AC Immune’s publication on the treatment of Down Syndrome

Patients with Down syndrome are often confronted with cognitive dysfunction right from childhood onwards and are predisposed to Alzheimer’s disease-like characteristics. The manuscript, An anti-Abeta-Amyloid Vaccine for Treating Cognitive Deficits in a Mouse Model of Down Syndrome, was published in PLOS ONE, a respected peer reviewed journal recognized for its high ethical standards and rigorous scientific approach.

Prof. Andrea Pfeifer, CEO of AC Immune, said: “This pioneering publication shows encouraging data on brain Abeta reduction and memory enhancement of our anti-Abeta vaccine in a pre-clinical model for Down syndrome. This research is the scientific basis for the first-ever clinical trial of an anti-Abeta vaccine in Down syndrome. People with Down syndrome are a genetically predisposed population showing early onset Alzheimer’s disease-like characteristics. Another such population is the extended family in Colombia, in which the antibody crenezumab is being evaluated in the world’s first preventive clinical trial for Alzheimer’s.”

The company also announced the start of patient recruitment for the first clinical trial for this anti-Abeta vaccine (ACI-24) targeting Alzheimer’s disease-like characteristics in those with Down syndrome. Announced earlier this year and being conducted in collaboration with the University of California San Diego (UC San Diego) Down Syndrome Research and Treatment Center, the phase 1b trial is expected to include 24 patients with a 12 month treatment period followed by a 12 month follow up.

Dr. Andreas Muhs, Chief Scientific Officer of AC Immune and corresponding author of the paper, observed: “Our anti-Abeta vaccine is a liposomal therapeutic vaccine that was discovered from our SupraAntigenTM technology platform. The vaccine stimulates the immune system to produce a non-inflammatory antibody response highly specific to misfolded Abeta. In this publication we prove it is potent, safe and effective in an established mouse model of Down syndrome. The promising data suggest the significant potential of the vaccine and are the basis for why we are running this novel clinical study in this specific patient population.”

Dr. William Mobley, Executive Director of the UC San Diego Down Syndrome Research and Treatment Center, and co-author of the paper, commented: “We see this work as both ground-breaking and as the next step in testing the idea that it will be possible to treat and eventually prevent Alzheimer’s disease in people with Down syndrome. The findings from the publication suggest vaccination is a good strategy to combat the effects of the elevated Abeta level in adults with Down syndrome. We are delighted to participate in a public-private partnership with AC Immune, the Lumind LuMind Research Down Syndrome Foundation and the NIH to test the effect of an anti-Abeta vaccination.”

About AC Immune
AC Immune is a leading Swiss-based biopharmaceutical company focused on neurodegenerative diseases with three products in clinical trials. The Company designs, discovers and develops therapeutic and diagnostic products to prevent and modify diseases caused by misfolding proteins. AC Immune’s two proprietary technology platforms create antibodies, small molecules and vaccines to address large markets across a broad spectrum of neurodegenerative indications. Alzheimer’s disease (AD) is the largest indication addressed by its products but the company’s innovative, highly differentiated and disease-modifying therapies are designed to shift the paradigm in the treatment of other neurodegenerative diseases such as Parkinson’s, Down syndrome, and glaucoma. The Company has a large, diversified and promising pipeline featuring seven therapeutic and three diagnostic products. The most advanced of these is crenezumab, an anti-Abeta antibody in phase 3 that is licensed to Genentech. Crenezumab was chosen by the US National Institute of Health for use in the first-ever AD prevention trial. The company has partnered three programs targeting Tau: ACI-35 with Janssen (therapeutic vaccine, phase 1b), Tau-PET imaging agent with Piramal (Alzheimer’s diagnostic agent) and anti-Tau-antibodies with Genentech (preclinical). The anti-Abeta vaccine ACI-24 phase 1/2a in AD and phase 1b in DS trials are run in house.

(Press release)