dm+d

Unassigned

New Medicines

Frontotemporal dementia, caused by mutations in the granulin (GRN) gene  

Information

New molecular entity
Prevail Therapeutics
Prevail Therapeutics

Development and Regulatory status

None
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Nov 20Granted orphan drug status in the EU [4].
Mar 20FDA grants fast track status to PR 006 for FTD-GRN [4].
Dec 19FDA grants orphan drug designation to PR 006 for the treatment of patients with FTD-GRN [4].

Category

A single dose in vivo gene therapy using an adeno-associated (AAV9) viral vector, PR 006 is designed to deliver healthy GRN gene to compensate deficiency of progranulin, a glycoprotein and demonstrates ability to slow or stop progression of FTD-GRN.
FTD is much less common than Alzheimers disease and vascular dementia. Figures vary, but it probably accounts for less than 5% of all dementia. The incidence of FTD is estimated to be 1.61 to 4.1 cases per 100,000 people annually [1]. GRN-FTD represents about 5% of all FTD, and 20% of FTD in which the family history is positive. It is inherited in an autosomal dominant manner [2].
Frontotemporal dementia, caused by mutations in the granulin (GRN) gene  
Intracisternal
Parenteral

Trial or other data

Mar 22PI/II PROCLAIM study is recruiting [3].
Nov 20PI/II PROCLAIM ascending dose trial to evaluate the safety and effects on progranulin levels of PR 006A in patients with fronto-temporal dementia with progranulin mutations (FTD-GRN)starts (NCT04408625; PRV-FTD101). The open-label, non randomised trial intends to enrol 15 participants aged 30 to 80 years in the US, Australia and Spain. Collection of primary outcome data is due to complete Sep 27 [3].