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Neurolixis today announced the publication of the results from its successful Phase 2A proof-of-concept clinical trial of NLX-112 (befiradol) in Parkinson’s disease. The findings, published in the prestigious journal 𝘔𝘰𝘷𝘦𝘮𝘦𝘯𝘵 𝘋𝘪𝘴𝘰𝘳𝘥𝘦𝘳𝘴, highlight NLX-112’s potential as a first-in-class, non-dopaminergic therapy with dual efficacy in addressing both levodopa-induced dyskinesia (LID) and motor impairment in Parkinson’s patients. The randomized, double-blind, placebo-controlled trial, supported by The Michael J. Fox Foundation and Parkinson’s UK, evaluated NLX-112 in patients with troubling LID. The study successfully met its primary endpoint of safety and tolerability, as well as its secondary endpoint of significantly reducing LID. Notably, NLX-112 also demonstrated anti-parkinsonian effects, significantly improving motor function in study participants.

See the full publication (Open Access): 𝗡𝗟𝗫-𝟭𝟭𝟮 𝗿𝗮𝗻𝗱𝗼𝗺𝗶𝘇𝗲𝗱 𝗣𝗵𝟮𝗔 𝘁𝗿𝗶𝗮𝗹: 𝘀𝗮𝗳𝗲𝘁𝘆, 𝘁𝗼𝗹𝗲𝗿𝗮𝗯𝗶𝗹𝗶𝘁𝘆, 𝗮𝗻𝘁𝗶-𝗱𝘆𝘀𝗸𝗶𝗻𝗲𝘁𝗶𝗰 𝗮𝗻𝗱 𝗮𝗻𝘁𝗶-𝗽𝗮𝗿𝗸𝗶𝗻𝘀𝗼𝗻𝗶𝗮𝗻 𝗲𝗳𝗳𝗶𝗰𝗮𝗰𝘆. 𝘔𝘰𝘷𝘦𝘮𝘦𝘯𝘵 𝘋𝘪𝘴𝘰𝘳𝘥𝘦𝘳𝘴, 2025 (https://doi.org/10.1002/mds.30175)

𝗔 𝗡𝗼𝘃𝗲𝗹 𝗠𝗲𝗰𝗵𝗮𝗻𝗶𝘀𝗺 𝗼𝗳 𝗔𝗰𝘁𝗶𝗼𝗻: Unlike current Parkinson’s treatments that target the dopamine system, NLX-112 acts on the serotonin (5-HT) system, as a highly selective full activator of 5-HT1A receptors. This unique mechanism differentiates NLX-112 from previous serotonergic drugs and is believed to underlie its dual efficacy in reducing dyskinesia and improving motor function. The promising results from this Phase 2A trial support further development of NLX-112 as a transformative therapy for movement disorders. Neurolixis is actively planning next steps in the clinical development program to advance NLX-112 toward potential regulatory approval.

𝗔𝗯𝗼𝘂𝘁 𝗣𝗮𝗿𝗸𝗶𝗻𝘀𝗼𝗻’𝘀 𝗗𝗶𝘀𝗲𝗮𝘀𝗲 𝗮𝗻𝗱 𝗥𝗲𝗹𝗮𝘁𝗲𝗱 𝗠𝗼𝘃𝗲𝗺𝗲𝗻𝘁 𝗗𝗶𝘀𝗼𝗿𝗱𝗲𝗿𝘀: Parkinson’s disease is the second most common neurodegenerative disorder, affecting over 10 million people worldwide. Levodopa, the mainstay treatment for Parkinson’s, often leads to debilitating dyskinesias (involuntary movements) after prolonged use. Up to 80% of patients develop LID within ten years of levodopa therapy. Current treatments for LID, such as amantadine, are limited by side effects and variable efficacy, underscoring the need for new therapeutic options.
As well as Parkinson’s disease, other disorders also involve dysfunction in the basal ganglia, a brain region critical for coordinating movement. Such disorders include spinocerebellar ataxia, Huntington’s disease, essential tremor and dystonia, and lead symptoms such as tremors, rigidity, and impaired motor control.

