As part of NeurologyLive®'s Year in Review, take a look at our most-read news in movement disorders in 2023.
The NeurologyLive® staff was hard at work in 2023, covering clinical news and data readouts from all over the United States and the world, across a number of key neurology subspecialty areas. From major study data and FDA decisions to medical society conference sessions and expert conversations, the team spent all year bringing the latest news and updates to the website's front page.
Among our key focus areas is movement disorders, which include a number of complex diseases that have benefitted greatly from recent advances in medical care and therapeutic development. Although major news items—such as first-time approvals or new guidelines—often appear among the top pieces our team produces, sometimes smaller stories reach those heights for other reasons, such as clinical impact and interest, or concerns about other facets 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.
Here, we'll highlight some of the most-read content on NeurologyLive® this year. Click the buttons to read further into these stories.
Exploratory findings from a phase 3 randomized, controlled trial (NCT03329508) assessing P2B001 (Pharma Two B), a low dose combination of extended-release pramipexole and rasagiline, in Parkinson disease (PD) showed efficacy that was comparable to extended-release pramipexole (Prami-ER) alone, but with reduced sleep-related and dopaminergic adverse events (AEs). Pharma Two B planned to submit a new drug application for P2B001 to the FDA in 2023.
Newly announced findings from a triple-blinded, randomized controlled trial showed that treatment with SYMBYX Neuro infrared light therapy helmet significantly improved symptoms of Parkinson disease (PD) in areas of facial expression, upper and lower limb coordination and movement, walking gait, and tremor. Using the standardized Movement Disorder Society Unified Parkinson’s Disease Rating Scale-III (UPDRS-III), compared with the placebo group, those on the light therapy improved 24% to 58% over baseline across all 5 areas tested, unlike the placebo group, which demonstrated statistically valid improvement in lower limb coordination and movement only.
After showing positive results in a phase 3 clinical program, the FDA has accepted Revance Therapeutic’s supplemental new biologics license application (sBLA) for daxibotulinumtoxinA injection (Daxxify), as a new treatment for adults with cervical dystonia. The agency ultimately approved the therapy on August 15, 2023. DaxibotulinumtoxinA is an acetylcholine release inhibitor and neuromuscular blocking agent indicated for the temporary improvement of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adults. To date, the therapy has shown promising results in 2 phase 3 studies of cervical dystonia, ASPEN-1 (NCT03608397) and ASPEN-OLS (NCT03617367).
Despite years of use of gold-standard therapy levodopa, therapeutic development in Parkinson disease has advanced rapidly and expanded to numerous novel pathways and targets. MedStar Georgetown's team of Katelynn Getchell; Gonul Ozay, MD; Brian Nagle, MD; Irma Zhang, MD; Luke Lovelace; Emma Waldon, RN; Yasar Torres-Yaghi, MD; and Fernando L. Pagán, MD explore this in depth.
Topline data from the Synuclein-One Study of CND Life Sciences’ Syn-One Test, an α-synuclein skin biopsy test used for the detection of the pathology in Parkinson disease (PD), dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF), suggest that the test is sensitive and specific in said detection of phosphorylated α-synuclein. As misdiagnosis remains a consistent challenge in neurodegenerative disorders—some estimates suggest a misdiagnosis rate of 30%—this represents an opportunity to address this clinical obstacle.
Using prospective cohort studies of community-dwelling elders followed up to 20 years, findings published in Neurology identified specific cognitive and functional declines in patients who developed incident Parkinson disease (PD). There were important sex differences as well, as men with incident PD had a steeper decline in executive function compared with women, but only women with incident PD exhibited detectably faster prediagnostic decline in global cognition.
BIAL R&D announced the dosing of the first patient in its phase 2 clinical trial, ACTIVATE (NCT05819359), to investigate BIA 28-6156, an allosteric activator of the enzyme beta-glucocerebrosidase (GCase), as a treatment of patients with genetically-mutated Parkinson disease (PD). The trial, which includes those with a mutation in the glucocerebrosidase 1 (GBA1) gene (GBA-PD), otherwise the most common genetic risk factor of the disease, is screening patients across sites in North America and with a Europe-based trial planned to initiate in the third quarter of 2023.
Biogen and Denali have announced that they are discontinuing a portion of the clinical development program for BIIB122 (also known as DNL151), an investigational small molecule inhibitor of LRRK2 in development for the treatment of Parkinson disease (PD). As a result of this decision, the phase 3 LIGHTHOUSE study (NCT05418673), which was initiated in September 2022, will be terminated.
New data from a first-in-human phase 1 study (NCT04802733) assessing bemdaneprocel (BlueRock Therapeutics/Bayer), an investigational cell therapy, showed that the agent met its primary objective of safety, with encouraging results on other measures of motor and nonmotor outcomes. Based on these results, the companies are planning for a phase 2 trial that is expected to begin enrolling patients in the first half of 2024.
The FDA has issued a complete response letter (CRL) to Amneal Pharmaceuticals for IPX203, an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules designed for the treatment of Parkinson disease. The reasons behind the decision were not based on efficacy or manufacturing for the agent, but rather established safety for an ingredient of the therapy. Amneal plans to work closely with the FDA to address the comments and align on the best path forward.