Aquinnah Pharmaceuticals Receives $750,000 Grant from the Alzheimer’s Association and Tau Consortium to Advance New Therapies for Tauopathy Diseases

CAMBRIDGE, Mass. — January 17, 2019 Aquinnah Pharmaceuticals, leaders in stress granule biology that afflict a wide range of neurodegenerative disorders, announced today that it has been awarded $750,000 from the Tau Pipeline Enabling Program (T-PEP) to advance its drug development programs in tauopathy diseases, including Progressive Supranuclear Palsy, Alzheimer’s Disease, and Fronto-temporal Dementia. T-PEP is a partnership between the Alzheimer’s Association and the Tau Consortium; a program of the Rainwater Charitable Foundation to explore new ways to prevent, reduce or remove pathological tau from the brain. Aquinnah Pharmaceuticals is the only company recipient and one of 5 awardees selected from 125 applications, to receive funding from the $3 million grant.

Glenn Larsen, Ph.D., President and CEO of Aquinnah said, “Pioneering work from Aquinnah’s co-founder Dr. Ben Wolozin, has shown that stress granule modulation in the brain represents a new and promising approach towards reducing effects of the toxic protein, tau, by showing improved lifespan and preservation of neurons and memory in mice, with potential application to a wide range of neurodegenerative diseases. Aquinnah has developed neuronal models that replicate human tau stress granule pathology and with this funding, we are screening chemical libraries containing over 250,000 small molecules, to identify molecules that have the potential to be developed as novel drugs. We are very pleased that the reviewers at the Alzheimer’s Foundation and Tau Consortium recognize the work we are doing to bring hope to the millions of people affected by these grave disorders.”

Aquinnah’s unique small molecule approach to the inhibition of tau pathology is through the stress granule protein TIA-1 pathway, where a 33% increase in mouse survival was observed, comparable to ~15 years of human life extension, and the best results ever reported in this human tauopathy model of Alzheimer’s Disease.

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop oral small molecule drug candidates in Neurodegenerative Orphan Diseases to prevent TDP-43 disease in amyotrophic lateral sclerosis (ALS); and tau disease pathology in Progressive Supranuclear Palsy, and other diseases including Alzheimer’s disease; by developing inhibitors of stress granule pathology. Aquinnah is financially backed by Pfizer, Inc., AbbVie, Inc. and Takeda Pharmaceuticals. www.aquinnahpharma.com

Media Contact
George MacDougall
MacDougall Biomedical Communications
gmacdougall@macbiocom.com
781-235-3060

Aquinnah Pharmaceuticals Receives $3.4M Grant from NINDS To Advance New Therapies for ALS

CAMBRIDGE, Mass. — May 15, 2018 Aquinnah Pharmaceuticals, leaders in stress granule biology, an exciting new target for the development of neurodegenerative therapeutics, announced today that it has been awarded $3.4 million from the National Institute of Neurological Disorders and Stroke (NINDS) in a competing grant to advance novel therapeutic drug candidates towards the clinic for treating patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

Aquinnah Pharmaceuticals is developing innovative drugs designed to slow or potentially reverse the progression of ALS. Currently there is no cure or effective treatment for slowing the progression of ALS, a lethal neurodegenerative disease that quickly stifles nerve cell function, leading to loss of muscle control throughout the body. ALS can progress rapidly with more than half of newly diagnosed patients not surviving beyond three years.This is Aquinnah’s second NINDS grant awarded to support its unique approach to treating ALS, which targets the prevention of pathological TDP-43 stress granule protein complexes found in the brains of most ALS patients. The accumulation of these disease-linked protein complexes is believed to drive the progression of ALS and other neurodegenerative diseases. Aquinnah will use newly identified small molecule compounds to attack and break down the complexes, with the goal of moving a novel and effective class of ALS drugs into clinical development.

Glenn Larsen, Ph.D., President and CEO of Aquinnah said, “Modulating stress granules in the brain represents a new and exciting approach to treating and potentially preventing a wide range of neurodegenerative diseases. We are very pleased that NINDS recognizes this, leading to funding for our innovative ALS program. Our mission is to revolutionize ALS treatment and change the clinical outcome of this devastating disease by developing a new drug to slow or potentially reverse the progression of ALS.”

