3 PolarisDMD trial. Their eligible siblings up to age 12 have the option to enroll as well.
    • Catabasis closed a $26.5 million underwritten public offering in February 2020. The proceeds will be used for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations through a potential NDA filing and into Q3 2021.
    • Catabasis plans to commercialize edasalonexent in the US and we are evaluating our commercialization strategy outside of the US. We are encouraged by the favorable feedback we have received from healthcare providers and payors on the potential profile for edasalonexent and the meaningful role it could play as a foundational therapy for all patients with DMD, regardless of mutation.
    • Clinical findings supporting edasalonexent treatment in young boys being associated with age-normative growth and normal adrenal function from the Phase 2 MoveDMD trial and open-label extension were presented at the Muscular Dystrophy Association Virtual Clinical Trials Session by Dr. Erika Finanger, M.D., Division of Neurology at Oregon Health and Science University and a Principal Investigator for the Phase 2 MoveDMD, Phase 3 PolarisDMD, and open-label extension GalaxyDMD trials of edasalonexent in DMD.
    • Catabasis and Duchenne UK entered into a partnership to evaluate edasalonexent in a Phase 2 trial in non-ambulatory DMD patients. Duchenne UK granted Catabasis over $600,000 in funding to support patient and clinical trial site costs. This planned Phase 2 trial is designed to assess safety and pharmacokinetics of edasalonexent and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.

    First Quarter 2020 Financial Results

    Cash Position: As of March 31, 2020, Catabasis had cash, cash equivalents and short-term investments of $55.1 million, compared to $36.2 million as of December 31, 2019. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations through a potential NDA filing and into Q3 2021. Net cash used in operating activities for the three months ended March 31, 2020 was $7.0 million, compared to $6.6 million for the three months ended March 31, 2019.

    R&D Expenses: Research and development expenses were $5.3 million for the three months ended March 31, 2020, compared to $4.2 million for the three months ended March 31, 2019.

    G&A Expenses: General and administrative expenses were $2.8 million for the three months ended March 31, 2020, compared to $2.1 million for the three months ended March 31, 2019.

    Operating Loss: Loss from operations was $8.0 million for the three months ended March 31, 2020, compared to $6.3 million for the three months ended March 31, 2019.

    Net Loss: Net loss was $8.0 million, or $0.50 per share, for the three months ended March 31, 2020, compared to a net loss of $6.0 million, or $0.62 per share, for the three months ended March 31, 2019.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the potential impact of the COVID-19 pandemic on the Company's business and operations, statements about future clinical trial plans including, among other things, statements about the potential commencement of the Company's planned Phase 2 trial in non-ambulatory patients, the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties related to the impact of the COVID-19 pandemic, uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the period ended March 31, 2020, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

    Catabasis Pharmaceuticals, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)

    (Unaudited)

     

     

     

     

     

    Three Months Ended March 31,

     

     

    2020

     

    2019

     
    Operating expenses:
    Research and development

     

    5,289

     

     

    4,197

     

    General and administrative

     

    2,753

     

     

    2,137

     

    Total operating expenses

     

    8,042

     

     

    6,334

     

    Loss from operations

     

    (8,042

    )

     

    (6,334

    )

    Other income (expense):
    Interest and investment income

     

    167

     

     

    226

     

    Other (expense) income, net

     

    (77

    )

     

    70

     

    Total other income, net

     

    90

     

     

    296

     

    Net loss

    $

    (7,952

    )

    $

    (6,038

    )

    Net loss per share - basic and diluted

    $

    (0.50

    )

    $

    (0.62

    )

    Weighted-average common shares outstanding used in net loss per share - basic and diluted

     

    15,898,664

     

     

    9,686,224

     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheets Data

    (In thousands)

    (Unaudited)

     

     

     

     

     

     

     

    March 31,

     

    December 31,

     

     

    2020

     

    2019

    Assets
    Cash and cash equivalents

    $

    13,344

    $

    9,899

    Short-term investments

     

    41,759

     

    26,345

    Right-of-use asset

     

    1,799

     

    2,349

    Other current and long-term assets

     

    2,274

     

    3,187

    Total assets

     

    59,176

     

    41,780

    Liabilities and stockholders' equity
    Current portion of operating lease liabilities

     

    879

     

    1,225

    Long-term portion of operating lease liabilities

     

    875

     

    1,028

    Other current and long-term liabilities

     

    3,711

     

    3,807

    Total liabilities

     

    5,465

     

    6,060

    Total stockholders' equity

    $

    59,176

    $

    41,780

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Statements of Cash Flows Data

    (In thousands)

    (Unaudited)

     

     

     

     

     

    Three Months Ended March 31,

     

     

    2020

     

    2019

    Net cash used in operating activities

    $

    (6,989

    )

    $

    (6,587

    )

    Net cash used in investing activities

     

    (15,432

    )

     

    (17,738

    )

    Net cash provided by financing activities

     

    25,624

     

     

    20,683

     

    Net increase (decrease) in cash, cash equivalents and restricted cash

    $

    3,203

     

    $

    (3,642

    )

     

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  16. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session. The three posters include an analysis of the baseline characteristics of patients enrolled in the Phase 3 PolarisDMD trial, age-normative growth and normal adrenal function seen in the Phase 2 MoveDMD trial and open-label extension, and experience with capsule swallowing in both clinical trials for edasalonexent in boys with DMD. Top-line results from the Phase 3 trial are expected in Q4 2020.

    "As we advance our Phase 3 PolarisDMD…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session. The three posters include an analysis of the baseline characteristics of patients enrolled in the Phase 3 PolarisDMD trial, age-normative growth and normal adrenal function seen in the Phase 2 MoveDMD trial and open-label extension, and experience with capsule swallowing in both clinical trials for edasalonexent in boys with DMD. Top-line results from the Phase 3 trial are expected in Q4 2020.

    "As we advance our Phase 3 PolarisDMD trial, we gain valuable insights into the full potential of edasalonexent," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. "We are pleased to share additional information supporting the design of the Phase 3 PolarisDMD trial and showing long-term safety of edasalonexent. In addition to being well-tolerated in over 100 cumulative years of patient exposure, edasalonexent has shown potential to both preserve muscle function as well as positive cardiac effects. Those living with Duchenne have limited treatment options, and our goal is for edasalonexent to be a new foundational therapy."

    An analysis of the baseline characteristics of the patients enrolled in the Phase 3 PolarisDMD trial was performed compared to the patients enrolled in the previous Phase 2 MoveDMD trial and found overall similar baseline characteristics in the patient populations in the two trials. Both the Phase 3 PolarisDMD trial and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4 to 7 (up to 8th birthday) with any mutation type who had not been on steroids for the previous 6 months. There were no significant differences between the two trials in baseline age, North Star Ambulatory Assessment (NSAA) score and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run). These findings are believed to support the assumptions on which the Phase 3 trial was powered.

    Catabasis also shared long-term safety and tolerability data from the MoveDMD trial and open-label extension. Edasalonexent was associated with age-normative growth, without negative impact on bone health, and normal adrenal function in boys with DMD as it does not impact the glucocorticoid receptor. There was no evidence of adrenal insufficiency for up to 150 weeks of edasalonexent treatment, and no clinically significant changes in cortisol or ACTH. Additionally, edasalonexent treatment in a mdx mouse model of DMD preserved bone length and bone density. In more than 100 years of cumulative patient exposure, edasalonexent has been well-tolerated, with no serious adverse events on treatment, and without the adverse effects associated with high-dose steroids. In both the Phase 2 MoveDMD trial and the ongoing Phase 3 PolarisDMD trial, 97% of 4 to 7 year-old boys with DMD screened for the studies were able to swallow soft-gel study drug capsules. There have been no discontinuations due to capsule burden. This demonstrates that edasalonexent has the potential to be broadly adopted by those affected by DMD and supports the long-term safety and tolerability of edasalonexent.

