Ayala Partnering with Novartis on Investigational AL102 for Treatment of Multiple Myeloma

In a new agreement, Ayala Pharmaceuticals will work with Novartis on the development and commercialization of AL102, an investigational treatment for multiple myeloma.

The option-to-license deal calls for Ayala to receive a $10 million equity investment from Novartis. Ayala, a clinical company that develops treatments for genetically identified cancers, also stands to receive development, clinical, regulatory, and commercial milestones, plus tiered sales royalties.

In return, Ayala will supply agents necessary for studies to investigate AL102 combined with B-cell maturation antigen (BCMA) therapies in multiple myeloma. Novartis will pay for study development costs. For all other indications, Ayala will retain global license rights.

“This collaboration is important to Ayala, as it immediately strengthens our balance sheet, further validates our technology, and accelerates the clinical development of AL102 as a combination therapy in hematologic cancers,” Roni Mamluk, Ayala’s CEO, said in a press release.

Ayala is evaluating AL102, an inhibitor of the Notch pathway, in blood cancers. The pathway regulates cell-fate determination during development, and maintains adult tissue balance. Cumulative evidence indicates that Notch is overactive in multiple myeloma and participates in its onset and progression.

BCMA is produced in most myeloma patients and is actively shed from multiple myeloma cells by gamma secretase — which the oral small-cell molecule AL102 inhibits. Thus, researchers believe that AL102 could be used to raise its levels in multiple myeloma, increasing the efficacy of BCMA-targeting agents.

In a Phase 1 trial (NCT01986218) conducted by Bristol-Myers Squibb — which first developed AL102 — researchers already tested the treatment in solid tumor patients. AL102 was safe and well-tolerated, and stabilized disease for more than six months in 14% of patients.

Multiple myeloma is a cancer that forms in a type of white blood cell called a plasma cell. These cells help fight infections by making antibodies that recognize and attack germs. Multiple myeloma causes cancer cells to accumulate in the bone marrow, crowding out healthy blood cells. Rather than produce beneficial antibodies, the cancer cells produce abnormal proteins that can cause complications.

According to the American Cancer Society, the lifetime risk for U.S. residents of developing multiple myeloma is 1 in 132. Roughly 30,770 new cases were expected to have been diagnosed in the U.S. last year.