Horizon Study Update for MSA Drug ION464, OnaboutlinumtoxinA to be Tested in Essential Tremor, Challenges With Ketogenic-Mediterranean Diets in PD

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

In an update of the HORIZON trial (NCT04165486), a phase 1/2 single- and multiple-ascending dose study of ION464 (Ionis Pharmaceuticals) in patients with multiple system atrophy (MSA), the investigational agent was shown to be safe and well tolerated thus far, with no serious adverse events (AEs) or deaths related to the study drug. ION464, also known as BIIB101, is an antisense medicine designed to inhibit the production of the alpha-synuclein protein as a potential therapy for a group of neurodegenerative disorders that include Parkinson disease (PD) and MSA.Presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held September 27-October 1, in Philadelphia, Pennsylvania, the poster included baseline characteristics of the first 22 participants from the study. Of these, 36% were female, 91% White, 45% with MSA-Parkinsonism, and 73% with probable MSA. To date, the treatment-emergent AEs recorded while on the study drug include falls (25), urinary tract infection (12), headache (6), contusion (5), and neck pain (3).

At the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held September 27-October 1, in Philadelphia, Pennsylvania, investigators presented the design of ELATE (NCT), a phase 2b clinical trial evaluating the safety and efficacy of onabotulinumtoxinA (onabotA) compared with placebo for the treatment of adult upper limb essential tremor (ULET). The trial, which seeks to enroll 174 participants across 40 sites in the United States and Canada, is expected to be completed in 2024.The hope is that the ELATE study results could reveal valuable insights into the effectiveness and safety of onabotA for ULET, potentially offering a better risk-benefit profile than current oral therapies. ELATE, an 18-week trial, will test 4 regimens, involving onabotA or placebo administered unilaterally or bilaterally. Study investigators will use changes on the Tremor Disability Scale-Revised (TREDS-R) score across 7 unliteral items over that time as the primary efficacy measure.

In an ongoing crossover study (NCT05469997) testing the effects of 2 dietary strategies on patients with Parkinson disease (PD), results showed that while participants embraced Mediterranean-style diets, there were several noted barriers to their combination with ketogenic-style interventions.In the trial, 50 participants were asked to follow two 8-week dietary interventions, separated by an 8-week washout: 1) a high-fat, low-carbohydrate Mediterranean diet (MEDI-KD) and 2) a standard Mediterranean diet supplemented with medium chain triglycerides. Throughout the study period, participant responses from interviews showed that patients took positively to the professional nutritional guidance, increased dietary mindfulness, and Mediterranean-style cuisine. In contrast, negative sentiments were focused on the restrictive nature and social impracticability of the ketogenic diet, as well as the gastrointestinal adverse effects of MCT oil.

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