A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Huntington's Disease (HD)

The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington's Disease (HD) in adult participants. It will also inform about the safety of pridopidine.
The main questions the study aims to answer are:
Does pridopidine slow the overall worsening of HD over 1 year? Does pridopidine slow the worsening of specific aspects of HD over 1 year, namely the clinical progression, the ability to perform daily life activities (functional capacity), the mind's ability to process information (cognition), working of the muscles (motor function), and quality of life?
Researchers will compare the drug pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works better than placebo to treat HD. During the first year of the study, participants will have the same chance to receive either pridopidine or placebo.
Participants will:
Take 1 pridopidine or placebo capsule twice daily for 12 months. Visit the clinic 6 times within 1 year for checkups and tests.
All participants who complete this 1-year placebo-controlled study period will roll over into an additional 2-year study period during which all participants will receive pridopidine treatment, including participants who had received placebo during the first year. During this additional 2-year treatment period participants will visit the clinic a total of 6 times for checkups and tests.

开始日期2026-07-01

申办/合作机构

Prilenia Therapeutics BV

[+1]

CTIS2025-524002-16-00

/ Recruiting临床3期

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants with Amyotrophic Lateral Sclerosis

开始日期2026-05-12

申办/合作机构

Ferrer Internacional SA

NCT07322003

/ Recruiting临床3期

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is:
Does pridopidine slow disease progression of ALS?
Researchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS.
Participants will:
Take pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks.
Visit the clinic once every 1-3 months for checkups and tests

开始日期2026-02-01

申办/合作机构

Prilenia Therapeutics BV

[+1]

100 项与普利多匹定相关的临床结果

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100 项与普利多匹定相关的转化医学

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100 项与普利多匹定相关的专利（医药）

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113

项与普利多匹定相关的文献（医药）

2026-04-03·Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Article

作者: Macklin, Eric A. ; Tan, Andrew M. ; Hand, Randal ; Cruz-Herranz, Andres ; Gershoni Emek, Noga ; Geva, Michal ; Paganoni, Sabrina ; Goldberg, Y. Paul ; Berry, James D. ; Cudkowicz, Merit E. ; Hayden, Michael R. ; Leitner, Melanie L. ; Shefner, Jeremy M. ; Chen, Kelly

Objectives: Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with limited treatment options. Pridopidine, a selective sigma-1 receptor agonist, was evaluated in Regimen D of the HEALEY ALS Platform Trial. Although the primary endpoint (ALS Functional Rating Scale-Revised (ALSFRS-R) total score accounting for survival at 24 weeks) was not met, a predefined subgroup analysis suggested slowed disease progression in ALS patients with definite and early disease (<18 months from onset). This report presents an exploratory analysis that further investigates pridopidine in rapidly progressing participants with definite/probable ALS and early-disease, where treatment effects may be more pronounced. Methods: The randomized, double-blind, placebo-controlled phase 2 trial assigned participants to pridopidine 45 mg bid or placebo, and placebo patients were shared across four trial regimens. The primary outcome was ALSFRS-R total score, with secondary outcomes assessing respiratory, bulbar, and speech functions. Results: Of 163 participants randomized to Regimen D, 72 met subgroup criteria (pridopidine: n = 37; shared placebo: n = 35). At week 24, pridopidine slowed ALSFRS-R total score decline (32%; Δ2.90, p = 0.03) and slowed decline of ALSFRS-R respiratory function (62%; Δ1.20, p = 0.03) and dyspnea (88%; Δ0.85, p = 0.005). ALSFRS-R-Bulbar function stabilized, with articulation and speaking rate declines reduced by 93% (Δ0.43, p = 0.0007) and 70% (Δ0.43, p = 0.002), respectively. Pridopidine was well-tolerated, with a safety profile comparable to placebo. All p values are nominal. Conclusion: Post hoc subgroup analysis suggests therapeutic benefits of pridopidine in patients that had definite/probable ALS and with early-disease progression, supporting further evaluation in a Phase 3 trial.

