APRINOIA Therapeutics' APN-1607 Gains FDA Fast Track for Progressive Supranuclear Palsy Diagnosis

CAMBRIDGE, Mass., May 20, 2024 -- APRINOIA Therapeutics, a clinical-stage biopharmaceutical company focusing on innovative treatments and diagnostics for neurodegenerative diseases, has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) for its product APN-1607 (florzolotau). This Positron Emission Tomography (PET) tracer is designed for imaging tau protein in patients suspected of having progressive supranuclear palsy (PSP).

PSP is a rare neurodegenerative illness characterized by the buildup of a particular form of tau protein in certain brain areas. Currently, there are no FDA-approved diagnostic markers for PSP or other rare tau-related disorders such as frontotemporal dementia. Diagnosis typically relies on clinical evaluations. APN-1607 aims to enable earlier and more accurate diagnoses, which could improve patient care and optimize clinical trial designs for new therapies.

Dr. Brad Navia, Chief Medical Officer of Aprinoia Therapeutics, expressed satisfaction with the FDA's decision, noting that it highlights the urgent need for a diagnostic marker for early-stage PSP and possibly other tau-related disorders, including Alzheimer’s disease. He emphasized that APN-1607 is a distinctive imaging agent created to detect specific tau forms implicated in these disorders. Early and accurate diagnosis of PSP can be challenging, as it is often mistaken for other Parkinson’s-like conditions. If approved, APN-1607 could provide physicians with a reliable diagnostic tool, enabling them to differentiate PSP from other disorders and enhance patient management.

The Fast Track Designation, along with a previously issued "Study May Proceed" letter from the FDA, underscores the significance of APN-1607's clinical development for early diagnosis of PSP and potentially other tau-related neurological disorders. Dr. Navia acknowledged the FDA’s support and the contributions from various investigators and partners, including the Alzheimer’s Drug Discovery Foundation and CurePSP. He noted that early and precise diagnosis is vital for optimizing treatment strategies and accelerating drug development via clinical trials.

Dr. Howard Fillit, Co-Founder and Chief Science Officer at the Alzheimer’s Drug Discovery Foundation, highlighted the importance of the FTD for Aprinoia’s PET tracer. He described it as a significant advancement for the field, marking it as the first tau imaging agent for PSP. Such tools are crucial for improving diagnostic accuracy and supporting precision medicine approaches that ensure patients receive appropriate treatments at the right time.

The Fast Track Designation is meant to speed up the development and review of product candidates that show promise in addressing unmet medical needs, aiming to bring new diagnostic and treatment options to patients more quickly than through traditional regulatory processes. This designation ensures early and frequent communication with the FDA about the product candidate’s development and review. If certain criteria are met, the product may qualify for Accelerated Approval and Priority Review.

APN-1607 is a radioactive fluorinated molecule designed to identify and measure 3R and 4R tau aggregates through PET imaging in various tau-related disorders, including PSP, frontotemporal dementia, and Alzheimer’s disease. Aprinoia has developed it as a pioneering radioactive diagnostic tracer for detecting these tau aggregates. The FDA has previously granted Orphan Drug Designation to APN-1607 for PSP diagnosis. It has been used in over 3,000 patients in clinical settings, demonstrating its potential as a diagnostic marker. In December 2023, Aprinoia completed enrollment for a Phase 3 trial in China to evaluate APN-1607 for Alzheimer’s diagnosis and received FDA approval to conduct a global Phase 3 study for PSP diagnosis.

APRINOIA Therapeutics Inc., headquartered in Cambridge, MA, is dedicated to developing sensitive and selective diagnostic and therapeutic agents for neurodegenerative diseases.

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