Study of P2B001 in PD Concludes, Verdiperstat Fails in MSA, Fenfluramine sNDA Submitted for LGS

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This week Neurology News Network covered the phase 3 study of P2B001 in patients with early-stage Parkinson disease, the failed trial of verdiperstat in patients with multiple system atrophy, and the newly announced supplemental drug application for fenfluramine for another rare epileptic condition.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

According to a recent announcement, the final patient in the phase 3 study of P2B001 (Pharma Two B), an investigational therapy for patients with Parkinson disease, has completed the study, with topline results expected to come in the fourth quarter of 2021. The multicenter, multinational, randomized, double-blind, double-dummy, active-controlled, parallel group study is evaluating the safety, efficacy, and tolerability of P2B001, a synergistic combination of low doses proprietary extended-release (ER) rasagiline and pramipexole, 2 previously approved drugs for PD. The investigational therapy is designed as a once-daily pill with no required titration to address the unmet needs of patients with early PD. The study spans 70 sites in North American and the EU and uses change from baseline to week 12 in total Unified Parkinson’s Disease Rating Scale (UPDRS) score as the primary end point.

Verdiperstat, Biohaven’s investigational first-in-class potent, selective, brain-penetrant, irreversible MPO enzyme inhibitor, failed to statistically differentiate from placebo on prespecified primary efficacy measures in the phase 3 M-STAR clinical trial (NCT03952806) of patients with multiple system atrophy. The company noted that additional analyses are still pending, with full results expected to be presented at an upcoming scientific meeting. A total of 250 patients, aged 40 to 80 years, with MSA were randomized 1:1:1 to verdiperstat 300 mg or 600 mg twice daily or placebo in the double-blind, parallel-group study. The primary efficacy end point was the change from baseline to week 48 on modified Unified MSA Rating Scale (UMSARS) score, which consists of a subset of items from the UMSARS Part I (Activities of Daily Living) and Part II (Motor Exam) that directly assessed clinically meaningful change in patients’ ability to function.

Zogenix has announced that it submitted a supplemental new drug application (sNDA) for fenfluramine (Fintepla), an already FDA-approved treatment for Dravet syndrome (DS), to treat seizures associated with Lennox-Gastaut syndrome. The application was backed by data from the phase 3 Study 1601, which met its primary end point, with the treatment demonstrating significant improvement in monthly drop in seizure frequency (MDSF) among a cohort of 263 patients with LGS. Long-term safety and efficacy of fenfluramine in ongoing open-label extension trials were also included in the submission.Gail Farfel, PhD, executive vice president and chief development officer, Zogenix, said in a statement, “As LGS is a severe, rare form of epilepsy that is not well-controlled by currently available anti-seizure medications, we believe that Fintepla, if approved, would become an important new treatment option that addresses a significant unmet need for this patient population.”

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