1. H.C. Wainwright 23rd Annual Global Investment Conference presentation and webcast to be available on September 13

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be presenting at the H.C. Wainwright 23rd Annual Global Investment Conference.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com, at 7:00 a.m. ET on September 13, 2021. A replay will also be available on Axcella's website for 90 days following the presentation.

    Internet Posting of Information

    Axcella uses its website…

    H.C. Wainwright 23rd Annual Global Investment Conference presentation and webcast to be available on September 13

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be presenting at the H.C. Wainwright 23rd Annual Global Investment Conference.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com, at 7:00 a.m. ET on September 13, 2021. A replay will also be available on Axcella's website for 90 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence and the treatment of non-alcoholic steatohepatitis (NASH). For more information, please visit www.axcellahealth.com.

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  2. AXA1125 demonstrates meaningful reductions in key measures of liver metabolism, inflammation, and fibrosis

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the publication of results from the company's AXA1125-003 clinical study in The American Journal of Gastroenterology entitled "Safety, Tolerability, and Biologic Activity of AXA1125 and AXA1957 in Subjects With Nonalcoholic Fatty Liver Disease." Based on the positive findings from this study, Axcella recently initiated its EMMPACT℠ Phase 2b clinical trial of AXA1125 in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH…

    AXA1125 demonstrates meaningful reductions in key measures of liver metabolism, inflammation, and fibrosis

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the publication of results from the company's AXA1125-003 clinical study in The American Journal of Gastroenterology entitled "Safety, Tolerability, and Biologic Activity of AXA1125 and AXA1957 in Subjects With Nonalcoholic Fatty Liver Disease." Based on the positive findings from this study, Axcella recently initiated its EMMPACT℠ Phase 2b clinical trial of AXA1125 in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH).

    "As our largest clinical investigation completed to date, AXA1125-003 served as an important confirmation of AXA1125's potential to generate meaningful benefits for patients with NASH utilizing a multi-targeted mechanism," says Dr. Alison Schecter, Axcella's President of Research and Development. "We are pleased to share data in this important publication to broaden awareness about this EMM composition's potential to serve as a compelling first-line treatment for NASH. On behalf of our team, I would like to thank the participants who took part in the study."

    AXA1125-003 was a placebo-controlled, randomized, multi-arm clinical study that enrolled 102 subjects with presumed NASH and assessed the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver, as measured by a comprehensive panel of imaging and soluble biomarkers related to metabolism, inflammation, and fibrosis. Study subjects were stratified based on the presence or absence of type 2 diabetes.

    Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to placebo, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers than AXA1957. Among subjects receiving AXA1125, 39% achieved a ≥30% relative reduction in liver fat content (MRI-PDFF), 39% achieved a ≥17 U/L reduction in alanine aminotransaminase (ALT; a marker of inflammation), and 35% achieved a ≥80 mSec reduction in corrected T1 (cT1; a marker of fibrosis). Among subjects with type 2 diabetes receiving AXA1125, a greater proportion achieved each of these thresholds. Emerging evidence suggests that these thresholds of activity increase the likelihood of histopathological improvement in NASH subjects. Notably, the above results were seen without impacting mean body weight or serum lipids.

    Initiated in April 2021, EMMPACT℠ is an ongoing randomized, double-blind, placebo-controlled, multi-center Phase 2b clinical trial that is evaluating the efficacy and safety of AXA1125 in patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients are being enrolled and randomized 1:1:1 to receive either 45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 48 weeks, with a four-week safety follow-up period. Patients are stratified based on the presence or absence of type 2 diabetes. Additional information can be found on https://clinicaltrials.gov/ via the identifier NCT04880187.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Development

    Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) "clinical studies" under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If and when Axcella decides to develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, the company will seek an IND to enable the initiation of "clinical trials" in patients.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of AXA1125 and the company's ability to enroll its EMMPACT clinical trial in a timely manner. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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    • Initiated EMMPOWER Phase 2 clinical trial of AXA1665 for the reduction in risk of recurrent OHE
    • Initiated EMMPACT Phase 2b clinical trial of AXA1125 for the treatment of NASH
    • Presented data at key medical congresses
    • Enhanced management team with the addition of Chief People Officer
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the second quarter ended June 30, 2021 and provided a business update.

    "Thanks to solid execution on the part of our team, Axcella has rapidly advanced the development of its…

    • Initiated EMMPOWER Phase 2 clinical trial of AXA1665 for the reduction in risk of recurrent OHE
    • Initiated EMMPACT Phase 2b clinical trial of AXA1125 for the treatment of NASH
    • Presented data at key medical congresses
    • Enhanced management team with the addition of Chief People Officer
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the second quarter ended June 30, 2021 and provided a business update.

    "Thanks to solid execution on the part of our team, Axcella has rapidly advanced the development of its multi-targeted EMM compositions in order to address significant unmet needs for patients with complex diseases," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "Our progress continued in the second quarter as we initiated the EMMPOWER Phase 2 clinical trial of AXA1665 in overt hepatic encephalopathy (OHE) as well as the EMMPACT Phase 2b clinical trial of AXA1125 in nonalcoholic steatohepatitis (NASH). With a high level of interest and engagement from the medical community and many clinical sites already activated for each of these global trials, I am pleased to report that we are off to a strong start. As we enter the second half of 2021, maximizing clinical trial enrollment and expanding Axcella's pipeline are our primary areas of focus."

    Recent Accomplishments

    AXA1665 for the Reduction in Risk of Recurrent OHE

    • Initiated EMMPOWER Clinical Trial: Axcella recently initiated its EMMPOWER Phase 2 clinical trial. This global 24-week, randomized, double-blind, placebo-controlled trial is evaluating the efficacy and safety of AXA1665 in approximately 150 patients who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening.
    • Presented Orally at DDW 2021: Data from Axcella's AXA1665-002 clinical study were highlighted in an oral presentation at the Digestive Disease Week (DDW) 2021 Annual Meeting by Dr. Arun Sanyal, Professor in the Virginia Commonwealth University (VCU) Department of Internal Medicine and Education Core Director in the VCU Center for Clinical and Translational Research.

    AXA1125 for the Treatment of NASH

    • Initiated EMMPACT Clinical Trial: In April, the U.S. Food and Drug Administration (FDA) cleared an Investigational New Drug (IND) application for AXA1125. Shortly thereafter, Axcella initiated its EMMPACT Phase 2b clinical trial. This global 48-week, randomized, double-blind, placebo-controlled trial is evaluating the efficacy and safety of AXA1125 in approximately 270 patients with biopsy-confirmed F2/F3 NASH.
    • Published Findings in Nature's Scientific Reports: Nature's Scientific Reports published findings from a systematic evaluation of AXA1125's EMM constituents across multiple primary human cell model systems demonstrating that they consistently and simultaneously impacted NASH-relevant metabolic, inflammatory and fibrotic processes.
    • Presented at ADA 81st Scientific Sessions: Clinical and nonclinical data regarding the effects from AXA1125 and its EMM constituents on insulin sensitivity were included in a poster presentation at the American Diabetes Association (ADA) 81st Scientific Sessions.

    Management Team

    • Added Virginia Dean as Chief People Officer: In June 2021, Axcella appointed Virginia Dean as the company's Senior Vice President and Chief People Officer. In this role, Ms. Dean is leading the company's organizational and cultural development initiatives with responsibility for all human resources functions. She previously headed up ClearSight Leadership, a consulting firm specializing in HR services, and led the human resources functions for several high-growth companies, including TESARO, Inc. and ARIAD Pharmaceuticals.

    Financial Results

    Cash Position: As of June 30, 2021, cash, cash equivalents, and marketable securities totaled $78.9 million, compared to $107.3 million at December 31, 2020. Axcella continues to expect that its existing cash balance will be sufficient to meet the company's operating needs into the third quarter of 2022.

    R&D Expenses: Research and development expenses for the quarter and six months ended June 30, 2021 were $10.3 million and $20.5 million, respectively. Research and development expenses for the same periods ended June 30, 2020 were $8.6 million and $18.9 million. These increases are primarily the result of work related to the initiations of the company's EMMPOWER and EMMPACT clinical trials.

    G&A Expenses: General and administrative expenses for the quarter and six months ended June 30, 2021 were $4.9 million and $9.2 million, respectively. General and administrative expenses for the same periods ended June 30, 2020 were $4.6 million and $8.7 million. These increases are primarily the result of greater non-cash stock-based compensation expenses and benefit-related costs.

    Net Loss: Net loss for the quarter and six months ended June 30, 2021 was $15.9 million, or $0.42 per basic and diluted share, and $31.1 million, or $0.83 per basic and diluted share, respectively. This compares with a net loss of $13.9 million, or $0.48 per basic and diluted share, and $28.9 million, or $1.10 per basic and diluted share, for the quarter and six months ended June 30, 2020.

    Conference Call Reminder

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the company's financial results and other recent business updates. The conference call webcast will be accessible in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Development

    Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) "clinical studies" under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If and when Axcella decides to develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, the company will seek an IND to enable the initiation of "clinical trials."

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of AXA1665, AXA1125 and potential future EMM compositions, the company's ability to enroll its EMMPOWER and EMMPACT clinical trials in a timely manner, its ability to expand the company's pipeline, and the company's expected cash runway. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    June 30,

     

    December 31,

     

     

    2021

     

    2020

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    33,940

     

     

    $

    71,590

     

    Marketable securities

     

    45,003

     

     

    35,739

     

    Other assets

     

    2,541

     

     

    2,263

     

    Total assets

     

    $

    81,484

     

     

    $

    109,592

     

    Liabilities and stockholders' equity:

     

     

     

     

    Liabilities

     

    $

    33,397

     

     

    $

    34,211

     

    Stockholders' equity

     

    48,087

     

     

    75,381

     

    Total liabilities and stockholders' equity

     

    $

    81,484

     

     

    $

    109,592

     

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations

    (in thousands, except share and per share data)

     

     

     

     

     

    Three Months Ended

    June 30,

     

    Six Months Ended

    June 30,

     

    2021

     

    2020

     

    2021

     

    2020

    Operating expenses:

     

     

     

     

     

     

     

    Research and development

    $

    10,298

     

     

    $

    8,565

     

     

    $

    20,538

     

     

    $

    18,900

     

    General and administrative

    4,946

     

     

    4,619

     

     

    9,202

     

     

    8,744

     

    Total operating expenses

    15,244

     

     

    13,184

     

     

    29,740

     

     

    27,644

     

    Loss from operations

    (15,244

    )

     

    (13,184

    )

     

    (29,740

    )

     

    (27,644

    )

    Other income (expense):

     

     

     

     

     

     

     

    Interest income (expense) and other income (expense), net

    (691

    )

     

    (708

    )

     

    (1,384

    )

     

    (1,257

    )

    Total other income (expense), net

    (691

    )

     

    (708

    )

     

    (1,384

    )

     

    (1,257

    )

    Net loss

    $

    (15,935

    )

     

    $

    (13,892

    )

     

    $

    (31,124

    )

     

    $

    (28,901

    )

    Net loss per share, basic and diluted

    $

    (0.42

    )

     

    $

    (0.48

    )

     

    $

    (0.83

    )

     

    $

    (1.10

    )

    Weighted average common shares outstanding, basic and diluted

    37,732,196

     

     

    29,202,367

     

     

    37,692,398

     

     

    26,195,591

     

     

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  3. Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its second quarter 2021 financial results and other business updates on July 29, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live…

    Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its second quarter 2021 financial results and other business updates on July 29, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

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    • Initial clinical sites activated and patient screening underway
    • Potential to improve the standard of care for cirrhotic patients by addressing multiple key drivers of overt hepatic encephalopathy (OHE)

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has activated initial clinical sites and patient screening for its global Phase 2 clinical trial of AXA1665, the company's multi-targeted oral product candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE).

    "OHE is a condition that has devastating consequences for patients and families and places a…

    • Initial clinical sites activated and patient screening underway
    • Potential to improve the standard of care for cirrhotic patients by addressing multiple key drivers of overt hepatic encephalopathy (OHE)

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has activated initial clinical sites and patient screening for its global Phase 2 clinical trial of AXA1665, the company's multi-targeted oral product candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE).

    "OHE is a condition that has devastating consequences for patients and families and places a substantial economic burden on the overall healthcare system," said Andres Duarte-Rojo, M.D., M.S., Ph.D., Department of Medicine, Division of Gastroenterology, Hepatology and Nutrition at the University of Pittsburgh and the trial's principal investigator. "Given the limitations of today's standards of care and the lack of clinical development activity beyond ammonia-focused agents, it also is an area in dire need of medical innovation. In prior clinical studies, AXA1665 has demonstrated the potential to have a multifactorial benefit to patients inclusive of improvements in amino acid balancing, ammonia metabolism, muscle function and neurocognition. I look forward to investigating AXA1665's ability to replicate these findings and reduce OHE events in EMMPOWER."

    Axcella has branded the Phase 2 trial EMMPOWER based on the potential for AXA1665, an EMM composition, to help patients, physicians and other caregivers overcome significant challenges associated with cirrhosis and OHE. This randomized, double-blind, placebo-controlled, multi-center investigation will evaluate the efficacy and safety of AXA1665 in patients who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening. Approximately 150 patients on lactulose ± rifaximin (stratified by rifaximin use) will be randomized 1:1 to receive either 53.8 grams per day of AXA1665 or a calorie-matched placebo in three divided doses for 24 weeks, with a four-week safety follow-up period.

    "The initiation of EMMPOWER is an important milestone, ushering in an exciting new era of clinical development for Axcella as we seek to tackle a variety of complex diseases utilizing proprietary, multi-targeted EMM compositions," said Bill Hinshaw, President and Chief Executive Officer of Axcella.

    The EMMPOWER trial will be conducted globally at more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a ≥2 point increase in the psychometric hepatic encephalopathy score (PHES) after the 24-week treatment period. The trial's key secondary endpoints will focus on the proportion of patients experiencing an OHE breakthrough event and time to first OHE breakthrough event, including time to hospitalization. Other secondary endpoints include changes in physical function, including the liver frailty index, and patient-reported outcomes. Measures of circulating ammonia, amino acid profile, and inflammation-related markers will also be included as endpoints.

    "By taking on the core drivers of OHE more comprehensively, we not only see the potential for AXA1665 to improve today's standard of care, but also to eventually become a first-line treatment option," said Alison Schecter, M.D., President of R&D at Axcella. "On behalf of the Axcella organization, we would like to express our gratitude to the many patients and dozens of clinical global sites that will be involved in this trial as we seek to transform the OHE treatment paradigm."

    Additional trial information can be found on https://clinicaltrials.gov/ via the identifier NCT04816916.

    About AXA1665 and Overt Hepatic Encephalopathy (OHE)

    Hepatic encephalopathy (HE), one of the most common complications of cirrhosis, is a condition involving amino acid imbalance, ammonia toxicity and muscle wasting, all of which contribute to diminished brain function. OHE refers to the presence of neurological abnormalities that are clinically apparent and do not require specialized psychometric testing. OHE is well established as a significant cause of morbidity and mortality in the cirrhotic population and is an area that continues to have unmet medical needs.

    AXA1665, Axcella's product candidate for reduction in risk of recurrent OHE, is a composition of eight amino acids and derivatives that is designed to target multiple metabolic pathways intersecting key organ systems, including the liver, muscle and gut. In prior clinical studies, this oral product candidate has been safe, well tolerated and has demonstrated the potential to improve ammonia handling, physical function, amino acid balance and neurocognition with a safe and well tolerated profile. AXA1665 is now in Phase 2 development.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position, and development potential of AXA1665, the potential for AXA1665 to improve upon the standard of care for OHE patients and reduce OHE events, and the company's ability to address other complex diseases utilizing EMM compositions. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trial of AXA1665, other potential impacts of COVID-19 on the company's our business and financial results, including with respect to the company's ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665, the clinical development and safety profile of AXA1665 and its therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  4. New Board Members Will Help Advance ‘Gene Writing' Platform and Mission to Cure Disease by Writing in the Code of Life

    Tessera Therapeutics, a biotech company pioneering Gene Writing, announced today the appointment of Elliott Sigal, M.D., Ph.D. and Mary Rozenman, Ph.D. to the company's Board of Directors. This announcement represents the latest key development for Tessera, following the March appointments of David Davidson, M.D. as Chief Medical and Development Officer, Hari Pujar, Ph.D. as Chief Operating Officer, and Lin Guey, Ph.D. as Senior Vice President of Rare Diseases Program Strategy and Operations, and a $230 million Series B fundraising round in January.

    "Elliott and Mary bring phenomenal leadership, experience, and creativity…

    New Board Members Will Help Advance ‘Gene Writing' Platform and Mission to Cure Disease by Writing in the Code of Life

    Tessera Therapeutics, a biotech company pioneering Gene Writing, announced today the appointment of Elliott Sigal, M.D., Ph.D. and Mary Rozenman, Ph.D. to the company's Board of Directors. This announcement represents the latest key development for Tessera, following the March appointments of David Davidson, M.D. as Chief Medical and Development Officer, Hari Pujar, Ph.D. as Chief Operating Officer, and Lin Guey, Ph.D. as Senior Vice President of Rare Diseases Program Strategy and Operations, and a $230 million Series B fundraising round in January.

    "Elliott and Mary bring phenomenal leadership, experience, and creativity to Tessera's Board of Directors," said Geoffrey von Maltzahn, Ph.D., CEO and Co-Founder of Tessera. "Elliott has overseen more than a dozen new medications on their way to market, and Mary has guided major strategic, fundraising, and partnership efforts. Together, they offer decades of experience that will help us rapidly grow Tessera and to fulfill our mission of curing diseases by writing in the code of life."

    "The next frontier in medicine will be therapeutics that cure and provide enduring protection against disease. I am excited to welcome Elliott and Mary to the team at Tessera and look forward to working closely with them as we advance Tessera's Gene Writing platform and pioneer the future of genetic medicine," said Noubar Afeyan, Ph.D., Co-founder and Chairman of the Board, Tessera and CEO of Flagship Pioneering.

    Dr. Sigal brings decades of clinical experience in drug discovery, drug development, and corporate strategy to Tessera's Board of Directors. He currently serves as a senior advisor to the healthcare team of New Enterprise Associates and is a member of the Board of Directors for Adaptimmune Therapeutics, Surface Oncology, Vir Therapeutics, and Affinia. He co-chairs the Scientific Advisory Board for Amgen and is a member of the Scientific Steering Committee for the Sean Parker Institute for Cancer Immunology. He previously served on the Board of Directors for the gene therapy company, Spark Therapeutics. Dr. Sigal is a former Executive Vice President and Director of Bristol-Myers Squibb, and served as the company's Chief Scientific Officer and President of R&D from 2004 until 2013. Under his leadership, fourteen new medicines came to market in multiple therapeutic areas including virology, oncology, immunology, psychiatry, metabolic diseases and cardiovascular disease. During his tenure, Dr. Sigal was named the best R&D chief in the pharmaceutical industry by Scrip Intelligence. Dr. Sigal received his M.D. from the University of Chicago and trained in Internal Medicine and Pulmonary Medicine at UCSF. Prior to medical school, he received his B.S., M.S., and Ph.D. in Industrial Engineering from Purdue University.

    "Tessera's Gene Writing technology represents a major breakthrough in the field with potential to fundamentally change the future of medicine across many therapeutic areas," said Dr. Elliott Sigal. "I am honored to join the Board and look forward to helping guide the company through continued growth and innovation."

    Dr. Rozenman brings more than 15 years of scientific expertise, company building, and transactions leadership in the biotech industry to Tessera's Board of Directors. She serves as the CFO and CBO of insitro, leading strategic and operational finance and accounting strategy, business development, investor relations, corporate communications, as well as project and portfolio management. Prior to insitro, Dr. Rozenman served as the Senior Vice President of Corporate Development and Strategy at Aimmune Therapeutics, where she oversaw business development and partnerships with companies including Regeneron/Sanofi and Nestle Health Science and supported the company's strategic finance and investor relations efforts. Across her time at both insitro and Aimmune, Dr. Rozenman has raised more than $1 billion in capital through a range of public and private transactions, including leadership of Aimmune's IPO in 2015. She received her Ph.D. in Organic Chemistry and Chemical Biology from Harvard University and her B.A. in Biochemistry and Russian Literature from Columbia University.

