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.

Start Date01 Jul 2026

Sponsor / Collaborator

Prilenia Therapeutics BV

[+1]

CTIS2025-524002-16-00

/ RecruitingPhase 3

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

Start Date12 May 2026

Sponsor / Collaborator

Ferrer Internacional SA

NCT07322003

/ RecruitingPhase 3

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

Start Date01 Feb 2026

Sponsor / Collaborator

Prilenia Therapeutics BV

[+1]

100 Clinical Results associated with Pridopidine

100 Translational Medicine associated with Pridopidine

100 Patents (Medical) associated with Pridopidine

107

Literatures (Medical) associated with Pridopidine

01 Jun 2026·EUROPEAN JOURNAL OF PHARMACOLOGY

Beyond motor symptoms: efficacy of pharmacotherapies on motor, functional, and cognitive domains in Huntington's disease - a systematic review

Review

Author: Teixeira, Amanda Gomes ; Wiprich, Melissa Talita ; Colonetti, Tamy ; Endler, Ana Thereza ; Bonan, Carla Denise ; Rosa, Maria Inês da ; Pereira, Wuesllen Dondossola ; Rico, Eduardo Pacheco ; Lodetti, Guilherme ; Pacagnan, Lucas de Oliveira ; Redivo, Henrique

INTRODUCTION:

Huntington's disease (HD) is a progressive neurodegenerative disorder characterized by motor, neuropsychiatric, cognitive, and peripheral alterations impairing functional capacity and quality of life (QoL). Pharmacotherapies, including dopaminergic and glutamatergic drugs, primarily target motor symptoms, while cholinergic modulators address cognitive decline. However, the literature lacks comprehensive reviews evaluating the combination of these three drug classes across a broad spectrum of clinical outcomes, extending beyond the total motor score (TMS).

OBJECTIVE:

This systematic review aimed to assess the efficacy of dopaminergic, glutamatergic, and cholinergic drugs on motor, functional, cognitive decline, and QoL in HD patients.

METHODOLOGY:

Following PRISMA guidelines, searches were performed in MEDLINE, Embase, Scopus, Web of Science, and the Cochrane Library until October 2025. Randomized clinical trials evaluating these three classes of drugs in adults with genetically confirmed HD were included. Primary outcomes were Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS), Total Functional Capacity (TFC), and cognitive performance. Secondary outcomes included the Independence Scale (IS) and QoL. Risk of bias was assessed using the Cochrane RoB 2 tool. Due to substantial heterogeneity, results were synthesized descriptively.

RESULTS:

From 32,746 studies, 15 were included in the final analysis. Findings showed that UHDRS TMS was improved with dopaminergics (tetrabenazine, deutetrabenazine, and pridopidine) and glutamatergic (riluzole) drugs. Only pridopidine treatment exhibited efficacy on UHDRS TFC, subsequently improving UHDRS IS. For QoL, deutetrabenazine treatment proved effective. In the UHDRS cognitive domain, tetrabenazine improved performance in the Stroop Color and Word test, while rivastigmine (cholinergic) improved verbal fluency.

CONCLUSION:

Dopaminergic, glutamatergic, and cholinergic drugs exhibit distinct efficacy profiles across various motor and non-motor UHDRS components. This positions them as therapeutic options for attenuating both the motor and non-motor symptoms in HD.

03 Apr 2026·Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Article

Author: 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.

01 Apr 2026·EUROPEAN NEUROPSYCHOPHARMACOLOGY

Emerging neurobiological targets in psychiatric treatment

Review

Author: 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.

