Novel AML Drug Receives FDA Fast Track Designation

The Food and Drug Administration is evaluating the novel drug DSP-5336 for acute myeloid leukemia treatment.

The novel drug DSP-5336 has received a fast track designation from the Food and Drug Administration (FDA) to treat patients with relapsed or refractory acute myeloid leukemia (AML) who have a KMT2A gene rearrangement.

DSP-5336 is a novel type of menin inhibitor, which may help prevent the growth of cancer, according to a news release from Sumitomo Pharma, the manufacturing company of DSP-5336.

A fast track designation, according to the FDA, is when a drug’s review will be expedited because it has potential to fulfill an unmet need.

The fast track designation was granted to DSP-5336 after updated data from an ongoing phase 1/2 trial that is evaluating dose escalation (finding the right dosage of the drug), the news release noted. The phase 2 portion of the trial will evaluate the safety of the drug, according to the listing on clinicaltrials.gov.

In phase 1 of the trial, patients are placed into two groups to either receive DSP-5336 with antifungal medications or DSP-5336 without antifungal medications. Patients in phase 2 of the trial are placed into groups depending on KMT2A rearrangements (group A) or nucleophosmin 1 (NPM1) mutations (group B), the listing explained.

Of note, an objective response (percentage of patients whose tumors partially or completely responded to treatment) was observed in 57% of patients (12 of 21) in the trial. Patients either had NPM1 mutations or KMT2A rearrangements. It was reported that 24% of patients (5 of 21) achieved complete remission (their cancer disappeared) or complete remission with partial hematologic (blood) recovery.

"For patients and families facing a diagnosis of relapsed or refractory acute myeloid leukemia, significant unmet medical needs remain — and we share in their urgency to identify and advance new treatment pathways," Tsutomu Nakagawa, president and chief executive officer of Sumitomo Pharma, said in the release. "We are encouraged by FDA's decision and look forward to working closely with the agency as we continue our clinical development of DSP-5336."

Regarding safety, DSP-5336 is considered well-tolerated and has not shown dose-limiting toxicity (serious side effects from the drug that prevent an increase in the dose), the news release stated.

READ MORE: Standard Treatment With Adjustments May Extend Survival in Older Patients With AML

"Management of AML continues to be challenging with limited options for which there are currently no approved targeted therapies to treat AML with KMT2A rearrangements or NPM1 mutations, leaving a serious unmet medical need," said Dr. Jatin Shah, chief medical officer — Oncology at Sumitomo Pharma, in the release. "DSP-5336 has shown promising clinical activity, and menin inhibitors have tremendous potential to impact the outcomes of these types of acute leukemia. We are excited by these early results and FDA fast track designation and look forward to working closely with the agency and our collaborators to rapidly advance this program with the goal of providing a well-tolerated and effective targeted treatment option for patients with relapsed or refractory [AML]."

AML is a cancer that starts in the bone marrow but also rapidly moves to the blood, as defined by the American Cancer Society. AML is reported to account for approximately one out of three leukemias in adults but accounts for approximately 1% of cancers in general.

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