AGLE Aeglea BioTherapeutics Inc.

7.65
-0.05  -1%
Previous Close 7.7
Open 7.71
52 Week Low 5.82
52 Week High 9.9
Market Cap $375,343,869
Shares 49,064,558
Float 43,497,685
Enterprise Value $250,095,286
Volume 11,935
Av. Daily Volume 147,606
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Upcoming Catalysts

Drug Stage Catalyst Date
Pegzilarginase (AEB1102) - PEACE
Arginase I deficiency
Phase 3
Phase 3
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AGLE-177
Homocystinuria
Phase 1/2
Phase 1/2
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Latest News

  1. AUSTIN, Texas, Sept. 1, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced its participation in the following medical and investor conferences: 

    Investor Meetings

    • 2021 Wells Fargo Virtual Healthcare Conference, September 9
    • H.C. Wainwright 23rd Annual Global Investment Conference, September 13
    • 2021 Cantor Virtual Global Healthcare Conference, September 27

    Medical Conferences

    • International Parkinson and Movement Disorder Society annual congress (MDS Virtual Congress 2021), September 17 - 22
    • 50th Child Neurology Society (CNS) Annual Meeting, September 29 - October 2

    At…

    AUSTIN, Texas, Sept. 1, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced its participation in the following medical and investor conferences: 

    Investor Meetings

    • 2021 Wells Fargo Virtual Healthcare Conference, September 9
    • H.C. Wainwright 23rd Annual Global Investment Conference, September 13
    • 2021 Cantor Virtual Global Healthcare Conference, September 27

    Medical Conferences

    • International Parkinson and Movement Disorder Society annual congress (MDS Virtual Congress 2021), September 17 - 22
    • 50th Child Neurology Society (CNS) Annual Meeting, September 29 - October 2

    At both the MDS Virtual Congress and the CNS Annual Meeting, a poster presentation will outline a subset analysis from the Company's previously reported Phase 1/2 and Phase 2 open label extension studies of pegzilarginase in patients with Arginase 1 Deficiency (ARG1-D). In the poster, analysis of gait kinematics and spasticity was completed using video compilations.

    To access live and/or archived Investor Conference webcasts, visit the Events & Presentations section of the Company's website. A replay of Company webcasts is archived on the website for 30 days following presentations.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. 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 began dosing patients in a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria in June 2021. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit http://aeglea.com.

    (PRNewsfoto/Aeglea BioTherapeutics, Inc.)

     

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  2. AUSTIN, Texas, Aug. 5, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced financial results for the second quarter ended June 30, 2021, and reviewed recent corporate updates and program highlights.

    "2021 is proving to be a pivotal year for Aeglea. In the second quarter, we achieved a significant milestone in our second clinical development program. With the initiation of dosing in our Phase 1/2 clinical trial of AGLE-177, Aeglea is making progress for patients who suffer from Homocystinuria, a rare and progressive disease which currently has limited treatment options…

    AUSTIN, Texas, Aug. 5, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced financial results for the second quarter ended June 30, 2021, and reviewed recent corporate updates and program highlights.

    "2021 is proving to be a pivotal year for Aeglea. In the second quarter, we achieved a significant milestone in our second clinical development program. With the initiation of dosing in our Phase 1/2 clinical trial of AGLE-177, Aeglea is making progress for patients who suffer from Homocystinuria, a rare and progressive disease which currently has limited treatment options," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "Additionally, with the strengthening of our leadership team, deepening relationship with the rare disease community and advancing our scientific communications platform, we continue to prepare for the data readout for our Phase 3 clinical trial of pegzilarginase in Arginase 1 Deficiency in the fourth quarter as well as lay the groundwork for potential approval and commercialization."

    Second Quarter and Recent Highlights and Updates

    AGLE-177 in Homocystinuria

    • Initiated dosing with AGLE-177 in a Phase 1/2 clinical trial in people with Homocystinuria. The trial is anticipated to enroll 16–20 patients at sites located in the United Kingdom and Australia. Aeglea expects to provide a clinical update prior to the end of 2021.

    Pegzilarginase in Arginase 1 Deficiency

    • Published 20-week data from a Phase 1/2 and open-label extension studies of pegzilarginase in the Journal of Inherited Metabolic Disease. The article, titled "Clinical Effect and Safety Profile of Pegzilarginase In Patients with Arginase 1 Deficiency," is available in the July issue.

