Antisense Therapeutics (ASX:ANP) Announces Positive Results for First Study in Rare Muscular Dystrophy – The Market Herald

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  • Antisense Therapeutics (ANP) announces positive results from its first animal study of its ATL1102 treatment for limb-girdle muscular dystrophy R2 (LGMDR2)
  • As part of the study, ANP treated mice with the rare disease with ATL1102 at three different dose levels
  • Results show that the lowest dose of the treatment caused a ‘significant’ decrease in RNA levels of some key immune cells in the quadriceps muscle
  • This essentially means that Antisense will now move its study into a second phase, scheduled for later this year, to test its product’s ability to reduce fat levels, muscle loss and damage.
  • ANP shares are up 1.19% and trading at 8.5 cents at 1:44 p.m. AEST

Antisense Therapeutics (ANP) announces positive results from its first animal study of its ATL1102 treatment for limb-girdle muscular dystrophy R2 (LGMDR2).

LGMDR2 is a rare genetic muscle disease caused by mutations in the dysferlin gene that lead to a significant reduction or absence of dysferlin protein levels in muscle fibers.

Also known as dysferlinopathy, the condition caused by this loss of dysferlin is characterized by muscle inflammation, fibrosis, adiposity and weakness in the hip and shoulder area.

According to Antisense, there is currently no treatment available for LGMDR2.

The company’s latest study, undertaken in collaboration with genetic muscle disease experts from the Murdoch Children’s Research Institute in Melbourne and the Jain Foundation in the US, tested its antisense product in a protein known as CD49d in mice with dysferlin loss.

As part of the study, the mice were treated with ATL1102 – an antisense inhibitor of the CD49d receptor – at three different dose levels, with treatment given once a week for six weeks.

Antisense said the results showed that the lowest dose of its treatment caused a “significant” decrease in RNA levels of CD49d and other key immune cells in the quadriceps muscle.

Antisense Managing Director and CEO Mark Diamond said the results mean the company can further explore its key treatment ATL1102 for a new muscle disease.

“This was a strategic decision on the part of the company to capitalize on the large dataset generated to date on ATL1102 and expand ANP’s product portfolio,” said Mr. Diamond.

“The results obtained in this first LGMDR2 animal study show the required effects on the targeted immune cells.”

The company said it now plans to move forward with a second phase of its LGMRD2 study to test its product’s ability to reduce fat levels, muscle loss and damage.

The second study is scheduled for the third or fourth quarter of 2022 and will last four months.

ANP shares rose 1.19% and were trading at 8.5 cents at 1:44 p.m. AEST.