Myotonic Dystrophy Type 1 (DM1) is a rare, multisystem genetic disorder that not only presents with diverse clinical symptoms but also mimics many of the biological hallmarks of aging.
Our Focus
At ElixIRA Pharma AG, our main leading asset EXL019 aims to extend healthy lifespan by targeting the fundamental biology of aging. DM1 mirrors key hallmarks of aging—mitochondrial decline, proteostasis loss, stem cell exhaustion—and serves as our proof-of-concept indication for platform validation.
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Individuals affected (US/EU)
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Approved disease-modifying therapies
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Aging hallmarks modeled
Pipeline
Our lead asset, ELX019, is a disease-modifying combination therapy addressing key cellular aging pathways (AMPK/mTORC1), with Myotonic Dystrophy Type 1 (DM1) as our lead indication.
| Program / Indication | Target | Preclinical | Phase 1/2 | Phase 3 | Registration |
|---|---|---|---|---|---|
| ELX019 – DM1 | AMPK / mTORC1 | ✔ | ● |
Join us in delivering affordable, accessible treatment to better serve the DM1 community.