Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative condition, mainly characterized by progressive weakness and wasting of the limbs, the respiratory and bulbar muscles. Respiratory insufficiency leads to a fatal outcome after a mean diseases duration of only three to five years. The disease is characterized by pathological accumulations of a protein called TDP-43, which can be found large cortical and sub-cortical areas of post-mortem ALS brains.
No causal treatment for this condition is known to date, and there is a large unmet need to develop new strategies in order to halt or slow down its progression.
The aim of this study is to test the safety and tolerability of Tideglusib, a treatment that is already in clinical trials for other neuromuscular conditions, in patients with ALS. It is assumed that this drug may have a significant therapeutic benefit in this population due to his mode of action: In the ALS mouse model, Tideglusib decreases significantly the amount of accumulated TDP-43 proteins within the cells.

Start Date01 Dec 2025

Sponsor / Collaborator

Les Hôpitaux Universitaires de Genève

[+4]

NCT06174220 / Not yet recruitingPhase 2

Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy

The TaRGET study is a multi-centre, prospective, randomized, double-blind, placebo-controlled trial designed to evaluate the potential therapeutic efficacy of tideglusib, a glycogen synthase kinase-3 β inhibitor, in genotype positive arrhythmogenic cardiomyopathy.

Start Date01 Oct 2024

Sponsor / Collaborator

Hamilton Health Sciences Corporation

[+3]

ChiCTR2200065870 / SuspendedIIT

Clinical Study on Tideglusib and Platelet Products Promoting Wound Healing

Start Date30 Nov 2022

Sponsor / Collaborator-

100 Clinical Results associated with Tideglusib

100 Translational Medicine associated with Tideglusib

100 Patents (Medical) associated with Tideglusib

113

Literatures (Medical) associated with Tideglusib

01 Feb 2025·JOURNAL OF DENTISTRY

Tideglusib enhances ALP activity and upregulates RANKL expression in Osteoblast-macrophage Co-cultures within a 3D collagen scaffold

Article

Author: Toledano, Raquel ; Yildirim-Ayan, Eda ; Toledano, Manuel ; Jacho, Diego ; Osorio, Raquel ; Osorio, María T

OBJECTIVES:

Tideglusib (Tx) is known for its osteogenic potential, yet its effects on the interplay between osteoblasts and M1 macrophages remain underexplored. This in vitro study aimed to isolate and evaluate both the individual and combined roles of M1 macrophages and osteoblasts in macrophage differentiation and osteoblast function, specifically focusing on how these interactions influence protein expression of osteogenesis and osteoclastogenesis in the presence or absence of Tx.

METHODS:

Osteoblast and macrophage cells were co-cultured in direct contact for 24 and 48 h, with or without the presence of Tx. ALP activity, the expression of inflammatory-related genes using RT-qPCR, and histological analyses were performed.

RESULTS:

Co-culturing osteoblasts and M1 macrophages with Tx increased alkaline phosphatase production, indicative of enhanced osteoblast activity. Histological assessments revealed that Tx treatment contributed to the stability and maintenance of cell morphology within the scaffold, suggesting a supportive environment for cell viability and function. Tx significantly reduced the expression of pro-inflammatory markers, such as TNF-α and IL-1β, in the co-culture at both 24 and 48 h Tx also effectively inhibited osteoclastogenic differentiation in macrophages, thereby diminishing their pro-inflammatory phenotype.

CONCLUSIONS:

Tx increased ALP activity and produced a significant up-regulation of RANKL expression, indicating enhanced osteoblast differentiation and osteoclast activation. Tx mitigates macrophage-driven inflammation.

CLINICAL SIGNIFICANCE:

Tx may enhance bone regeneration by modulating inflammatory responses and preserving cell integrity.

01 Nov 2024·DENTAL MATERIALS

Effect of the anti-Alzheimer drug GSK-3β antagonist on numerical modeling of the energy dissipation through the resin-dentin interface

Article

Author: Fernández-Romero, Enrique ; Toledano, Raquel ; Toledano, Manuel ; Osorio, María T ; Lynch, Christopher D ; Aguilera, Fátima S. ; Osorio, María T. ; Osorio, Raquel ; Osorio, Estrella ; Aguilera, Fátima S ; Lynch, Christopher D.

OBJECTIVES:

The aim of this study was to determine the viscoelastic performance and energy dissipation of conditioned dentin infiltrated with polymeric nanoparticles (NPs) doped with tideglusib (TDg) (TDg-NPs).