    "Tessera is pioneering Gene Writing at a pivotal moment for drug discovery, as we rapidly expand our understanding of the genetic drivers of disease. Tessera's work to write into the genome may open opportunities to cure and prevent a broad range of diseases. I am thrilled to join the Board during such an exciting time in the company's growth and look forward to partnering with Geoff and the world class Tessera team," said Dr. Mary Rozenman.

    For more information on Tessera, including how Gene Writing works, partnership opportunities, and job openings, visit www.tesseratherapeutics.com.

    About Tessera Therapeutics

    Tessera Therapeutics is an early-stage life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability.

    About Flagship Pioneering

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life science platform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $90 billion in aggregate value. To date, Flagship has deployed over $2.4 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ:AXLA), Codiak BioSciences (NASDAQ:CDAK) Denali Therapeutics (NASDAQ:DNLI), Evelo Biosciences (NASDAQ:EVLO), Foghorn Therapeutics (NASDAQ:FHTX), Indigo Ag, Kaleido Biosciences (NASDAQ:KLDO), Moderna (NASDAQ:MRNA), Rubius Therapeutics (NASDAQ:RUBY), Sana Biotechnology (NASDAQ:SANA), Seres Therapeutics (NASDAQ:MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

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  5. CAMBRIDGE, Mass., June 14, 2021 /PRNewswire/ -- Flagship Pioneering, the bioplatform innovation company, today announced a significant expansion of its capital base to support founding and growing its next generation of human health and sustainability companies. Flagship re-opened Fund VII in April 2021 to current Limited Partners and a select group of new investors, raising an additional $2.23 billion for a total capital pool of $3.37 billion. This brings together for the first time its origination and growth strategies under one investment vehicle with a long-term focus on the creation, development, and growth of its first-in-category bioplatform companies.

    CAMBRIDGE, Mass., June 14, 2021 /PRNewswire/ -- Flagship Pioneering, the bioplatform innovation company, today announced a significant expansion of its capital base to support founding and growing its next generation of human health and sustainability companies. Flagship re-opened Fund VII in April 2021 to current Limited Partners and a select group of new investors, raising an additional $2.23 billion for a total capital pool of $3.37 billion. This brings together for the first time its origination and growth strategies under one investment vehicle with a long-term focus on the creation, development, and growth of its first-in-category bioplatform companies.

    "At Flagship Pioneering, we are re-imagining how to prevent and treat disease, feed a growing population, and sustain our planet. We do this by harnessing chemistry, biology, and machine learning/AI to build cohesive, integrated platforms, each with the potential for multiple transformative products," said Noubar Afeyan, Ph.D., Founder and Chief Executive Officer of Flagship Pioneering. "There is no better example of the power of bioplatform companies than Flagship-founded Moderna, which was able to rapidly shift its focus in 2020 to develop, test, and deploy its COVID-19 vaccine in record time."

    Dr. Afeyan continued, "In the last year alone, breakthroughs at Rubius Therapeutics, Denali Therapeutics, Seres Therapeutics, and others demonstrated the exponential impact bioplatforms can have on health. Biotechnology is at the leading edge of technological progress, challenging us to think beyond incremental advances and take big leaps – and Flagship Pioneering is focused on actively leaping to the scientific spaces ripe for disruption."

    Financial Update

    After Fund VII's close, Flagship is now operating with an aggregate capital pool of $6.7 billion, with $14.1 billion assets under management. Over the past four quarters, Flagship deployed $370 million in capital to its companies, combined with more than $4.4 billion from other capital providers, for a total of $4.8 billion in new capital fueling Flagship companies.

    "We are grateful to our longstanding Limited Partners and our new investors for their support as we pioneer life science solutions to tackle the world's most urgent issues," said Stephen Berenson, Managing Partner, Flagship Pioneering. "This expanded capital pool will enable us to support first-in-category platform companies, spanning human therapeutics, agriculture, and nutrition, as well as our soon-to-be-launched Preemptive Medicine and Health Security Initiative, and the development of a robust pipeline of clinical assets through our Pioneering Medicines Division."

    Driving Ground-breaking Multi-Product Bioplatforms                                      

    Flagship Pioneering is dedicated to exploring and innovating in uncharted spaces, as well as founding, launching, and advancing new companies to develop and commercialize these innovations.

    "The biological century is an era of new possibility. Today, the opportunity to harness biology for the benefit of humanity and the world is unprecedented," said Doug Cole, M.D., Managing Partner, Flagship Pioneering. "At Flagship, we challenge ourselves daily to discover what is not yet visible, invent what has previously not been possible, and imagine and realize new ways to treat, cure, diagnose, and prevent diseases."

    Flagship's team of entrepreneurial scientists, executives, and operations experts, as well as its companies, achieved important milestones in the last year, as follows:

    • Unveiled Generate Biomedicines, Invaio Sciences, Inzen Therapeutics, Laronde, and Valo Health
    • Raised $1.8 billion in private investments for early growth-stage companies: Cellarity, Inari Agriculture, Indigo Ag, Invaio Sciences, Omega Therapeutics, Repertoire Immune Medicines, Ring Therapeutics, Senda Biosciences, Tessera Therapeutics, and Valo Health
    • Completed Initial Public Offerings at Codiak Biosciences (NASDAQ:CDAK), Foghorn Therapeutics (NASDAQ:FHTX), Sana Biotechnology (NASDAQ:SANA), and Sigilon Therapeutics (NASDAQ:SGTX)
    • Raised follow-on public market capital in Axcella (NASDAQ:AXLA), Codiak Biosciences (NASDAQ:CDAK), Evelo (NASDAQ:EVLO), Kaleido (NASDAQ:KLDO), Moderna (NASDAQ:MRNA), Rubius (NASDAQ:RUBY), and Seres (NASDAQ:MCRB)
    • Conducted more than 60 new explorations, the first step of our company origination process
    • Filed more than 300 patent applications from Flagship Labs
    • Flagship ecosystem companies have 53 clinical programs and 150 pre-clinical programs currently underway

    Assembling a World-Class Team

    Flagship is committed to bringing on the highest caliber industry executives with track records of exceptional performance, as evidenced in the company's new hires over the past year. These have included the recent additions of Christopher P. Austin, M.D., as CEO-Partner, Ara Darzi, M.D., as Chair, Preemptive Medicine and Health Security Initiative, Michael T. Nally, as CEO-Partner and CEO of Generate Biomedicines, and Louisa Salter-Cid, Ph.D., as Chief Scientific Officer for Pioneering Medicines. In addition, Flagship has hired a number of world class executives, scientists, and operations leaders to roles across the company and its ecosystem of companies. 

    Introducing Preemptive Medicine and Health Security, a New, More Comprehensive Approach to Health Security

    Flagship is actively building a division tasked with pioneering a new field that aims to protect, maintain, or improve people's health before they get sick by proactively intervening in those who are seemingly healthy but at risk. It will encompass interventions that protect health from external threats (health security) as well as to prevent or delay the onset of disease (preemptive health) to better prepare for the next infectious disease threats, and to pursue bolder treatments for existing pandemic diseases such as obesity, cancer, and neurodegeneration. Learn more about Flagship's perspective in this recent whitepaper, "Health Security and Beyond: A New Paradigm for a Post-Pandemic World."

    "The pandemic has laid bare the critical nature of health, not only to individuals, but to economies and societies," said Ara Darzi, M.D., Chair of Flagship's Preemptive Medicine and Health Security Initiative. "Flagship is pioneering a new field that aims to protect, maintain, and improve people's health by providing security against threats for everyone, and preventing or postponing the onset of disease for 'seemingly healthy' people, before they experience any recognisable symptoms at all."

    Building the Pioneering Medicines Division

    Flagship launched its Pioneering Medicines Division earlier this year to harness the power of Flagship's scientific and development innovation to create and advance novel therapeutic medicines that benefit more patients sooner. Given the broad applicability of many Flagship platforms, Pioneering Medicines is focused on expanding the reach of these scientific innovations by creating medicines in disease areas where the companies are not yet focused and working across platforms in unprecedented ways.

    "Pioneering Medicines is focused on innovative approaches to achieving greater impact for patients at faster speeds," said Paul Biondi, Executive Partner, Flagship Pioneering and President of Pioneering Medicines. "We are leveraging the innovative scientific platforms and technologies within Flagship Pioneering's ecosystem of companies to conceive and develop a unique portfolio of life-changing treatments for patients."

    About Flagship Pioneering

    Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $90 billion in aggregate value. To date, Flagship has deployed over $2.4 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Denali Therapeutics (NASDAQ:DNLI), Indigo Ag, Moderna (NASDAQ:MRNA), Rubius Therapeutics (NASDAQ:RUBY), Sana Biotechnology (NASDAQ:SANA), Seres Therapeutics (NASDAQ:MCRB), and Valo Health.

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    SOURCE Flagship Pioneering

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  6. CAMBRIDGE, Mass., May 25, 2021 /PRNewswire/ -- Invaio Sciences, Inc., a Flagship Pioneering company focused on unlocking the potential of the planet's interdependent natural systems to solve pressing agriculture, nutrition and environmental challenges, today announced the closing of a Series C financing round totaling $88.9 million. Joining founder and principal backer Flagship Pioneering is Stage 1 Ventures, Bluwave Capital, Alexandria Venture Investments, and others. With this financing, Invaio has raised more than $142 million since its founding in 2018.

    "Our ambition is to build the most impactful and valuable crop health company on the planet," said Ignacio Martinez, Co-Founder and CEO of Invaio. "The world is changing quickly, and food production practices must adapt to keep pace with evolving consumer demands, changing environmental conditions and increased stress on ecological resources."

    "This commitment by new and existing investors enables Invaio to accelerate the expansion of our integrated technology platforms that will solve performance and delivery challenges facing the agriculture industry. Our approach to reducing the use of fertilizers and chemicals will revolutionize the industry and guide the production of food to biological practices in less than a decade," explained Martinez.

    Teeming with pioneering technologies, extraordinary talent, and an insurgent mindset, Invaio Sciences has focused research and development in large addressable perennial, row crop and vegetable marketplaces. With this most recent financing, Invaio will continue to delve deep into diverse areas of discovery including tree nutritional health, insect management, vector and disease control, transformative herbicides and fungicides replacements, advanced seed technologies, carbon cycling, and more.

    "This is an exciting time to be in agricultural science. Within the next decade, a new playbook for producing food will evolve and take root, and Invaio is at the forefront of this discovery, actively contributing to developing the model for agricultural production of the future," said Gerardo Ramos, Chief Science Officer, Invaio Sciences. "At Invaio, we take a systems approach: controlling the disease with minimal environmental impact by reducing the use of pesticides by more than 90 percent and increasing carbon capture while improving the health of the system as a whole. This is just the beginning."

    Already addressing some of the biggest concerns plaguing crop health throughout the planet, Invaio Sciences is gaining a foothold within perennial production. Resources from the Series C capital raise will immediately be used to roll out solutions for problems including HLB in the citrus industry, Xylella in Olive production, and to institute more refined practices in managing vineyards and orchards. Invaio is also advancing a new carbon offering for perennial producers and is working with the USDA to bolster the vitality of beehives through ensuring pollinator health and the longevity of flowering real food trees in the future.

    "Invaio is transitioning from an R&D-focused organization to a fully commercial enterprise," commented Peleg Chevion, President and Chief Commercial Officer of Invaio. "We're building an economically attractive approach to institute sustainable agriculture at scale for diverse growers around the world."

    Chevion continued, "We are deeply committed to delivering the right solutions for producers now, as well as taking a generational view of their operations and the entire value chain. Our use of precision delivery offers a cleaner, safer and more mindful approach to manage the need for healthier crops and more nutritious and efficient food production in the future."

    About Invaio Sciences:

    Invaio Sciences is a multi-platform technology company that unlocks the potential of the planet's interdependent systems to address pressing agricultural, nutritional, and environmental challenges. Founded by Flagship Pioneering in 2018, Invaio leverages discoveries from diverse fields including human therapeutics, agriculture, environmental science, and advanced manufacturing. The company's deep understanding of the physiology of insects, plants and trees, together with its novel integrated solutions approach, promises to refine agricultural practices and reduce the need for pesticides globally. Invaio Sciences is dedicated to developing solutions that are mindful of beneficial insects, bad for pests, and safer for us all.  For more information, please visit www.invaio.com

    About Flagship Pioneering:

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life science platform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $80 billion in aggregate value. To date, Flagship has deployed over $2.3 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ: AXLA), Codiak BioSciences (NASDAQ: CDAK) Denali Therapeutics (NASDAQ: DNLI), Evelo Biosciences (NASDAQ: EVLO), Foghorn Therapeutics (NASDAQ: FHTX), Indigo Ag, Inari Agriculture, Kaleido Biosciences (NASDAQ: KLDO), Moderna (NASDAQ: MRNA), Rubius Therapeutics (NASDAQ: RUBY), Sana Biotechnology (NASDAQ: SANA), Seres Therapeutics (NASDAQ: MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

     

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    SOURCE Invaio Sciences

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  7. Activity demonstrated across targeted biologies, including amino acid balance, ammonia handling, muscle function and neurocognition

    Presentation includes new subject-specific neurocognition and muscle function data

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that positive data from the company's AXA1665-002 clinical study were presented last night at the Digestive Disease Week (DDW) 2021 Annual Meeting by Dr. Arun Sanyal, Professor in the Virginia Commonwealth University (VCU) Department of Internal Medicine and Education Core Director in the VCU Center for Clinical and Translational…

    Activity demonstrated across targeted biologies, including amino acid balance, ammonia handling, muscle function and neurocognition

    Presentation includes new subject-specific neurocognition and muscle function data

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that positive data from the company's AXA1665-002 clinical study were presented last night at the Digestive Disease Week (DDW) 2021 Annual Meeting by Dr. Arun Sanyal, Professor in the Virginia Commonwealth University (VCU) Department of Internal Medicine and Education Core Director in the VCU Center for Clinical and Translational Research.

    AXA1665-002 was a placebo-controlled, randomized clinical study that investigated the safety, tolerability and physiological impact of AXA1665 in 60 subjects with mild (Child Pugh A) and moderate (Child Pugh B) hepatic insufficiency. Subjects in the study were randomized in a 2:2:1 ratio to receive either 29.4 g or 53.8 g of AXA1665 or a matched placebo in three divided doses per day for 12 weeks with a four-week follow up.

    Both doses of AXA1665 were shown to be safe and well tolerated in the study. Additionally, activity was demonstrated across multiple targeted biologies, including amino acid balance, ammonia handling and muscle structure and function. Most importantly, dose dependent, directionally consistent changes were noted across all three psychometric tests that were utilized in AXA1665-002. These included a statistically significant change in the psychometric hepatic encephalopathy score (PHES) for patients receiving the 53.8 g dose of AXA1665 versus placebo. PHES is a highly specific assessment to diagnose hepatic encephalopathy.

    The DDW oral presentation included new subject-level data in certain measures of neurocognition and muscle function. Specifically, a positive change in PHES was seen from baseline to week 12 in a dose-proportional manner in subjects receiving AXA1665 compared with placebo, with a majority of subjects in the high dose arm achieving a clinically relevant ≥2 point improvement in PHES. Additionally, while this study primarily enrolled a non-sarcopenic population, a higher proportion of AXA1665 treated subjects (26-40%) versus placebo (14%) achieved a ≥0.3 unit decrease in the liver frailty index. Previous studies suggest that a ≥0.3 reduction in the LFI score may correlate with an improved ability to conduct activities of daily living in subjects with end-stage liver disease.

    "Given the clinical evidence supporting the role of muscle dysfunction and sarcopenia in neurocognitive impairment and morbidity in cirrhosis, there is a need for treatments that can provide OHE patients with benefits beyond those provided by conventional approaches," said Dr. Sanyal. "Based on the finding from this most recent clinical study, I believe AXA1665 provides a novel approach to the treatment of OHE and holds the potential to address important unmet needs."

    A video recording of Dr. Sanyal's presentation at DDW 2021 is now available in the Publications section of Axcella's website at https://axcellahealth.com/publications/.

    Axcella is now initiating a 24-week Phase 2 clinical trial that will compare the 53.8 g/day dose of AXA1665 versus placebo in approximately 150 patients with more advanced liver disease who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening. Additional trial information can be found on https://clinicaltrials.gov/ via the identifier NCT04816916.

    About AXA1665 and Overt Hepatic Encephalopathy (OHE)

    Hepatic encephalopathy (HE), one of the most common complications of cirrhosis, is a condition involving amino acid imbalance, ammonia toxicity and muscle wasting, all of which contribute to diminished brain function. OHE refers to the presence of neurological abnormalities that are clinically apparent and do not require specialized psychometric testing. OHE is well established as a significant cause of morbidity and mortality in the cirrhotic population and is an area that continues to have unmet medical needs.

    AXA1665, Axcella's product candidate for reduction in risk of recurrent OHE, is a composition of eight amino acids and derivatives that is designed to target multiple metabolic pathways intersecting key organ systems, including the liver, muscle and gut. In prior clinical studies, this oral product candidate has been safe, well tolerated and has demonstrated the potential to improve ammonia handling, physical function, amino acid balance and neurocognition with a safe and well tolerated profile. AXA1665 is now in Phase 2 development.

    About Axcella's Clinical Studies

    Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position, and development potential of AXA1665, the potential for AXA1665 to improve upon the standard of care for OHE patients and address unmet patient needs, and the company's ability to address other complex diseases utilizing EMM compositions. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trial of AXA1665, other potential impacts of COVID-19 on the company's our business and financial results, including with respect to the company's ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665, the clinical development and safety profile of AXA1665 and its therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  8. CAMBRIDGE, Mass., May 19, 2021 /PRNewswire/ -- Invaio Sciences, Inc., a Flagship Pioneering company focused on unlocking the potential of the planet's interdependent natural systems to solve pressing agriculture, nutrition and environmental challenges, announced today an agreement with the world-renowned Fundecitrus, Fund for Citrus Protection, located in Araraquara, Brazil.  The agreement focuses on the collaboration to evaluate the use of a novel natural peptide to control the devastating disease Citrus Greening (HLB) in Brazilian citrus trees.

    The agreement will leverage Fundecitrus' deep expertise in Citrus grove and pest management to evaluate application details of the novel peptide maSAMP in locally relevant conditions.  The peptide, developed by Dr. Hailing Jin of the University of California Riverside, and licensed for global commercialization by Invaio, has previously been proven under greenhouse conditions to reduce the multiplication of pathogen that causes HLB/Citrus Greening – a severe plant disease carried by an insect called the Asian citrus psyllid that has dramatically and rapidly threatened the citrus industry by devastating millions of acres of citrus crops throughout the world.

    Fundecitrus is a non-profit private Brazilian Citrus Industry Association that works for the interests of the citrus growers and industry. Founded 44 years ago in the state of São Paulo, the organization promotes the sustainable development of citrus production in Brazil.  Through its cutting-edge research facilities, hundreds of hectares of field trials in commercial orchards, and more than 130 staff and research members, Fundecitrus has been leading efforts to ensure that Brazilian citrus growers have access to the latest technologies, techniques, and information enabling the sustainable production of citrus products.

    "Brazil is currently the largest producer of oranges in the world, with 17 million tons of the fruit produced annually (21% of global production) and supplying over 50% of global juice production," explains Peleg Chevion, President & Chief Commercial Officer at Invaio Sciences.

    Chevion continues, "The collaboration will focus on the application of Invaio's pioneering technologies to evaluate efficacy and assess measurable improvements to tree health.

    We are already testing the technology in field trials in Florida and the goal of the Fundecitrus collaboration is to bring the benefit of this game changing technology to Brazil, as well as to other Citrus growing regions, which have already suffered, or are expected to suffer immense losses from the devastating effects of the HLB disease."

    "We are very pleased to work with some of the world's experts in HLB management in Brazil, to best understand how to apply this sustainable and innovative technology and improve the economic conditions of citrus growers", commented Avram Slovic, Sr. Commercial Director of Latin America.  "While we understand these studies are preliminary, we are confident that together with Fundecitrus we have the right team in place in order to integrate our technologies with current practices."

