AC Immune’s phosphorylated Tau (pTau)-targeting vaccine has secured a fast track designation from the US Food and Drug Administration (FDA) to treat Alzheimer’s disease.
The JNJ-2056 vaccine, previously named ACI-35.030, is being investigated in the Phase IIb ReTain clinical trial. As per AC Immune, the trial is recruiting participants with preclinical Alzheimer’s disease, where patients have yet to show clinical symptoms. According to the company, this is the first active immunotherapy tested in a preclinical Alzheimer’s population.
JNJ-2056 is designed to reduce the spread of a protein called tau, which clumps together and tangles up in the brains of patients with Alzheimer’s. The candidate has shown promising efficacy in a Phase Ib/IIa trial (NCT04445831), where it was able to activate patients’ immune systems, producing a strong antibody response against both pTau and its harmful aggregated form.
The investigational vaccine is being developed as part of a global license, development, and commercialisation agreement with Johnson & Johnson Innovation Medicine (J&J). Back in 2022, the two companies teamed up to advance the development of the vaccine.
Another one of AC Immune’s vaccine candidates ACI-24.060 was swept up by Japanese pharma company Takeda in May 2024. Takeda signed an exclusive, worldwide option and licence agreement for the amyloid beta-targeting Alzheimer’s disease immunotherapy, in a deal worth up to $2.2bn.
The Alzheimer’s disease treatment space is currently dominated by monoclonal antibodies (mAbs) like Eli Lily’s Kisunla (donanemab), which finally got an FDA approval earlier this month following a series of setbacks. The leading drug in the space is Biogen’s Leqembi (lecanemab), which is expected to generate $6.1bn in 2030, according to GlobalData consensus forecasts. However, experts have said that if Alzheimer’s vaccines were proven effective, they could be cheaper, safer, and more convenient than mAbs.
GlobalData is the parent company of Pharmaceutical Technology.
A spotlight will be on Alzheimer’s disease research in a few days, with the start of the Alzheimer's Association International Conference (AAIC) 2024 in Philadelphia, US taking place 28 July – 1 August. The Swiss biotech will hold three presentations at the conference, including one on ACI-24.060.
In the announcement accompanying the fast track designation, AC Immune’s CEO Andrea Pfeifer said: “We believe this modality has the potential to offer therapeutic advantages, as well as benefits in terms of convenience and access. Fast Track designation offers opportunities for more efficient development and regulatory review.”
