Aquinnah Pharmaceuticals has received $10 million from two pharmaceutical companies, Pfizer and AbbVie, to advance its treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and other neurodegenerative diseases toward clinical trials.
Together with a previous investment from Takeda Pharmaceuticals, Aquinnah’s research and development support totals $15 million.
“We are excited to have the support of three world-class pharmaceutical companies to expand our drug development efforts from ALS into new RNA binding proteins known to modulate Tau, one of the hallmark pathological proteins involved in Alzheimer’s disease,” Glenn Larsen, Aquinnah’s president and chief executive officer, said in a press release.
Many therapeutic approaches for neurodegenerative diseases focus on targeting neurofibrillary tangles and beta-amyloid plaques. Although these are major features of ALS and Alzheimer’s, other elements are common in these diseases.
“There continues to be a substantial need to combat neurodegenerative diseases like Alzheimer’s disease and ALS,” said Scott Brun, Vice president, Scientific Affairs and Head at AbbVie Ventures. “Aquinnah’s research approach … could offer valuable advances to treat these debilitating, progressive brain diseases and complements AbbVie’s ongoing neuroscience research efforts.”
Aquinnah is targeting complex structures of proteins and RNA molecules called stress granules. In healthy people, stress granules are a mechanism of self-repair. But these complex structures can accumulate in tissues and contribute to the development and progression of diseases such as Alzheimer’s.
“We discovered that RNA binding proteins play a critical role in brain deterioration, cognitive loss and life expectancy reduction in mouse models of Alzheimer’s disease. Targeting these pathways are expected to yield new therapies for the treatment of Alzheimer’s disease”, said Ben Wolozin, Aquinnah’s Chief Scientific Officer.
Still in preclinical animal studies, the company’s candidate drugs have shown themselves to be potent, and capable of getting past the blood brain barrier. These characteristics make them promising therapeutic drugs. Aquinnah plans to start human clinical trials in two to three years, first targeting ALS and then Alzheimer’s.
“We believe Aquinnah’s novel approach to ALS and Alzheimer’s disease is one of the most promising developments in neurodegenerative disease today,” said Morris Birnbaum, senior vice president and chief scientific officer of Internal Medicine at Pfizer. “We see the potential for multiple approaches to these diseases based on the company’s work. Neurodegenerative disease remains one of Pfizer’s top priorities and we are committed to supporting Aquinnah’s research.”
Aquinnah was named the Most Innovative Neurodegenerative Research Company in 2016 by New Economy Magazine.