    About Invaio Sciences:

    Invaio Sciences is a multi-platform technology company that unlocks the potential of the planet's interdependent systems to address pressing agricultural, nutritional, and environmental challenges. Founded by Flagship Pioneering in 2018, Invaio leverages discoveries from diverse fields including human therapeutics, agriculture, environmental science, and advanced manufacturing. The company's deep understanding of the physiology of insects, plants and trees, together with its novel integrated solutions approach, promises to refine agricultural practices and reduce the need for pesticides globally. Invaio Sciences is dedicated to developing solutions that are mindful of beneficial insects, bad for pests, and safer for us all.  For more information, please visit www.invaio.com

    About Flagship Pioneering:

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life science platform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $80 billion in aggregate value. To date, Flagship has deployed over $2.3 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ: AXLA), Codiak BioSciences (NASDAQ: CDAK) Denali Therapeutics (NASDAQ: DNLI), Evelo Biosciences (NASDAQ: EVLO), Foghorn Therapeutics (NASDAQ: FHTX), Indigo Ag, Inari Agriculture, Kaleido Biosciences (NASDAQ: KLDO), Moderna (NASDAQ: MRNA), Rubius Therapeutics (NASDAQ: RUBY), Sana Biotechnology (NASDAQ: SANA), Seres Therapeutics (NASDAQ: MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/invaio-sciences-announces-key-agreement-with-fundecitrus-in-brazil-to-combat-against-citrus-greening-301294961.html

    SOURCE Invaio Sciences

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    • Initial clinical sites activated and patient screening underway
    • Potential for AXA1125 to serve as a first-line therapy for patients with nonalcoholic steatohepatitis (NASH)

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has activated initial clinical sites and begun patient screening for its EMMPACT Phase 2b clinical trial of AXA1125, the company's multi-targeted oral product candidate for the treatment of NASH.

    "NASH is the quintessential complex disease, involving the dysregulation of numerous biological pathways and impacting an enormous, heterogenous global population…

    • Initial clinical sites activated and patient screening underway
    • Potential for AXA1125 to serve as a first-line therapy for patients with nonalcoholic steatohepatitis (NASH)

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has activated initial clinical sites and begun patient screening for its EMMPACT Phase 2b clinical trial of AXA1125, the company's multi-targeted oral product candidate for the treatment of NASH.

    "NASH is the quintessential complex disease, involving the dysregulation of numerous biological pathways and impacting an enormous, heterogenous global population," said Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK and the principal investigator of EMMPACT. "These factors have confounded many physicians and drug developers in the past. Given its multi-modal mechanism, the activity and tolerability seen in past clinical studies and the presumed safety of its underlying amino acids, AXA1125 holds the potential to serve as an ideal first-line NASH agent. We are excited to have EMMPACT underway and are eager to gauge AXA1125's histological impact."

    Axcella has branded this global trial EMMPACT based on the potential for AXA1125, an EMM composition, to deliver meaningful, multifactorial clinical benefits to patients with NASH. This randomized, double-blind, placebo-controlled, multi-center investigation will evaluate the efficacy and safety of AXA1125 in patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients will be enrolled and randomized 1:1:1 to receive either 45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 48 weeks, with a four-week safety follow-up period. Patients will be stratified based on the presence or absence of type 2 diabetes.

    "The initiation of EMMPACT is the latest in a series of exciting recent milestones for Axcella and comes just one month after clearing our IND application for AXA1125, which is a testament to our team's strong preparation and execution as well as our investigators' interest and engagement," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "I am confident in our ability to extend our successful track record and rapidly enroll this clinical trial."

    EMMPACT will be conducted globally across more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a biopsy-confirmed ≥2 point improvement in NAFLD Activity Score (NAS) after the 48-week treatment period. Secondary endpoints will include the proportion of patients achieving biopsy-confirmed resolution of NASH without worsening of fibrosis and the proportion of patients achieving a ≥1 stage improvement in fibrosis without worsening of NASH. A range of non-invasive biomarkers, including MRI-PDFF and Fibroscan, will be utilized for additional endpoints and an interim analysis in the trial.

    "In two consecutive clinical studies of AXA1125 in subjects with presumed NASH, meaningful improvements were seen in non-invasive measures of hepatic fat, insulin resistance, inflammation and fibrosis," said Alison Schecter, M.D., President of R&D at Axcella. "We expect EMMPACT to provide robust insights on AXA1125's dose response, longer-term effects, histological impact and potential differentiation in type 2 diabetics that can be leveraged to expand our development efforts."

    Additional trial information can be found on https://clinicaltrials.gov/ via the identifier NCT04880187.

    About AXA1125 and Nonalcoholic Steatohepatitis (NASH)

    NASH is the most severe form of fatty liver disease and is driven by multifactorial systemic dysregulation of pathways associated with metabolism, inflammation and fibrosis. If left untreated, this disease may ultimately lead to life-threatening conditions such as cirrhosis or liver cancer, requiring liver transplant. According to the Global Liver Institute's U.S. NASH Action Plan published in December 2020, up to 40 million people in the U.S. alone are living with NASH and approximately 10% of U.S. children are afflicted with this disease. Incidence is expected to continue increasing in parallel with the obesity and type 2 diabetes epidemics. Currently, there are no approved drug therapies for NASH in the United States.

    AXA1125, Axcella's product candidate for treatment of NASH, is a composition of six amino acids and derivatives that is designed to target multiple metabolic pathways known to affect the pathogenesis of fatty liver disease. In prior clinical studies, this oral product candidate has been safe, well tolerated and has demonstrated the potential to reduce liver fat, inflammation and fibrosis with a safe and well tolerated profile while avoiding an impact on lipids and weight. AXA1125 is now in Phase 2b development.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the clinical investigations that the company has completed to date have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position, and development potential of AXA1125, the potential for AXA1125 to serve as a first-line NASH agent, the company's ability to rapidly enroll its clinical trials, and the company's ability to address other complex diseases utilizing EMM compositions. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trial of AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to the company's ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1125, the clinical development and safety profile of AXA1125 and its therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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    • AXA1665 IND application cleared by FDA for the reduction in risk of recurrent OHE
    • AXA1125 IND application cleared by FDA for the treatment of NASH
    • Initiation of AXA1665 Phase 2 and AXA1125 Phase 2b clinical trials expected shortly
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the first quarter ended March 31, 2021 and provided a business update.

    "Already in 2021, we have made tremendous progress in advancing our EMM platform and our mission to bring new multi-targeted therapies to patients with complex…

    • AXA1665 IND application cleared by FDA for the reduction in risk of recurrent OHE
    • AXA1125 IND application cleared by FDA for the treatment of NASH
    • Initiation of AXA1665 Phase 2 and AXA1125 Phase 2b clinical trials expected shortly
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the first quarter ended March 31, 2021 and provided a business update.

    "Already in 2021, we have made tremendous progress in advancing our EMM platform and our mission to bring new multi-targeted therapies to patients with complex diseases and conditions," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "The recent clearance of our first two investigational new drug (IND) applications has extended our strong track record of execution. Additionally, these milestones validate how we can efficiently compile a significant amount of clinical data and then rapidly progress into later-stage development. We expect to continue our momentum as we get our later-stage clinical trials in overt hepatic encephalopathy (OHE) and nonalcoholic steatohepatitis (NASH) underway this quarter, ramp up enrollment leveraging a physician network that is excited by our approach and modality, and expand Axcella's pipeline of EMM compositions."

    Recent Accomplishments and Next Steps

    AXA1665 for the Reduction in Risk of Recurrent OHE

    • Cleared IND: In January 2021, Axcella's IND application was cleared by the U.S. Food and Drug Administration (FDA) for AXA1665, the company's oral product candidate for OHE.
    • Readied for Phase 2 Clinical Trial: During the first quarter of 2021, Axcella finalized plans for a 24-week Phase 2 clinical trial of AXA1665 that will enroll patients with a history of OHE. Initiation of this trial is expected in the near future.
    • Accepted for DDW 2021 Oral Presentation: Data from Axcella's AXA1665-002 clinical study were accepted for an oral presentation at the Digestive Disease Week (DDW) 2021 Annual Meeting, which will be held virtually from May 21 to 23, 2021.

    AXA1125 for the Treatment of NASH

    • Cleared IND: In April 2021, Axcella's investigational new drug (IND) application was cleared by the FDA for AXA1125, the company's oral product candidate for NASH.
    • Readied for Phase 2b Clinical Trial: During the first quarter of 2021, Axcella finalized plans for a 48-week Phase 2b serial biopsy clinical trial of AXA1125 that will enroll adult patients with NASH, with primary and secondary endpoints based on liver histology. Initiation of this trial is expected in the near future.
    • Presented Data at NASH-TAG 2021: In March 2021, multiple presentations regarding AXA1125 were included in NASH-TAG 2021, an event bringing clinicians and researchers together to focus on the diagnosis and treatment of NASH and liver fibrosis. Among them was a poster presentation providing novel insights regarding AXA1125's multi-targeted mechanism of action and a AXA1125-003 clinical study data presentation that was recognized with a Distinguished Abstract and Poster Award.

    Management Team

    • Added Dr. Alison Schecter as President of R&D: In March 2021, Axcella announced the appointment of Alison D. Schecter, M.D., as the company's President of Research and Development. In this role, Dr. Schecter oversees all of the company's research, product candidate design, clinical and regulatory efforts. She brings more than 20 years of research, clinical and regulatory experience to Axcella at companies such as Sanofi-Genzyme, Johnson & Johnson, Selecta Biosciences, and Novartis.

    Financial Results

    Cash Position: As of March 31, 2021, cash, cash equivalents, and marketable securities totaled $93.0 million, compared to $107.3 million at December 31, 2020. Axcella expects that its cash balance will be sufficient to meet the company's operating needs into the third quarter of 2022.

    R&D Expenses: Research and development expenses were $10.2 million and $10.3 million for the quarters ended March 31, 2021 and 2020, respectively.

    G&A Expenses: General and administrative expenses were $4.3 million and $4.1 million for the quarters ended March 31, 2021 and 2020, respectively.

    Net Loss: Net loss for the quarter ended March 31, 2021 was $15.2 million, or $0.40 per basic and diluted share. This compares with a net loss of $15.0 million, or $0.65 per basic and diluted share, for the quarter ended March 31, 2020.

    Conference Call Reminder

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the company's financial results and other recent business updates. The conference call webcast will be accessible in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They were not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. INDs were recently cleared for Axcella's lead product candidates, AXA1665 and AXA1125, allowing for the investigation of efficacy, safety and tolerability in subsequent clinical trials.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of AXA1665, AXA1125 and potential future EMM compositions, the potential for our product candidates to improve the quality of life for patients with complex diseases, the status and timing of the company's planned Phase 2 clinical trial of AXA1665 and planned Phase 2b clinical trial of AXA1125, and the company's expected cash runway into the third quarter of 2022. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    March 31,

     

    December 31,

     

     

    2021

     

    2020

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    43,049

     

     

    $

    71,590

     

    Marketable securities

     

    49,909

     

     

    35,739

     

    Other assets

     

    1,522

     

     

    2,263

     

    Total assets

     

    $

    94,480

     

     

    $

    109,592

     

    Liabilities and stockholders' equity:

     

     

     

     

    Liabilities

     

    $

    32,822

     

     

    $

    34,211

     

    Stockholders' equity

     

    61,658

     

     

    75,381

     

    Total liabilities and stockholders' equity

     

    $

    94,480

     

     

    $

    109,592

     

     

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations

    (in thousands, except share and per share data)

     

     

     

     

     

    Three Months Ended

    March 31,

     

     

    2021

     

    2020

    Operating expenses:

     

     

     

     

    Research and development

     

    $

    10,240

     

     

    $

    10,335

     

    General and administrative

     

    4,256

     

     

    4,125

     

    Total operating expenses

     

    14,496

     

     

    14,460

     

    Loss from operations

     

    (14,496

    )

     

    (14,460

    )

    Other income (expense):

     

     

     

     

    Interest income (expense) and other income (expense), net

     

    (693

    )

     

    (549

    )

    Total other income (expense), net

     

    (693

    )

     

    (549

    )

    Net loss

     

    $

    (15,189

    )

     

    $

    (15,009

    )

    Net loss per share, basic and diluted

     

    $

    (0.40

    )

     

    $

    (0.65

    )

    Weighted average common shares outstanding, basic and diluted

     

    37,652,158

     

     

    23,188,816

     

     

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  9. Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its first quarter 2021 financial results and other business updates on May 6, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast…

    Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its first quarter 2021 financial results and other business updates on May 6, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

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  10. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the following upcoming oral presentation at the DDW 2021 regarding AXA1665, the company's product candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE):

    • Title: A Novel, Precision-Engineered Amino Acid Composition, AXA1665, is Safe, Well-Tolerated and Improves Neurocognition and Physical Function in Child-Pugh A and B Subjects
      Abstract Number: 848
      Presenter: Dr. Arun Sanyal, Professor in the Virginia Commonwealth University (VCU) Department of Internal Medicine and Education Core Director in the VCU Center for…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the following upcoming oral presentation at the DDW 2021 regarding AXA1665, the company's product candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE):

    • Title: A Novel, Precision-Engineered Amino Acid Composition, AXA1665, is Safe, Well-Tolerated and Improves Neurocognition and Physical Function in Child-Pugh A and B Subjects

      Abstract Number: 848

      Presenter: Dr. Arun Sanyal, Professor in the Virginia Commonwealth University (VCU) Department of Internal Medicine and Education Core Director in the VCU Center for Clinical and Translational Research

      Session: #4450 Portal Hypertension

      Date/Time: Sunday, May 23rd from 5:45-6:00 p.m. ET

    DDW 2021 is being held virtually from May 21 to 23, 2021. Abstracts are expected to appear on DDW's website in late April and in the May online supplement to Gastroenterology. ePosters and ePapers will be made available on this website during the congress. DDW is the world's leading educational forum for clinicians, researchers, students and trainees working in gastroenterology, hepatology, GI endoscopy, gastrointestinal surgery and related fields.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

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    • Multi-targeted oral candidate with the potential to be a first-line treatment option for adult and pediatric patients with NASH
    • Phase 2b clinical trial in adults expected to begin in Q2

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has achieved a key milestone with U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for AXA1125, enabling the company to proceed directly into a Phase 2b clinical trial. AXA1125 is Axcella's multi-targeted oral product candidate for nonalcoholic steatohepatitis (NASH), a chronic and progressive liver…

    • Multi-targeted oral candidate with the potential to be a first-line treatment option for adult and pediatric patients with NASH
    • Phase 2b clinical trial in adults expected to begin in Q2

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it has achieved a key milestone with U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for AXA1125, enabling the company to proceed directly into a Phase 2b clinical trial. AXA1125 is Axcella's multi-targeted oral product candidate for nonalcoholic steatohepatitis (NASH), a chronic and progressive liver disease impacting up to 40 million people in the U.S. alone.

    "This IND follows close on the heels of AXA1665's IND clearance earlier this year, ushering in an exciting new era for Axcella as we seek to tackle a variety of complex diseases and address important unmet needs for patients utilizing multi-targeted EMM compositions," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "I am pleased by our team's strong execution in completing two robust FDA submissions and readying us for the start of our highly efficient, later-stage clinical trials."

    Axcella expects to initiate its Phase 2b clinical trial in the second quarter of 2021. This randomized, double-blind, placebo-controlled, multi-center trial will evaluate the efficacy, safety and tolerability of AXA1125 in adult patients with biopsy-confirmed F2/F3 NASH. Approximately 270 patients will be enrolled and randomized 1:1:1 to receive either 45.2 or 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 48 weeks, with a four-week safety follow-up period. Patients will be stratified based on the presence or absence of type 2 diabetes.

    The trial will be conducted globally across more than 70 clinical sites with a primary endpoint assessing the proportion of patients with a biopsy-confirmed ≥2 point improvement in NAFLD Activity Score (NAS) after the 48-week treatment period. Secondary endpoints will include the proportion of patients achieving biopsy-confirmed resolution of NASH without worsening of fibrosis and the proportion of patients achieving a ≥1 stage improvement in fibrosis without worsening of NASH. A range of non-invasive biomarkers, including MRI-PDFF and Fibroscan, will be utilized for additional endpoints and an interim analysis in the trial.

    "AXA1125 leverages a mechanism that engages multiple pathophysiologic pathways involved in the development of NASH using a modality with well-precedented safety, which is particularly important given the many comorbidities associated with this chronic disease," said Alison Schecter, M.D., President of R&D at Axcella. "In past clinical studies, compelling activity has been seen with AXA1125 across a range of non-invasive biomarkers, with enhanced effects in type 2 diabetics. Our goal in the Phase 2b is to affirm AXA1125's impact via histology and further strengthen its profile as a compelling candidate for first-line treatment in NASH."

    The upcoming Phase 2b clinical trial follows two earlier non-IND clinical studies in which reductions were seen with AXA1125 in key measures of hepatic fat, insulin resistance, inflammation and fibrosis with a safe and well tolerated profile. Presentations containing those findings can be found by visiting https://axcellahealth.com/publications/.

    About AXA1125 and Nonalcoholic Steatohepatitis (NASH)

    NASH is the most severe form of non-alcoholic fatty liver disease (NAFLD). This chronic, complex disease is associated with significant morbidity and mortality globally, and it is estimated to impact up to 40 million Americans, including up to 10% of American children. Despite its severity and increasing prevalence, there are currently no approved therapies for NASH in the United States.

    AXA1125, Axcella's NASH product candidate, is a composition of six amino acids and derivatives that is designed to target multiple metabolic pathways involved in NASH's progression from metabolism to inflammation to fibrosis. In prior clinical studies, this oral product candidate has been safe and well tolerated and has demonstrated clinically meaningful reductions in a range of well-accepted, non-invasive NASH biomarkers, with the most pronounced activity noted in subjects with type 2 diabetes. AXA1125 is now entering Phase 2b development in adults with NASH. Axcella also plans to investigate AXA1125 in pediatric NASH.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of naturally occurring molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the underlying causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They were not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. INDs were recently cleared for Axcella's lead product candidates, AXA1665 and AXA1125, allowing for the investigation of these candidates' efficacy, safety and tolerability in subsequent clinical trials.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position, and development potential of AXA1125, the design, status and timing of the company's planned Phase 2b clinical trial of AXA1125, and the company's ability to address other complex diseases utilizing EMM compositions. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trial of AXA1125, other potential impacts of COVID-19 on the company's our business and financial results, including with respect to the company's ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1125, the clinical development and safety profile of AXA1125 and its therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  11. David Davidson, M.D. to Serve as Chief Medical and Development Officer; Hari Pujar, Ph.D. to Serve as Chief Operating Officer and Lin Guey, Ph.D. to Serve as Senior Vice President of Rare Diseases Program Strategy and Operations

    Tessera Therapeutics, a biotech company pioneering Gene Writing, announced today the appointment of David Davidson, M.D. as Chief Medical and Development Officer, Hari Pujar, Ph.D. as Chief Operating Officer, and Lin Guey, Ph.D. as Senior Vice President of Rare Diseases Program Strategy and Operations. The senior appointments come just two months after the company announced over $230 million in Series B fundraising in January and will be key to advancing the company's Gene Writing platform and accelerating development…

    David Davidson, M.D. to Serve as Chief Medical and Development Officer; Hari Pujar, Ph.D. to Serve as Chief Operating Officer and Lin Guey, Ph.D. to Serve as Senior Vice President of Rare Diseases Program Strategy and Operations

    Tessera Therapeutics, a biotech company pioneering Gene Writing, announced today the appointment of David Davidson, M.D. as Chief Medical and Development Officer, Hari Pujar, Ph.D. as Chief Operating Officer, and Lin Guey, Ph.D. as Senior Vice President of Rare Diseases Program Strategy and Operations. The senior appointments come just two months after the company announced over $230 million in Series B fundraising in January and will be key to advancing the company's Gene Writing platform and accelerating development of multiple therapeutic programs.

    "David, Hari and Lin all bring a wealth of strategic leadership and deep expertise to Tessera at an exciting time in our growth," said Dr. Geoffrey von Maltzahn, CEO and Co-Founder of Tessera. "Together and alongside our current senior leadership team, they will be instrumental in advancing Tessera's mission to bring forth innovative and curative medicines that go beyond the limitations of current technologies."