“This support from the NINDS will greatly help advance Aquinnah's science toward IND-enabling activities and our goal of developing novel ALS therapies,” said co-founder Ben Wolozin, M.D., Ph.D. "Our work remains urgent knowing that our efforts could radically change the outcomes for ALS patients within a matter of years."

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop oral small molecule drug candidates to prevent TDP-43 pathology in amyotrophic lateral sclerosis (ALS); and with other programs targeting Alzheimer’s disease by developing inhibitors of tau stress granule pathology. Aquinnah is financially backed by AbbVie, Inc., Pfizer, Inc. and Takeda Pharmaceuticals.

ABOUT NINDS   

NINDS is the nation’s leading funder of research on the brain and nervous system. The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

Company Contact
Glenn Larsen, President & CEO
Aquinnah Pharmaceuticals
news@aquinnah.com
617-250-8157

Media Contact
George MacDougall
MacDougall Biomedical Communications
gmacdougall@macbiocom.com
781-235-3060

Aquinnah Pharmaceuticals Appoints Travis Wager as Vice President of Research

Former Pfizer research lead to drive development of Aquinnah’s neurodegenerative therapeutic development programs

CAMBRIDGE, Mass. — May 15, 2018 Aquinnah Pharmaceuticals, leaders in stress granule biology, an exciting new approach in the development of neurodegenerative therapeutics for ALS and Alzheimer’s diseases, today announced the appointment of Travis T. Wager, Ph.D., as Vice President of Research. Dr. Wager brings almost 20 years of leadership experience driving neuroscience research and drug discovery programs at Pfizer. Dr. Wager will oversee all discovery biology and chemistry activities for Aquinnah’s growing pipeline of neurodegenerative therapeutics.

“We are very excited to welcome Travis to our growing team,” said Glenn Larsen, Ph.D., Co-founder, President and CEO of Aquinnah. “His leadership and experience at Pfizer, where he helped advance eight neuroscience programs to clinical development, and his deep and extensive research and development experience will be invaluable as we advance our therapeutic programs targeting stress granules in both ALS, tau biology in Alzheimer’s, and other neurodegenerative diseases.”

Prior to joining Aquinnah, Dr. Wager held various leadership positions at Pfizer including Project Leader for Neuroscience and Head of small molecule design for the Neuroscience and Pain Research Unit. Dr. Wager is an internationally recognized expert in the field of brain-penetrant small molecule design. His seminal contributions to the understanding of physicochemical properties and the development of the CNS MPO desirability tool has enabled drug discovery design across the industry. He has been published extensively in ACS Chemical Neuroscience and Journal of Medicinal Chemistry, authored nearly 40 scientific papers and holds over 30 patents. While at Pfizer, Dr. Wager’s eight small molecule programs advanced to clinical development candidates, including the first kinase inhibitor for a CNS indication. Beyond developing new medicines targeting Alzheimer’s disease, Dr. Wager has developed compounds to treat Schizophrenia, Attention Deficit Hyperactivity Disorder (ADHD), narcolepsy and allergic rhinitis. He has received numerous awards including the W.E. Upjohn Award from Pfizer, the Cheves T. Walling Award from University of Utah and is also a member of the Editorial Advisory Board for ACS Chemical Neuroscience. Dr. Wager obtained a Ph.D. in Organic Chemistry from the University of Utah with Professor G. E. Keck, where he also conducted his postdoctoral research.

“The field of neurodegenerative diseases is vast and includes a number of conditions with high, unmet medical needs, such as ALS and Alzheimer’s, where I have dedicated my education and career,” said Dr. Wager. “I look forward to leading the research and development team at Aquinnah and advancing our first-in-class medicines to accomplish Aquinnah’s mission to provide life-changing impact for patients around the world.”

About Aquinnah Pharmaceuticals

Aquinnah Pharmaceuticals is harnessing the power of stress granule biology to develop a new class of drug candidates with the potential to prevent and possibly reverse the pathology of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, as well as Alzheimer’s disease. Aquinnah is financially backed by Abbvie, Inc., Pfizer, Inc. and Takeda Pharmaceuticals.

Company Contact
Glenn Larsen, President & CEO
Aquinnah Pharmaceuticals
news@aquinnah.com
617-250-8157

Media Contact
George MacDougall
MacDougall Biomedical Communications
gmacdougall@macbiocom.com
781-235-3060