Glimpse of optimism in desmoid tumor
Sep 12, 2022 | DelveInsight
Desmoid tumors (DT) are rare benign monoclonal fibroblastic tumors that constitute less than 5 % of all soft tissue tumors. Due to the high probability of postoperative recurrence, the current scenario illustrates that surgery is not regarded as a feasible treatment option. The most advisable treatment modality for the desmoid tumor is primary surgery with negative surgical margins, currently, there are no FDA-approved systemic therapies available for the treatment of untreatable, recurrent, or developing desmoid tumors. As part of the evolution of therapy advancement, apart from the primary surgical intervention approach less invasive techniques such as image-guided ablation consisting of cryoablation and high-intensity focused ultrasound has been considered less morbid procedure.
To fill the treatment gaps in desmoids tumors, addressed above, two of the emerging promising players in the market such as Ayala pharmaceuticals (AL102) and SpringWorks Therapeutics (nirogacestat) presented the updated data readouts for their emerging assets for the treatment of DT at the conference.
Ayala pharmaceutical’s AL102, demonstrated excellent therapeutic response by gamma secretase inhibition. Ayala pharmaceuticals presented interim results of pivotal Phase II/III, clinical study (RINGSIDE), evaluating efficacy and safety of AL102 vs. placebo in patients with progressing desmoid tumors, at ESMO 2022:
- Treatment with the AL102 at varying doses, 1.2mg (once daily), 2mg (2 days on, 5 days off), or 4mg (2 days on, 5 days off), resulted in tumor shrinkage in 13 of the patients, who were evaluable at 16 weeks. Moreover, 1 patient experienced an unconfirmed partial response at week 16
- AL102 was well tolerated, and no dose-limiting toxicities were observed, at all of the examined dose levels
Expert Opinion: “The results presented at ESMO from the RINGSIDE study are very encouraging. AL102 demonstrated an early and meaningful effect on tumors within a 16-week period and was well tolerated, which could be allowed for long term treatment of patients. AL102 has the potential to significantly improve the lives of patients suffering from desmoid tumors who currently have no approved therapy”.