    Dr. Davidson brings over 20 years of expertise in clinical drug development focused on rare diseases and gene therapy to his new role as Tessera's Chief Medical and Development Officer. For nearly a decade, Dr. Davidson served as Chief Medical Officer of bluebird bio, where he played a critical role in advancing a broad genetic medicine pipeline across blood disorders, rare diseases and oncology, successfully leading numerous genetic medicine programs into the clinic and delivering multiple first-in-class approvals for genetic medicines in the U.S. and Europe. Prior to bluebird, Dr. Davidson led clinical research for a wide range of therapeutic programs at Genzyme and GelTex, spanning biologics, polymers, and gene therapy. He completed a fellowship in infectious diseases at the Harvard Longwood Combined ID Program, his residency training in internal medicine, and an endocrinology research fellowship at the University of Chicago Hospitals. Dr. Davidson received his M.D. from New York University and B.A. from Columbia University.

    "I have been privileged to lead the clinical development of gene therapy programs from inception through approval to deliver these transformative medicines to patients with life-threatening diseases. I am excited to join the team at Tessera Therapeutics to bring innovative Gene Writing therapies into the clinic," said Dr. David Davidson. "By harnessing the power of mobile genetic elements, Tessera's Gene Writing platform is poised to realize the full potential of genetic medicine and make an unprecedented impact on human health. I am honored to help move this vision forward."

    Dr. Pujar, an Operating Partner at Flagship Pioneering since October 2020, will also serve as Tessera's Chief Operating Officer. Dr. Pujar brings more than 20 years of drug development, manufacturing and commercialization experience spanning AAV gene therapy, mRNA vaccines and therapeutics, and traditional vaccines and biologics. Most recently, Dr. Pujar served as Chief Technology Officer at Spark Therapeutics, a pioneer in AAV gene therapy, and Head of Technical Development and Manufacturing at Moderna, where he led mRNA and lipid nanoparticle development and manufacturing. Previously, Dr. Pujar spent 18 years at Merck & Co., developing and commercializing vaccines and biologics. Dr. Pujar received his Ph.D. in Chemical Engineering from the University of Delaware and his M.B.A. from the University of Pennsylvania.

    "As medicine continues to move up the central dogma of molecular biology from proteins to mRNA to DNA, the opportunity to cure diseases will continue to expand. I'm excited to help shape Tessera Therapeutics, whose Gene Writing technology has the potential to address many of the limitations of current genetic medicine technologies," said Dr. Hari Pujar.

    Dr. Guey, Tessera's Senior Vice President of Rare Diseases Program Strategy and Operations, will oversee the execution of Tessera's genetic medicine programs in Rare Diseases. Dr. Guey brings over 13 years of drug development experience in program leadership, research and nonclinical development, pharmacogenomics and epidemiology. Prior to joining Tessera, Dr. Guey served in senior leadership roles for Moderna, Xilio Therapeutics, Shire, and Pfizer. Dr. Guey received her Ph.D. in Statistics and B.S. in Mathematics from Stony Brook University.

    "With its pioneering and versatile Gene Writing technology, Tessera Therapeutics is at the very forefront of genetic medicine," said Dr. Lin Guey. "I look forward to working with the Tessera team to find new ways to cure rare genetic diseases at their source."

    For more information on Tessera, including how Gene Writing works, partnership opportunities, and job openings, visit www.tesseratherapeutics.com.

    About Tessera Therapeutics

    Tessera Therapeutics is a life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability.

    About Flagship Pioneering

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life science platform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $80 billion in aggregate value. To date, Flagship has deployed over $2.3 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ:AXLA), Codiak BioSciences (NASDAQ:CDAK) Denali Therapeutics (NASDAQ:DNLI), Evelo Biosciences (NASDAQ:EVLO), Foghorn Therapeutics (NASDAQ:FHTX), Indigo Ag, Kaleido Biosciences (NASDAQ:KLDO), Moderna (NASDAQ:MRNA), Rubius Therapeutics (NASDAQ:RUBY), Sana Biotechnology (NASDAQ:SANA), Seres Therapeutics (NASDAQ:MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

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  12. CAMBRIDGE, Mass., March 30, 2021 /PRNewswire/ -- Invaio Sciences, Inc., a Flagship Pioneering company focused on unlocking the potential of the planet's interdependent natural systems to solve pressing agriculture, nutrition and environmental challenges, announced today the appointment of Peleg Chevion as President and Chief Commercial Officer.  Chevion, who joined the company in late 2020, will focus on accelerating the commercial activities of the company.

    "Our goal at Invaio is to accelerate the transition in agriculture from a chemical-based industry to a biological-based industry that addresses the needs of farmers and society, while protecting the environment," said Ignacio Martinez, Co-Founder and CEO, Invaio. "The company is now transitioning from an R&D-focused organization to a full commercial enterprise."

    Martinez continued, "Peleg has already made a significant impact at Invaio since he joined the company in late 2020. He brings a very unique set of experiences, commercial leadership, and an entrepreneurial mindset perfectly suited for the next phase of the company."

    Prior to joining Invaio, Chevion was President and Chief Commercial Officer of Blendhub, a global leader in food and ingredients production. Before Blendhub, Peleg worked with Lindsey Goldberg, a leading global private equity firm based in New York. Chevion also worked for seven years as a senior executive at Syngenta where he started and then led the Abiotic Stress Management indication across the spectrum of chemical, biological, genetics and digital platforms. Prior to Syngenta, Chevion spent several years leading Business Development at Crucell before the company was acquired by J&J.

    Chevion began his career as an officer in the Israeli Air Force, and later held diverse roles in the Israeli startup ecosystem.  Chevion graduated from the IDF Computer Academy. He received an L.L.B from Tel Aviv University, an MBA from INSEAD and is a licensed attorney in Israel.

    "Since joining Invaio last year, I have been impressed by the breakthrough platform technologies we are developing, our integrated solutions approach and the commercial traction of the company," said Peleg Chevion, President and Chief Commercial Officer, Invaio. "We are curing diseases like Greening in Citrus and Xylella in Olives, significantly reducing the use of chemicals with our precision delivery systems, and discovering biological products with consistent performance using proprietary Artificial Intelligence and Machine Learning technologies"

    Chevion continues, "We are laser focused on developing unique solutions for our partner growers and other stakeholders in three segments: perennials, row crops and vegetable crops. We see the opportunity and have a sense of urgency to deliver."

    About Invaio Sciences:

    Invaio Sciences is a multi-platform technology company that unlocks the potential of the planet's interdependent systems to address pressing agricultural, nutritional, and environmental challenges. Founded by Flagship Pioneering in 2018, Invaio leverages discoveries from diverse fields including human therapeutics, agriculture, environmental science, and advanced manufacturing. The company's deep understanding of the physiology of insects, plants and trees, together with its novel integrated solutions approach, promises to refine agricultural practices and reduce the need for pesticides globally. Invaio Sciences is dedicated to developing solutions that are mindful of beneficial insects, bad for pests, and safer for us all.  For more information, please visit www.invaio.com

    About Flagship Pioneering:

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life science platform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $80 billion in aggregate value. To date, Flagship has deployed over $2.3 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ:AXLA), Codiak BioSciences (NASDAQ:CDAK) Denali Therapeutics (NASDAQ:DNLI), Evelo Biosciences (NASDAQ:EVLO), Foghorn Therapeutics (NASDAQ:FHTX), Indigo Ag, Inari Agriculture, Kaleido Biosciences (NASDAQ:KLDO), Moderna (NASDAQ:MRNA), Rubius Therapeutics (NASDAQ:RUBY), Sana Biotechnology (NASDAQ:SANA), Seres Therapeutics (NASDAQ:MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/invaio-sciences-strengthens-leadership-team-with-appointment-of-peleg-chevion-as-president-and-chief-commercial-officer-301258544.html

    SOURCE Invaio Sciences

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    • First IND application cleared by FDA for AXA1665
    • Initiation of AXA1665 Phase 2 clinical trial in OHE expected in Q2 2021
    • Initiation of AXA1125 Phase 2b clinical trial in NASH expected in Q2 2021
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the fourth quarter and full year ended December 31, 2020 and provided a business update.

    "The year 2020 was a key inflection point for Axcella as we completed multiple clinical studies with positive results and shifted our full attention to our upcoming later-stage…

    • First IND application cleared by FDA for AXA1665
    • Initiation of AXA1665 Phase 2 clinical trial in OHE expected in Q2 2021
    • Initiation of AXA1125 Phase 2b clinical trial in NASH expected in Q2 2021
    • Company to host conference call at 8:30 a.m. ET today

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the fourth quarter and full year ended December 31, 2020 and provided a business update.

    "The year 2020 was a key inflection point for Axcella as we completed multiple clinical studies with positive results and shifted our full attention to our upcoming later-stage clinical trials," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "We were pleased to advance our preparations in the fourth quarter with a successful Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding our nonalcoholic steatohepatitis (NASH) candidate, AXA1125, and began 2021 with our first investigational new drug (IND) clearance for AXA1665, our candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE). Axcella's strong execution has positioned us for an exciting year ahead as we seek to swiftly enroll our upcoming trials, expand our pipeline of EMM compositions and further our mission to improve the lives of patients with complex diseases."

    Recent Accomplishments and Next Steps

    AXA1665 for OHE

    • Cleared IND Application: In January 2021, Axcella's IND application was cleared by the FDA for AXA1665. This first IND clearance marks a key milestone and validates the company's clinical approach with its EMM compositions.
    • Planned Phase 2 Clinical Trial Initiation: Axcella plans to initiate a 24-week Phase 2 clinical trial of AXA1665 in the second quarter of 2021. This will be a randomized, double-blind, placebo-controlled, multi-center trial evaluating the efficacy and safety of AXA1665 in patients who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening. Approximately 150 patients on lactulose ± rifaximin (stratified by rifaximin use) will be enrolled and randomized 1:1 to receive either 53.8 grams per day of AXA1665 or a calorie-matched placebo in three divided doses for 24 weeks, with a four-week safety follow-up period. The trial will be conducted globally with a primary endpoint assessing the proportion of subjects with a ≥2 point increase in the psychometric hepatic encephalopathy score (PHES) after the 24-week treatment period. Secondary endpoints will include the proportion of patients experiencing an OHE breakthrough event; time to first OHE breakthrough event, including time to hospitalization; changes in physical function; and patient-reported outcomes. Other endpoints include measures of circulating ammonia, amino acids, and inflammation-related markers.

    AXA1125 for NASH

    • Advanced IND Preparations: Axcella participated in a Type B pre-IND meeting with the FDA regarding AXA1125 in the fourth quarter of 2020. This engagement provided the insight required for the company to advance its Phase 2b clinical trial design and finalize its preparation of an IND submission.
    • Planned Phase 2b Clinical Trial: Following IND clearance by the FDA, Axcella plans to proceed directly into a 48-week Phase 2b serial biopsy clinical trial of AXA1125 enrolling adult patients with NASH, with a primary endpoint based on liver histology. This trial is expected to be initiated in the second quarter of 2021.
    • Presented Data at The Liver Meeting 2020: Two posters regarding AXA1125 were presented in November 2020 at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD). Both presentations featured data from AXA1125-003, a placebo-controlled, randomized clinical study that enrolled 102 subjects with presumed nonalcoholic steatohepatitis (NASH) to assess the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver for 16 weeks.
    • Presented Data at NASH-TAG 2021: Last week, multiple presentations regarding AXA1125 were included in NASH-TAG 2021, an event bringing clinicians and researchers together to focus on the diagnosis and therapy of NASH and liver fibrosis. Among them was a poster presentation providing novel insights regarding AXA1125's multi-targeted mechanism of action and a AXA1125-003 clinical study data presentation that was recognized with a Distinguished Abstract and Poster Award.

    Management Team

    • Added President of R&D: Axcella announced the appointment of Alison D. Schecter, M.D., as the company's President of Research and Development. In this role, Dr. Schecter oversees all of the company's research, product candidate design, clinical and regulatory efforts. She brings more than 20 years of research, clinical and regulatory experience to Axcella at companies such as Selecta Biosciences, Sanofi-Genzyme, Baxalta, Novartis and Johnson & Johnson.

    Financial Results

    Cash Position: As of December 31, 2020, cash, cash equivalents, and marketable securities totaled $107.3 million, compared to $92.1 million at December 31, 2019. The increase is primarily the result of net proceeds from the company's follow-on stock offering that was completed in May 2020. Axcella expects that its cash balance will be sufficient to meet the company's operating needs into the third quarter of 2022.

    R&D Expenses: Research and development expenses were $10.6 million and $10.8 million for the quarters ended December 31, 2020 and 2019, respectively. For the years ended December 31, 2020 and 2019, research and development expenses were $37.0 million and $41.7 million, respectively. The year-over-year decrease is primarily due to the completion of the company's AXA1665-002 and AXA1125-003 clinical studies.

    G&A Expenses: General and administrative expenses were $3.9 million and $4.6 million for the quarters ended December 31, 2020 and 2019, respectively. For the years ended December 31, 2020 and 2019, general and administrative expenses were $16.8 million and $15.8 million, respectively. The year-over-year increase is primarily due to greater costs associated with becoming a public company.

    Net Loss: Net loss for the quarter ended December 31, 2020 was $15.2 million, or $0.40 per basic and diluted share. This compares with a net loss of $15.7 million, or $0.68 per basic and diluted share, for the quarter ended December 31, 2019. Net loss for the year ended December 31, 2020 was $56.5 million, or $1.78 per basic and diluted share. This compares with a net loss of $59.0 million, or $3.55 per basic and diluted share, for the year ended December 31, 2019.

    Conference Call Reminder

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the company's financial results and other recent business updates. The conference call webcast will be accessible in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical trials will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of AXA1665, AXA1125 and potential future EMM compositions, the potential for our product candidates to improve the quality of life for patients with complex diseases, the status and timing of the company's planned Phase 2 clinical trial of AXA1665 and planned Phased 2b clinical trial of AXA1125, the timing and outcome of IND application submissions and the company's expected cash runway into the third quarter of 2022. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    December 31,

    2020

     

    December 31,

    2019

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    71,590

     

    $

    92,053

    Marketable securities

     

     

    35,739

     

     

    Other assets

     

     

    2,263

     

     

    2,306

    Total assets

     

    $

    109,592

     

    $

    94,359

    Liabilities and stockholders' equity:

     

     

     

     

    Liabilities

     

    $

    34,211

     

    $

    34,135

    Stockholders' equity

     

     

    75,381

     

     

    60,224

    Total liabilities and stockholders' equity

     

    $

    109,592

     

    $

    94,359

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations

    (in thousands, except share and per share data)

     

     

     

     

     

    Three Months Ended

    December 31,

     

    Year Ended

    December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    Operating expenses:

     

     

     

     

     

     

     

    Research and development

    $

    10,598

     

     

    $

    10,758

     

     

    $

    37,039

     

     

    $

    41,658

     

    General and administrative

     

    3,869

     

     

     

    4,582

     

     

     

    16,797

     

     

     

    15,781

     

    Total operating expenses

     

    14,467

     

     

     

    15,340

     

     

     

    53,836

     

     

     

    57,439

     

    Loss from operations

     

    (14,467

    )

     

     

    (15,340

    )

     

     

    (53,836

    )

     

     

    (57,439

    )

    Other income (expense):

     

     

     

     

     

     

     

    Interest income (expense) and other income (expense), net

     

    (722

    )

     

     

    (373

    )

     

     

    (2,691

    )

     

     

    (1,598

    )

    Total other income (expense), net

     

    (722

    )

     

     

    (373

    )

     

     

    (2,691

    )

     

     

    (1,598

    )

    Net loss

    $

    (15,189

    )

     

    $

    (15,713

    )

     

    $

    (56,527

    )

     

    $

    (59,037

    )

    Net loss per share, basic and diluted

    $

    (0.40

    )

     

    $

    (0.68

    )

     

    $

    (1.78

    )

     

    $

    (3.55

    )

    Weighted average common shares outstanding, basic and diluted

     

    37,536,350

     

     

     

    23,137,014

     

     

     

    31,747,676

     

     

     

    16,624,941

     

     

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  13. Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its fourth quarter and full year 2020 financial results and other business updates on March 17, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen…

    Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its fourth quarter and full year 2020 financial results and other business updates on March 17, 2021. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (844) 808-7139 (U.S. toll free) or (412) 902-0127 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 90 days following the call.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    View Full Article Hide Full Article
  14. Experienced biopharma leader to oversee research, candidate development, clinical and regulatory functions

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the appointment of Alison D. Schecter, M.D., as the company's President of Research and Development. In this role, Dr. Schecter will oversee all of the company's research, product candidate design, clinical and regulatory efforts. Additionally, Manu Chakravarthy, M.D., Ph.D., has decided to step down as the company's Chief Medical Officer to pursue another opportunity.

    "We are excited to welcome Alison to Axcella and view her unique…

    Experienced biopharma leader to oversee research, candidate development, clinical and regulatory functions

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced the appointment of Alison D. Schecter, M.D., as the company's President of Research and Development. In this role, Dr. Schecter will oversee all of the company's research, product candidate design, clinical and regulatory efforts. Additionally, Manu Chakravarthy, M.D., Ph.D., has decided to step down as the company's Chief Medical Officer to pursue another opportunity.

    "We are excited to welcome Alison to Axcella and view her unique skillsets as a great match for our innovative, efficient and highly informative development model. Her impressive experience across the drug development lifecycle will be valuable as we enter late-stage development with our lead candidates and execute our pipeline expansion plans," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "On behalf of Axcella's employees and board, I would like to extend our appreciation to Manu for his collaboration and many contributions over the past few years, culminating in the finalization of our AXA1125 IND submission and clearance of our AXA1665 IND. We wish him the very best in his new venture."

    Dr. Schecter brings more than 20 years of R&D, clinical and regulatory experience to Axcella. Previously, she served as Chief Medical Officer at Selecta Biosciences, Inc. and Global Project Head, Rare Diseases at Sanofi-Genzyme. Prior to this, Dr. Schecter was Global Program Head at Baxalta, where she was instrumental in obtaining multinational approvals for Adynovate and advancing the company's other hemophilia candidates. Earlier, she held roles of increasing responsibility in translational medicine, cardiovascular and metabolism product innovation at the Northeast J&J Innovation Center and the Novartis Institutes of Biomedical Research (NIBR). Dr. Schecter started her career in academia as Associate Professor in Immunology and Medicine and co-founder and co-director of the Cardiovascular Research Institute at the Icahn School of Medicine at Mount Sinai. She is a boarded cardiologist and internist who completed an Internal Medicine residency at The Johns Hopkins Hospital, a Cardiology fellowship at Massachusetts General Hospital and a Research Fellowship at Mount Sinai School of Medicine. Dr. Schecter earned her medical degree from SUNY Downstate Health Sciences University.

    "With strong clinical data, recent regulatory milestones and a plethora of R&D opportunities afforded by its unique platform, I could not be more excited to take on this role at Axcella," said Dr. Schecter. "I am looking forward to working with the team as we strive to improve the lives of patients with complex diseases utilizing novel EMM compositions."

    Dr. Chakravarthy said, "My time at Axcella has been extremely gratifying as we were able to pioneer a new class of multi-targeted therapeutic candidates, demonstrate their potential, replicate our findings in multiple clinical studies, and advance two liver disease candidates into Phase 2/2b trials within four years of their design. It has been a privilege to be a part of this organization, and I am looking forward to helping ensure a smooth transition as Axcella enters its next exciting phase of development."

