A growing body of research recognizes that microglia, the resident immune cells of the brain, are a potential driver of disease pathology in those suffering from neurodegenerative diseases such as ALS, Parkinson's, Alzheimer's, and our lead indication, ccALD (click here to learn about Our Target).


Orpheris' clinical focus will be to advance OP-101 to proof-of-concept in in its first indication, childhood cerebral adrenoleukodystrophy (ccALD). We will assess the ability of OP-101 to restore microglia to a normal state, using neural imaging techniques. We intend to employ both radioligands for reactive microglia using positron emission tomography (PET), and gadolinium enhancement using magnetic resonance imaging (MRI). Orpheris has discussed the phase 1/2/3 clinical study protocol with FDA and expects to begin that study upon completion of the ongoing Phase 1 study in healthy normal volunteers.

In parallel to our clinical efforts with OP-101 in ccALD, we are also conducting additional preclinical studies to investigate the potential of OP-101 in other neurological diseases. We have already demonstrated a significant reduction in reactive microglia by OP-101 in an established animal model of Parkinson's disease. We intend to further explore the effectiveness of OP-101 in Parkinson's disease and ALS animal models to enable a rapid transition upon completion of the Phase 1/2 stage ccALD study proof of concept.

We are also exploring the potential to link other disease modifying agents to our platform hydroxyl dendrimer technology. We have several research studies ongoing with our founding scientists at the Johns Hopkins University.

Patient Advocacy

Orpheris works closely with patient advocacy groups in the indications on which we are focusing.

A Mother's Letter to the FDA: Jean Kelley, Mother of Brian Kelley, a ccALD patient

"Our family has lived with this insidious disease for 20 years. In 1995, at the age of 6, our happy, energetic, athletic, thoughtful, loving, son was diagnosed as follow-up of a head injury. Brian hit a woodpile while sled riding. It was an accident that turned out to be a blessing. Despite a bone marrow transplant Brian progressed rapidly. You cannot fathom, until you live it, how upsetting and painful it is to watch your child lose his abilities and skills, one after another after another, relentlessly. On July 24, 1995, Brian's last words to me were a labored and whispered, "I love you". He last saw me smile at him the following February, when he lost his vision. Next came the spasticity that engulfed his body rendering him dependent on others forever. It was a cavalcade of maladies that in retrospect, gave little time for reflection, only reaction. Problems and losses continue to arise. The many children who struggle daily with no treatment deserve a chance at any improvement in their quality of life."

Visit www.brianshope.org »