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Spur Therapeutics to begin Phase III gene therapy trial in Gaucher disease

Spur Therapeutics has announced that its gene therapy candidate for Gaucher disease will soon be moving to a Phase III trial after positive early data from a Phase I/II trial.

Spur, which is a portfolio company of Syncona, will begin the Phase III study in 2025 and announced it has completed enrolment for the Phase I/II trial (NCT05324943).

Gaucher disease is a rare, genetic, metabolic disorder where the enzyme Glucocerebroside builds up in areas such as the spleen, liver and bone marrow. Common manifestations include an abnormally enlarged liver and/or spleen, low levels of circulating red blood cells, low levels of platelets, and skeletal abnormalities.

The GALILEO-1 trial is investigating FLT201, an adeno-associated virus (AAV) gene therapy candidate, as a treatment for Gaucher disease. FLT-201 acts as a glucosylceramidase beta (GBA1) gene activator. Mutations in this gene cause Gaucher disease. The drug restores the function of the defective GBA1 gene and alleviates the condition.

Six patients have been dosed in the study and two of those patients have completed the full nine months follow-up. One patient has been excluded from efficacy analysis after it was found they had pre-existing neutralising antibodies to the AAV3 capsid used to deliver FLT201 below the protocol cut-off.

Phase I/II improvement in various biomarkers

Results show levels of Lyso-Gb1, an established biomarker of clinical response in Gaucher disease, were substantially reduced in four patients.

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The levels of haemoglobin and platelets were deemed normal or improved in five patients, another well-established regulatory endpoint of Gaucher disease.

Bone marrow burden also improved, and patients reported reduced fatigue and pain.

All six patients were included in the safety analysis, with FLT201 continuing to demonstrate a favourable safety and tolerability profile.

Further data from the Phase I/II trial of FLT201 is expected in the second half of the year.

Spur Therapeutics chair Chris Hollowood said: “We continue to be highly encouraged by the data coming from the FLT201 programme in Gaucher disease. The safety and tolerability of the treatment, alongside these compelling efficacy signals, suggest FLT201 has the potential to play an important role in the treatment of patients with Gaucher disease.”

In November 2023, Syncona acquired Freeline Therapeutics in a $28.3m deal. In June 2024, Syncona committed $50m as it merged Freeline Therapeutics with another biotech SwanBio to create Spur Therapeutics.

Based on data announced earlier this year, Spur received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation by the European Medicines Agency (EMA) for the therapy.

The Gaucher disease market is expected to reach $1.3bn across the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Israel) by 2032, according to a GlobalData report.

GlobalData is the parent company of the Clinical Trials Arena.

The pipeline includes therapies such as Sanofi’s venglustat malate, a substrate reduction therapy that GlobalData expects to secure a substantial share of the Gaucher disease market upon its launch.

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