News (Medical) associated with Pridopidine

07 Jul 2026

·www.biospace.com

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

PRECISE-HD (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease (HD)) is designed to further evaluate the efficacy and safety of pridopidine, an oral investigational drug, on disease progression, functional capacity, motor function, cognition, speech and Quality of life Focused on generating the data to further support regulatory evaluation, PRECISE-HD will be undertaken in up to 75 sites globally, including the US, EU, UK and Canada. Recruitment has now commenced in the US, with other countries expected to follow later in the year NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain--(BUSINESS WIRE)-- #HD -- Prilenia Therapeutics B.V. and Ferrer i today announced initiation of the confirmatory PRECISE-HD study (Pridopidine Phase 3 Study to Establish Clinical Impact and Safety in Huntington’s Disease (HD) ( NCT07609108 )). Recruitment has now commenced in the US, with other countries expected to follow on a rolling basis later in the year. Focused on further evaluating the efficacy and safety of pridopidine, an investigational medicinal product (taken as an oral capsule twice a day), and the generation of data to support registration, the confirmatory 400-participant randomized, double-blind, placebo-controlled PRECISE-HD study will assess the clinical impact of pridopidine on disease progression, functional capacity, motor function, cognition, speech and quality of life (QoL). Incorporating learnings from prior research and advice and input from the HD patient and research community and regulatory authorities, the study will focus on people living with HD, from early to mid-stage disease (defined as Total Functional Capacity (TFC) score of 7-13), a Total Motor Score (TMS) ≥20 and Independence Scale score ≤ 90%, meaning people who have experienced some motor changes or impact on independent functioning. This will allow for appropriate assessment of any potential effect of the therapy on disease progression in comparison to placebo. PRECISE-HD will be undertaken in up to 75 sites globally, including the US, EU, UK and Canada. Katie Jackson, President/CEO of Help 4 HD International, remarked : "For families affected by Huntington's disease, each day is a race against time. There are currently no available treatments to slow the progression of HD, which gradually diminishes every bit of quality of life. Despite these challenges, the therapeutic needs of the HD community remain significantly underserved. Every study aimed at changing this reality is critically important. It is essential that this study be completed promptly so its results—and their potential impact on individuals with HD and their families, who have already waited far too long for progress—can be fully evaluated.” Victor Sung, M.D. Director of the Division of Movement Disorders in the University of Alabama, Director of the UAB Huntington’s Disease Clinic, Director of the HDSA Center of Excellence and PRECISE-HD Steering Committee Member said : “Across prior studies, pridopidine has shown meaningful clinical effects in specific circumstances ii , and this study has been carefully designed to confirm those effects, and uniquely incorporates prior learnings and novel elements specifically designed to provide treatment effect clarity.” PRECISE-HD is comprised of two consecutive stages: A 52-week placebo-controlled stage with participants randomized to receive either pridopidine or placebo on a 1:1 basis. This will be followed by a 104-week open-label extension stage, in which all eligible participants will receive pridopidine, allowing researchers to assess any potential effect on disease progression for up to three years in total, compared to matched external control cohorts from longitudinal, multinational observational studies iii . PRECISE-HD will evaluate a range of HD-related outcomes, with the primary endpoint being the change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale (cUHDRS) score. Further details on the PRECISE-HD study can be found at: NCT07609108 . A PRECISE-HD study website ( ) will also be made available shortly. ENDS [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 pridopidine Pridopidine (taken as an oral capsule twice a day) 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) iv . In clinical studies to date, pridopidine 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 v . In addition to HD, pridopidine is in late-stage clinical development for ALS. The currently recruiting pivotal Phase 3 PREVAiLS trial in ALS is guided by findings from the Phase 2 HEALEY ALS Platform trial in patients with rapidly progressing disease early in their disease (defined as definite or probable ALS by El Escorial Criteria (EEC) and <18 months since symptom onset) vi . The investigational drug pridopidine has Orphan Drug designation in HD and ALS in the US and EU, and FDA Fast Track designation for the treatment of HD. These designations do not imply that pridopidine has been approved or that safety and efficacy have been established. About Huntington’s Disease 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 vii , and each child of a parent with HD has a 50 percent chance of developing the disease. viii Across the world an estimated 100,000 people have HD ix,x , with an additional 300,000 people at risk of developing HD xi,xii . It is usually diagnosed between the ages of 30 and 50, 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 15 to 20 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 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 partnered with Ferrer for the commercialization and co-development of pridopidine. 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) . 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 Reilmann, R., Feigin, A., Rosser, A.E. et al. Pridopidine in early-stage manifest Huntington’s disease: a phase 3 trial. Nat Med 31, 3780–3789 (2025). iii ENROLL-HD and TRACK-HD iv 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. v 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. vi Writing Committee for the HEALEY ALS Platform Trial, HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025;333(13):1128–1137. doi:10.1001/jama.2024.26429 vii 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. viii Myers RH. Huntington's disease genetics. NeuroRx. 2004 Apr;1(2):255-62. doi: 10.1602/neurorx.1.2.255. PMID: 15717026; PMCID: PMC534940.) ix x 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. xi 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. xii 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. 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

