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NAARDEN, Netherlands & WALTHAM, Mass.--( BUSINESS WIRE )-- Prilenia Therapeutics B.V. , a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, announced its plan to submit a Marketing Authorization Application (MAA) for pridopidine for the treatment of Huntington’s disease (HD) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). This decision follows positive pre-submission meetings with regulatory authorities in the European Union. Submission is planned for mid-2024. “Pridopidine demonstrates consistent treatment benefits across independent measures that are important to patients and families. These measures include day-to-day function, cognition, motor and clinical progression in people living with HD. The benefits are clearly evident in those with HD who are not taking anti-dopaminergic medications (ADM),” said Dr. Michael R. Hayden, CEO of Prilenia. “We appreciate the constructive discussions with European regulators regarding our data for pridopidine. This provides a pathway to potential approval of a therapy for HD, a rare neurodegenerative disease with a predictable decline, and no currently approved treatments that address progression. We are now finalizing our MAA submission for mid-2024 and preparing to make pridopidine commercially available for patients in Europe, if approved.” ADMs include neuroleptics (also known as antipsychotics) and VMAT2 inhibitor (anti-chorea) drugs. Prilenia intends to discuss with the U.S. Food and Drug Administration (FDA) a potential path forward for pridopidine as a possible treatment for those living with HD in the United States. The Company will also consider global regulatory submissions for additional countries and regions following the regulatory review process in Europe. In addition to the progress in HD, Prilenia announced in January 2024 that it had completed discussions with global regulatory agencies regarding the next stage of development of pridopidine for amyotrophic lateral sclerosis (ALS) and is planning for a single pivotal Phase 3 study. About Pridopidine Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability profile similar to placebo in clinical studies to date. The safety dataset includes approximately 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. About Huntington’s disease Huntington’s disease (HD) is a rare inherited (genetic) neurodegenerative disorder caused by a mutation in the huntingtin gene. Each child of a parent with HD has a 50 percent chance of developing the disease. HD causes neurons in the brain to degenerate and lose their ability to communicate with each other, resulting in functional, motor, cognitive and behavioral symptoms. HD affects about 100,000 people around the world with an additional 300,000 people at risk of developing HD. It is usually diagnosed between the ages of 30 and 50, although HD can also occur at any age including 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. Currently, there is no treatment to address the progression of HD. About PROOF-HD PROOF-HD, a Phase 3, randomized, double-blind, placebo-controlled study, evaluated the safety and efficacy of pridopidine (45 mg twice daily for 65-78 weeks), in individuals with HD ( NCT04556656 ). The study enrolled 499 individuals and was conducted in the U.S., Canada, Austria, Czech Republic, France, Germany, Italy, the Netherlands, Poland, Spain and the United Kingdom. Pridopidine (45 mg bid) has shown consistent and sustained benefit on multiple, independent, clinically meaningful measures in HD participants not taking ADMs. These benefits were seen across multiple clinical endpoints that matter to people with HD and their families (function, cognition, motor, global disease progression and quality of life). In pre-specified analyses, there were improvements compared to placebo across progression, motor, cognition and HD QoL (as measured by TFC, cUHDRS, SWR, Q-Motor, HDQoL). These treatment benefits were sustained for up to 78 weeks at all time points. There was also unprecedented improvement from baseline for a minimum of 1 year in cUHDRS, cognition and motor function (Q-Motor). These PROOF-HD results are further supported by an independent integrated efficacy analysis from a 4 placebo-controlled, double-blind studies of pridopidine in HD which showed significance for cUHDRS, TFC and Q-Motor in patients off ADMs. In clinical studies to date, pridopidine’s safety and tolerability profile was comparable to placebo. The PROOF-HD study was conducted in collaboration with the Huntington Study Group (HSG) , a world leader in clinical research for HD and valued collaborator for Prilenia. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of well-respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S. For more information, visit www.prilenia.com and follow us on LinkedIn and Twitter . © 2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at www.prilenia.com .

