Aliqopa Granted FDA’s Breakthrough Therapy Status for Previously Treated Marginal Zone Lymphoma

Aliqopa (copanlisib) has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed marginal zone lymphoma (MZL) who have received at least two prior therapies.

The designation is intended to accelerate the development and review of a potential therapy for a serious or life-threatening disease, with clinical evidence of substantial improvement over existing options. The program’s features include more intensive FDA guidance throughout development — such as ensuring efficient design of clinical trials — and eligibility for priority review.

MZL accounts for about 10% of all non-Hodgkin’s lymphomas in the U.S. Patients are often treated with either chemotherapy, immunotherapy, or a combination of both, but after an initial response, a fraction of the patients relapse.

Aliqopa, being developed by Bayer, is an inhibitor of PI3K delta, a family of proteins important in the proliferation and survival of B-cells that can promote the development of B-cell cancers, such as MZL. The therapy is given into the blood.

The FDA’s decision was based on interim data from the CHRONOS-1 Phase 2 clinical trial (NCT01660451), where the treatment was deemed safe, with an overall response rate of 70% in MZL patients. In 13% of MZL patients, the cancer had been totally eliminated.

CHRONOS-1 included 142 patients with relapsed or refractory indolent non-Hodgkin lymphoma, including 23 patients with MZL, all of whom had received at least two prior therapies.

Its main goal was to determine the proportion of patients achieving a reduction in tumor burden after Aliqopa treatment. Duration of response, time to disease progression or death, and overall survival were assessed as secondary measures.

Data from this trial also led to the approval of Aliqopa for follicular lymphoma patients who relapsed after at least two prior therapies, after 61 of the 104 included patients (59%) responded to the treatment, including 21 (20%) with a total tumor eradication.

“The clinical evidence suggests that copanlisib may address an unmet medical need by providing physicians and MZL patients with a therapy in a relapsed setting,” Scott Z. Fields, MD, senior vice president and head of oncology development at Bayer’s Pharmaceutical Division, said in a press release. “We will continue working closely with the FDA in order to bring copanlisib to these underserved patients as soon as possible.”

Bayer is now conducting two additional Phase 3 studies — CHRONOS-3 (NCT02367040) and CHRONOS-4 (NCT02626455) — that will examine Aliqopa in combination with other cancer therapies for indolent non-Hodgkin’s lymphomas.

Both trials are recruiting participants who received at least one prior line of treatment. For more information, click on the trials’ registry numbers.