On May 7, 2019 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that engineers next generation human enzymes to provide solutions for diseases with unmet medical need, reported financial results for the first quarter ended March 31, 2019 and corporate highlights (Press release, Aeglea BioTherapeutics, MAY 7, 2019, View Source [SID1234535843]).

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"Aeglea built on its momentum from 2018 with a terrific start to the year, including the presentation of new compelling clinical data from the Phase 1/2 trial of pegzilarginase for Arginase 1 Deficiency," said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer. "We see marked and sustained reductions in plasma arginine in all patients with clinically meaningful improvements in mobility and adaptive behavior. We believe the emergent data from this trial reaffirms the design of our upcoming pivotal Phase 3 PEACE trial, with first patient dosing on track for this quarter."

Corporate Highlights

Pegzilarginase in Arginase 1 Deficiency: Continued progress towards commercialization of the Company’s lead program

Aeglea presented new positive Phase 1/2 data for pegzilarginase in patients with Arginase 1 Deficiency (ARG1-D) at the 2019 Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD). The oral presentation was delivered by Dr. George Diaz, M.D., Ph.D., Division Chief of Medical Genetics in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine at Mt. Sinai, New York, NY, and a Principal Investigator on the pegzilarginase Phase 1/2 trial. Highlights of the presentation included the following:

Plasma arginine reduction was statistically significant (p<0.001) at eight weeks with sustained control through longer-term dosing.
Five of five (100%) and eight of 14 (57%) patients showed overall clinical response (mobility or adaptive behavior) at 20 weeks and eight weeks, respectively. Clinical responses were effectively captured using mobility and adaptive behavior assessments.
Pegzilarginase was generally well tolerated. Serious adverse events included hypersensitivity and hyperammonemia. Hypersensitivity reactions were infrequent, managed with standard treatment and did not lead to any patient discontinuations.
Upcoming Events

Aeglea will present at the following conferences, with details regarding the date and time of the presentations and webcasts to be announced prior to the events.

Jefferies 2019 Healthcare Conference to be held June 4-7 in New York, NY.
BMO 2019 Prescriptions for Success Healthcare Conference to be held June 25 in New York, NY.
First Quarter 2019 Financial Results

As of March 31, 2019, Aeglea had available cash, cash equivalents and marketable securities of $123.7 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.

Research and development expenses totaled $14.4 million for the first quarter of 2019, compared with $6.9 million for the first quarter of 2018. The increase was primarily due to expanded clinical development activity, investment in manufacturing and pre-commercial activities for Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for our Homocystinuria program.

General and administrative expenses totaled $3.3 million for the first quarter of 2019, compared with $2.9 million for the first quarter of 2018. This increase was primarily due to additional employee headcount and compensation to support company growth.

Net loss totaled $17.2 million and $8.1 million for the first quarter of 2019 and 2018, respectively, with non-cash stock compensation expense of $1.1 million and $0.8 million for the first quarter of 2019 and 2018, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, 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 replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s Phase 1/2 data for pegzilarginase in patients with Arginase 1 Deficiency demonstrated clinical improvements and sustained lowering of plasma arginine. The Company intends to initiate its single, global pivotal Phase 3 PEACE trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction.