Antisense Therapeutics Limited announced the commencement of the second phase (chronic setting) of its program to study the effects of an antisense oligonucleotide (ASO) to CD49d (mouse equivalent of ATL1102) in a LGMDR2 animal model of dysferlin deficiency. Having previously successfully demonstrated drug activity (reducing target and immune cell RNA in muscle) in the first study in the dysferlin deficient animals, this follow-on chronic study will assess longer duration treatment effects on key disease progression endpoints including reduction in muscle adipose (fat) levels. The study is being undertaken in collaboration with experts in genetic muscle disease at the Murdoch Children's Research Institute (MCRI) in Melbourne and the Jain Foundation in the USA.

Suitably aged mice with the dysferlin mutation and related disease progression characteristics have been sourced via the Jain Foundation for use in the study. In this blinded and controlled study, mice will be treated for four months with results to follow mid-2023. LGMDR2 (also known as dysferlinopathy) is a rare genetic muscle disease that is caused by mutations in the dysferlin gene that leads to significant reduction or absence of dysferlin protein levels in muscle fibers.

LGMDR2 is characterized by muscle inflammation, fibrosis, adiposity (fat) and progressive weakness in the hip and shoulder area (the limb girdle) proximal muscles (those closest to the center of the body) with loss of ambulation and upper limb function in adulthood. LGMDR2 affects ~ 1 in 125,000 people. To date, no treatments have proven to be beneficial in slowing LGMDR2 disease progression.

The use of ATL1102 as a treatment for dysferlinopathy is covered in ANP's patent application PCTAU2020/050445 directed at modifying muscle performance by reducing muscle adiposity and provisional application 2021903024 that claims the use of ATL1102 to reduce thrombospondin-1 reported to be beneficial in treating the disease. As LGMDR2 is a rare disease, the Company expects to be eligible to apply for additional market exclusivity protection via Orphan Drug Designation in the US and Europe.