- Antisense Therapeutics (ANP) announces positive results from its first animal study of its ATL1102 treatment against limb-girdle muscular dystrophy R2 (LGMDR2)
- As part of the study, ANP treated mice that had the rare disease with ATL1102 at three different dose levels
- The results show that the lowest dose of the treatment brought about a “significant” decrease in the RNA levels of some key immune cells in the quadriceps muscle
- This essentially means Antisense will now progress its study to a second phase, slated for later this year, to test its product’s ability to reduce fat levels, muscle loss and damage
- Shares in ANP are up 1.19 per cent and trading at 8.5 cents at 1:44 pm AEST
Antisense Therapeutics (ANP) has announced positive results from its first animal study of its ATL1102 treatment against 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 in or absence of dysferlin protein levels in muscle fibres.
Also known as dysferlinopathy, the condition brought about by this dysferlin loss is characterised by muscle inflammation, fibrosis, adiposity, and weakness in the hip and shoulder area.
According to Antisense, there are currently no available treatments for LGMDR2.
The company’s latest study, taken on in collaboration with genetic muscle disease experts at 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, mice were treated with ATL1102 — an antisense inhibitor of the CD49d receptor — at three different dose levels, with treatment administered once a week for six weeks.
Antisense said results showed that the lowest dose of its treatment brought about a “significant” decrease in the RNA levels of CD49d and other key immune cells in the quadriceps muscle.
Antisense Managing Director and CEO Mark Diamond said these results meant the company could further explore its key ATL1102 treatment against a new muscle disease.
“This was a strategically-minded move on behalf of the company to capitalise on the extensive data package generated to date on ATL1102 and to broaden ANP’s product pipeline,” Mr Diamond said.
“The results achieved in this first LGMDR2 animal study are showing the requisite effects on targeted immune cells.”
The company said it now planned 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 planned for either the third or fourth quarter of 2022 and will run for four months.
Shares in ANP were up 1.19 per cent and trading at 8.5 cents at 1:44 pm AEST.