According to a story from Newswise, the early results of a recent phase 1/2 clinical trial have revealed the potential of an experimental treatment that was being tested for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare form of blood cancer. The investigational therapy is called IMGN632; the results were presented recently at the Annual 2020 Meeting of the American Society of Hematology.

About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare blood disorder that affects plasmacytoid dendritic cells. It is projected that this disease comprises less than 0.5% of all blood disease cases. Plasmacytoid dendritic cells are a rare type of immune system cell that play a role in combating viral infections. The disease presents with characteristics similar to both leukemia and lymphoma. Although risk factors remain unknown, the BPDCN appears after mutations build up in plasmacytoid dendritic cell (pDC) precursors. Symptoms of the disease include ulcers, bruise-like patches, or skin lesions, swollen lymph nodes, liver, and/or spleen, anemia, and the presence of malignant pDCs throughout the body. Treatment often includes chemotherapy and stem cell transplant, but the disease almost always relapses after this treatment. Consequently, the five year survival rate for BPDCN is low. There is an urgent need for more effective therapies for this disease. To learn more about BPDCN, click here.

About The Trial

The trial included a total of 28 adult patients. Most patients had received previous treatment and were presenting with relapse or refractory disease. Previous treatment included stem cell transplant (24 percent), chemo (52 percent), and tagraxofusp (45 percent), which is the only FDA approved therapy specifically for this cancer. This was the largest trial to date that consisted primarily of relapsed/refractory BPDCN patients. Treatment with IMGN623 produced a 29 percent overall response rate and a 31 percent overall response rate in patients that were previously treated with tagraxofusp. Five patients were able to achieve a complete response.

This trial is continuing to enroll and will also be made available to previously untreated patients as it provides a unique opportunity for those living with this cancer to try a different treatment option.

Click here to learn more about these findings.

About IMGN623

These numbers may not seem too impressive, but in a deadly disease such as BPDCN with limited treatment options, they are an encouraging sign. IMGN623 also appeared to have favorable safety characteristics compared to tagraxofusp. IMGN623 is categorized as a antibody-drug conjugate targeting CD123. The therapy earned Breakthrough Therapy designation from the US Food and Drug Administration (FDA) in October.