    Posters from the MDA Virtual Poster Session are available under "Our Science" at www.catabasis.com.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, anticipated timing for top-line results and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-K for the year ended December 31, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  17. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report first quarter 2020 financial results after the Nasdaq Global Market close on Tuesday, May 12, 2020.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report first quarter 2020 financial results after the Nasdaq Global Market close on Tuesday, May 12, 2020.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  18. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session later this month.

    During the Virtual Poster Session, Catabasis and collaborators will present three posters entitled:

    • Baseline Characteristics of Patients Enrolled in PolarisDMD, a Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy. Richard S. Finkel; Craig McDonald; H. Lee Sweeney; Kathryn Wagner; Erin Neil; Maria Mancini; James MacDougall; Joanne M. Donovan.
    • Edasalonexent Treatment in Young Boys with Duchenne

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session later this month.

    During the Virtual Poster Session, Catabasis and collaborators will present three posters entitled:

    • Baseline Characteristics of Patients Enrolled in PolarisDMD, a Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy. Richard S. Finkel; Craig McDonald; H. Lee Sweeney; Kathryn Wagner; Erin Neil; Maria Mancini; James MacDougall; Joanne M. Donovan.
    • Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy is Associated with Age-Normative Growth and Normal Adrenal Function. Erika L. Finanger; Richard S. Finkel; Gihan I. Tennekoon; Krista Vandenborne; H. Lee Sweeney; Perry Shieh; Sabrina W. Yum; Maria Mancini; James MacDougall; Joanne M. Donovan.
    • Experience with Edasalonexent Demonstrates Ability of 4 to 7 Year Old Boys with Duchenne Muscular Dystrophy to Take Soft-gel Capsules in Trials. Maria Mancini; Gigi Shafai; Joanne M. Donovan; Sachin Chandran; Richard S. Finkel.

    The poster session will be available to registered conference attendees and will be made available under "Our Science" on the company website at www.catabasis.com.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  19. NEW YORK, NY / ACCESSWIRE / April 6, 2020 / Solebury Trout, a boutique investor relations and corporate communications firm, announced today that it will be hosting a fourth day of presentations as part of a Virtual Conference Series featuring leading companies from the life sciences industry. At the event, taking place on April 7, 2020, management teams will present a business overview and highlights using an online, digital meeting format.

    The April 7 schedule follows, with presentations available at https://78449.themediaframe.com/dataconf/productusers/solebury/mediaframe/36850/indexl.html:

    10:00 a.m. EST, Ascentage Pharma (6855-HK)
    10:30 a.m. EST, Maxcyte (MXCT-GB)
    11:00 a.m. EST, CASI Pharmaceuticals (CASI)
    11:30 a.m. EST, BioXcel Therapeutics…

    NEW YORK, NY / ACCESSWIRE / April 6, 2020 / Solebury Trout, a boutique investor relations and corporate communications firm, announced today that it will be hosting a fourth day of presentations as part of a Virtual Conference Series featuring leading companies from the life sciences industry. At the event, taking place on April 7, 2020, management teams will present a business overview and highlights using an online, digital meeting format.

    The April 7 schedule follows, with presentations available at https://78449.themediaframe.com/dataconf/productusers/solebury/mediaframe/36850/indexl.html:

    10:00 a.m. EST, Ascentage Pharma (6855-HK)
    10:30 a.m. EST, Maxcyte (MXCT-GB)
    11:00 a.m. EST, CASI Pharmaceuticals (CASI)
    11:30 a.m. EST, BioXcel Therapeutics (BTAI)
    12:00 p.m. EST, Pharming Group NV (PHARM-NL)
    12:30 p.m. EST, I-Mab Biopharma (IMAB)
    1:00 p.m. EST, Incysus Therapeutics (private)
    1:30 p.m. EST, Engage Therapeutics (private)
    2:00 p.m. EST, NervGen Pharma (NGEN-CA)
    2:30 p.m. EST, Camallergy (private)
    3:00 p.m. EST, Yiviva (private)

    Investors and others can register and access the conference at the Solebury Trout Virtual Global Healthcare Conference Series website: https://troutaccess.com/investor.php/c/Solebury_Trout_Virtual_Global_Healthcare_Conference.

    Companies that have presented at prior days of the conference series on March 26, March 31, and April 2, 2020, have included:

    Adrenomed (private), Affimed (NASDAQ:AFMD), AM-Pharma (private), ANTEV Limited (private), Apexigen (private), Aptorum Group (NASDAQ:APM), BerGenBio (BGBIO-NO), Catabasis Pharmaceuticals (NASDAQ:CATB), CerSci Therapeutics (private), ESSA Pharma (NASDAQ:EPIX), Evofem Biosciences (NASDAQ:EVFM), Faraday Pharma (private), Forbius (private), HaemaLogiX (private), Impact Therapeutics (private), Junshi Biosciences (1877-HK), Kineta (private), Landos Biopharma (private), Lineage Cell Therapeutics (NYSE:LCTX), NeuroRx (private), Neximmune (private), NorthSea Therapeutics (private), OnKure Therapeutics (private), Paion (PA8-DE), Paradigm Biopharma (PAR-AU), PharmaMar (PHM-SE), Pieris Pharmaceuticals (NASDAQ:PIRS). Ryvu Therapeutics (RVU-PL), Sol-Gel Technologies (NASDAQ:SLGL), Targovax (TRVX-NO), Teon Therapeutics (private), and Vaccitech Limited (private).

    More information on the event, including archived presentations when available and a list of presenters and companies potentially available for engagement with the investment community, can be found at www.troutaccess.com.

    About Solebury Trout

    Solebury Trout is a well-established Investor Relations and Corporate Communications firm. For more than 20 years, Solebury Trout has partnered with private and public companies across all sectors, offering fully outsourced investor relations, perception surveys, environmental, social and governance (ESG) evaluations, C-suite visibility programs, content development, media relations, digital and social media communications, crisis preparation and media and presentation training.

    Solebury Trout is affiliated with Solebury Capital and is a subsidiary of The PNC Financial Services Group, Inc. Solebury Capital a premier equity capital markets advisory firm providing management teams and owners with independent advice and judgment honed in decades of experience in capital markets at leading investment firms across the globe. Since its founding in 2005, Solebury Capital has advised on over 700 successful transactions representing nearly $300B in proceeds.

    Contact:

    Thomas Hoffmann
    646-378-2931

    Disclaimer

    "Solebury" is a marketing name for Solebury Trout LLC and Solebury Capital LLC. The services described herein may be performed by a combination of Solebury Trout LLC or Solebury Capital LLC. Solebury deal team members may be employees of either entity. Solebury is an affiliate of The PNC Financial Services Group, Inc., a diversified financial services company. Equity capital markets advisory and related services are provided by Solebury Capital LLC. Solebury Capital LLC, is a registered broker-dealer and member of FINRA and SIPC. This material does not constitute an offer to sell or a solicitation of an offer to buy any investment product or security.

    SOURCE: Solebury Trout



    View source version on accesswire.com:
    https://www.accesswire.com/584081/Solebury-Trout-Announces-Additional-Virtual-Conference-Presentations-from-Life-Science-Companies-on-April-7-2020

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  20. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, plans to present a corporate overview and provide a business update at the upcoming Solebury Trout Virtual Investor Conference on Tuesday, March 31, 2020 at 12:30pm EDT.

    A webcast of the presentation will be available in the investors section of the Company's website www.catabasis.com, and will be archived for 30 days following the presentation.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Jill C. Milne, Ph.D., Chief Executive Officer, plans to present a corporate overview and provide a business update at the upcoming Solebury Trout Virtual Investor Conference on Tuesday, March 31, 2020 at 12:30pm EDT.

