AGLE Aeglea BioTherapeutics Inc.

6.74
-0.24  -3%
Previous Close 6.98
Open 6.97
52 Week Low 3.5
52 Week High 11.38
Market Cap $300,602,969
Shares 44,599,847
Float 41,600,144
Enterprise Value $267,422,932
Volume 257,523
Av. Daily Volume 302,015
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Upcoming Catalysts

Drug Stage Catalyst Date
Pegzilarginase (AEB1102)
Arginase I deficiency
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ACN00177
Homocystinuria
Phase 1/2
Phase 1/2
Phase 1/2 trial initiated June 2020.
Pegzilarginase (AEB1102) + KEYTRUDA
Small cell lung cancer (SCLC)
Phase 2
Phase 2
Phase 1/2 trial ongoing.

Latest News

  1. Pegzilarginase Showed Durable Clinical Response and Sustained Reduction in Plasma Arginine at 56 Week Analysis of Phase 1/2 Open-Label Extension Study

    Initiated Phase 1/2 Clinical Trial of ACN00177 for the Treatment of Homocystinuria

    AUSTIN, Texas, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today reported its second quarter 2020 financial results, and provided recent corporate and program highlights.

    "Despite the operating challenges posed by the global pandemic, we continued to advance our pegzilarginase program in the first half of the…

    Pegzilarginase Showed Durable Clinical Response and Sustained Reduction in Plasma Arginine at 56 Week Analysis of Phase 1/2 Open-Label Extension Study

    Initiated Phase 1/2 Clinical Trial of ACN00177 for the Treatment of Homocystinuria

    AUSTIN, Texas, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today reported its second quarter 2020 financial results, and provided recent corporate and program highlights.

    "Despite the operating challenges posed by the global pandemic, we continued to advance our pegzilarginase program in the first half of the year. The presentation of long-term data showing sustained lowering of arginine levels and durable clinical response with pegzilarginase treatment, as well as progress in our patient identification efforts, reinforce our belief in its potential as a life-changing therapy for those with Arginase 1 Deficiency and lay a strong foundation for the commercial launch of pegzilarginase," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "Additionally, we initiated our Phase 1/2 clinical trial of ACN00177 for Homocystinuria in the second quarter. We are continuing our patient identification activities and look forward to dosing the first patient once the clinical sites are able to begin screening patients."

    Recent Highlights and Updates



    Pegzilarginase in Arginase 1 Deficiency

    • In May, Aeglea presented results from its 56 week analysis from the Company's completed Phase 1/2 clinical trial and ongoing open-label extension study during a late-breaking oral presentation at the 6th Congress of the European Academy of Neurology. Key results include:
      • Treatment with pegzilarginase resulted in a significant reduction in plasma arginine from baseline with all 13 patients achieving plasma arginine levels within the target range (<200 µM).
      • 85% (11 of 13) of patients were clinical responders based on mobility improvements evaluated using three assessments: 6MWT (6 Minute Walk Test), GMFM (Gross Motor Function) Part D (standing) and Part E (walking, running, and jumping).
      • Pegzilarginase was shown to have a favorable safety profile with more than 750 doses administered.
    • To date, Aeglea has identified more than 240 Arginase 1 Deficiency patients. The number of identified patients represents more than 50% and 30% of the estimated genetic prevalence patient population in the U.S. and key European markets (France, Germany, Spain, Italy and the United Kingdom), respectively.

    ACN00177 in Homocystinuria

    • Aeglea initiated its Phase 1/2 clinical trial for ACN00177, a novel engineered human enzyme therapy designed to treat Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy.

    Corporate

    • Eric Bradford, M.D., M.Sc., M.B.A. has been promoted to Chief Development Officer. Dr. Bradford will oversee the clinical programs for pegzilarginase and ACN00177 as well as shape the clinical development strategy for future programs from the Company's platform of novel human enzymes.
    • Chief Medical Officer Ravi M. Rao, M.B BChir, Ph.D., will depart the company to pursue other opportunities. Dr. Rao will continue to support Aeglea in a medical advisor role through a transitional period.

