PPMD is thrilled to welcome Antisense Therapeutics to the Duchenne community. Antisense is developing a new potential therapy, ATL1102, using an antisense oligonucleotide (ASO) inhibitor aimed at treating muscle inflammation and associated muscle wasting.

Although our community is largely familiar with ASOs in the context of Duchenne exon skipping where a functional truncated (shortened) dystrophin protein is restored, this ASO is aimed at inhibiting function of a protein involved in the immune response. The ATL1102 ASO is not mutation specific and is an immunomodulatory product that will bind messenger RNA (mRNA) to prevent translation of a functional CD49d protein, which may help to reduce inflammation.

Antisense has conducted a small non-ambulatory study with matched natural history controls which showed encouraging results. They plan to pursue their next study in Europe in 2021, as well as move forward discussions with FDA to begin planning a trial in the US.

PPMD has invited Antisense to join us for a community webinar once they have more details on timing for the US trials. We look forward to working with the team at Antisense and learning more.

Read the Community Letter from Antisense

Dear Duchenne Community,

We are excited to introduce our company to the Community! We are dedicated to developing our therapeutic, ATL1102, an antisense inhibitor of the CD49d receptor, to help treat the muscle inflammation and associated muscle wasting in patients with DMD. We recently announced promising positive results from our Phase II study in non-ambulant boys compared to a matched natural history control from a database of patients in Rome, Italy. This is encouraging data as we move forward to test our therapy in a controlled trial in Europe.

Some recent company highlights include:

• Received a positive opinion from EMA on Orphan Drug Designation
• Granted Orphan Drug Designation by FDA
• Granted Rare Pediatric Disease Designation by FDA
• New ATL1102 data presented at the 25th International Annual Congress of the World Muscle Society
  • ATL1102 treated patients showed a statistically significant mean improvement in PUL2.0 scores (assessment of muscle function) at 24 weeks compared to a matched natural history control

We believe ATL1102 is presenting as an exciting prospect for the treatment of DMD patients and look forward to its continued development and progress. For further details on these new results and recent announcements please refer to our press releases. We plan to pursue our next study in Europe in 2021as well as moving forward with discussions with FDA to move toward studies in the US. We will keep the Duchenne community updated on our progress.

We look forward to hosting a webinar in the near future to go through the data and future plans for our Phase IIb trial. Please do not hesitate to reach out to us should you have any questions on our recent announcements or on the ATL1102 program in DMD generally.

Sincerely,
Your Antisense Therapeutics team

Contact Information:
Erin Cox
Patient Advocacy
Erin.cox@antisense.com.au

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