A Randomized, Double-Blind, Placebo-Controlled Parallel Group Study to Investigate the Safety and Efficacy of Arbaclofen Extended-Release Tablets for the Treatment of Spasticity in Patients With Multiple Sclerosis (Study OS440-3006)

This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety and efficacy of oral arbaclofen ER tablets in MS patients with spasticity. Arbaclofen ER will be compared with placebo.

Start Date01 Jan 2026

Sponsor / Collaborator

RVL Pharmaceuticals, Inc.

NCT04271332

/ Active, not recruitingPhase 2IIT

An Exploratory, Randomized, Double-Blind, Placebo-Controlled and Open-label Extension Study of the Safety, Tolerability, and Efficacy of Arbaclofen in Subjects With 16p11.2 Deletion

This Phase 2 study examines the safety, tolerability, and efficacy of arbaclofen in pediatric subjects with 16p11.2 deletion. Male or female subjects aged 5 to 17 years of age will be randomized to receive either placebo or arbaclofen in a double-blind study design. If the subject completes all study requirements through Visit 4 (Close-out Visit), he/she may be eligible for an optional open-label study with arbaclofen.

Start Date01 Sep 2022

Sponsor / Collaborator

Clinical Research Associates, LLC

NCT03682978

/ CompletedPhase 2IIT

A Phase II Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy, Safety, and Tolerability of Arbaclofen Administered for the Treatment of Social Function in Children and Adolescents With Autism Spectrum Disorders

AIMS-2-CT-01 is a randomized, double-blind, placebo controlled, study to explore the efficacy, safety and tolerability of Arbaclofen administered to children and adolescents (ages 5-17) for the treatment of social adaptive function in participants with ASD. The effects of Arbaclofen on social function in children and adolescents with ASD will be evaluated in a randomized, placebo controlled, parallel-group study of 16 weeks duration. Subjects who meet protocol criteria will be randomly allocated to receive either Arbaclofen or placebo in a 1:1 ratio in the Treatment Period. There will be 7 recruiting sites and randomization will be stratified by site. A sample of 130 patients will be recruited. Blinding will be maintained by utilizing identical tablets containing either Arbaclofen or placebo.

Start Date19 Sep 2019

Sponsor / Collaborator

University Medical Center of Utrecht

100 Clinical Results associated with Arbaclofen

100 Translational Medicine associated with Arbaclofen

100 Patents (Medical) associated with Arbaclofen

367

Literatures (Medical) associated with Arbaclofen

26 Mar 2025·Journal of the American Chemical Society

Computational Identification of Potential Organocatalysts (CIPOC) Reveals a 2-aminoDMAP/Urea Catalyst Superior to Its Thiourea Analogue

Article

Author: Işık, Murat ; Bakırcı, İrem ; Çelebi-Ölçüm, Nihan ; Tanyeli, Cihangir ; Alsancak-Koca, Sezen ; Çamlısoy, Yeşim

Asymmetric organocatalysis by bifunctional acid- and base-type small organic molecules has emerged as a promising way to enhance stereoselective organic transformations since the beginning of this millennium. Takemoto's tert-amine/thiourea catalyst, an archetype in these endeavors, has encouraged many to design new multifunctional alternatives. However, the discovery of efficient catalysts in a library of thousands of candidates containing the desired functionalities in their structures remains a great challenge both synthetically and computationally. We, toward these ends, developed a computational protocol (CIPOC─Computational Identification of POtential (Organo)Catalysts), which discovered a chiral 2-aminoDMAP/urea catalyst among 1600 multifunctional catalyst candidates enabling conjugate addition of malonates to trans-β-nitroalkenes rapidly (in a few hours) with exquisite selectivities and yields, producing superior results than that of Takemoto's. The unique activity of this chiral 2-aminoDMAP/urea is attributed to the dual function of the 2-aminoDMAP unit (double H-bonding and π-stacking interactions) in addition to the exceptional performance of the urea unit compared to thiourea, as a result of a lower energetic penalty required to distort the catalyst to its active conformation to provide optimal catalytic interactions.

01 Mar 2025·JOURNAL OF PSYCHOPHARMACOLOGY

Navigating agitation in neurodevelopmental disorders: A comparative study of pharmacotherapies via network meta-analysis in children and adults with autism spectrum disorder or intellectual disabilities

Review

Author: Hawken, Emily R ; Waqas, Ahmed ; Ayub, Muhammad ; Nasar, Amina ; Bahji, Anees ; Forth, Evan

Importance::

Individuals with autism spectrum disorder (ASD) and intellectual disability often experience persistent challenges related to aggressive behaviour and agitation, highlighting the critical need for evidence-based pharmacological interventions among other strategies. Despite previous network meta-analyses (NMAs), the rapidly evolving landscape of treatment options necessitates ongoing and updated assessments.