    "Ravi has been a valued and impactful member of the Aeglea team. While we are disappointed by his planned departure, we wish him the best as he returns to his roots in immunology research and development," said Dr. Quinn. "I look forward to working more closely with Eric as we continue to strengthen our capabilities and advance pegzilarginase towards potential approval and launch."

    Upcoming Events

    Aeglea will be attending the following virtual investor conferences in the coming quarter.

    • Wells Fargo Securities Healthcare Conference, September 9-10
    • H.C. Wainwright Healthcare Conference, September 13-15
    • Cantor Fitzgerald Global Healthcare Conference, September 15-17

    Further, Aeglea's leadership looks forward to participating in dialogue about the Company's enzyme therapeutics platform during the following industry events, with additional details to be announced.

    • World Orphan Drug Congress USA 2020, August 24-26
    • Child Neurology Society Annual Meeting-International Child Neurology Congress 2020, October 19-23

    Second Quarter 2020 Financial Results

    As of June 30, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $159.2 million. Based on Aeglea's current operating plans, management believes it has sufficient capital resources to fund anticipated operations through 2022.

    Research and development expenses totaled $16.9 million for the second quarter of 2020 and $14.8 million for the second quarter of 2019. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea's lead product candidate, pegzilarginase; ramp-up in manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses offset by decreasing clinical development expenses as a result of completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency and closing out cancer trials. 

    General and administrative expenses totaled $4.7 million for the second quarter of 2020 and $3.8 million for the second quarter of 2019. This increase was primarily due to additional employee headcount, ramping up commercial capabilities, and additional facilities to support company growth.

    Net loss totaled $21.4 million and $18.0 million for the second quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.6 million and $1.2 million for the second quarter of 2020 and 2019, respectively.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating, progressive disease presenting in childhood with persistent hyperargininemia, spasticity, developmental delay, intellectual disability, seizures and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea's Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company's single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

    About ACN00177 in Homocystinuria



    Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company initiated a Phase 1/2 trial in the second quarter of 2020 and continues patient identification and administrative activities. The timing of first patient dosing in this Phase 1/2 trial will depend on determinations by individual sites as they adjust to impacts from COVID-19.

    About Aeglea BioTherapeutics



    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. The Company initiated a Phase 1/2 clinical trial of ACN00177 for the treatment of Homocystinuria in the second quarter of 2020. Aeglea has an active discovery platform, with the most advanced program for Cystinuria. For more information, please visit http://aegleabio.com.



    Safe Harbor / Forward Looking Statements



    This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our cash forecasts, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the potential addressable markets of the our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

    Media Contact:

    Kelly Boothe, Ph.D.

    Director, Corporate Communications

    Aeglea BioTherapeutics

    512.399.5458

    Investor Contact:

    Joey Perrone

    Senior Director, Finance & Investor Relations

    Aeglea BioTherapeutics



    Financials

    Aeglea BioTherapeutics, Inc.

    Condensed Consolidated Balance Sheets

    (Unaudited)

    (In thousands, except share and per share amounts)

      June 30,  December 31, 
      2020  2019 
    ASSETS        
    CURRENT ASSETS        
    Cash and cash equivalents $65,713  $19,253 
    Marketable securities  92,017   52,696 
    Prepaid expenses and other current assets  4,136   2,556 
    Total current assets  161,866   74,505 
    Restricted cash  1,500   1,500 
    Property and equipment, net  4,896   2,385 
    Operating lease right-of-use assets  4,557   4,726 
    Other non-current assets  92   67 
    TOTAL ASSETS $172,911  $83,183 
             
    LIABILITIES AND STOCKHOLDERS' EQUITY        
    CURRENT LIABILITIES        
    Accounts payable $4,151  $3,154 
    Operating lease liabilities  228   351 
    Accrued and other current liabilities  11,511   14,854 
    Total current liabilities  15,890   18,359 
    Non-current operating lease liabilities  4,695   4,712 
    Other non-current liabilities  68   31 
    TOTAL LIABILITIES  20,653   23,102 
             
