On August 5, 2021 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, reported financial results for the second quarter ended June 30, 2021, and reviewed recent corporate updates and program highlights (Press release, Aeglea BioTherapeutics, AUG 5, 2021, View Source [SID1234585789]).

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"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.