Aquinnah Pharmaceuticals Announces Alzheimer's Clinical Trial Support

Cambridge, MA – Aquinnah Pharmaceuticals is a biotechnology company developing innovative oral small-molecule therapeutics for neurodegenerative diseases. Today Aquinnah announced that it has been granted a $1 million award from the Alzheimer's Association's Part the Cloud program. The grant will help advance the company's novel oral therapeutic, AQV-8741, in Phase 1 clinical trials for use in Alzheimer's disease and related tauopathy diseases. 

The Aquinnah drug targets prevention of pathological tau protein, which is the main protein that damages neurons as it accumulates with progression of Alzheimer’s disease.  AQV-8741 was developed using Aquinnah's proprietary platform that targets the toxic interactions between pathologic tau protein and stress granules—dynamic biomolecular condensates that drive protein aggregation and neurodegeneration.

AQV-8741 is a first-in-class, brain-penetrant oral small molecule that has demonstrated compelling preclinical efficacy, including a ~70% reduction in brain tau pathology in advanced Alzheimer's disease models; with tau pathology determined by measuring phosphorylated and misfolded tau, accepted measures of human disease. Thus, the Aquinnah therapeutic AQV-8741 demonstrates the power to not just slow, but potentially reverse disease progression.

This novel approach addresses a critical root cause of disease progression, offering the potential to be a disease modifying drug for neurodegeneration in Alzheimer's, frontotemporal dementia  and  other tauopathy diseases.

"This prestigious Part the Cloud award validates our innovative stress granule-targeted mechanism and provides crucial funding to bridge our program into human trials," said Glenn Larsen, Ph.D., Co-Founder, President, and CEO of Aquinnah Pharmaceuticals. "With tau emerging as a key therapeutic target in the post-amyloid era, we are excited to accelerate this oral therapy toward the clinic, bringing hope to millions affected by Alzheimer's and related diseases."

Part the Cloud, founded by philanthropist Michaela "Mikey" Hoag, accelerates transformative Alzheimer's research by funding early-phase clinical studies that fill critical gaps in drug development. Since its inception, the program has invested in high-impact projects, many of which have attracted substantial follow-on funding, including support for Aquinnah Pharmaceuticals first in human, phase 1 studies.

Aquinnah's tau program builds on foundational research by Co-Founder and Chief Scientific Officer Benjamin Wolozin, M.D., Ph.D., a pioneer in linking stress granules to neurodegenerative pathology. “This compound opens up a new approach to the treatment of Alzheimer’s disease targeting but not binding to pathological tau, while at the same time dampening the chronic stress response that underlies aging and Alzheimer’s disease. I am very excited to move this promising therapeutic into clinical trials and beyond to help all those suffering from this dreaded disease.” 

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power by integrating the fields of stress granule biology, RNA binding proteins & bimolecular condensate formation to develop oral small molecule drug candidates in Neurodegenerative Diseases.  Aquinnah’s therapeutics are designed to stop or even reverse diseases such as Alzheimer's Disease, amyotrophic lateral sclerosis (ALS), and frontotemporal disease (FTD) by eliminating pathologies caused by the proteins tau and/or TDP-43. Aquinnah has received numerous awards, including from the ALS Association, National Institutes of Health, the Alzheimer’s Association, Rainwater Foundation and the Alzheimer's Drug Discovery Foundation.  See the website, www.aquinnahpharmaceuticals.com, for more information.

About the Alzheimer's Association and Part the Cloud

The Alzheimer's Association is the leading voluntary health organization in Alzheimer's care, support, and research. Part the Cloud accelerates global progress toward treatments by funding early-phase clinical trials of novel therapies. See their press release at www.alz.org/news/2026/part-the-cloud-grants-11-million-to-develop-innovative-treatments.

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, MA – October 30, 2023 – 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.” 

Aquinnah Pharmaceuticals is also focused on innovative neurodegenerative research to advance oral small molecules for ALS 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.

Dr. Benjamin Wolozin to Present “Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology” at CTAD 2023

Cambridge, MA – October 24, 2023 – Aquinnah Pharmaceuticals, leaders in stress granule biology that afflict a wide range of neurodegenerative disorders, today announced that the company’s Co-Founder and Chief Scientific Officer, Dr. Benjamin Wolozin MD, Ph.D., will be presenting new preclinical research at the 2023 Clinical Trials on Alzheimer’s Disease (CTAD) conference. CTAD is the premier industry event highlighting new research and breakthroughs in the areas of disease-modifying agents, gene therapy, vaccines and multimodal interventions.

Dr. Wolozin’s session – “Development of Orally Available, Brain Penetrant Compound Reducing Tau Pathology” – is part of CTAD’s late breaking track and will take place on Friday, October 27th at 4:45 p.m. in the Grand Ballroom AB on Floor 2 of the Boston Park Plaza hotel. CTAD 23 is sold out, but attendees can still register for virtual attendance.

In addition to his role at Aquinnah, Dr. Wolozin is also a professor of Anatomy & Neurobiology and Neurology at the Boston University School of Medicine. A distinguished scientist, Dr. Wolozin identified one of the first molecular markers for the tangle pathology that accumulates in neurons in Alzheimer’s disease, the first to identify the link between the use of statins and reduced rates of Alzheimer’s disease, and more recently he discovered the role of persistent stress granules in Alzheimer’s disease and ALS.

“Alzheimer’s disease is devastating for patients and their families,” said Dr. Wolozin. “Aquinnah is excited to be at CTAD presenting a novel therapeutic approach for Alzheimer’s disease. Our therapeutic removes abnormal tau protein, which is the key protein contributing to loss of memory and cognition in Alzheimer’s disease. If successful in clinical trials, Aquinnah’s treatment offers the hope of reducing the brain damage and correspondingly restoring brain function for patients suffering from Alzheimer’s disease.”

Aquinnah’s CEO, Glenn Larsen, Ph.D., emphasized the significance of Aquinnah’s results that will be presented at CTAD, “In the treatment and prevention of Alzheimer’s disease progression, two proteins have received significant attention – beta-amyloid and Tau. The FDA recently approved two anti-amyloid biologic drugs. The approval of these drugs was very important for the field, but both drugs offer only modest benefits and require intravenous administration. Aquinnah’s approach will allow patients to orally take a pill that targets the Tau protein pathology that is also involved in Alzheimer’s disease progression and that potentially can be used alone or in combination with anti-amyloid drugs.” 

Focused on innovative neurodegenerative research, Aquinnah Pharmaceuticals is funded by AbbVie, Pfizer 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. Based in Kendall Square, the company operates from The Engine at MIT and is an alumni company of LabCentral.

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.