    STOCKHOLDERS' EQUITY        
    Preferred stock, $0.0001 par value; 10,000,000 shares authorized

    as of June 30, 2020 and December 31, 2019; no shares issued and

    outstanding as of June 30, 2020 and December 31, 2019
          
    Common stock, $0.0001 par value; 500,000,000 shares authorized

     as of June 30, 2020 and December 31, 2019; 44,599,847 shares and

     29,084,437 shares issued and outstanding as of June 30, 2020

     and December 31, 2019, respectively
      5   3 
    Additional paid-in capital  387,475   255,142 
    Accumulated other comprehensive income  39   51 
    Accumulated deficit  (235,261)  (195,115)
    TOTAL STOCKHOLDERS' EQUITY  152,258   60,081 
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $172,911  $83,183 



    Aeglea BioTherapeutics, Inc.

    Condensed Consolidated Statements of Operations

    (Unaudited)

     (In thousands, except share and per share amounts)

      Three Months Ended

    June 30,
      Six Months Ended

    June 30,
     
      2020  2019  2020  2019 
    Operating expenses:                
    Research and development  16,869   14,806   31,431   29,195 
    General and administrative  4,691   3,816   9,151   7,084 
    Total operating expenses  21,560   18,622   40,582   36,279 
    Loss from operations  (21,560)  (18,622)  (40,582)  (36,279)
                     
    Other income (expense):                
    Interest income  161   619   461   1,126 
    Other expense, net  (19)  (16)  (25)  (33)
    Total other income  142   603   436   1,093 
    Net loss $(21,418) $(18,019) $(40,146) $(35,186)
                     
    Net loss per share, basic and diluted $(0.40) $(0.55) $(0.93) $(1.14)
    Weighted-average common shares outstanding, basic and

      diluted
      52,941,603   32,840,357   43,019,670   30,936,623 

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  2. AUSTIN, Tx., May 28, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced that, due to the public health impact of the coronavirus (COVID-19) pandemic, the location of the Company's 2020 annual meeting of stockholders has been changed and will be held in a virtual meeting format only. As previously announced, the annual meeting will be held on Monday, June 8, 2020 at 9:00 a.m. Central Time. To be admitted to the annual meeting at www.virtualshareholdermeeting.com/AGLE2020, stockholders must enter the control number found on their proxy card, voting instruction…

    AUSTIN, Tx., May 28, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced that, due to the public health impact of the coronavirus (COVID-19) pandemic, the location of the Company's 2020 annual meeting of stockholders has been changed and will be held in a virtual meeting format only. As previously announced, the annual meeting will be held on Monday, June 8, 2020 at 9:00 a.m. Central Time. To be admitted to the annual meeting at www.virtualshareholdermeeting.com/AGLE2020, stockholders must enter the control number found on their proxy card, voting instruction form or notice previously received. Further information regarding this change to the location of the annual meeting can be found in the supplemental proxy materials filed by Aeglea with the Securities and Exchange Commission on May 28, 2020.

    About Agelea BioTherapeutics, Inc.

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. The Company received approval of its Clinical Trial Application (CTA) for ACN00177 for the treatment of Homocystinuria by the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA). Aeglea has an active discovery platform, with the most advanced program for Cystinuria. For more information, please visit http://aegleabio.com.

