NVSEF - Novartis drops plans to develop Huntington's disease candidate

• Novartis ( NYSE: NVS ) ( OTCPK:NVSEF ) has halted its development program for Huntington’s disease (HD) candidate branaplam after an analysis of mid-stage data, the Swiss pharma giant said Wednesday with its Q4 2022 results.
• The company attributed the decision to a benefit-risk assessment of its Phase 2b VIBRANT-HD study, which was previously linked to safety concerns prompting Novartis ( NVS ) to pause its dosing last year.
• The randomized placebo-controlled study was designed to evaluate the oral therapy in 75 patients with HD, a rare, hereditary disease that leads to the breakdown of nerve cells in the brain.
• The development of HD drugs has a checkered past. In October, PTC Therapeutics ( PTCT ) said it paused enrollment in the Phase 2 PIVOT-HD trial for HD candidate PTC518 in the U.S. following a request from the FDA.
• In March 2021, Wave Life Sciences ( WVE ) announced a halt to the clinical development of HD candidates VE-120102 and WVE-120101, noting a lack of statistically significant changes in disease pathology.

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