Aquinnah Pharmaceuticals Advances Oral Small Molecule Program Targeting Alzheimer's and Other Tauopathy Diseases

New preclinical data presented at CTAD demonstrates exciting potential for a new drug approach for Alzheimer's disease, reducing brain tau pathology by ~70%

CAMBRIDGE, Mass., Oct. 30, 2023 /PRNewswire/ -- Aquinnah Pharmaceuticals announced preclinical research findings for a novel therapeutic designed to slow or stop the progression of Alzheimer's disease and related disorders. Aquinnah is a leader in pharmaceutical approaches targeting stress granule biology, which underlies the pathology of a wide range of neurodegenerative disorders. The Aquinnah team announced today that their lead compound targets the interaction of tau with stress granules and removes ~70% of tau pathology in an Alzheimer's animal model with advanced stage disease, measured using three different markers of pathological tau that also increase in patients as their disease progresses. Importantly, the Aquinnah compound is expected to be administered orally as a pill, which is generally preferred by patients over injections that are required currently for approved Alzheimer's immunotherapies.

Administration of the compound once daily in preclinical models was sufficient to achieve a strong reduction of abnormal tau protein, which is the key protein contributing to loss of memory and cognition in Alzheimer's disease. Because Aquinnah's compound acts through a unique approach, this new compound also has the potential to be used in combination with anti-amyloid drugs, recently approved by the FDA. If successful in clinical trials, Aquinnah's treatment could benefit patients suffering from Alzheimer's disease by reducing brain damage, restoring memory capabilities, improving brain function and slowing disease progression.

The company's Co-Founder and Chief Scientific Officer, Dr. Benjamin Wolozin MD, Ph.D., presented the research – "Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology" – at the 2023 Clinical Trials on Alzheimer's Disease (CTAD) conference last week in Boston.

"As the number of patients and families impacted by Alzheimer's disease continues to grow worldwide, new medical approaches to target tau pathology, alone or in combination with anti-amyloid therapeutics, will benefit these patients greatly. Research from around the world, including the numerous presentations at CTAD, show that decreasing pathological tau, correlates strongly with improved cognitive function," said Dr. Wolozin. "We're excited about these results and the potential to give Alzheimer's patients a therapeutic agent that will not just delay the disease's progression, but potentially improve their cognitive functioning. Our success with animal models, equivalent to advanced stage Alzheimer's patients, suggests that our compound has exactly this ability to turn the tide for patients suffering from cognitive loss and have or are facing impending dementia caused by Alzheimer's disease."

In addition to its application to Alzheimer's disease, Aquinnah's therapeutic is a potential treatment for other diseases with tauopathies, including Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD), Frontotemporal Lobar Dementia (FTD) and Traumatic Brain Injury (TBI).

Aquinnah's CEO, Glenn Larsen, Ph.D., added, "Alzheimer's disease affects millions of patients and their families each year, so these results and their potential impact are a significant and promising step forward in the fight against this pervasive disease. More importantly, our approach contrasts to existing and developing therapies which are administered through injections. As a once-a-day pill, it will be a preferred method for patients, easier to take early in disease to prevent progression and be less costly compared to injectable drugs. Our strong results have attracted significant interest from the industry to collaborate with us in advancing the development of this promising therapeutic." 

Focused on innovative neurodegenerative research, Aquinnah Pharmaceuticals signed a collaboration agreement with Roche in 2022 to advance oral small molecules for ALS and other neurodegenerative diseases, by modulating TDP-43 pathology, which is the hallmark pathology in more than 95% of ALS patients. Aquinnah has received funding from Pfizer, AbbVie and Takeda, with additional grant funding from the National Institute of Health, the Alzheimer's Association, The Rainwater Foundation and the Mass Life Sciences Center.

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop oral small molecule therapeutics in Neurodegenerative Diseases, focusing on Alzheimer's disease and other tauopathy diseases such as Progressive Supranuclear Palsy and Frontotemporal dementia and ALS, also known as Lou Gehrig's disease, by developing chemical modulators of stress granule pathology. More information is available at www.aquinnahpharma.com.

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