    A webcast of the presentation will be available in the investors section of the Company's website www.catabasis.com, and will be archived for 30 days following the presentation.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    View Full Article Hide Full Article
  21. NEW YORK, NY / ACCESSWIRE / March 24, 2020 / Solebury Trout, a boutique investor relations and corporate communications firm, announced today that it will be hosting a Virtual Conference Series on March 26, March 31, and April 2, 2020, featuring leading companies from the life sciences industry. At the event, management teams will present a business overview and highlights using an online, digital meeting format.

    Investors and others can register at the Solebury Trout Virtual Global Healthcare Conference Series homepage to access the conference and presentation schedule.

    "We are pleased to introduce a virtual meeting platform to highlight the progress and potential of leading life sciences companies," commented Jonathan Fassberg, Co-CEO of Solebury…

    NEW YORK, NY / ACCESSWIRE / March 24, 2020 / Solebury Trout, a boutique investor relations and corporate communications firm, announced today that it will be hosting a Virtual Conference Series on March 26, March 31, and April 2, 2020, featuring leading companies from the life sciences industry. At the event, management teams will present a business overview and highlights using an online, digital meeting format.

    Investors and others can register at the Solebury Trout Virtual Global Healthcare Conference Series homepage to access the conference and presentation schedule.

    "We are pleased to introduce a virtual meeting platform to highlight the progress and potential of leading life sciences companies," commented Jonathan Fassberg, Co-CEO of Solebury Trout. "This platform enables investors to continue to have direct access to the many companies in our industry advancing innovative, life-saving technologies and products worldwide."

    The companies to present at the conference series include:
    Adrenomed (private), Affimed (NASDAQ:AFMD), AM-Pharma (private), ANTEV Limited (private), Apexigen (private), Aptorum Group (NASDAQ:APM), BerGenBio (BGBIO-NO), Catabasis Pharmaceuticals (NASDAQ:CATB), CerSci Therapeutics (private), ESSA Pharma (NASDAQ:EPIX), Evofem Biosciences (NASDAQ:EVFM), Faraday Pharma (private), Forbius (private), HaemaLogiX (private), Impact Therapeutics (private), Junshi Biosciences (1877-HK), Kineta (private), Landos Biopharma (private), Lineage Cell Therapeutics (NYSE:LCTX), NeuroRx (private), Neximmune (private), NorthSea Therapeutics (private), OnKure Therapeutics (private), Paion (PA8-DE), Paradigm Biopharma (PAR-AU), PharmaMar (PHM-SE), Pieris Pharmaceuticals (NASDAQ:PIRS). Ryvu Therapeutics (RVU-PL), Sol-Gel Technologies (NASDAQ:SLGL), Targovax (TRVX-NO), Teon Therapeutics (private), and Vaccitech Limited (private).

    More information on the event, including a list of presenters and companies potentially available for engagement with the investment community, can be found at www.troutaccess.com.

    About Solebury Trout

    Solebury Trout is a well-established Investor Relations and Corporate Communications firm. For more than 20 years, Solebury Trout has partnered with private and public companies across all sectors, offering fully outsourced investor relations, perception surveys, environmental, social and governance (ESG) evaluations, C-suite visibility programs, content development, media relations, digital and social media communications, crisis preparation and media and presentation training.

    Solebury Trout is affiliated with Solebury Capital and is a subsidiary of The PNC Financial Services Group, Inc. Solebury Capital a premier equity capital markets advisory firm providing management teams and owners with independent advice and judgment honed in decades of experience in capital markets at leading investment firms across the globe. Since its founding in 2005, Solebury Capital has advised on over 700 successful transactions representing nearly $300B in proceeds.

    Contact:

    Thomas Hoffmann

    Disclaimer

    "Solebury" is a marketing name for Solebury Trout LLC and Solebury Capital LLC. The services described herein may be performed by a combination of Solebury Trout LLC or Solebury Capital LLC. Solebury deal team members may be employees of either entity. Solebury is an affiliate of The PNC Financial Services Group, Inc., a diversified financial services company. Equity capital markets advisory and related services are provided by Solebury Capital LLC. Solebury Capital LLC, is a registered broker-dealer and member of FINRA and SIPC. This material does not constitute an offer to sell or a solicitation of an offer to buy any investment product or security.

    SOURCE: Solebury Trout

    View source version on accesswire.com:
    https://www.accesswire.com/582334/Solebury-Trout-Announces-Virtual-Conference-Series-with-Presentations-from-Life-Science-Companies

    View Full Article Hide Full Article
  22. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) during the Muscular Dystrophy Association (MDA) Virtual Clinical Trials Session on Tuesday, March 24, 2020.

    Erika L. Finanger, MD, Division of Neurology at Oregon Health and Science University and a Principal Investigator for the Phase 2 MoveDMD, Phase 3 PolarisDMD, and open-label extension GalaxyDMD trials, will give an oral presentation titled "Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy Is Associated with Age-Normative Growth and Normal Adrenal Function" during the Clinical…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) during the Muscular Dystrophy Association (MDA) Virtual Clinical Trials Session on Tuesday, March 24, 2020.

    Erika L. Finanger, MD, Division of Neurology at Oregon Health and Science University and a Principal Investigator for the Phase 2 MoveDMD, Phase 3 PolarisDMD, and open-label extension GalaxyDMD trials, will give an oral presentation titled "Edasalonexent Treatment in Young Boys with Duchenne Muscular Dystrophy Is Associated with Age-Normative Growth and Normal Adrenal Function" during the Clinical Trial Presentations session from 11am to 4pm EDT on Tuesday, March 24, 2020. The webcast will be available to registered conference attendees and presented material will be made available on the company website after the session at www.catabasis.com.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  23. -- Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy Top-Line Results Expected in Q4 2020 --

    -- Conference Call and Webcast Today at 8:30am ET --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the fourth quarter and full year ended December 31, 2019 and reviewed recent business progress.

    "We made excellent progress with our edasalonexent program in 2019 and are in a great place coming into 2020 with the Phase 3 PolarisDMD trial in Duchenne fully enrolled. We also strengthened our financial position. We are preparing for top-line Phase 3 results in the fourth quarter and a subsequent NDA filing in 2021 and have initial commercialization…

    -- Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy Top-Line Results Expected in Q4 2020 --

    -- Conference Call and Webcast Today at 8:30am ET --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the fourth quarter and full year ended December 31, 2019 and reviewed recent business progress.

    "We made excellent progress with our edasalonexent program in 2019 and are in a great place coming into 2020 with the Phase 3 PolarisDMD trial in Duchenne fully enrolled. We also strengthened our financial position. We are preparing for top-line Phase 3 results in the fourth quarter and a subsequent NDA filing in 2021 and have initial commercialization and supply chain preparations underway," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are also building for the future with our partnership with Duchenne UK to explore edasalonexent in non-ambulatory Duchenne patients and our ongoing preclinical research to explore the potential for broad benefits of edasalonexent in Duchenne and also in additional neuromuscular diseases."

    Recent and Upcoming Corporate Highlights

    • Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) on track to report top-line results in Q4 2020.
      • Patient enrollment is complete and has exceeded the enrollment target with 131 boys enrolled. Patients are enrolled across all 8 countries where the trial is active.
      • The Phase 3 trial is intended to support a new drug application (NDA) for commercial registration of edasalonexent in 2021.
    • An analysis of the baseline characteristics of the patients enrolled in the Phase 3 PolarisDMD trial was performed compared to the patients enrolled in the previous Phase 2 MoveDMD trial and found overall similar characteristics in the patient populations in the two trials.
      • Both the Phase 3 PolarisDMD trial and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4 to 7 (up to 8th birthday) with any mutation type who had not been on steroids for the previous 6 months.
      • Baseline age, North Star Ambulatory Assessment (NSAA) score and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run) were similar in both the Phase 3 and Phase 2 trials and there were no significant differences in these baseline characteristics between the two trials.
      • These findings are believed to support the assumptions on which the Phase 3 trial was powered.
    • Catabasis and Duchenne UK entered into a partnership to evaluate edasalonexent in a Phase 2 trial in non-ambulatory DMD patients. Duchenne UK granted Catabasis over $600,000 in funding to support patient and clinical trial site costs. This exploratory Phase 2 trial is planned to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.
    • Catabasis closed a $26.5 million underwritten public offering in February 2020. The proceeds will be used for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations through a potential NDA filing and into Q3 2021.

