APRINOIA Therapeutics Announces Fast Track Designation Granted by U.S. FDA to APN-1607 (florzolotau) for the Diagnosis of Progressive Supranuclear Palsy

Fast Track Designation follows announcement in January 2024 that the U.S. Food and Drug Administration (FDA) granted a “Study May Proceed” letter for Aprinoia’s planned Phase 3 study of APN-1607 (florzolotau) in the U.S.,U.K., Europe and Asia

CAMBRIDGE, Mass., May 20, 2024 (GLOBE NEWSWIRE) — APRINOIA Therapeutics (“Aprinoia” or the “Company”), a clinical-stage biopharmaceutical company developing novel therapeutics and precision diagnostics for the treatment of neurodegenerative diseases, today announced that on May 8, 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation (FTD) to APN-1607 (florzolotau), a Positron Emission Tomography (PET) tracer for imaging tau protein in patients with suspected progressive supranuclear palsy (PSP).

PSP is a rare neurodegenerative disorder caused primarily by the accumulation of a specific form of tau in subcortical brain regions. There are no FDA-approved diagnostic markers for PSP or any other rare tau-related disorder such as frontotemporal dementia, and until now, diagnosis has primarily relied on clinical assessment. APN-1607 may enable more accurate diagnosis at earlier disease stages, potentially improving patient management and resulting in more efficient clinical trial designs for novel therapies.

“We are very pleased with the FDA’s decision to grant APN-1607 Fast Track Designation as it underscores the significant unmet medical need for a diagnostic marker for the early diagnosis of PSP and potentially other tau-related disorders, including Alzheimer’s disease. APN1607 is a unique imaging agent as it was designed to detect specific forms of tau implicated in PSP and other related disorders. Sadly, patients with PSP can remain undiagnosed for several years as it is often confused with other Parkinson’s like disorders, especially during the early stages. If approved, APN-1607 would provide physicians with an important diagnostic tool that will allow them to diagnose PSP with greater confidence and differentiate it from other disorders, thereby improving the management of these patients,” stated Dr. Brad Navia, Chief Medical Officer of Aprinoia Therapeutics.

“Receiving Fast Track Designation, along with the previously announced “Study May Proceed” letter from the FDA, reinforces the importance of our work related to APN-1607, and specifically, the clinical development plan to advance the asset into the clinic for the early diagnosis of PSP, and potentially other tau-related neurological disorders, including frontotemporal dementia and Alzheimer’s disease. We look forward to our further engagement with the FDA as a part of APN-1607’s Fast Track Designation, as we seek to accelerate the clinical development program for this important diagnostic tracer. We are grateful to our many investigators and partners, including the Alzheimer’s Drug Discovery Foundation and CurePSP for their continued support in our efforts to advance APN-1607 for the diagnosis of PSP and related disorders,” said Dr. Navia.

“Providing patients with neurodegenerative disorders, such as Alzheimer’s, and PSP with an early and accurate diagnosis is critical for determining the best course of treatment as well as accelerating drug development via clinical trials. The Fast-Track Designation for Aprinoia’s PET tracer is a milestone for the field that will serve as the first tau imaging agent for PSP and will add to the arsenal of tau imaging tools for Alzheimer’s. This new generation PET Tracer – in addition to other biomarkers – will move us closer to the day when we can treat the right patients with the right drugs at the right time through a precision medicine approach,” said Dr. Howard Fillit, Co-Founder and Chief Science Officer at the Alzheimer’s Drug Discovery Foundation (ADDF).

Fast Track Designation is designed to facilitate the development and expedite the review of product candidates that demonstrate the potential to address an unmet medical need, with the goal of advancing important new diagnostic and treatment options to patients more quickly than traditional regulatory routes.

Once a drug candidate receives Fast Track Designation, early and frequent communication with the FDA, including discussions around the product candidate’s development plan and regulatory review process are ensured. If the relevant criteria are met, the product candidate may be eligible for Accelerated Approval and Priority Review by the FDA.

About APN-1607
APN-1607 is a radioactive fluorinated molecule developed to visualize and quantify 3R and 4R tau aggregates by PET imaging across a number of diverse tau-related disorders, including PSP, frontotemporal dementia and Alzheimer’s disease, among others. APRINOIA is developing APN-1607 as a first-in-class radioactive diagnostic tracer for the detection of 3R and 4R tau aggregates, which contribute to the pathogenesis of various tau-related neurological disorders. APRINOIA previously received an Orphan Drug Designation from the FDA for APN-1607 as a diagnostic agent for PSP. APN-1607 has been clinically utilized in over 3,000 patients through investigator-initiated and sponsor trials, supporting its potential clinical utility as a diagnostic marker for tau-related disorders. In December 2023, enrollment of a Phase 3 trial to evaluate the efficacy and safety of APN-1607 for the diagnosis of Alzheimer’s disease was completed in China and on December 8, 2023, Aprinoia received a “Study May Proceed” letter from the U.S. FDA to conduct a global Phase 3, multicenter, open-label study to evaluate the efficacy and safety of APN-1607 as a diagnostic marker in patients suspected to have PSP.

APRINOIA Therapeutics Inc. is a clinical-stage biotechnology company, headquartered in Cambridge, MA, committed to developing highly sensitive and selective diagnostic and therapeutic agents for a broad range of neurodegenerative diseases. To learn more, please visit http://www.aprinoia.com and follow us on LinkedIn.

Forward-Looking Statement
This communication contains forward-looking statements which reflect APRINOIA’s current expectations regarding future events, including its expectations for the future development of the Company. APRINOIA’s actual results may differ from its expectations, estimates and projections and consequently, you should not rely on these forward-looking statements as predictions of future events. Forward-looking statements include statements concerning plans, objectives, goals, strategies, future events or performance, and underlying assumptions and other statements that are other than statements of historical facts. No representations or warranties, express or implied are given in, or in respect of, this communication. When APRINOIA uses words such as “may,” “will,” “intend,” “should,” “believe,” “expect,” “anticipate,” “project,” “estimate” or similar expressions that do not relate solely to historical matters, it is making forward-looking statements. Forward-looking statements speak only as of the date they are made and factors that may cause actual results to differ materially from current expectations include, but are not limited to: the performance of APRINOIA’s business; the risk that preclinical studies and early-stage clinical trials may not be predictive of future results; the risk that regulatory approvals for APRINOIA’s product development are not obtained or are delayed, and the timing, success and cost of APRINOIA’s pipeline development activities and clinical trials. There may be additional risks that APRINOIA does not presently know, or that APRINOIA currently believes are immaterial, that could cause actual results to differ from those contained in the forward-looking statements. APRINOIA undertakes no obligation to publicly revise these forward-looking statements to reflect events or circumstances that arise after the date of this communication, except as required by applicable law.

Nick Colangelo