• Investment Supports APRINOIA’s Effort to Bring Precision Medicine to Neuroscience, Advancing APRINOIA’s Diagnostic and Therapeutic Pipelines.
  • Funding Supports APRINOIA’s Goal to Bring a Central Nervous System (CNS) Protein Degrader Candidate to Investigational New Drug (IND)-Enabling Study.
  • Proceeds also Intended to Advance APRINOIA’s Late-Stage Tau Positron Emission Tomography (PET) to a Global Single Pivotal Phase 3 Trial for Progressive Supranuclear Palsy (PSP).

CAMBRIDGE, Mass., September 11, 2023 — APRINOIA Therapeutics (“APRINOIA” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapeutics and precision diagnostics for the treatment of neurodegenerative diseases, headquartered in Cambridge, MA, is pleased to announce the strategic investment by the Alzheimer’s Drug Discovery Foundation (ADDF) of approximately $4.4M.  This funding is designed to advance APRINOIA’s late-stage diagnostic product candidate, a Phase 3 Tau PET tracer 18F-APN-1607 (INN: florzolotau (18F)) (“APN-1607”), as well as its emerging pre-clinical platform of CNS-targeting protein degraders, which target pathogenic tau proteins. 

“APRINOIA is taking a potentially promising approach to personalized medicine in Alzheimer’s disease (AD) through the development of a novel tau PET biomarker and CNS penetrant tau protein degraders that may be able to safely cross the blood-brain barrier,” says Howard Fillit, MD, Co-Founder and Chief Science Officer at the ADDF.  “The ultimate goal in treatment of Alzheimer’s is to develop new tools that support this precision medicine approach, and the advancement of new diagnostics and therapeutics in tandem will be essential to advancing the next generation of treatments for Alzheimer’s.”

Microtubule-associated protein tau is one of the most abundant neuronal proteins with complex biology and pathophysiology.  Accumulation of misfolded tau aggregates is a pathological hallmark of AD and several neurodegenerative diseases collectively known as tauopathies, including PSP.  With the recent approval of lecanemab (LeqembiTM), the anti-amyloid antibody of Eisai Co., Ltd., and positive results from the Phase 3 trial of donanemab by Eli Lilly, another anti-amyloid antibody, patient disease staging and monitoring using tau PET imaging, will continue to grow in their importance to the field.

There is a significant unmet need to develop biomarkers that can aid in the early diagnosis and differentiation of neurological disorders, attributed to the accumulation of abnormal forms of tau. With the exception of AD, there are no FDA approved biomarkers for the diagnosis of any of the other tauopathies.  Physicians, patients, and their families usually wait several years before a diagnosis can be made. APRINOIA is developing APN-1607 as a novel PET imaging agent that, unlike other tau tracers in development or approved, binds to different abnormal forms of tau with a high degree of selectivity and sensitivity.  Based on a large body of clinical images acquired in over 3,000 patients with various tauopathies, APRINOIA plans to develop APN-1607 as a first-in class PET imaging agent for the detection of tau aggregates in the brains of patients with tauopathies. Funding support from ADDF will enable APRINOIA to initiate a single pivotal, global Phase 3 study to develop APN-1607 as a novel biomarker for the early diagnosis of PSP and related tauopathies.

Beyond innovative diagnostics, APRINOIA is developing innovative therapeutics.  In addition to evaluating the safety of APN-mAb005 in healthy volunteers in a Phase 1 clinical trial, it is optimizing its protein degrader candidates to advance the treatment of neurodegenerative disorders.  APRINOIA has assembled a library of proprietary small molecules that can bind to aggregates of tau and alpha-synuclein to enable their degradation inside brain cells.  There are very few drug development efforts in neuroscience that are pursuing this potentially transformative approach.

With the ADDF’s investment, APRINOIA plans to produce clinical-stage protein degraders capable of penetrating the blood-brain-barrier (BBB), to enter brain cells and clear toxic aggregated forms of tau.  Specifically, the ADDF investment will support the screening and lead optimization of APRINOIA’s degrader library of small molecules that target tau in cellular assays as well as their evaluation of efficacy in animal models.  The objective of this program is to select a lead candidate in preparation for IND-enabling studies in the next 12 months.  

ARPINOIA’s Chief Medical Officer, Bradford Navia, M.D., Ph.D., commented, “We are very excited to receive this generous investment and vote of confidence from the ADDF. This funding will further enable us to advance these critical programs toward their respective milestones. We believe both platforms will be first-in-class in the diagnosis and treatment of neurodegenerative disorders, including AD which afflicts over 50 million people worldwide.  By combining highly selective diagnostics and therapeutics, we believe that precision medicine, which has transformed the diagnosis and treatment of cancer, may finally be realized for neurological disorders.”

About The Alzheimer’s Drug Discovery Foundation (ADDF)

Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer’s Drug Discovery Foundation is dedicated to rapidly accelerating the discovery of drugs to prevent, treat and cure Alzheimer’s disease. The ADDF is the only public charity solely focused on funding the development of drugs for Alzheimer’s, employing a venture philanthropy model to support research in academia and the biotech industry. The ADDF’s leadership and contributions to the field have played a pivotal role in bringing the first Alzheimer’s PET scan (Amyvid®) and blood test (PrecivityAD®) to market, as well as fueling the current robust and diverse drug pipeline. Through the generosity of its donors, the ADDF has awarded more than $250 million to fund over 720 Alzheimer’s drug discovery programs, biomarker programs and clinical trials in 19 countries. To learn more, please visit: http://www.alzdiscovery.org/.

About APRINOIA Therapeutics Inc.

APRINOIA Therapeutics Inc., is a global clinical-stage biopharmaceutical company, headquartered in Cambridge, MA, developing novel therapeutics and precision diagnostics for the treatment of neurodegenerative diseases of the brain.  To learn more, please visit http://www.aprinoia.com and follow us on LinkedIn.

APRINOIA Investor Contact

Matt Hughes