The Desmoid Tumor Research Foundation

*Big News*: Exciting Results Announced For Two Promising Desmoid Tumor Clinical Trials At ESMO Conference In France

AL102 RINGSIDE

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*Update: Watch the 2022 DTRF Annual Patient Meeting presentations on the Nirogacestat results and the AL102 results. For all Patient Meeting recordings, visit Patient Meeting.*

BIG NEWS this Desmoid Tumor Awareness Month!

Presentations on the latest data from two promising clinical trials – both studying gamma secretase inhibitor drugs in desmoid tumors – were given this past weekend at the 2022 European Society for Medical Oncology (ESMO) conference in Paris, France.

PLEASE NOTE: Presentations tailored for the patient community on these two trials and their data will be given at the Annual Patient Meeting Webinar #2 (of 3) on Sept. 24th during the DTRF Together We Will Virtual Weekend. Register at no cost at DTRF.org/weekend, and bring your questions!

Both of these presentations have left us encouraged and hopeful. The interest in desmoid tumor research is higher than ever before and we are making significant progress in finding more effective and more manageable therapies. If you have fundraised for or donated to the DTRF, you have helped make this progress possible by supporting our patient outreach, advocacy, research, awareness, and scientific collaboration efforts. Give today.

Here are the details from the ESMO presentations:

  • NIROGACESTAT – Phase 3 DeFi Clinical Trial (SPRINGWORKS THERAPEUTICS)

On Saturday, Principal Investigator Bernd Kasper, MD, (University of Heidelberg, Maneheim) presented the latest data from the SpringWorks Therapeutics Phase 3 DeFi clinical trial for nirogacestat (a gamma secretase inhibitor) in desmoid tumor patients during the Presidential Symposium session of ESMO.

“Results showed a statistically significant improvement in progression-free survival in patients randomised to nirogacestat compared to the placebo group, with a 71% lower risk of disease progression on average. The response rate was also much higher – 41% in the nirogacestat arm and only 8% in the placebo arm; nearly one in ten patients (7%) showed a complete response with the agent.” 

– Bernd Kasper, MD, PhD, University of Heidelberg, Principal Investigator of the DeFi trial

For more information, see the SpringWorks’ press release

…and the ESMO article.

  • AL102 – RINGSIDE Phase 2/3 Clinical Trial (AYALA PHARMACEUTICALS)

On Monday, Prof. Robin Jones, MD, (The Royal Marsden) presented the latest data from the Ayala Pharmaceuticals RINGSIDE Phase 2/3 clinical trial for AL102 (a gamma secretase inhibitor) in desmoid tumor patients.

“Efficacy was demonstrated across all cohorts of Part A of the study, with early responses that deepened over time. The results also showed that AL102 was well-tolerated across all doses.” 

– Roni Mamluk, PhD, CEO of Ayala


For more information, read Ayala’s press release