Ann: Dosing commenced in the ATL1102 toxicology study, page-2

Antisense Therapeutics Limited [ASX:ANP | US OTC:ATHJY | FSE:AWY] (ANP or Company) today
announced the commencement of dosing in the nine-month chronic monkey toxicology study of
ATL1102. Dosing of all animals will be completed in November this year with study outcomes on track
for reporting in 1H’24 as previously advised1
. The toxicology study is intended to support the
advancement of the ATL1102 program in the US for Duchenne muscular dystrophy (DMD) or any other
clinical application of ATL1102.
Successful completion of the toxicology study is expected to be the final requisite step for the FDA to
allow dosing of ATL1102 for a term longer than six months in the US. Successful completion of the
study should also allow ANP to apply for expedited program status with the US Food and Drug
Administration (FDA) including Fast Track or potential Breakthrough Therapy designation. US FDA has
already granted ATL1102 an Orphan Drug Designation and a Rare Pediatric Disease Designation for
the treatment of DMD.
The reporting of key study findings from the nine-month chronic monkey toxicology study in 1H’24 is
due around the same time as the results from the blinded phase of the ATL1102 Phase IIb DMD clinical
study are expected, which could then allow the Company to share with FDA and other regulatory
bodies a compelling data package of clinical and toxicology study results for potential discussions on
accelerated regulatory pathways to registration. In addition, and subject to it meeting the eligibility
criteria, the Company may also be in a position to receive a future Pediatric Review Voucher (PRV)2,3.
In recent years the price paid for PRVs has ranged between US$95 million and US$110 million4
.