Bloomsbury Genetic Therapies Completes Successful Scientific Advice Meeting with the MHRA regarding its BGT-INAD Program

– Company intends to move forward with toxicology and biodistribution study in rodents ahead of a single, Phase 1/2/3 trial in Infantile Neuroaxonal Dystrophy (INAD) –

 

London, UK, 26 June 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, today announced another successful scientific advice meeting with the UK Medicines and Healthcare products Regulatory Agency (MHRA) confirming the formal toxicology and biodistribution requirements to commence a single, Phase 1/2/3 clinical trial with the Company’s product candidate, BGT-INAD, as a potential new therapeutic for the treatment of INAD.

The Company discussed its preclinical data package and plans for BGT-INAD with the MHRA during the Scientific Advice meeting on 28 April 2023. The MHRA confirmed that it was supportive of the Company’s proposal to conduct a single toxicology and biodistribution study in rodents supplemented by published data from precedent intra-brain AAV9 gene therapies, similar to its feedback on the Company’s other intra-brain AAV9 gene therapy program, BGT-NPC, prior to initiating a registrational, Phase 1/2/3 clinical trial for BGT-INAD in the UK. There was no requirement to conduct studies in a larger animal model.

The Company is currently completing efficacy studies for BGT-INAD in collaboration with its partner University College London (UCL). Following the advice and formal feedback received from the MHRA, subject to completing a manufacturing campaign with its chosen vector Contract Development and Manufacturing Organisation (CDMO) partner, the Company intends to initiate a toxicology & biodistribution study in 2024.

There are currently no disease-modifying treatments for INAD and affected patients derive limited clinical benefit from available medical therapies used for symptom control. With a view to designing an appropriate Phase 1/2/3 clinical trial to assess BGT-INAD in this high unmet need population, the MHRA also provided initial advice on disease rating scales and biomarkers currently being investigated by the Company and its  collaborators at UCL.

“This positive regulatory interaction with the MHRA once again validates Bloomsbury’s strategy of leveraging clinically proven technologies to accelerate the development of AAV gene therapy programs for rare diseases,” said Adrien Lemoine, Co-Founder & Chief Executive Officer of Bloomsbury. “We now have a clear view on the regulatory pathways to clinical translation for our three programs for neurological diseases, and we are committed to expeditiously bringing them to patients.”

 

– ENDS –

 

Enquiries

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About Bloomsbury

Bloomsbury is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. The Company was spun out of University College London and launched in October 2022 with funding from UCL Technology Fund. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs. For more information, please visit www.bloomsburygtx.com

 

About BGT-INAD 

BGT-INAD is an AAV9 investigational gene therapy designed to provide a potentially curative solution to Infantile Neuroaxonal Dystrophy (INAD) patients following a one-time injection in the cerebrospinal fluid (CSF). BGT-INAD is currently completing preclinical studies, with compelling preclinical efficacy data already demonstrated.

 

About Infantile Neuroaxonal Dystrophy (INAD)

INAD is a devastating and fatal autosomal recessive paediatric neurodegenerative disease caused by mutations in the PLA2G6 gene that affects the catalytic activity of its protein product, a calcium-independent phospholipase A2 protein located in mitochondria and axons. When PLA2G6 is defective, mitochondrial inner membrane integrity is damaged, and phospholipid accumulation in axons causes neuronal damage. Given greater disease awareness and advances in next generation sequencing technologies, INAD is increasingly diagnosed at a younger age, with the majority of affected children now diagnosed by 2 years of age. Symptoms usually begin between 6 months and 3 years of age and include a rapid decline in previously acquired skills such as walking and talking, with the subsequent evolution of seizure, axonal neuropathy and generalised spasticity leading to loss of neurodevelopmental skills and complete dependence for all daily living activities. From presentation, there is gradual loss of motor and cognitive skills, most losing ambulation by 5 years of age with the average age of death around 10 years of age. There are currently no disease-modifying treatments for INAD and affected patients derive limited clinical benefit from available medical therapies used for symptom control. The prevalence of INAD is poorly documented, it is estimated to be approximately 0.3/100,000 worldwide.