The U.S. Food and Drug Administration (FDA) has responded to Affiris‘ pre-investigational new drug (IND) submission regarding a planned Phase 2 clinical trial of the potential Parkinson’s vaccine Affitope (PD01A).
After reviewing previous preclinical and clinical data, the FDA answered questions posed by the company and provided guidance related to designing the upcoming trial.
Based on that response, Affiris intends to initiate the Phase 2 study in the U.S. and Europe in the second half of this year.
In the brains of people with Parkinson’s disease, the protein alpha-synuclein forms into spherical clumps called Lewy Bodies, which are toxic to neurons. It is widely believed that therapies that reduce this accumulation of alpha-synuclein could be beneficial for Parkinson’s.
Affitope is designed to induce the body’s immune system to make antibodies — molecules that recognize specific targets — against alpha-synuclein. By encouraging one’s body to develop its own defenses against molecules that contribute to Parkinson’s, Affitope works like a vaccine against the disease.
In theory, this could cause the immune system to reduce alpha-synuclein, without the need for repeated administration of medication.
The experimental therapy was previously tested in a series of Phase 1 clinical trials in patients with Parkinson’s disease (NCT01568099, NCT01885494, NCT02618941, NCT02758730, and NCT02216188). Results showed Affitope was well-tolerated, with the only reported treatment-related adverse side effects being mild injection site reactions. Lab tests also showed that the vaccine did trigger the production of antibodies against alpha-synuclein.
Those early trials were not designed to detect a change in Parkinson’s symptoms.
Details of the planned Phase 2 trial. trial design and objectives have not yet been released by Affiris, but in general, Phase 1 trials are designed primarily to evaluate safety, whereas Phase 2 trials are more concerned with finding appropriate dosages and obtaining preliminary data on effectiveness.
“We are pleased to have completed the pre-IND process, with the FDA providing helpful guidance for the continued advancement of Affitope PD01 for the treatment of Parkinson’s disease, an indication with an urgent and significant unmet medical need,” Noel Barrett, PhD, CEO at Affiris, said in a press release. “We appreciate the FDA’s feedback as we endeavour to provide patients with a potentially disease-modifying immunotherapy utilizing our proprietary patented Affitome technology.”