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the company's ability to progress into late-stage development with its lead candidates and execute its pipeline expansion plans. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  15. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced three upcoming presentations at the NASH-TAG 2021 Conference, which is taking place March 11-13. Among the information to be shared by Axcella at this event will be clinical data regarding AXA1125, the company's multi-targeted product candidate for nonalcoholic steatohepatitis (NASH) that is entering Phase 2b clinical development. Additionally, new in-depth insights into the mechanism of action for AXA1125 will be provided. The presentation details are as follows:

    • Abstract #22: "Mechanistic insights into AXA1125, a novel endogenous metabolic…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced three upcoming presentations at the NASH-TAG 2021 Conference, which is taking place March 11-13. Among the information to be shared by Axcella at this event will be clinical data regarding AXA1125, the company's multi-targeted product candidate for nonalcoholic steatohepatitis (NASH) that is entering Phase 2b clinical development. Additionally, new in-depth insights into the mechanism of action for AXA1125 will be provided. The presentation details are as follows:

    • Abstract #22: "Mechanistic insights into AXA1125, a novel endogenous metabolic modulator composition, targeting multiple NASH drivers"
    • Abstract #33: Distinguished Abstract and Poster Award to be presented by Nadege T. Gunn, M.D., Lead Physician Investigator at Pinnacle Clinical Research in Austin, TX and Adjunct Assistant Professor at Texas A&M University College of Medicine. "Safety, tolerability, and biological activity of AXA1125 and AXA1957 in a prospective 16-week randomized, placebo-controlled study in subjects with NAFLD with and without type 2 diabetes"
    • Abstract #34: "Biological activity of AXA1125 and AXA1957 on glucose, insulin, HOMA-IR, and HbA1c and measures of liver fat and fibroinflammation in a prospective 16-week randomized, placebo-controlled study in subjects with NAFLD and type 2 diabetes"

    NASH-TAG brings together clinicians and researchers from academia and the biopharmaceutical industry for interactive educational presentations highlighting the most relevant advances and challenges in the diagnosis and therapy of NASH and liver fibrosis. Internationally renowned faculty assist in the development of the educational content and serve as faculty at the conference. Given the hybrid nature of the conference in 2021, all presentations will be in poster format.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

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  16. CAMBRIDGE, Mass., Feb. 25, 2021 /PRNewswire/ -- Cellarity, a life sciences company founded by Flagship Pioneering to develop a new method of drug discovery targeting the cell, announced today that it raised $123 million in Series B financing. The round included contributions from funds and accounts managed by BlackRock, The Baupost Group, Banque Pictet on behalf of their clients and eight other investors alongside Flagship Pioneering.

    Founded in 2017 within Flagship Labs, Cellarity's new approach to drug discovery is based on the computational modeling of cell behavior, leading to a more complete understanding of system and network biology to uncover cell-behavior-targeted medicines for a wide variety of diseases.

    Cellarity's medicines are developed to address disease at the level of the cell and are not aimed at single molecular targets, the approach pursued in much of traditional drug discovery. The successful financing round validates the strength of Cellarity's science and its potential to redefine drug discovery through its cell-centric approach.

    "All diseases stem from a disorder at the cellular level, but until now most drug discovery efforts have relied upon single molecular targets that overlook the potent networked relationships of biology," said Fabrice Chouraqui, CEO of Cellarity and a CEO-Partner at Flagship Pioneering. "By working at the level of the entire cell, Cellarity's drug discovery platform harnesses the complexity of human biology to uncover breakthrough treatments in a wide range of disease areas."

    Cellarity is generating unprecedented biological insights by combining its unique expertise in network biology, high-resolution data, and machine learning. The result is a new understanding of the cell's trajectory from health to disease, and how cells relate to one another in tissues. Because the cell and its network of transcripts and proteins offer a more complete view of the complexity of human biology than any individual molecular target, Cellarity's approach allows for more efficient drug discovery and is designed to drive higher rates of clinical success. Already, the company has seven drug discovery programs underway in diverse therapeutic areas that range from metabolic disease to immuno-oncology.

    "Cellarity's cell-centric platform has the potential to engineer a shift in how the world approaches the discovery of medicine," said Noubar Afeyan, CEO of Flagship Pioneering and Co-founder and Chairman of the Board of Cellarity. "It is another example of Flagship Pioneering's relentless efforts to push the boundaries of the life sciences to uncover breakthrough technologies and identify innovative treatments that will change the lives of patients."

    About Cellarity

    Cellarity's mission is to bring breakthrough medicines to patients by completely redefining the way drugs are discovered. Founded by Flagship Pioneering in 2017, Cellarity is designing medicines against the cell as opposed to a single molecular target. The company has developed a unique combination of expertise across system and network biology, high-resolution data, and machine learning to unlock treatments in a large number of therapeutic areas.

    About Flagship Pioneering

    Flagship Pioneering conceives, creates, resources, and develops first-in-category life sciences companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $50B in aggregate value. To date, Flagship has deployed over $2.2B in capital toward the founding and growth of its pioneering companies alongside more than $18B of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Axcella Health (NASDAQ:AXLA), Codiak Biosciences (NASDAQ:CDAK), Denali Therapeutics (NASDAQ:DNLI), Evelo Biosciences (NASDAQ:EVLO), Foghorn Therapeutics (NASDAQ:FHTX), Indigo Ag, Kaleido Biosciences (NASDAQ:KLDO), Moderna (NASDAQ:MRNA), Rubius Therapeutics (NASDAQ:RUBY), Sana Biotechnology (NASDAQ:SANA), Seres Therapeutics (NASDAQ:MCRB), and Sigilon Therapeutics (NASDAQ:SGTX).

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/cellarity-raises-123-million-in-series-b-funding-to-pioneer-a-new-approach-to-drug-discovery-treating-disease-at-the-level-of-the-cell-as-opposed-to-a-single-molecular-target-301235332.html

    SOURCE Cellarity

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  17. Webcasts scheduled for February 26, March 9 and March 15

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will participate in the following upcoming investor conferences:

    • SVB Leerink 10th Annual Global Healthcare Conference: The company's fireside chat will be webcast on February 26, 2021 at 9:20 a.m. ET.
    • H.C. Wainwright Global Life Sciences Conference: The company's presentation will be webcast on March 9, 2021 at 7:00 a.m. ET.
    • 33rd Annual Roth Conference: The company's fireside chat will be webcast on March 15, 2021 at 9:00 a.m. ET.

    The audio webcasts will be accessible…

    Webcasts scheduled for February 26, March 9 and March 15

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will participate in the following upcoming investor conferences:

    • SVB Leerink 10th Annual Global Healthcare Conference: The company's fireside chat will be webcast on February 26, 2021 at 9:20 a.m. ET.
    • H.C. Wainwright Global Life Sciences Conference: The company's presentation will be webcast on March 9, 2021 at 7:00 a.m. ET.
    • 33rd Annual Roth Conference: The company's fireside chat will be webcast on March 15, 2021 at 9:00 a.m. ET.

    The audio webcasts will be accessible on the "Investors & News" section of the company's website, www.axcellahealth.com, for 90 days following the presentations.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

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    • AXA1665 Phase 2 trial initiation planned for Q2
    • AXA1125 Phase 2b trial initiation planned for Q2
    • Company decides not to expand enrollment in AXA4010-001 following cohort 1 readout
    • Pipeline expansion opportunities to be announced in 2021

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced clearance of the company's first investigational new drug (IND) filing and provided an update on its product candidates, research and development activities and expected milestones for 2021.

    "The year 2020 was a time of tremendous accomplishment for Axcella as we reported positive data for our lead…

    • AXA1665 Phase 2 trial initiation planned for Q2
    • AXA1125 Phase 2b trial initiation planned for Q2
    • Company decides not to expand enrollment in AXA4010-001 following cohort 1 readout
    • Pipeline expansion opportunities to be announced in 2021

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced clearance of the company's first investigational new drug (IND) filing and provided an update on its product candidates, research and development activities and expected milestones for 2021.

    "The year 2020 was a time of tremendous accomplishment for Axcella as we reported positive data for our lead candidates, AXA1665 and AXA1125, and prepared for their later stage clinical trials," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "We are excited to begin 2021 by announcing the clearance of our first investigational new drug (IND) filing, which enables us to advance AXA1665 into Phase 2 with a wealth of clinical data just four years after this candidate was designed. This validates our accelerated, highly informed development approach, which enables us to de-risk candidates and maximize our resources. As we enter what we expect to be a momentous 2021, our attention is firmly focused on clinical trial execution for our lead candidates and the expansion of our EMM composition pipeline."

    AXA1665 Program Update

    Axcella announced today that its IND application has been cleared by the U.S. Food and Drug Administration (FDA) for AXA1665, the company's oral product candidate for the reduction in risk of recurrent overt hepatic encephalopathy (OHE). In the second quarter of 2021, Axcella plans to initiate a Phase 2 trial of AXA1665.

    The Phase 2 trial will be a randomized, double-blind, placebo-controlled, multi-center study evaluating the efficacy and safety of AXA1665 in patients who have experienced at least one prior OHE event and have neurocognitive dysfunction at screening. Approximately 150 patients on either lactulose ± rifaximin (stratified by rifaximin use) will be enrolled and randomized 1:1 to receive either 53.8 grams per day of AXA1665 or a calorie-matched placebo in three divided doses for 24 weeks, with a two-week safety follow-up period.

    The trial will be conducted globally with the primary endpoint assessing change in the psychometric hepatic encephalopathy score (PHES) after 24 weeks of treatment. Secondary endpoints will include the proportion of patients experiencing an OHE breakthrough event; time to first OHE breakthrough event, including time to hospitalization; changes in physical function; and patient-reported outcomes. Other endpoints include measures of circulating ammonia, amino acids, and inflammation-related markers.

    "Over the course of the past few years, we have advanced AXA1665 from initial concept and design through two clinical studies enrolling more than 70 subjects with liver disease, demonstrating its multifactorial therapeutic potential," said Manu Chakravarthy, M.D., Ph.D., Chief Medical Officer of Axcella. "We are excited to now initiate a global Phase 2 clinical trial assessing AXA1665's efficacy in patients with OHE. Ultimately, our goal is to bring a new multi-targeted oral treatment option that improves upon today's standard of care for OHE patients and more comprehensively addresses their unmet needs."

    In 2020, Axcella reported positive top-line data from AXA1665-002. In this clinical study, AXA1665 was observed to be well tolerated with a strong safety profile. Dose-dependent changes were noted across measures of amino acid metabolism and neurocognition over 12 weeks in subjects with mild and moderate hepatic insufficiency. These included statistically-significant (p <0.05) improvements in the Fischer Ratio and the psychometric hepatic encephalopathy score (PHES) in the AXA1665 high dose arm vs. placebo. Additionally, clinically-relevant trends were seen in certain measures of nitrogen/ammonia handling and physical function in the AXA1665 arms versus placebo.

    AXA1125 Program Update

    Axcella completed a successful Type B pre-IND meeting with the FDA in late 2020 and is now working actively on its IND application for AXA1125, the company's oral product candidate for nonalcoholic steatohepatitis (NASH). Subject to FDA clearance, Axcella plans to proceed directly into a 48-week placebo-controlled Phase 2b paired biopsy clinical trial enrolling adult patients with biopsy-proven NASH, with the primary endpoint based on liver histology. This trial is expected to be initiated in the second quarter of 2021.

    In 2020, Axcella reported positive top-line data from AXA1125-003. In this clinical study, AXA1125 was observed to be well tolerated with a strong safety profile in subjects with presumed NASH. Additionally, sustained reductions versus placebo over 16 weeks were noted in virtually all key biomarkers of metabolism, inflammation and fibrosis, including MRI-PDFF, HOMA-IR, ALT and ProC3. Among subjects with type 2 diabetes enrolled in the study, reductions in most biomarkers were even greater versus placebo. Based on AXA1125's multi-targeted design and these data, Axcella believes this candidate holds the potential to serve as a first-line NASH monotherapy for both adult and pediatric patients and may be used in combination with other agents if required.

    AXA4010 Program Update

    AXA4010 has been under investigation in an open label pilot clinical study, AXA4010-001. An initial cohort of nine subjects with sickle cell disease (SCD) was enrolled and received AXA4010 in two 26-gram doses per day for up to 12 weeks. This first cohort was intended to allow the company to a) efficiently assess this candidate's safety, tolerability and effects on blood structure and function, including red blood cell metabolism/hemolysis, inflammation and vascular function/adhesion, and b) decide whether to expand enrollment to additional cohorts.

    Clinical data from this first cohort were recently received. Most of the reported adverse events, including all serious adverse events, were considered to be associated with underlying disease. Five subjects reported mild to moderate gastrointestinal events (diarrhea and nausea) that were considered to be possibly related to AXA4010. Biomarker data from this cohort suggest activity in targeted biologies, including generally positive trends in markers related to inflammation. Other markers related to hemolysis and vascular adhesion demonstrated greater variability. Based on the totality of the findings to date, Axcella has decided not to expand enrollment in the AXA4010-001 study.

    R&D Update

    Axcella sees the potential to develop a range of EMM compositions for a variety of diseases and conditions, including those related to metabolism, muscle atrophy, mitochondrial biology, neuroprotection, inflammation and immunology. The company has characterized approximately 75 potential therapeutic applications for EMMs and is now applying platform advances and program learnings to assess pipeline expansion opportunities.

    Axcella plans to provide an update on its research and development activities and share details about opportunities it has identified for pipeline expansion later in 2021. As demonstrated with AXA1125 and AXA1665, the company sees the opportunity for rapid, efficient and highly informed clinical development for its future product candidates.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical trials will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of AXA1665, AXA1125 and potential future EMM compositions, the potential for AXA1665 to reduce OHE events and improve the quality of life for cirrhotic patients, the potential for AXA1125 to serve as a first-line NASH monotherapy for both adult and pediatric patients and be used in combination with other agents if required, the design, status and timing of the company's planned Phase 2 clinical trial of AXA1665 and planned Phased 2b clinical trial of AXA1125, the timing and outcome of IND application submissions, the intended results of the company's strategy and approach and the company's ability to address other complex diseases and conditions utilizing EMM compositions. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its planned clinical trials of AXA1665 and AXA1125, other potential impacts of COVID-19 on the company's business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company's clinical trial initiation plans and timing, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of AXA1665 and AXA1125 and their therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company's target indications, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  18. Presentation and webcast to take place at 10 a.m. ET on January 14

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be presenting at the J.P. Morgan 2021 Healthcare Conference on January 14, 2021 at 10:00 a.m. ET.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 90 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic…

    Presentation and webcast to take place at 10 a.m. ET on January 14

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be presenting at the J.P. Morgan 2021 Healthcare Conference on January 14, 2021 at 10:00 a.m. ET.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 90 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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  19. Fireside chat at Piper Sandler 32nd Annual Virtual Healthcare Conference to be webcast on November 24

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be participating in the Piper Sandler 32nd Annual Virtual Healthcare Conference. The company will be webcasting its fireside chat at this event on November 24, 2020 at 9:00 a.m. ET.

    This webcast will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its…

    Fireside chat at Piper Sandler 32nd Annual Virtual Healthcare Conference to be webcast on November 24

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be participating in the Piper Sandler 32nd Annual Virtual Healthcare Conference. The company will be webcasting its fireside chat at this event on November 24, 2020 at 9:00 a.m. ET.

    This webcast will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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  20. Poster presentations highlight AXA1125's multifactorial activity; potentially differentiating and enhanced effects in subjects with type 2 diabetes

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today shared details about the company's poster presentations at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place virtually today through November 16, 2020. Both presentations feature data from AXA1125-003, a placebo-controlled, randomized clinical study that enrolled 102 subjects with presumed nonalcoholic steatohepatitis…

    Poster presentations highlight AXA1125's multifactorial activity; potentially differentiating and enhanced effects in subjects with type 2 diabetes

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today shared details about the company's poster presentations at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place virtually today through November 16, 2020. Both presentations feature data from AXA1125-003, a placebo-controlled, randomized clinical study that enrolled 102 subjects with presumed nonalcoholic steatohepatitis (NASH) to assess the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver for 16 weeks.

    "The results of AXA1125-003 were a key milestone for Axcella in which we were able to demonstrate clinically relevant multifactorial effects with AXA1125 in subjects with presumed NASH," said Manu Chakravarthy, M.D., Ph.D., Axcella's Chief Medical Officer and Executive Vice President of Clinical Development. "At The Liver Meeting this week, we are sharing further data from this study. Based on AXA1125's data to date and its multi-targeted mechanism of action, we believe this candidate is well positioned to be a first-line NASH therapy with potentially differentiating effects in type 2 diabetic subjects. We look forward to investigating its impact on liver histology in our upcoming serial biopsy Phase 2b clinical trial in patients with biopsy-proven NASH."

    Abstract 1663 is entitled "Biological Activity of AXA1125 and AXA1957 on Glucose, Insulin, HOMA-IR, and HbA1c and Measures of Liver Fat and Fibroinflammation in a Prospective 16-Week Randomized, Placebo-Controlled Study in Subjects with NAFLD and Type 2 Diabetes." This presentation highlights reductions seen in subjects receiving AXA1125 vs. placebo in markers of metabolism (MRI-PDFF and HOMA-IR) and fibroinflammation (cT1, ProC3 and FIB-4) as well as the reduction over time in ALT through 16 weeks. Additionally, the presentation shows the increased proportion of subjects receiving AXA1125 vs. placebo that achieved clinically relevant thresholds of improvement in specific markers, including:

    Threshold

    Subjects on Placebo

    Subjects on AXA1125

    ≥30% reduction in MRI-PDFF

    8.3%

    38.5%

    ≥17 U/L reduction in ALT

    25.0%

    38.5%

    ≥80 mSec reduction in cT1

    16.7%

    34.6%

    ≥2 ng/mL reduction in ProC3

    33.3%

    50.0%

    Abstract 1694 is entitled "Safety, Tolerability, and Biological Activity of AXA1125 and AXA1957 in a Prospective 16-Week Randomized, Placebo-Controlled Study in Subjects with NAFLD with and without Type 2 Diabetes." Focusing on data for subjects with both presumed NASH and type 2 diabetes (T2D), the presentation highlights reductions seen in those receiving AXA1125 vs. placebo in measures of fasting glucose, fasting insulin, HOMA-IR, HbA1C, MRI-PDFF, ALT, cT1 and ProC3. It also features the increased proportion of these subjects that achieved clinically relevant thresholds of improvement in specific markers, including:

    Threshold

    T2D Subjects on Placebo

    T2D Subjects on AXA1125

    ≥30% reduction in MRI-PDFF

    0%

    54.5%

    ≥17 U/L reduction in ALT

    33.3%

    63.6%

    ≥80 mSec reduction in cT1

    33.3%

    45.5%

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics and development potential of the company's EMM product candidates and the company's characterization of the results from its clinical studies and future clinical trials, including for AXA1125, the company's anticipated program milestones, including the design, status and timing of the planned Phase 2b clinical trial of AXA1125 in adult NASH, the subject and timing of the company's planned interactions with the FDA, including the timing of IND submissions, and the potential of the company's product candidates to impact health and/or disease, including AXA1125's potential in NASH. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical studies or IND-enabled clinical trials, other potential impacts of COVID-19 on business and financial results, including with respect to the ability to raise additional capital, make planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support the company's IND submission and clinical trial initiation plans and timing, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, past results from clinical studies not being representative of future results, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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    • Plan to advance AXA1125 directly into a Phase 2b biopsy clinical trial in NASH under IND following successful Type B pre-IND meeting with FDA
    • Plan to advance AXA1665 directly into a Phase 2 clinical trial in OHE under IND following positive top-line data from AXA1665-002
    • Published peer-reviewed manuscripts highlighting therapeutic benefit of EMMs and results from AXA1665-001 clinical study
    • Presented AXA1125 late-breaker at EASL Digital International Liver Congress
    • Company to hold conference call today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial…

    • Plan to advance AXA1125 directly into a Phase 2b biopsy clinical trial in NASH under IND following successful Type B pre-IND meeting with FDA
    • Plan to advance AXA1665 directly into a Phase 2 clinical trial in OHE under IND following positive top-line data from AXA1665-002
    • Published peer-reviewed manuscripts highlighting therapeutic benefit of EMMs and results from AXA1665-001 clinical study
    • Presented AXA1125 late-breaker at EASL Digital International Liver Congress
    • Company to hold conference call today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced financial results for the third quarter ended September 30, 2020 and provided a business update.

    "Recent accomplishments serve as further validation of the strength of Axcella's execution amid the COVID-19 pandemic as well as its clinical and regulatory approach," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "We are pleased to report that we recently completed a successful Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) that enabled us to affirm our plans to submit an Investigational New Drug (IND) application and initiate a Phase 2b biopsy clinical trial of AXA1125, our product candidate for nonalcoholic steatohepatitis (NASH). We also presented positive top-line data in the third quarter for AXA1665, our product candidate for the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Having already completed our pre-IND FDA engagement for AXA1665, preparations for its IND submission and Phase 2 clinical trial are now underway."