2026-04-01·EUROPEAN NEUROPSYCHOPHARMACOLOGY

Emerging neurobiological targets in psychiatric treatment

Review

作者: Berk, Michael ; Govinnage, Janani ; Giorlando, Frank ; De Zoysa, Agampodi Ishan ; Narayanaswamy, J C ; Dodd, Seetal

Despite significant advancements in psychopharmacology, there are inadequate treatment options for many psychiatric disorders, including major depressive disorder, bipolar disorder, schizophrenia, and anxiety disorders. This review explores emerging neurobiological targets beyond conventional monoaminergic approaches, focusing on sodium channels, Neuropeptide Y (NPY), Neurokinin 1 (NK1) receptors, P2 × 7 purinergic receptors, Sigma-1 receptors, and Orexin. Recent evidence suggests that sodium channel modulators, such as evenamide, may offer therapeutic benefits for treatment-resistant schizophrenia by stabilizing glutamatergic neurotransmission. NPY-based therapies have potential in stress-related disorders, foreshadowing rapid anxiolytic and antidepressant effects through modulation of the stress response. NK1 receptor antagonists, although inconsistent in mood disorders, show promise in addiction treatment by reducing substance cravings. The P2 × 7 receptor, a key regulator of neuroinflammation, has been implicated in mood disorders, and its pharmacological inhibition may provide neuroprotective benefits. Additionally, Sigma-1 receptor agonists, including Blarcamesine and Pridopidine, have shown neuroprotective and cognitive-enhancing properties, making them attractive candidates for psychiatric and neurodegenerative disorders. Orexin receptor antagonists, such as suvorexant and seltorexant, have potential in mood disorders and substance dependence, highlighting the broader therapeutic applications of targeting the orexinergic system. While these emerging therapeutic targets hold promise, challenges remain in translating preclinical findings into effective clinical applications. Large-scale, placebo-controlled trials are necessary to establish their efficacy and safety. The identification of biomarkers for patient stratification will be critical in the hitherto elusive goal of developing precision medicine approaches. Targeted pharmacological interventions offer a path toward more effective, well-tolerated, and potentially individualized treatment options for patients with severe mental illness.

2026-03-01·NEUROBIOLOGY OF DISEASE

Corrigendum to ‘Pridopidine stabilizes mushroom spines in mouse models of Alzheimer's disease by acting on the sigma-1 receptor’ [Neurobiology of Disease Volume 124 April 2019 Pages 489–504]

作者: Kim, Dabin ; Ryskamp, Daniel ; Hayden, Michael ; Wu, Jun ; Geva, Michal ; Rammes, Gerhard ; Bezprozvanny, Ilya ; Wu, Lili

项与普利多匹定相关的新闻（医药）

2026-10-22

·BioSpace

Prilenia and Ferrer Announce Authorization for EU Initiation of Confirmatory PRECISE-HD Study of Pridopidine in Huntington’s Disease