Objective::

To evaluate the efficacy and tolerability of various pharmacotherapies in managing agitation in children and adults with ASD or intellectual disabilities (ID).

Methods::

Employing a systematic review and network meta-analysis methodology, we conducted an exhaustive search across multiple databases for double-blind, randomized controlled trials focusing on pharmacotherapies targeting agitation in these neurodevelopmental disorders. Adhering to Preferred Reporting Items for Systematic Reviews and Meta-analysis guidelines, our assessment of study quality utilized the Cochrane Risk of Bias Tool to ensure methodological rigour and accuracy in data synthesis. Primary outcomes encompassed measures of reduced agitation, as indicated by treatment response on standardized agitation scales, alongside dropout rates, providing a comprehensive overview of treatment efficacy and tolerability.

Results::

Our analysis included data from 38 eligible trials, involving 2503 participants across both pediatric and adult populations. Key pharmacological interventions, such as arbaclofen, risperidone plus buspirone, omega-3 fatty acids, risperidone plus palmitoylethanolamide, aripiprazole and risperidone, demonstrated significant efficacy in reducing agitation compared to placebo. Importantly, these treatments were generally well-tolerated, with no significant increase in all-cause dropouts compared to placebo, highlighting their suitability for clinical use in managing agitation in individuals with ASD or ID.

Conclusions::

This study underscores the efficacy and tolerability of several pharmacotherapies in managing agitation among children and adults with ASD or ID. Our findings provide robust evidence that specific treatments, such as arbaclofen, risperidone plus buspirone and omega-3 fatty acids, are both effective and well-tolerated, offering valuable therapeutic options for clinicians. The study emphasizes the need for ongoing research to ensure that treatment strategies remain aligned with the evolving clinical landscape, ultimately improving patient outcomes in this challenging population.

01 Feb 2025·ANALYTICA CHIMICA ACTA

Preparation of novel chiral stationary phases based on chiral metal-organic cages enable extensive HPLC enantioseparation

Article

Author: Luo, Aiqin ; Duan, Juntao ; Tang, Bo ; Wang, Wei ; Zhang, Yukui ; Wang, Zhen ; Sun, Liquan ; Liang, Axin

BACKGROUND:

The metal organic cages (MOCs) are an emerging type of porous material that has attracted considerable research interest due to their unique properties, including good stability and well-defined intrinsic cavities. The chiral MOCs with porous structures have broad application prospects in enantiomeric recognition and separation. However, there are almost no relevant reports on chiral MOCs as chiral stationary phases (CSPs) for enantioseparation by high-performance liquid chromatography (HPLC).

RESULTS:

Here, we report for the first time, the chiral MOCs, MOC-PA was "wrapped" onto the silica gel to create a novel HPLC CSP of chiral MOC-PA@SiO₂ through wrapped in net method. There are 24 and 20 chiral compounds or chiral drugs, including alcohols, diols, esters, ketones, alkene, epoxides, ethers, organic acids, phenols and amines, were enantioseparated by vary degrees on MOC-PA@SiO₂ packed column under both normal phase (NP) and reversed phase (RP) modes, respectively. Furthermore, compared with commercial Chiralpak AD-H and Chiralcel OD-H columns, the MOC-PA@SiO₂ packed column exhibits well chiral separation complementarity to them. It can separate some racemates that unable to be or not to be well separated by the Chiralpak AD-H and Chiralcel OD-H columns. Besides, there are four positional isomers were also well separated by MOC-PA@SiO₂ packed column.

SIGNIFICANCE:

The effects of temperature and injection volume on separation of MOC-PA@SiO₂ packed column were further investigated. This column also exhibited excellent repeatability, stability and column-to-column reproducibility on separation in HPLC. This work indicates that chiral MOCs are a promising class of chiral materials to prepare new CSPs for HPLC enantioseparation.

