An Open-label Study to Investigate the Safety, Tolerability and Efficacy of a Single 6-hour Intravenous Infusion of AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy

The purpose of this study is to investigate the safety, tolerability and efficacy of a single 6-hour intravenous infusion of AMO-01 to treat adolescents and adults with PMS and co-morbid epilepsy. Phelan-McDermid Syndrome (PMS) is a neurodevelopmental disorder characterized by a chromosomal deletion or mutation at 22q13.3 that contains the SHANK3/ProSAP2 gene. A key co-morbidity in PMS is the presence of epilepsy. Currently there are no approved treatments for PMS. Furthermore, there has been relatively little clinical study of pharmacological interventions for PMS. AMO-01 may provide benefit to PMS patients exhibiting behavioral abnormalities and seizures.

Start Date30 May 2018

Sponsor / Collaborator

Icahn School of Medicine at Mount Sinai

NCT00730262 / TerminatedPhase 2

A Phase II Study of TLN-4601 in Patients With Glioblastoma Multiforme

The objective of this study is to assess the efficacy and safety of TLN-4601 used to treat patients with Glioblastoma Multiforme(GBM) that recur/progress after receiving first line systemic therapy post surgery/radiotherapy.

Start Date01 Aug 2008

Sponsor / Collaborator

Thallion Pharmaceuticals, Inc.

NCT00338026 / CompletedPhase 1

A Phase I, Dose Finding, Pharmacokinetic, and Safety Study of Continuous IV Infusion of ECO-4601 in Patients With Advanced Cancer Including an Extension Portion to Obtain Safety Data at the Highest Tolerable Dose

The purpose of this study was to determine the maximum tolerated dose (MTD) and the recommended dose for future studies of ECO-4601 administered as a continuous IV infusion for 14 days with 7 days recovery (21 day cycle) in patients with histologically confirmed solid tumors (high grade glioma, colorectal, lung, breast, ovarian, pancreatic and prostate). This study was also designed to determine the clinical pharmacokinetic profile, safety of multiple cycles of administration, and document the antitumor activity of ECO-4601.

Start Date01 Feb 2006

Sponsor / Collaborator

Thallion Pharmaceuticals, Inc.

100 Clinical Results associated with Diazepinomicin

100 Translational Medicine associated with Diazepinomicin

100 Patents (Medical) associated with Diazepinomicin

Literatures (Medical) associated with Diazepinomicin

01 Jan 2022·Bioorganic & Medicinal Chemistry Letters

Synthesis and biological evaluation of BU-4664L derivatives as potential anticancer agents

Article

Author: Dong, Yue-Hui ; Liu, Zhao-Peng ; Wen, Zhao-Hui ; Liu, Chao ; Xu, Yuan-Yuan

BU-4664L is a naturally occurring N-farnesylated dibenzodiazepinone with important biological activities. Herein, we report the synthesis and antitumor evaluation of two series of BU-4664L derivatives bearing different substituent patterns on the dibenzodiazepinone core and with diverse side chains. All of the derivatives displayed micromolar activity against the human prostate cancer PC-3 cells, while lower or no activity against the human lung H460 cells. The most active derivatives were 10a and 16c which exerted antiproliferative activity against PC-3 cells with GI₅₀ values of 5.66 and 5.94 μM, respectively, and thus represent promising lead compounds for further development.

01 Dec 2018·Cell Communication and SignalingQ2 · BIOLOGY

Overexpression of miR-1 in the heart attenuates hippocampal synaptic vesicle exocytosis by the posttranscriptional regulation of SNAP-25 through the transportation of exosomes

Q2 · BIOLOGY

ArticleOA

Author: Zhang, Xin-Yu ; Su, Dan ; Sun, Lin-Lin ; Duan, Ming-Jing ; Ma, Ji-Chao ; Ai, Jing ; Mao, Meng ; Wang, Qin ; Huang, Si-Yu ; Yan, Mei-Ling ; Qu, Yang ; Sun, Qiang ; Li, Ke-Xin ; Ban, Tao

