- Positive results suggest NLX-112 is safe and efficacious for people with Parkinson’s with levodopa-induced dyskinesias
- These challenging involuntary movements have been identified as a priority for new treatments by people with Parkinson’s
- The trial, carried out by Neurolixis, is the first funded by the Parkinson’s Virtual Biotech to complete, marking a milestone for the international drug development program
Monday, March 20 2023 - A clinical trial looking at whether an experimental drug could tackle the challenging side effect of dyskinesia has shown initial promising results. The phase 2a trial suggests that NLX-112 is safe for people with Parkinson’s, and has shown efficacy in treating dyskinesia, giving hope a new treatment could be in the pipeline.
This clinical trial, carried out by Neurolixis, has been made possible by co-funding from the Parkinson’s Virtual Biotech and The Michael J Fox Foundation for Parkinson’s Research (MJFF). The Parkinson’s Virtual Biotech, led by charity Parkinson’s UK in partnership with the Parkinson’s Foundation, is plugging the funding gap in drug development. It fast-tracks the projects with the greatest scientific potential to transform the lives of people with Parkinson’s, focusing on treatments that will meet the most pressing needs identified by the Parkinson’s community.
Over the last 5 years, the Parkinson’s Virtual Biotech has provided US based biotech company, Neurolixis, with a total of £1.57 million ($2 million) in funding to support the progress of NLX-112 through preclinical research and clinical trials. This is the first completed clinical trial made possible by the Parkinson’s Virtual Biotech, and shows the exciting potential for patient initiatives to bring forward new treatments that would not have otherwise progressed to this stage.
Neurolixis has been researching the effect of the drug NLX-112 on dyskinesia, a common side effect experienced by people with Parkinson’s who have been taking levodopa-based medications for several years. It causes involuntary movements that can affect various parts of the body, making everyday tasks impossible. The main medication available to manage dyskinesia is amantadine, which can have side effects and does not work for everyone.
NLX-112 works by targeting serotonin cells inside the brain which are believed to contribute to the development of dyskinesia, by releasing dopamine in an erratic manner. It aims to reduce dyskinesia by decreasing the amount of dopamine the cells release.
This latest phase 2a study firstly looked at how safe and well tolerated NLX-112 was in people with Parkinson’s and also how well NLX-112 improved their dyskinesia symptoms. Of the 22 participants with levodopa-induced dyskinesia who completed the treatment process, 15 participants received NLX-112 and 7 participants received a placebo.
Participants either received NLX-112 or placebo in increasing doses during the initial 4 weeks, to minimise the potential side effects. After 2 weeks at the maximum dose, they decreased the dosage over 2 weeks.
The results achieved the first objective to suggest that NLX-112 was safe and well tolerated in people with Parkinson’s. The second aim of the study was to show that NLX-112 was effective in treating dyskinesia. The results suggest participants who received NLX-112 showed significant reduction in their scores for dyskinesia, whereas those who received the placebo did not show significant reduction in their scores.
The side effects of participants who received NLX-112 were mild, confirming previous observations on the compound.
Full analysis of efficacy measures is underway and will be disclosed in further announcements.
Dr Arthur Roach, Director of Research at Parkinson’s UK, said: “We’re incredibly proud and excited by these early results from Neurolixis. They were one of the first companies that the Parkinson’s Virtual Biotech invested in, in partnership with The Michael J Fox Foundation, so to see it making positive progress just reiterates why this brave and innovative approach is right for the Parkinson’s community. It really is bringing us closer to new treatments that address the symptoms that the Parkinson’s community have told us are the most urgent and LID is one of those.
“The Parkinson’s Virtual Biotech is a global partnership between Parkinson’s UK, the largest charitable funder of Parkinson’s research in Europe, and the Parkinson’s Foundation, but it is entirely driven by the Parkinson’s community. Further studies will be necessary for regulatory approval and routine clinical use of NLX-112. But now people with Parkinson’s can have hope that a much needed new treatment for LID may be coming to them soon, and know that their support of the Parkinson’s Virtual Biotech has made this possible.”