METHODS:

Dentin conditioned surfaces were infiltrated with NPs and TDg-NPs. Bonded interfaces were created, stored for 24 h and submitted to mechanical and thermal challenging. Resin-dentin interfaces were evaluated through nano-DMA/complex-loss-storage moduli-tan delta assessment and atomic force microscopy (AFM) analysis.

RESULTS:

Dentin infiltrated with NPs and load cycled attained the highest complex modulus at hybrid layer and bottom of hybrid layer. Intertubular dentin treated with undoped NPs showed higher complex modulus than peritubular dentin, after load cycling, provoking energy concentration and breakdown at the interface. After infiltrating with TDg-NPs, complex modulus was similar between peri-intertubular dentin and energy dissipated homogeneously. Tan delta at intertubular dentin was higher than at peritubular dentin, after using TDg-NPs and load cycling. This generated the widest bandwidth of the collagen fibrils and bridge-like mineral structures that, as sight of energy dissipation, fastened active dentin remodeling. TDg-NPs inducted scarce mineralization after thermo-cycling, but these bridging processes limited breakdown zones at the interface.

SIGNIFICANCE:

TDg-based NPs are then proposed for effective dentin remineralization and tubular seal, from a viscoelastic approach.

01 Nov 2024·JOURNAL OF DENTISTRY

Investigation of the effect of Tideglusib on the hydroxyapatite formation, crystallinity and elasticity of conditioned resin-dentin interfaces

Article

Author: Fernández-Romero, Enrique ; Osorio, María T. ; Toledano, Manuel ; Toledano, Raquel ; Aguilera, Fátima S. ; Aguilera, Fátima S ; Osorio, Raquel ; Osorio, Estrella ; Osorio, María T

OBJECTIVES:

To investigate the effect of dentin infiltration with polymeric nanoparticles (NPs) doped with tideglusib (TDg) (TDg-NPs) on hydroxyapatite formation, crystallinity and elasticity of conditioned resin-dentin interfaces.

METHODS:

Dentin conditioned surfaces were infiltrated with NPs or TDg-NPs. Bonded interfaces were created, stored for 24 h and submitted to mechanical and thermal challenging. Resin-dentin interfaces were evaluated through nanoindentation to determine the modulus of elasticity, X-ray diffraction and transmission electron microscopy through selected area diffraction and bright-filed imaging.

RESULTS:

TDg-NPs provoked peaks narrowing after the diffraction-intensity analysis that corresponded with high crystallinity, with an increased modulus of Young after load cycling in comparison with the samples treated with undoped NPs. New minerals, in the group of TDg-NPs, showed the greatest both deviation of line profile from perfect crystal diffraction and dimension of the lattice strain, i.e., crystallite, grain size and microstrain and 002 plane-texture. The new minerals generated after TDg-NPs application and mechanical loading followed a well defined lineation. Undoped NPs mostly produced small hydroxyapatite crystallites, non crystalline or amorphous in nature with poor maturity.

CONCLUSIONS:

Tideglusib promoted the precipitation of hydroxyapatite, as a major crystalline phase, at the intrafibrillar compartment of the collagen fibrils, enabling functional mineralization. TDg-NPs facilitated nucleation of crystals randomly oriented, showing less structural variation in angles and distances that improved crystallographic relative order of atoms and maturity. Nanocrystals inducted by TDg-NPs were hexagonal prisms of submicron size. Thermal challenging of dentin treated with TDg-NPs have provoked a decrease of functional mineralization and crystallinity, associated to immature hydroxyapatite.

CLINICAL SIGNIFICANCE:

New polycrystalline lattice formation generated after TDg-NPs infiltration may become correlated with high mechanical performance. This association can be inferred from the superior crystallinity that was obtained in presence of tideglusib. Immature crystallites formed in dentin treated with undoped NPs will account for a high remineralizing activity.

