Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
In recent news, Sarepta revealed new positive findings from its long-term phase 3 EMBARK study (NCT05096221) testing delandistrogene moxeparvovec, or Elevidys, in ambulatory patients with Duchenne muscular dystrophy (DMD). All told, patients from Part 1 of the study on the gene therapy demonstrated notable motor milestone achievements over a 3-year study period, further adding to the drug’s long-term efficacy profile. EMBARK was a double-blind, placebo-controlled, 2-part crossover trial testing a single dose of Elevidys in boys aged 4 to 7 years with DMD. In Part 1, 125 participants were randomized by age and baseline North Star Ambulatory Assessment (NSAA) score to receive Elevidys (1.33 x 1014 vg/kg) or placebo for 52 weeks. In Part 2, groups crossed over, with prior placebo recipients receiving Elevidys and prior Elevidys recipients receiving placebo, each followed for another 52 weeks while maintaining blinding throughout both study phases.
A recent study published in Alzheimer’s & Dementia found that patients with stage III or IV chronic traumatic encephalopathy (CTE) face a significantly higher risk of developing dementia. While prospective studies are needed to validate these associations, the study is among the first to provide evidence for advanced CTE neuropathology alone as being associated with dementia. Funded by the National Institutes of Health, the analysis featured 614 brain donors with (n = 366) and without (n = 248) autopsy-confirmed CTE. All told, patients with stage III CTE were 2.12 times more likely to develop dementia (95% CI = 1.91–3.77, P = 0.011), the primary outcome, while those with stage IV CTE faced an even greater risk, displaying a 4.48-fold increase compared to patients without CTE (95% CI, = 1.97–10.90, P = 0.001).
According to a recent company update, Lexicon Pharmaceuticals completed an End-of-Phase 2 meeting with the FDA to advance pilavapadin, an investigational agent, as a potential treatment for diabetic peripheral neuropathic pain (DPNP). The agency had no concerns over the phase 3 program, which will include two 12-week, placebo-controlled studies evaluating a 10-mg daily dose versus placebo, using change in average daily pain score (ADPS) over a 12-week period as the primary end point. Previously reported topline data from the phase 2b PROGRESS study (NCT06203002), a large-scale study of nearly 500 participants, demonstrated that treatment with pilavapadin led to reductions in pain compared with placebo in adults with moderate to severe DPNP.2 Along with findings from the earlier RELIEF-DPN-1 study (NCT04455633), these results supported selecting a 10-mg once-daily dose for advancement into phase 3 trials.
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