Spur Therapeutics Reports Positive Phase 1/2 GALILEO-1 Trial Results for FLT201 at WORLDSymposium

Spur Therapeutics has unveiled promising data from its Phase 1/2 GALILEO-1 trial concerning FLT201, a gene therapy candidate targeting Gaucher disease type 1. This investigational therapy employs an adeno-associated virus (AAV) to potentially offer a novel treatment approach for this chronic condition. The study results highlighted significant and lasting improvements in glucosylsphingosine (lyso-Gb1) levels, a critical marker for Gaucher disease, alongside enhancements or stabilization in patients' blood counts, organ volumes, and bone marrow burden following a single infusion of FLT201. The research findings were recently shared through oral and poster presentations at the 21st Annual WORLD Symposium, underlining FLT201’s potential to elevate treatment standards for Gaucher disease.

Gaucher disease is a serious genetic disorder characterized by a deficiency in the enzyme glucocerebrosidase (GCase), necessary for lipid metabolism. This deficiency leads to the accumulation of harmful lipid substrates in the body’s tissues and organs, resulting in a variety of symptoms including organ enlargement, fatigue, bone pain, and diminished lung function. Traditional treatments have provided some relief, but many patients continue to experience significant symptoms. Gaucher disease affects around 18,000 individuals across several countries including the United States, the United Kingdom, and various European nations.

Pamela Foulds, M.D., the Chief Medical Officer at Spur, emphasized the potential benefits demonstrated in the trial, noting the therapy’s ability to address persistent symptoms in patients who had been on existing treatments for several years. The therapy's safety profile and the durability of the response were particularly promising, suggesting that FLT201 could reduce the treatment burden for Gaucher disease patients. Michael Parini, the company's CEO, highlighted FLT201's design to overcome limitations of current therapies by using a more stable GCase enzyme variant packaged in an efficient capsid, potentially offering advantages over other gene therapies in development.

The Phase 1/2 GALILEO-1 study involved six patients receiving a low dose of FLT201, with follow-ups ranging from nine to 17 months. The trial’s outcome showed improved biomarker levels and sustained safety and efficacy, leading to the anticipation of a Phase 3 study. This forthcoming study, set to begin in the latter half of 2025, aims to further investigate FLT201's potential for accelerated approval based on key outcomes such as lyso-Gb1 reduction and hemoglobin level improvements.

FLT201 seeks to deliver a one-time treatment option by utilizing Spur’s AAVS3 capsid technology to deliver GCase85, an engineered enzyme variant designed to prevent disease progression and alleviate symptoms. The goal is to allow patients to discontinue lifelong treatments currently required to manage the disease.

Spur Therapeutics, the company behind FLT201, is dedicated to developing transformative gene therapies for chronic conditions. Their research extends beyond Gaucher disease, with initiatives targeting adrenomyeloneuropathy and explorations into more prevalent conditions like Parkinson’s disease, dementia, and cardiovascular diseases. Their mission centers on unlocking the potential of gene therapy to achieve significant clinical outcomes and improve patient lives.