RARE Daily

Orbus Enters Exclusive License Agreement for IP Related to Treatment of Rare Pediatric Syndrome

January 11, 2023

Orbus Therapeutics said it has entered into an exclusive license agreement with Michigan State University and Helen DeVos Children’s Hospital that grants Orbus exclusive rights to intellectual property related to the method of use of eflornithine in the treatment of Bachmann-Bupp Syndrome, a rare pediatric condition.

Photo: Bob Myers, CEO of Orbus

Bachmann-Bupp Syndrome (BABS) is a newly recognized autosomal dominant genetic disorder caused by a heterozygous de novo variant in the ornithine decarboxylase 1 (ODC1) gene. ODC1 produces an enzyme that is integral in putrescine biosynthesis, which is involved in cell division. BABS is a rare condition, where a gain-of-function mutation in ODC1 causes an increase in ODC expression and levels of putrescine in patients that leads to disabling neurodevelopmental deficits. Researchers at Michigan State University and Helen DeVos Children’s Hospital have shown that oral delivery of eflornithine improves physical coordination, attention, and interaction in patients with BABS.

Eflornithine is a novel cytostatic agent that irreversibly inhibits ornithine decarboxylase, a key enzyme in mammalian polyamine biosynthesis that is up-regulated in certain types of cancer. Eflornithine injection was approved by the U.S. Food and Drug Administration in 1990 for the treatment of African trypanosomiasis (sleeping sickness), but it was never made commercially available in the United States. In 2000, the FDA approved a topical eflornithine cream for the reduction of unwanted facial hair in women.

Eflornithine has been granted Orphan Drug Designation and Breakthrough Therapy Designation for the treatment of patients with anaplastic glioma by the U.S. Food and Drug Administration and has also been granted Orphan Medicinal Product status for the treatment of glioma by the Committee for Medicinal Products for Human Use at the European Medicines Agency.

Orbus Therapeutics is currently conducting a phase 3 clinical trial, referred to as the STELLAR study, to evaluate its eflornithine oral solution in patients with anaplastic astrocytoma whose cancer has recurred following radiation and adjuvant temozolomide chemotherapy. The company plans to expand its activities to include development and future commercialization of its eflornithine oral solution in BABS in parallel with its ongoing efforts in malignant glioma.

“These children face significant neurological and development hurdles as a result of this gain-of-function mutation,” said Bob Myers, CEO of Orbus. “We have been very impressed by the results of the ongoing work at MSU and HDVCH, and the positive impact that has been seen in the development of these children following treatment with eflornithine.”

Author: Rare Daily Staff

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