    Fourth Quarter and Full Year 2019 Financial Results

    Cash Position: As of December 31, 2019, Catabasis had cash, cash equivalents and short-term investments of $36.2 million, compared to $40.6 million as of September 30, 2019. Following December 31, 2019, Catabasis raised an additional $27.6 million from equity financings for $25.6 million in net proceeds. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations through a potential NDA filing and into Q3 2021. Net cash used in operating activities for the three months ended December 31, 2019 was $7.8 million, compared to $5.3 million for the three months ended December 31, 2018. Net cash used in operating activities for the full year 2019 was $26.6 million, compared to $23.5 million for the full year 2018.

    R&D Expenses: Research and development expenses were $4.3 million for the three months ended December 31, 2019, compared to $3.7 million for the three months ended December 31, 2018 and $18.3 million for the full year 2019, compared to $17.0 million for the full year 2018.

    G&A Expenses: General and administrative expenses were $2.5 million for the three months ended December 31, 2019, compared to $2.4 million for the three months ended December 31, 2018 and $8.8 million for the full year 2019, compared to $9.3 million for the full year 2018.

    Operating Loss: Loss from operations was $6.7 million for the three months ended December 31, 2019, compared to $6.1 million for the three months ended December 31, 2018 and $27.1 million for the full year 2019, compared to $26.4 million for the full year 2018.

    Net Loss: Net loss was $6.6 million, or $0.55 per share, for the three months ended December 31, 2019, compared to a net loss of $6.1 million, or $0.85 per share, for the three months ended December 31, 2018 and $26.3 million for the full year 2019, compared to $25.9 million for the full year 2018.

    Conference Call and Webcast

    Catabasis will host a conference call and webcast at 8:30am ET today to provide an update on corporate developments and to discuss fourth quarter and full year 2019 financial results.

     

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    3621269

     

    Please specify to the operator that you would like to join the "Catabasis Fourth Quarter and Full Year 2019 Results Call."

    Interested parties may access a live audio webcast of the conference call via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the potential commencement of the Company's planned Phase 2 trial in non-ambulatory patients, the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-K for the year ended December 31, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

     

    Catabasis Pharmaceuticals, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)

    (Audited)

     
    Year Ended December 31,

    2019

    2018

     
    Operating expenses:
    Research and development

    18,317

    17,042

    General and administrative

    8,771

    9,329

    Total operating expenses

    27,088

    26,371

    Loss from operations

    (27,088)

    (26,371)

    Other income (expense):
    Interest expense

    -

    (100)

    Interest and investment income

    845

    425

    Other (expense) income, net

    (50)

    176

    Total other income, net

    795

    501

    Net loss

    $ (26,293)

    $ (25,870)

    Net loss per share - basic and diluted

    $ (2.35)

    $ (5.12)

    Weighted-average common shares outstanding used in net loss per share - basic and diluted

    11,199,057

    5,054,823

     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheets Data

    (In thousands)

    (Audited)

     
    December 31, December 31,

    2019

    2018

    Assets
    Cash and cash equivalents

    $ 9,899

    $ 15,294

    Short-term investments

    26,345

    22,276

    Right-of-use asset

    2,349

    -

    Other current and long-term assets

    3,187

    1,599

    Total assets

    41,780

    39,169

    Liabilities and stockholders' equity
    Current portion of operating lease liabilities

    1,225

    -

    Long-term portion of operating lease liabilities

    1,028

    -

    Other current and long-term liabilities

    3,807

    4,227

    Total liabilities

    6,060

    4,227

    Total stockholders' equity

    $ 35,720

    $ 34,942

     
     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Statements of Cash Flows Data

    (In thousands)

    (Audited)

     
    Year Ended December 31,

    2019

    2018

    Net cash used in operating activities

    $

    (26,569

    )

    $

    (23,465

    )

    Net cash used in investing activities

     

    (4,082

    )

     

    (21,905

    )

    Net cash provided by financing activities

     

    25,620

     

     

    44,295

     

    Net decrease in cash, cash equivalents and restricted cash

    $

    (5,031

    )

    $

    (1,075

    )

     

     

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  24. The company will host a conference call at 8:30am ET

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report fourth quarter and full year 2019 financial results before the Nasdaq Global Market open on Tuesday, March 10, 2020. Jill C. Milne, Chief Executive Officer, will host a conference call and webcast at 8:30am ET to provide an update on corporate developments and to discuss fourth quarter and last year's financial results.

    Conference Call Dial-In Information:

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    3621269

    Please specify to the operator that you would like to join the "Catabasis…

    The company will host a conference call at 8:30am ET

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report fourth quarter and full year 2019 financial results before the Nasdaq Global Market open on Tuesday, March 10, 2020. Jill C. Milne, Chief Executive Officer, will host a conference call and webcast at 8:30am ET to provide an update on corporate developments and to discuss fourth quarter and last year's financial results.

    Conference Call Dial-In Information:

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    3621269

    Please specify to the operator that you would like to join the "Catabasis Fourth Quarter and Full Year 2019 Results Call."

    Interested parties may access a live audio webcast of the conference via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

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  25. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy at the XVIII International Conference on Duchenne and Becker Muscular Dystrophy, organized by Parent Project, in Rome, Italy.

    Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis Pharmaceuticals will give a presentation titled "Edasalonexent, an oral NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy, regardless of mutation" on Sunday, March 1st at 12:30pm CET.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it will present the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy at the XVIII International Conference on Duchenne and Becker Muscular Dystrophy, organized by Parent Project, in Rome, Italy.

    Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis Pharmaceuticals will give a presentation titled "Edasalonexent, an oral NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy, regardless of mutation" on Sunday, March 1st at 12:30pm CET.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes and the GalaxyDMD open-label extension trial, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-Q for the year ended September 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  26. Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the closing of its previously announced underwritten public offering of an aggregate of 5,290,000 shares of common stock at a public offering price of $5.00 per share, including 690,000 shares issued upon the exercise in full by the underwriter of its over-allotment option.

    Oppenheimer & Co. Inc. acted as sole underwriter for the offering.

    The gross proceeds of the offering are expected to be approximately $26.5 million, prior to deducting the underwriting discount and other estimated offering expenses.

    Catabasis intends to use the net proceeds from the offering for clinical trial…

    Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the closing of its previously announced underwritten public offering of an aggregate of 5,290,000 shares of common stock at a public offering price of $5.00 per share, including 690,000 shares issued upon the exercise in full by the underwriter of its over-allotment option.

    Oppenheimer & Co. Inc. acted as sole underwriter for the offering.

    The gross proceeds of the offering are expected to be approximately $26.5 million, prior to deducting the underwriting discount and other estimated offering expenses.

    Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes.

    The securities described above were offered pursuant to a prospectus supplement and an accompanying base prospectus forming part of a shelf registration statement on Form S-3 (File No. 333-231441), which was declared effective by the Securities and Exchange Commission ("SEC") on May 23, 2019. A final prospectus supplement relating to the offering was filed with the SEC on January 30, 2020 and is available on the SEC's website located at http://www.sec.gov. Copies of the final prospectus supplement and the accompanying base prospectus may be obtained for free by contacting Oppenheimer & Co. Inc. Attention: Syndicate Prospectus Department, 85 Broad St., 26th Floor, New York, NY 10004, by telephone at (212) 667-8055 or by email at .

