A note to our families
This morning, Fulcrum released top-line results of its phase 3 trial for Losmapimod. The results showed no difference between placebo and active ingredient groups. Therefore, Fulcrum is suspending future Losmapimod development.
This news is disheartening and as a community, we will need time to process this development. But we will also look to the hope and promise of treatments on the horizon. Avidity Biosciences, Dyne Therapeutics, Arrowhead Pharmaceuticals, Roche, miRecule, and other companies are actively pursuing FSHD therapies.
On a personal note, allow me to express my appreciation to the hundreds who took part in the Fulcrum trials. You are pioneers who have marked the trail others will follow and future trials will benefit from your participation. I also want to thank Fulcrum. I am grateful for their years of commitment to improving the lives of patients with FSHD.
To our families living with FSHD, you are the most courageous and resilient people I know. While this news will cause us to pause, it will not stop us from pressing forward toward our collective goals: treatments and a cure for FSHD.
-Mark Stone, CEO, FSHD Society
I am beyond crushed. Not for me as there is nothing but a miracle for me and my siblings now. Its but my nephews now 30+ that would need intervention sooner than later. When do we switch gears? When do we start thinking outside the box a little more? Has transplantation of healthy muscle tissue ever been considered? Where could that lead us? Am I so far off target it’s not even technically feasible. Remembering a story about a little girl who had her face se disfigured after a dog attack. European country, I believe.
If it doesn’t work, it doesn’t work. And we can accept that. What has been totally unacceptable is the amount of time it took to find out. This should have been a 2 or 3 year process tops not 10 years like it has taken.
Is this successful?? Please reply, we are going to get treatment or not?? Losmopimod is successful??
This was the only drug from FSHD clinical trials that reached Phase 3 and just like that this happened. This is not just heartbreaking but very frustrating as we were hoping for launch of treatment with less than 3 years, now the other remaining drugs in development perhaps we are looking for at least 3-7 years or even a decade.
I hope someone from the Fshd Society can communicate with the higher-ups at fulcrum. We have gotten absolutely no direct communication from the company as study participants. There was no effort to reach out to participants to tell us about any next steps, no hotline to ask questions about what to do with our medication, next appointments. It’s so distressing, disrespectful and unethical. I have no idea what to do to try and rectify the situation. Perhaps somebody at the society can speak with the CEO or patient representatives there. They do not provide emails to reach out directly. I also hope that there can be discussions with other drug companies so that something like this doesn’t happen again. Those of us who have participated in the study as subjects have given so much time and effort, traveled all over, sat through hours of testing and MRIs. The lack of care for the patient, the people for the drugs are meant to help, and without whom, there would be no research studies is unbelievable, disrespectful and unethical.