Alzheimer’s disease can be treated with antibody drugs that reduce levels of amyloid beta protein that build up in the brain. Clinical-stage biotech company AC Immune is developing a novel therapy that sparks the immune system to clear away amyloid plaque, and Takeda Pharmaceutical sees enough promise in this approach to commit $100 million for the opportunity to pick up this experimental therapy.
The AC Immune drug, code-named ACI-24.060, is currently being evaluated in the Phase 2 portion of a Phase 1b/2 study. Under the deal terms announced Monday, Takeda’s upfront payment gives it an exclusive option to license global rights to this immunotherapy.
ACI-24.060 is an immunogenic peptide that is presented to the immune system’s B cells in a way that mimics the pathological form of amyloid beta. This presentation is intended to spark activity from antibodies that bind to pathological forms of the protein. In preclinical research, Lausanne, Switzerland-based AC Immune has reported that the drug led to higher blood levels of immunoglobulin G antibodies that were associated with reduction in amyloid plaque. In monkey tests, AC Immune has reported a favorable safety profile for the drug.
AC Immune is responsible for completing the placebo-controlled Phase 1b/2 study, which is evaluating for ACI-24.060 in patients with early Alzheimer’s. The study is also testing the immunotherapy as a treatment for patients with Down’s syndrome. Under the Takeda agreement, the Japanese pharmaceutical giant may exercise its option after it receives predefined clinical data on the first three Alzheimer’s patient cohorts from the ongoing study, AC Immune said in a regulatory filing. The first Phase 2 data on amyloid plaque reduction will be reported in the current quarter, potentially paving the way for Phase 3 testing, AC Immune said Monday in its report of financial results for the first quarter of 2024.
“We believe the maximum impact of ACI-24.060 can best be realized by partnering with Takeda at this critical juncture in its development, which will help us move rapidly into Phase 3,” AC Immune CEO Andrea Pfeifer said in a prepared statement. “This agreement allows us to leverage the developmental expertise, strategic vision and financial capacity of an accomplished organization that has demonstrated its ability to execute the type of comprehensive global program required for Phase 3 trials in Alzheimer’s disease while allowing us to focus on completing Phase 1b/2 development and accelerating our efforts to replicate this success with enhanced funding for our early-stage pipeline.”
The agreement makes AC Immune eligible for up to $2.1 billion in milestone payments, which includes an option exercise fee in the low-to-mid-hundred millions of dollars, according to the regulatory filing . After exercising that option, Takeda will be responsible for clinical development of the Alzheimer’s drug. Takeda will also handle regulatory submissions as well as commercialization if the drug is approved.
ACI-24.060 would give Takeda another opportunity to address Alzheimer’s, but with a drug candidate that has been de-risked with clinical data. The company previously partnered with Denali Therapeutics on the development of an antibody engineered to penetrate the brain to hit and activate a target called TREM2. Last August, the two companies announced they would discontinue clinical development of this Alzheimer’s drug after safety signals emerged in Phase 1 testing. Takeda also has a research collaboration with Cure Network Dolby Acceleration Partners focused on developing small molecules that target tau in Alzheimer’s and other brain disorders.
AC Immune has other alliances in Alzheimer’s. Its anti-tau immunotherapy ACI-35.030 is currently in mid-stage clinical development under a partnership with Johnson & Johnson subsidiary Janssen Pharmaceuticals. A tau aggregation inhibitor is in preclinical development under a partnership with Eli Lilly. Two AC Immune antibodies, semorinemab and crenezumab, were partnered with Roche’s Genentech division. But after disappointing clinical trial results, Genentech terminated the alliance this year, returning to AC Immune rights to both antibodies.
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