    This press release does not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the anticipated use of net proceeds from the offering, among other things, statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties associated with the Company's intended use of proceeds, as well as uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the prospectus supplement and the accompanying base prospectus to the Company's Registration Statement on Form S-3 relating to this offering, and the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, each of which is on file with the SEC, and in other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  27. Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the pricing of an underwritten public offering of an aggregate of 4,600,000 shares of common stock at a public offering price of $5.00 per share. The offering is expected to close on or about February 3, 2020, subject to customary closing conditions. Catabasis has granted the underwriters a 30-day option to purchase up to 690,000 additional shares of common stock at the public offering price, less the underwriting discount, to cover over-allotments, if any.

    Oppenheimer & Co. Inc. is acting as sole underwriter for the offering.

    The gross proceeds of the offering are expected to be approximately…

    Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the pricing of an underwritten public offering of an aggregate of 4,600,000 shares of common stock at a public offering price of $5.00 per share. The offering is expected to close on or about February 3, 2020, subject to customary closing conditions. Catabasis has granted the underwriters a 30-day option to purchase up to 690,000 additional shares of common stock at the public offering price, less the underwriting discount, to cover over-allotments, if any.

    Oppenheimer & Co. Inc. is acting as sole underwriter for the offering.

    The gross proceeds of the offering are expected to be approximately $23 million, prior to deducting the underwriting discount and other estimated offering expenses.

    Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes.

    The securities described above are being offered pursuant to a prospectus supplement and an accompanying base prospectus forming part of a shelf registration statement on Form S-3 (File No. 333-231441), which was declared effective by the Securities and Exchange Commission ("SEC") on May 23, 2019. A final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC's website located at http://www.sec.gov. When available, copies of the final prospectus supplement and the accompanying base prospectus may be obtained for free by contacting Oppenheimer & Co. Inc. Attention: Syndicate Prospectus Department, 85 Broad St., 26th Floor, New York, NY 10004, by telephone at (212) 667-8055 or by email at .

    This press release does not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the anticipated completion, timing and size of the public offering and the anticipated use of net proceeds therefrom, among other things, statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks and uncertainties associated with market conditions, the satisfaction of customary closing conditions relating to the public offering and the Company's intended use of proceeds, as well as uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the prospectus supplement and the accompanying base prospectus to the Company's Registration Statement on Form S-3 relating to this offering, which will be filed with the SEC, and the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, which is on file with the SEC, and in other filings that the Company may make with the SEC in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  28. Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it intends to offer its securities in an underwritten public offering. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Oppenheimer & Co. Inc. is acting as sole underwriter for the offering.

    Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes.

    The securities…

    Catabasis Pharmaceuticals, Inc. ("Catabasis," the "Company," "we," "our," or "us") (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it intends to offer its securities in an underwritten public offering. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

    Oppenheimer & Co. Inc. is acting as sole underwriter for the offering.

    Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital and other general corporate purposes.

    The securities described above will be offered pursuant to a shelf registration statement on Form S-3 (No. 333-231441), which was previously filed with and declared effective by the Securities and Exchange Commission ("SEC") on May 23, 2019. A preliminary prospectus supplement and accompanying base prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC's website located at http://www.sec.gov, copies of which may be obtained, when available, from Oppenheimer & Co. Inc. Attention: Syndicate Prospectus Department, 85 Broad St., 26th Floor, New York, NY 10004, by telephone at (212) 667-8055 or by email at . The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release does not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the anticipated public offering and the anticipated use of net proceeds therefrom, among other things, statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The offering is subject to market and other conditions and there can be no assurance as to whether or when the offering may be completed or as to the actual size or terms of the offering. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: market conditions that may affect the timing, terms and conditions of the offering, the Company's ability to satisfy the conditions to closing of the offering and other matters affecting our ability to consummate the offering on terms acceptable to us. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  29. -- Duchenne UK Grants Over $600,000 in Funding to Support Patient and Clinical Trial Site Costs --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), have entered into a partnership for a Phase 2 trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory DMD patients. This exploratory Phase 2 trial, which is subject to the receipt of adequate funding, is designed to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.

    "We are thrilled to announce plans to expand our…

    -- Duchenne UK Grants Over $600,000 in Funding to Support Patient and Clinical Trial Site Costs --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), have entered into a partnership for a Phase 2 trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory DMD patients. This exploratory Phase 2 trial, which is subject to the receipt of adequate funding, is designed to assess safety, pharmacokinetics and exploratory measures of function including cardiac, skeletal muscle and pulmonary function in non-ambulatory DMD patients.

    "We are thrilled to announce plans to expand our knowledge of edasalonexent to non-ambulatory boys and men affected by Duchenne. We recognize the urgent need for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function by benefitting both skeletal muscle as well as cardiac function," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. "We are incredibly fortunate to have the opportunity to partner with Duchenne UK for this important work and appreciate their deep commitment as we work together to bring treatment options to all patients."

    "We first approached Catabasis last year to ask if we could encourage them to advance a trial to look at the non-ambulant patient population and we are delighted to be able to announce this collaboration today," said Emily Crossley and Alex Johnson, Duchenne UK cofounders. "Duchenne UK is committed to developing medicines for all boys and men with DMD, regardless of their physical stage, mutation or age. This trial will represent an important step in that direction."

    The Phase 2 trial is designed to be a one-year, randomized, double-blind, placebo-controlled trial evaluating safety, pharmacokinetics and exploratory measures of function with edasalonexent in non-ambulatory boys and men affected by DMD. The trial expects to enroll approximately 16 non-ambulatory patients ages 10 and older regardless of mutation type who have not been on steroids for at least 6 months at clinical trial sites in the United Kingdom. The exploratory functional endpoints are anticipated to include assessments of cardiac function, upper limb skeletal muscle function and pulmonary function. In addition, the trial is also expected to explore patient reported outcomes. The intention is that upon completing this trial, patients will have the option to transition to the GalaxyDMD open-label extension trial and receive edasalonexent.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the potential commencement of the Company's planned Phase 2 trial in non-ambulatory patients, the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes and the GalaxyDMD open-label extension trial, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-Q for the year ended September 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  30. -- Analysis Shows Patients Enrolled in Phase 2 MoveDMD and Phase 3 PolarisDMD Trials of Edasalonexent in Duchenne Muscular Dystrophy Have Similar Baseline Characteristics --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the results from an analysis of the baseline characteristics of the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The analysis shows that overall the patients enrolled in the Phase 3 trial have similar characteristics to the patients that enrolled in the previous Phase 2 MoveDMD trial. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing…

    -- Analysis Shows Patients Enrolled in Phase 2 MoveDMD and Phase 3 PolarisDMD Trials of Edasalonexent in Duchenne Muscular Dystrophy Have Similar Baseline Characteristics --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the results from an analysis of the baseline characteristics of the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The analysis shows that overall the patients enrolled in the Phase 3 trial have similar characteristics to the patients that enrolled in the previous Phase 2 MoveDMD trial. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing in 2021.

    Both the Phase 3 PolarisDMD trial and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4 to 7 (up to 8th birthday) with any mutation type whom had not been on steroids for the previous 6 months. The Phase 3 trial enrolled 131 boys at 37 sites in the United States, Canada, Europe, Israel and Australia and 98% were steroid-naïve. The Phase 2 trial enrolled 31 boys in the United States, all of whom were steroid-naïve. A comparison was made between the populations at the baseline of each trial. Baseline age, North Star Ambulatory Assessment (NSAA) score and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run) were similar in both trials; there were no significant differences in these baseline characteristics between the two trials. Distribution of baseline NSAA and timed function tests was less variable in the Phase 3 trial than in the Phase 2 trial. These findings support the assumptions on which the Phase 3 trial was powered. Boys in the Phase 3 trial had an elevated heart rate and elevated muscle enzyme levels at baseline, which was also consistent with the Phase 2 trial population.

