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: catalin_adminHow often do patients complain of symptoms, such as Lethargy, Dehydration, Hypotonia, Stunted growth, Microcephaly, Seizures
Added By: catalin_adminHow 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: catalin_adminCan 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?
Are You Interested In These Questions?
Slingshot Insights Explained
Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.
Reason
*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.
