Officials with the FDA have granted fast track designation to BerGenBio’s bemcentinib for the treatment of elderly patients with acute myeloid leukemia (AML) whose disease has relapsed, according to a press release.

Bemcentinib, formerly known as BGB324, is a potentially first-in-class selective AXL inhibitor, according to BerGenBio. The drug works by targeting and binding the intracellular catalytic kinase domain of AXL receptor tyrosine kinase and inhibiting its activity.

AML is the most common type of aggressive leukemia in adults and has the lowest survival rate for all leukemias. Oftentimes, patients older than 60 years of age are unable to tolerate intensive induction chemotherapy. There are currently no treatments specifically approved for all relapsed patients with AML.

“We are thrilled that bemcentinib has been granted fast track designation,” Richard Godfrey, chief executive officer of BerGenBio, said in a statement. “Not only does this make us eligible for accelerated approval and priority review, but it serves as an important validation in this significant unmet medical need we are trying to address. Currently, bemcentinib is in expanded phase 2 trials in the US and Europe for the treatment of AML and the company has recently announced positive interim top line data.”

At the 2019 annual congress of the European Hematology Association in June, investigators presented data from a phase 1b trial and preliminary results from a phase 2 trial of bemcentinib plus cytarabine or decitabine in patients with AML who are unfit for intensive chemotherapy.

The study evaluated the efficacy and safety of bemcentinib and also looked at an experimental arm with bemcentinib as a single agent. Sixteen patients with AML were enrolled in the combination portion of the phase 2 study and received bemcentinib 200 mg daily plus low-dose cytarabine in 21-day cycles. Among the 14 patients evaluable for efficacy, 6 patients achieved responses, 4 achieved complete remission/complete remission with incomplete hematologic recovery (CR/CRi), and 2 patients achieved partial remission. Five of the 6 responses occurred among elderly patients with AML.

Additionally, the study also showed that 2 patients achieved durable stable disease for more than 3 months. The relapse-free survival rate for patients with CR/CRi is 7.9 months (range: 0.7 to 9.6 months) and continues to mature.

Overall, the treatment was considered tolerable, with the most common adverse events being anemia, neutropenia, and diarrhea.

Bemcentinib is currently being evaluated in ongoing clinical trials for multiple solid and hematological tumors, in combination with current and emerging therapies, and as a single agent.

References

BerGenBio Receives FDA Approval of Fast Track Designation for Bemcentinib [news release]. BerGenBio’s website. https://www.bergenbio.com/bergenbio-receives-fda-approval-of-fast-track-designation-for-bemcentinib/. Accessed October 23, 2019.

BerGenBio Presents Preliminary Phase 2 Clinical Data at EHA 24: Bemcentinib in Combination with Low Dose Chemotherapy Yields Durable Responses in AML Patients Unfit for Intensive Chemotherapy [news release]. BerGenBio’s website. https://www.bergenbio.com/bergenbio-to-present-preliminary-phase-ii-clinical-data-showing-bemcentinib-in-combination-with-low-dose-chemotherapy-yields-durable-responses-in-aml-patients-unfit-for-intensive-chemotherapy-at-the-2/. Accessed October 23, 2019.