    "Just three years ago, Axcella finalized the designs of these differentiated, multi-targeted product candidates, and we now are prepared to enter later-stage clinical trials. Better still, we have compiled far more human data than would normally be expected at this stage of development, which we believe increases the probability of success in our next phase of development as we seek to make a difference for patients," Mr. Hinshaw continued. "With key catalysts for AXA1125 and AXA1665 as well as our decision regarding enrollment expansion for our AXA4010-001 clinical study on the near-term horizon, our excitement continues to build."

    Recent Accomplishments and Next Steps

    AXA1125

    • Completed Successful Type B Meeting: Axcella recently participated in a Type B pre-IND meeting with the FDA regarding AXA1125, focusing on key elements of the company's development strategy. This engagement enables Axcella to proceed with its IND submission and Phase 2b clinical trial design as planned.
    • Planned Phase 2b Clinical Trial: Axcella plans to submit an IND for AXA1125 and proceed directly into a 48-week Phase 2b serial biopsy clinical trial enrolling adult patients with NASH, with a primary endpoint based on liver histology. This trial is expected to be initiated in the first half of 2021.
    • Presented Late-Breaker at EASL for AXA1125: A late-breaker poster containing key findings from Axcella's AXA1125-003 clinical study was presented at the EASL Digital International Liver Congress by Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX and visiting professor of Hepatology at the University of Oxford, UK.
    • Planned Presentations at AASLD: Posters regarding AXA1125-003 will be presented at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place virtually November 13-16, 2020.

    AXA1665

    • Reported Positive Top-Line Data: Axcella reported top-line data from AXA1665-002, a 12-week clinical study assessing the impact of AXA1665 on safety, tolerability and physiology in subjects with mild and moderate hepatic insufficiency. Results from the study showed that AXA1665 was generally well-tolerated, with dose dependent changes noted across measures of amino acid metabolism and neurocognition. These included statistically significant (p <0.05) improvements in the Fischer Ratio and the psychometric hepatic encephalopathy score (PHES) in the AXA1665 high dose arm vs. placebo. Additionally, clinically relevant trends were seen in certain measures of nitrogen/ammonia handling and physical function in the AXA1665 arms versus placebo.
    • Planned Phase 2 Clinical Trial Initiation: Axcella plans to submit an IND for AXA1665 and proceed directly into a 24-week Phase 2 clinical trial enrolling subjects with liver cirrhosis who have experienced at least one prior OHE event. This trial is expected to be initiated in the first half of 2021.
    • Published Findings from Initial Investigation of AXA1665: Clinical and Translational Gastroenterology published a peer-reviewed report detailing results from Axcella's AXA1665-001 clinical study, the initial clinical investigation of AXA1665's effect on safety, tolerability, and clinically relevant biomarkers related to hepatic and muscle metabolism and function.

    AXA4010

    • Planned Enrollment Decision for AXA4010-001: Following Axcella's receipt of Cohort 1 data in December 2020 from the ongoing AXA4010-001 clinical study, the company expects to communicate its decision about whether to enroll additional subjects with sickle cell disease in the study by early 2021.

    EMM Platform

    • Published Manuscript Elucidating the Therapeutic Potential of EMMs: iScience published a peer-reviewed manuscript entitled "Endogenous Metabolic Modulators: Emerging Therapeutic Potential of Amino Acids" that detailed clinical precedents for EMMs as therapeutics and discussed the potential to develop EMM compositions that simultaneously target multiple biological pathways to address unmet needs in a range of complex diseases.

    Financial Results

    Cash Position: As of September 30, 2020, cash, cash equivalents and marketable securities totaled $117.3 million, compared to $92.1 million as of December 31, 2019. The increase is primarily the result of net proceeds from the company's follow-on stock offering that was completed in May 2020.

    R&D Expenses: Research and development expenses for the quarter and nine months ended September 30, 2020 were $7.5 million and $26.4 million, respectively. Research and development expenses for the same periods ended September 30, 2019 were $12.2 million and $29.1 million. The decrease for the three and nine months ended September 30, 2020 is primarily due to the completion of the company's AXA1125-003 and AXA1665-002 clinical studies.

    G&A Expenses: General and administrative expenses for the quarter and nine months ended September 30, 2020 were $4.2 million and $12.9 million, respectively. General and administrative expenses for the same periods ended September 30, 2019 were $4.8 million and $13.0 million.

    Net Loss: Net loss for the quarter and nine months ended September 30, 2020 was $12.4 million, or $0.34 per basic and diluted share, and $41.3 million, or $1.39 per basic and diluted share, respectively. This compares with a net loss of $17.3 million, or $0.75 per basic and diluted share, and $43.3 million, or $3.01 per basic and diluted share, for the quarter and nine months ended September 30, 2019. Included in the net loss for the quarter and the nine months ended September 30, 2020 was $1.4 million and $4.9 million, respectively, of non-cash expense related to stock-based compensation, as compared to $1.7 million and $4.3 million, respectively, for the same periods in 2019.

    Conference Call Reminder

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the company's financial results and other recent business updates. The conference call webcast will be accessible in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 30 days following the call.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of the company's product candidates and the company's characterization of the results from its clinical studies and future clinical trials, including for AXA1125 and AXA1665, the design, status and timing of the company's ongoing clinical study and planned IND-enabled clinical trials, the company's anticipated program milestones, including the timing of data readout from Cohort 1 of AXA4010-001, the subject and timing of the company's planned interactions with the FDA on the AXA1665 and AXA1125 programs, including the potential timing of IND application submissions for its product candidates, including AXA1125 and AXA1665, the potential of the company's product candidates to impact health and/or disease, including AXA1125's potential in NASH and AXA1665's potential in OHE, and the importance of any intellectual rights granted to the company. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials and planned interactions and submissions to FDA or other regulatory authorities, including planned IND submissions for AXA1125 and AXA1665, in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its ongoing AXA4010-001 clinical study and potential delays in disclosure of the same, other potential impacts of COVID-19 on the company's our business and financial results, including with respect to the company's ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support the company's IND submission and clinical trial initiation plans and timing, clinical trial design and target indications for AXA1125 and AXA1665, the clinical development and safety profile of the company's product candidates and their health or therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, past results from clinical studies not being representative of future results in clinical studies or IND-enabled clinical trials, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    September 30,

     

    December 31,

     

     

    2020

     

    2019

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    114,063

     

     

    $

    92,053

     

    Marketable securities

     

    3,192

     

     

     

    Other assets

     

    2,898

     

     

    2,306

     

    Total assets

     

    $

    120,153

     

     

    $

    94,359

     

    Liabilities and stockholders' equity:

     

     

     

     

    Liabilities

     

    $

    31,391

     

     

    $

    34,135

     

    Stockholders' equity

     

    88,762

     

     

    60,224

     

    Total liabilities and stockholders' equity

     

    $

    120,153

     

     

    $

    94,359

     

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations

    (in thousands, except share and per share data)

     

     

     

     

     

     

     

    Three Months Ended

    September 30,

     

    Nine Months Ended

    September 30,

     

    2020

     

    2019

     

    2020

     

    2019

    Operating expenses:

     

     

     

     

     

     

     

    Research and development

    $

    7,541

     

     

    $

    12,157

     

     

    $

    26,441

     

     

    $

    29,063

     

    General and administrative

    4,184

     

     

    4,840

     

     

    12,928

     

     

    13,036

     

    Total operating expenses

    11,725

     

     

    16,997

     

     

    39,369

     

     

    42,099

     

    Loss from operations

    (11,725)

     

     

    (16,997)

     

     

    (39,369)

     

     

    (42,099)

     

    Other income (expense), net

    (712)

     

     

    (307)

     

     

    (1,969)

     

     

    (1,225)

     

    Net loss

    $

    (12,437)

     

     

    $

    (17,304)

     

     

    $

    (41,338)

     

     

    $

    (43,324)

     

    Net loss per share, basic and diluted

    $

    (0.34)

     

     

    $

    (0.75)

     

     

    $

    (1.39)

     

     

    $

    (3.01)

     

    Weighted average common shares outstanding, basic and diluted

    36,942,475

     

     

    23,083,367

     

     

    29,804,034

     

     

    14,430,397

     

     

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  21. Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its third quarter financial results and other business updates on November 12, 2020. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international) five minutes prior to the start time. For those unable to listen in live, a…

    Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that it plans to report its third quarter financial results and other business updates on November 12, 2020. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast will be available in the Investors & News section on the company's website at www.axcellahealth.com. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 30 days following the call.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    View Full Article Hide Full Article
  22. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced two upcoming poster presentations at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place virtually November 13-16, 2020. These presentations will include key data from the company's AXA1125-003 clinical study, including in subjects with type 2 diabetes as well as the overall study population.

    • Abstract 1663: "Biological Activity of AXA1125 and AXA1957 on Glucose, Insulin, HOMA-IR, and HbA1c and Measures of Liver Fat and Fibroinflammation in a Prospective 16-Week…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced two upcoming poster presentations at The Liver Meeting® 2020, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place virtually November 13-16, 2020. These presentations will include key data from the company's AXA1125-003 clinical study, including in subjects with type 2 diabetes as well as the overall study population.

    • Abstract 1663: "Biological Activity of AXA1125 and AXA1957 on Glucose, Insulin, HOMA-IR, and HbA1c and Measures of Liver Fat and Fibroinflammation in a Prospective 16-Week Randomized, Placebo-Controlled Study in Subjects with NAFLD and Type 2 Diabetes"
    • Abstract 1694: "Safety, Tolerability, and Biological Activity of AXA1125 and AXA1957 in a Prospective 16-Week Randomized, Placebo-Controlled Study in Subjects with NAFLD with and without Type 2 Diabetes"

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor this portion of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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  23. iScience review paper details use of amino acids in medicine

    Axcella focused on designing novel EMM compositions with the potential to tackle complex diseases; plans to advance lead candidates into later-stage clinical trials

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced a new publication detailing the potential for the development of EMM compositions that simultaneously target multiple biological pathways and address unmet needs in a range of complex diseases. The publication in iScience is now available online.

    Amino acids and related metabolites and precursors are a class of EMMs…

    iScience review paper details use of amino acids in medicine

    Axcella focused on designing novel EMM compositions with the potential to tackle complex diseases; plans to advance lead candidates into later-stage clinical trials

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced a new publication detailing the potential for the development of EMM compositions that simultaneously target multiple biological pathways and address unmet needs in a range of complex diseases. The publication in iScience is now available online.

    Amino acids and related metabolites and precursors are a class of EMMs that have diverse biological functions. Entitled "Endogenous Metabolic Modulators: Emerging Therapeutic Potential of Amino Acids," the new publication reviews literature on the use of this EMM class to treat diseases, with a focus on single and combination agents targeted to a single biology.

    "This manuscript sheds light on a wealth of clinical precedents demonstrating the ability to utilize single and simple combinations of EMMs to reprogram biological pathways," said Michael Hamill, Head of Research at Axcella and the publication's lead author. "It also describes how, utilizing a rational design approach, compositions of EMMs provide the potential for compounding multifactorial effects on a range of complex diseases."

    Complex diseases, which tend to involve the dysregulation of a variety of underlying pathways and biologies, also are discussed in the publication. These diseases would be best addressed by safe multifactorial treatments rather than combination therapies that can raise the risk of drug-drug interactions and adverse effects.

    Axcella's EMM compositions are designed to enable a comprehensive approach, with the potential to simultaneously and safely target multiple pathways underlying diseases and regulate biological processes to foster overall health.

    "Individual EMMs have been utilized for decades to drive therapeutic activity with a well-established safety track record," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "At Axcella, we are seeking to harness the full power of EMMs by developing multi-targeted agents with the potential to tackle complex diseases like non-alcoholic steatohepatitis (NASH) and serious complications such as overt hepatic encephalopathy (OHE). Each of our five clinical studies to date investigating multiple product candidates have generated positive results, validating our approach and providing us with confidence in our ability to address unmet needs for patients with a range of diseases. We look forward to furthering our leadership in EMMs as we advance our product candidates into later-stage clinical trials."

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Approach

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics and development potential of the company's EMM product candidates, as well as compositions of EMMs in general, the company's characterization of the results from its clinical studies and future clinical trials, and the potential of the company's product candidates to impact health and/or treat disease, including NASH and OHE. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical studies or IND-enabled clinical trials, other potential impacts of COVID-19 on business and financial results, including with respect to the ability to raise additional capital, make planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support the company's IND submissions and clinical trial initiation plans and timing, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, past results from clinical studies not being representative of future results, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  24. Fireside chat and webcast at H.C. Wainwright 4th Annual NASH Investor Conference to take place on October 5

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be participating in a fireside chat at the H.C. Wainwright 4th Annual NASH Investor Conference on October 5, 2020 at 1:30 p.m. ET.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its…

    Fireside chat and webcast at H.C. Wainwright 4th Annual NASH Investor Conference to take place on October 5

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions, today announced that management will be participating in a fireside chat at the H.C. Wainwright 4th Annual NASH Investor Conference on October 5, 2020 at 1:30 p.m. ET.

    A live audio webcast of this discussion will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases and improve health using endogenous metabolic modulator (EMM) compositions. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    View Full Article Hide Full Article
  25. Axcella (NASDAQ:AXLA) is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Company:

    Axcella Health Inc.

     

     

    Axcella (NASDAQ:AXLA) is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Company:

    Axcella Health Inc.

     

     

    Headquarters Address:

    840 Memorial Dr., Third Floor

     

    CAMBRIDGE, MA 02139

     

     

    Main Telephone:

    857-320-2200

     

     

    Website:

    www.axcellahealth.com

     

     

    Ticker:

    AXLA(NASDAQ)

     

     

    Type of Organization:

    Public

     

     

    Industry:

    Biotechnology

     

     

    Key Executives:

    CEO: Bill Hinshaw

     

     

    Investor Relations

     

    Contact:

    Jason Fredette

    Phone:

    7814240946

    Email:

    jfredette@axcellahealth.com

     

    View Full Article Hide Full Article
  26. Presentations and webcasts to take place on September 14 and 15

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that management will be presenting at the following upcoming conferences:

    • H.C. Wainwright 22nd Annual Global Investment Conference, on September 14, 2020, at 2:00 p.m. ET
    • Cantor Virtual Global Healthcare Conference, on September 15, 2020, at 4:40 p.m. ET

    Live audio webcasts of these presentations will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. Replays will also be available on Axcella's website for 30 days…

    Presentations and webcasts to take place on September 14 and 15

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that management will be presenting at the following upcoming conferences:

    • H.C. Wainwright 22nd Annual Global Investment Conference, on September 14, 2020, at 2:00 p.m. ET
    • Cantor Virtual Global Healthcare Conference, on September 15, 2020, at 4:40 p.m. ET

    Live audio webcasts of these presentations will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. Replays will also be available on Axcella's website for 30 days following the presentations.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor this portion of the company's website, in addition to its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    View Full Article Hide Full Article
  27. Poster presentation contains data demonstrating AXA1125's multifactorial effects on markers of metabolism, inflammation and fibrosis in subjects with NAFLD

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, announced that data are being presented today in a late-breaker poster regarding key findings from its AXA1125-003 clinical study by Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK. The poster presentation is entitled "Multifactorial Effects of AXA1125 and AXA1957 Observed on Markers…

    Poster presentation contains data demonstrating AXA1125's multifactorial effects on markers of metabolism, inflammation and fibrosis in subjects with NAFLD

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, announced that data are being presented today in a late-breaker poster regarding key findings from its AXA1125-003 clinical study by Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK. The poster presentation is entitled "Multifactorial Effects of AXA1125 and AXA1957 Observed on Markers of Metabolism, Inflammation and Fibrosis: A 16-Week Randomized Placebo-Controlled Study in Subjects With Nonalcoholic Fatty Liver Disease (NAFLD) With and Without Type 2 Diabetes (T2D)."

    "Based on the multifactorial effects seen in initial clinical investigations and its well-tolerated profile to date, AXA1125 is among the most intriguing candidates in development for nonalcoholic steatohepatitis (NASH)," said Dr. Harrison. "Notable reductions in virtually all non-invasive biomarkers – including liver fat content (MRI-PDFF), alanine aminotransferase (ALT) and corrected T1 (cT1) – were seen in subjects receiving AXA1125 versus placebo in the 003 clinical study. Interestingly, mean reductions in these measures were even greater among subjects with T2D, providing the potential for a highly differentiated profile within the NASH field."

    AXA1125-003 was a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver. A total of 102 adult subjects with presumed NASH were enrolled and dosed in a 2:2:2:1 ratio to receive AXA1125, one of two AXA1957 doses, or placebo administered twice daily for 16 weeks. Study subjects were stratified based on the presence or absence of T2D, with approximately 40 percent of the subjects having T2D.

    Highlights from the presentation include:

    • The AXA1125 study arm consistently achieved numerically greater reductions from baseline in biomarkers of liver fat and fibroinflammation versus placebo, with AXA1957 showing activity in a number of key biomarkers, but with less consistent directional change than AXA1125
    • A reduction from baseline was seen in the AXA1125 arm compared with placebo at weeks 8 and 16 in:
      • Liver fat biomarker MRI-PDFF
      • HOMA-IR
      • ALT
      • CK-18 M65
      • ProC3
      • cT1
    • In the AXA1125 arm, 39 percent of subjects achieved a ≥30 percent relative reduction in MRI-PDFF, 39 percent achieved a ≥17 U/L reduction in ALT from baseline, and 54 percent achieved a ≥40 millisecond absolute reduction in cT1
    • AXA1125 and AXA1957 were both generally well tolerated in the study, with low rates of discontinuations and with all product-emergent adverse events being mild or moderate

    As announced earlier this year in conjunction with Axcella's top-line data announcement, the company plans to advance AXA1125 into a Phase 2b clinical trial in adult NASH in the first half of 2021.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Ongoing Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics and development potential of the company's EMM product candidates and the company's characterization of the results from its clinical studies and future clinical trials, including for AXA1125, the company's anticipated program milestones, including the design, status and timing of the planned Phase 2b clinical trial of AXA1125 in adult NASH, the subject and timing of the company's planned interactions with the FDA, including the timing of IND submissions, and the potential of the company's product candidates to impact health and/or disease, including AXA1125's potential in NASH. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical studies or IND-enabled clinical trials, other potential impacts of COVID-19 on business and financial results, including with respect to the ability to raise additional capital, make planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support the company's IND submission and clinical trial initiation plans and timing, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, past results from clinical studies not being representative of future results, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  28. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced an upcoming late-breaker poster presentation at The Digital International Liver Congress™ 2020 (EASL), which is being held virtually from August 27 to 29. This presentation will discuss key data from AXA1125-003, a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver in 102 adult subjects with non-alcoholic fatty liver disease (NAFLD).

    Details of the presentation are as follows:

    Title: Multifactorial…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced an upcoming late-breaker poster presentation at The Digital International Liver Congress™ 2020 (EASL), which is being held virtually from August 27 to 29. This presentation will discuss key data from AXA1125-003, a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver in 102 adult subjects with non-alcoholic fatty liver disease (NAFLD).

    Details of the presentation are as follows:

    Title: Multifactorial Effects of AXA1125 and AXA1957 Observed on Markers of Metabolism, Inflammation and Fibrosis: A 16-Week Randomized Placebo-Controlled Study in Subjects With Nonalcoholic Fatty Liver Disease (NAFLD) With and Without Type 2 Diabetes (T2D)

    Abstract Number: 4470

    Presenter: Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK

    Session: Late-Breaker Poster

    Date/Time: August 28, 9:30 a.m. to 7:30 p.m. CEST

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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  29. Data support AXA1665's potential to address hyperammonemia, amino acid dysregulation and physical dysfunction in subjects with cirrhosis

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the publication of results from the company's AXA1665-001 clinical study in Clinical and Translational Gastroenterology entitled "Safety, tolerability, and physiological effects of AXA1665, a novel composition of amino acids, in subjects with Child–Pugh A and B cirrhosis." The AXA1665-001 study, which was completed in late 2018, investigated AXA1665's effect on safety, tolerability, and biomarkers…

    Data support AXA1665's potential to address hyperammonemia, amino acid dysregulation and physical dysfunction in subjects with cirrhosis

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the publication of results from the company's AXA1665-001 clinical study in Clinical and Translational Gastroenterology entitled "Safety, tolerability, and physiological effects of AXA1665, a novel composition of amino acids, in subjects with Child–Pugh A and B cirrhosis." The AXA1665-001 study, which was completed in late 2018, investigated AXA1665's effect on safety, tolerability, and biomarkers related to hepatic and muscle metabolism and function. Axcella also recently reported positive top-line data from its second clinical study of AXA1665, AXA1665-002.

