Our Pipeline

Pediatric Brain Stem Cancer


Our initial drug products are Orphan Drugs for the treatment of intractable, high-grade gliomas in the brain. Diffuse Intrinsic Pontine Glioma (DIPG) and glioblastoma multiforme (GBM) are two primary brain cancers that have an extraordinarily low patient survival rate. There is currently no effective therapy to treat or control either of these aggressive tumors.

Our first drug product (ORX-101) is being developed for Diffuse Intrinsic Pontine Glioma (DIPG), a glioblastoma-like tumor in the brain stem and one of the most resistant of all cancers. DIPG primarily affects young children between 5 and 7 years old. Most of these brain stem tumors cannot be surgically removed because the migrating tumors become entangled around the brain stem nerves. Fewer than 10% of these children will live longer than 18 months from their initial diagnosis because there are no DIPG drugs that can penetrate the brain stem. ORX-101 easily penetrates the blood-brain-barrier (BBB).

Glioblastoma multiforme


Our second drug product (ORX-102) is being developed for glioblastoma multiforme (GBM), a tumor that is difficult to treat with drug therapy and one which is associated with poor patient survival. Over 90% of patients with astrocytomas have high-grade glioblastoma multiforme tumors when initially diagnosed; and these patients have a median survival of about 1 year from diagnosis with standard-of-care surgery, and radiation therapy with Temodar®. The five-year survival rate for patients with GBM and associated regional metastases using currently available therapies is less than 3%. ORX-102 easily penetrates the blood-brain-barrier (BBB).


Our follow-on indications for the treatment of tumors that readily metastasize to the brain include malignant melanoma, lung cancer, colon cancer, pancreatic cancer and estrogen-negative breast cancer. These are expected to provide the company with significant growth opportunities.