News (Medical) associated with Arbaclofen

21 Aug 2025

·www.prnewswire.com

Allos Pharma Acquires Global Rights to Arbaclofen, Reports Positive ASD Trial Results and Grant Award

CAMBRIDGE, Mass., Aug. 21, 2025 /PRNewswire/ -- Allos Pharma Inc., announces key milestones advancing its mission to deliver effective treatments for Autism Spectrum Disorder (ASD), Fragile X Syndrome, and related neurodevelopmental disorders. Global Rights to Arbaclofen Allos has secured worldwide rights to arbaclofen, a selective GABA-B receptor agonist previously evaluated in multiple Phase II and III trials in ASD, Fragile X syndrome (FXS) and 16p11.2 deletion syndrome. This transfer includes clinical data, regulatory assets, and manufacturing capabilities. "This marks a foundational moment for Allos," said Dr. Randall Carpenter, Co-founder and CMO of Allos Pharma. "Owning full rights to arbaclofen enables us to move swiftly and with strategic clarity as we build a sustainable pipeline targeting core features of ASD and genetically defined neurodevelopmental syndromes." Encouraging ASD Clinical Results Recently completed placebo-controlled trials in Canada and the EU, sponsored by the POND Network and the EU AIMS-2-Trials Consortium respectively, independently replicate the clinically meaningful improvements in social function observed in the Phase II Seaside Therapeutics clinical trial. Significant improvements were also observed in the core ASD domains of communication and restricted/repetitive behaviors. These results provide the strongest efficacy seen to date in ASD pharmacotherapy. 16p11.2 Data Analysis The recently completed L16hthouse study, sponsored by Clinical Research Associated LLC, was motivated by strong efficacy in preclinical models of 16p11.2 deletion syndrome, the most common genetic copy number variant cause of ASD. The results of this pilot study inform selection of primary endpoints and patient stratification strategies for future precision trials in this genetically defined orphan disease. Grant Awarded Allos has also received a non-dilutive grant from the Simons Foundation. The grant will support further clinical development of arbaclofen and enable biomarker-driven analyses in ASD. About Arbaclofen Arbaclofen is a GABA-B receptor agonist that modulates excitatory/inhibitory balance and metabolic pathways in the brain to correct core molecular pathophysiology in preclinical models of ASD, FXS, and 16p11.2 deletion syndrome. It has been administered to over 700 children, adolescents, and adults with ASD and FXS in previous trials and demonstrated exceptional safety and tolerability, including in long-term, open-label studies. About Allos Pharma Allos Pharma is a biopharmaceutical company developing novel therapies for autism and genetically defined neurodevelopmental disorders. The company's lead candidate, arbaclofen, targets core domains and associated symptoms of ASD, Fragile X syndrome, and 16p11.2 deletion syndrome. Allos is committed to rigorous science and clinical excellence, backed by leading philanthropic and academic partners. CONTACT: [email protected]. SOURCE Allos Pharma Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Clinical Result

