DT-216 Reduces Frataxin Levels in FA, Valbenazine FDA-Approved for Huntington Chorea, EU Approves Atogepant as Migraine Preventive

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

Recently announced data from a phase 1, multiple-ascending dose study showed that treatment with DT-216, an investigational agent developed by Design Therapeutics, was generally well tolerated, with statistically significant and dose-related increases in frataxin (FXN) levels in skeletal muscle biopsies among patients with Friedreich ataxia. The company plans to begin another multiple-dose phase 1 trial assessing an improved DT-216 formulation in the second half of 2024, with initial clinical data expected in the first half of 2025. The double-blind, placebo-controlled trial included 29 adults with genetically confirmed FA who received 3 weekly injections of either DT-216 or placebo at doses of 100 mg (n = 4), 200 mg (n = 11), and 300 mg (n = 14). Using a cutoff of August 7, 2023, findings showed no serious treatment-related adverse events (TEAEs) and no treatment-related discontinuations.

The FDA has approved an expanded indication for Neurocrine Biosciences’ therapy valbenazine (Ingrezza) to include the treatment of chorea associated with Huntington disease (HD). The therapy becomes the only selective VMAT2 inhibitor approved for the condition, offering an effective starting dosage that can be adjusted according to response and tolerability with no complex titration. Data from the phase 3 KINECT-HD study, a double-blind trial, and its ongoing open-label extension, KINECT-HD2, served as the basis for the approval. Similar in design, each study featured adults aged 18 to 75 years who had been diagnosed with either manifest HD or motor manifest HD who have sufficient chorea symptoms. In KINECT-HD, the agent met its primary end point, demonstrating a statistically significant placebo-adjusted reduction in the Unified Huntington’s Disease Rating Scale (UHDRS) Total Maximal Chorea score of 3.2 units from baseline to weeks 10 and 12.

The European Commission recently approved AbbVie’s atogepant (Aquipta) for the prophylaxis of migraine in adults who have 4 or more migraine days per month, becoming the first and only calcitonin gene-related peptide (CGRP) agent indicated for prevention of both episodic and chronic migraine. The approval was based on data from 2 phase 3 studies, PROGRESS and ADVANCE, in which atogepant-treated patients showed greater reduction of monthly migraine days than placebo. Atogepant originally received FDA approval as a preventive for episodic migraine in September 2021, and had its label expanded to include chronic migraine earlier this year. It remains approved in the US for both chronic and episodic migraine and in Canada for episodic migraine under the brand name Qulipta.

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