AviadoBio Receives FDA IND Clearance and Fast Track Designation for AVB-101 in the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations
Tuesday, November 07, 2023
AviadoBio, an innovative gene therapy company dedicated to developing groundbreaking treatments for neurodegenerative disorders, has officially announced that the U.S. Food and Drug Administration (FDA) has granted clearance for the Investigational New Drug (IND) application for AVB-101. This clearance allows for the clinical investigation of AVB-101 in individuals with frontotemporal dementia (FTD) who carry mutations in the progranulin (GRN) gene. Additionally, AVB-101 has received Fast Track designation from the FDA for the treatment of FTD-GRN, aimed at slowing disease progression. The Fast Track designation is an expedited process designed to accelerate the development and evaluation of new drugs targeting serious medical conditions with unmet medical needs, with the goal of delivering these potential treatments to patients more swiftly.
AVB-101 has been specifically designed as a potential one-time therapy to halt the progression of FTD by delivering a functional copy of the GRN gene, thus restoring appropriate progranulin levels in the brain regions affected by FTD. Both the U.S. FDA and the European Commission (EC) granted orphan drug designation to AVB-101 for FTD treatment in 2022. The company has recently initiated patient enrollment in European countries for ASPIRE-FTD, an open-label, multi-center dose-escalation study designed to assess the safety and initial effectiveness of AVB-101 in patients with FTD-GRN.
Lisa Deschamps, Chief Executive Officer, expressed, "The clearance of the IND and the Fast Track designation for AVB-101 represent significant milestones for both the FTD community and our organization. Unfortunately, there are currently no approved therapies that can modify the course of frontotemporal dementia in individuals with progranulin mutations. Receiving Fast Track designation underscores the pressing need for treatment options for these patients, and we eagerly anticipate the opening of clinical trial sites in the U.S. to provide an innovative option for eligible FTD-GRN patients."
Dr. David Cooper, Chief Medical Officer, explained the innovative approach, saying, "We are leveraging adeno-associated virus (AAV) technology and adopting a novel method to directly deliver a functional copy of the GRN gene to the brain through bilateral, MRI-guided intrathalamic infusion, with the goal of reinstating normal progranulin protein levels. Our preclinical studies indicate robust distribution to the specific brain regions where progranulin restoration is required, potentially slowing or halting the progression of FTD-GRN, with minimal to no impact on progranulin levels elsewhere in the body, where it could have adverse effects."
FTD is a devastating form of early-onset dementia, typically leading to death within seven to 13 years of symptom onset and three to 10 years from diagnosis. Individuals with FTD commonly experience personality changes, behavioral disturbances, loss of language, apathy, and reduced mobility. FTD is a leading cause of dementia in individuals under the age of 65, with an estimated prevalence of up to 4.6 cases per 1,000 people at any given time. Genetic FTD cases account for about one-third of cases and are most frequently associated with autosomal dominant mutations in three genes, including the progranulin gene.
ASPIRE-FTD is an open-label, multi-center study designed to assess the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In this study, AVB-101 will be administered as a one-time treatment into the thalamus through a stereotactic neurosurgical procedure at specialized neurosurgical centers in Europe and the United States.
AVB-101 is an experimental gene therapy that contains a correct, non-mutated version of the GRN gene. It is designed to restore progranulin levels in the brain, potentially slowing or halting the progression of FTD-GRN. AVB-101 will be administered as a one-time dose directly into the brain through a minimally invasive surgical procedure conducted by a study neurosurgeon at a specialized neurosurgical center.
Source: businesswire.com
