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Exhibit 99.1
Aeglea BioTherapeutics Reports First Quarter 2022 Financial Results and Provides Program Updates
Pegzilarginase BLA submitted to the FDA; if approved, pegzilarginase would be the first FDA-approved treatment for Arginase 1 Deficiency
Additional PEACE data supporting potential efficacy of pegzilarginase presented at SIMD
Enrollment continues in Phase 1/2 trial of AGLE-177; IND now open for U.S. site initiation
Austin, Texas, May 5, 2022 -
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 first quarter 2022 and provided program updates.“We have started off 2022 with significant progress in both our clinical programs. With AGLE-177, we were pleased to see total homocysteine lowering in all patients in our first, low dose cohort and we look forward to sharing clinical data later this year. This program is another example of innovation with human enzyme therapies and has the potential to address a significant unmet need,” said Anthony Quinn, M.B., Ch.B., Ph. D., president and chief executive officer of Aeglea. “I’m also proud of the work we have done with our pegzilarginase program enabling our recent BLA submission to the FDA with what we believe are very compelling data to support approval for the treatment of Arginase 1 Deficiency. Some of these data were presented recently at the SIMD Annual Meeting, providing additional insight into the potential positive impact of pegzilarginase treatment. We look forward to working with the FDA throughout the review of our BLA as they assess the potential effectiveness of pegzilarginase in addressing the unmet need that impacts the lives of Arginase 1 Deficiency patients and their families.”
Program Updates
Pegzilarginase in Arginase 1 Deficiency
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Submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D). |
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Requested Priority Review at the time of submission. |
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Presented new data from the PEACE Phase 3 clinical trial at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting, including patient-level outcomes, results from additional secondary endpoints and previously announced topline results. Highlights of the data presented include: |
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76.7% reduction in mean plasma arginine in pegzilarginase treated patients compared to placebo (p<0.0001); normal plasma arginine levels achieved in 90.5% of pegzilarginase treated patients compared to no patients receiving placebo. |
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Eleven patients (65%) treated with pegzilarginase reached or exceeded response criteria for ≥1 mobility assessment compared to four patients (44%) receiving placebo. |
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Eight patients (47%) treated with pegzilarginase met or exceeded prespecified clinical response criteria for ≥2 mobility assessments compared to no patients receiving placebo. |
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Aeglea Biotherapeutics, Inc.'s Definitive Proxy Statement (Form DEF 14A) filed after their 2022 10-K Annual Report includes:
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In July 2020, we filed and the SEC declared effective a shelf registration statement on Form S-3, or the 2020 Registration Statement, for the potential offering, issuance and sale by us of up to $400.0 million of our common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock and debt securities, subscription rights to purchase common stock and units consisting of all or some of these securities.
The shares of common stock and pre-funded warrants sold in the 2020 Public Offering were offered pursuant to a shelf registration statement on Form S-3, or the 2019 Registration Statement, declared effective in February 2019 by the SEC for the potential offering, issuance and sale by us of up to $200.0 million of our common stock, warrants to purchase common stock, and other security types and subscription rights.
Pegzilarginase treated patients also showed statistically significant biochemical improvements in measures of ornithine and guanidino compounds compared to the placebo arm, consistent with the pegzilarginase mechanism of action.
The net change in operating assets and liabilities of $2.9 million was primarily related to a $7.8 million increase in deferred revenue due to receiving a $21.5 million upfront payment under the Immedica Agreement offset by the recognition of revenue allocated to the license, PEACE trial and BLA filing.
These increases will likely include higher costs related to the hiring of additional personnel and fees to outside consultants, lawyers and accountants, among other expenses.
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Financial Statements, Disclosures and Schedules
Inside this 10-Q Quarterly Report
Material Contracts, Statements, Certifications & more
Aeglea Biotherapeutics, Inc. provided additional information to their SEC Filing as exhibits
Ticker: AGLE
CIK: 1636282
Form Type: 10-Q Quarterly Report
Accession Number: 0001564590-22-027895
Submitted to the SEC: Thu Aug 04 2022 6:04:25 AM EST
Accepted by the SEC: Thu Aug 04 2022
Period: Thursday, June 30, 2022
Industry: Pharmaceutical Preparations