Neurolixis is making strides in developing first-in-class treatments for rare neurological disorders. The lead drug candidate, NLX-112, is Phase 2-ready and has received Orphan Drug designation for treating Spinocerebellar Ataxia. With a proven safety record in over 600 subjects and beneficial motor effects in people with Parkinson’s disease, NLX-112 could be a breakthrough in the movement disorders field.
Additionally, NLX-101 (Phase 1) has Orphan Drug designation for treating Autism Spectrum Disorders: Fragile X syndrome and Rett syndrome, two debilitating neurodevelopmental conditions with high medical need.
Both NLX-112 and NLX-101 have neuroprotective and neuroplastogenic effects, suggesting disease modifying therapeutic activity.
Neurolixis CEO, Adrian Newman-Tancredi, recently presented our rare disease pipeline at the Executive Roundtable organized by WuXi Apptec..

👉 See his 3-minute summary of the Neurolixis program: https://wxpress.wuxiapptec.com/ExecutiveRoundtables.html?id=216

NLX-112, a highly selective serotonin 5-HT1A receptor agonist, has been granted Orphan Medicinal Product (OMP) Designation by the European Commission for the treatment of Spinocerebellar Ataxia (SCA; approval #EU/3/24/2951). This decision follows a positive recommendation from the Committee for Orphan Medicinal Products (COMP). SCA is a group of rare genetic disorders, most notably Machado-Joseph Disease (SCA3), which leads to progressively worsening neurological symptoms such as clumsiness, muscle weakness, and tremors, often resulting in severe disability. The OMP designation for NLX-112 was awarded following a successful collaboration between Neurolixis and Professor Patricia Maciel's team at the University of Minho in Portugal. This partnership, funded by the U.S. Department of Defense, showed that NLX-112 significantly reduces motor dysfunction in a transgenic mouse model of SCA3.

👉 Full Press Release: https://t.ly/hUL6R 
👉 European Medicines Agency Announcement: https://t.ly/qkjCd
👉 More on Spinocerebellar Ataxias: https://t.ly/UoIFL
👉 Info on NLX-112 and Related Compounds: https://t.ly/hCB8y

United States Patent and Trademark Office (USPTO) granted a patent (US11974992B2) covering the use of NLX-101, a clinical phase drug candidate, for treatment of Fragile X syndrome (FXS). FXS is a rare genetic autism spectrum disorder which affects about 1 in 5000 individuals and is a leading cause of inherited intellectual disability. There are no approved treatments for Fragile X syndrome. Neurolixis has already shown that NLX-101 has robust activity in transgenic models of another autism spectrum disorder (Rett syndrome) and the new patent expands the company's presence in the orphan autism disorder indication space.  

Fragile X syndrome is a genetic disorder caused by a mutation in the FMR1 gene. It is the most common form of inherited intellectual disability and the leading cause of autism-like symptoms in males. Symptoms include intellectual disability, physical features, and behavioral symptoms. Genetic testing and physical examination are used for diagnosis. Supportive care and medications can help manage symptoms but there is no cure or approved treatment. The incidence of Fragile X syndrome is approximately 1 in 4,000 males and 1 in 8,000 females; in the United States 60 to 80 thousand individuals are affected.

Read the full press release.

The exciting results of the recent Phase 2A proof-of-concept clinical trial on NLX-112, a new drug candidate for the treatment of Parkinson's disease are discussed in a new interview between Arthur Roach, director of Parkinson's UK Virtual Biotech, and Adrian Newman-Tancredi, CEO of Neurolixis. NLX-112 is a first-in-kind non-dopaminergic drug for treatment of dyskinesia and other symptoms of Parkinson's disease and exhibited positive effects against dyskinesia and parkinsonism in a recent study supported by The Michael J. Fox Foundation for Parkinson's Research and Parkinson's UK research foundations. Neurolixis is proud to have joined forces with these leading research foundations. Parkinson's disease affects 9 million people worldwide and is the second most common neurodegenerative disease after Alzheimer's. 