26 May 2026

·www.businesswire.com

Prilenia Donates Clinical Trial Datasets to CHDI Foundation to Advance Huntington's Disease Research

NAARDEN, The Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)--Prilenia Therapeutics B.V. today announced a Data Use Agreement (DUA) under which Prilenia will provide CHDI with clinical data from the placebo arms of its PRIDE and PROOF studies for the purpose of advancing research into treatments for Huntington's disease (HD). “Patients are at the center of this initiative; it is vital to foster an ecosystem where we can all work together to fully utilize high-quality data to enable future innovation for the benefit of people living with HD.” “Collaboration which balances scientific rigor with data openness is an ethical and scientific imperative. Sharing data is key to accelerating and advancing knowledge and understanding and accelerates progress aimed at the development of much needed treatments capable of diminishing the effect of intractable diseases such as HD,” said Dr. Michael R. Hayden, Prilenia’s Chief Executive Officer. “Patients are at the center of this initiative; it is vital to foster an ecosystem where we can all work together to fully utilize high-quality data to enable future innovation for the benefit of people living with HD.” Robi Blumenstein, the president of CHDI Management, the company that manages the scientific activities of CHDI Foundation, remarked: “Prilenia’s gifting of these data will help us better understand how placebo-related effects influence clinical trial outcomes, which could improve the design and interpretation of future trials and ultimately support the development of more effective approaches to slow the progression of this devastating disease. A better understanding of placebo effects could also help strengthen the validation of external comparator approaches, including those derived from the Enroll-HD dataset.” Under the terms of the agreement, CHDI receives a worldwide, non-exclusive license to use the individual patient level data for the placebo patients from the two studies, full ownership of any research results it generates using the data, and the option to also make the data available to qualified third-party research institutions and companies. Prilenia retains ownership of the underlying data itself, and all data remains securely coded and anonymized to protect the identity of study participants. Any publications arising from the research may include a Prilenia author and will acknowledge Prilenia as the source of the data. About Huntington’s Disease 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 genei, and each child of a parent with HD has a 50 percent chance of developing the diseaseii. Across the world an estimated 100,000 people have HDiii,iv, with an additional 300,000 people at risk of developing HDv,vi. It is usually diagnosed between the ages of 30 and 50, 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 15 to 20 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 nothing impacting measures of overall progression. A potential registrational Phase 3 study of pridopidine in early to mid-stage HD is planned to commence in the second quarter of 2026. 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 partnered with Ferrer for the commercialization and co-development of pridopidine. The company is incorporated in the Netherlands and backed by leading life sciences investors. For more information, please visit www.prilenia.com, and connect with us on LinkedIn or X (Twitter). About CHDI Foundation, Inc. CHDI Foundation is a privately-funded, nonprofit biomedical research organization devoted to a single disease – Huntington’s disease (HD). Its mission is to collaboratively develop therapeutics that will provide meaningful clinical benefit to patients as quickly as possible. To achieve this CHDI manages a diverse portfolio of research projects through a novel virtual model that encourages scientific collaboration to more directly connect innovative research, drug discovery, and clinical development. This helps bridge the translational gap that often exists between academic and industrial research pursuits, and which adds costly delays to therapeutic development. CHDI’s activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials. i Eddings, C. R., Arbez, N., Akimov, S., Geva, M., Hayden, M. R., & Ross, C. A. (2019). Pridopidine protects neurons from mutant-huntingtin toxicity via the sigma-1 receptor. Neurobiology of disease, 129, 118–129. https://doi.org/10.1016/j.nbd.2019.05.009 ii Myers RH. Huntington's disease genetics. NeuroRx. 2004 Apr;1(2):255-62. doi: 10.1602/neurorx.1.2.255. PMID: 15717026; PMCID: PMC534940.) iii https://medically.roche.com/global/en/medical-material/HSG-2019-poster-yohrling-prevalence-of-huntington-s-disease-in-the-US-pdf.html 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. https://doi.org/10.3390/ijms241613021

