Healx
IMPACT-FXS clinical trial
Fragile X syndrome (FXS) is a rare neurodevelopmental condition and is the most common genetic cause of learning difficulties in the world, but there are currently no effective or approved therapies for the condition available.
We are sponsoring a Phase 2 clinical trial (clinicaltrials.gov identifier NCT04823052) to explore the safety and efficacy of several potential medications for fragile X syndrome.
The IMPACT-FXS (Investigating Multiple Pathways And Combined Treatments for FXS) study is a 14 week randomised, placebo-controlled, parallel-group study that consists of a 2-week screening period, continued by 10-week period of treatment (placebo or one of the potential medications) and closed with a 2-week follow-up period.
This trial has an umbrella design, meaning that not one, but several drug candidates will be evaluated under the same protocol and against a placebo for FXS. The first stage will investigate two drug candidates, HLX-0201 and HLX-0206, and then, in the next stage, we are aiming to add other candidates. This innovative approach and trial design allows us to identify the best mono- and/or combination therapy candidates and evaluate them in upcoming trials.
Where is the trial taking place?
Across several sites in the United States.
Recruiting
Not yet recruiting
University of Colorado
School of Medicine,
Children’s Hospital Colorado (Colorado)
School of Medicine,
Children’s Hospital Colorado (Colorado)
Rush University
Medical Center
(Illinois)
Medical Center
(Illinois)
University of Massachusetts
Medical School (Massachusetts)
Medical School (Massachusetts)
Emory University
(Georgia)
Mt Sinai Hospital
(Miami)
University of Miami,
Mailman Centre for Child Development
(Miami)
Where can I learn more?
If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.
The science behind our FXS programme
We use artificial intelligence (AI) and machine learning algorithm methods, as well as in-house drug discovery expertise and patient group insights, to identify novel therapeutic opportunities from known compounds. This enables us to rapidly progress therapies to patients.
HLX-0201 was identified as a potential therapy for FXS by one of our proprietary drug discovery methods, Drug-Gene Expression Matching (DGEM). DGEM compares the gene expression profile for a disease with the gene expression profiles from our curated drug database to identify entirely novel connections between the two. The method works on the premise that a drug mechanism with the opposite mechanism profile to a disease would be a strong candidate for an effective treatment. HLX-0206 was identified as a potential combination partner to HLX-0201 by our proprietary combination prediction methods.
Several other compounds identified by our AI methods are currently being progressed through preclinical validation to uncover possible combinations and novel mechanisms of action. These compounds will join the umbrella study in the coming months.
Throughout the drug discovery phase we worked closely with a number of patient groups who contributed invaluable disease expertise, connections with fragile X researchers and a process for preclinical and clinical validation.
Several other compounds identified by our AI methods are currently being progressed through preclinical validation to uncover possible combinations and novel mechanisms of action. These compounds will join the umbrella study in the coming months.
Throughout the drug discovery phase we worked closely with a number of patient groups who contributed invaluable disease expertise, connections with fragile X researchers and a process for preclinical and clinical validation.
Where can I learn more?
If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.
What is fragile X syndrome?
Fragile X syndrome is a rare neurodevelopmental condition and the most common inherited cause of learning difficulties in the world, affecting roughly 1 in 4,000 males and 1 in 8,000 females.
It is caused by a fault in the FMR1 gene, which is responsible for making a protein (FMRP) that is needed for normal brain development. In patients with fragile X syndrome, the protein isn’t made and the brain doesn’t develop as it should.
Prevalence
Affects 1 / 4,000 males
and 1 / 8,000 females
Causes
Caused by a DNA repeat in FMR1 gene (X chromosome) that means a protein required for normal brain development isn’t made
Therapies
There are no approved treatments but there is medication to minimise certain symptoms
Symptoms
Common symptoms of fragile x include intellectual disabilities; attention deficit and hyperactivity; anxiety; speech delays; and sensory integration problems.
Where can I learn more?
If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.
US patient groups
Publications
1 November 2019
Deep bidirectional transformers for relation extraction without supervision
1 March 2019
Repurposing available drugs for neurodevelopmental disorders: the fragile X experience
1 October 2018
Using machine learning to predict synergistic antimalarial compound combinations with novel structures
30 August 2018
Understanding and predicting disease relationships through similarity fusion
1 June 2018
The antidepressant tianeptine reverts synaptic AMPA receptor defects caused by deficiency of CDKL5
1 February 2018
Integrating splice-isoform expression into genome-scale models characterizes breast cancer metabolism
More resources
Where can I learn more?
If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.
Dr Mike TranfagliaWe are excited about the potential of HLX-0201: a proven, safe drug with a long track record of effectiveness. It never would have occurred to us to investigate the compound for fragile X without Healx’s innovative technology pointing us in the right direction, but the preclinical studies have shown remarkable effectiveness.