BACKGROUNDThe link between cardiac diseases and cognitive deterioration has been accepted from the concept of "cardiogenic dementia", which was proposed in the late 1970s. However, the molecular mechanism is unclarified.METHODSThe two animal models used in this study were cardiac-specific overexpression of microRNA-1-2 transgenic (Tg) mice and a myocardial infarction mouse model generated by left coronary artery ligation (LCA). First, we observed the microRNA-1 (miR-1) level and synaptic vesicles (SV) distribution in the hippocampus using in situ hybridization and transmission electron microscopy (TEM) and evaluated the expression of vesicle exocytosis related proteins by western blotting. Second, we used dual luciferase reporter assay as well as antagonist and miRNA-masking techniques to identify the posttranscriptional regulatory effect of miR-1 on the Snap25 gene. Third, FM1-43 staining was performed to investigate the effect of miR-1 on synaptic vesicle exocytosis. Lastly, we used GW4869 to inhibit the biogenesis and secretion of exosomes to determine the transportation effect of exosomes for miR-1 from the heart to the brain.RESULTSCompared with the levels in age-matched WT mice, miR-1 levels were increased in both the hearts and hippocampi of Tg mice, accompanied by the redistribution of SVs and the reduction in SV exocytosis-related protein SNAP-25 expression. In vitro studies showed that SNAP-25 protein expression was down- or upregulated by miR-1 overexpression or inhibition, respectively, however, unchanged by miRNA-masking the 3'UTR of the Snap25 gene. SV exocytosis was inhibited by miR-1 overexpression, which could be prevented by co-transfection with an anti-miR-1 oligonucleotide fragment (AMO-1). The knockdown of miR-1 by hippocampal stereotaxic injection of AMO-1 carried by a lentivirus vector (lenti-pre-AMO-1) led to the upregulation of SNAP-25 expression and prevented SV concentration in the synapses in the hippocampi of Tg mice. The application of GW4869 significantly reversed the increased miR-1 level in the blood and hippocampi as well as reduced the SNAP-25 protein levels in the hippocampi of both Tg and LCA mice.CONCLUSIONThe overexpression of miR-1 in the heart attenuated SV exocytosis in the hippocampus by posttranscriptionally regulating SNAP-25 through the transportation of exosomes. This study contributes to the understanding of the relationship between cardiovascular disease and brain dysfunction.

28 Sep 2018·Journal of Natural ProductsQ2 · BIOLOGY

Complete Genome of Micromonospora sp. Strain B006 Reveals Biosynthetic Potential of a Lake Michigan Actinomycete

Q2 · BIOLOGY

Article

Author: Crnkovic, Camila M. ; Eustáquio, Alessandra S. ; Orjala, Jimmy ; Maienschein-Cline, Mark ; Green, Stefan J. ; Kunstman, Kevin J. ; Murphy, Brian T. ; Braesel, Jana

Actinomycete bacteria isolated from freshwater environments are an unexplored source of natural products. Here we report the complete genome of the Great Lakes-derived Micromonospora sp. strain B006, revealing its potential for natural product biosynthesis. The 7-megabase pair chromosome of strain B006 was sequenced using Illumina and Oxford Nanopore technologies followed by Sanger sequencing to close remaining gaps. All identified biosynthetic gene clusters (BGCs) were manually curated. Five known BGCs were identified encoding desferrioxamine, alkyl- O-dihydrogeranylmethoxyhydroquinone, a spore pigment, sioxanthin, and diazepinomicin, which is currently in phase II clinical trials to treat Phelan-McDermid syndrome and co-morbid epilepsy. We report here that strain B006 is indeed a producer of diazepinomicin and at yields higher than previously reported. Moreover, 11 of the 16 identified BGCs are orphan, eight of which were transcriptionally active under the culture condition tested. Orphan BGCs include an enediyne polyketide synthase and an uncharacteristically large, 36-module polyketide synthase-nonribosomal peptide synthetase BGC. We developed a genetics system for Micromonospora sp. B006 that will contribute to deorphaning BGCs in the future. This study is one of the few attempts to report the biosynthetic capacity of a freshwater-derived actinomycete and highlights this resource as a potential reservoir for new natural products.