License out/inPhase 3

01 May 2026

·endpoints.news

Boehringer appoints pharma veteran for corporate affairs post; BlueRock CSO heads for the exit

→ Boehringer Ingelheim has brought in Christie Bloomquist as SVP, corporate affairs, US human pharma, and she’ll be president of the Boehringer Cares Foundation. Peer Review informed you about her move to Astellas in 2023, and she had been VP, US strategy and government affairs at the Japanese drugmaker since January 2025. She was also in charge of US corporate and government affairs at AstraZeneca . “What drew me in was how both comfortable and challenged I felt with everyone I met during the interview process,” Bloomquist wrote in a LinkedIn post . “These are people I genuinely want to work with, in a place where I know I can contribute.” → In another LinkedIn discovery, Stefan Irion is stepping down as CSO of Bayer ’s BlueRock Therapeutics . Irion was promoted to this position in 2023 and started working at BlueRock “in its earliest days, when the idea was audacious and the roadmap mostly lived on whiteboards and in late‑night conversations,” he wrote . BlueRock has moved into Phase 3 with bemdaneprocel , a cell therapy to treat Parkinson’s disease. → Not only did Incyte get a pair of Phase 3 wins in nonsegmental vitiligo this week, its search for a CFO is over . Suky Upadhyay replaced Dan Florin as CFO of Zimmer Biomet in 2019 and would tack on the role of EVP, global operations. Upadhyay is currently on the board of directors at Vertex and led global financial operations at Bristol Myers Squibb from 2016-19. You may recall that Christiana Stamoulis left Incyte in September to become CFO at Mallinckrodt , which is now known as Keenova after the merger with Endo . Zimmer Biomet said that Paul Stellato will fill in as interim finance chief. → Shortly before landing on the Nasdaq in a $255 million IPO this week, Seaport Therapeutics snagged neuroscience veteran Sharon Mates for its board. The mid-stage neuroscience biotech now has the former chair and CEO of Intra-Cellular Therapies to help guide it toward commercialization. Mates led Intra-Cellular all the way from the ground up, put it on a commercial footing, and took it to a $14.6 billion exit to Johnson & Johnson . → Jennifer Smoter said in a LinkedIn post that she’s “thrilled to return to the biopharmaceutical industry” as chief communications & governance officer at Takeda . Smoter led the communications team during her four and a half years with UnitedHealth Group , and she was chief external affairs & communications officer at Intellia Therapeutics . As part of its multiyear restructuring , Takeda recently ended its partnership with Denali Therapeutics . The Japanese pharma reports its FY2025 earnings on May 13. → A spokesperson tells Peer Review that AI-focused startup Basecamp Research has tapped Richard Pearce as CBO after raising the curtain on its Trillion Gene Atlas project last month. Basecamp plucked him from Biogen , where he was head of business development, strategy and portfolio management in the back half of his 11-year career with the company. Earlier, Pearce served as business and operations director of the Biodevelopment Centre at Merck KGaA . “By training foundation models on the largest evolutionary dataset ever assembled, Basecamp is building something fundamentally new for biology,” Basecamp CEO Glen Gowers said in a statement. “Richard brings a rare combination of deep pharma experience and first-principles thinking — exactly what’s needed to translate this technology into real-world impact.” → Boston-based mRNA startup Strand Therapeutics has hired its first-ever CFO. Kunal Bhatia started his career at Credit Suisse and Jefferies , and has been a managing director in the healthcare investment banking groups at Oppenheimer and TD Cowen . Kinnevik , Regeneron Ventures , Amgen Ventures and Eli Lilly participated in Strand’s $153 million Series B last summer. → Coy Stout is retiring after 30 years in industry, with the last year and a half as executive director of US patient support at