MedPath

ATL1102

Generic Name
ATL1102
Drug Type
Small Molecule
Background

ATL1102 is a second-generation antisense inhibitor of CD49d, an immunesystem protein known as VLA-4, an immune cell molecule. It works by entering cells and targeting genes.

Indication

Investigated for use/treatment in asthma and multiple sclerosis.

Percheron Therapeutics' Duchenne Muscular Dystrophy Trial Fails to Meet Primary Endpoint

• Percheron Therapeutics' Phase 2b trial of avicursen (ATL-1102) for Duchenne muscular dystrophy (DMD) failed to meet its primary endpoint, leading to a significant drop in share value. • The trial, involving wheelchair-bound boys, showed no statistically significant difference between avicursen and placebo in improving upper limb function at week 25. • Percheron plans a strategic review of its pipeline, exploring alternative indications for avicursen and considering new programs to restore shareholder value. • Despite the trial's failure, avicursen was reported to be safe and well-tolerated, prompting the company to explore its potential in other conditions.

Percheron Therapeutics to Broaden Pipeline Following Avicursen Phase IIb Failure in DMD

• Percheron Therapeutics will explore new indications for avicursen (ATL1102) after Phase IIb trial failure in non-ambulatory Duchenne muscular dystrophy (DMD) patients. • The company plans a strategic review of its pipeline to identify alternative value sources within its current assets, including atesidorsen (ATL1103) for acromegaly. • Percheron is also considering adding new programs to its portfolio and has initiated discussions with potential partners to enhance shareholder value. • Further data analysis from the avicursen trial is expected by Q1 2025 to determine future development in DMD, while conserving cash reserves.

Immutep's LAG-3 Agonist Shows Promise, Percheron and PYC Update on Neurological Disease Trials

• Immutep's IMP-761, a LAG-3 agonist, demonstrates a favorable safety profile in Phase I trials, potentially addressing autoimmune diseases like rheumatoid arthritis. • Percheron Therapeutics anticipates Phase 2b trial results for ATL-1102 in Duchenne muscular dystrophy, a genetic condition affecting muscle protein production. • PYC Therapeutics reports positive pre-clinical data for PYC-002, targeting the underlying cause of Phelan-McDermid syndrome, with human trials expected in 2026.

Percheron Therapeutics' Avicursen Shows Promise in Primate Study, Awaits FDA Discussion

• Percheron Therapeutics completed a nine-month toxicology study of avicursen in non-human primates, a key step toward US clinical trials. • The study results were consistent with earlier findings, showing no unexpected toxicities and full reversibility of observed effects. • Percheron plans to discuss the data with the FDA in early 2025, aiming to lift the clinical hold and advance avicursen's development. • A Phase IIb trial of avicursen in Duchenne muscular dystrophy is ongoing, with initial data expected to strengthen the FDA submission.

Avicursen Shows Promise in Phase II Trial for Duchenne Muscular Dystrophy

• Avicursen, developed by Percheron Therapeutics, is currently in Phase II clinical trials for Duchenne Muscular Dystrophy (DMD). • Phase II drugs for DMD have a benchmark of 61% for progressing into Phase III trials, according to GlobalData. • Avicursen (ATL-1102) is a second-generation antisense inhibitor of CD49d, administered subcutaneously or via inhalation. • Percheron Therapeutics focuses on developing antisense drugs for immunology disorders and rare diseases, including DMD and multiple sclerosis.
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