Takeda Pharmaceuticals is expanding its presence in the neurodegenerative diseases space – investing $5 million in Cambridge startup Aquinnah Pharmaceuticals to expand research on a small molecule drug for amyotrophic lateral sclerosis, or ALS.
At present, there are no effective drugs on the market to treat or slow the progression of ALS – but Aquinnah wagers that it has designed compounds that could slow or even reverse the progression of the disease.
There are still many questions on what causes the nerve cells of patients with ALS to slowly die off – but one potential cause is the buildup of abnormally formed proteins.
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Aquinnah’s approach targets a protein called TDP-43 that has been found to accumulate in patients with neurodegenerative conditions like ALS, Alzheimer’s disease and certain dementias. The Aquinnah platform works by breaking down these protein complex buildups.
The science is based on the work of Dr. Benjamin Wolozin, a Boston University researcher whose primary interests lie in Alzheimer’s disease and Parkinson’s disease.
Aquinnah has also received funding from the Massachusetts Life Sciences Center, the ALS Association and the National Institute of Neurological Disorders and Stroke.
Notably, Takeda recently announced a research partnership with University College London to study gene identification and signaling pathways that modify neurodegenerative diseases like Motor Neuron Disease and ALS. It also has an experimental Alzheimer’s disease drug in Phase 3 trials, partnering with Zinfandel Pharmaceuticals.
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“We believe that Aquinnah’s program is among the most promising new approaches to treating neurodegenerative diseases,” Dr. Ceri Davies, head of central nervous system drug discovery at Takeda, said in a statement.
[Image courtesy of Flickr user LeeAnder]