    Corporate

    • Strengthened the leadership team with appointment of Jonathan Alspaugh as chief financial officer and Jim Kastenmayer as general counsel.
      • Mr. Alspaugh joined Aeglea from Evercore, where he most recently served as a managing director in the firm's corporate advisory business.
      • Mr. Kastenmayer joined Aeglea from Viela Bio, where he provided strategic guidance and legal advice, including advising Viela in connection with the FDA approval and launch of its first commercial product. 
    • Expanded the board of directors with the appointment of Marcio Souza. Mr. Souza serves as the president & chief executive officer of Praxis Precision Medicines.
    • Participated in Rare Disease Awareness Week on Capitol Hill 2021, meeting with patient advocates and law makers to raise awareness and discuss the specific needs facing the rare disease community.
    • Supported the launch of the Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, of which Aeglea is a founding member.

    Upcoming Investor Events

    • Wells Fargo 2021 Virtual Healthcare Conference, September 9–10
    • HC Wainwright 23rd Annual Global Investment Conference, September 13–15
    • 2021 Cantor Virtual Healthcare Conference, September 27–30

    Second Quarter 2021 Financial Results

    As of June 30, 2021, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $130.4 million inclusive of the $21.5 million upfront cash payment received from Immedica Pharma AB pursuant to the March 2021 license and supply agreement. The Company expects its cash, cash equivalents and investments will enable it to fund its operating expenses and capital expenditure requirements into 2023.

    Aeglea recognized license and development revenues of $13.7 million in the second quarter of 2021, as a result of its license and supply agreement with Immedica for the commercial rights of pegzilarginase in certain territories outside the United States. The revenues recorded in the second quarter of 2021 are related to the transfer of the license and delivery of trial and regulatory services. Aeglea recognized no revenue for the corresponding period of 2020.

    Research and development expenses totaled $13.6 million for the second quarter of 2021 and $16.9 million for the second quarter of 2020. The decrease was primarily associated with completing certain pre-commercial manufacturing activities for Aeglea's lead product candidate, pegzilarginase. 

    General and administrative expenses totaled $6.8 million for the second quarter of 2021 and $4.7 million for the second quarter of 2020. This increase was primarily due to ramping-up the Company's commercial capabilities and infrastructure.

    Net loss totaled $6.8 million and $21.4 million for the second quarter of 2021 and 2020, respectively, with non-cash stock compensation expense of $2.1 million and $1.6 million for the second quarter of 2021 and 2020, respectively.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is a novel recombinant human enzyme, which has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. 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 ongoing 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. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough, Fast Track and Orphan Drug Designations from the FDA as well as Orphan Drug Designation from the European Medicines Agency.

    About AGLE-177 in Homocystinuria

    AGLE-177 is a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of homocysteine and homocystine. 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. Preclinical data demonstrated that AGLE-177, which is designed to lower abnormally high blood levels of homocysteine, improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. 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 began dosing patients in a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria in June 2021. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit http://aeglea.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 ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, 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 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 Annual Report on Form 10-Q for the quarter ended June 30, 2021 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.

    Financials

    Aeglea BioTherapeutics, Inc.

    Condensed Consolidated Balance Sheets

    (Unaudited)

    (In thousands, except share and per share amounts)