NAARDEN, Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)-- Prilenia Therapeutics B.V., a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, will present clinical data supporting a future Phase 3 study in amyotrophic lateral sclerosis (ALS) during the 14th Annual California ALS Research Summit in Los Angeles. The Company has completed discussions with global regulatory agencies regarding the next stage of development of pridopidine for ALS and is planning for a single pivotal Phase 3 study to start in H2 2024. Pridopidine is an investigational oral, small molecule, highly selective and potent Sigma-1 Receptor (S1R) agonist being studied as a potential treatment for ALS and Huntington’s disease (HD). The S1R is highly expressed in the brainstem and spinal cord, areas implicated in ALS and important for bulbar function and speech. “Based on the clinical data generated to date, pridopidine has the potential to become a meaningful treatment for ALS, slowing key measures of disease progression, including function, respiration, quality of life and impact on speech, which is very meaningful for patients and caregivers, as well as prolonging overall survival,” said Dr. Michael R. Hayden, CEO and Founder of Prilenia. “We believe it is important for Prilenia to advance our clinical program in ALS, and we are actively planning a single, global, pivotal Phase 3 clinical trial. Our sincere appreciation to the many ALS patients, caregivers and healthcare providers who have contributed so much to our clinical studies as well as to those who will participate in future trials. We also are grateful to Dr. Merit Cudkowicz and the entire team at the Sean M. Healey & AMG Center for ALS for their innovative and passionate approach to evaluating pridopidine as we continue our close working relationship.” “In the Phase 2 clinical trial, pridopidine showed encouraging results for the potential treatment of ALS across multiple secondary and exploratory measures with an excellent safety profile, and we are pleased to work closely with Prilenia on the advancement of this program into Phase 3,” said Merit Cudkowicz, M.D., MSc, principal investigator and sponsor of the HEALEY ALS Platform Trial, Director of the Sean M. Healey & AMG Center for ALS, Chair of the Department of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. While pridopidine did not meet the primary outcome measure, data from the Phase 2 HEALEY ALS Platform Trial evaluating the safety and efficacy of pridopidine as a potential treatment for ALS show in the pre-specified subgroup of participants with definite ALS who were also early in the course of the disease (less than 18 months from symptom onset) that pridopidine was associated with slower disease progression relative to placebo in ALSFRS-R (Δ2.4, p=0.19), respiratory domain (Δ1.04, p=0.18), and dyspnea (Δ1.35, p=0.014). Less worsening was observed on the ALSAQ-40 quality of life scale (Δ-10.83, p=0.018) and its eating and drinking (Δ-19.18, p=0.015) and communication (Δ-13.04, p=0.12) domains. Pridopidine was also observed to have beneficial effects compared to placebo in speech in the full analysis set (speaking rate, p=0.027, articulation rate p=0.0129, phonation time p=0.076, and articulation precision p=0.1138). Speech is a highly clinically relevant endpoint in ALS, and measures of speech are associated with overall ALS and bulbar disease severity. As ALS progresses, it is common for patients to have difficulty speaking due to weakening muscles (dysarthria). This poses significant communications challenges with family, friends and healthcare providers. Additional analyses from the same Phase 2 study of participants with definite or probable ALS who were also early and fast progressors were conducted. In this subgroup, there was a significant and meaningful (41 percent) slowing of disease progression compared to placebo at 24 weeks in ALSFRS-R (Δ5.2, p=0.04). This slowing of disease progression was already observed at 8 weeks (44.5 percent, p=0.02) and 16 weeks (52 percent, p=0.014). This group also showed greatest improvements vs placebo in ALSFRS-R respiratory domain (Δ1.81, p=0.08), dyspnea (Δ1.41, p=0.019), speaking rate (Δ1.08, p=0.00004) and articulation rate (Δ1.03, p=0.00002). Furthermore, survival benefits from post-hoc analyses of the placebo-controlled and open-label extension portions of the study show that pridopidine provided an increase in survival time for the specific subgroup consisting of participants with definite or probable ALS who were also early in the course of the disease. A Kaplan-Meier survival analysis showed a prolongation of median survival time from ~300 to 600 days in these participants compared to the delayed-start placebo participants (log rank test: p=0.069). The Cox Proportional Hazard Ratio (HR), adjusted for baseline characteristics, was 0.429 (p=0.052), representing a 57 percent improvement in survival benefit. Altogether, these results are encouraging and further inform the ongoing planning of a Phase 3 study. In collaboration with Massachusetts General Hospital and the Healey Center and through the ACT for ALS (Accelerating Access to Critical Therapies for ALS Act), the National Institutes of Health (NIH) has provided a grant to support expanded access to pridopidine for 200 persons with ALS in the U.S. who are not eligible for active clinical trials. Preliminary topline results in the full analysis set of this Phase 2 study were previously announced. Pridopidine was well-tolerated with no serious treatment-related adverse events, with a safety and tolerability pro to placebo and consistent with previous clinical studies. About Pridopidine and the Sigma-1 Receptor (S1R) Pridopidine (45 mg twice daily) is an oral, highly selective, and potent investigational S1R agonist that has exhibited a safety and tolerability pro to placebo in clinical studies to date. