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Upcoming Clinical Trials

Phase III Trial for Nirogacestat

A new company named SpringWorks Therapeutics has been formed as a spinoff of Pfizer to focus on investigational therapies that hold 
significant promise for underserved populations. 

SpringWorks will now take the gamma-secretase inhibitor that showed promising results in treating desmoid tumors in Phase I and Phase II trials, into a Phase III trial. Formerly known as PF-03084014, the drug is now named Nirogacestat (pronounced “nye-roe-gay-se-stat”). 

We have been working for several years to bring this drug to patients. We will continue working collaboratively with SpringWorks Therapeutics and the investigator community as they design Phase III trials that serve both adult and pediatric desmoid patients.

No details have been announced about the trials or their timing. Rest assured that we will keep you informed as soon as any information is released in the upcoming months.

Please see the press release that provides further details about this exciting development.

 

Phase I Trial for Tegavivint 

At our 11th DTRF Annual Patient Meeting in Philadelphia, we were pleased to have Beta Cat Pharmaceuticals’ Jon Northrup, Chief Executive Officer, and Casey Cunningham, Chief Medical Officer, present an outline of the planned clinical trial of Tegavivint, a new drug that directly interferes with beta-catenin stabilization. Targeting beta-catenin in this way may hold promise for desmoid sufferers, as the science that has evolved shows that desmoid tumors are due to mutations in the beta-catenin gene, CTNNB1, which cause improper degradation of beta-catenin. Tegavivint has shown promise in preclinical studies of desmoid tumors and this trial will be the drug’s first trial in patients.

Beta Cat anticipates clinical sites at the Harvard Hospital system, Memorial Sloan Kettering in NYC, Northwell Hospital on Long Island, Princess Margaret in Toronto, Canada, University of Washington in Seattle, and MD Anderson in Houston.

We will keep you posted as more details are finalized for this trial. Until then, we recommend watching the video of Dr. Sunil Sharma (University of Utah’s Huntsman Cancer Institute) discussing the drug at last year’s Patient Meeting and perusing the slides from Beta Cat Pharmaceuticals.

 
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