AstraZeneca. He’s had stops along the way at Lykos , Brii Biosciences and nearly two decades at Gilead . “While I expected this part of my journey as a market access leader to last a little bit longer,” Stout wrote on LinkedIn, “life has a way of making priorities clear. I’m stepping away from the industry to focus on work-life balance and on one of the most important roles of my life: being a spouse and caregiver to someone living with advancing Parkinson’s disease.” → Now under the direction of ex- bluebird bio boss Andrew Obenshain , Inventiva has welcomed three new execs, including former CureVac finance chief Axel-Sven Malkomes . He’ll be the first person besides Jean Volatier to step into the CFO slot in the company’s history, and Volatier will still have a presence at Inventiva as EVP of finance & corporate social responsibility. Meanwhile, Inventiva has recruited Susan Coles as chief legal officer and Pamela Herbster as chief people officer. Coles has already been general counsel at Inventiva twice, and Herbster was elevated to chief people & experience officer at Sage before Supernus bought the embattled drug developer for $561 million. → Newly public Avalyn Pharma has recruited Adam Golden as general counsel and head of business development, and Frank Salisbury as SVP of commercial. Golden comes to Avalyn from London-based law firm Freshfields , where he was a partner and global head of life sciences. Salisbury is a Sage and Genentech alum who spent the last four years with PureTech Health as head of commercial and product strategy. → Lis Leiderman has returned to Peer Review for her fourth CFO job this decade. Her LinkedIn page indicates that she succeeded Rob Lauzen at Dutch neuro biotech Prilenia Therapeutics in March. Leiderman worked for Decibel Therapeutics (2020-22), Atsena Therapeutics (2022-23) and Dewpoint Therapeutics (2024-25), and she sits on the board of directors at Autolus Therapeutics . Prilenia’s only candidate is an S1R agonist called pridopidine for ALS and Huntington’s disease. → Sanofi Genzyme vet Michael Panzara has replaced co-founder Andreas Muehler as CMO of Immunic . Panzara had been CMO of Blackstone ’s Neurvati Neurosciences and its subsidiary GRIN Therapeutics , which collected $50 million upfront from Angelini Pharma for the ex-North America rights to radiprodil . He also vaulted to CMO and head of therapeutics discovery & development during a six-year run at Wave Life Sciences . → Radiotherapy shop Convergent Therapeutics has selected Merck vet Andrew Davis as chief business and strategy officer. Davis concluded his stint as CBO of Dynavax after it was purchased by Sanofi for $2.2 billion, and he held that same role at Ironwood Pharmaceuticals . → Christian Schade has been named chairman of the board at German biotech CatalYm . Since selling Halda Therapeutics to J&J for north of $3 billion last fall, Schade joined Frazier Life Sciences as a senior advisor. After a stint as CEO of Aprea Therapeutics , he landed at Flagship Pioneering in early 2023 as a growth partner. → Marea Therapeutics CEO Josh Lehrer has locked up a spot on the board of directors at Fulcrum Therapeutics . With a series of roles over a six-year period at Global Blood Therapeutics , including CMO, his input on Fulcrum’s lead sickle cell asset pociredir should be beneficial. Lehrer ran Graphite Bio until it merged with Lenz Therapeutics . → Deborah Birx ’s bacteriophage biotech Armata Pharmaceuticals has appointed Daniel Gilmer to its board of directors. Gilmer was elevated to senior director, commercial quality US team lead at Pfizer last year. → In other boardrooms, Raymond Dwek will retire at United Therapeutics , but he’ll stay on the scientific advisory board; Boehringer Ingelheim’s Lykke Hinsch Gylvin is headed to ProQR ; Troy Cox and Alumis CEO Martin Babler are in line to replace Robert Malenka and Jim Trenkle at Maplight Therapeutics , according to an SEC filing ; Santhera has added ex- Idorsia CEO Srishti Gupta ; and InduPro Therapeutics CEO Prakash Raman has picked up a board seat at Olema Oncology .