    June 30,





    December 31,







    2021





    2020



    ASSETS

















    CURRENT ASSETS

















    Cash and cash equivalents



    $

    34,765





    $

    90,095



    Marketable securities





    93,813







    56,178



    Prepaid expenses and other current assets





    5,790







    3,516



    Total current assets





    134,368







    149,789



    Restricted cash





    1,846







    1,842



    Property and equipment, net





    5,208







    5,642



    Operating lease right-of-use assets





    4,012







    4,230



    Other non-current assets





    1,548







    115



    TOTAL ASSETS



    $

    146,982





    $

    161,618





















    LIABILITIES AND STOCKHOLDERS' EQUITY

















    CURRENT LIABILITIES

















    Accounts payable



    $

    2,221





    $

    2,254



    Operating lease liabilities





    412







    319



    Deferred revenue





    5,530









    Accrued and other current liabilities





    12,441







    13,870



    Total current liabilities





    20,604







    16,443



    Non-current operating lease liabilities





    4,880







    5,129



    Deferred revenue, net of current portion





    2,274









    Other non-current liabilities





    200







    214



    TOTAL LIABILITIES





    27,958







    21,786





















    STOCKHOLDERS' EQUITY

















    Preferred stock, $0.0001 par value; 10,000,000 shares authorized

    as of June 30, 2021 and December 31, 2020; no shares issued and

    outstanding as of June 30, 2021 and December 31, 2020













    Common stock, $0.0001 par value; 500,000,000 shares authorized

    as of June 30, 2021 and December 31, 2020; 49,025,956 shares and

    47,959,086 shares issued and outstanding as of June 30, 2021

    and December 31, 2020, respectively





    5







    5



    Additional paid-in capital





    420,064







    415,824



    Accumulated other comprehensive income





    11







    11



    Accumulated deficit





    (301,056)







    (276,008)



    TOTAL STOCKHOLDERS' EQUITY





    119,024







    139,832



    TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY



    $

    146,982





    $

    161,618



     

    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,









    2021





    2020





    2021





    2020





    Revenue:



































    License



    $

    12,000





    $





    $

    12,000





    $





    Development fee





    1,696













    1,696











    Total revenue





    13,696













    13,696















































    Operating expenses:



































    Research and development





    13,579







    16,869







    25,434







    31,431





    General and administrative





    6,822







    4,691







    13,176







    9,151





    Total operating expenses





    20,401







    21,560







    38,610







    40,582





    Loss from operations





    (6,705)







    (21,560)







    (24,914)







    (40,582)









































    Other income (expense):



































    Interest income





    19







    161







    41







    461





    Other expense, net





    (52)







    (19)







    (83)







    (25)





    Total other income (expense)





    (33)







    142







    (42)







    436





    Loss before income tax expense





    (6,738)







    (21,418)







    (24,956)







    (40,146)





    Income tax expense





    (92)













    (92)











    Net loss



    $

    (6,830)





    $

    (21,418)





    $

    (25,048)





    $

    (40,146)









































    Net loss per share, basic and diluted



    $

    (0.10)





    $

    (0.40)





    $

    (0.38)





    $

    (0.93)





    Weighted-average common shares outstanding, basic and

       diluted





    65,631,906







    52,941,603







    65,618,207







    43,019,670





     

     

    (PRNewsfoto/Aeglea BioTherapeutics, Inc.)

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  3. AUSTIN, Texas, July 15, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the appointment of Jim Kastenmayer, Ph.D., J.D., as general counsel.

    "With his broad experience at both big pharma and emerging biotech and a strong intellectual property background, Jim brings a depth of experience that will be valuable as we advance our two clinical programs and evolve to a commercial-stage company," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "I'm pleased to welcome Jim to the Aeglea team and look forward to working with him closely…

    AUSTIN, Texas, July 15, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the appointment of Jim Kastenmayer, Ph.D., J.D., as general counsel.

    "With his broad experience at both big pharma and emerging biotech and a strong intellectual property background, Jim brings a depth of experience that will be valuable as we advance our two clinical programs and evolve to a commercial-stage company," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "I'm pleased to welcome Jim to the Aeglea team and look forward to working with him closely as we continue to execute on our corporate strategy."

    Mr. Kastenmayer joins Aeglea from Viela Bio, where, as general counsel, he provided strategic guidance and legal advice including advising the company in connection with the FDA approval and launch of Uplizna® as well as the company's acquisition by Horizon Therapeutics. Prior to Viela Bio, he served in roles of increasing responsibility at AstraZeneca including global legal director, where he advised on commercialization and market access strategies, collaboration agreements and handled legal proceedings, as well as senior patent director, accountable for delivering global IP estates for small and large molecule therapies. Earlier in his career, Mr. Kastenmayer served as IP counsel at MedImmune, and as an Associate at an IP boutique firm. Mr. Kastenmayer earned a J.D. from Georgetown University Law Center, a Ph.D. in biochemistry and cell & molecular biology from Michigan State University and a B.A. in biology from the University of Virginia and is a registered patent attorney.