    Mean ± sd

    PolarisDMD

    (n=131)

    MoveDMD
    (n=23)
    Age (years) 5.7 ± 1.0 6.0 ± 1.1
    Percent enrolled patients that had not taken steroids

    98%

    100%

    NSAA score 20.8 ± 4.7 20.1 ± 5.5
    10-meter walk/run speed (1/s) 0.181 ± 0.037 0.168 ± 0.045
    4-stair climb speed (1/s) 0.265 ± 0.097 0.254 ± 0.110
    Time to stand speed (1/s) 0.212 ± 0.070 0.193 ± 0.080

    "We are pleased to see that as expected, the patient population enrolled in our Phase 3 PolarisDMD trial is consistent with the patient population enrolled in the Phase 2 MoveDMD trial, which supports our design for the Phase 3 trial. The overwhelming positive interest from physicians and families and the rapid enrollment of the trial reinforces the strong demand for a well-tolerated treatment like edasalonexent with the potential to slow disease progression and preserve muscle function," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. "We look forward to sharing results from the trial next year and are dedicated to bringing hope and life changing therapies to patients and their families."

    "The baseline age and functional abilities of the boys enrolled in the Phase 3 trial confirm that we have enrolled the expected patient population and also that this patient population is similar to published natural history studies of boys in this age range who are not on steroids," said Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System and a Principal Investigator for the Phase 2 and Phase 3 trials with edasalonexent. "There is a clear need for a therapy that could benefit all patients affected by Duchenne, regardless of mutation type, by slowing disease progression while being well tolerated. I am pleased to be participating in this important Phase 3 trial of edasalonexent."

    In the earlier MoveDMD trial and open-label extension, edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in the off-treatment control period, significantly improved biomarkers of muscle health and inflammation and was well-tolerated. In more than 60 cumulative patient years of exposure, the majority of adverse events were mild in nature, and the most common treatment-related adverse event was diarrhea, generally mild and transient. There were no serious adverse events observed on treatment, and no adverse trends in chemistry, hematology, or measures of adrenal function.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Phase 3 PolarisDMD Trial

    The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of edasalonexent in patients with DMD. The trial enrolled patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who had not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen were also eligible to enroll. The primary efficacy endpoint is change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints include the age-appropriate timed function tests: time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also included as important potential areas of differentiation. For each boy that receives placebo, two boys are receiving 100 mg/kg/day of edasalonexent and after 12 months, all boys are expected to receive edasalonexent in the open-label extension study GalaxyDMD. The PolarisDMD trial design was informed by discussions with regulators as well as input from treating physicians, patient organizations and families of boys affected by Duchenne. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020. More information about the Phase 3 PolarisDMD clinical trial is available on clinicaltrials.gov.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes and the open-label extension trial GalaxyDMD, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-Q for the year ended September 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  31. -- Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy Fully Enrolled with Top-Line Results Expected in Q4 2020 --

    -- Conference Call and Webcast Today at 8:30am ET --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the third quarter ended September 30, 2019 and reviewed recent business progress.

    "We have made significant clinical progress with our lead program edasalonexent, a potential foundational therapy for the treatment of Duchenne muscular dystrophy. We are excited that enrollment is complete and target enrollment was exceeded for our Phase 3 PolarisDMD trial of edasalonexent. We very much appreciate the strong interest from…

    -- Edasalonexent Global Phase 3 PolarisDMD Trial in Duchenne Muscular Dystrophy Fully Enrolled with Top-Line Results Expected in Q4 2020 --

    -- Conference Call and Webcast Today at 8:30am ET --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported financial results for the third quarter ended September 30, 2019 and reviewed recent business progress.

    "We have made significant clinical progress with our lead program edasalonexent, a potential foundational therapy for the treatment of Duchenne muscular dystrophy. We are excited that enrollment is complete and target enrollment was exceeded for our Phase 3 PolarisDMD trial of edasalonexent. We very much appreciate the strong interest from physicians and the Duchenne community that propelled trial enrollment. We look forward to reporting top-line results in the fourth quarter of next year," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "In preparation for the potential commercialization of edasalonexent, we strengthened our executive team with the addition of Andrew Komjathy as Chief Commercial Officer."

    Recent and Upcoming Corporate Highlights

    • Phase 3 PolarisDMD of edasalonexent in Duchenne muscular dystrophy (DMD)
      • Trial enrollment is complete and has exceeded the enrollment target with 131 boys enrolled. Patients are enrolled across all 8 countries where the trial is active.
      • Top-line results from the Phase 3 PolarisDMD trial are expected in Q4 of 2020, and the trial is intended to support an application for commercial registration of edasalonexent in 2021.
    • Clinical findings supporting edasalonexent as a potential foundational therapy for the treatment of DMD from the Phase 2 MoveDMD trial and open-label extension were presented at the 24th International Annual Congress of the World Muscle Society by Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the MoveDMD and Phase 3 PolarisDMD trials of edasalonexent in DMD. Edasalonexent slowed disease progression compared to the off-treatment control period and was well tolerated through 60 cumulative years of patient exposure in boys affected by DMD.
    • Catabasis named Andrew A. Komjathy as its Chief Commercial Officer, adding deep rare disease commercial experience to the executive team.
    • Catabasis and the Jain Foundation announced a preclinical research collaboration to study edasalonexent in Dysferlinopathy, which includes Limb-girdle muscular dystrophy type 2B and Miyoshi myopathy. Edasalonexent has the potential to benefit patients with other diseases beyond DMD, such as Dysferlinopathy.

    Third Quarter 2019 Financial Results

    Cash Position: As of September 30, 2019, Catabasis had cash, cash equivalents and short-term investments of $40.6 million, compared to $46.1 million as of June 30, 2019. Based on the Company's current operating plan, Catabasis expects that it has sufficient cash to fund operations beyond top-line Phase 3 results and through 2020. Net cash used in operating activities for the three months ended September 30, 2019 was $6.5 million, compared to $5.8 million for the three months ended September 30, 2018.

    R&D Expenses: Research and development expenses were $4.7 million for the three months ended September 30, 2019, compared to $3.9 million for the three months ended September 30, 2018.

    G&A Expenses: General and administrative expenses were $2.0 million for the three months ended September 30, 2019, compared to $2.1 million for the three months ended September 30, 2018.

    Operating Loss: Loss from operations was $6.7 million for the three months ended September 30, 2019, compared to $6.0 million for the three months ended September 30, 2018.

    Net Loss: Net loss was $6.5 million, or $0.56 per share, for the three months ended September 30, 2019, compared to a net loss of $5.7 million, or $0.80 per share, for the three months ended September 30, 2018.

    Conference Call and Webcast

    Catabasis will host a conference call and webcast at 8:30am ET today to provide an update on corporate developments and to discuss third quarter 2019 financial results.

     

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    4740699

     

    Please specify to the operator that you would like to join the "Catabasis Third Quarter 2019 Results Call."