News (Medical) associated with Tideglusib

27 Nov 2024

·www.prnewswire.com

Myotonic Dystrophy Clinical Trial Pipeline Boom as Over 20 Companies Leading the Charge in Research and Development | DelveInsight

An increase in research and development (R&D) activities in myotonic dystrophy is expected to drive market growth by enhancing the understanding of the disease, accelerating drug discovery, and creating innovative treatments. With more R&D, new therapeutic options such as gene therapies, antisense oligonucleotides, and targeted small molecules can be developed, addressing the unmet needs in myotonic dystrophy care. Increased R&D also attracts funding and partnerships, fostering a competitive environment that can lead to improved patient outcomes and market expansion. LAS VEGAS, Nov. 27, 2024 /PRNewswire/ -- DelveInsight's ' Myotonic Dystrophy Pipeline Insight 2024 ' report provides comprehensive global coverage of pipeline myotonic dystrophy therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the myotonic dystrophy pipeline domain. Key Takeaways from the Myotonic Dystrophy Pipeline Report DelveInsight's myotonic dystrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for myotonic dystrophy treatment. Key myotonic dystrophy companies such as Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others are evaluating new myotonic dystrophy drugs to improve the treatment landscape. Promising myotonic dystrophy pipeline therapies such as AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others are under different phases of myotonic dystrophy clinical trials. On November 7, 2024, ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of gene expression, announced that the US Food and Drug Administration has granted Rare Pediatric Designation (RPD) to ATX-01 for the treatment of myotonic dystrophy type 1. In November 2024, Dyne Therapeutics announced that the US Food and Drug Administration has cleared the Investigational New Drug application for DYNE-101, which is being evaluated in the ongoing, global Phase I/II ACHIEVE trial in adults with myotonic dystrophy type 1 (DM1). The ACHIEVE trial currently includes 56 participants and is fully enrolled through the 6.8 mg/kg Q8W cohort (approximate ASO dose). In August 2024, PepGen announced that both Health Canada and the United Kingdom Medicines and Healthcare products Regulatory Agency have cleared the Company's clinical trial application (CTA) submissions for the FREEDOM2 trial, and PepGen expects to initiate patient dosing in the second half of 2024. FREEDOM2 is a Phase 2 randomized, double-blind, placebo-controlled, MAD clinical trial evaluating PGN-EDODM1 in approximately 24 adult patients with DM1 in Canada, the United Kingdom, and in the United States, subject to regulatory clearance for the treatment of myotonic dystrophy. In May 2024, Avidity Biosciences announced that the US Food and Drug Administration granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1. In April 2024, PepGen presented a poster on the PGN-EDODM1 program at the 14th International Myotonic Dystrophy Consortium (IDMC-14) Meeting titled "FREEDOM-DM1: Phase 1 Study Design to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 for Myotonic Dystrophy Type 1." In March 2024, Entrada achieved a milestone under its global collaboration with Vertex related to the clinical advancement of Vertex's Phase I/II clinical trial of VX-670, which triggered a USD 75 million payment. The Company expects to receive this payment in the second quarter of 2024. The Company is eligible to receive up to USD 485 million, inclusive of milestones achieved to date, for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that may result from this collaboration agreement. In February 2024, PepGen Inc. announced that the US Food and Drug Administration had granted Fast Track designation to PGN-EDODM1, an investigational candidate for the treatment of myotonic dystrophy type 1. In January 2024, Juvena Therapeutics receives the FDA Orphan Drug Designation for JUV-161 for the treatment of Myotonic Dystrophy Type 1. Request a sample and discover the recent advances in myotonic dystrophy treatment drugs @ Myotonic Dystrophy Pipeline Report The myotonic dystrophy pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage myotonic dystrophy drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the myotonic dystrophy clinical trial landscape. Myotonic Dystrophy Overview Myotonic dystrophy is a genetic disorder characterized by progressive muscle wasting and weakness. It is caused by mutations in specific genes that lead to an abnormal expansion of repetitive DNA sequences. The two main types of myotonic dystrophy are Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2). DM1 is caused by a CTG repeat expansion in the DMPK gene, while DM2 is linked to a CCTG repeat expansion in the CNBP gene. Symptoms of myotonic dystrophy can vary but often include muscle stiffness (myotonia), weakness, and atrophy, primarily affecting the distal muscles and those of the face. Additional symptoms may involve cataracts, cardiac arrhythmias, endocrine issues, and cognitive difficulties. Diagnosis typically involves a combination of clinical evaluation, family history assessment, and genetic testing to identify the specific mutations. Electromyography can also be used to detect myotonia. Currently, there is no cure for myotonic dystrophy, and treatment focuses on managing symptoms. This may include physical therapy to maintain muscle function, medications to alleviate myotonia, and regular monitoring for cardiac issues. Genetic counseling is recommended for affected individuals and their families to understand the inheritance patterns and potential risks. Find out more about myotonic dystrophy treatment drugs @ Drugs for Myotonic Dystrophy Treatment A snapshot of the Myotonic Dystrophy Pipeline Drugs mentioned in the report: Learn more about the emerging myotonic dystrophy pipeline therapies @ Myotonic Dystrophy Clinical Trials Myotonic Dystrophy Therapeutics Assessment The myotonic dystrophy pipeline report proffers an integral view of the myotonic dystrophy emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Myotonic Dystrophy Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Oral, Intravenous, Subcutaneous, Parenteral, Topical Therapeutics Assessment By Molecule Type: Recombinant fusion proteins, Small molecule, Monoclonal antibody, Peptide, Polymer, Gene therapy Therapeutics Assessment By Mechanism of Action: Myotonin protein kinase expression inhibitors, RNA interference, Sodium channel antagonists, Glycogen synthase kinase 3 beta inhibitors, Histamine H3 receptor antagonists, Histamine H3 receptor inverse agonists, MicroRNA inhibitor, RNA inhibitors, RNA splicing modulators Key Myotonic Dystrophy Companies: Avidity Biosciences, Lupin, AMO Pharma, Harmony Biosciences, Arthex Biotech, Dyne Therapeutics, Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, Inc., Pepgen Corporation, Juvena Therapeutics, EditForce, Inc., Kinea Bio, and others. Key Myotonic Dystrophy Pipeline Therapies: AOC 1001, Mexiletine, Tideglusib, Pitolisant, ATX-01, DYNE-101, VX-670, ARO-DM1, PGN EDODM1, JUV 161, EF-210, KNA129, and others. Dive deep into rich insights for new drugs for myotonic dystrophy treatment, visit @ Myotonic Dystrophy Drugs Table of Contents For further information on the myotonic dystrophy pipeline therapeutics, reach out @ Myotonic Dystrophy Treatment Drugs Related Reports Myotonic Dystrophy Market Myotonic Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myotonic dystrophy companies, including Lupin, AMO Pharma, Harmony Biosciences, Avidity Biosciences, Dyne Therapeutics, Nexien BioPharma, Locana Inc., Entrada Therapeutics, Arthex Biotech, NeuBase Therapeutics, NeuBase Therapeutics, Enzerna, Astellas Gene Therapies, Dyne Therapeutics, Pepgen Corporation, Sangamo Therapeutics, Syros Pharmaceuticals, among others. Myotonic Dystrophy Epidemiology Forecast Myotonic Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted myotonic dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Duchenne muscular dystrophy companies, including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics , among others. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Fast TrackPhase 2Orphan Drug

