PYC 2.50% 10.3¢ pyc therapeutics limited

Ann: Q3 Investor Call Presentation, page-14

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    Yes, the higher dose will be subject to discussion and agreement with the FDA but I would think it should be fine.

    PYC’s NHP toxicology testing demonstrated no adverse events at 50mcg which is the equivalent of 150mcg in humans.

    90mcg is also certainly well below the doses used in ProQR’s Phase 1/2 clinical trial of sepofarsen in LCA. In multiple dose testing their low dose was 160mcg loading followed by 80mcg maintenance; mid-dose was 320mcg loading followed by 160mcg maintenance and the high dose was 500mcg loading followed by 270mcg dosing. Macular edema was seen in the high dose group (500/270mcg). Pro QR ultimately ran with 80/40mcg and 160/80mcg dosing in their pivotal trial.
 
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