Our Pipeline

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Our initial drug products are Orphan Drugs for the treatment of intractable, high-grade gliomas in the brain. 

Diffuse Intrinsic Pontine Glioma (DIPG) and recurrent medulloblastoma 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-102) 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.

Upon completion of our preclinical studies, we plan to conduct a clinical trial in patients with DIPG. We further plan to conduct a clinical trial with ORX-102 in patient with recurrent medulloblastoma who have DIPG-like characteristics. Despite the success of current therapies, medulloblastoma patients who survive live with major disabilities caused by those therapies.

Upon completion of our initial clinical trials, we plan to conduct a clinical trial with ORX-102 for the treatment of glioblastoma multiforme (GBM). GBM is difficult to treat with drug therapy and 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%.  

In addition to gliomas that occur as primary tumors in the brain, we plan to develop ORX-102 for the treatment of  metastatically aggressive tumors that share strikingly similar phenotypical and morphological characteristics. 

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