04 Oct 2024

·www.pharmaceutical-technology.com

FDA lifts clinical hold on Avidity’s lead antibody conjugate therapy trial

Earlier this year, Avidity started a pivotal Phase III HARBOR trial evaluating del-desiran inpatients with DM1. Image credit: JHVEPhoto /Shutterstock. The US Food and Drug Administration (FDA) has lifted the partial clinical hold on Avidity Biosciences ’ investigational drug, delpacibart etedesiran (del-desiran). In 2022, the US regulatory agency placed a partial hold after a report of a serious adverse event in a newly enrolled patient in the Phase I/II MARINA trial (NCT05027269), which evaluated del-desiran in patients myotonic dystrophy type 1 (DM1). The company did not disclose the details of the adverse event and the US FDA eased the partial clinical hold the following year, allowing the company to recruit new patients. The clinical hold has not slowed down Avidity’s efforts to advance the clinical development of del-desiran in DM1. Earlier this year, Avidity started a pivotal placebo-controlled Phase III HARBOR trial (NCT06411288). The study is expected to enrol 150 participants with DM1 who will be assessed over 54 weeks. The trial’s primary endpoint is the improvement of myotonia over 30 weeks, measured using video Hand Opening Time (vHOT), the average time to extend the right third and first digits. The study’s secondary endpoints also include the functional assessment of myotonia across 30 weeks, including hand grip strength, quantitative muscle testing composite score and Myotonic Dystrophy Type 1 activity and participation scale c. Del-desiran is an antibody oligonucleotide conjugate (AOC) made up of a monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting myotonic dystrophy protein kinase (DMPK) mRNA, responsible for producing DMPF protein. If approved, GlobalData expects del-desiran to pull in over $1.2bn in sales by 2030. See Also: BMS receives FDA approval for Opdivo to treat NSCLC Novaliq and Laboratoires Théa announce EC approval for dry eye disease therapy GlobalData is the parent company of Pharmaceutical Technology . mRNA vaccines were instrumental during the Covid-19 pandemic. Since then, there has been increased interest in mRNA therapies beyond the vaccines. GlobalData forecasts the mRNA non-vaccine therapeutics field to reach $2bn by 2028. DM1 is a genetic disorder caused by a triplet-repeat in the DMPK gene. It primarily affects skeletal and cardiac muscle causing muscle weakness and wasting leading to respiratory problems, and cardiac abnormalities. There are no approved treatments for DM1 and management is mostly aimed at symptomatic treatment. Another therapy in development for treating DM1 is AMO Pharma’s AMO-02 (tideglusib). The therapy is an irreversible inhibitor of glycogen synthase kinase 3 (GSK-3), it has the potential to disrupt the RNA repeats that cause congenital DM1. In May, AMO announced plans to start a Phase III trial for AMO-02 in DM1 patients.

