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The orphan medicinal product designation given by the European Commission was based on findings produced in research funded by the United States Department of Defense.
Patricia Maciel, PhD
(Credit: ICVS)
Following a recent positive recommendation from the Committee for Orphan Medicinal Products, the European Commission has granted NLX-112 (Neurolixis), a highly selective serotonin 5-HT1A receptor agonist, orphan medicinal product designation for the treatment of spinocerebellar ataxia (SCA).1
The decision behind this designation was based on a successful partnership between Neurolixis and Patricia Maciel’s, PhD, team at the University of Minho in Portugal. This collaboration, which was funded by the United States Department of Defense, showed that NLX-112 significantly reduced motor dysfunction in preclinical models of SCA type 3 (SCA3), also known as Machado-Joseph Disease.
“Serotonin plays a crucial role in motor control, and our research shows that NLX-112 can mitigate the motor impairments seen in transgenic models of SCA3. This finding suggests that NLX-112 has the potential to improve the lives of patients suffering from this devastating disorder,” Maciel, an associate professor at the University of Minho School of Medicine and director of the MSc program in Health Sciences and Senior Researcher at the Life and Health Sciences Research Institute, said in a statement.1
The orphan medicinal product designation offers many significant benefits for NLX-112, including 10 years of market exclusivity after marketing approval by the European Union. An additional 2 years could be granted to Neurolixis if it successfully complies with a pediatric assessment plan which the company noted intends to submit.
“This [orphan medicinal product] designation is a critical milestone for Neurolixis as we expand our drug development programs targeting rare neurological disorders. With NLX-112, we are making strides in the field of movement disorders, including Spinocerebellar Ataxia, and this designation further solidifies our commitment to advancing treatments for rare diseases,” Adrian Newman-Tancredi, PhD, DSc, chief executive officer at Neurolixis, said in a statement.1
SCA, a group of rare genetic disorders, causes progressively debilitating neurological symptoms in patients including clumsiness, muscle weakness, and tremors. Patients with SCA, most notably SCA3, can ultimately experience severe disability if not given effective treatment for these symptoms. SCA3 is defined as a rare, inherited motor disorder characterized by symptoms such as difficulty with speech and swallowing, a staggering gait and in some cases, dystonia or symptoms resembling PD. SCA3, a condition with no cure or approved treatment currently, typically starts in adolescence and can worsen over time to eventually lead to paralysis.
NLX-112, known also as befiradol, is an oral drug that selectively activates serotonin 5 HT1A receptors, which displayed significant efficacy in preclinical models of motor disorders. To date, the treatment has been tested in over 600 patients with various nonmotor indications, which further supports its potential in neurological treatments. Previously, in a phase 2a trial (NCT05148884) of Parkinson disease (PD), the compound revealed positive findings among patients who presented with troubling levodopa-induced dyskinesia (LID).
Presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders, held August 27-31, in Copenhagen, Denmark, NLX-112 met the primary end point of safety and tolerability, and also met its secondary end point of significantly reducing LID.2 Additionally, NLX-112 demonstrated robust antiparkinsonian efficacy, significantly reducing motor impairment in the study participants. Overall, the data point to a promising dual-acting activity of NLX-112, combining both anti-LID and antiparkinsonian properties.
The study was a randomized, double-blind, placebo-controlled trial performed at 5 centers in Sweden. The company noted that 22 patients (NLX-112, n = 15; placebo, n = 7) completed the 8-week treatment according to the protocol. Neurolixis also reported that safety results were positive and did not differ between the NLX-112 and placebo groups. In addition, the company noted that tolerability was favorable, with no serious adverse events reported in the NLX-112 group.3
Besides NLX-112, the company is advancing NLX-101 in a phase 1 study for treating rare autism spectrum disorders, such as Fragile X and Rett syndromes. Neurolixis is also developing NLX-204, a preclinical candidate that shows promise as a rapid-acting antidepressant and analgesic, using a non-opioid mechanism.
