Pretzel Therapeutics has received Fast Track Designation from the US Food and Drug Administration (FDA) for PX578, an investigational treatment for POLG-mediated primary mitochondrial disorders, also known as POLG disease.

POLG disease is a rare genetic condition that affects mitochondrial function and can progressively damage multiple organs. There are currently no approved disease-modifying treatments for the condition.

The FDA designation follows clearance for Pretzel Therapeutics to begin a clinical trial of PX578 in patients. The Phase 1/2 POLARIS study will assess the treatment in adults with POLG disease, following results from a Phase 1 study in healthy volunteers conducted in New Zealand.

Fast Track Designation is intended to support the development of medicines for serious conditions where there is an unmet medical need. It allows closer communication between the developer and FDA during the development process and may support a faster review of the treatment if relevant requirements are met.

POLG disease is caused by changes in the POLG gene, which plays an important role in maintaining mitochondrial DNA (mtDNA). Impaired mitochondrial DNA maintenance can reduce cellular energy production and lead to progressive damage across different organs.

The condition can affect people at different ages, with symptoms and disease progression varying according to the age of onset. Childhood-onset disease can involve liver problems, seizures and cognitive decline. Juvenile and adult-onset disease may cause problems such as ataxia, peripheral neuropathy and seizures, while later-onset disease can involve muscle weakness, drooping eyelids and restricted eye movement.

Because of its wide range of symptoms, POLG disease can be difficult to diagnose and is believed to be underdiagnosed or misdiagnosed. The condition can cause significant disability and may lead to early mortality.

PX578 is an investigational small-molecule therapy designed to enter the central nervous system and activate the mitochondrial DNA polymerase POLG. Pretzel Therapeutics is developing the treatment to increase mitochondrial DNA levels and improve mitochondrial function.

Preclinical studies and findings from the Phase 1 healthy volunteer study are being used to support the continued development of PX578. The company plans to evaluate the candidate further in patients with POLG disease as it progresses towards Phase 2 development.

 

Source: pretzeltx.com

Report Abuse