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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.
Newly announced interim data from a phase 1 study showed that treatment with salanersen (Biogen), an investigational antisense oligonucleotide administered once a year, was safe and led to slowing of neurodegeneration in patients with spinal muscular atrophy (SMA) previously on gene therapy. Based on these findings, Biogen plans to test the therapy in phase 3 studies, the design of which is being discussed with the FDA. Building on the mechanism of action of nusinersen (Spinraza; Biogen), salanersen is designed to enhance potency and allow for once-yearly dosing, offering potential improvements in convenience and efficacy. Mechanistically, salanersen targets the SMN2 gene, modulating its splicing to increase production of functional survival motor neuron (SMN) protein, which is deficient in SMA.
Edgewise Therapeutics has announced positive topline data from the MESA open-label extension study, with results showing that treatment with investigational sevasemten led to notable improvements in North Star Ambulatory Assessment (NSAA) scores over an 18-month period in patients with Becker muscular dystrophy (BMD). In addition, the company shared encouraging data from its phase 2 LYNX and FOX trials of Duchenne muscular dystrophy (DMD), as well as completion of a Type C meeting with the FDA, paving a path for sevasemten to become the first approved therapy for BMD. MESA, an open-label extension, featured 99% of eligible participants from the previously completed ARCH, CANYON, GRAND CANYON, or DUNE trials. In the latest data update, results showed a 0.8-point increase in NSAA scores among participants from CANYON, the major phase 2 trial of sevasemten, after 18 months of treatment. More notably, those who switched from placebo to sevasemten demonstrated a 0.2-point improvement since their crossover.
In a new announcement from UCB, fenfluramine (Fintepla), an FDA-approved antiseizure medication, met its primary and secondary end points in the phase 3 GEMZ trial of patients with CDKL5 deficiency disorder (CDD). Based on these data, the company plans to submit an application for fenfluramine to become a potential treatment option for patients living with CDD. Between baseline and the titration plus maintenance phase, fenfluramine demonstrated statistically significant changes relative to placebo on the primary end point of percent change in countable motor seizure frequency. In this phase 3, double-blind, placebo-controlled, fixed-dose study, fenfluramine continued to show a safety profile that was consistent with its previous indications in Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).
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