We’re always encouraged to see new research efforts focused on improving the lives of individuals and families living with Fragile X syndrome (FXS). One company taking an exciting new approach is CONNECTA Therapeutics, a biotech company developing treatments that aim to help the brain strengthen its own connections.
A New Way to Support the Fragile X Brain
CONNECTA’s lead medicine, called CTH120, takes a fresh approach to treating FXS. It works by supporting a natural brain pathway called the BDNF pathway, which helps brain cells grow, communicate, and form new connections. In studies using FXS mouse models, CTH120 helped improve how brain cells connect to each other and led to better thinking and behavior — two areas that can often present challenges for people living with FXS.
Early Signs of Success
CONNECTA recently finished a phase 1 clinical trial in 76 healthy volunteers. The results were very positive: the medicine was safe and well-tolerated, and it even showed early signs of reducing anxiety, which is common in many individuals living with FXS.
Why This Matters
For families affected by FXS, this research represents a new opportunity, and, perhaps, a new kind of hope. Most current treatments focus on easing symptoms, but CTH120 aims to modulate neuroplasticity, the ability of the brain to adapt its activity in response to internal or external stimuli by reorganizing its structure, functions, and connections.
What’s Next for CONNECTA and FXS
The next big step will be a Phase 2a clinical trial, set to begin in early 2026 in Europe. This study will test how well CTH120 works for adults living with FXS and continue to confirm its safety. Through the FRAXCURE project, co-funded by the European Union, CONNECTA is also preparing to expand its work to include children and teens in future studies. The company eventually plans to run clinical trials in Europe, the U.S., and Japan.
Looking Ahead
While it’s still early, CONNECTA’s work is an exciting step forward for Fragile X research. By focusing on the brain’s natural ability to rebuild and reconnect, CONNECTA is opening a new path that could one day help individuals with Fragile X.
We’ll continue to follow CONNECTA’s progress and share updates as their research moves forward. Every step like this brings us closer to more options — and more hope — for our community.