Recruitment is expected to start this month, rolling out to upwards of 31 sites across nine EU countries, and joining the ongoing enrollment in the US and UK, with Canada set to follow Prilenia and Ferrer will share new data analyses and clinical trial updates at the European Huntington’s Disease Network (EHDN) Clinical Research Congress NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain--(BUSINESS WIRE)-- #HD -- Prilenia Therapeutics B.V. and Ferrer i today announced that the confirmatory Phase 3 PRECISE-HD study of investigational pridopidine in Huntington's disease (HD) has received authorization to begin in participating EU Member States (clinicaltrialsregister.eu 2026-526093-16-00 , clinicaltrials.gov NCT07609108 ). Recruitment in the EU is expected to commence this month, joining currently recruiting sites in the US and UK, with Canada to follow. Designed to evaluate the efficacy and safety of pridopidine (administered as an oral capsule twice a day), and to generate data for regulatory evaluation, the global, approximately 400-participant, randomized, double-blind, placebo-controlled PRECISE-HD study will assess the clinical effect of the investigational treatment on disease progression, functional capacity, motor function, cognition, speech and quality of life. “The initiation of PRECISE-HD in Europe marks a milestone for the Huntington’s disease research community. This confirmatory Phase 3 study has been designed to build on learnings from previous research and to rigorously evaluate the efficacy and safety of pridopidine in people living with HD. As investigators, we look forward to working with participating centers and the HD community to generate robust evidence that can help us better understand the potential role of the study treatment in HD,” said Dr. Ralf Reilmann, Principal Investigator and member of the PRECISE-HD Steering Committee . “For people living with Huntington’s disease and their families, every step toward a better understanding of the disease and potential treatment options matters. We welcome the involvement of European centers in PRECISE-HD and the continued focus on outcomes that reflect the realities and priorities of people living with HD and their families,” s aid Astri Arnesen, President of the European Huntington Association . EHDN 2026 Clinical Research Congress Prilenia and Ferrer will participate at the European Huntington's Disease Network (EHDN) Clinical Research Congress 2026, taking place October 22–24, 2026, in Kraków, Poland, with Prilenia’s Chief Executive Officer, Dr. Michael R. Hayden, giving an oral presentation on clinical trial design and determination of clinical meaningfulness, in addition to four poster presentations, describing clinically meaningful change in early manifest HD, placebo response in the PROOF-HD clinical trial, and the designs of the PREVAiLS and PRECISE-HD studies of pridopidine in ALS and HD respectively. [This press release is intended for informational purposes only. Pridopidine is an investigational medicine and is not approved for commercial use by any regulatory authority. Its safety and efficacy have not been established. Information contained herein does not constitute medical advice. Patients should consult their healthcare provider for guidance regarding diagnosis or treatment options. Local regulations may vary; this release is not intended to promote or advertise any product.] About HD Huntington’s disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in functional, motor, cognitive and behavioral symptoms, and ultimately leads to death. HD is caused by a mutation in the huntingtin (HTT) gene ii , and each child of a parent with HD has a 50 percent chance of developing the disease iii . Across the world an estimated 100,000 people have HD iv , with an additional 300,000 people at risk of developing HD v,vi . It is usually diagnosed between the ages of 35 and 55 vii viii , although HD can occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 10 to 30 ix years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death. The only currently available treatments for HD focus on symptomatic relief and palliative care, with no approved therapies shown to impact measures of overall disease progression. About pridopidine Pridopidine is an investigational highly selective, oral sigma-1 receptor (S1R) agonist. S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS) x . In clinical studies to date, pridopidine (taken as an oral capsule twice a day) has shown a generally favorable safety and tolerability profile, with data from more than 1,600 people (mostly from HD studies), some of whom have received active treatment for up to seven years xi . Pridopidine is currently in late-stage clinical development for HD and ALS. It has Orphan Drug Designation in HD and ALS in the US and EU, and FDA Fast Track designation in the US for the treatment of HD. These designations do not imply that pridopidine has been