AC Immune Receives Second Milestone Payment for Phase 2b ACI-35.030 Alzheimer's Trial Progress

AC Immune SA, a biopharmaceutical company based in Lausanne, Switzerland, announced on September 17, 2024, that it has received a milestone payment of CHF 24.6 million under its agreement with Janssen Pharmaceuticals, Inc., a Johnson & Johnson company. This payment is related to the Phase 2b ReTain trial of their active-immunotherapy candidate ACI-35.030, now called JNJ-2056, aimed at treating preclinical Alzheimer's disease (AD).

The milestone was achieved due to the rapid prescreening rate in the potentially registrational trial. Combined with a previous payment made in December, AC Immune has now received a total of CHF 40 million for ACI-35.030 related to this study.

Dr. Andrea Pfeifer, CEO of AC Immune, emphasized the importance of this milestone, noting that it reflects the trust the medical community places in new therapeutic approaches for early-diagnosed Alzheimer’s disease. The company aims to combat neurodegeneration through early diagnosis and treatment, specifically targeting the pathological Tau protein (pTau).

Additionally, this payment confirms the effectiveness of AC Immune’s technology platforms and drug development capabilities. To date, the company has secured approximately CHF 425 million from various collaboration deals, with potential future milestones exceeding CHF 4.3 billion, plus royalties. This strong financial position ensures operational funding for the next three years, during which several significant milestones are expected.

JNJ-2056 has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in July, highlighting its potential value for patients. This is the second AC Immune’s active immunotherapy to receive such recognition, following ACI-24.060, which targets Abeta. Furthermore, the company's PI-2620 Tau-PET diagnostic, currently in Phase 3, also received Fast Track designation in August.

The Phase 1b/2a clinical trials of ACI-35.030 have demonstrated the candidate’s ability to induce an antibody response targeting pTau without affecting normal forms of Tau. The ReTain trial has garnered significant interest, with prescreening rates surpassing expectations.

Dr. Pfeifer acknowledged the importance of the Phase 2b ReTain trial as it is the first to test an active immunotherapy in a preclinical AD population. Active immunotherapies like ACI-35.030 potentially offer therapeutic benefits coupled with better convenience and accessibility. The recent Fast Track designation underscores its potential value for patients.

The Phase 2b ReTain trial is a randomized, multicenter, double-blind, placebo-controlled study involving participants with preclinical AD to assess the clinical effects of immunization with JNJ-2056. The study aims to demonstrate that JNJ-2056 can delay or prevent cognitive impairment or other symptoms in individuals with preclinical AD by inhibiting the spread of pathological Tau. Approximately 500 participants will be enrolled, with a 1:1 randomization for either JNJ-2056 or placebo, administered as intramuscular injections over four years. The primary endpoint will measure cognitive decline using the PACC-5 score, and a secondary endpoint will analyze the effect of JNJ-2056 on Tau pathology through Tau PET imaging. This trial is fully funded and conducted by Janssen.

ACI-35.030, derived from AC Immune’s SupraAntigen® platform, has shown promise in clinical studies by inducing strong polyclonal antibody responses against pathological Tau forms. This investigational candidate aims to reduce Tau spreading in AD’s early stages, potentially slowing or preventing disease progression.

AC Immune SA is a leader in precision prevention for neurodegenerative diseases. Its technology platforms, SupraAntigen® and Morphomer®, support a broad pipeline of therapeutic and diagnostic programs, including multiple Phase 2 and Phase 3 developments. The company has a history of forming strategic partnerships, securing over $4.5 billion in potential milestone payments plus royalties.