Examining Promising Phase 2b PARADIGM Findings of ALS Agent PrimeC: Merit Cudkowicz, MD, MSc

WATCH TIME: 4 minutes

"It’s important for clinicians to know that there is a big pipeline of therapies coming for trials in ALS, and they can become part of those clinical trials if they would like to. Also, it is essential for clinicians to make sure their patients know that there still is a lot of hope and options for them coming up.”

PrimeC (NeuroSense) is a novel extended-release oral formulation composed of a unique fixed-dose combination of 2 FDA-approved drugs: ciprofloxacin and celecoxib. The therapy, an investigational amyotrophic lateral sclerosis (ALS) agent, has been granted orphan drug designation by the FDA and the European Medicines Agency in the past. The phase 2b PARADIGM study (NCT0535790) assessing the agent included 69 patients with ALS who were randomly assigned 2:1 to either PrimeC or placebo for a 6-month double-blind period, followed by a 12-month open-label extension.

At the 6-month timepoint in PARADIGM, PrimeC achieved its primary safety and tolerability end points, along with secondary clinical efficacy end points. All told, PrimeC-treated patients demonstrated a slowing of disease progression in comparison with placebo at 6 months, explained by a 29% difference in ALS Functional Rating Scale. In addition, investigators recorded a 13% treatment difference favoring the investigational agent on slow vital capacity, a measure of respiratory function. All told, the safety and tolerability profile of PrimeC was considered comparable to the placebo.¹

These findings were presented at the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, by Merit Cudkowicz, MD, MSc. Cudkowicz, chair of neurology at Massachusetts General Hospital, sat down with NeurologyLive^® at the meeting to further discuss the primary drugs used in the combination therapy of PrimeC, and why they were chosen for ALS treatment. In addition, she talked about the key findings of the phase 2 trial regarding safety, clinical effects, and biomarkers. She also spoke about the expectations and plans for the upcoming phase 3 trial of PrimeC for ALS.

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REFERENCES
1. Cudkowicz M, Chio A, Lunetta C, et al. PrimeC, An Oral Candidate for Amyotrophic Lateral Sclerosis, Meets Primary Safety and Secondary End Points in the Phase 2b PARADIGM Trial. Presented at: 2024 AAN Annual Meeting; April 13-18; Denver, CO.