approved or that safety and efficacy have been established. About the PRECISE-HD Study PRECISE-HD (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease) is a confirmatory, global, 400-participant, randomized, double-blind, placebo-controlled study designed to further evaluate the efficacy and safety of pridopidine, an investigational medicinal product, in people living with Huntington’s disease (HD), from early to mid-stage disease (TFC 7-13). Focused on generating data to further support regulatory evaluation, the study will assess the clinical effect of pridopidine on disease progression, functional capacity, motor function, cognition, speech and quality of life. It incorporates learnings from prior research, advice and input from the HD patient and research community, and regulatory authorities. PRECISE-HD comprises two consecutive stages: a 52-week placebo-controlled stage, with participants randomized to receive either pridopidine or placebo on a 1:1 basis, followed by a 104-week open-label extension stage in which all eligible participants will receive pridopidine. The primary endpoint is the change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale (cUHDRS) score. PRECISE-HD will be undertaken at up to 75 sites globally, including sites in the United States, European Union, United Kingdom and Canada. Further details are available at clinicaltrials.gov ( NCT07609108 ) and clinicaltrialsregister.eu ( 2026-526093-16-00 ). About Prilenia Prilenia is a private biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Our mission is simple but urgent: to develop and provide sustainable access to transformative medicines for people affected by devastating neurodegenerative diseases. Prilenia is incorporated in the Netherlands and backed by leading life sciences investors. Prilenia and Ferrer are partners in co-development and commercialisation of pridopidine. For more information, please visit , and connect with us on LinkedIn , X or Instagram . About Ferrer At Ferrer, we use business to fight for social justice. We have long been a company that wants to do things differently; instead of maximizing shareholder returns, we reinvest much of our profit in initiatives that give back to society. Back where it belongs. We go beyond compliance and are guided by the highest standards of sustainability, ethics and integrity. As such, since 2022, we are a B Corp. Founded in Barcelona in 1959, Ferrer offers transformative solutions for life-threatening diseases in more than one hundred countries. In line with our purpose, we have an increasing focus on pulmonary vascular and interstitial lung diseases and rare neurological disorders. Our 1,800-strong team is driven by a clear conviction: our business is not an end in itself, but a way to change lives. We are Ferrer. Ferrer for good. i In April 2025, Prilenia and Ferrer signed a commercialization and co-development agreement for pridopidine. ii Eddings CR, Arbez N, Akimov S, Geva M, Hayden MR, Ross CA. Pridopidine protects neurons from mutant-huntingtin toxicity via the sigma-1 receptor. Neurobiol Dis. 2019 Sep;129:118-129. doi: 10.1016/j.nbd.2019.05.009. Epub 2019 May 17. PMID: 31108174; PMCID: PMC6996243. iii Myers RH. Huntington's disease genetics. NeuroRx. 2004 Apr;1(2):255-62. doi: 10.1602/neurorx.1.2.255. PMID: 15717026; PMCID: PMC534940.) iv Pringsheim T, Wiltshire K, Day L, Dykeman J, Steeves T, Jette N. The incidence and prevalence of Huntington's disease: a systematic review and meta-analysis. Mov Disord. 2012 Aug;27(9):1083-91. doi: 10.1002/mds.25075. Epub 2012 Jun 12. PMID: 22692795. v Medina et al., Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022 Dec;37(12):2327-2335. vi Jiang, A., Handley, R. R., Lehnert, K., & Snell, R. G. (2023). From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington’s Disease Research. International Journal of Molecular Sciences, 24(16), 13021. vii EHDN. About Huntington’s Disease. April 2024. Available at: (accessed April 2025). viii Petracca M, et al. Clinical and genetic characteristics of late-onset Huntington's disease in a large European cohort. Eur J Neurol. 2022;29:1940–51. ix Mayo Clinic. Huntington’s disease. Available at: x Lachance V, Bélanger S-M, Hay C, Le Corvec V, Banouvong V, Lapalme M, Tarmoun K, Beaucaire G, Lussier MP, Kourrich S. Overview of Sigma-1R Subcellular Specific Biological Functions and Role in Neuroprotection. International Journal of Molecular Sciences. 2023; 24(3):1971. xi Goldberg YP, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan AM, Schuring H, Geva M, Hayden MR. The Safety Pro Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington's Disease. CNS Drugs. 2025 May;39(5):485-498. doi: 10.1007/s40263-025-01171-x. Epub 2025 Mar 7. PMID: 40055280; PMCID: PMC11982116. Contacts Media Contacts: Prilenia Contact Rob Cohen, Head of Corporate Communications rob.cohen@prilenia.com Ferrer Contact Alba Soler, Director of Communication asolerc@ferrer.com