    Safe Harbor / Forward Looking Statements

    This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our plans to hold the 2020 annual meeting of stockholders. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

    Media Contact:
    Kelly Boothe, Ph.D.
    Director, Corporate Communications
    Aeglea BioTherapeutics
    512.399.5458

    Investor Contact:
    Joey Perrone
    Senior Director, Finance & Investor Relations
    Aeglea BioTherapeutics


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  3.   Pegzilarginase Showed Durable Clinical Response at 56 Week Analysis

    All Patients Demonstrated a Marked and Sustained Reduction in Plasma Arginine

    Favorable Safety Profile, Consistent with Previously Reported Results

    AUSTIN, Texas, May 26, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced a new 56 week analysis on Arginase 1 Deficiency (ARG1-D) patients who have been treated with pegzilarginase from the Company's completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. The data were shared yesterday in a virtual, late-breaking…

      Pegzilarginase Showed Durable Clinical Response at 56 Week Analysis

    All Patients Demonstrated a Marked and Sustained Reduction in Plasma Arginine

    Favorable Safety Profile, Consistent with Previously Reported Results

    AUSTIN, Texas, May 26, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced a new 56 week analysis on Arginase 1 Deficiency (ARG1-D) patients who have been treated with pegzilarginase from the Company's completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study. The data were shared yesterday in a virtual, late-breaking oral presentation at the 6th Congress of the European Academy of Neurology.

    "Arginase 1 Deficiency is a devastating disease that is frequently under diagnosed or misdiagnosed as more common neurological conditions, such as cerebral palsy, due to lack of awareness of this rare condition," said George Diaz, M.D., Ph.D., division chief of medical genetics in the Division of Medical Genetics and Genomics and Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai, New York, NY. "Because of the condition's progressive nature, it is essential that patients be diagnosed early, and there is an urgent need for a therapy that addresses the underlying cause of the disease and improves clinical manifestations."

    "The results of this long-term data demonstrate that treatment with pegzilarginase resulted in a durable clinical response, which is a critical factor in effectively treating a life-long, progressive condition," said Ravi M. Rao, M.B Ch.B PhD, chief medical officer of Aeglea. "We are also pleased to see that the lowering of arginine levels observed in the 20 week analysis were maintained through the 56 week analysis. These results align with the primary endpoint of PEACE, our ongoing pivotal Phase 3 clinical trial, and together with the durable clinical response bolsters our belief that pegzilarginase has the potential to be an impactful treatment for people living with Arginase 1 Deficiency."

    The presentation, titled "1 Year Data from First in Human Study of Pegzilarginase for the Treatment of Arginase 1 Deficiency (ARG1-D)," includes data on 13 patients treated with pegzilarginase who completed the 56 week treatment period (8 weeks Part 2 repeat dosing + 48 weeks open-label extension).

    Highlights from the 56 week analysis include:

    • A statistically significant reduction in plasma arginine from baseline was observed with a single dose, with continued improvement through the 20 and 56 week analyses
      • All 13 patients achieved plasma arginine levels within the target range (<200µM)
      • The median plasma arginine level was 99µM (normal range: 40-115µM)
    • Eleven patients overall were clinical Responders (85%)
      • Mobility improvements were evaluated using three assessments: 6MWT (6 Minute Walk Test), GMFM (Gross Motor Function) Part D (standing) and Part E (walking, running, and jumping)
      • Mean change in 6MWT showed progressive improvement from baseline through 8, 20 and 56 week analyses
      • All six patients with significant mobility impairment at baseline (GMFCS [Gross Motor Function Classification System] Levels II and III) improved in the GMFM Part E; four out of six patients also improved in the GMFM Part D
    • Pegzilarginase was shown to have a favorable safety profile with more than 750 doses administered
      • Safety profile is consistent with previously reported data
      • The most common treatment-related serious adverse events were hypersensitivity and hyperammonemia, both of which were infrequent, expected and managed with standard care; no treatment-related serious adverse events led to patient discontinuation
      • Most treatment-related adverse events were mild and decreased in frequency over time

    The presentation is available for download on the Presentations & Events section of the Company's website.

    About the Phase 1/2 and Open-Label Extension Trial

    The Phase 1/2, multicenter, single arm, open-label extension study of pegzilarginase enrolled patients aged 2 years and older with Arginase 1 Deficiency in the United States, Canada, and Europe. The trial investigates single ascending doses (Part 1), repeated weekly dosing for eight weeks (Part 2). The trial enrolled 16 adult and pediatric patients and 14 patients rolled over to the open-label extension. The primary endpoint of the trial is safety and tolerability of intravenous administration of pegzilarginase in patients with Arginase 1 Deficiency. The trial also evaluated the pharmacokinetic and pharmacodynamic effects of repeated doses of pegzilarginase on plasma arginine levels, and evaluation of clinical outcomes using several mobility assessments.

