NEW YORK – AviadoBio on Monday said the US Food and Drug Administration has cleared its investigational new drug (IND) application allowing it to start testing the gene therapy AVB-101 in certain patients with dementia.
London-based AviadoBio is developing AVB-101 as a one-time treatment for patients with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. The gene therapy is designed to deliver a normal version of GRN to patients' brains through a bilateral, MRI-guided intrathalamic infusion. Based on preclinical studies, the company is hoping AVB-101 will restore levels of the progranulin glycoprotein lacking in patients with FTD-GRN.
"Our preclinical program shows robust biodistribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects," AviadoBio Chief Medical Officer David Cooper said in a statement.
In the Phase I/II dose-escalation trial, dubbed ASPIRE-FTD, AviadoBio is assessing the safety and preliminary efficacy of AVB-101 in this setting. The trial is already underway in Europe, and the latest IND clearance from the FDA will pave the way for the firm to open additional study sites in the US next year.
The FDA has also granted AVB-101 fast-track status, which allows sponsors to meet with the agency more frequently to discuss development plans, submit data on a rolling basis, and apply for accelerated approval or priority review. The gene therapy also has orphan drug status from regulators in the US and EU.
FTD is an early-onset form of dementia that manifests in people under age 65. People typically die within three to 10 years of diagnosis. Around one-third of patients have FTD due to inherited mutations in genes such as GRN.
"Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations," said AviadoBio CEO Lisa Deschamps. "Receiving fast-track designation underscores the significant need for treatment options for these patients, and we are eager to soon open US clinical trial sites and offer an innovative option for eligible patients with FTD-GRN."