-Enigma Biomedical USA (EB USA) today announced the U.S. Food and Drug Administration (FDA) approval of TAUKLARIFY™ (florquinitau F 18 injection, also previously referred to as MK-6240), a radiodiagnostic agent indicated for positron emission tomography (PET) of the brain in adults with cognitive impairment who are being evaluated for Alzheimer’s disease to identify patients with tau neurofibrillary tangle (NFT) pathology.

In 2023, EB USA sold its subsidiary, Cerveau Technologies, to Lantheus to complete development of and commercialization of TAUKLARIFY. The companies have continued to collaborate to advance product development, which has culminated in this FDA Approval.

Rick Hiatt, President and CEO of EB USA stated: “The FDA granted Fast Track designation to TAUKLARIFY, a status to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. With the approval of TAUKLARIFY, EB USA demonstrates its ongoing commitment to be the premier developer of imaging biomarkers for neurological pathologies to accelerate the development, approval, and adoption of effective therapies to treat neurodegenerative diseases.”

“TAUKLARIFY imaging will enable improved understanding of the status of Alzheimer’s disease patients across the spectrum of cognitive impairment, including those in the early stages of the disease,” said Samantha Budd Haeberlein, PhD, Chief Medical Officer of EB USA. “Two large, independent clinical studies demonstrated the efficacy and safety of TAUKLARIFY in assessing the status of NFTs in patients being evaluated for Alzheimer’s disease. As Alzheimer’s research moves towards an increased focus on early disease detection and treatment, TAUKLARIFY is uniquely suited to address those emerging needs.”

“This approval is an important and critical step forward in the management of patients being evaluated for Alzheimer’s disease,” said Sterling Johnson, PhD, Jean R. Finley Professor of Geriatrics and Dementia, Associate Director and Core Leader of the Wisconsin Alzheimer’s disease Research Center, and Associate Director of the Wisconsin Alzheimer’s institute. “The University of Wisconsin Alzheimer’s disease Research Center is proud to have been one the earliest adopters of MK-6240 in the academic research setting. We have continued to grow our use of TAUKLARIFY as our imaging agent of choice for assessment of tau neurofibrillary tangles, based on several clinically important features, particularly its high dynamic range. We find it particularly useful in elucidating the status of AD patients with mild cognitive symptoms. We are excited to witness the transition of this valuable tool from research to broad community clinical use enabled by this approval.”

TAUKLARIFY was evaluated in two clinical studies. Five independent blinded readers in each study classified scans as positive or negative for tau neurofibrillary tangle (NFT) pathology and each scan was then compared against a pre-established reference standard based on cognitive status and amyloid beta PET pathology status. The studies included scans from a total of 617 subjects, including 152 with mild Alzheimer's disease dementia, 157 with mild cognitive impairment, and 308 cognitively unimpaired individuals. Both studies met their pre-specified success criteria:

- Study 1, (n=279), with reader Positive Percent Agreement* (PPA) (95% CI) ranging from 80% (72, 86) to 88% (82, 93), and Negative Percent Agreement (NPA) from 98% (94, 99) to 99% (95, 100).
- Study 2, (n=338), with reader PPA (95% CI) ranging from 68% (61, 75) to 82% (76, 87), and NPA from 93% (89, 96) to 99% (96, 100).

Inter-reader agreement was high in both studies, with a generalized Fleiss' kappa of 0.92 in Study 1, and 0.86 in Study 2. Safety was evaluated in 1,734 subjects. The most commonly reported adverse reactions were headache (0.7%), nausea (0.2%), injection site reactions (0.1%), dizziness (0.1%), and abdominal discomfort (0.1%)¹.

TAUKLARIFY™ Indication
TAUKLARIFY is indicated for positron emission tomography (PET) of the brain in adults with cognitive impairment who are being evaluated for Alzheimer’s disease to identify patients with tau neurofibrillary tangle (NFT) pathology.

TAUKLARIFY Limitations of Use
The safety and effectiveness of TAUKLARIFY have not been established for the evaluation of non-Alzheimer’s disease tauopathies.