    Please visit www.clinicaltrials.gov for more information.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea's Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company's single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. The Company received approval of its Clinical Trial Application (CTA) for ACN00177 for the treatment of Homocystinuria by the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA). Aeglea has an active discovery platform, with the most advanced program for Cystinuria. For more information, please visit http://aegleabio.com.

    Safe Harbor / Forward Looking Statements

    This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our cash forecasts, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the potential addressable markets of the our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

    Media Contact:
    Kelly Boothe, Ph.D.
    Director, Corporate Communications
    Aeglea BioTherapeutics
    512.399.5458

    Investor Contact:
    Joey Perrone
    Senior Director, Finance & Investor Relations
    Aeglea BioTherapeutics

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  4. AUSTIN, Texas, May 19, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced it will present a new 56 week analysis on patients who have been treated with pegzilarginase from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study in a late-breaking oral presentation at the 6th Congress of the European Academy of Neurology being held virtually May 23-26, 2020.

    Presentation Details:

    • Title: 1 Year Data from First in Human Study of Pegzilarginase for the Treatment of Arginase 1 Deficiency (ARG1-D)
    • Session: Late Breaking…

    AUSTIN, Texas, May 19, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today announced it will present a new 56 week analysis on patients who have been treated with pegzilarginase from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study in a late-breaking oral presentation at the 6th Congress of the European Academy of Neurology being held virtually May 23-26, 2020.

    Presentation Details:

    • Title: 1 Year Data from First in Human Study of Pegzilarginase for the Treatment of Arginase 1 Deficiency (ARG1-D)
    • Session: Late Breaking News
    • Presentation Date: Monday, May 25, 2020, 5:15-6:30pm CEST (11:15am-12:30pm EDT)

    An electronic version of the presentation will be available for download on the Presentations & Events section of the Company's website.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea's Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company's single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. The Company received approval of its Clinical Trial Application (CTA) for ACN00177 for the treatment of Homocystinuria by the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA). Aeglea has an active discovery platform, with the most advanced program for Cystinuria. For more information, please visit http://aegleabio.com.

    Safe Harbor / Forward Looking Statements

    This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our cash forecasts, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the potential addressable markets of the our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

    Media Contact:
    Kelly Boothe, Ph.D.
    Director, Corporate Communications
    Aeglea BioTherapeutics
    512.399.5458

    Investor Contact:
    Joey Perrone
    Senior Director, Finance & Investor Relations
    Aeglea BioTherapeutics

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  5. Gross Proceeds of $138 Million from April 2020 Public Offering Extends Cash Runway Through 2022

    Clinical Trial Application for ACN00177 Approved by MHRA; Progress Toward Phase 1/2 Clinical Trial in Homocystinuria Initiation

    AUSTIN, Texas, May 07, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today reported its first quarter 2020 financial results, and provided recent corporate and program highlights.

    "These past few months have brought unique challenges as we navigate the impact of COVID-19, and reminds us all of the critical need for healthcare innovation and…

    Gross Proceeds of $138 Million from April 2020 Public Offering Extends Cash Runway Through 2022

    Clinical Trial Application for ACN00177 Approved by MHRA; Progress Toward Phase 1/2 Clinical Trial in Homocystinuria Initiation

    AUSTIN, Texas, May 07, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare and other high-burden diseases, today reported its first quarter 2020 financial results, and provided recent corporate and program highlights.