News (Medical) associated with Diazepinomicin

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

15 Feb 2024

·www.prnewswire.com

AMO Pharma Announces Collaboration with Population Health Research Institute to Advance Proof of Concept Clinical Trial to Assess Efficacy of Tideglusib in Treatment of Arrhythmogenic Cardiomyopathy

LONDON, Feb. 15, 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 announced a collaboration with Population Health Research Institute (PHRI), a joint institute of McMaster University and Hamilton Health Sciences in Canada, to advance a clinical proof of concept trial to assess the efficacy of AMO-02 (tideglusib), AMO Pharma's investigational oral glycogen synthase kinase 3 beta (GSK3β) inhibitor, in the treatment of genotype positive arrhythmogenic cardiomyopathy (ACM). The study, entitled "Targeted Therapy with Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy" (TaRGET), will be led by the research team at PHRI and will involve collaboration with multiple organizations in addition to AMO Pharma including the Hearts in Rhythm Organization (HiRO). Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio. "It can be a helpless feeling to carry a gene for ACM and know that, no matter how you live your life, you may someday develop a deadly form of heart disease," said Jason Roberts, scientist at PHRI and TaRGET study principal investigator, adding, "Encouraged by earlier studies in animal models, we are hopeful that tideglusib may serve as an effective treatment that can dramatically improve the health of people living with ACM. The TaRGET trial is a critical first step in evaluating this potential." ACM is a form of genetic heart disease characterized by scarring in the heart muscle. This scarring increases the risk of dangerously fast heart rhythms that can lead to sudden cardiac death (SCD). There are currently no approved medical therapies that have been shown to prevent progression of ACM. An implantable cardioverter defibrillator (ICD) is recommended for prevention of SCD for ACM patients considered at high risk for SCD. In prior research, a high-throughput screen of bioactive compounds against a zebrafish model of ACM identified a small molecule classified as a GSK3β inhibitor that successfully prevented and rescued the phenotype, findings that have subsequently been reproduced in a series of ACM murine models. GSK3β is an enzyme that modulates the activity of a broad spectrum of intracellular signaling pathways, including the canonical Wnt/β-catenin pathway whose suppression has been suggested to exert an important role in ACM pathogenesis. "As we continue to advance our research to assess the potential benefits of AMO-02 in the treatment of myotonic dystrophy, we are very pleased to be supporting this landmark research effort in ACM with the outstanding team at PHRI," said Mike Snape, chief scientific officer at AMO Pharma. "Based on extensive research that highlights the proven MOA of AMO-02 as a clinically safe and well tolerated GSK3β inhibitor, we look forward to progress in advancing this promising program that can bring new hope to people living with ACM and their families in the years ahead. This new data has the potential to build on the cardiac efficacy data previously reported in studies conducted in Duchenne muscular dystrophy by Professor Val Fajardo's team at Brock University." 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 myotonic dystrophy, 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, neither on the part of MHRA/Commission on Human Medicines (CHM) nor on the Company. 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 212 253 8881 [email protected] Logo -

Clinical Study

19 Sep 2023

·www.prnewswire.com

AMO Pharma Announces Preclinical Data Showing AMO-02 Improves Muscle Function, Glucose Handling and CNS function in Mouse Models of Duchenne Muscular Dystrophy

