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CONNECTA Therapeutics to Advance CTH120 into Phase II Pediatric Development for Fragile X Syndrome

CONNECTA Therapeutics is making strides in pediatric medical advancements with its innovative treatment program for Fragile X Syndrome (FXS). The clinical-stage biotech company has recently announced the impending Phase II development of its investigational drug, CTH120, aimed specifically at addressing the needs of young patients affected by this rare neurodevelopmental disorder. Supported by a substantial grant of €2.5 million from the European Innovation Council (EIC) Accelerator program, this initiative seeks to set new standards in the treatment of FXS, which currently lacks approved disease-modifying therapies.

Understanding Fragile X Syndrome

FXS is recognized as one of the most common genetic causes of intellectual disabilities, presenting significant challenges for those affected. Approximately 1 in 7,000 males and 1 in 11,000 females are diagnosed with this disorder, leading to cognitive delays and behavioral issues that begin in early childhood. Common symptoms include anxiety, social withdrawal, and aggression, considerably impacting the quality of life not only for patients but also for their families. Studies indicate that nearly 80% of families dealing with FXS experiences financial strain due to the costs associated with care and therapy, highlighting the urgent need for effective treatment options.

CTH120: A Promising Treatment

CTH120 is a pioneering neuroplasticity modulator that has demonstrated strong safety and tolerability in initial Phase I trials. This small-molecule compound operates by targeting the TrkB receptor, facilitating improvements in brain development and function by restoring disrupted neural connections. Unlike existing therapies that only address symptoms, the goal of CTH120 is to modify the disease process itself, providing a transformative approach to treatment.

The EIC Accelerator grant, awarded amidst stiff competition— where only 71 companies were chosen out of more than 1,200 applicants—will enable CONNECTA to finalize essential regulatory steps. These include developing an appropriate formulation for younger patients and submitting the Pediatric Investigation Plan necessary for future Phase II trials. Additionally, CONNECTA is poised to initiate a Phase IIb trial for adult patients to broaden the clinical evidence surrounding CTH120’s effectiveness.

Jordi Fàbrega, Co-Founder and CEO of CONNECTA Therapeutics, emphasized the significance of this funding, stating, "The EIC Accelerator grant is a game-changer - accelerating our mission to deliver hope and lasting impact to families". He underscored the critical nature of the treatment given the absence of approved therapies for FXS, describing CTH120 as a means of redefining care in a field that has yet to see meaningful progress.

Building on Success

The promising data from CTH120's trials build upon CONNECTA’s broader vision to utilize its neuroplasticity modulation platform to develop solutions for a variety of CNS disorders. The company is eyeing treatments for conditions including autism spectrum disorder (ASD), Rett syndrome, and attention deficit hyperactivity disorder (ADHD). CTH120 serves as a proof of concept for extending this innovative treatment modality beyond FXS, indicating the potential for further advancements in the understanding and management of several neurodevelopmental disorders.

CONNECTA is dedicated to reshaping the future of neurological treatments—an aim fueled by both its scientific innovations and the newfound support from entities like the European Union. As the world watches this groundbreaking initiative unfold, many in the FXS community remain hopeful that new therapies will emerge to enhance the lives of those affected by this challenging disorder, thereby fostering a brighter future for children and families dealing with Fragile X Syndrome.

As CONNECTA accelerates its development plans for CTH120, the healthcare community remains alert to the potential of this innovative treatment in making strides against FXS, a disorder that has remained largely unaddressed for far too long. From fostering early diagnosis to ensuring quality treatment availability, initiatives like these represent the starting point for tangible change in neurology.

Conclusion

The strategic advancement of CTH120 is not just a step for CONNECTA but a profound leap for pediatric care in fragile neurodevelopmental disorders. Through commitment to innovation and an understanding of the scientific landscape, CONNECTA Therapeutics paves the way for a future where treatment options for FXS are not only available but also effective in restoring hope and enhancing quality of life for patients and their families.