Neurodegenerative disease specialist InBrain Pharma has received a positive opinion from the European Medicines Agency (EMA) on its plans to conduct a Phase III trial on A-dopamine.
The 100mg/ml dopamine hydrochloride solution, which is administered via the intracerebroventricular route for the management of patients with advanced-stage Parkinson's disease, will now be tested in a single pivotal Phase III trial.
Named DEEP-DIVE, the trial has been found to meet current EMA requirements for the submission of an MA application for a new anti-Parkinsonian drug under the centralised European procedure.
The Committee for Medicinal Products for Human Use (CHMP) validated the overall clinical and regulatory strategy, including the conduct of a single pivotal trial for submission of a centralised marketing authorisation application at the European level.
This randomized, double-blind, placebo-controlled, parallel-group trial will enroll 170 patients and features an adaptive design, allowing for sample size adjustments if necessary.
Patients in the placebo arm will receive a brain infusion of saline solution on top of an optimised oral anti-Parkinsonian treatment based on L-dopa or a L-dopa derivative, for a maximum of 24 weeks — ensuring ethical acceptability.
At the end of the double-blind evaluation phase, all patients, including those in the placebo group, will enter a 52-week open-label extension phase during which they will receive A-dopamine.
“This positive feedback from EMA marks a major regulatory and strategic milestone for InBrain Pharma,” said Dr. Véronique Foutel, CEO of InBrain Pharma.
“It validates the preclinical and clinical programmes carried out to date and officially brings the program into the late stage of clinical development.”
“Our therapy is not only designed to relieve symptoms, but to fundamentally personalise dopamine supplementation for every patient,” commented Professor Caroline Moreau, co-founder and medical advisor at InBrain Pharma.
“This approach paves the way for precision medicine in neurology – delivering treatment directly to the right brain location, at the right dose. With the EMA’s green light, we are one step closer to our goal: delivering this therapeutic breakthrough to patients with advanced Parkinson’s disease, for whom current options are no longer satisfactory,” added Professor David Devos, co-founder and scientific advisor at InBrain Pharma.
The phase III pivotal trial is planned to take place in both Europe and the United-States.
