AbbVie and Roche says they plan to file for approval of their chronic lymphocytic leukaemia (CLL) candidate venetoclax by the end of the year after encouraging phase II data.
Venetoclax (also known as ABT-199) demonstrated a significant improvement in overall response rate (ORR) in CLL patients with a specific genetic mutation – 17p deletion – who are known to be have a poor prognosis.
The drug is an inhibitor of a protein called BCL-2 that is associated with B cell lymphomas and was awarded breakthrough status by the FDA earlier this year for this form of CLL.
The companies did not reveal details of the preliminary trial results but said they would “serve as the pivotal registration data for applications to the FDA, EMA and other health authorities.”
The ongoing phase II trial (Study M13-982) assessed the safety and efficacy of venetoclax monotherapy in 157 patients with relapsed or refractory CLL, and in addition to ORR is also measuring complete and partial responses and progression-free survival (PFS).
It is scheduled to complete at the end of 2016 with full data available in May 2017, although AbbVie and Roche hope that the serious unmet medical need in CLL will allow them to get accelerated approval.
CLL is a slow-progressing cancer of the bone marrow and blood and is the most common leukaemia diagnosed in adults in western countries, with around 14,600 cases diagnosed in the US each year.
The 17p deletion mutation is present in between 3% and 10% of all CLL patients, but is disproportionately found in relapsed and refractory cases, where the incidence is 30% to 50% and reveals just how hard this form of the disease is to treat effectively.
If approved, venetoclax will compete in the market with other new therapies for CLL, including Johnson & Johnson’s first-in-class BTK inhibitor Imbruvica (ibrutinib) and Gilead Sciences’ Zydelig (idelalisib), which is specifically indicated for use in 17p deletion-positive patients.
Both Imbruvica and Zydelig were approved for CLL last year and have been tipped to become $1bn-plus products at peak. J&J booked $270m in Imbruvica sales in the first six months of this year, while Zydelig made $56m.