    Interested parties may access a live audio webcast of the conference call via the investor section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes and the open-label extension trial GalaxyDMD, including the anticipated timing for top-line results, the potential timing for the filing of an NDA, the Company's cash expectations, the Company's planned transition to a commercial-stage organization and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-Q for the year ended September 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

     

    Catabasis Pharmaceuticals, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)

    (Unaudited)

     
    Three Months Ended September 30, Nine Months Ended September 30,

    2019

    2018

    2019

    2018

     
    Operating expenses:
    Research and development

     

    4,697

     

    3,897

     

    14,054

     

    13,383

    General and administrative

     

    1,985

     

    2,111

     

    6,287

     

    6,900

    Total operating expenses

     

    6,682

     

    6,008

     

    20,341

     

    20,283

    Loss from operations

     

    (6,682)

     

    (6,008)

     

    (20,341)

     

    (20,283)

    Other income (expense):
    Interest expense

     

    -

     

    (10)

     

    -

     

    (100)

    Interest and investment income

     

    214

     

    177

     

    697

     

    252

    Other (expense) income, net

     

    (46)

     

    162

     

    (39)

     

    321

    Total other income, net

     

    168

     

    329

     

    658

     

    473

    Net loss

    $

    (6,514)

    $

    (5,679)

    $

    (19,683)

    $

    (19,810)

    Net loss per share - basic and diluted

    $

    (0.56)

    $

    (0.80)

    $

    (1.80)

    $

    (4.54)

    Weighted-average common shares outstanding used in net loss per share - basic and diluted

     

    11,624,232

     

    7,103,842

     

    10,945,765

     

    4,360,395

     
     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Balance Sheets Data

    (In thousands)

    (Unaudited)

     

    September 30,

    December 31,

    2019

    2018

    Assets
    Cash and cash equivalents

    $

    17,765

    $

    15,294

    Short-term investments

     

    22,850

     

    22,276

    Right-of-use asset

     

    965

     

    -

    Other current and long-term assets

     

    2,254

     

    1,599

    Total assets

     

    43,834

     

    39,169

    Liabilities and stockholders' equity
    Current portion of operating lease liabilities

     

    929

     

    -

    Other current and long-term liabilities

     

    4,632

     

    4,227

    Total liabilities

     

    5,561

     

    4,227

    Total stockholders' equity

    $

    38,273

    $

    34,942

     
     

    Catabasis Pharmaceuticals, Inc.

    Selected Consolidated Statements of Cash Flows Data

    (In thousands)

    (Unaudited)

     
    Nine Months Ended September 30,

    2019

    2018

    Net cash used in operating activities

    $

    (18,799

    )

    $

    (18,196

    )

    Net cash used in investing activities

     

    (578

    )

     

    (22,999

    )

    Net cash provided by financing activities

     

    21,848

     

     

    44,702

     

    Net increase in cash, cash equivalents and restricted cash

    $

    2,471

     

    $

    3,507

     

     

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  32. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report third quarter 2019 financial results before the Nasdaq Global Market open on Thursday, November 7, 2019. Jill C. Milne, Ph.D., Chief Executive Officer, will host a conference call and webcast at 8:30am ET to provide an update on corporate developments and to discuss third quarter financial results.

    Conference Call Dial-In Information:

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    7757757

    Please specify to the operator that you would like to join the "Catabasis Third Quarter 2019 Results Call."

    Interested parties may access a live audio…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, will report third quarter 2019 financial results before the Nasdaq Global Market open on Thursday, November 7, 2019. Jill C. Milne, Ph.D., Chief Executive Officer, will host a conference call and webcast at 8:30am ET to provide an update on corporate developments and to discuss third quarter financial results.

    Conference Call Dial-In Information:

    Participant Toll-Free Dial-In Number:

    (877) 388-2733

    Participant International Dial-In Number:

    (541) 797-2984

    Pass Code:

    7757757

    Please specify to the operator that you would like to join the "Catabasis Third Quarter 2019 Results Call."

    Interested parties may access a live audio webcast of the conference call via the investors section of the Catabasis website, www.catabasis.com. Please connect to the Catabasis website several minutes prior to the start of the webcast to ensure adequate time for any software download that may be necessary. The webcast will be archived for 90 days.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    View Full Article Hide Full Article
  33. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that results from the Phase 2 MoveDMD trial and open-label extension with edasalonexent in boys affected by Duchenne muscular dystrophy (DMD) will be presented at the Child Neurology Society 48th Annual Meeting to be held October 23-26 in Charlotte, North Carolina.

    Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System and a Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent, will give an oral presentation titled "Treatment of Young Boys with Duchenne Muscular Dystrophy with the NF-kB Inhibitor Edasalonexent Showed a Slowing of Disease…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that results from the Phase 2 MoveDMD trial and open-label extension with edasalonexent in boys affected by Duchenne muscular dystrophy (DMD) will be presented at the Child Neurology Society 48th Annual Meeting to be held October 23-26 in Charlotte, North Carolina.

    Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System and a Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent, will give an oral presentation titled "Treatment of Young Boys with Duchenne Muscular Dystrophy with the NF-kB Inhibitor Edasalonexent Showed a Slowing of Disease Progression as Assessed by MRI and Functional Measures" during Platform Session 1 on Friday, October 25th at 9:00am ET.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  34. -- Differentiating Safety and Tolerability Profile of Edasalonexent Through More Than 55 Patient Years of Exposure --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today presented the findings from the MoveDMD trial of edasalonexent. In the Phase 2 MoveDMD trial and open-label extension, edasalonexent slowed disease progression compared to the off-treatment control period and was well tolerated through more than 55 cumulative patient years of exposure in boys affected by Duchenne muscular dystrophy (DMD). These data were presented by Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the Phase 2 MoveDMD…

    -- Differentiating Safety and Tolerability Profile of Edasalonexent Through More Than 55 Patient Years of Exposure --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today presented the findings from the MoveDMD trial of edasalonexent. In the Phase 2 MoveDMD trial and open-label extension, edasalonexent slowed disease progression compared to the off-treatment control period and was well tolerated through more than 55 cumulative patient years of exposure in boys affected by Duchenne muscular dystrophy (DMD). These data were presented by Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent in DMD at the 24th International Annual Congress of the World Muscle Society.

    "Our goal is to provide a therapy for DMD that slows disease progression, has a compelling safety profile and can be used in boys regardless of mutation," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. "The safety and tolerability data from the MoveDMD trial support the potential of edasalonexent to become a foundational therapy for those with Duchenne, from the time of diagnosis onwards. Edasalonexent has broad potential for benefit and can be used as a monotherapy as well as potentially with other therapies. Our hope is to improve the quality of life for those affected by Duchenne."

    In the MoveDMD trial and open-label extension, edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in the off-treatment control period, significantly improved biomarkers of muscle health and inflammation and was safe and well-tolerated. In more than 55 cumulative patient years of exposure, the majority of adverse events were mild in nature, and the most common treatment-related adverse event was diarrhea, generally mild and transient. There were no serious adverse events observed on treatment, and no adverse trends in chemistry, hematology, or measures of adrenal function. Edasalonexent is not a steroid and has not shown the known side effects of corticosteroids.

    Edasalonexent is an investigational oral small molecule designed to inhibit NF-kB. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Edasalonexent is currently being studied in the Phase 3 PolarisDMD trial, which has fully enrolled 130 boys with DMD, ages 4 to 7 (up to 8th birthday) with any mutation type and who had not been on steroids for the past 6 months. After the completion of 52 weeks of treatment, all boys and their eligible siblings are expected to have the option to enroll in GalaxyDMD, an open-label extension study designed to assess the long-term safety of edasalonexent. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020, and the trial is anticipated to support an NDA filing in 2021.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general

    economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  35. -- Top-line Results Expected Fourth Quarter of 2020 --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today the completion of enrollment for the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The target enrollment of 125 boys was exceeded due to strong interest from our 40 clinical sites in 8 countries and the support of patient advocacy organizations. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing in 2021.

    "We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive. At a…

    -- Top-line Results Expected Fourth Quarter of 2020 --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today the completion of enrollment for the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The target enrollment of 125 boys was exceeded due to strong interest from our 40 clinical sites in 8 countries and the support of patient advocacy organizations. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020 and the trial is anticipated to support an NDA filing in 2021.

    "We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive. At a time when there are multiple trials for Duchenne, we are very pleased that physicians and families chose the Phase 3 PolarisDMD trial for edasalonexent," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. "Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health. We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients."

