Multiple Myeloma Therapy Gets Orphan Drug Designation

Last night, SELLAS Life Sciences Group, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.

GPS is licensed from Memorial Sloan Kettering Cancer Center and targets the Wilms Tumor 1 (WT1) protein, which is present in an array of tumor types.

The news comes less than a month after Phase 2 clinical and immunological data was presented at the 44th Annual European Society for Blood and Marrow Transplantation (EBMT) Meeting, revealing that novel WT1 gene-targeting direct immunizer is safe and effective in high-risk multiple myeloma.

“We are delighted to receive this orphan drug designation as it underscores the great need for innovative, effective treatments for this rare cancer, and recognizes the potential benefits that GPS may provide for patients with MM,” said Angelos Stergiou, MD, ScD h.c., President & Chief Executive Officer of SELLAS in a press release.

In the Phase 2, GPS was evaluated as consolidation therapy to potentially stimulate a highly-specific immune response against WT1 to stop or slow progression of myeloma.

The results demonstrated that median progression-free survival (PFS) in the high-risk disease setting was 23.6 months, versus historically inferior outcomes of patients on an immunomodulatory drug (IMID) or proteasome inhibitor post-autologous stem cell transplant (ASCT) maintenance.

“Receiving orphan drug designation for the treatment of MM is a significant regulatory milestone in the development of GPS,” continued Stergiou. “We have reported median progression-free survival (PFS) of 23.6 months in the high-risk MM disease setting, compared to historically inferior outcomes in such a patient cohort of around 12 months, and GPS stimulated time-dependent and robust CD4+ T cell or CD8+ T cell immune responses as well as multifunctional cross-epitope T cell reactivity.”

Because the WT1 antigen is overexpressed in several malignancies and it is not found in most normal tissues, GPS is believed to have the potential to be a broad immunotherapy, effective across several different cancer types and patient populations.

GPS has also previously received orphan drug designation for the treatment of acute myeloid leukemia (AML) and malignant plural mesothelioma (MPM). SELLAS has Phase 3 clinical trials planned for GPS in both AML and MPM and is developing GPS as a potential treatment for a broad range of other cancer indications.

For more news from the rare disease community, subscribe to Rare Disease Report’s e-newsletter.