Our Expanded Access Policy

Last updated: September 2026

Pretzel Therapeutics is a clinical-stage biotechnology company advancing a new class of medicines designed to restore mitochondrial function, improve cellular energetics and impede disease progression across a range of neurological and rare diseases. We are committed to conducting clinical trials to evaluate the safety and efficacy of our investigational therapies and to generating the evidence needed to obtain regulatory approval and, ultimately, make these therapies available to as many patients as possible.

Expanded access

New investigational therapies must be reviewed and approved by a regulatory authority, such as the U.S. Food and Drug Administration (FDA), before they become commercially available. In some circumstances, a treating physician may request access to an investigational therapy for an individual patient who cannot participate in a clinical trial. This process is called expanded access, which includes compassionate use. Expanded access is not offered by every pharmaceutical or biotechnology company or for every investigational therapy.

Our current policy

Because our investigational therapy has not yet been clinically evaluated in patients, and much remains unknown about its safety and efficacy, Pretzel Therapeutics does not currently offer an expanded access program.

Clinical trials are designed to evaluate whether an investigational therapy is safe and may benefit patients, while generating data that regulatory authorities use to determine whether the therapy can be approved for broader use. Pretzel Therapeutics believes that participation in a clinical trial is the most appropriate way for patients to access our investigational therapy and that this approach best supports the goal of advancing its development as quickly as possible and making it available to the greatest number of people living with rare mitochondrial disease.

We encourage patients and their families to speak with their physicians about potential clinical trial participation. Information about our investigational therapy can be found at www.pretzeltx.com.

We reserve the right to review and amend this policy as our clinical development program progresses and as additional information becomes available.