    The PolarisDMD trial enrolled 130 boys ages 4 to 7 (up to 8th birthday) with any mutation type and who had not been on steroids for the past 6 months. The trial is a randomized, double-blind, placebo-controlled trial with 2 to 1 randomization such that two boys receive edasalonexent for each boy that receives placebo. At the completion of 52 weeks, all boys and their eligible siblings are expected to have the option to enroll in GalaxyDMD, an open-label extension study designed to assess the long-term safety of edasalonexent. Boys can begin or continue treatment with an approved exon skipping therapy in the GalaxyDMD trial, which has a streamlined schedule with visits to trial sites every six months.

    About Edasalonexent (CAT-1004)

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Phase 3 PolarisDMD Trial

    The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of edasalonexent in patients with DMD. The trial enrolled patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who had not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen were also eligible to enroll. The primary efficacy endpoint is change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints include the age-appropriate timed function tests: time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also included as important potential areas of differentiation. For each boy that receives placebo, two boys are receiving 100 mg/kg/day of edasalonexent and after 12 months, all boys are expected to receive edasalonexent in the open-label extension study GalaxyDMD. The PolarisDMD trial design was informed by discussions with regulators as well as input from treating physicians, patient organizations and families of boys affected by Duchenne. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020. More information about the Phase 3 PolarisDMD clinical trial is available on clinicaltrials.gov.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and our Phase 3 trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  36. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Phase 2 MoveDMD trial and open-label extension with edasalonexent in boys affected by Duchenne muscular dystrophy (DMD) will be presented at the 24th International Congress of the World Muscle Society, being held October 1-5, 2019, at the Tivoli Garden Concert Hall in Copenhagen, Denmark.

    Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent in DMD will present "Treatment of Young Boys with Duchenne Muscular Dystrophy with the NF-kB Inhibitor Edasalonexent Showed a…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that the Phase 2 MoveDMD trial and open-label extension with edasalonexent in boys affected by Duchenne muscular dystrophy (DMD) will be presented at the 24th International Congress of the World Muscle Society, being held October 1-5, 2019, at the Tivoli Garden Concert Hall in Copenhagen, Denmark.

    Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent in DMD will present "Treatment of Young Boys with Duchenne Muscular Dystrophy with the NF-kB Inhibitor Edasalonexent Showed a Slowing of Disease Progression as Assessed by MRI and Functional Measures." The presentation will take place at the Concert Hall on Saturday, October 5th at 1pm local time.

    About Edasalonexent

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the year ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  37. -- Edasalonexent Inhibits NF-kB, a Potential Driver of Disease Progression in Dysferlinopathy --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and the Jain Foundation, a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy. Dysferlinopathy (Limb-girdle muscular dystrophy type 2B / Miyoshi myopathy) is a serious rare disease that causes progressive muscle weakness for which there are currently no approved treatment options. Edasalonexent is in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) and has the potential to benefit…

    -- Edasalonexent Inhibits NF-kB, a Potential Driver of Disease Progression in Dysferlinopathy --

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and the Jain Foundation, a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, announced a preclinical research collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy. Dysferlinopathy (Limb-girdle muscular dystrophy type 2B / Miyoshi myopathy) is a serious rare disease that causes progressive muscle weakness for which there are currently no approved treatment options. Edasalonexent is in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) and has the potential to benefit patients with other diseases, such as Dysferlinopathy.

    In Dysferlinopathy, muscles lack dysferlin and as a result NF-kB is chronically activated. Edasalonexent, an oral small molecule designed to inhibit NF-kB, has the potential to slow disease progression in dysferlin-deficient populations. Under this collaboration, Catabasis and the Jain Foundation are conducting a preclinical study to evaluate the potential of edasalonexent as a therapeutic intervention for Dysferlinopathy by measuring disease progression in dysferlin-deficient mice treated with edasalonexent. The study will utilize magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) to measure muscle volume, fat accumulation, and other changes in the dysferlin-deficient mice. Initial results are expected in the first half of 2020.

    "We look forward to working with Catabasis to advance research for Dysferlinopathy," said Laura Rufibach, Ph.D., and Doug Albrecht, Ph.D., Co-Presidents of the Jain Foundation. "Patients with Dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a progressive and debilitating decline in muscle function which significantly impacts their lives. As there are currently no treatment options, we are excited to explore the potential of edasalonexent to benefit those living with this disease."

    "The chronic activation of NF-kB is a key driver in many neuromuscular disorders, including Duchenne muscular dystrophy. Evidence of NF-kB activation in Dysferlinopathy suggests a similar disease mechanism and opportunity for intervention. In the MoveDMD trial and open-label extension, treatment with edasalonexent slowed disease progression compared to the off-treatment control period. Through this collaboration, we look forward to learning more about the potential of edasalonexent in Dysferlinopathy, where, similar to Duchenne, there is also a significant unmet need," said Andrew Nichols, Ph.D., Chief Scientific Officer at Catabasis Pharmaceuticals.

    About Edasalonexent

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    About the Jain Foundation

    The Jain Foundation is a non-profit foundation whose mission is to cure muscular dystrophies caused by dysferlin protein deficiency, which includes the clinical presentations Limb-girdle muscular dystrophy type 2B (LGMD2B) and Miyoshi muscular dystrophy 1 (MMD1). Collectively these are referred to as Dysferlinopathy. The foundation's focused strategy includes financing and actively directing the progress of projects in key pathways towards a cure. These include identification and assessment of promising drug candidates, the development and maintenance of a global patient registry (the Dysferlin Registry), as well as a natural history study of patients with Dysferlinopathy to better understand disease progression and identify the best outcome measures for use in clinical trials. For more information about the Jain Foundation, the Dysferlin Registry, and the projects we support, please visit www.jain-foundation.org.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the year ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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  38. Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has named Andrew A. Komjathy as Chief Commercial Officer. Mr. Komjathy brings to Catabasis more than 30 years of experience in commercial operations and sales including extensive experience in rare diseases.

    "Andrew brings valuable rare disease commercial experience including building high performing commercial teams and strategies for successful product launches," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are looking forward to leveraging his expertise as we prepare for the potential commercialization of edasalonexent for the treatment of Duchenne muscular dystrophy."

    "Catabasis is in an exciting…

    Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announced today that it has named Andrew A. Komjathy as Chief Commercial Officer. Mr. Komjathy brings to Catabasis more than 30 years of experience in commercial operations and sales including extensive experience in rare diseases.

    "Andrew brings valuable rare disease commercial experience including building high performing commercial teams and strategies for successful product launches," said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. "We are looking forward to leveraging his expertise as we prepare for the potential commercialization of edasalonexent for the treatment of Duchenne muscular dystrophy."

    "Catabasis is in an exciting period progressing its Phase 3 trial of edasalonexent and preparing to potentially bring edasalonexent to market," said Mr. Komjathy. "I am proud to be part of a team that puts patients first and thrilled to be joining their efforts to make a new treatment option available to the Duchenne community."

    Mr. Komjathy brings deep rare disease and commercial experience, most recently from his role as Vice President, Commercial Sales at Alkermes, Inc. He has also held senior level commercial operating positions in the U.S. Multiple Sclerosis Business Unit at Genzyme, in the North American and Asia-Pacific regions for Shire Human Genetic Therapies, as Shire Global Franchise Leader for Fabry/Gaucher in Switzerland, and at Biogen. Mr. Komjathy holds an MBA from New York University Stern School of Business and his BS in Business Administration from Bucknell University.

    About Edasalonexent

    Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

    About Catabasis

    At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com.

    Forward Looking Statements

    Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company's global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, including the anticipated timing for top-line results, potential timing for the filing of an NDA, and other statements containing the words "believes," "anticipates," "plans," "expects," "may" and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company's product candidates; whether interim results from a preclinical or clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company's product candidates; and general economic and market conditions and other factors discussed in the "Risk Factors" section of the Company's Quarterly Report on Form 10-Q for the year ended June 30, 2019, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company's views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this release.

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