Healx has just announced that they’ve been given Investigational New Drug FDA approval for HLX-0201 for Fragile X syndrome. This means they are able to move forward with their Phase 2a investigation of the compound. The hope is that they will uncover a combination therapy that is effective in treating this rare disease.
Healx is an AI-powered company which has uncovered many potential drug candidates for Fragile x syndrome. The purpose of the Phase 2a trial is to understand which combination of these produces the strongest effect. Recruitment will begin for this trial in the next few months.
Considering that there are unfortunately no approved and effective treatments for Fragile X, let alone a cure, this trial is incredibly meaningful for patients. Healx is striving to bring at minimum one effective combination treatment to this patient population.
HLX-0201 was first given Orphan Drug Designation in 2020 from the European Union. It then received this designation in the United States in 2021.
This compound was first approved as an anti-inflammatory and nonsteroidal drug.
It was first identified by Healx as a possible treatment for Fragile X with the company’s omic-based drug matching technology. This technology looks at the gene expression of a certain disease and compares it to the gene expression of different treatment options. This way, common pathways can be found.
Preclinical studies of this compound have demonstrated that it is effective at a much lower dose than it would be as a monotherapy. These studies also found that the compound was able to modify many of the behaviors that are very similar to those patients with Fragile X experience. The therapeutic effect acted in a newly discovered secondary form of action.
This isn’t the only compound Healx has uncovered. Now, they’re investigating which of these compounds may be combined to have synergistic mechanisms of action.
A combination therapy could very well be the best option for Fragile X because the symptoms are so diverse. If different treatments can target different pathways and ease different symptoms, patients may have the best outcomes.
This company is very excited about the potential of combination therapies to treat not simply Fragile X, but many rare diseases. They hope to expand their clinical programs. The goal is to have 50 by the year 2025.
To do so, they plan to collaborate with patient advocacy groups, academic centers, industry professionals, and more. To learn about partnering opportunities, visit this website. For instance, through their work with Fragile X, they have maintained close communication with the FRAXA Research Foundation.
You can read more about this upcoming Phase 2a trial here.