Strong KRRO-111 Preclinical Data De-Risks AATD Development & NYC Lunch May 26th

研报英文原文证据摘录

Strong KRRO-111 Preclinical Data De-Risks AATD Development & NYC Lunch May 26th

Sandler & Co.

AATD program, where this asset is a potential best-in-class SC GalNAc-conjugated 646 951-0692, dominic.lorenzi@psc.com

oligonucleotide designed to impact liver and lung biology. Specifically, KRRO-111 is

Changes Previous Current engineered to deliver the oligonucleotide to liver cells where it will co-opt the hepatocytes' Rating — Overweight

endogenous ADAR enzyme and repair pathogenic SNV on AAT mRNA to restore normal Price Tgt — US$30.00

protein production. Altogether, KRRO believes KRRO-111 may be the key to unlock FY26E Rev (mil) — US$0.0

clear efficacy with strong safety in AATD, especially considering its highly competitive FY27E Rev (mil) — US$0.0

preclinical evidence in the disease. In fact, KRRO-111 demonstrated best-in-class RNA FY26E EPS — US$(6.61)

editing in in-vivo studies where it corrected >90% of AAT transcripts in the liver cells FY27E EPS — US$(6.48)

that translated into ~90% M-AAT protein (Exhibit 1). Moreover, repeat-dosing models 52-Week High / Low US$55.89 / US$5.20

showed that 3 mg/kg dose of KRRO-111 at Q2W (after loading dose) nearly eliminated Shares Out (mil) 14.4

active Z protein production in PiZZ mice, with reduction of non-inclusion Z-AAT by Market Cap. (mil) US$143.8

~95% vs vehicle at Day 28 and 56 (Exhibit 2). Accordingly, this reflects near-complete Avg Daily Vol (000) 177

cessation of Z protein synthesis across the hepatocyte population, where it is important Div Yield 0.00%

to recognize >95% of severe clinical cases of AATD are homozygous for the PiZZ Fiscal Year End Dec

genotype. Of note, recall that AATD represents a highly debilitating disease where the

current SOC is QW IV augmentation therapy for AAT, where this only targets serum AAT