McQuade Center, Sentinel Oncology Working on New Therapy
The McQuade Center for Strategic Research and Development (MSRD) will collaborate with Sentinel Oncology for the clinical development of a potential breakthrough treatment for fragile X syndrome (FXS).
Under agreement terms, the MSRD, which funds innovative early stage research and development programs, will work with the United Kingdom-based treatment discovery company Sentinel to advance investigational compound SOL784 to Phases 1a and 1b clinical trials. Those studies may be followed by an expanded collaboration, according to a description of the agreement.
SOL784 is aimed at the underlying cause of fragile X — mutations in the FMR1 gene, which is on the X chromosome. Because there currently is no cure for the genetic condition, treatments generally seek to ease symptoms, especially those associated with behavior and cognitive challenges, and to improve patients’ quality of life.
The goal of clinical trials is to determine whether, in this case, a pharmaceutical therapy works and is safe. Both people with the disease and healthy volunteers, known as controls, may be enrolled in clinical studies, which are regulated by law and require government approval before they can commence.
New medications typically undergo three trial “phases.” In the first phase, which is pertinent here, a relatively small number of participants would test the first-in-class therapeutic candidate for safety, tolerability, and other properties.
“Although there are treatment options for the symptoms of fragile X syndrome available today, none have been approved by regulatory to address the underlying cause of this rare disorder,” Robert McQuade, PhD, said in a press release. McQuade is president of the McQuade Center, and chief strategic officer for Otsuka Pharmaceutical Development & Commercialization.
“Our two organizations share like-minded missions of delivering treatments to patients with unmet medical needs. We are hopeful that our collaboration can lead to the development of a potential therapeutic for people living with this condition.”
The McQuade Center is a member of the global Otsuka family of pharmaceutical companies. Sentinel Oncology seeks to develop novel therapies for disorders with unmet needs.
“We are delighted to be collaborating with the MSRD team to advance SOL784 into clinical development,” said Robert G. Boyle, founder and CEO of Sentinel Oncology. “SOL784 targets the underlying disease biology of FXS and we are excited for the prospects of a drug which has the potential to provide real benefits to patients for whom there is an unmet medical need.”
Between 38,000 and 87,000 individuals in the U.S. are thought to be living with fragile X.