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As part of NeurologyLive®'s Year in Review, take a look at the top most-read news in movement disorders in 2022.
The NeurologyLive® staff spent 2022 covering clinical news and data readouts from around the world across a number of key neurology subspecialty areas—and there was plenty to keep us busy 2022. From major study publications and FDA decisions to societal conference sessions and expert interviews, the team spent all year bringing the latest information to the website's front page.
Among our key focus areas is movement disorders, a field that experienced a turbulent 12 months in the drug development pipeline amid progress in other areas. Although major news items—which can include disappointing updates—often appear among the top pieces our team produces, sometimes smaller stories reach those heights for other reasons—clinical impact and interest, or concerns about the small- or big-picture parts of care, for example. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in this field over the course of 2022.
Here, we'll highlight some of the most-read content on NeurologyLive® this year. Click the buttons to read further into these stories.
Confirming previous reports, new post hoc data from a recently published study in Movement Disorders by Thomas Foltynie, PhD, and colleagues showed an association between Parkinson disease, diabetes mellitus, and more severe neuroaxonal damage.
According to an announcement by Amneal Pharmaceuticals in November, the FDA accepted its new drug application for IPX203, a novel oral formulation of carbidopa/levodopa extended-release capsules intended to treat patients with Parkinson disease, with the agency scheduling a PDUFA date of June 30, 2023, to complete its evaluation of the NDA.
Findings from a case-control study published by Anette Schrag, MD, PhD, in JAMA Neurology using insurance claims of outpatient consultations highlighted several previously known early features associated with subsequent diagnosis of Parkinson disease, which may reflect possible early extrastriatal and extracerebral pathology of the disease.
In October, the US enrollment for the phase 2 PIVOT-HD trial (NCT05358717) assessing PTC Therapeutics' investigational agent PTC518 in patients with Huntington disease was paused, according to the company, as the FDA continued to seek more data about the drug. Although stopped in the US, the study of the oral, small molecule splicing modifier was still enrolling participants at sites in several European countries and in Australia.
According to a community update from August, Novartis had temporarily suspended dosing of the phase 2 VIBRANT-HD study (NCT05111249) evaluating its experimental Huntington disease drug branaplam, citing issues with nerve damage that were reported in treated participants.
In January, almost a year after news that the phase 3 GENERATION HD1 study (NCT03761849) was discontinuing dosing of tominersen, an investigational agent for Huntington disease, its developer Roche announced it was designing a new phase 2 trial after a post hoc analysis showed a possible benefit for subgroup of patients who are younger with less disease burden, according to an announcement from partner company Ionis.
In a presentation given by Rajesh Pahwa, MD, FANA, FAAN, on emerging therapies at the 2022 Advanced Therapeutics in Movement and Related Disorders Congress, four specific therapies on the horizon were highlighted that might be able to address, at least in part, some of the shortcomings of the available options.
In July, Biogen and Alectos Therapeutics entered into a license and collaboration agreement to develop and commercialize AL01811, a novel GBA2 inhibitor designed to treat patients with Parkinson disease. The two companies will collaborate on plans for preclinical testing of the drug; however, Biogen will be solely responsible for all development, manufacturing, and commercial activities and costs.
Six-month interim results from a phase 3, open-label, single-arm study (NCT03781167) presented at the 2022 American Academy of Neurology (AAN) Annual Meeting by Jason Aldred, MD, FAAN, showed that 24-hour/day subcutaneous foslevodopa/foscarbidopa—ABBV-951 (AbbVie)—significantly reduced motor complications, improved morning akinesia, sleep disturbances, and quality of life in patients with advanced Parkinson disease.
Dosing for the large-scale, phase 3 LIGHTHOUSE study (NCT05418673) evaluating the safety and efficacy of BIIB122 (Biogen/Denali Therapeutics), an investigational agent for patients with Parkinson disease with a confirmed pathogenic mutation in the LRRK2 gene, commenced in October.