Phase 3Drug ApprovalOligonucleotidemRNAVaccine

02 May 2024

·www.prnewswire.com

AMO Pharma Completes Meeting with U.S. FDA and Outlines Plans to Advance Clinical Development of AMO-02 (tideglusib) in Treatment of Myotonic Dystrophy

Company will conduct Phase 3 trial of AMO-02 (tideglusib) in adults to support a future submission for marketing authorization in Type 1 myotonic dystrophy in both adult and pediatric indications. LONDON, May 2, 2024 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare genetic disorders with limited or no treatment options, today provided an update on plans to advance the clinical development program for AMO-02 (tideglusib), the company's investigational therapy in development for the treatment of Type 1 myotonic dystrophy (DM1), following a recent meeting with the U.S. Food and Drug Administration (FDA). Based on the input from the meeting, AMO Pharma will conduct a Phase 3 clinical trial to assess the efficacy and safety of AMO-02 in adult patients with adult-onset DM1 to support a future submission for marketing authorization for the treatment of DM1 in children, adolescents and adults. Continue Reading REACH-CDM (PRNewsfoto/AMO Pharma Limited) Results from AMO Pharma's previous REACH-CDM Phase 2/3 clinical study (the "REACH-CDM Study") involving children and adolescents with congenital-onset DM1 showed that AMO-02 was generally safe and well tolerated during the study, with no reported severe adverse events (SAEs) related to treatment. The primary and secondary outcome measures based on clinician administered subjective ratings showed an unexpected placebo effect that may have masked some beneficial effects of treatment. Further post hoc analysis as reviewed with the FDA indicates that there is sufficient evidence to support the continued development of tideglusib for the treatment of DM1 with agreement that AMO Pharma will now advance a Phase 3 study in adults with the adult-onset version of DM1. During the meeting, FDA reviewed data from the REACH-CDM Study to assess the efficacy and safety of treatment with AMO-02 in children and adolescents with congenital DM1. The FDA also gave feedback on the design of the new Phase 3 clinical trial for adult patients with adult onset DM1. In this study in adult onset DM1, outcome measures will be similar to those that showed benefit in the REACH-CDM Study. Following discussion with the FDA, AMO Pharma will plan a future submission for approval in children, adolescents and adults with myotonic dystrophy based on a package of safety and efficacy data from the new Phase 3 trial in adults combined with efficacy, pharmacokinetic and safety data from AMO Pharma's REACH-CDM Study. "We are pleased with the outcome of the productive discussions we had with the FDA regarding next steps for AMO-02 and continue to feel strongly that this innovative therapy can represent a significant advance in the treatment of patients with DM1, whether they are children, adolescents or adults," said Alan Rubino, executive chair at AMO Pharma. "We are now focused on progressing a new clinical study in adults with adult-onset DM1. We are very grateful to the FDA and to the patients, families, clinicians and investors who have supported our research thus far and we look forward to working with the community to advance this program as rapidly as possible. We will make an announcement regarding the start date and site locations for the adult onset DM1 study shortly." "The results of the REACH-CDM Study showed measures of efficacy benefit across multiple symptom areas, and we are very excited that this clinical development program will continue to move forward," said Hanns Lochmuller, investigator in the REACH-CDM Study at Children's Hospital of Eastern Ontario, Canada. "We remain very hopeful that this investigational therapy can have a transformative impact on the health of patients living with this ultra-rare, severe muscular dystrophy in the years ahead." About AMO-02 AMO-02 (tideglusib) is an investigational therapy in development for the treatment of DM1 with a dual mechanism disrupting the pathogenic RNA repeat in DM1and inhibiting excess levels of the kinase GSK3β. The REACH-CDM Study was a double-blind, placebo-controlled, randomized study in children and adolescents with congenital DM1 intended to support a future submission for marketing authorization in congenital DM1. The study included 56 participants at leading specialist sites in the US, Canada, New Zealand and Australia. Data associated with functional and objective measures were collected during the study, including motor and muscle assessments (10 meter walk/run, myometry, lip strength and lean muscle mass as assessed by DXA scan), cognitive performance (Peabody Picture Vocabulary Test, NIH Toolbox Picture Sequence Memory Test, NIH Toolbox Dimensional Change Card Sort Test), adaptive (real world) function (Vineland Adaptive Behavior Communication Scale, Vineland Adaptive Behavior Daily Living Scale, Vineland Adaptive Behavior Socialization Scale), bone density (DXA scan) and creatine kinase measurements. In post-hoc analyses more participants showed a positive response following AMO-02 treatment than placebo on 10 of the 12 of these measures. Treatment with AMO-02 was associated with clinically significant improvements in walking and cognition and significant effects on objective biomarkers of muscle and neuronal integrity. AMO-02 also showed significant improvement compared to placebo on a Multi-Domain Responder Index integrating clinically significant responses on measures of ambulation, muscle strength, cognitive performance, activities of daily living and an objective biomarker. Primary analyses of subjective rating assessments showed a placebo effect. Patients from this study and treatment naïve patients with congenital or juvenile onset DM1 are currently being supported in the REACH CDM-X open label extension safety study. About AMO Pharma AMO Pharma is a clinical-stage specialty biopharmaceutical company working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare and severe childhood onset neurogenetic disorders with limited or no treatment options. In addition to developing AMO-02 for DM1, the company is also progressing AMO-01 as a clinical stage treatment for Phelan-McDermid syndrome and AMO-04 as a clinic-ready potential medicine for Rett syndrome and related disorders. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world. Advice provided to AMO Pharma by regulators is under the condition that any scientific advice given is not legally binding with regards to any future application for the product concerned. Furthermore, advice cannot be taken as indicative of any future agreed position. For more information, please visit the AMO Pharma website at . Media contact: Holly Stevens Berry & Company Public Relations [email protected] 212-253-8881 SOURCE AMO Pharma Limited

