Using pioneering science to strengthen our multi-franchise portfolio, we have made significant progress in expanding our extensive research pipeline. Our pursuit of multiple modalities may have the ability to slow or halt the progression of neurological diseases.
In our core growth area of Alzheimer’s disease and dementia, we plan to file for U.S. Food and Drug Administration approval of aducanumab, an investigational treatment for early Alzheimer’s disease that we are developing in collaboration with Eisai Co., Ltd. This decision was based on a new analysis of a larger dataset from the Phase 3 EMERGE and ENGAGE Phase 3 studies that had been discontinued in March 2019 following a prespecified futility analysis. One of the Phase 3 studies, EMERGE, met its prespecified primary and secondary endpoints by showing a significant reduction in clinical decline. We believe that results from a subset of patients from ENGAGE who received sufficient exposure to high dose aducanumab support the findings of from EMERGE, though ENGAGE did not meet its primary endpoint. If approved, aducanumab would be the first therapy to reduce clinical decline in the early stages of Alzheimer’s disease.
We also exercised our option with Ionis Pharmaceuticals, Inc. and obtained a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB080, a tau-targeting antisense investigational treatment in early Alzheimer’s disease. This investigational drug is designed to cut down on the production of tau proteins, formally known as MAPT (microtubule-associated protein tau), in the central nervous system. These proteins can form tangles in the brain and are thought to cause neurodegenerative diseases, like Alzheimer’s, and some other types of dementia. This investigational treatment expands our broad Alzheimer's disease portfolio and pipeline, which includes two studies of anti-tau antibodies (Phase 1 and 2) in addition to BIIB080, BIIB076 and gosuranemab (BIIB092).
Our success in neurology gives our researchers insights into other diseases with shared commonalities, such as immunology. In our investigations of therapies to potentially treat lupus, our Phase 2 LILAC study results showed that BIIB059, a monoclonal antibody, demonstrated a statistically significant reduction of disease activity in people with cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE) compared to those who received placebo. We are planning to advance BIIB059 to Phase 3.
Also in 2019 we completed several strategic acquisitions and partnerships to bolster our pipeline:
- In June, we acquired Nightstar Therapeutics plc, a clinical-stage gene therapy company focused on adeno-associated virus (AAV) treatments for inherited retinal disorders. As a result, we added two mid- to late-stage clinical assets, BIIB111 (timrepigene emparvovec) in choroideremia and BIIB112 (RPGR gene therapy) in X-linked retinitis pigmentosa, along with preclinical programs in other inherited retinal disorders (Stargardt’s disease, Best disease and Retinitis Pigmentosa).
- In November, we completed enrollment in the global Phase 3 STAR study, which is evaluating BIIB111 for the potential treatment of choroideremia, a rare, degenerative, X-linked inherited retinal disorder that leads to blindness and currently has no approved treatments. Learn more about our expanded focus in Ophthalmology here.
- In December, we licensed an anti-C3 protease from Catalyst Biosciences, a compound in preclinical development for the potential treatment of geographic atrophy.
- Our continued collaborations with C4 Therapeutics (C4T) and Skyhawk Therapeutics, Inc. are showing great progress. Our strategic collaboration with C4T is focused on the use of its novel protein degradation platform for the discovery and development of potential new treatments for neurological conditions such as Alzheimer’s disease and Parkinson’s disease. Through our agreement with Skyhawk Therapeutics, the companies are leveraging Skyhawk Therapeutics’ SkySTAR technology platform with the goal of discovering innovative small molecule treatments for patients with neurological diseases, including MS and SMA.
We will continue to pursue collaboration in 2020 and beyond that reinforce our leadership and strengthen our pipeline. In February 2020, Biogen and Sangamo Therapeutics announced a global collaboration to develop gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurological diseases.
Making an impact beyond our portfolio
At Biogen, we are committed to understanding and addressing the issue of health disparities and inequity in the disease areas we treat. We support underserved, vulnerable and minority populations both now and in the future. We are proud to collaborate with the n-Lorem Foundation, a non-profit focused on creating individual treatments for patients with ultra-rare diseases caused by genetic mutations that affect approximately 1 to 10 patients in the world.
Learn more about our collaboration with n-Lorem Foundation.