Trial to Assess Safety and Efficacy of Novel Drug for Extremely Rare Cancer Begins Enrolling Patients

The first five patients have been enrolled in a phase 1 trial to assess the safety and efficacy of AL101, an experimental gamma-secretase inhibitor, for the treatment of an extremely rare cancer, according to the agent’s manufacturer, Ayala Pharmaceuticals.

The disease, known as adenoid cystic carcinoma, is often found in a person’s head and neck area — such as the mouth, throat and salivary glands — as well as other areas of the body including the tear and sweat glands.

Fewer than 1,500 people in the United States are diagnosed with this cancer each year, according to the American Society of Clinical Oncology (ASCO). Although five-year survival rates (defined as the percentage of individuals alive five years after diagnosis or start of treatment) are close to 90%, the long-term survival outcomes continue to decrease. According to information released from ASCO, the 15-year survival rate for this patient population is approximately 40%.

For this particular trial (NCT04973683), Ayala Pharmaceuticals is collaborating with The University of Texas MD Anderson Cancer Center in Houston and the Adenoid Cystic Carcinoma Research Foundation. Participants will have to undergo a biopsy to confirm a diagnosis of adenoid cystic carcinoma with Notch activation (which is the presence of a receptor associated with the development of malignancies).

Once the presence of adenoid cystic carcinoma is confirmed, up to 12 patients be delivered study drug intravenously for 60 minutes once a week for a duration of six to eight weeks. Surgery will then be performed within one to three days after the last infusion of the study drug.

The main objective of this phase 1b trial is to assess the safety and overall feasibility of administering the study drug prior to surgical removal of the tumor/tumors.

Other goals include measuring the objective response rate (which is the percentage of patients who achieve a complete or partial response to treatment) at six to eight weeks after the start of treatment.

Of note, a patient may continue to receive study drug following the completion of their surgery, however that decision is up to the discretion of the investigators.

The plan is to then follow-up with patients within six weeks of the surgery or 30 days after last dose of study drug. After those follow-ups, patients will return for a follow-up every six months. The estimated date of study completion is April 2023.

“(Adenoid cystic carcinoma) can be treated with surgery and radiation following initial diagnosis, resulting in relatively high five-year survival rates,” Jeffrey Kaufman, executive director of the Adenoid Cystic Carcinoma Foundation, said in a news release announcing the enrollment of the first five patients. “However, late disease recurrence is common and can occur many years after initial treatment. Unfortunately, there are no approved therapies for patients with recurrent/metastatic disease, and the 10- to 20-year survival rates for (adenoid cystic carcinoma) remain very low. We are very encouraged by the excellent work being conducted by Ayala and MD Anderson Cancer Center and the promise that AL101 holds in improving outcomes for patients. We are pleased to support this important study.”

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