Pretzel Therapeutics Answers Key Questions from the POLG Community

2 Sep, 2026

Following the POLG Foundation Summit, Pretzel Therapeutics has shared updated answers to community questions about PX578, their first‑in‑class investigational therapy for POLG disease. They emphasise that patient partnership is essential: families can support the programme by joining registries, participating in natural history studies such as PIONEER, and engaging in community forums — including the externally‑led POLG PFDD Meeting on 17 November, where patient voice will help inform the FDA.

Pretzel reaffirmed its commitment to developing PX578 for children, while explaining the regulatory need for a stepwise approach: testing first in adults, then moving carefully into paediatric studies once foundational safety data is established. They are also exploring outcome measures and biomarkers relevant to both adults and children, including gait, neuropathy, seizure activity, liver function, developmental regression, and mitochondrial stress markers such as GDF‑15 and FGF‑21.

Over the next 1–3 years, the community can expect completion of the Phase 1 healthy volunteer study, initiation of a Phase 2 trial in adults with POLG disease, and continued progress toward regulatory approval. Pretzel closed by recognising families as experts in their own right and encouraging ongoing dialogue as development continues.