See the interview on YouTube.

Neurolixis is proud to win the Occitanie Silver Trophées 2023 contest in the 'Innovation' category. Neurolixis was rewarded for the development of NLX-112, a new drug candidate for the treatment of Parkinson's disease which gave positive results in a Phase 2A proof-of-concept clinical trial. Parkinson's disease affects 9 million people worldwide and is the second most common neurodegenerative disease after Alzheimer's. Many thanks to La Région Occitanie / Pyrénées-Méditerranée and to The Michael J. Fox Foundation for Parkinson's Research and Parkinson's UK research foundations for their support!

NLX-112, a ground-breaking, non-dopaminergic drug, exhibited both anti-dyskinetic and anti-parkinsonian properties in a proof-of-concept Phase 2A clinical trial. The positive findings were presented in July as a ‘Hot Topics’ oral presentation at the World Parkinson’s Congress in Barcelona, Spain (see video). They will also be presented at the forthcoming Movement Disorders Society Congress in Copenhagen, Denmark, on Wednesday, August 30 (Poster #1435). The dual clinical efficacy profile of NLX-112 arises from its innovative mechanism of action. Unlike most current anti-parkinsonian medications, NLX-112 does not target the dopaminergic system, and is a highly selective, serotonin 5-HT1A receptor activator. 
See the full press release.

Neurolixis to Present Positive Proof-of-Concept Results on NLX-112 at World Parkinson’s Congress  (WPC).

NLX-112, a first-in-kind, highly selective, serotonin 5-HT1A receptor activator, recently underwent a successful Phase 2A clinical trial for treatment of levodopa-induced dyskinesia in Parkinson's disease. The detailed results from the trial, which met its primary and secondary endpoints, will be presented at the WPC in Barcelona as a poster on Wednesday July 5^th 2023 (poster board # LBP38.31) and in an oral presentation on Friday July 7^th 2023 (morning Hot Topics session). If you are attending this meeting, come and meet us!

See the full press release.

Neurolixis, Inc. today announced the positive results of its Phase 2A clinical trial with NLX-112 (befiradol), for treatment of levodopa-induced dyskinesias (LID) in Parkinson’s disease (PD).
NLX-112, a first-in-kind, highly selective serotonin 5-HT1A receptor activator, met the primary outcome of safety and tolerability and, in addition, showed statistically significant efficacy in reducing LID symptoms in Parkinson’s disease patients.

The study (ClinicalTrials.gov ID: NCT05148884) was a randomized, double-blind, placebo-controlled trial conducted at 5 centers in Sweden. 22 patients (15 on NLX-112, 7 on PBO) completed the 8-week treatment according to the protocol. Safety was good and did not differ between NLX-112 and placebo groups. Tolerability was favorable, there were no serious adverse events in the NLX-112 group. Beyond meeting the primary outcome of safety and tolerability, NLX-112 also achieved significant reductions in LID scores, whereas placebo group changes were not significant. Full analysis of efficacy measures is underway and will be disclosed in further announcements and at upcoming scientific conferences. 

"We are excited about the positive results of this proof-of-concept study," said Prof. Per Svenninsson, Principal Clinical Investigator at the Karolinska institute in Stockholm. "The findings indicate that NLX-112 can be safely administered to people with PD and alleviates their troublesome LID. If these findings are confirmed in larger clinical trials, NLX-112 could become a promising new treatment option for this indication."

The study was financially supported by grants from Parkinson's UK and The Michael J. Fox Foundation, the two largest foundations for research on Parkinson's disease  in the world.
Neurolixis plans to advance NLX-112 to a Phase 2B study to further evaluate the drug's efficacy and safety in a larger patient population.