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways, including autophagy, axonal transport, mitochondrial energy production and calcium homeostasis, which are essential to neuronal function and survival, and may lead to neuroprotective effects. Mutations in the S1R gene cause ALS with the time of onset related to the expression of the S1R gene. For example, patients with no S1R protein have juvenile-onset ALS, while patients with diminished S1R expression have adult-onset ALS. The S1R positively regulates key cellular pathways that are commonly impaired in ALS and other neurodegenerative diseases. In ALS cell culture and mouse models, pridopidine has been shown to be neuroprotective, specifically mediated via the activation of S1R. Prilenia holds Orphan Drug designation for pridopidine in ALS and Huntington’s Disease (HD) in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. Pridopidine is not yet approved in the U.S. or EU. About the HEALEY ALS Platform Study – Pridopidine Regimen The Phase 2 clinical study, led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, was designed to evaluate the potential safety and efficacy of pridopidine, along with multiple other investigational products simultaneously, for the treatment of ALS. Eligible participants had El Escorial possible, probable, or definite ALS, symptom onset <36mo and vital capacity >50%-predicted. Pridopidine 45 mg bid (n=121) was compared to a shared placebo (n=164). The primary endpoint was change from baseline through 24 weeks in ALSFRS-R total (Full Analysis Set, FAS). Secondary and exploratory endpoints included speech, respiration, and quality of life measurements. Prespecified and post-hoc subgroups included definite or probable ALS, early (<18mo symptom onset) and fast progressors (pre-baseline slope>=-1). Nominal p-values are reported. About Expanded Access for Pridopidine The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital has been awarded a grant from the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS) to conduct an intermediate size Expanded Access Protocol (EAP) in ALS in collaboration with Prilenia Therapeutics. The EAP made possible by this award will allow ALS individuals who are otherwise not eligible for participation in clinical trials of pridopidine to access this investigational drug. Additionally, this EAP will provide real world safety, biomarker, and clinical data in up to two hundred (200) ALS individuals treated with pridopidine for up to two (2) years, thus contributing to our understanding of the effects of pridopidine in a broad population. The pridopidine EAP will be conducted at up to 45 enrolling centers across the U.S. The enrollment phase is anticipated to start in the Spring of 2024. Additional information about Prilenia’s EAP policy is available on our website. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of well-respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in Naarden, the Netherlands, Herzliya, Israel and Waltham, Massachusetts in the U.S. For more information, visit and follow us on LinkedIn and Twitter. ©2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at .

VANCOUVER, British Columbia--(BUSINESS WIRE)-- Prominent contract research organization, Purpose Life Sciences (PLS) is set to launch a groundbreaking Phase III clinical trial in conjunction with Dr. Gilmar Reis of Card Research, an expansive medical clinic network based in Brazil. The trial was designed by researchers at McMaster University in Hamilton, Canada, and is aimed at addressing the persisting challenges posed by Long-COVID. As a multi-center study beginning with 1,500 patients, the trial will rigorously assess the efficacy of multiple potential treatment options – beginning with fluvoxamine and metformin – in alleviating the symptoms associated with Long-COVID. The study will be run internationally, including medical centers across Brazil. The trial will leverage state-of-the-art data management via an EDC developed by Yonalink, to ensure the highest caliber of data integrity. Long-COVID, a condition characterized by an array of debilitating symptoms, has continued to affect a significant number of individuals even after the acute phase of the disease has passed. Therapeutic research for both acute and Long-COVID has faced challenges marked by inconsistent methodologies, biases, and flawed results. This study aims to rectify this situation by leveraging a robust study infrastructure, efficient control arm utilization, and streamlined intervention launches. The new approach seeks to swiftly identify effective Long-COVID-19 treatments and assess comparative efficacy without compromising the rigorous standards of a randomized clinical trial. Metformin, a well-established drug in the treatment of type 2 diabetes, presents an intriguing potential for Long-COVID treatment. While initial clinical trials employing metformin in acute COVID-19 cases did not demonstrate significant efficacy in reducing clinically relevant events or symptoms, recent data suggests that prolonged use and follow-up of patients on metformin may lead to a lower incidence of shortness of breath, fatigue, and nonspecific neurological symptoms. The trial seeks to explore the potential benefit of metformin in Long-COVID cases. Fluvoxamine, a known SSRI and S1R receptor agonist has shown potential in preliminary tests to reduce excessive inflammation in those with the virus. New information published in Cell reported that researchers studying people with symptoms months after infection had reduced rates of serotonin—a chemical messenger involved in regulating mood and digestion, among myriad other functions—which in turn causes cognitive problems. “Everyone knows someone affected by COVID-19,” says Dr. Edward Mills, Founder of PLS. “It’s a true privilege to be able to spearhead a project that will make a difference for people around the globe. COVID knows no geographic boundaries; it doesn’t differentiate between gender, ethnicity, or socioeconomic status, and finding a solution to such a prevalent disease will have a significant impact on those suffering from Long-COVID and their families.” PLS and Card Research are committed to delivering meaningful advancements in Long-COVID treatment and to using the most advanced data management tools to ensure that the trial is run with the utmost efficiency and concern for data quality and integrity. The research subject participation period extends up to 90 days, allowing for rigorous data collection and analysis, ensuring that the clinical results are robust and reliable. About PLS (Purpose Life Sciences): PLS is a leading research organization dedicated to advancing medical science and improving patient outcomes. With a strong commitment to research and innovation, PLS conducts clinical trials and studies aimed at addressing some of the most pressing health challenges of our time. About Yonalink Yonalink, with offices in Boston and Tel Aviv, streamlines clinical trial operations globally by seamlessly integrating patient data from Electronic Health Records (EHR) into a real-time Electronic Data Capture (EDC) system. Their full-service data management platform offers EHR-to-EDC capabilities, EDC, eConsent, ePRO/eCOA, and more, into one convenient and easy-to-use platform. Learn more at .