FDA Grants Fast Track to AC Immune's JNJ-2056 for Alzheimer's

AC Immune SA, a clinical-stage biopharmaceutical company specializing in precision medicine for neurodegenerative diseases, announced that its active-immunotherapy candidate, ACI-35.030 (now named JNJ-2056), has received Fast Track designation from the U.S. Food and Drug Administration (FDA). This candidate targets the pathological form of the Tau protein, specifically phosphorylated Tau (pTau), which is linked to the progression of Alzheimer's disease (AD). The Phase 2b clinical trial, known as ReTain, is actively recruiting participants and is fully funded by Janssen Pharmaceuticals, Inc., a subsidiary of Johnson & Johnson.

Dr. Andrea Pfeifer, CEO of AC Immune, emphasized the significance of the Fast Track designation, highlighting the potential therapeutic advantages and increased accessibility of their anti-pTau active immunotherapy. She noted that this is the first time active immunotherapy is being tested in a preclinical AD population, aiming to prevent the onset of cognitive decline by targeting pTau. The Phase 1b/2a clinical trials demonstrated that ACI-35.030 specifically targets toxic forms of Tau while sparing normal protein forms. Dr. Pfeifer underscored the importance of this innovation in the treatment and potential prevention of Alzheimer's disease, aiming to develop new, safe, and user-friendly therapies.

JNJ-2056 is being developed under a global license, development, and commercialization agreement with Janssen Pharmaceuticals. The ReTain trial is a potentially registration-enabling study, designed to assess the efficacy of active immunization with JNJ-2056 in delaying or preventing cognitive impairment in individuals with preclinical AD. This multicenter, double-blind, placebo-controlled study will include approximately 500 participants, randomized to receive either JNJ-2056 or a placebo through intramuscular injections over a period of up to four years.

The primary endpoint of the ReTain trial is to measure cognitive decline using the Preclinical AD Cognitive Composite 5 (PACC-5) score. The key secondary endpoint will evaluate the effect of JNJ-2056 on the propagation and accumulation of Tau pathology, using Tau PET imaging to compare with the placebo group.

JNJ-2056, derived from AC Immune’s SupraAntigen® platform, has shown promise in clinical studies by inducing a robust polyclonal antibody response against pTau. This investigational candidate aims to reduce the spread of pathological Tau in the early stages of AD, potentially preventing disease progression.

AC Immune SA, headquartered in Lausanne, Switzerland, is a global leader in precision medicine for neurodegenerative diseases such as Alzheimer’s and Parkinson’s disease. The company utilizes two clinically validated technology platforms, SupraAntigen® and Morphomer®, which support a diverse pipeline of therapeutic and diagnostic programs. Currently, AC Immune is conducting five Phase 2 clinical trials and one Phase 3 trial. The company has a history of forming strategic partnerships with leading pharmaceutical companies, securing substantial non-dilutive funding through potential milestone payments and royalties.