ORPHERIS RECEIVES ORPHAN DRUG DESIGNATION FROM THE U.S. FDA FOR ITS LEAD DRUG CANDIDATE, OP-101, FOR THE TREATMENT OF ccALD
REDWOOD CITY, Calif., Nov. 27, 2017 – Orpheris Inc., a biopharmaceutical company dedicated to developing dendrimer technology to treat neuroinflammatory orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to OP-101, the Company's lead drug candidate, for the treatment of patients with childhood cerebral andrenoleukodystrophy (ccALD), not eligible for bone marrow transplant.
"We are very pleased to receive Orphan Drug Designation from the FDA for OP-101," said Sujatha Kannan M.D., Orpheris' co-founder and chief medical officer and associate professor of anesthesiology and critical care medicine and pediatrics at the Johns Hopkins University School of Medicine. "This is an important milestone, not only for the Company, but for patients with ccALD and their families."
"Being granted Orphan Drug Designation is a significant step forward for us and speaks to the potential of the drug and the high unmet medical need of this disease," said Jeffrey L. Cleland Ph.D., Orpheris' co-founder and executive chair. "We plan to complete a normal healthy volunteer study in Q1 2018 and begin a clinical trial in ccALD in Q3 2018. Orphan drug status should enable us to streamline the regulatory path for OP-101, helping us provide hope to families and children that currently have no available treatment options."
The FDA's Orphan Drug Program offers orphan status to drugs and biologics that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. The designation provides development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
Adrenoleukodystrophy, or ALD, is a deadly genetic disease that affects 1 in 18,000 people. It most severely affects boys and men. The cerebral form of the disease, ccALD, appears in childhood, generally between the ages of four and ten years old. This is the most common form of ALD, representing about 45% of all ALD cases. It is characterized by a severe loss in brain function and eventual death in most cases. Currently, the standard of care for preadolescent patients in the early stages of ccALD pathology is hematopoietic stem cell transplantation (HSCT). Orpheris believes that OP-101 will be effective in increasing survival and reducing the loss of neurological function in ccALD patients not eligible for bone marrow transplant.
Orpheris' lead product, OP-101, is a new chemical entity containing an approved anti-inflammatory drug that is effective at reducing inflammation and oxidative stress. OP-101 is selectively targeted to activated microglia and astrocytes in the brain and releases the drug upon internalization. OP-101 consists of a hydroxyl poly (amidoamine) (PAMAM) dendrimer conjugated to an anti-inflammatory drug with a disulfide bond that is designed to be cleaved only under intracellular conditions. In clinical studies, the free drug is administered IV to ccALD patients before and after bone marrow transplant resulting in increased survival with minimal impact on progressive loss of neurological function. By directly targeting the drug to activated microglia in the brain, Orpheris believes that OP-101 will be effective in increasing survival and reducing the loss of neurological function in ccALD patients not eligible for bone marrow transplant.
Orpheris is focused on the treatment of neuroinflammatory orphan diseases that result in death or severe disabilities, including childhood cerebral adrenoleukodystrophy (ccALD), Parkinson's disease, amyotrophic lateral sclerosis (ALS), Huntington's disease, Rett syndrome and autism spectrum disorders. Orpheris' patented hydroxyl dendrimer technology selectively targets activated microglia implicated in a number of brain diseases. Controlling neuroinflammation through targeting activated microglia with OP-101 is a potent weapon against CNS disorders. OP-101 is the company's first potential drug candidate utilizing hydroxyl dendrimer technology to deliver therapeutic doses of an anti-inflammatory drug across the blood brain barrier to reduce neuroinflammation.