    "These past few months have brought unique challenges as we navigate the impact of COVID-19, and reminds us all of the critical need for healthcare innovation and new medicines. These needs are all too familiar for people with rare diseases, like Arginase 1 Deficiency, where adequate treatment options are often not available," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "With our recently completed financing, we have the resources to advance our pegzilarginase program for Arginase 1 Deficiency through regulatory submission and potential FDA approval. With the recent approval of our Clinical Trial Application for ACN00177 for the treatment of Homocystinuria, the company is positioned to bring forward its second clinical-stage human enzyme – both with the potential to be transformative solutions for rare genetic disorders."

    Recent Highlights & COVID-19 Update

    Pegzilarginase in Arginase 1 Deficiency

    • Aeglea is working with treating physicians to implement individual treatment plans and potentially developing a home healthcare option for patients enrolled in the Phase 3 PEACE trial.
    • To date, most enrolled patients have continued to receive treatment. The Company is developing a plan to analyze results for patients that are missing data points. The Phase 3 PEACE trial protocol is designed in such a way that a patient may miss a few doses without being disqualified from the trial.
    • The supply chain has not experienced any significant impact at this time and the Company currently has sufficient supply available for completion of its ongoing clinical trials.
    • The Company expects to complete enrollment of its Phase 3 PEACE trial in the third quarter of 2020 and to provide topline data in the first quarter of 2021.

    ACN00177 in Homocystinuria

    • In April, Aeglea announced the approval of its Clinical Trial Application (CTA) by the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177, a novel engineered human enzyme therapy designed to treat Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy.
    • The Company continues its patient identification and administrative activities to quickly move forward with dosing patients once trial sites are able to initiate clinical trials.
    • While Aeglea continues to prepare to initiate a Phase 1/2 trial for ACN00177 in the second quarter of 2020, the timing will depend on determination by individual sites that each is ready to open for recruitment in light of COVID-19; the Company's priorities at this time are to avoid further overburdening hospital staff and to minimize the risk of trial participants exposure to COVID-19.

    Corporate Highlights

    • In April, the Company strengthened its financial position with a public offering resulting in gross proceeds of $138 million, which extended its cash runway through 2022.
    • The Company implemented policies and practices to protect the health and wellbeing of the Company's employees and communities, including asking employees to work from home and implementing a work rotation for essential lab employees.
    • Aeglea suspended all business travel and transitioned all meetings, including conference attendance and industry events, to virtual meetings. 

    First Quarter 2020 Financial Results

    As of March 31, 2020, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $50.5 million. In addition, in April 2020 the Company raised approximately $129.6 million in net proceeds from a public offering. Based on Aeglea's current operating plan, and taking into account the net offering proceeds, management believes it has sufficient capital resources to fund anticipated operations through 2022.

    Research and development expenses totaled $14.6 million for the first quarter of 2020 and $14.4 million for the first quarter of 2019. The increase was primarily associated with investing in manufacturing and pre-commercial activities for Aeglea's lead product candidate, pegzilarginase; ramp-up in toxicology, nonclinical studies, and manufacturing activities for ACN00177 in Homocystinuria; and personnel-related expenses. The increase was offset by a decrease in clinical development expenses as a result of completing a Phase 1/2 clinical trial in patients with Arginase 1 Deficiency, a Phase 1 clinical trial in patients with advanced solid tumors, and concluding enrollment of a Phase 1/2 combination trial in patients with small cell lung cancer. 

    General and administrative expenses totaled $4.5 million for the first quarter of 2020 and $3.3 million for the first quarter of 2019. This increase was primarily due to additional employee headcount, ramping up commercial capabilities, and additional facilities to support company growth.

    Net loss totaled $18.7 million and $17.2 million for the first quarter of 2020 and 2019, respectively, with non-cash stock compensation expense of $1.3 million and $1.1 million for the first quarter of 2020 and 2019, respectively.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is an enhanced human arginase that enzymatically lowers levels of the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme therapy to reduce elevated blood arginine levels in patients with ARG1-D. Aeglea's Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company's single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.

