APRINOIA Therapeutics Inc. receives “Study May Proceed” letter from FDA for a Phase 3 study of APN-1607 (florzolotau) for the diagnosis of Progressive Supranuclear Palsy

Cambridge, MA, January 3, 2024 — APRINOIA Therapeutics Inc. (“APRINOIA” or the “Company”) is pleased to announce that on December 8, 2023, the United States Food and Drug Administration (“FDA”) has issued a “Study May Proceed” letter for APRINOIA’s newly filed investigational new drug application for the clinical investigation of a Phase 3, open-label, multicenter, prospective study of the diagnostic efficacy and safety of 18F-APN-1607 (INN: florzolotau (18F)) (“APN-1607”) positron emission tomography (“PET”) in patients suspected to have progressive supranuclear palsy (“PSP”). APRINOIA is developing APN-1607 as a PET imaging tracer for the detection of 3R and 4R tau aggregates, which contribute to the pathogenesis of various tau-related neurological disorders, including PSP, frontotemporal dementia (“FTD”), and Alzheimer’s disease (“AD”). The FDA’s Study May Proceed decision enables APRINOIA to conduct a single, global Phase 3 trial to evaluate the performance of APN-1607 as a biomarker for the early diagnosis of PSP. If the results from this single trial are positive, they will serve as the primary basis of approval of APN-1607 in the United States.

PSP is a rare neurodegenerative disorder caused primarily by the accumulation of tau in subcortical brain regions. There are no FDA-approved diagnostic markers for PSP or for any other rare tau-related disorders such as FTD. Until now, diagnosis has primarily relied on clinical assessment. APN-1607 may enable a more accurate diagnosis at earlier stages of the disease, potentially resulting in more efficient clinical trial designs for novel therapies and potentially to a more personalized approach to treatment. We expect that the Phase 3 trial will be a global study and include sites in the United States, Europe, the United Kingdom and Asia.

“This is an exciting and important milestone for APRINOIA, and for the field. Developing a reliable diagnostic marker for PSP and related disorders is a huge unmet need. APRINOIA is committed to developing APN-1607 as a diagnostic marker that will enable early diagnosis of PSP, a devastating disorder, and help identify patients at earlier stages of their disease for clinical trials evaluating the efficacy and safety of novel disease modifying therapies. The FDA’s Study May Proceed letter represents a critical milestone in developing APN-1607 as an effective and safe diagnostic marker for PSP”, said Dr. Bradford A. Navia, Chief Medical Officer of the Company.

About APN-1607

APN-1607 is a radioactive fluorinated molecule developed to visualize and quantify tau aggregates by PET imaging across a number of diverse tau-related disorders, including PSP, FTD, and AD, among others. 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 2,800 patients through investigator initiated and sponsor trials, supporting its potential clinical utility as a diagnostic marker for tau-related disorders.


APRINOIA Therapeutics Inc., is a clinical-stage biotechnology company, headquartered in Cambridge, MA, committed to protecting patients’ brain health and changing clinical outcomes for a broad range of neurodegenerative diseases by developing novel, highly sensitive and selective diagnostic tools and novel therapeutics.  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.