FDA OKs Phase 2 Trial Design for Xanamem
The U.S. Food and Drug Administration (FDA) has given positive feedback to Actinogen’s pre-investigational new drug (pre-IND) submission regarding Xanamem, its experimental oral therapy for fragile X syndrome.
In a written response following a pre-IND meeting with Actinogen, the FDA agreed the data gathered and the proposed trial design would be sufficient to support an IND application, depending on the review of additional documentation.
A pre-IND meeting helps create a strategy for the therapy’s development process by reviewing the clinical trial design, sharing ideas, and understanding the specific expectations and requirements for a future IND application.
“The FDA’s positive Pre-IND advice for FXS marks a significant milestone in the clinical development of our 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,” Actinogen CEO Steven Gourlay, MD, said in a press release.
Actinogen plans to submit an IND for a Phase 2 trial — to be called XanaFX and to take place in Australia — that will test Xanamem in adolescents with fragile X by the third quarter of this year. The trial’s start date is scheduled for the end of the year.
The 12-week trial will assign up to 40 boys with fragile X, ages 12–18, randomly to one of two different doses of Xanamem or a placebo. The goal is to assess the safety and efficacy of Xanamem. Effectiveness will be measured by changes in levels of anxiety, sleep patterns, and behavioral issues.
According to Actinogen, approval of its IND by the FDA, despite not being mandatory for conducting the trial in Australia, makes sure it follows all the agency’s requirements and is in agreement with international regulatory standards.
“There are no currently approved therapies for FXS. The Company is advancing the planning for its Phase II XanaFX trial which is expected to commence by the end of the year. Actinogen is now positioned to progress its clinical programs with multiple Phase II trials and is well funded to advance the development pipeline,” Gourlay said.
Xanamem works by blocking the cortisol-activating enzyme 11beta-HSD1, which is found at high levels in brain regions associated with cognition and memory.
Elevated cortisol can cause profound alterations to the brain that affect cognition and behavior. By blocking 11beta-HSD1, Xanamem is expected to inhibit cortisol production.