Ayala Pharmaceuticals reports promising data from Desmoid Tumors trial

The trial is focused on Desmoid tumors: Rare, connective tissue tumors that can have aggressive infiltrative growth and high risk of local recurrence. Image © janiecbros / Getty Images

Updated interim results from Part A the RINGSIDE pivotal Phase 2/3 study of the company’s AL102 new drug showed consistent early tumor shrinkage with measures deepening over time, plus good tolerability at all doses.

Data from the randomized global multi-center trial was presented at the recent European Society for Medical Oncology (ESMO) Congress 2022 by Prof. Robin Jones, M.D., Head of the Sarcoma Unit at The Royal Marsden, London, UK.

“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 allow 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,” said Prof. Robin Jones.

Ayala has also announced that Part B and the Open-Label Extension are being initiated with the selected dose of 1.2mg once daily.

“We are excited to share strong interim results from the AL102 RINGSIDE study at this year’s ESMO Congress which continue to demonstrate early and meaningful anti-tumor activity as monotherapy in patients with desmoid tumors,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala.

“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. We are advancing to Part B of RINGSIDE with a selected dose of 1.2mg once daily, as well as enrolling patients in the open label extension study. The results presented today give us increased confidence in the potential for AL102 to improve the lives of patients with desmoid tumors.”

Key data points as of the Cut-Off Date of July 14, 2022

Part A involved dosing patients in the AL102 monotherapy cohorts with 1.2mg (once daily), 2mg (2 days on, 5 days off), or 4mg (2 days on, 5 days off). AL102’s activity is being evaluated by change in tumor volume (central MRI readings) and response (per RECIST 1.1) determined by blinded independent central review.

Part A interim efficacy results of the 1.2mg daily dose:

- At week 16 there were 9 evaluable patients for RECIST in the selected dose of 1.2mg once daily with one PR observed, confirmed at week 28. The remaining 8 patients had stable disease, of which 7 patients had a tumor reduction.

- At week 28 there were three patients evaluable for RECIST in the selected dose of 1.2mg daily with one confirmed and one unconfirmed PR and one stable disease with all patients showing tumor reduction and deepening of tumor shrinkage since previous scan.

- At the selected dose of 1.2mg once daily, at week 16 there were 9 evaluable patients for volume change with 7 patients experiencing tumor volume reduction. At week 28 there were three evaluable patients for volume change in the selected dose of 1.2mg once daily with all three patients experiencing continuous tumor shrinkage.

In terms of safety, the results showed that AL102 was generally well tolerated at all doses and that most adverse events were grade 1 or 2 and included mainly diarrhea. There were no grade 4 or 5 events observed and low rates of grade 3 events.

Encouraging

Commenting on the results, Jeanne Whiting, Executive Director & Co-Founder of the Desmoid Tumor Research Foundation, stated: “Desmoid tumors are rare, connective tissue tumors that can have aggressive infiltrative growth and high risk of local recurrence. Patients and physicians struggle with the fact that there are no FDA-approved therapies.

“We are very encouraged by the results presented by Ayala and the opportunity that AL102 holds in improving outcomes for patients with this rare condition.”