11 Dec 2023

·www.prnewswire.com

Arbaclofen ER Emerging Drug Insight and Market Forecast to 2032

DUBLIN, Dec. 11, 2023 /PRNewswire/ -- The "Arbaclofen ER Emerging Drug Insight and Market Forecast - 2032" report has been added to ResearchAndMarkets.com's offering. This report provides comprehensive insights about Arbaclofen ER for spasticity in the seven major markets. A detailed picture of the Arbaclofen ER for spasticity in the 7MM, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan for the study period 2019 -2032 is provided in this report along with a detailed description of the Arbaclofen ER for spasticity. The report provides insights about mechanism of action, dosage and administration, as well as research and development including regulatory milestones, along with other developmental activities. Further, it also consists of future market assessments inclusive of the Arbaclofen ER market forecast analysis for spasticity in the 7MM, SWOT, analysts' views, comprehensive overview of market competitors, and brief about other emerging therapies in spasticity. Drug Summary Arbaclofen ER is an extended-release formulation of arbaclofen, the R isomer of baclofen that leverages the proprietary for osmodex drug delivery system and is being studied for the treatment of spasticity resulting from multiple sclerosis. It acts upstream of the mGluR5 receptor to increase inhibitory neurotransmission. It is the isomer of baclofen which harbors antispastic activity. The company is exploring opportunities to sell or out-license late-stage product candidate arbaclofen ER tablets designed to alleviate signs and symptoms of spasticity resulting from multiple sclerosis, for which they have completed Phase III clinical trials. The report provides insights into: A comprehensive product overview including the Arbaclofen ER description, mechanism of action, dosage and administration, research and development activities in spasticity. Elaborated details on Arbaclofen ER regulatory milestones and other development activities have been provided in this report. The report also highlights the Arbaclofen ER research and development activities in spasticity across the United States, Europe and Japan. The report also covers the patents information with expiry timeline around Arbaclofen ER. The report contains forecasted sales of Arbaclofen ER for spasticity till 2032. Comprehensive coverage of the late-stage emerging therapies for spasticity. The report also features the SWOT analysis with analyst views for Arbaclofen ER in spasticity. In-depth Arbaclofen ER Market Assessment This report provides a detailed market assessment of Arbaclofen ER for spasticity in the seven major markets, i.e., the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. This segment of the report provides forecasted sales data from 2026 to 2032. Arbaclofen ER Clinical Assessment The report provides the clinical trials information of Arbaclofen ER for spasticity covering trial interventions, trial conditions, trial status, start and completion dates. Report Highlights In the coming years, the market scenario for spasticity is set to change due to the extensive research and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market. The companies are developing therapies that focus on novel approaches to treat/improve the disease condition, assess challenges, and seek opportunities that could influence Arbaclofen ER dominance. Other emerging products for spasticity are expected to give tough market competition to Arbaclofen ER and launch of late-stage emerging therapies in the near future will significantly impact the market. A detailed description of regulatory milestones, and developmental activities, provide the current development scenario of Arbaclofen ER in spasticity. This in-depth analysis of the forecasted sales data of Arbaclofen ER from 2026 to 2032 will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the Arbaclofen ER in spasticity. Key Questions What is the product type, route of administration and mechanism of action of Arbaclofen ER? What is the clinical trial status of the study related to Arbaclofen ER in spasticity and study completion date? What are the key collaborations, mergers and acquisitions, licensing and other activities related to the Arbaclofen ER development? What are the key designations that have been granted to Arbaclofen ER for spasticity? What is the forecasted market scenario of Arbaclofen ER for spasticity? What are the forecasted sales of Arbaclofen ER in the seven major countries, including the United States, Europe (Germany, France, Italy, Spain) and the United Kingdom, and Japan? What are the other emerging products available and how are these giving competition to Arbaclofen ER for spasticity? Which are the late-stage emerging therapies under development for the treatment of spasticity? Key Topics Covered: 1. Report Introduction 2. Arbaclofen ER Overview in Spasticity 2.1. Product Detail 2.2. Clinical Development 2.2.1. Clinical studies 2.2.2. Clinical trials information 2.2.3. Safety and efficacy 2.3. Regulatory Milestone 2.4. Other Developmental Activities 2.5. Product Profile 3. Competitive Landscape (Marketed Therapies) 4. Competitive Landscape (Late-stage Emerging Therapies) 5. Arbaclofen ER Market Assessment 5.1. Market Outlook of Arbaclofen ER in Spasticity 5.2. 7MM Analysis 5.2.1. Market Size of Arbaclofen ER in the 7MM for Spasticity 5.3. Country-wise Market Analysis 6. SWOT Analysis 7. Analysts' Views For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo - SOURCE Research and Markets

Phase 3

18 Sep 2023

·www.prnewswire.com

Allos Pharma Inc. Expands Intellectual Property Rights for Arbaclofen

CAMBRIDGE, Mass., Sept. 18, 2023 /PRNewswire/ -- Allos Pharma Inc. ("Allos"), a biopharmaceutical company specializing in the development of treatments for rare neurological disorders, announced today that it has expanded its intellectual property rights with a new patent family. A new patent family was filed to the United States Patent Trademark & Office earlier in 2023. This new patent family comes in addition to an existing granted patent family. Continue Reading Allos also announces today the worldwide exclusive rights to granted patents and clinical programs for arbaclofen in further indications, including Autism Spectrum Disorder. This is in addition to the current Fragile X Syndrome development program, for which there are no approved medications, and where arbaclofen has Orphan Drug Designation. Expanded arbaclofen rights with a newly filed patent family and with exclusive rights in all indications, incl. Autism. Tweet this "This license builds upon the progress in our Fragile X Syndrome program, where we have achieved alignment with the fragile X community and the FDA on the design of the confirmatory Phase 3 clinical trial. We are excited to have the rights to develop arbaclofen in additional indications as clear efficacy signals were observed in recent trials in individuals with idiopathic Autism Spectrum Disorder." said Dr. Randall Carpenter, MD, co-founder of Allos Pharma Inc. In May 2023, the results of two autism studies, in Canada (n=90) and EU (n=122), were presented at the annual meeting of the International Society for Autism Research. The two studies had nearly identical protocols and endpoints. Improvement was seen in both studies on the primary endpoint (Socialization Domain, Vineland-3) but did not reach statistical significance. Nominally significant improvements were seen in one or both studies on other measures of social skill (Social Responsiveness Scale, Aberrant Behavior Checklist-Social Withdrawal, Autism Impact Measure). A subject-level meta-analysis is planned. "We remain committed to providing new treatment options for patients and families affected by Fragile X Syndrome and Autism Spectrum Disorder, and we believe that arbaclofen has the potential to make a significant, positive impact on their lives." Dr. Randall Carpenter, MD, co-founder of Allos Pharma Inc About Allos Pharma Inc. Allos Pharma Inc. is a biopharmaceutical company dedicated to the development of treatments for rare neurological disorders. Allos' investigational drug, arbaclofen, is currently in development for the treatment of Fragile X Syndrome and Autism Spectrum Disorder. For more information, please visit Allos Pharma Inc.'s website at SOURCE Allos Pharma Inc.

