- Actinogen Medical (ACW) receives FDA support for its pre-investigational drug (pre-IND) application for its Fragile X Syndrome (FXS) program
- The clinical study will test ACW’s Xanamem lead molecule in treating FXS — an inherited disease that causes developmental and intellectual disabilities
- The FDA says ACW’s proposed data package and trial design are sufficient, subject to a final review of full documentation, to be submitted in Q3 this year
- The parties have also agreed on an adolescent patient population to undergo observation in ACW’s phase two XanaFX study in Australia later this year
- Shares are trading 10.3 per cent higher at 16 cents apiece at 1:36 pm AEST
Actinogen Medical (ACW) has received written support from the US Food and Drug Administration (FDA) in response to the company’s pre-investigational new drug (pre-IND) application for its Fragile X Syndrome (FXS) program.
FXS is an inherited genetic disease that causes developmental and intellectual disabilities and, presently, has no approved therapies.
The positive advice follows a pre-IND meeting with the FDA to discuss the use of Actinogen’s Xanamem lead molecule in the context of the FXS clinical study.
The FDA has determined that ACW’s proposed data package and trial design are sufficient, subject to a final review of complete documentation, which the company plans to submit in the third quarter of 2021.
Further, the two parties have agreed on an adolescent patient population to be observed in Actinogen’s phase two XanaFX study.
This will comprise a randomised, placebo-controlled, double-blind, 12-week trial to investigate the safety and efficacy of Xanamem in male adolescents who have FXS.
Actinogen Medical CEO Steven Gourlay said receiving the FDA’s support was a milestone in the development of its second disease program.
“It provides greater confidence that we will obtain support from the FDA for the investigation of Xanamem in this disease that has a high unmet medical need for effective therapies,” Dr Gourlay said.
“Actinogen is now positioned to progress its clinical programs with multiple Phase two trials and is well-funded to advance the development pipeline.”
The phase two study will commence in Australia during the fourth quarter of calendar year 2021.
Interestingly, US IND approval is not required to conduct the trials in Australia. However, the biotechnology developer said it would ensure the studies incorporated all FDA requirements to an international regulatory standard.
Shares in Actinogen Medical were trading 10.3 per cent higher at 16 cents apiece at 1:36 pm AEST.