Findings from research collaboration with Brock University also show improved function in metabolic area and skeletal and cardiac muscle. LONDON, Sept. 19, 2023 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held clinical-stage specialty biopharmaceutical company focusing on rare childhood-onset neurogenetic disorders with limited or no treatment options, today announced initial preclinical data from a study of the use of the company's investigational therapy AMO-02 in the treatment of Duchenne muscular dystrophy (DMD). In a mouse-model study conducted in collaboration with Brock University, treatment with AMO-02 was associated with improvements in muscle function and glucose handling as well as improvements in cardiac muscle function and CNS function. In January 2023, Brock University and AMO Pharma announced initiation of a research partnership supported by a grant from the Brock-Niagara Validating, Prototyping and Manufacturing Institute (VPMI) to investigate the potential utility of GSK3β inhibitors in the treatment of DMD, a rare genetic disorder characterized by progressive muscle degeneration and weakness. A team of researchers at Brock University led by professors Val Fajardo, PhD, and Rebecca MacPherson, PhD, investigated the effects of the GSK3β inhibitor AMO-02 (tideglusib) and a follow-on compound in the mdx.D2 mouse, a well characterized, naturally occurring murine model of DMD. Studies were conducted with one month of oral treatment initiated at either the start of the myodegenerative phase in mice (six to eight weeks) or at an advanced stage of disease progression (24 to 26 weeks). Assessments during treatment investigated the entire disease phenotype rather than being limited to solely muscle function and biomarker expression. Initial studies showed that unlike chronic glucocorticoid treatment in mdx mice, which can acutely improve muscle function while dampening insulin sensitivity, AMO-02 treatment improved both muscle function and glucose handling and showed improved function in metabolic areas, muscle fat deposition and cardiac muscle. These benefits were seen in both early and late stage mdx mice. An additional study led by Dr. MacPherson examined the effects of AMO-02 treatment on cognitive function, as pathology seen in disorders similar to Alzheimer's disease has been previously detected in mdx mice2. These results showed that short-term treatment with AMO-02 significantly improved cognitive function. "These data are important in several ways, particularly in showing the broad profile of efficacy in the mdx mouse at all stages of the degenerative process. Benefit was seen in multiple organ domains including heart and brain as well as in metabolic function, skeletal muscle, and glucose and fat handling," said Dr. Fajardo. "These studies further reaffirm the strong potential of AMO-02 in treating the muscle damage and weakness that occurs with Duchenne muscular dystrophy and other muscle-wasting conditions, as well as the potential to improve cardiac and skeletal muscle health and function. We look forward to building a larger research collaboration with Brock and extend our gratitude to Drs. Fajardo and MacPherson and their teams for their outstanding work," said Michael Snape, PhD, AMO Pharma Chief Scientific Officer. AMO Pharma also recently announced results from the REACH-CDM clinical study of the investigational therapy AMO-02 in the treatment of children and adolescents with congenital myotonic dystrophy that showed statistically and clinically significant efficacy benefits in multiple areas including cognitive performance, reduction in biomarkers and improvements in walk/run measurements. The company plans to meet with regulators to outline a path forward for the development of AMO-02, including plans for study in patients with adult-onset myotonic dystrophy. 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 congenital myotonic dystrophy, 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, neither on the part of MHRA/Commission on Human Medicines (CHM) nor on the Company. Furthermore, advice cannot be taken as indicative of any future agreed position. For more information, please visit the AMO Pharma website at . References Bianca M. Marcella, Briana L. Hockey, Jessica L. Braun, Kennedy C. Whitley, Mia S. Geromella, Ryan W. Baranowski, Colton J.F. Watson, Sebastian Silvera, Sophie I. Hamstra, Luc J. Wasilewicz, Robert W.E. Crozier, Amelie Marais, Rene Vandenboom, Brian D. Roy, Adam J. MacNeil, Rebecca E.K. MacPherson, Val A. FajardoGSK3 inhibition improves skeletal muscle function and whole-body metabolism in the severe DBA/2J mdx mouse model bioRxiv 2022.02.16.480726 Hayward GC, Caceres D, Copeland EN, Baranowski BJ, Mohammad A, Whitley KC, Fajardo VA, MacPherson REK. Characterization of Alzheimer's disease-like neuropathology in Duchenne's muscular dystrophy using the DBA/2J mdx mouse model. FEBS Open Bio. 2022 Jan;12(1):154-162. doi: 10.1002/2211-5463.13317. Epub 2021 Nov 11. PMID: 34668666; PMCID: PMC8727939. Media contact: Bill Berry Berry & Company Public Relations [email protected] 212 253 8881 Logo - SOURCE AMO Pharma Limited

Clinical Result

100 Deals associated with Diazepinomicin

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB12420

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Phelan-McDermid syndrome	Phase 2	-	BELLUS Health, Inc.	-
Glioblastoma Multiforme	Phase 1	CA	Thallion Pharmaceuticals, Inc.	01 Aug 2008
Glioblastoma Multiforme	Phase 1	US	Thallion Pharmaceuticals, Inc.	01 Aug 2008
Recurrent Glioblastoma	Phase 1	US	Thallion Pharmaceuticals, Inc.	01 Aug 2008
Recurrent Glioblastoma	Phase 1	CA	Thallion Pharmaceuticals, Inc.	01 Aug 2008
Fragile X Syndrome	Phase 1	-	BELLUS Health, Inc.	-
Epilepsy	Discovery	US	Icahn School of Medicine at Mount Sinai	30 May 2018
Breast Cancer	Discovery	CA	Thallion Pharmaceuticals, Inc.	01 Feb 2006
Ovarian Cancer	Discovery	CA	Thallion Pharmaceuticals, Inc.	01 Feb 2006
Fragile X Syndrome	Discovery	US	AMO Pharma Ltd.Startup	-

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03493607 (CTgov)	Phase 2	Epilepsy \| Phelan-McDermid syndrome	6	AMO-01	olxvodozpj(ghowkdspqr) = qvemseqpvd lyofpijqzh (yqhwhndcqn, oqijtzspsl - wvaekjsxlw) View more	-	22 Jun 2021

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