PROJECT SUMMARY
Autoimmune inner ear disease (AIED) is the name given to describe a disorder where the ear is the target of
an overactive immune system, causing symptoms of progressive hearing loss and/or dizziness. Most cases of
AIED are accompanied by hearing loss and tinnitus that occur over a course of a few months1,2 that progresses
rapidly to the second ear, indicative of AIED.3 There are currently no FDA-approved medications for the
treatment of AIED, or any type of inner ear hearing loss. The use of high dose oral or intratympanic injection
corticosteroids to suppress immune function is the most common treatment for AIED. However, the use of high
doses of corticosteroids leads to significant side effects. In response to this unmet medical need, O-Ray
Pharma has developed a lead formulation of sustained-release fluticasone pellets (OR-102) for the treatment
of AIED that will provide a consistent low dose of corticosteroid to the cochlea over a period of three months.
The pellet is implanted directly into the cochlea and releases steroid at the affected site to protect against
hearing loss. The OR-102 formulation has demonstrated its safety, pharmacokinetics (PK), and efficacy in
multiple guinea pig studies.
To test the safety and preliminary efficacy of OR-102 in a first-in-human clinical trial in AIED-affected patients,
O-Ray Pharma has submitted an IND application to the FDA. The FDA provided tacit approval of the majority
of the application save for a request to perform an additional animal study and suggestions for additional
Chemistry/Manufacturing/Control (CMC) work. In response, there were no clinical holds pertaining to the
clinical protocol and the chemistry/manufacturing/controls procedures. Thus, the IND is on clinical hold pending
the completion of a Good Laboratory Practices (GLP) compliant toxicity and pharmacokinetics study. This IND
correspondence has provided us with a clear framework towards an IND allowance. The Specific Aims of this
Direct to Phase II proposal are, therefore, to complete the following IND-enabling activities: 1) Drug product
manufacturing in accordance with current Good Manufacturing Practices (cGMP) for animal and human trials;
2) Performance of a GLP-compliant guinea pig study as guided by FDA feedback, 3) Preparation and
submission of an IND response. The milestone for the successful completion of this grant will be the granting
of an IND approval.
The team of investigators at O-Ray is uniquely qualified to perform the work proposed herein, and has
expertise in otology, regulatory affairs, and business development. O-Ray’s scientists have successfully
developed other therapeutic formulations from concept to FDA approval, including an intraocular sustained-
release steroid implant capable of maintaining anti-inflammatory intravitreal drug levels for up to three years
from a single implantation. O-Ray’s management has previously out-licensed three drug delivery products to
larger pharmaceutical companies upon demonstration of proof-of-concept in the target patient populations.
