The U.S. Food and Drug Administration (FDA) has granted fast track designation to BerGenBio‘s bemcentinib as a treatment for elderly people with acute myeloid leukemia (AML) whose disease has relapsed, the company announced.
The designation facilitates the development and approval of therapies that can treat serious conditions and fill an unmet medical need, either because there are no other treatments available or the new treatment offers significant benefits over approved therapies.
Treatments with fast track designation are eligible for benefits such as more frequent meetings with the FDA to discuss the development plan and clinical trial design for the therapy; accelerated approval and priority review; and “rolling review,” which means a company can submit completed sections of its application for consideration by the FDA, rather than waiting for the full application to be completed before starting the review process.
“We are thrilled that bemcentinib has been granted Fast Track Designation. Not only does this make us eligible for accelerated approval and priority review, but it serves as an important validation of bemcentinib in this significant unmet medical need we are trying to address,” Richard Godfrey, CEO of BerGenBio, said in a press release.
Current options for people with AML consist of intensive chemotherapy or autologous stem cell transplant. But some patients, including the elderly, often cannot tolerate the side effects of these treatments and have no other approved therapies for their condition.
Bemcentinib (formerly known as BGB324) inhibits the action of a protein called AXL kinase. When expressed by cancer cells, AXL reduces the immune response against the tumor, which has been associated with treatment failure, more aggressive cancer, and increased metastasis.
That is why AXL inhibitors such as bemcentinib are potential cancer treatments that can act either alone or in combination with other therapies.
In a recent Phase 2 clinical trial (NCT02488408), researchers evaluated bemcentinib, in combination with low-intensity chemotherapy, in adults with AML unsuitable for treatment with intensive chemotherapy, either due to advanced age or to co-existing diseases.
Results presented at the European Hematology Association 2019 Annual Congress in June showed that six of 14 evaluable patients (43%) responded to treatment with bemcentinib plus low-dose cytarabine. Of them, four (29%) had complete responses, or the elimination of all tumor signs. This is significantly better than responses seen with low-dose cytarabine in similar patient populations.
Researchers also reported that five of the six responses occurred in patients older than 75, and that two additional patients had stable disease for more than three months.
Another group of patients received bemcentinib in combination with decitabine. Results presented at the American Society of Clinical Oncology (ASCO) 2019 Annual Meeting showed that of the 12 patients evaluated for efficacy, three (25%) responded to treatment, including one with a complete response. Also, five patients had stable disease for more than three months.
The combination was well-tolerated, with the most common adverse side effects being anemia, low levels of neutrophils (an immune cell type) and diarrhea.
BerGenBio is currently overseeing another Phase 2 clinical trial (NCT03824080), which is still recruiting participants in Europe, to test the safety and effectiveness of bemcentinib as a second-line treatment in adults with AML or myelodysplastic syndrome, another type of blood cancer.
Other ongoing Phase 2 trials are investigating bemcentinib as a treatment for different solid tumors and blood cancers, either alone or in combination with immunotherapies, targeted therapies, or chemotherapies.