RARE Daily

Syncona Merges Two Biotechs Into Spur Therapeutics, Moving Gene Therapy Beyond Rare Diseases

June 18, 2024

Rare Daily Staff

Spur Therapeutics, formerly Freeline Therapeutics, said the new name and brand reflect a research strategy to unlock the promise of gene therapy beyond rare diseases into more prevalent conditions.

The move builds on compelling data for lead program FLT201, a highly differentiated gene therapy candidate for Gaucher disease, which is poised to enter phase 3 development in 2025.

“Our new name and new brand reflect our determination to alter the course of a disease with a single dose of genetic medicine and change the course of people’s lives,” said Michael Parini, Spur’s CEO. “By optimizing every component of our product candidates to get just the right expression, packaged and delivered to the body in just the right way, we are working to develop a new generation of therapies that spur gene therapy forward to transform the lives of even more patients.”

Spur also acquired SwanBio Therapeutics, which adds a potential first-in-class gene therapy program for adrenomyeloneuropathy (AMN), a devastating neurodegenerative disease, to its clinical-stage pipeline, as well as strengthened capabilities in central nervous system disorders that can be leveraged across both its AMN and Parkinson’s disease programs. The AMN program, SBT101, is currently in a phase 1/2 clinical trial, and Spur plans to report an initial safety update from the higher-dose cohort in this trial in the first half of next year. There are no approved treatments for AMN, and SBT101 is the only gene therapy candidate in development for the disease.

Life sciences investment firm Syncona, the founding shareholder in both Freeline and SwanBio, has committed an additional $50 million (£40 million) to support development of the expanded pipeline. Syncona Executive Partner and former SwanBio Executive Chair John Tsai has joined Spur’s Board of Directors.

Spur expects to initiate a phase 3 trial for FLT201 in 2025 in Gaucher disease. There is no cure for Gaucher disease, and even with current treatments, many patients continue to experience debilitating symptoms. Recently reported data from its phase 1/2 GALILEO-1 show that a single dose of FLT201 resulted in dramatic reductions in the toxic buildup of glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response and disease severity in Gaucher disease, in patients who have had persistently high levels despite years of treatment on currently approved therapies. Early signs of clinical improvement in fatigue and bone marrow burden were also observed. FLT201 has demonstrated a favorable safety and tolerability profile.

Building on its work in Gaucher disease, Spur’s research program in Parkinson’s disease is focused on a subset of patients with mutations in the GBA1 gene, the same gene implicated in Gaucher disease. The program leverages the same transgene as FLT201. Spur is further optimizing the transgene for expression in the brain and identifying the best capsid and route of administration to deliver its proprietary GBA1-85 transgene to key areas of the brain affected by Parkinson’s disease. Spur expects to select a development candidate later this year to progress into preclinical studies designed to support the program’s advancement into clinical trials. No disease-modifying therapies currently exist for Parkinson’s disease, and this program could be a first step toward a gene therapy for hundreds of thousands of people with GBA1 Parkinson’s worldwide.

Additionally, Spur has a research program, leveraging a suite of promising cardioprotective proteins to develop gene therapy candidates for cardiovascular diseases, starting with a severe subset of chronic heart failure.

Photo: Michael Parini, CEO of Spur Therapeutics

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