Dr. Adrian Newman-Tancredi, CEO of Neurolixis, commented: “PD is the fastest growing neurodegenerative disease and these results suggest that NLX-112 could help mitigate the medical and societal burden caused by this disease. We are grateful to the Parkinson’s Virtual Biotech and the Michael J. Fox Foundation for financially supporting investigation of NLX-112 from preclinical studies through to this clinical trial, and their recognized expertise in the PD field provides a strong validation for the program. These positive findings further support the development of NLX-112 as a potential treatment for LID and other movement disorders with large market potential.”
Prof. Per Svenningsson, Principal Clinical Investigator, said: “We are excited about the positive results of this proof-of-concept study. 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.”
Katharina Klapper, Director of Clinical Research at The Michael J. Fox Foundation, said: “For people and families living with Parkinson’s, levodopa-induced dyskinesia can be challenging and have a significant impact on the ability to perform daily tasks. We are thankful for the contributions of study participants, the collaboration of the Parkinson’s Virtual Biotech and the efforts of the Neurolixis team. Together, we work toward our shared goal of delivering more options from the lab to the clinic – options that offer patients more opportunities for successful management of their most bothersome symptoms.”
Neurolixis, Inc:
For more information, contact Pim Cerutti, Head of US & EU Operations, Liberi Group Life Science Business Development & Strategy; pimcerutti@LiberiGroup.com
About dyskinesia
Dyskinesia is a common side effect experienced by people with Parkinson's who have been taking levodopa-based medications — a standard treatment for Parkinson's — for several years. Between 40 and 50 percent of people with Parkinson's will experience dyskinesia after just five years of taking levodopa. After ten years of taking the medication, this figure jumps to up to 80 percent. The main medication available to manage dyskinesia is amantadine, which can have challenging side effects and does not work for everyone.
About Neurolixis and NLX-112
Neurolixis is a biopharmaceutical company focused on the discovery and development of novel drugs for the treatment of central nervous system disorders with unmet medical needs and sizeable market opportunity, including movement disorders, autism spectrum disorders, depression and pain. Neurolixis is developing a platform of drug candidates based on the concept of serotonin 5-HT1A receptor ‘biased agonism’ which enables drugs to more precisely target specific brain regions controlling CNS disorders.
For more information, contact Pim Cerutti, Head of US & EU Operations, Liberi Group Life Science Business Development & Strategy; pimcerutti@LiberiGroup.com
NLX-112 (also known as befiradol) acts on the brain’s serotonin system and is a particularly specific and efficacious activator of neuronal proteins known as 5-HT1A receptors. By targeting these receptors, NLX-112 potently and efficaciously reduces dyskinesias in rodent and non-human primate models of Parkinson’s disease. NLX-112 is orally administered and has been safely tested in over 500 human subjects. Neurolixis plans to investigate the antidyskinetic activity of NLX-112 in patients with L-DOPA-induced Parkinson’s disease in a Phase 2b clinical trial.
About Parkinson’s and Parkinson’s UK
Parkinson’s is the fastest growing neurological condition in the world. Around 145,000 people in the UK have Parkinson’s. We are Parkinson's UK. Here for everyone affected by the condition. Funding research into the most promising treatments, taking us closer to a cure every day. Fighting for fair treatment and better services.
Read more facts and statistics.
Further information, advice and support is available on our website, www.parkinsons.org.uk or our free, confidential helpline on 0808 800 0303.
About the Parkinson’s Virtual Biotech
Founded by Parkinson’s UK in 2017, the Parkinson’s Virtual Biotech is now an international programme in partnership between Parkinson’s UK and the Parkinson’s Foundation in the US. We believe we’ll get to a cure faster by joining forces and collaborating. Like other biotechs, The Parkinson’s Virtual Biotech uses cutting edge biological and chemical research to come up with new treatments, and testing them in clinical trials. But it’s driven by people with Parkinson’s, not profit. Collaborative and agile, it adapts successful methods from the business world to deliver new treatments faster. The innovative approach is working. The next treatment is closer than ever, thanks to this groundbreaking global movement to deliver life-changing new treatments in years not decades.
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $1.75 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; creates a robust open- access data set and biosample library to speed scientific breakthroughs and treatment with its landmark clinical study, PPMI; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
For more information, visit https://michaeljfox.org.
Media Contact:
Kristina Magana
The Michael J. Fox Foundation
kmagana@michaeljfox.org
212-509-0995
Parkinson’s UK: Emma Smith, Senior Media and PR Officer on 020 7932 1361 or email esmith@parkinsons.org.uk