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ORPHERIS OBTAINS FDA AGREEMENT ON SINGLE, SEAMLESS PHASE 1/2/3 TRIAL TO REGISTRATION FOR ITS LEAD DRUG CANDIDATE, OP-101, AS A TREATMENT FOR CHILDHOOD CEREBRAL ANDRENOLEUKODYSTROPHY (ccALD)

REDWOOD CITY, Calif., Nov. 29, 2017 – Orpheris Inc., a biopharmaceutical company dedicated to developing dendrimer technology to treat neuroinflammatory orphan diseases, today announced the Company held a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss trial design and regulatory path for OP-101, the Company's lead drug candidate, for the treatment of patients with childhood cerebral andrenoleukodystrophy (ccALD), not eligible for bone marrow transplant.

The Company held a meeting with the FDA early this year to review the clinical trial protocol for OP-101, including a single, seamless Phase 1/2/3 trial design and study endpoints. The FDA provided recommendations on the trial design and agreed that a single Phase 1/2/3 trial comprised of approximately 30 patients could be sufficient for registration, given the lack of treatment options for these patients and the fact that the drug candidate is targeting a rare pediatric disease. A follow up meeting with FDA is scheduled in the first quarter of 2018 to review the statistical plan and confirm the regulatory path.

"We are pleased with the outcome of our Type C meeting with the FDA," said Jeffrey L. Cleland Ph.D., Orpheris' co-founder and executive chair. "We are encouraged by the FDA's initial response to a possible single, combined registration trial in ccALD patients, as this could streamline the registration process and significantly reduce trial costs. With orphan drug designation granted earlier this year for OP-101, we remain on target to submit an IND and initiate clinical trials early next year."

"Patients with ccALD typically die within 2-5 years of diagnosis due to progressive neurodegeneration caused primarily by neuroinflammation and oxidative stress," continued Dr. Cleland. "We believe by directly targeting the drug to activated microglia in the brain, OP-101 will be effective in increasing survival and reducing the loss of neurological function in ccALD patients."  

As recently reported by the Company, the FDA also granted Orphan Drug Designation to its lead drug candidate, OP-101, in the first quarter of this year. The Company expects to complete a normal healthy volunteer study in the first quarter of 2018 and begin a clinical trial in ccALD in the third quarter of 2018.

About ccALD
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.

About OP-101
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.  

About Orpheris
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.

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