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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.

Our IMPACT-FXS clinical trial

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)
Rush University
Medical Center
(Illinois)
University of Massachusetts
Medical School
(Massachusetts)
Emory University (Georgia)
Mt Sinai Hospital (Miami)
University of Miami, Mailman Centre for Child Development (Miami)
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Where can I learn more?

If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.

Background to the trial

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.

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Where can I learn more?

If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.

About fragile X syndrome

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.

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Where can I learn more?

If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.

Resources

US patient groups

FRAXA Research Foundation
USA
National Fragile X Foundation
USA

Publications

21 April 2022

Slot Filling for Biomedical Information Extraction

Yannis Papanikolaou
Marlene Staib,
Justin Grace
Francine Bennett
Read More

25 June 2020

Data augmented relation extraction (DARE) with GPT-2

Yannis Papanikolaou
Andrea Pierleoni
Read More

20 April 2020

Drug combination modeling

Anna H C Vlot
Daniel J Mason
Krishna C Bulusu
Andreas Bender
Read More

1 November 2019

Reasoning over paths via knowledge base completion

Saatviga Sudhahar
Ian Roberts
Andrea Pierleoni
Read More

1 November 2019

Deep bidirectional transformers for relation extraction without supervision

Yannis Papanikolaou
Ian Roberts
Andrea Pierleoni
Read More

1 March 2019

Repurposing available drugs for neurodevelopmental disorders: the fragile X experience

Michael R Tranfaglia
Clare Thibodeaux
Daniel J Mason
David Brown
Ian Roberts
Tim Guilliams
Patricia Cogram
Read More

1 November 2018

The poly-omics of ageing through individual-based metabolic modelling

Elisabeth Yaneske
Claudio Angione
Read More

1 October 2018

Drug repurposing: progress, challenges and recommendations

Sudeep Pushpakom
Francesco Iorio
Patrick A. Eyers
K. Jane Escott
Shirley Hopper
Andrew Wells
Andrew Doig
Tim Guilliams
Joanna Latimer
Christine McNamee
Alan Norris
Philippe Sanseau
David Cavalla
Munir Pirmohamed
Read More

1 October 2018

Using machine learning to predict synergistic antimalarial compound combinations with novel structures

Daniel J Mason
Richard T Eastman
Rich Lewis
Ian P Stott
Rajarshi Guha
Andreas Bender
Read More

30 August 2018

Understanding and predicting disease relationships through similarity fusion

Erin Oerton
Ian Roberts
Patrick S H Lewis
Tim Guilliams
Andreas Bender
Read More

1 June 2018

The antidepressant tianeptine reverts synaptic AMPA receptor defects caused by deficiency of CDKL5

Marco Tramarin
Laura Rusconi
Lara Pizzamiglio
Isabella Barbiero
Diana Peroni
Linda Scaramuzza
Tim Guilliams
David Cavalla
Flavia Antonucci
Charlotte Kilstrup-Nielsen
Read More

1 February 2018

Integrating splice-isoform expression into genome-scale models characterizes breast cancer metabolism

Claudio Angione
Read More

More resources

Prediction to patient: learn more about our drug discovery and development process

Listen to the podcast featuring Healx and FRAXA Research Foundation for more on the fragile X project

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Where can I learn more?

If you’re interested in learning more about the study, take a look at ClinicalTrials.gov.

Vector

We 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.

Dr Mike Tranfaglia
Chief Scientific Officer and Co-Founder, FRAXA Research Foundation