临床3期孤儿药快速通道临床结果

2026-10-22

·BioSpace

Ferrer and Prilenia Announce Authorization for EU Initiation of Confirmatory PRECISE-HD Study of Pridopidine in Huntington’s Disease

BARCELONA, Spain--(BUSINESS WIRE)-- Ferrer i and Prilenia Therapeutics B.V. today announced that the confirmatory Phase 3 PRECISE-HD study of investigational pridopidine in Huntington's disease (HD) has received authorization to begin in participating EU Member States (EU CT number: 2026-526093-16-00, NCT07609108 ). Recruitment in the EU is expected to commence this month, joining currently recruiting sites in the US and UK, with Canada to follow. Designed to evaluate the efficacy and safety of pridopidine (administered as an oral capsule twice a day), and to generate data for regulatory evaluation, the global, approximately 400-participant, randomized, double-blind, placebo-controlled PRECISE-HD study will assess the clinical effect of the investigational treatment on disease progression, functional capacity, motor function, cognition, speech and quality of life. “The initiation of PRECISE-HD in Europe marks a milestone for the Huntington’s disease research community. This confirmatory Phase 3 study has been designed to build on learnings from previous research and to rigorously evaluate the efficacy and safety of pridopidine in people living with HD. As investigators, we look forward to working with participating centers and the HD community to generate robust evidence that can help us better understand the potential role of the study treatment in HD,” said Dr. Ralf Reilmann , Principal Investigator and member of the PRECISE-HD Steering Committee. “For people living with Huntington’s disease and their families, every step toward a better understanding of the disease and potential treatment options matters. We welcome the involvement of European centers in PRECISE-HD and the continued focus on outcomes that reflect the realities and priorities of people living with HD and their families,” said Astri Arnesen , President of the European Huntington Association. “At Ferrer, putting our purpose into practice also means taking on complex scientific challenges, while recognizing the uncertainty inherent in research. The authorization to initiate PRECISE-HD in participating EU Member States is a further step in our work with Prilenia. We approach that work with a clear sense of responsibility: to conduct research with integrity and let the evidence lead the way,” said Oscar Pérez , Chief Scientific Officer at Ferrer. About HD Huntington’s disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in functional, motor, cognitive and behavioral symptoms, and ultimately leads to death. HD is caused by a mutation in the huntingtin (HTT) gene ii , and each child of a parent with HD has a 50 percent chance of developing the disease iii . Across the world an estimated 100,000 people have HD iv , with an additional 300,000 people at risk of developing HD v,vi . It is usually diagnosed between the ages of 35 and 55 vii viii , although HD can occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 10 to 30 ix years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death. The only currently available treatments for HD focus on symptomatic relief and palliative care, with no approved therapies shown to impact measures of overall disease progression. About pridopidine Pridopidine is an investigational highly selective, oral sigma-1 receptor (S1R) agonist. S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS) x . In clinical studies to date, pridopidine (taken as an oral capsule twice a day) has shown a generally favorable safety and tolerability profile, with data from more than 1,600 people (mostly from HD studies), some of whom have received active treatment for up to seven years xi . Pridopidine is currently in late-stage clinical development for HD and ALS. It has Orphan Drug Designation in HD and ALS in the US and EU, and FDA Fast Track designation in the US for the treatment of HD. These designations do not imply that pridopidine has been approved or that safety and efficacy have been established. About the PRECISE-HD Study PRECISE-HD (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease) is