    "I'm thrilled to be joining such a strong team, particularly at this pivotal time as we prepare for Phase 3 data later this year and advance our second program through the clinic," said Mr. Kastenmayer. "I believe Aeglea has the science, strategy and personnel in place to deliver on its mission of having an impact on the lives of patients with rare metabolic diseases and I look forward to being part of the journey."

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and devastating metabolic diseases with limited treatment options. 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 began dosing patients in a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria in June 2021. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have big impact on the lives of patients and their families. For more information, please visit http://aeglea.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 ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, 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, our cash forecasts, the potential addressable markets of 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, 2021 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.

     

    (PRNewsfoto/Aeglea BioTherapeutics, Inc.)

     

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  4. AUSTIN, Texas, July 14, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the publication of 20 week data from the Phase 1/2 clinical trial of pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D), a rare, progressive and devastating disease characterized by high levels of the amino acid arginine. Pegzilarginase is a novel, recombinant human arginase 1 enzyme designed to lower levels of arginine that is also being investigated in PEACE, an ongoing Phase 3 pivotal trial for the treatment of ARG1-D.

    The article, titled "Clinical Effect and Safety Profile…

    AUSTIN, Texas, July 14, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the publication of 20 week data from the Phase 1/2 clinical trial of pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D), a rare, progressive and devastating disease characterized by high levels of the amino acid arginine. Pegzilarginase is a novel, recombinant human arginase 1 enzyme designed to lower levels of arginine that is also being investigated in PEACE, an ongoing Phase 3 pivotal trial for the treatment of ARG1-D.

    The article, titled "Clinical Effect and Safety Profile of Pegzilarginase In Patients with Arginase 1 Deficiency," was lead authored by Dr. George Diaz, Division Chief of Medical Genetics in the Department of Genetics & Genomic Sciences at the Icahn School of Medicine at Mt. Sinai, New York, NY, and has been published in the July issue of the Journal of Inherited Metabolic Disease. The full publication can be accessed at https://onlinelibrary.wiley.com/doi/epdf/10.1002/jimd.12343.

    "This trial is the first time clinical outcomes have been used to assess the impact of ARG1-D and provides critical information in our understanding of the disease burden and potential for its treatment," said Dr. Diaz. "Given the serious and progressive nature of this condition, understanding how to limit or even reverse the course of the disease has the potential to fundamentally alter how we treat ARG1-D and improve the lives of patients and their families."

    The Phase 1/2 trial was designed to assess the safety and efficacy of pegzilarginase for the treatment of ARG1-D. These results represent data after only 20 doses of pegzilarginase. Key results from the study include:

    • Pegzilarginase was well tolerated, and the rates of treatment-related adverse events decreased over time.
      • Common treatment-related adverse events included hypersensitivity, vomiting, hyperammonemia, pruritus and abdominal pain.
      • Serious treatment-related adverse events included hypersensitivity and hyperammonemia, which were infrequent, managed with standard treatment and did not lead to any patient discontinuations.
    • All patients demonstrated a marked and sustained reduction in plasma arginine.
      • By Dose 20, 13 of 14 (93%) patients had arginine levels <200 µM and seven patients had arginine levels within the normal range (40-115 µM). 200 µM is the clinical goal level used at present, which is rarely achieved through currently available treatment options.
    • Eleven of 14 (79%) patients demonstrated improvements equal to or greater than the minimal clinically important difference (MCID) in one or more of three mobility assessments.

    "People living with ARG1-D face an immense burden from their disease every day with mobility challenges, intellectual disability, and apprehension about disease progression and a shortened life. The publication of these results in a peer reviewed journal mark an important validation for this program and we are very pleased to see that 79% of patients treated with pegzilarginase showed evidence of clinical improvements," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "The very high clinical responder rate in this Phase 1/2 trial using these assessments similar to those in PEACE, our pivotal Phase 3 study, gives us high confidence in our clinical program and its potential to demonstrate the effectiveness of pegzilarginase as a treatment option for people living with ARG1-D."

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

    The multicenter, global clinical trial enrolled 16 patients diagnosed with Arginase 1 Deficiency aged 2 years or older. Data are reported from the 12-week Phase 1/2 study (n=16) and the first 12 weeks of an open-label extension study. The Phase 1/2 study was conducted in two parts. In Part 1, patients received single ascending intravenous (IV) doses of pegzilarginase at 2-week intervals. In Part 2, patients received eight weekly repeat IV doses of pegzilarginase. After completion of the Phase 1/2 study, patients were eligible to enroll in the open-label extension study which consisted of weekly administration of subcutaneous (SC) doses of pegzilarginase. Patients in the trial continued to receive standard treatment approaches. The open-label extension study is ongoing.