Executive ChangeIPOPhase 3

100 Deals associated with Pridopidine

External Link

KEGG	Wiki	ATC	Drug Bank
D09953	Pridopidine	-	DB11947

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Huntington Disease	NDA/BLA	European Union	Prilenia Therapeutics BV	03 Sep 2024
Amyotrophic Lateral Sclerosis	Phase 3	United States	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Belgium	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Canada	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	France	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Germany	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Ireland	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Israel	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Italy	Prilenia Therapeutics BV	01 Feb 2026
Amyotrophic Lateral Sclerosis	Phase 3	Netherlands	Prilenia Therapeutics BV	01 Feb 2026

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04556656 (PROOF-HD, Company_Website) Manual	Phase 3	Huntington Disease	-	Pridopidine	xxyczjjbuc(ddqqwcmvul) = 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. wupdlghked (lnyvwjehdi ) View more	Positive	10 Oct 2025
NCT04556656 (PROOF-HD, Pubmed \| Nat Med) Manual	Phase 3	Huntington Disease	-	Pridopidine (45 mg)	uwlatipdsc(pmcedcitpf) = 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. zrdepmvbmf (qdbgwophuk ) Not Met View more	Positive	05 Sep 2025
				Placebo
NCT04556656 (PROOF-HD, EAN2025)	Phase 3	Huntington Disease	245	Participants on ADMs	xdkzoymvfa(epmjjerenh) = spdxlsjzkp mspieuhtrf (mkocqqzgmv )	Negative	21 Jun 2025
				Participants off ADMs	xdkzoymvfa(epmjjerenh) = qqhwregyrh mspieuhtrf (mkocqqzgmv )
NCT04297683 \| NCT04615923 (HEALEY ALS Platform Trial, Pubmed \| JAMA)	Phase 2/3	Amyotrophic Lateral Sclerosis sigma-1 (σ1) receptor	163	Pridopidine 45 mg twice daily	wdlgradote(qrzfacbkse): DRR = 0.99 (95% CI, 0.8 - 1.21) View more	Negative	01 Apr 2025
				Placebo
NCT04556656 (PROOF-HD, CTgov)	Phase 3	Huntington Disease	499	Pridopidine (Pridopidine)	ufywutpwyn(ohnlagjynr) = eetuvrrsvg nkapqquhnx (jmetqveaoh, 0.119) View more	-	12 Mar 2025
				Placebo (Placebo)	ufywutpwyn(ohnlagjynr) = pssumvfasr nkapqquhnx (jmetqveaoh, 0.119) View more
NCT00724048 (HART, CTgov)	Phase 2	Huntington Disease	227	Placebo (Placebo)	vwopxaogyx(zzxjvududw) = yqtnfhwatu kuwsmjvbls (vadrtvwhhq, 3.72) View more	-	31 Aug 2023
				ACR16 (ACR16 10 mg BID)	vwopxaogyx(zzxjvududw) = jjkelhlxko kuwsmjvbls (vadrtvwhhq, 3.33) View more
NCT04615923 (HEALEY ALS Platform Trial, CTgov)	Phase 2/3	Amyotrophic Lateral Sclerosis	163	Pridopidine (Pridopidine)	rjbsscfnfj(axipmbctzk) = hlxxkjvzuj fllrelapwa (feqlqkyfwt, 0.077) View more	-	23 Aug 2023
				Matching Placebo (Matching Placebo)	rjbsscfnfj(axipmbctzk) = skeehsmypk fllrelapwa (feqlqkyfwt, 0.070) View more
NCT04615923 (HEALEY ALS Platform, NEWS) Manual	Phase 2	Amyotrophic Lateral Sclerosis Nfl	-	Pridopidine	utwnuluroi(aogencesdt) = kqbqycskno kysvntumwd (rinerlvgmp )	Positive	23 Feb 2023
				Placebo	utwnuluroi(aogencesdt) = zbossqzbyt kysvntumwd (rinerlvgmp )
NCT00724048 \| EUCTR2007-004988-22-AT \| NCT02006472 \| NCT00665223 \| EUCTR2013-001888-23-AT (PRIDE-HD, Pubmed)	Phase 2	Huntington Disease	402	Pridopidine 45 mg bid	xhofbxgnce(twymqxlzwy) = pjoezhqvie xgenpmkjor (bcmldftomq, 8.0)	-	22 Feb 2023
NCT03922711 (CTgov)	Phase 2	Dyskinesia, Drug-Induced \| Parkinson Disease	23	Placebo	sifdzdpqyo(kjpbarxens) = wmqssjlqrq btcmznnmus (rzdykaowzh, 6.44) View more	-	01 Jul 2022

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