Spur Therapeutics Unveils New Name and Brand

Spur Therapeutics, previously known as Freeline Therapeutics, has rebranded to better reflect its commitment to revolutionizing gene therapy and advancing genetic medicine. The company has announced significant progress in its mission to develop next-generation gene therapies. Highlighting its efforts, Spur is focusing on optimizing every aspect of its product candidates to achieve the best possible therapeutic outcomes.

Spur's lead program, FLT201, targets Gaucher disease and is expected to enter Phase 3 clinical trials by 2025. Gaucher disease, a rare genetic disorder, currently has no cure, and existing treatments often fail to alleviate the symptoms fully. Recent data from Spur's Phase 1/2 GALILEO-1 trial have shown promising results, with a single dose of FLT201 significantly reducing the toxic buildup of glucosylsphingosine (lyso-Gb1), a key indicator of the disease's severity. Patients in the trial also exhibited early signs of clinical improvement, such as reduced fatigue and bone marrow burden. The therapy has demonstrated a favorable safety and tolerability profile, positioning FLT201 as a potential new standard of care for Gaucher disease.

In addition to its work on Gaucher disease, Spur is broadening its research to address more prevalent chronic conditions. The company is developing a gene therapy program for Parkinson’s disease, particularly focusing on patients with mutations in the GBA1 gene, which is also implicated in Gaucher disease. This program utilizes the same transgene as FLT201, modified for optimal expression in the brain. Spur is currently identifying the best method to deliver this transgene to affected brain areas and expects to select a development candidate for preclinical studies later this year. If successful, this could lead to the first disease-modifying gene therapy for Parkinson’s disease, potentially benefiting hundreds of thousands of patients globally.

Furthermore, Spur is expanding its pipeline to include treatments for cardiovascular diseases. The company is leveraging a suite of cardioprotective proteins to develop gene therapy candidates for chronic heart failure, starting with a severe subset of this condition.

Spur's ambitions have been bolstered by the acquisition of SwanBio Therapeutics, which adds a novel gene therapy program for adrenomyeloneuropathy (AMN) to its clinical-stage pipeline. AMN is a severe neurodegenerative disease with no approved treatments. The acquired program, SBT101, is currently in a Phase 1/2 clinical trial, and Spur plans to report initial safety data from this trial's higher-dose cohort in the first half of next year. This acquisition also enhances Spur's expertise in central nervous system disorders, benefitting both its AMN and Parkinson’s disease programs.

Syncona Ltd., a leading life science investor and founding shareholder in both Freeline and SwanBio, has committed an additional $50 million to support the development of Spur's expanded pipeline. Syncona Executive Partner and former SwanBio Executive Chair John Tsai has joined Spur’s Board of Directors, reinforcing the company's strategic direction.

According to Chris Hollowood, CEO of Syncona Investment Management Limited and Chairman of Spur's Board of Directors, Spur's broadened pipeline shows great promise. With its differentiated lead program and the addition of a potentially first-in-class clinical asset, Spur is well-positioned to become a leader in gene therapy, developing one-time treatments for debilitating chronic diseases and potentially setting new standards of care.

Spur Therapeutics remains dedicated to its mission of creating life-changing therapies that can transform the lives of patients with chronic conditions. By optimizing their product candidates and expanding their research focus, Spur aims to push the boundaries of gene therapy and bring hope to more patients worldwide.