    About ACN00177 in Homocystinuria

    Aeglea is developing ACN00177 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities including severe osteoporosis, developmental delay, intellectual disability, lens dislocation and severe near-sightedness. ACN00177 has been designed as a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. With this mechanism, ACN00177 is intended to lower the abnormally high blood levels of homocysteine in patients with Homocystinuria. Preclinical data demonstrated that ACN00177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. The Company received approval from the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) for its Clinical Trial Application (CTA) and continues to prepare to initiate a Phase 1/2 trial in the second quarter of 2020.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and other high burden diseases. Aeglea's lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. Aeglea has an active discovery platform with programs for Homocystinuria and Cystinuria. The Company received approval of its Clinical Trial Application (CTA) for ACN00177 for the treatment of Homocystinuria by the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) and continues to prepare to initiate a Phase 1/2 trial in the second quarter of 2020. For more information, please visit http://aegleabio.com.

    Safe Harbor / Forward Looking Statements

    This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our cash forecasts, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the potential addressable markets of the our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

    Media Contact:
    Kelly Boothe, Ph.D.
    Director, Corporate Communications
    Aeglea BioTherapeutics
    512.399.5458

    Investor Contact:
    Joey Perrone
    Senior Director, Finance & Investor Relations
    Aeglea BioTherapeutics


    Financials

    Aeglea BioTherapeutics, Inc.
    Condensed Consolidated Balance Sheets
    (Unaudited)

    (In thousands, except share and per share amounts)

        March 31,     December 31,  
        2020     2019  
    ASSETS                
    CURRENT ASSETS                
    Cash and cash equivalents   $ 12,594     $ 19,253  
    Marketable securities     36,398       52,696  
    Prepaid expenses and other current assets     5,273       2,556  
    Total current assets     54,265       74,505  
    Restricted cash     1,500       1,500  
    Property and equipment, net     4,244       2,385  
    Operating lease right-of-use assets     4,712       4,726  
    Other non-current assets     60       67  
    TOTAL ASSETS   $ 64,781     $ 83,183  
                     
    LIABILITIES AND STOCKHOLDERS' EQUITY                
    CURRENT LIABILITIES                
    Accounts payable   $ 3,618     $ 3,154  
    Operating lease liabilities     293       351  
    Accrued and other current liabilities     13,227       14,854  
    Total current liabilities     17,138       18,359  
    Non-current operating lease liabilities     4,815       4,712  
    Other non-current liabilities     23       31  
    TOTAL LIABILITIES     21,976       23,102  
                     
    STOCKHOLDERS' EQUITY                
    Preferred stock, $0.0001 par value; 10,000,000 shares authorized
    as of March 31, 2020 and December 31, 2019; no shares issued and outstanding as of March 31, 2020 and December 31, 2019
               
    Common stock, $0.0001 par value; 500,000,000 shares authorized
     as of March 31, 2020 and December 31, 2019; 29,147,461 shares and
     29,084,437 shares issued and outstanding as of March 31, 2020
     and December 31, 2019, respectively
        3       3  
    Additional paid-in capital     256,779       255,142  
    Accumulated other comprehensive (loss) income     (134 )     51  
    Accumulated deficit     (213,843 )     (195,115 )
    TOTAL STOCKHOLDERS' EQUITY     42,805       60,081  
    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY   $ 64,781     $ 83,183  



    Aeglea BioTherapeutics, Inc.
    Condensed Consolidated Statements of Operations
    (Unaudited)

     (In thousands, except share and per share amounts)

        Three Months Ended
    March 31,
     
        2020     2019  
    Operating expenses:                
    Research and development     14,562       14,389  
    General and administrative     4,460       3,268  
    Total operating expenses     19,022       17,657  
    Loss from operations     (19,022 )     (17,657 )
                     
    Other income (expense):                
    Interest income     300       507  
    Other expense, net     (6 )     (17 )
    Total other income     294       490  
    Net loss   $ (18,728 )   $ (17,167 )
                     
    Net loss per share, basic and diluted   $ (0.57 )   $ 0.59  
    Weighted-average common shares outstanding, basic and diluted     33,097,736       29,011,737  

     

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