Expert Interview
Analyzing Aeglea BioTherapeutics’s Pegzilarginase in Patients with Arginase 1 Deficiency, following Phase 3 Data Release
Ticker(s): AGLETo understand the needs of argininemia patients, and assess Pegzilarginase’s potential.
Please tell us about your clinical experience. How many patients with argininemia do you see on a yearly basis? What is the standard of care and what treatments are available?
Added By: slingshot_insightsHow often do patients complain of symptoms, such as Lethargy, Dehydration, Hypotonia, Stunted growth, Microcephaly, Seizures
Added By: slingshot_insightsHow much of a need is there for novel treatments ? Where do you see Pegzilarginase fitting in this space? What other upcoming therapies are you excited about?
Added By: slingshot_insightsCan you tell us more about the urea cycle disorder class of genetic diseases?
How often do any of the following treatments get prescribed:Sodium benzoateSodium phenylbutyrateCarglumic acidGlycerol phenylbutyratePalonosetronOndansetron hydrochloride
Could you please discuss the trial results where:- normal plasma arginine levels (40-115µM) were achieved in 90.5% of pegzilarginase treated patients compared to none of the patients in the placebo arm.-he least squares mean GMFM-E score improved by 4.2 units for pegzilarginase treated patients and worsened by 0.4 units in the placebo arm (p=0.1087; 95% CI [-1.1, 10.2],. The least squares mean 2MWT distance increased 7.4 meters in pegzilarginase treated patients and 1.9 meters in the placebo arm (p=0.5961; 95% CI [-15.6, 26.7]).
The company’s management stated that it was challenging to design a study where there’s no clinical precedent? How much does this statement ring true to you?
How likely is the positive biomarker to eventually lead to significant clinical benefit even without the actual benefits shown in patients?
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