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."
