Key Advances in Neuromuscular and Movement Disorder Treatments Highlighted in 2024
• AbbVie's Vyalev, a 24-hour subcutaneous levodopa infusion, gained FDA approval for managing motor fluctuations in advanced Parkinson's, offering a novel therapeutic approach.
• Intellia's CRISPR therapy, NTLA-2001, demonstrated safe redosing in ATTR amyloidosis patients, achieving additive pharmacodynamic effects on the target protein.
• A phase 3 study revealed that buntanetap is a safe and well-tolerated drug which improves motor, nonmotor, and cognitive symptoms of Parkinson's disease.
• The FDA supported using αSyn-SAA biomarker in Parkinson's clinical trials, enhancing therapeutic development through improved diagnostic precision.
Prasinezumab Phase IIb Trial Fails Primary Endpoint, Shows Promise in Parkinson's Subgroup
• Genentech's prasinezumab Phase IIb PADOVA study in early-stage Parkinson's missed its primary endpoint of delaying motor progression.
• A pre-specified analysis showed a more pronounced effect in patients treated with levodopa (HR=0.79), suggesting a potential benefit in this subgroup.
• The investigational monoclonal antibody continues to be well-tolerated, with ongoing open-label extension studies to further explore observed effects.
• Roche plans to present full PADOVA study results at an upcoming medical meeting and will work with health authorities to determine next steps.
Amlenetug Receives FDA Fast Track Designation for Multiple System Atrophy
• The FDA has granted Fast Track Designation to Lundbeck's amlenetug for the potential treatment of Multiple System Atrophy (MSA).
• The designation is supported by Phase II AMULET trial outcomes and will expedite the drug development process through rolling reviews and intensive guidance.
• Amlenetug, a human monoclonal antibody, targets extracellular α-synuclein to prevent uptake and aggregation, potentially slowing MSA progression.
• Lundbeck has initiated the Phase III MASCOT trial to evaluate the efficacy, safety, and tolerability of amlenetug in MSA patients across North America, Europe, and Asia.
FDA Approves Supernus' ONAPGO for Motor Fluctuations in Advanced Parkinson's Disease
• The FDA has approved ONAPGO (apomorphine hydrochloride) as the first subcutaneous apomorphine infusion device for treating motor fluctuations in advanced Parkinson's disease.
• ONAPGO, a wearable device, provides continuous subcutaneous apomorphine infusion, offering more consistent control of OFF time for Parkinson's patients.
• Clinical trials demonstrated that ONAPGO significantly reduced daily OFF time by 2.6 hours compared to placebo, with improvements seen as early as week 1.
• Supernus plans to launch ONAPGO in the U.S. in the second quarter of 2025, supported by a dedicated nurse education program and access support.
Lundbeck to Acquire Longboard Pharmaceuticals for $2.6 Billion, Bolstering Epilepsy Pipeline
• Lundbeck will acquire Longboard Pharmaceuticals for $2.6 billion, gaining bexicaserin, a promising drug for developmental and epileptic encephalopathies (DEEs).
• Bexicaserin, a 5-HT2C receptor superagonist, has shown encouraging results in clinical trials for reducing seizure frequency in Dravet syndrome and other DEEs.
• The acquisition aligns with Lundbeck's strategy to expand its neuro-rare disease portfolio, with bexicaserin potentially launching in 2028 and reaching peak sales of $1.5-2 billion.
• The deal is expected to close in the fourth quarter of 2024, pending regulatory approvals and tender of Longboard's outstanding shares.
Lu AF82422 Shows Potential in Slowing MSA Progression in Phase 2 Trial
• Lu AF82422, an anti-alpha-synuclein antibody, showed promise in slowing disease progression in patients with multiple system atrophy (MSA) in the Phase 2 AMULET trial.
• Although the primary endpoint was not met, a more pronounced efficacy signal was observed in a less impaired subgroup of MSA patients.
• The investigational agent was generally safe and well-tolerated, with a significant number of patients opting to continue treatment in an open-label extension.
• These findings support further investigation of Lu AF82422 in a Phase 3 trial for MSA, targeting the underlying pathophysiology of the disease.
Lundbeck Presents Promising Data on Amlenetug for Multiple System Atrophy at MDS Congress
• Lundbeck presented data from the TALISMAN natural history study, enhancing the understanding of Multiple System Atrophy (MSA) progression in early-stage patients.
• The AMULET trial of amlenetug (Lu AF82422) in MSA showed a consistent slowing of clinical progression, with a post-hoc analysis revealing a 42% slowing in a less impaired subgroup.
• Based on the AMULET trial's encouraging results, Lundbeck is set to initiate a Phase III trial of amlenetug at the beginning of 2025, marking a significant step in MSA treatment.
• Amlenetug (Lu AF82422), a human monoclonal antibody, targets extracellular α-synuclein, potentially preventing uptake and inhibiting aggregation in MSA patients.
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