NFXF Update on Seaside Therapeutics

Since many of you have been following developments regarding the financial challenges faced by Seaside Therapeutics, we want to update you on what we understand to be the current situation. Seaside has been an important catalyst in the effort to develop new treatments for fragile X syndrome, and the clinical trial of their STX209 medication (Arbaclofen) has involved many of our families. The unexpected cancellation of that trial has been a source of distress and confusion for many in the Fragile X community, and as the underlying financial situation plays out, we want to share what we know. The situation can be summarized in six key points.

  1. Based upon the best information we have at this time, it appears Seaside has thus far been unable to secure needed financing that would allow it to fully resume operations and reestablish their clinical trials. We are aware of numerous attempts to secure needed capital, but to date no such efforts appear to have been successful.
  2. There is one remaining clinical trial for which the results have not yet been made public. It is possible, of course, that those results could be better than the results of the previous two Seaside trials for which results were announced in May, but that is simply unknown at this time. It is also unknown whether even a positive result would be enough to attract new funding sources for the company.
  3. Seaside owns the patent on Arbaclofen. If the company does discontinue operations it is possible that it could sell the rights to Arbaclofen, and the purchasing company could continue its development. The NFXF has no information regarding what kinds of discussions have been taking place with possible purchasers. It is reasonable, given the encouraging results in some aspects of the clinical trial, to expect that such efforts are under active consideration, but we have no information with respect to their status. If and when such information becomes available and we are in a position to publicly disclose it, we will do so.
  4. We are aware that some Fragile X clinics may be affected financially by developments in this case, given their active participation in the clinical trial. Each participating clinic has its own financial arrangement with Seaside, and if the company were to cease operations the clinics would be impacted to varying degrees. Most of these clinics are also associated with the Fragile X Clinical & Research Consortium (FXCRC), which is coordinated through the NFXF. The NFXF is committed to the viability of the FXCRC clinics and will do everything possible to assure their continued success. The most we can do now is to monitor this situation closely, commit to acting in the community’s best interests, and to keep families and clinics fully informed of what we know.
  5. Based upon our ongoing communications with families, we believe that most individuals who were participating in the continuation of the STX209 trials have now completed the process of weaning off of the medication. We fully understand and sympathize that this has been a very difficult process, both for many of the individuals taking the medication and their families. The NFXF will continue to be available to provide support to those working through this process. We think it important to note that while the events of the last few months have produced great distress, this is only so because for many families, Arbaclofen was bringing about positive changes. As explained above, it is impossible to say right now whether Arbaclofen may be available again at some point in the future, but it is our strong belief that the experience with it and other compounds coming up through the drug trials pipeline demonstrates that the researchers who have brought us to this point really are onto something significant. This remains cause for optimism that effective treatments are possible and will be available in coming years.
  6. While individuals, their families and the doctors and clinics involved in the Arbaclofen saga have been negatively affected in their own ways by this experience, it is important that the entire Fragile X community not only continues to support ongoing and future trials, but redoubles our efforts to do so. We are in a vital time in the development of targeted medications for Fragile X, with researchers hoping to work with the FDA to define new measures of success for trials. New classes of compounds are being studied that target the underlying brain disorder. Many viable and committed companies are involved in these efforts. It is our intention to work as closely as possible with all involved parties to meet everyone’s objective that safe and effective compounds can and will be approved and brought to market. For these reasons, we urge all those involved at every level of this process to continue supporting and participating in this important effort. Thank you!