a confirmatory, global, 400-participant, randomized, double-blind, placebo-controlled study designed to further evaluate the efficacy and safety of pridopidine, an investigational medicinal product, in people living with Huntington’s disease (HD), from early to mid-stage disease (TFC 7-13). Focused on generating data to further support regulatory evaluation, the study will assess the clinical effect of pridopidine on disease progression, functional capacity, motor function, cognition, speech and quality of life. It incorporates learnings from prior research, advice and input from the HD patient and research community, and regulatory authorities. PRECISE-HD comprises two consecutive stages: a 52-week placebo-controlled stage, with participants randomized to receive either pridopidine or placebo on a 1:1 basis, followed by a 104-week open-label extension stage in which all eligible participants will receive pridopidine. The primary endpoint is the change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale (cUHDRS) score. PRECISE-HD will be undertaken at up to 75 sites globally, including sites in the United States, European Union, United Kingdom and Canada. Further details are available at ( NCT07609108 ), (EU CT number: 2026-526093-16-00). About Ferrer At Ferrer, we use business to fight for social justice. We have long been a company that wants to do things differently; instead of maximizing shareholder returns, we reinvest much of our profit in initiatives that give back to society. Back where it belongs. We go beyond compliance and are guided by the highest standards of sustainability, ethics and integrity. As such, since 2022, we are a B Corp. Founded in Barcelona in 1959, Ferrer offers transformative solutions for life-threatening diseases in more than one hundred countries. In line with our purpose, we have an increasing focus on rare pulmonary and neurological diseases. Our 1,800-strong team is driven by a clear conviction: our business is not an end in itself, but a way to change lives. We are Ferrer. Ferrer for good. i In April 2025, Prilenia and Ferrer signed a commercialization and co-development agreement for pridopidine. ii Eddings CR, Arbez N, Akimov S, Geva M, Hayden MR, Ross CA. Pridopidine protects neurons from mutant-huntingtin toxicity via the sigma-1 receptor. Neurobiol Dis. 2019 Sep;129:118-129. doi: 10.1016/j.nbd.2019.05.009. Epub 2019 May 17. PMID: 31108174; PMCID: PMC6996243. iii Myers RH. Huntington's disease genetics. NeuroRx. 2004 Apr;1(2):255-62. doi: 10.1602/neurorx.1.2.255. PMID: 15717026; PMCID: PMC534940.) iv Pringsheim T, Wiltshire K, Day L, Dykeman J, Steeves T, Jette N. The incidence and prevalence of Huntington's disease: a systematic review and meta-analysis. Mov Disord. 2012 Aug;27(9):1083-91. doi: 10.1002/mds.25075. Epub 2012 Jun 12. PMID: 22692795. v Medina et al., Prevalence and Incidence of Huntington's Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022 Dec;37(12):2327-2335. vi Jiang, A., Handley, R. R., Lehnert, K., & Snell, R. G. (2023). From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington’s Disease Research. International Journal of Molecular Sciences, 24(16), 13021. vii EHDN. About Huntington’s Disease. April 2024. Available at: (accessed April 2025). viii Petracca M, et al. Clinical and genetic characteristics of late-onset Huntington's disease in a large European cohort. Eur J Neurol. 2022;29:1940–51. ix Mayo Clinic. Huntington’s disease. Available at: x Lachance V, Bélanger S-M, Hay C, Le Corvec V, Banouvong V, Lapalme M, Tarmoun K, Beaucaire G, Lussier MP, Kourrich S. Overview of Sigma-1R Subcellular Specific Biological Functions and Role in Neuroprotection. International Journal of Molecular Sciences. 2023; 24(3):1971. xi Goldberg YP, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan AM, Schuring H, Geva M, Hayden MR. The Safety Pro Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington's Disease. CNS Drugs. 2025 May;39(5):485-498. doi: 10.1007/s40263-025-01171-x. Epub 2025 Mar 7. PMID: 40055280; PMCID: PMC11982116. Contacts Ferrer media: Alba Soler, Director of Communication asolerc@ferrer.com Paula Bonet pbonetg@ferrer.com