Phase 3Orphan Drug

100 Deals associated with Arbaclofen

External Link

KEGG	Wiki	ATC	Drug Bank
-	Arbaclofen	-	DB08891

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Multiple sclerosis relapse	Phase 3	United States	Osmotica Pharmaceutical US LLC	03 Apr 2018
Multiple Sclerosis	Phase 3	Belarus	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis	Phase 3	Bosnia and Herzegovina	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis	Phase 3	Bulgaria	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis	Phase 3	Croatia	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis	Phase 3	Poland	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis	Phase 3	Serbia	Osmotica Pharmaceutical US LLC	28 Jan 2018
Multiple Sclerosis, Primary Progressive	Phase 3	United States	Osmotica Pharmaceutical US LLC	01 Jul 2016
Multiple Sclerosis, Relapsing-Remitting	Phase 3	United States	Osmotica Pharmaceutical US LLC	01 Jul 2016
Multiple Sclerosis, Secondary Progressive	Phase 3	United States	Osmotica Pharmaceutical US LLC	01 Nov 2012

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03319732 (OS440-3005, CTgov)	Phase 3	Muscle Spasticity \| Multiple Sclerosis \| Multiple Sclerosis, Secondary Progressive ... View more	323	Arbaclofen	nzizkhorsx = ajwppgxmor popyhyhiap (epivhdchjg, infoksnrax - jtpxrrfhwv) View more	-	15 Jul 2022
NCT03290131 (OS440-3004, CTgov)	Phase 3	Muscle Spasticity \| Multiple Sclerosis	536	Arbaclofen (AERT 40 mg)	hxxinxdjkb(dtrmqnxxtl) = huksjniase ebbykdlsis (pxpgbpiyam, 1.97) View more	-	15 Jul 2022
				Arbaclofen (AERT 80 mg)	hxxinxdjkb(dtrmqnxxtl) = ltwlcayuec ebbykdlsis (pxpgbpiyam, 1.78) View more
NCT01325220 (Biospace) Manual	Phase 3	Fragile X Syndrome	-	Arbaclofen	etzfpwxiof(ftwwudammm) = uvnrkwnteh inzlerovme (pgcponppti )	Positive	14 Jul 2022
				Placebo	etzfpwxiof(ftwwudammm) = ciciclhkov inzlerovme (pgcponppti )
NCT01282268 (Pubmed \| J Neurodev Disord) Manual	Phase 3	Fragile X Syndrome	119	Arbaclofen	mbvrkavwci(xjiufekeqp) = bxswnzdhyg mgrjchztbv (cfrsrsaozp )	Negative	12 Jun 2017
				Placebo	mbvrkavwci(xjiufekeqp) = rrtgfrwncs mgrjchztbv (cfrsrsaozp )
NCT00846547 (CTgov)	Phase 2	Autism Spectrum Disorder \| Irritable Mood	32	Arbaclofen	gttocpvwux(gxgfovqmxz) = afiitdnqsn xvyttczdua (ywqwruvquq, 1.73) View more	-	14 Jun 2013
NCT00788073 (CTgov)	Phase 2	Irritable Mood \| Fragile X Syndrome	63	STX209 (STX209)	dhmedzgxpl(rplctxnwye) = icqforuccn apiswdklek (ziuqzuxtbs, jvcuwypxzy - qwgiwtewjd) View more	-	06 May 2013
				Placebo (Placebo)	dhmedzgxpl(rplctxnwye) = lymvgvxbch apiswdklek (ziuqzuxtbs, mmepaahaef - hcxudnknwn) View more

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