AC Immune has secured Fast Track designation and U.S. FDA clearance of its Investigational New Drug (IND) application for ACI-7104, its experimental anti-alpha-synuclein immunotherapy for early-stage Parkinson’s disease.

 

The regulatory milestone strengthens the company’s bid to develop ACI-7104 as a potential treatment for Parkinson’s, a disease with a major unmet medical need.

 

ACI-7104 is designed to trigger antibodies that selectively target aggregated alpha-synuclein, a protein associated with neuronal damage. The candidate is already being tested in the Phase 2 VacSYn trial, an adaptive, biomarker-based study involving patients with early-stage Parkinson’s disease.

 

Final Part 1 results at 100 weeks remain on track for release in the second half of the year.

 

“Fast Track designation and IND clearance from the FDA are important achievements for ACI-7104, one of our wholly-owned programs, given its potential to address the significant unmet need in Parkinson’s. We look forward to working closely with the FDA as we advance ACI-7104 through its ongoing clinical development towards potentially delivering a much-needed treatment option for patients,” said Martin Zügel, interim CEO of AC Immune.

 

The FDA’s Fast Track decision follows interim Phase 2 data showing that ACI-7104 was safe and well tolerated at 76 weeks, with no clinically relevant safety issues reported. The trial also met its immunogenicity target, with a 100% responder rate.

 

The company said the interim findings provide early signals that targeting underlying alpha-synuclein pathology through active immunotherapy could potentially affect the course of Parkinson’s disease.

 

AC Immune has also secured a $4 million research grant from the Vijay and Marie Goradia Charitable Foundation to extend Part 1 of the VacSYn trial. The funding will support an additional two years of follow-up to assess the long-term safety and efficacy of ACI-7104.

 

“ACI-7104 represents a promising active immunotherapy designed to target pathological a-syn for the treatment of prodromal and early PD. The FDA’s decision signifies the growing importance of the program including the positive interim Phase 2 findings. This is a key regulatory milestone for the program, and we look forward to delivering the full results from Part 1 of the VacSYn trial in the coming months,” said Günther Staffler, Executive Vice President, Development at AC Immune.

 

ACI-7104 is an optimized version of AC Immune’s earlier anti-alpha-synuclein active immunotherapy. The approach is designed to generate an antibody response against pathological oligomeric alpha-synuclein, with the goal of limiting its spread and downstream neurodegeneration.