FDA Supports A-Synuclein Assay for PD Trials, VES001 Meets Primary End Point, Digital Migraine Therapeutic CT-132 Performs

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio.

According to a new announcement, the FDA has issued a letter of support for using the α-synuclein seed amplification assay (αSyn-SAA) biomarker in clinical trials for Parkinson disease (PD) and related diseases. This decision comes following a multi-stakeholder collaboration between The Michael J. Fox Foundation for Parkinson's Research (MJFF) and the Critical Path Institute (C-Path) to advance PD therapeutics using this recently discovered biomarker.In the letter, the agency highlighted that its support comes from evidence gathered in the Parkinson's Progression Markers Initiative (PPMI), a longitudinal observational study sponsored by MJFF. The FDA noted that its decision to encourage use of αSyn-SAA also relied on other global studies across industry and academia, which represented the large amount of data as well as the applied collaboration used to validate the tool.

New findings from a phase 1 study of healthy volunteers showed that all end points were successfully met with treatment of Vesper Bio’s VES001, a potentially disease-modifying treatment for frontotemporal dementia with mutations in the progranulin gene (FTD-GRN). The company has completed a clinical trial application seeking to advance the investigational agent into a phase 2a proof-of-concept trial in a relevant patient population, with dosing expected to begin in Q4 2024.In the 2-part study, topline data from the 7-day multiple ascending dose (MAD) portion reaffirmed findings seen in the single-ascending dose (SAD) stage, announced earlier this year. Using a cohort of 78 healthy volunteers, VES001 was considered safe and tolerated across multiple doses, with no serious or treatment-emergent adverse events reported. VES001, a patient friendly, first-in-class, brain penetrant, oral treatment which targets progranulin deficiency, an underlying cause of FTD, demonstrated strong target engagement, evidenced by an increased and accumulating level of progranulin in the volunteers dosed once or twice daily in the MAD stage.

According to an announcement, CT-132 (Click Therapeutics), an investigational prescription digital therapeutic, met its primary end point in the phase 3 ReMMi-D randomized trial (NCT05853900) of patients with episodic migraine, with results revealing a significant reduction in monthly migraine days (MMDs) among treated patients over a 12-week treatment period. ReMMi-D included 568 adults with migraine who were randomly assigned to CT-132 or sham digital control intervention for a 12-week treatment period. The study, a decentralized, double-blind study, enrolled patients on the most commonly prescribed acute and preventive migraine medications. CT-132 delivers a proprietary clinical intervention aimed at reducing brain hypersensitivity implicated in migraine by modulating patient responses to environmental and internal stimuli to help restore patients’ ability to engage fully in daily activities.

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