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news.NOXXON Pharma has received orphan drug designation from the US Food and Drug Administration (FDA) for one of its Spiegelmer therapeutics, olaptesed pegol (NOX-A12), indicated to treat glioblastoma in conjunction with radiotherapy.
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Olaptesed pegol is a PEGylated mirror-image (L-) oligonucleotide, which binds and neutralizes the chemokine CXCL12/SDF-1, preventing interaction with its receptors CXCR4 & CXCR7.
The company develops a new class of proprietary therapeutics, Spiegelmers, which are chemically synthesized L-stereoisomer oligonucleotide aptamers, a non-immunogenic alternative to antibodies.
Standard treatments for glioblastoma include chemotherapy, radiation and surgery, but treatment is very difficult due to resistance of tumor cells, an almost inevitable relapse following initial treatment and the risk of damaging the brain with conventional therapies.
According to the company, the CXCL12/CXCR4/CXCR7 pathway directly affects tumor progression by controlling cancer cell survival, proliferation and migration, and also has indirect effects through angiogenesis and recruitment of immune cells.
At present, olaptesed pegol is being evaluated in Phase IIa studies in two hematological cancers: multiple myeloma (MM) and chronic lymphocytic leukemia (CLL).
The FDA’s orphan drug status will support the company’s further development of olaptesed pegol for the treatment of glioblastoma.
NOXXON Pharma chief medical officer Dr Matthias Baumann said the FDA orphan drug designation for olaptesed pegol in the US is a significant regulatory milestone for the company.
"In conjunction with interim results of our ongoing Phase IIa studies in multiple myeloma and chronic lymphocytic leukemia, the preclinical glioblastoma findings further support the broad therapeutic potential of olaptesed pegol," Baumann said.