    "Excess ammonia, amino acid imbalance and muscle wasting are often underlying contributing factors that impact various complications of cirrhosis, including overt hepatic encephalopathy, which is typically marked by significant cognitive dysfunction," says Dr. Manu Chakravarthy, Chief Medical Officer of Axcella. "We believe that AXA1665 holds the potential to address these metabolic derangements in an integrated and well-tolerated manner. In this paper, we report on the findings from our initial clinical experience with this oral product candidate in subjects with Child-Pugh A and B cirrhosis."

    AXA1665-001 was a domiciled and controlled crossover clinical study that examined two AXA1665 doses (14.7g/day and 44.1g/day), each over a two-week period. Overall, the results showed that both doses of AXA1665 were well tolerated, with no product-related adverse events observed. AXA1665's effects on hepatic and muscle physiology were measured using clinically relevant biomarkers. Results suggest AXA1665's potential to mitigate the multifactorial defects of hyperammonemia, dysregulated amino acid metabolism, and physical dysfunction. Key physiological findings in subjects receiving the higher dose of AXA1665 for 15 days as compared to the control group were:

    • An increase in Fischer Ratio (branched chain amino acids ÷ aromatic amino acids) of approximately 40%, indicating improved amino acid balance.
    • A decreased trend in mean fasted plasma ammonia concentration despite provision of additional nitrogen via the amino acids in AXA1665, suggesting improved nitrogen handling.
    • A decrease in the liver frailty index, suggesting improved physical function, accompanied by trends toward a leaner body composition.

    "This publication follows our presentations at EASL and AASLD last year and the recent positive data readout for our second clinical study of AXA1665, helping to raise further awareness about Axcella's pioneering work to develop multi-targeted agents utilizing distinct compositions of EMMs," explains Bill Hinshaw, President and CEO of Axcella. "The AXA1665-001 study was our first opportunity to demonstrate this product candidate's multifactorial potential, and we were excited to build on this body of knowledge in AXA1665-002 by also showing positive changes in neurocognitive measures. We look forward to initiating our planned Phase 2 clinical trial of AXA1665 in patients with advanced liver disease in the first half of 2021."

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Ongoing Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics and development potential of the company's EMM product candidates and the company's characterization of the results from its clinical studies and future clinical trials, including for AXA1665, the company's anticipated program milestones, including the design, status and timing of an IND-enabled clinical trial of AXA1665, the subject and timing of the company's planned interactions with the FDA on its AXA1665 program, including the timing of IND submissions, and the potential of the company's product candidates to impact health and/or disease, including AXA1665's potential in advanced liver disease. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its AXA1665-002 clinical study, other potential impacts of COVID-19 on business and financial results, including with respect to the ability to raise additional capital, make planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support our IND submission and clinical trial initiation plans and timing, clinical trial design and target indication for AXA1665, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, past results from clinical studies not being representative of future results, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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    • Top-line data from AXA1665-002 show dose dependent improvements in markers of cognitive function and amino acid metabolism; both AXA1665 doses safe and well tolerated for 12 Weeks; plan to initiate Phase 2 clinical trial under IND in 1H 2021
    • Reported positive top-line data for AXA1125, Axcella's NASH product candidate, with clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation; plan to initiate Phase 2b clinical trial under IND in 1H 2021
    • Completed follow-on equity offering, raising approximately $60 million in gross proceeds
    • Company to hold conference call today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators…

    • Top-line data from AXA1665-002 show dose dependent improvements in markers of cognitive function and amino acid metabolism; both AXA1665 doses safe and well tolerated for 12 Weeks; plan to initiate Phase 2 clinical trial under IND in 1H 2021
    • Reported positive top-line data for AXA1125, Axcella's NASH product candidate, with clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation; plan to initiate Phase 2b clinical trial under IND in 1H 2021
    • Completed follow-on equity offering, raising approximately $60 million in gross proceeds
    • Company to hold conference call today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today reported positive top-line 12-week data from AXA1665-002, a placebo-controlled clinical study of AXA1665, and financial results for the second quarter ended June 30, 2020.

    "The second quarter of 2020 was a time of significant accomplishment for Axcella," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "This period was highlighted by positive top-line data from our AXA1125-003 clinical study, which served as another strong validator for our novel EMM platform. Preparations are now well underway for our engagement with the U.S. Food and Drug Administration (FDA) regarding our adult and pediatric NASH programs as we seek to finalize the design of our proposed Phase 2b clinical trial of AXA1125 and initiate enrollment in the first half of 2021. We are appreciative of the support from the investors that enabled us to bolster our balance sheet via a follow-on equity offering in May 2020."

    "Today, we are pleased to share our AXA1665-002 top-line data that once again demonstrate safety, tolerability and activity across multiple biologies and, for the first time, show positive changes in neurocognitive measures. After additional data analyses and consultation with external medical experts, we plan to submit an IND and initiate a Phase 2 clinical trial of AXA1665 in patients with advanced liver disease. Ultimately, our goal is to provide a much-needed new treatment option for the many patients who have experienced an overt hepatic encephalopathy (OHE) event. We extend our sincere thanks to all of the subjects and investigators involved in AXA1665-002 for their participation in and commitment to this study, particularly in light of the ongoing global pandemic," Mr. Hinshaw concluded.

    AXA1665-002 Top-Line Data

    AXA1665-002 was a placebo-controlled, randomized clinical study that was designed to investigate the safety, tolerability and physiological impact of AXA1665, a proprietary composition of eight amino acids, in 60 subjects with mild (Child Pugh A) and moderate (Child Pugh B) hepatic insufficiency. Subjects in the study were randomized in a 2:2:1 ratio to receive either 29.4 g or 53.9 g of AXA1665 or a matched placebo in three divided doses per day for 12 weeks with a four-week follow up. In addition to safety and tolerability, the study evaluated plasma amino acid and ammonia levels as well as markers of neurocognition, muscle structure and function.

    "We believe the data from the AXA1665-002 study show AXA1665's potential to address multiple fundamental dysregulations associated with cirrhosis and hepatic encephalopathy," said Manu Chakravarthy, M.D., Ph.D., Chief Medical Officer of Axcella. "This study replicated findings on amino acid metabolism from our previous short-term study, AXA1665-001, and we were pleased to see those effects sustained through 12 weeks. We also noted dose dependent, directionally consistent changes across all three psychometric tests that were utilized, which help to bolster our confidence in AXA1665's potential to reduce OHE events. We look forward to initiating a Phase 2 clinical trial to investigate this hypothesis and further evaluate this candidate's impact on measures of physical function and related patient reported outcomes in patients with advanced liver disease."

    Key results from AXA1665-002 include:

    • Safety/Tolerability: Both doses of AXA1665 were safe and well-tolerated. Rates of adverse events (AEs) were low, mostly unrelated to study product and generally mild or moderate. There were four serious adverse events reported in the study and two deaths (one due to complications of COVID-19; one due to a myocardial infarction during the study run-in period prior to dosing), none of which were determined to be related to AXA1665.
    • Neurocognitive Function: Positive, dose dependent trends were observed in the AXA1665 arms across all three psychometric tests: Stroop EncephalApp, critical flicker frequency, and psychometric hepatic encephalopathy score (PHES). In PHES, a highly specific assessment to diagnose minimal hepatic encephalopathy (MHE), a statistically significant (p <0.05) improvement of a clinically relevant magnitude was observed in the AXA1665 high dose arm vs. placebo. Additionally, the proportion of subjects achieving a clinically relevant threshold of PHES improvement was higher in the AXA1665 arms relative to placebo.
    • Amino Acid Metabolism: A dose dependent and statistically significant (p <0.05) percentage increase from baseline in Fischer Ratio (FR; a measure of branched chain amino acids ÷ aromatic amino acids) was seen in the AXA1665 arms relative to placebo (low dose: 21%, high dose: 44%), which was sustained over 12 weeks. Observed changes in FR were accompanied by concomitant decreases in circulating aromatic amino acids (Phe and Tyr), which may suggest their incorporation into protein synthesis and an improved metabolic state. Published studies suggest that aromatic amino acids may contribute to impaired neurotransmission and have correlated lower FR with poor clinical outcomes in patients with cirrhosis and end-stage liver disease.
    • Ammonia Handling: Despite the increased nitrogen load delivered via the amino acids in AXA1665, fasted plasma ammonia levels remained stable in the active arms over the 12-week dosing duration. In a subset of subjects with evidence of MHE at baseline as assessed by PHES, a mean reduction from baseline of approximately 7% in fasted plasma ammonia levels was observed in subjects receiving both doses of AXA1665 at week 12.
    • Muscle Structure and Function: Key measures of muscle structure (e.g. lean mass) and function (e.g. gait speed, liver frailty index, or LFI) remained essentially stable in all groups from baseline to week 12. This observation may reflect the mild hepatic insufficiency and lack of overt sarcopenia in nearly all enrolled subjects at baseline. A higher proportion of subjects in the AXA1665 arms achieved a ≥0.3 absolute reduction in LFI (i.e. less frailty) versus placebo. Previous studies suggest that a ≥0.3 reduction in the LFI score may correlate with an improved ability to conduct activities of daily living in subjects with end-stage liver disease.

    "Overt hepatic encephalopathy is a complex disease that involves dysregulation across multiple organ systems, including altered amino acids, elevated ammonia levels, dysregulated muscle metabolism, and cognitive dysfunction," said Dr. Arun Sanyal, Professor of Medicine, Physiology and Molecular Pathology at Virginia Commonwealth University School of Medicine, and an investigator in AXA1665-002. "While previous approaches have focused on reducing ammonia load from the bowel alone, future approaches should incorporate the evolving knowledge of the role of other organs, such as muscle and the brain, in the development of encephalopathy. I am encouraged by the multifactorial activity seen with AXA1665 in the 002 study and look forward to its continued investigation in a subsequent Phase 2 trial to evaluate AXA1665's therapeutic potential to prevent OHE recurrence in advanced liver disease patients who are in dire need of new treatment options."

    Additional data will be discussed during the company's conference call at 8:30 a.m. ET today and will be included in a presentation that will be posted to "Investors & News" section of Axcella's website prior to the call. Details about how to access this conference call are included below.

    Other Recent Developments

    • AXA1125 Top-Line Data: Reported positive top-line data from AXA1125-003, a clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and physiology in subjects with non-alcoholic fatty liver disease (NAFLD). Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to both placebo and AXA1957, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers, with a greater magnitude noted among subjects with type 2 diabetes.
    • AXA1665 Patents: Announced the issuance of two key patents: U.S. Patent 10,682,325 and U.S. Patent 10,660,870. These are the first patents related to Axcella's family of applications for AXA1665, the company's product candidate for the reduction in risk of overt hepatic encephalopathy recurrence, covering both its composition and methods of use. These patents follow the issuance of composition and methods of use patents for Axcella's other lead product candidate, AXA1125, in 2019.
    • Follow-On Stock Offering: Axcella closed an underwritten public offering of an aggregate of 12,650,000 shares of its common stock, including the full exercise of the underwriters' option to purchase additional shares. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Axcella, were approximately $60.1 million.
    • Addition to Board of Directors: The company also today announced that Chief Development Officer Shreeram Aradhye, M.D., will be stepping down from his full-time role to accept another opportunity and will be appointed to Axcella's Board of Directors, effective on September 1, 2020. Dr. Aradhye has more than 20 years of pharmaceutical industry experience in clinical development and medical affairs, having previously served as Chief Medical Officer and Global Head, Medical Affairs for Novartis Pharmaceuticals. In previous roles at Novartis and Sandoz, he provided functional leadership for clinical development and medical affairs teams working on novel and biosimilar medicines across multiple indications, including multiple sclerosis, Alzheimer's Disease, neuropathic pain, muscle disease and migraine.
    • Management Update: Chief Medical Officer Manu Chakravarthy, M.D., Ph.D., has been promoted to Executive Vice President and will assume Dr. Aradhye's day-to-day responsibilities. Additionally, Andrew Suchoff joined Axcella as Chief People Officer in June 2020. He is driving initiatives aimed at the company's culture, talent, learning and development, employee engagement and compensation. Mr. Suchoff brings more than 20 years of human resources leadership experience to Axcella, having most recently served as Global Head of People Operations and Talent Development at Stallergenes Greer, a healthcare company with more than 1,200 employees and operations throughout the world.

    Upcoming Planned Milestones

    Liver Programs

    • Q3 2020: Present AXA1125-003 data (late-breaker poster presentation) at The Digital International Liver Congress 2020 (EASL)
    • 2H 2020: Engage with the FDA regarding the company's planned IND application for AXA1125, proposed Phase 2b clinical trial in adults and pediatric development program
    • 1H 2021: Initiate a Phase 2b clinical trial of AXA1125 in adult NASH under an IND
    • 1H 2021: Initiate a Phase 2 clinical trial of AXA1665 in patients with advanced liver disease (i.e. in cirrhotic subjects with at least one prior episode of OHE) under an IND

    Blood Program

    • Q4 2020: Report top-line data from Cohort 1 of AXA4010-001, a clinical study on safety, tolerability and blood physiology in subjects with sickle cell disease

    Financial Results

    R&D Expenses: Research and development expenses were $8.6 million and $9.3 million for the quarters ended June 30, 2020 and 2019, respectively. The change was primarily related to the completion of the company's AXA1125-003 clinical study.

    G&A Expenses: General and administrative expenses were $4.6 million and $4.7 million for the quarters ended June 30, 2020 and 2019, respectively.

    Net Loss: Net loss for the quarter ended June 30, 2020 was $13.9 million, or $0.48 per basic and diluted share. This compares with a net loss of $14.4 million, or $0.95 per basic and diluted share, for the quarter ended June 30, 2019.

    Cash Position: Cash and cash equivalents at June 30, 2020 were $121.3 million, which compares with $92.1 million at December 31, 2019. The increase is primarily the result of net proceeds from the company's follow-on stock offering that was completed in May 2020.

    Conference Call Reminder

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the top-line data from AXA1665-002 and other recent business updates. The conference call webcast and accompanying slides will be made available shortly before the start of the call on the company's website at www.axcellahealth.com in the Investors & News section. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 30 days following the call.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of the company's EMM product candidates and the company's characterization of the results from its clinical studies and future clinical trials, including for AXA1125 and AXA1665, the design, status and timing of the company's ongoing clinical studies and planned IND-enabled clinical trials, the company's anticipated program milestones, including the timing of data readout from Cohort 1 of AXA4010-001, the subject and timing of the company's planned interactions with the FDA on the AXA1665 and AXA1125 programs, including the potential timing of IND application submissions, the potential of the company's product candidates to impact health and/or disease, including AXA1125's potential in NASH and AXA1665 potential in OHE, and the importance of any intellectual rights granted to the company. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND-enabled clinical trials and planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data we are able to collect in our ongoing AXA4010-001 clinical study and potential delays in disclosure of the same, other potential impacts of COVID-19 on our business and financial results, including with respect to our ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support our IND submission and clinical trial initiation plans and timing, clinical trial design and target indications for AXA1125 and AXA1665, the clinical development and safety profile of the company's product candidates and their health or therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, past results from clinical studies not being representative of future results in clinical studies or IND-enabled clinical trials, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    June 30,

     

    December 31,

     

     

    2020

     

    2019

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    121,326

     

     

    $

    92,053

     

    Other assets

     

    3,222

     

     

    $

    2,306

     

    Total assets

     

    $

    124,548

     

     

    $

    94,359

     

    Liabilities and stockholders' equity (deficit):

     

     

     

     

    Liabilities

     

    $

    31,585

     

     

    $

    34,135

     

    Stockholders' equity (deficit)

     

    92,963

     

     

    $

    60,224

     

    Total liabilities and stockholders' equity

     

    $

    124,548

     

     

    $

    94,359

     

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss

    (in thousands, except share and per share data)

     

     

     

     

     

     

     

    Three Months Ended

    June 30,

     

    Six Months Ended

    June 30,

     

    2020

     

    2019

     

    2020

     

    2019

    Operating expenses:

     

     

     

     

     

     

     

    Research and development

    $

    8,565

     

     

    $

    9,343

     

     

    $

    10,335

     

     

    $

    7,563

     

    General and administrative

    4,619

     

     

    4,728

     

     

    4,125

     

     

    3,468

     

    Total operating expenses

    13,184

     

     

    14,071

     

     

    14,460

     

     

    11,031

     

    Loss from operations

    (13,184

    )

     

    (14,071

    )

     

    (14,460

    )

     

    (11,031

    )

    Other income (expense), net

    (708

    )

     

    (376

    )

     

    (549

    )

     

    (542

    )

    Net loss and comprehensive loss

    $

    (13,892

    )

     

    $

    (14,447

    )

     

    $

    (15,009

    )

     

    $

    (11,573

    )

    Net loss per share, basic and diluted

    $

    (0.48

    )

     

    $

    (0.95

    )

     

    $

    (0.65

    )

     

    $

    (2.43

    )

    Weighted average common shares outstanding, basic and diluted

    29,202,367

     

     

    15,230,815

     

     

    23,188,816

     

     

    4,775,828

     

     

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  30. Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that it plans to report its second quarter financial results and other business updates on August 5, 2020. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast and accompanying slides will be made available shortly before the start of the call on the company's website at www.axcellahealth.com in the Investors & News section. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international…

    Conference call to take place at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that it plans to report its second quarter financial results and other business updates on August 5, 2020. The company will host a conference call at 8:30 a.m. ET that morning.

    The conference call webcast and accompanying slides will be made available shortly before the start of the call on the company's website at www.axcellahealth.com in the Investors & News section. To access the call via telephone, please dial (866) 652-5200 (U.S. toll free) or (412) 317-6060 (international) five minutes prior to the start time. For those unable to listen in live, a webcast archive will be available on the company's website for 30 days following the call.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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    • Both of Axcella's lead product candidates, AXA1665 and AXA1125, now covered under U.S. patents for composition of matter and methods of use
    • Expansion of Axcella's patent estate continues

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the issuance of two new patents: U.S. Patent 10,682,325 and U.S. Patent 10,660,870. These are the first patents related to Axcella's family of applications for AXA1665, the company's product candidate for the reduction in risk of overt hepatic encephalopathy recurrence.

    U.S. Patent 10,682,325 covers the composition of AXA1665 as well as other…

    • Both of Axcella's lead product candidates, AXA1665 and AXA1125, now covered under U.S. patents for composition of matter and methods of use
    • Expansion of Axcella's patent estate continues

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the issuance of two new patents: U.S. Patent 10,682,325 and U.S. Patent 10,660,870. These are the first patents related to Axcella's family of applications for AXA1665, the company's product candidate for the reduction in risk of overt hepatic encephalopathy recurrence.

    U.S. Patent 10,682,325 covers the composition of AXA1665 as well as other related EMM compositions. U.S. Patent 10,660,870 focuses on methods of use for these EMM compositions in treating a liver disease or disorder involving hyperammonemia and/or muscle wasting.

    "Axcella's goal is to build the world's leading portfolio of EMM compositions, backed by strong intellectual property," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "Over the course of the past 18 months, we have grown our number of patent families to 19, covering both our product candidates as well as our platform. We also have more than tripled our number of patent applications to nearly 200 and received our first six patent issuances covering AXA1665, AXA1125, AXA4010 and AXA2678. These accomplishments position us well as we approach potential advanced clinical trials for our lead candidates, AXA1665 and AXA1125."

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's clinical studies to date, including AXA1125-003, are or have been conducted as non-investigational new drug application (IND) clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, any subsequent clinical studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of the company's EMM product candidates and its platform, the coverage that the company's expanded patent portfolio provides to its product candidates and platform and the potential to approach advanced clinical trials for AXA1665 and AXA1125. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of any future challenges to the company's patents, the scope of and ability to maintain patent protection, the ability of the patent portfolio to protect the company's product candidates and platform, the potential impacts of COVID-19 on our business and financial results, whether data readouts and/or U.S. Food and Drug Administration feedback support our plans and timing for IND filings, clinical trial design and target indications for AXA1665 and AXA1125, the clinical development and safety profile of the company's product candidates and their health or therapeutic potential, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  31. Presentation and webcast to take place at 3:50 p.m. ET on June 10

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that management will be presenting at the Goldman Sachs 41st Annual Global Healthcare Conference, which is being held virtually, on June 10, 2020 at 3:50 p.m. ET.

