November 25, 2020
Specialist development pharmaceutical company AMZELL B.V has concluded discussions with the FDA ahead of initiating a Phase 3 trial of its new treatment for infantile epileptic disease, AMZ002.
The Phase 3 trial will compare AMZ002 to another active treatment in a superiority study, as a placebo-controlled trial would not be ethical for this patient population. AMZ002 is a sterile injectable hormone and would offer a purified synthetic alternative to an existing FDA-approved treatment for epileptic seizures.
Infantile epileptic disease can cause global neurodevelopmental delay, significant intellectual disability, and currently has limited treatment options and a poor prognosis. In most cases, the initial age of onset is between 3 and 12 months of age, and over 90% of cases begin before 12 months of age. Infantile epileptic disease is an orphan indication, affecting fewer than 200,000 patients in the United States. The lack of treatment options for the condition means that there is a considerable unmet need.
Dario N. Carrara PhD, Chief Scientific Officer commented: “We are delighted to have had such positive feedback from the FDA on our proposed Phase 3 trial of AMZ002, and hope to initiate the trial in the coming months. Our purified, synthetic injectable should provide a treatment option for epileptic seizures, suited to best meet the needs of this highly vulnerable patient population.”
Following the Type B End of Phase 2 meeting with the FDA Division of Neurology for AMZ002, AMZELL has agreed the general study design, dosing, primary and secondary endpoints and the estimative sample size for the Phase 3 trial, and has now submitted the protocol utilizing a Special Protocol Agreement.
Further details on this Phase 3 trial will be confirmed following the FDA’s approval of AMZELL’s proposed protocol.
AMZ002 is the latest product to reach Phase 3 from AMZELL, which specialises in developing well-characterized active substances through to proof-of-concept or registration for sale to commercial partners. AMZELL’s innovative platform drug delivery technologies provide effective drug delivery, increased efficacy, and improved safety and compliance.
AMZELL B.V is a specialist, virtual development pharmaceutical company which takes candidate or repurposed drugs and devices either through to proof of concept, or through to registration for sale to commercial healthcare companies.
AMZELL B.V has particular interest in developing well-characterized active substances utilizing innovative platform drug delivery technologies, with the aim to provide more effective drug delivery, increased efficacy, and improved safety and compliance.