临床3期孤儿药快速通道

2026-09-08

·BioSpace

Prilenia Appoints Frederick (Rick) Munschauer, MD, FAAN, as Chief Medical Officer

NAARDEN, Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)-- #ALS -- Prilenia Therapeutics B.V. , a biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD) and Amyotrophic Lateral Sclerosis (ALS), today announced the appointment of Frederick (Rick) E. Munschauer III, MD, MSc, FAAN, as Chief Medical Officer (CMO). “Rick is a renowned neurologist and seasoned pharmaceutical executive who has spent his entire career at the front line of neurodegenerative disease – firstly, as an academic clinician and researcher, and then as a leader behind some of the most consequential neurology drug development programs of the past three decades,” said Dr. Michael R. Hayden, Chief Executive Officer of Prilenia Therapeutics . “He has designed and delivered trials from first-in-human through to approval and beyond, and he has done it repeatedly and successfully. As we execute PRECISE-HD and PREVAiLS, our two registrational Phase 3 studies in HD and ALS respectively, that combination of clinical judgement, regulatory experience and operational discipline will bring significant value to Prilenia, and to patients, and I am delighted to welcome Rick to our leadership team.” Rick has served as CMO at three biopharmaceutical companies, most recently at Emalex Biosciences. There he led Clinical Development, Clinical Operations, Pharmacovigilance, Biostatistics and Medical Affairs, leading development of ecopipam to FDA NDA submission for pediatric Tourette syndrome in June 2026. Previously he was CMO at MedDay Pharmaceuticals and at Marathon Pharmaceuticals, where he led the pre- and post-approval strategy in multiple sclerosis (MS) and Duchenne muscular dystrophy, acting as medical lead in all FDA interactions. Previously, whilst at Biogen, Rick served as Vice President and Chief Medical Advisor and as Vice President, Global Head of Medical Affairs, leading a global organization of more than 250 people and directing the integrated medical launches of important MS therapies, Tecfidera and Plegridy. He also held senior US medical affairs leadership roles at EMD Serono. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); His academic career spans more than two decades at the State University of New York at Buffalo, where he was the Irvin and Rosemary Smith Professor and Chair of Neurology and Chief of the Jacobs Neurological Institute. There he held the original IND for interferon beta-1a in multiple sclerosis and executed the NIH-funded pivotal study that was the first to demonstrate a slowing of disability progression — work that led to the approval of Avonex. He has served as Principal or Co-Principal Investigator on more than 50 Phase 1–4 clinical trials in multiple sclerosis and cerebrovascular disease and has authored more than 90 peer-reviewed publications. “Prilenia is currently tackling two devastatingly intractable diseases, in HD and ALS, where the unmet need is stark, and where we now have a possibility of changing the course of illness,” said Rick Munschauer, MD, Prilenia’s Chief Medical Officer . “What drew me here is the clarity of the mission and the pridopidine story, which has already garnered compelling data. My focus will be on execution — running our pivotal HD and ALS studies to the highest standard, generating the evidence regulators need, and staying close to the clinical and patient communities whose participation makes all this possible.” Rick holds an MD, CM from McGill University, an MSc in Mechanical Engineering from the Massachusetts Institute of Technology, and a BS in Electrical and Mechanical Engineering from Duke University. He completed his residency in neurology at The Johns Hopkins Hospital and a fellowship in neurologic and neurosurgical intensive care at The National Hospital for Nervous Diseases, London. He is a Fellow of the American Academy of Neurology and has served on the National Board of Directors and the National Medical Advisory Board of the National Multiple Sclerosis Society. About Prilenia Prilenia is a private biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by HD, ALS and other neurodegenerative disorders. Our mission is simple but urgent: to develop and provide sustainable access to transformative medicines for people affected by devastating neurodegenerative diseases. ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); The company is incorporated in the Netherlands and backed by leading life sciences investors. For more information, please visit , and connect with us on LinkedIn or X (Twitter) . For a copy of this release, visit Prilenia’s website at . Contacts Prilenia Rob Cohen, Head of Corporate Communications, Prilenia rob.cohen@prilenia.com

