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Aeglea BioTherapeutics (AGLE) Stock: Why The Price Increased Over 20% TodayApr 12th, 2022 5:43 EST
- The stock price of Aeglea BioTherapeutics, Inc. (Nasdaq: AGLE) increased by over 20% pre-market today. This is why.
The stock price of 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 – increased by over 20% pre-market today. Investors are responding positively to Aeglea BioTherapeutics announcing that they shared additional data from the PEACE Phase 3 study during a poster presentation at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting being held in Orlando, FL from April 10-13. And the poster, titled Pegzilarginase in Arginase 1 Deficiency: Results of the PEACE Pivotal Phase 3 Clinical Trial (Abstract #30), will also be available on the publications section of Aeglea’s website and includes new data on patient-level outcomes, additional secondary endpoints, and previously announced topline results.
Pegzilarginase is known as a novel, recombinant human arginase 1 enzyme that has been shown in clinical trials to normalize the elevated levels of the amino acid arginine in patients with Arginase 1 Deficiency (ARG1-D), a rare, progressive disease characterized by high levels of arginine. And people living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.
Results from the PEACE Phase 3 Study
PEACE is known as a global, randomized, double-blind, placebo-controlled trial that enrolled 32 patients with ARG1-D aged two years and older. And the study was designed to assess the effects of treatment with pegzilarginase (n=21) versus placebo (n=11) from baseline through a prespecified 24-week treatment period.
Previously Announced Topline Results
— Achieved the primary endpoint with a highly statistically significant 76.7% reduction in mean plasma arginine in pegzilarginase treated patients (p<0.0001) compared to placebo.
— Normal plasma arginine levels (40-115µM) were achieved in 90.5% of pegzilarginase treated patients compared to no patients in the placebo arm.
— Accompanying improvements in the key secondary mobility assessments in pegzilarginase treated patients compared to patients in the placebo arm.
— Gross Motor Function Measure Part E (GMFM-E): The least squares mean score improved by 4.2 units for pegzilarginase treated patients and worsened by 0.4 units in the placebo arm (p=0.1087), establishing a positive trend.
— 2-minute walk test (2MWT): The least squares mean distance increased 7.4 meters in pegzilarginase treated patients and 1.9 meters in the placebo arm (p=0.5961).
— Pegzilarginase was well-tolerated and safety data were consistent with results from previous clinical trials. Adverse events were generally mild to moderate in severity. And there were no study discontinuations due to adverse events.
Patient-Level Outcomes Analysis
In an analysis of individual patients that were Gross Motor Function Classification System (GMFCS) Level I-III with predefined clinical response criteria there were clinically important differences between the pegzilarginase treated patients (n=17) and placebo (n=9).
— Eleven patients (65%) treated with pegzilarginase reached or exceeded prespecified response criteria for at least one mobility assessment compared to four patients (44%) receiving placebo.
— Eight patients (47%) met or exceeded prespecified clinical response criteria for at least two of the mobility outcomes compared to no patients receiving placebo.
— Six of the patients meeting or exceeding the clinical response threshold for at least two mobility outcomes also showed no worsening on any other mobility endpoint. Additional analysis was conducted on these six patients to compare improvement to age- and sex-matched norms. (Table 1 below)
Additional Secondary Efficacy Endpoints
— In a post hoc analysis correcting for a missed assessment that was improperly scored as 0 rather than “not assessed,” the least-squares mean GMFM-D score of patients treated with pegzilarginase improved from baseline by 2.25 units compared to placebo (p=0.0896).
— Pegzilarginase treated patients also showed statistically significant biochemical improvements in measures of ornithine and guanidino compounds compared to placebo, consistent with pegzilarginase mechanism of action. (Table 2 below)
“Our clinical development program for pegzilarginase has provided the first quantitative insights into the disease burden of ARG1-D and sheds new light on both the severity of the disease and the devastating effect of uncontrolled arginine levels in these patients. The data from PEACE show the ability of pegzilarginase to markedly improve arginine control and its impact on a broad range of disease-related abnormalities. We are pleased with these results and believe pegzilarginase has the potential to change the lives of the patients and families living with ARG1-D.”
— Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea
“It is great to see these additional results and the consistency of the effects as we conduct further analysis of the data from the PEACE Phase 3 study. The lowering of guanidino compounds, downstream metabolites of arginine, supports the mechanism of action of pegzilarginase in lowering arginine levels, the key driver of disease. In rare diseases, and in particular trials conducted in small patient populations, much of the story unfolds in looking at the individual patient responses. We are excited about the results that we see from PEACE with nearly half of the evaluable pegzilarginase treated patients demonstrating clinically meaningful improvement in two or more mobility assessments. This is a remarkable result given that ARG1-D is a progressive disease with a devastating impact on mobility.”
— Eric Bradford, M.D., M.B.A., chief development officer at Aeglea
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