Phase 3Clinical ResultPhase 2

100 Deals associated with Tideglusib

External Link

KEGG	Wiki	ATC	Drug Bank
-	Tideglusib	-	DB12129

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myotonic Dystrophy	Phase 3	US	AMO Pharma Ltd.Startup	03 Mar 2021
Myotonic Dystrophy	Phase 3	AU	AMO Pharma Ltd.Startup	03 Mar 2021
Myotonic Dystrophy	Phase 3	CA	AMO Pharma Ltd.Startup	03 Mar 2021
Myotonic Dystrophy	Phase 3	NZ	AMO Pharma Ltd.Startup	03 Mar 2021
Myotonic Dystrophy	Phase 3	GB	AMO Pharma Ltd.Startup	03 Mar 2021
Alzheimer Disease	Phase 2	BE	Noscira SA	01 Apr 2011
Alzheimer Disease	Phase 2	FI	Noscira SA	01 Apr 2011
Alzheimer Disease	Phase 2	FR	Noscira SA	01 Apr 2011
Alzheimer Disease	Phase 2	DE	Noscira SA	01 Apr 2011
Alzheimer Disease	Phase 2	ES	Noscira SA	01 Apr 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03692312 (REACH CDM, PRNewswire) Manual	Phase 2/3	Myotonic Dystrophy	56	AMO-02	aaaahixbcd(dyjabbozfh): P-Value = <0.05	Positive	06 Sep 2023
				placebo
NCT02858908 (Pubmed \| Pediatr Neurol) Manual	Phase 2	Myotonic Dystrophy	16	tideglusib	clevlhktgp(dowwmzwepl) = oqjikpwxwu rzusbdoaow (vvymgcvsdf )	Positive	01 Nov 2020
TAUROS trial (AAIC2012)	Not Applicable	Supranuclear Palsy, Progressive	-	Tideglusib	xkvizygzba(xzgpairbcc) = zcrackviax qozkoopebh (pbjrthxwta ) View more	-	01 Jul 2012
				Placebo	xkvizygzba(xzgpairbcc) = npnudbcdpr qozkoopebh (pbjrthxwta ) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis