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Advancements in Aquinnah Pharmaceuticals' Oral Small Molecule Program for Alzheimer's and Tauopathy Diseases

Monday, October 30, 2023

Aquinnah Pharmaceuticals has revealed promising preclinical research findings for a novel therapy designed to slow or halt the progression of Alzheimer's disease and related neurodegenerative disorders. The company, known for its expertise in pharmaceutical approaches targeting stress granule biology, has developed a lead compound that specifically targets the interaction between tau and stress granules. This compound was found to reduce approximately 70% of tau pathology in advanced-stage Alzheimer's animal models, as measured by three distinct markers of pathological tau. This achievement is significant, given that these markers tend to increase as the disease progresses. Notably, Aquinnah's compound can be administered orally as a pill, which is generally preferred by patients over the injection-based delivery methods currently used for approved Alzheimer's immunotherapies.

In preclinical models, a once-daily administration of the compound demonstrated a robust reduction in abnormal tau protein, a key contributor to memory and cognition loss in Alzheimer's disease. What makes Aquinnah's approach unique is its potential compatibility with anti-amyloid drugs recently approved by the FDA. If successful in clinical trials, this treatment could offer Alzheimer's patients substantial benefits, including reduced brain damage, memory restoration, improved brain function, and a slower disease progression.

Alzheimer's disease (CTAD) conference in Boston under the title "Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology." Dr. Wolozin highlighted the growing global impact of Alzheimer's disease and the potential of targeting tau pathology, either alone or in combination with anti-amyloid therapies, to significantly enhance cognitive function.

Aquinnah's success in advanced-stage animal models, equivalent to late-stage Alzheimer's patients, suggests that their compound may have the capacity to reverse cognitive decline and dementia caused by Alzheimer's disease, not merely delaying its progression.

Aside from its potential application in Alzheimer's disease, Aquinnah's therapeutic shows promise in treating other tauopathies, such as Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Lobar Dementia (FTD), and Traumatic Brain Injury (TBI).

Alzheimer's disease, a condition that affects millions of patients and their families each year. Furthermore, the oral, once-daily administration of this therapy makes it a more patient-friendly and cost-effective option compared to injectable drugs. As a result, Aquinnah has garnered considerable industry interest in collaborating to further develop this promising therapeutic.

Aquinnah Pharmaceuticals, dedicated to cutting-edge neurodegenerative research, entered into a collaboration agreement with Roche in 2022 to advance oral small molecules for ALS and other neurodegenerative diseases by addressing TDP-43 pathology, a hallmark in over 95% of ALS patients. The company has received funding from Pfizer, AbbVie, Takeda, and grants from esteemed organizations like the National Institute of Health, the Alzheimer's Association, The Rainwater Foundation, and the Mass Life Sciences Center.



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