US-based drug development firm Ionis Pharmaceuticals has launched a Phase I/IIa clinical trial of IONIS-MAPT to treat patients having mild Alzheimer's disease (AD).
IONIS-MAPT is an antisense drug being developed to minimise the production of all forms of microtubule-associated protein tau (MAPT) in the brain.
The randomised, placebo-controlled, dose escalation Phase I/IIa trial will assess the safety and activity of the investigational drug in around 44 subjects over a period of three months.
Patients with mild AD will be injected with once-monthly intrathecal IONIS-MAPT directly into the cerebral spinal fluid.
Ionis Pharmaceuticals chief operating officer Lynne Parshall said: "Targeting MAPT represents a unique opportunity to address the unmet need in both rare and more prevalent tauopathies.
“The first clinical study with IONIS-MAPT is in patients with AD, however, in parallel, we also plan to develop IONIS-MAPT for patients with FTD, a rare disease population.”
The trial is part of a broad strategic collaboration under which Ionis’ antisense technology will be combined with Biogen’s neurology expertise to develop new therapies for neurological conditions.
While Biogen holds an option for the development and commercialisation of IONIS-MAPT, the firm has made a $10m milestone payment to Ionis, associated with the initiation of the Phase I/IIa trial.
Under the collaboration, the firms have developed Spinraza drug for spinal muscular atrophy (SMA), and are additionally developing IONIS-SOD1 to treat amyotrophic lateral sclerosis (ALS), along with IONIS-BIIB5, IONIS-BIIB6, and IONIS-BIIB7 for undisclosed neurodegenerative disorders.