    A live audio webcast of the presentation will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com

    Presentation and webcast to take place at 3:50 p.m. ET on June 10

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced that management will be presenting at the Goldman Sachs 41st Annual Global Healthcare Conference, which is being held virtually, on June 10, 2020 at 3:50 p.m. ET.

    A live audio webcast of the presentation will be available on the "Investors & News" section of the company's website, www.axcellahealth.com. A replay will also be available on Axcella's website for 30 days following the presentation.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors & News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

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  32. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the closing of its underwritten public offering of an aggregate of 12,650,000 shares of its common stock, including the full exercise of the underwriters' option to purchase additional shares, at a public offering price of $4.75 per share, before deducting underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Axcella, were approximately $60.1 million.

    Axcella intends to use the net proceeds from the offering…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the closing of its underwritten public offering of an aggregate of 12,650,000 shares of its common stock, including the full exercise of the underwriters' option to purchase additional shares, at a public offering price of $4.75 per share, before deducting underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Axcella, were approximately $60.1 million.

    Axcella intends to use the net proceeds from the offering, together with its existing cash and cash equivalents, to advance its current liver programs, including its planned IND filing for AXA1665 and ensuing initiation of a Clinical Trial and its planned IND filing for AXA1125 in adults and pediatric patients and ensuing initiation of Clinical Trials; advance its product candidate AXA4010, including the conclusion of its ongoing Clinical Study; advance its development platform and discovery efforts; and support organizational growth and for working capital and other general corporate purposes. J.P. Morgan and SVB Leerink acted as joint book-running managers for the offering. Wedbush PacGrow acted as lead manager and Roth Capital Partners acted as co-manager for the offering.

    A registration statement relating to these securities was filed with the Securities and Exchange Commission ("SEC") on May 11, 2020 and was declared effective by the SEC on May 13, 2020. The offering was made only by means of a prospectus. These documents were filed with the SEC and may be obtained on the SEC's website at http://www.sec.gov. You can also obtain the prospectus by contacting J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, telephone: (866) 803-9204, or email: prospectus-eq_fi@jpmchase.com; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6218, or email: syndicate@svbleerink.com.

    This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any offer or sale of these securities in any state or 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 jurisdiction.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development.

    Forward Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the use of proceeds of the offering. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the success, cost, and timing of the company's product candidate development activities and planned clinical studies and clinical trials; the company's ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies or clinical trials; regulatory developments in the United States and foreign countries; the company's estimates regarding expenses, future revenue, and capital requirements, and other financial results; as well as those risks and uncertainties set forth in the company's most recent Quarterly Report on Form 10-Q and subsequent filings with the Securities and Exchange Commission. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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  33. Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the pricing of its underwritten public offering of 11,000,000 shares of its common stock at a public offering price of $4.75 per share, before deducting underwriting discounts and commissions. In addition, Axcella has granted the underwriters a 30-day option to purchase up to an additional 1,650,000 shares of common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other estimated offering expenses…

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced the pricing of its underwritten public offering of 11,000,000 shares of its common stock at a public offering price of $4.75 per share, before deducting underwriting discounts and commissions. In addition, Axcella has granted the underwriters a 30-day option to purchase up to an additional 1,650,000 shares of common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Axcella, are expected to be approximately $52.3 million, excluding any exercise of the underwriters' option to purchase additional shares. The offering is expected to close on May 18, 2020, subject to the satisfaction of customary closing conditions.

    Axcella intends to use the net proceeds from the offering, together with its existing cash and cash equivalents, to advance its current liver programs, including its planned IND filing for AXA1665 and ensuing initiation of a Clinical Trial and its planned IND filing for AXA1125 in adults and pediatric patients and ensuing initiation of Clinical Trials; advance its product candidate AXA4010, including the conclusion of its ongoing Clinical Study; advance its development platform and discovery efforts; and support organizational growth and for working capital and other general corporate purposes. J.P. Morgan and SVB Leerink are acting as joint book-running managers for the offering. Wedbush PacGrow is acting as lead manager and Roth Capital Partners is acting as co-manager for the offering.

    A registration statement relating to these securities was filed with the Securities and Exchange Commission ("SEC") on May 11, 2020 and was declared effective by the SEC on May 13, 2020. The offering is being made only by means of a prospectus. Before you invest, you should read the prospectus and the other documents the issuer has filed with the SEC for more complete information about the issuer and the risks associated with investing in the offering. These documents have been filed with the SEC and may be obtained on the SEC's website at http://www.sec.gov. You can also obtain the prospectus, when available, by contacting J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, telephone: (866) 803-9204, or email: prospectus-eq_fi@jpmchase.com; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6218, or email: syndicate@svbleerink.com.

    This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any offer or sale of these securities in any state or 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 jurisdiction.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development.

    Forward Looking Statements

    Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the expected gross proceeds, closing and use of proceeds of the offering. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, market risks and uncertainties and the satisfaction of customary closing conditions related to the offering; the success, cost, and timing of the company's product candidate development activities and planned clinical studies and clinical trials; the company's ability to execute on its strategy; positive results from a clinical study may not necessarily be predictive of the results of future or ongoing clinical studies or clinical trials; regulatory developments in the United States and foreign countries; the company's estimates regarding expenses, future revenue, and capital requirements, and other financial results; as well as those risks and uncertainties set forth in the company's most recent Quarterly Report on Form 10-Q and subsequent filings with the Securities and Exchange Commission. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

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    • Reported positive top-line data from AXA1125-003, with clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation observed in subjects with NAFLD receiving AXA1125
    • Completed enrollment of AXA1665-002, with data readout expected in Q3 2020
    • Announced AXA2678 patent issuance and oral presentation at ICSFR
    • Launched new corporate website at www.axcellahealth.com

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced financial results for the first quarter ended March 31, 2020 and provided a business update.

    "These past few months have been a…

    • Reported positive top-line data from AXA1125-003, with clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation observed in subjects with NAFLD receiving AXA1125
    • Completed enrollment of AXA1665-002, with data readout expected in Q3 2020
    • Announced AXA2678 patent issuance and oral presentation at ICSFR
    • Launched new corporate website at www.axcellahealth.com

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced financial results for the first quarter ended March 31, 2020 and provided a business update.

    "These past few months have been a particularly exciting period for Axcella, culminating in our recent report of positive top-line data for AXA1125, our NASH product candidate, that we believe further validates our platform," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "In addition to these recent data, we also completed enrollment in AXA1665-002 in the first quarter and plan to provide a data readout from that ongoing clinical study in the third quarter of 2020 despite the COVID-19 pandemic. With this and other milestones expected in the quarters ahead, including expected regulatory engagements regarding our lead product candidates, 2020 has the potential to be a transformational year for Axcella."

    Recent Highlights

    Liver Programs

    • Reported positive top-line data from AXA1125-003, a clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and physiology in subjects with non-alcoholic fatty liver disease (NAFLD). Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to both placebo and AXA1957, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers.
    • Completed enrollment of AXA1665-002, an ongoing clinical study assessing the impact of AXA1665 on safety, tolerability and physiology in adult subjects with mild and moderate hepatic insufficiency. AXA1665 has been generally safe and well tolerated in this study to date. The company expects to report data from this study in the third quarter of 2020.

    Muscle Program

    • Discussed Axcella's AXA2678 muscle product candidate during an oral presentation at the 2020 International Conference on Frailty and Sarcopenia Research (ICFSR) in March 2020.
    • Announced the issuance of U.S. Patent 10,596,136, which broadly covers the use of AXA2678 and related EMM compositions for the reduction of fat infiltration in muscle.

    Corporate

    • Launched a new corporate website at www.axcellahealth.com that includes additional detail about the company's platform, product candidates and culture.

    Anticipated 2020 Milestones

    Liver Programs

    • Q3 2020: Report data from AXA1665-002 in adult subjects with mild and moderate hepatic insufficiency.
    • Q4 2020: Submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) and initiate a potential Phase 2b/3 registrational clinical trial of AXA1665 for the reduction in risk of OHE recurrence.
    • 2H 2020: Engage with the FDA to discuss the company's planned IND application for AXA1125, proposed Phase 2b clinical trial in adults and pediatric development program.

    Blood Program

    • Q4 2020: Report top-line data from Cohort 1 of AXA4010-001, a clinical study on safety, tolerability and blood physiology in subjects with sickle cell disease.

    Financial Results

    R&D Expenses: Research and development expenses were $10.3 million and $7.6 million for the quarters ended March 31, 2020 and 2019, respectively. The change was primarily related to greater costs associated with the advancement of the company's product candidates and ongoing clinical studies.

    G&A Expenses: General and administrative expenses were $4.1 million and $3.5 million for the quarters ended March 31, 2020 and 2019, respectively. The change was primarily related to higher professional services and employee-related costs associated with being a public company.

    Net Loss: Net loss for the quarter ended March 31, 2020 was $15.0 million, or $0.65 per basic and diluted share. This compares with a net loss of $11.6 million, or $2.43 per basic and diluted share, for the quarter ended March 31, 2019.

    Cash Position: Cash and cash equivalents at March 31, 2020 were $75.5 million, which compares with $92.1 million at December 31, 2019. Axcella expects that its cash and cash equivalents will be sufficient to meet the company's operating needs into the second quarter of 2021.

    About Endogenous Metabolic Modulators (EMMs)

    EMMs are a broad family of molecules, including amino acids, that regulate human metabolism. Axcella is developing a range of novel product candidates that are comprised of multiple EMMs engineered in distinct combinations and ratios to simultaneously impact multiple metabolic pathways to modify the root causes of various complex diseases and improve health.

    About Axcella's Clinical Studies

    Each of the company's ongoing clinical studies are being conducted as non-investigational new drug (IND) application clinical studies under U.S. Food and Drug Administration regulations and guidance supporting research with food. These studies evaluate product candidates for safety, tolerability and effects on the normal structures and functions in humans, including in individuals with disease. They are not designed or intended to evaluate a product candidate's ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop a product candidate as a potential therapeutic, as is the case with AXA1665 and AXA1125, subsequent studies will be conducted under an IND.

    Internet Posting of Information

    Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the company's website in the "Investors and News" section. Accordingly, investors should monitor such portions of the company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.

    About Axcella

    Axcella is a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health. The company's product candidates are comprised of EMMs and their derivatives that are engineered in distinct combinations and ratios to simultaneously impact multiple biological pathways. Axcella's pipeline includes lead therapeutic candidates for non-alcoholic steatohepatitis (NASH) and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. Additional muscle- and blood-related programs are in earlier-stage development. For more information, please visit www.axcellahealth.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the characteristics, competitive position and development potential of the company's EMM product candidates, including for AXA1125 and AXA1665, the design, status and timing of the company's ongoing clinical studies and planned IND-enabled clinical trials, the company's anticipated program milestones, including the timing of AXA1665-002 and AXA4010-001 data readouts, the subject and timing of the company's planned interactions with the FDA on the AXA1665 and AXA1125 programs including potential timing of IND application submissions, and the potential of the company's product candidates to impact health and/or disease, including AXA1125's potential in NASH and AXA1665 potential in OHE. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company's ability to conduct and complete its ongoing or planned clinical studies and IND enabled clinical trials and planned interactions and submissions to FDA or other regulatory authorities in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data we are able to collect in our ongoing AXA1665-002 and AXA4010-001 clinical studies and potential delays in disclosure of the same, other potential impacts of COVID-19 on our business and financial results, including with respect to our ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts and/or FDA feedback support our IND submission and clinical trial initiation plans and timing, clinical trial design and target indication for AXA1125 and AXA1665, the clinical development and safety profile of the company's product candidates and their health or therapeutic potential, whether and when, if at all, the company's product candidates will receive approval from the FDA or other comparable regulatory authorities, and for which, if any, indications, competition from other biotechnology companies, past results from clinical studies not being representative of future results, and other risks identified in the company's SEC filings, including Axcella's Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

    Axcella Health Inc.

    Unaudited Condensed Consolidated Balance Sheets

    (in thousands)

     

     

     

     

     

     

     

    March 31,

     

    March 31,

     

     

    2020

     

    2019

    Assets:

     

     

     

     

    Cash and cash equivalents

     

    $

    75,522

     

     

    $

    66,769

     

    Other assets

     

    2,060

     

     

    $

    4,149

     

    Total assets

     

    $

    77,582

     

     

    $

    70,918

     

    Liabilities and stockholders' equity (deficit):

     

     

     

     

    Liabilities

     

    $

    30,768

     

     

    $

    33,253

     

    Preferred stock

     

     

     

    $

    197,888

     

    Stockholders' equity (deficit)

     

    46,814

     

     

    $

    (160,223

    Total liabilities and stockholders' equity

     

    $

    77,582

     

     

    $

    70,918

     

    Axcella Health Inc.

    Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss

    (in thousands, except share and per share data)

     

     

     

     

     

    Three Months Ended

     

    March 31,

     

     

    2020

     

    2019

    Operating expenses:

     

     

     

     

    Research and development

     

    $

    10,335

     

     

    $

    7,563

     

    General and administrative

     

    4,125

     

     

    3,468

     

    Total operating expenses

     

    14,460

     

     

    11,031

     

    Loss from operations

     

    (14,460)

     

     

    (11,031)

     

    Other income (expense), net

     

    (549)

     

     

    (542)

     

    Net loss and comprehensive loss

     

    $

    (15,009)

     

     

    $

    (11,573)

     

    Net loss per share, basic and diluted

     

    $

    (0.65)

     

     

    $

    (2.43)

     

    Weighted average common shares outstanding, basic and diluted

     

    23,188,816

     

     

    4,775,828

     

     

    View Full Article Hide Full Article
    • Clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation markers observed with AXA1125 along with favorable tolerability, supporting its potential to be a first-line NASH therapy
    • Greater activity in key markers seen among subjects with type 2 diabetes receiving AXA1125
    • Company plans to engage with FDA regarding IND submission for AXA1125, proposed Phase 2b clinical trial in adult NASH and pediatric development program
    • Conference call to be held today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced positive top-line…

    • Clinically relevant reductions in liver fat content, insulin resistance and fibroinflammation markers observed with AXA1125 along with favorable tolerability, supporting its potential to be a first-line NASH therapy
    • Greater activity in key markers seen among subjects with type 2 diabetes receiving AXA1125
    • Company plans to engage with FDA regarding IND submission for AXA1125, proposed Phase 2b clinical trial in adult NASH and pediatric development program
    • Conference call to be held today at 8:30 a.m. ET

    Axcella (NASDAQ:AXLA), a clinical-stage biotechnology company focused on leveraging endogenous metabolic modulators (EMMs) to pioneer a new approach for treating complex diseases and improving health, today announced positive top-line data from AXA1125-003.

    AXA1125-003 is a placebo-controlled, randomized, multi-arm clinical study assessing the impact of AXA1125 and AXA1957 on safety, tolerability and effects on structures and functions of the liver, as measured by a comprehensive panel of imaging and soluble biomarkers related to metabolism, inflammation and fibrosis. Both of these distinct product candidates are proprietary compositions of amino acids and derivatives that have been designed to support liver health. In this non-IND study, 102 adult non-alcoholic fatty liver disease (NAFLD) subjects with presumed nonalcoholic steatohepatitis (NASH), based on inclusion criteria, were enrolled and dosed in a 2:2:2:1 ratio to receive AXA1125, one of two AXA1957 doses, or placebo administered twice daily for 16 weeks. Study subjects were stratified based on the presence or absence of type 2 diabetes.

    Results from the study showed that AXA1125 and AXA1957 were generally well-tolerated, with sustained reductions noted for both product candidates versus placebo in key biomarkers of metabolism, inflammation and fibrosis over 16 weeks. Overall, as compared to placebo, AXA1125 demonstrated larger and more consistent reductions in clinically relevant biomarkers than AXA1957. Among subjects receiving AXA1125, 39% achieved a ≥30% relative reduction in liver fat content (MRI-PDFF), 39% achieved a ≥17 U/L reduction in alanine aminotransaminase (ALT), and 35% achieved a ≥80 mSec reduction in corrected T1 (cT1). Among the 11 subjects with type 2 diabetes receiving AXA1125, a greater proportion achieved each of these thresholds. Emerging evidence suggests that these thresholds of activity increase the likelihood of histopathological improvement in NASH subjects. Notably, the above results were seen without impacting mean body weight or serum lipids.

    Manu Chakravarthy, M.D., Ph.D., Chief Medical Officer of Axcella, said, "In AXA1125-003, we were seeking to evaluate safety and tolerability while also determining what differential responses may be seen from AXA1125 and AXA1957 across markers of metabolism, inflammation and fibrosis. It is gratifying that multifactorial activity and a favorable tolerability profile were noted for AXA1125 in this multi-arm, placebo-controlled randomized study, which replicates findings from our previous clinical study in subjects with NAFLD and type 2 diabetes. We believe that these data, coupled with AXA1125's oral route of administration and favorable tolerability profile to date, reinforce its potential to meaningfully improve the lives of patients with NASH."

    "These data indicate that AXA1125 holds the potential to be a first-line therapy in NASH, with impressive, concordant metabolism, inflammation and fibrosis activity as well as a favorable tolerability profile with no meaningful changes to serum lipids and body weight," said Stephen A. Harrison, M.D., Medical Director of Pinnacle Clinical Research in San Antonio, TX, visiting professor of Hepatology at the University of Oxford, UK and the principal investigator of AXA1125-003. "Additionally, in the majority of subjects with type 2 diabetes receiving AXA1125, clinically relevant thresholds of activity were observed in non-invasive tests that suggest a higher probability of positive outcomes histopathologically. This is a potential differentiator and is particularly encouraging given that nearly 40% of the NASH population is diabetic and the disease is known to be more severe in these patients."

    Select Measures of Relevance at 16 Weeks

    Measure

    Placebo

    AXA1125

    AXA1957 Low

    AXA1957 High

    Subjects dosed

    15

    29

    26

    32

    Mean relative reduction in liver fat content (MRI-PDFF)

    -6%

    -23%

    -20%

    -8%

    Subjects with ≥30% relative reduction in liver fat content

    8%

    39%

    23%

    19%

    Mean relative reduction in ALT

    -7%

    -22%

    -19%

    -21%

    Subjects with ≥17 U/L reduction in ALT

    25%

    39%

    32%

    37%

    Subjects with ≥80 mSec reduction in cT1

    17%

    35%

    23%

    23%

    Mean absolute change in proC3 (ng/mL)

    -0.7

    -3.4

    -3.1

    -4.1

    * Mean values and percentages above only include subjects for whom data was available at the week 16 timepoint.

    AXA1125 and AXA1957 were both generally well tolerated in the study. The adverse events (AEs) experienced in ≥10% of subjects were gastrointestinal (diarrhea, nausea, reduced appetite) and upper respiratory infection. Gastrointestinal AEs were generally mild and transient, self-resolving in two to three weeks on average. Two serious adverse events were reported, both of which were determined to be unrelated to study product administration.

    "The findings from this clinical study further validate the strength of our EMM platform and its ability to identify product candidates with the potential to address complex diseases in a multi-targeted manner," said Bill Hinshaw, President and Chief Executive Officer of Axcella. "Given the strength and consistency of data on AXA1125, we have selected it as our product candidate for NASH and have decided that we will not reinitiate our AXA1957-002 pediatric study, which had recently been suspended due to COVID-19. In the months ahead, we plan to engage with the U.S. Food and Drug Administration (FDA) to discuss our investigational new drug (IND) application for AXA1125, proposed Phase 2b clinical trial in adults and pediatric development program. We give our thanks to the many subjects and investigators who participated in AXA1125-003 and will continue working diligently toward our goal of providing them with an effective and safe treatment option."

    Conference Call Information

    Axcella will host a conference call today at 8:30 a.m. ET to discuss the top-line data from AXA1125-003. The conference call webcast and accompanying slides will be made available shortly before the start of the call on the company's website at