高管变更临床3期临床1期

100 项与普利多匹定相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09953	普利多匹定	N07XX26	DB11947

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
亨廷顿舞蹈病	申请上市	欧盟	Prilenia Therapeutics BV	2024-09-03
肌萎缩侧索硬化	临床3期	美国	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	比利时	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	加拿大	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	法国	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	德国	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	爱尔兰	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	以色列	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	意大利	Prilenia Therapeutics BV	2026-02-01
肌萎缩侧索硬化	临床3期	荷兰	Prilenia Therapeutics BV	2026-02-01

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适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04556656 (PROOF-HD, Company_Website) 人工标引	临床3期	亨廷顿舞蹈病	-	Pridopidine	糧構醖築餘窪憲獵築繭(齋齋憲願鏇齋顧淵糧糧) = Compared to natural history cohort from ENROLL-HD, data at 2 years shows slowing of disease progression by 65% for TFC (p = <0.0001) for pridopidine-treated patients off ADMs. Similar results were seen compared to history cohort TRACK-HD. 觸構遞選鏇簾鬱構齋鹹 (築獵簾顧壓製網鏇糧齋 ) 更多	积极	2025-10-10
NCT04556656 (PROOF-HD, Pubmed \| Nat Med) 人工标引	临床3期	亨廷顿舞蹈病	-	Pridopidine (45 mg)	廠齋積範廠淵艱襯蓋蓋(齋膚窪觸糧鬱鬱製襯鑰) = Sensitivity analysis in a subgroup of participants not treated with antidopaminergic medications at any time demonstrated a consistent pattern favoring pridopidine across multiple measures, including TFC. 簾齋顧糧繭顧壓糧醖簾 (選夢繭構繭廠獵醖遞膚 ) 未达到更多	积极	2025-09-05
				Placebo
NCT04556656 (PROOF-HD, EAN2025)	临床3期	亨廷顿舞蹈病	245	Participants on ADMs	齋積積夢觸獵憲繭夢窪(艱鏇淵範衊鹹齋築觸膚) = 繭淵齋觸獵衊鹽餘鬱衊憲襯範衊築範壓簾衊獵 (糧鹹築網餘廠網壓範鹹 )	不佳	2025-06-21
				Participants off ADMs	齋積積夢觸獵憲繭夢窪(艱鏇淵範衊鹹齋築觸膚) = 鑰夢製襯製襯糧窪簾艱憲襯範衊築範壓簾衊獵 (糧鹹築網餘廠網壓範鹹 )
NCT04297683 \| NCT04615923 (HEALEY ALS Platform Trial, Pubmed \| JAMA)	临床2/3期	肌萎缩侧索硬化 sigma-1 (σ1) receptor	163	Pridopidine 45 mg twice daily	夢製襯築製餘窪憲蓋壓(築壓繭鏇觸鹽構遞醖築): DRR = 0.99 (95% CI, 0.8 ~ 1.21) 更多	不佳	2025-04-01
				Placebo
NCT04556656 (PROOF-HD, CTgov)	临床3期	亨廷顿舞蹈病	499	Pridopidine (Pridopidine)	選齋構憲願顧簾網壓選(鹹製繭獵顧鬱鹹鹹衊蓋) = 憲鬱鹹廠憲構觸構願餘繭願窪艱簾顧鬱淵簾艱 (鏇糧繭壓選構襯願膚鹹, 0.119) 更多	-	2025-03-12
				Placebo (Placebo)	選齋構憲願顧簾網壓選(鹹製繭獵顧鬱鹹鹹衊蓋) = 壓襯繭淵襯壓簾襯鑰淵繭願窪艱簾顧鬱淵簾艱 (鏇糧繭壓選構襯願膚鹹, 0.119) 更多
NCT00724048 (HART, CTgov)	临床2期	亨廷顿舞蹈病	227	Placebo (Placebo)	壓襯築衊蓋願膚顧構鑰(醖餘網襯齋構餘獵選鏇) = 鏇齋廠製鏇選繭鏇築壓醖築鏇衊鏇簾鑰顧築齋 (鏇蓋鬱艱窪築繭膚遞簾, 3.72) 更多	-	2023-08-31
				ACR16 (ACR16 10 mg BID)	壓襯築衊蓋願膚顧構鑰(醖餘網襯齋構餘獵選鏇) = 遞醖製鏇襯築積糧淵獵醖築鏇衊鏇簾鑰顧築齋 (鏇蓋鬱艱窪築繭膚遞簾, 3.33) 更多
NCT04615923 (HEALEY ALS Platform Trial, CTgov)	临床2/3期	肌萎缩侧索硬化	163	Pridopidine (Pridopidine)	選艱襯鹽襯壓顧願顧範(鑰築窪觸鹽獵鹹鹹窪膚) = 鏇積窪窪範憲鑰壓遞糧鹹廠膚範膚顧憲製壓襯 (廠網醖夢築顧鑰鏇襯網, 0.077) 更多	-	2023-08-23
				Matching Placebo (Matching Placebo)	選艱襯鹽襯壓顧願顧範(鑰築窪觸鹽獵鹹鹹窪膚) = 糧襯繭膚餘膚簾蓋鑰鑰鹹廠膚範膚顧憲製壓襯 (廠網醖夢築顧鑰鏇襯網, 0.070) 更多
NCT04615923 (HEALEY ALS Platform, NEWS) 人工标引	临床2期	肌萎缩侧索硬化 Nfl	-	Pridopidine	淵憲選艱衊簾獵觸範觸(選製壓願膚築獵簾淵鏇) = 鹹鹽鬱窪艱醖繭醖鏇構餘鹽襯夢壓鹹醖壓齋簾 (夢鏇膚醖艱蓋網衊鹽廠 )	积极	2023-02-23
				Placebo	淵憲選艱衊簾獵觸範觸(選製壓願膚築獵簾淵鏇) = 衊選構願廠衊壓築顧壓餘鹽襯夢壓鹹醖壓齋簾 (夢鏇膚醖艱蓋網衊鹽廠 )
NCT00724048 \| EUCTR2007-004988-22-AT \| NCT02006472 \| NCT00665223 \| EUCTR2013-001888-23-AT (PRIDE-HD, Pubmed)	临床2期	亨廷顿舞蹈病	402	Pridopidine 45 mg bid	簾顧網願願網鹹齋淵壓(糧鏇窪積膚鹹淵網艱鹹) = 鹹選獵鏇艱壓糧襯願淵範選顧餘簾積膚選範鏇 (願淵簾製網齋鹹鏇襯觸, 8.0)	-	2023-02-22
NCT03922711 (CTgov)	临床2期	药物性运动障碍 \| 帕金森病	23	Placebo	淵鹹鏇衊鹹鬱製觸築窪(積鏇築襯製選夢網觸蓋) = 齋遞蓋襯夢築鏇遞鹽壓鬱獵遞淵鑰願鹽壓醖繭 (餘顧範齋製製積鏇壓範, 6.44) 更多	-	2022-07-01