    The primary endpoint of the study was assessment of safety and tolerability of pegzilarginase. Additional endpoints included decrease from baseline in plasma arginine level as well as mobility assessments, including 6MWT (Six-minute Walk Test) and GMFM (Gross Motor Function Measure) Part D and Part E. A Responder was defined based on a ≥1 MCID improvement in at least one of the three key mobility assessments.

    About Pegzilarginase in Arginase 1 Deficiency

    Pegzilarginase is a novel recombinant human enzyme, which has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. Aeglea's Phase 1/2 and Phase 2 open-label extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company's ongoing 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. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough, Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. 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 began dosing patients in a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria in June 2021. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit http://aeglea.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 ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, 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, our cash forecasts, the potential addressable markets of 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, 2021 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.

    (PRNewsfoto/Aeglea BioTherapeutics, Inc.)

     

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  5. AUSTIN, Texas, June 24, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the dosing of the first patient in the Company's first-in-human Phase 1/2 clinical trial investigating AGLE-177 for the treatment of Homocystinuria. The markedly elevated plasma homocysteine levels lead to a wide range of life-altering complications and reduced life expectancy in people with Homocystinuria. AGLE-177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma. Aeglea anticipates providing a clinical update on the program prior to…

    AUSTIN, Texas, June 24, 2021 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the dosing of the first patient in the Company's first-in-human Phase 1/2 clinical trial investigating AGLE-177 for the treatment of Homocystinuria. The markedly elevated plasma homocysteine levels lead to a wide range of life-altering complications and reduced life expectancy in people with Homocystinuria. AGLE-177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma. Aeglea anticipates providing a clinical update on the program prior to the end of 2021.

    "AGLE-177 is designed to address the markedly elevated homocysteine levels that are believed to be a key driver of the risk of serious complications in Homocystinuria, and has the potential to transform management of this burdensome and life-threatening condition," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "We believe this clinical trial will provide us with proof of concept of the homocysteine lowering effects of AGLE-177 in patients and provide us with the information needed to transition rapidly into a Phase 3 registrational trial."

    Cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria, is an inherited disorder of methionine metabolism that results in elevated homocysteine and homocystine in the plasma and urine. Disease management strategies, which are limited and include dietary protein restriction with amino acid replacement either alone or with vitamin B6, and betaine supplementation, are challenging, have poor adherence and many patients are not responsive to treatment. It is estimated that there are more than 30,000 people living with Homocystinuria in the global addressable markets, which includes approximately 15,000 to 18,000 B6-non-responsive patients.

    "Untreated Homocystinuria is a severe disease as a result of which individuals can develop significant complications, including visual impairment, skeletal abnormalities, reduced intellectual capacity, and recurrent thromboembolic events which may be life-threatening," said Principal Investigator Dr. Elaine Murphy, Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK. "Currently available treatment options are complex and do not always result in optimal metabolic control of the condition, leaving patients at risk of lifelong complications. There is an unmet need, therefore, for new therapies that can improve the outcomes and quality of life for this patient community."

    The multicenter, open label, dose escalation Phase 1/2 trial is anticipated to enroll 16-20 patients diagnosed with Homocystinuria aged 12 years or older at sites located in the United Kingdom and Australia. The primary endpoint is the safety and tolerability of AGLE-177. Secondary endpoints include pharmacokinetic assessments as well as reduction in plasma total homocysteine levels. Patients will be dosed weekly for four weeks, with four patients in each of four dosing cohorts and an option to include a fifth cohort as needed.

    About AGLE-177 in Homocystinuria

    AGLE-177 is a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of homocysteine and homocystine. 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. Preclinical data demonstrated that AGLE-177, which is designed to lower abnormally high blood levels of homocysteine, improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.

    About Aeglea BioTherapeutics

    Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. 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 began dosing patients in a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria in June 2021. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit http://aeglea.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 ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, 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, our cash forecasts, the potential addressable markets of 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, 2021 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.

    (PRNewsfoto/Aeglea BioTherapeutics, Inc.)

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