SAN DIEGO, CALIFORNIA — Otonomy, Inc. (Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical proof-of-concept results for OTO-825 presented at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting. OTO-825 is an AAV-mediated gene therapy targeting the gap junction beta-2 (GJB2) gene developed under the company’s collaboration with Applied Genetic Technologies Corporation (Nasdaq: AGTC).
These results demonstrate that a single administration of OTO-825 rescues hearing loss and cochlear damage in two preclinical models that represent a range of hearing loss severity caused by GJB2 deficiency.
“The treatment of GJB2 deficiency is an important unmet medical need since it is the most common genetic mutation causing congenital hearing loss with many patients experiencing significant impairment beginning at birth. These proof-of-concept results in two independent preclinical models are an important milestone for the program because they validate the therapeutic potential of OTO-825 across a range of hearing loss levels observed in patients and support its advancement into IND enabling activities, which are currently underway.”
–Alan C. Foster, Ph.D., Chief Scientific Officer at Otonomy
The joint oral presentation by Otonomy and AGTC demonstrates the following:
- OTO-825 induces expression of Connexin26, the protein product of the GJB2 gene, in target cochlear cells including support cells of the organ of Corti and spiral limbus, and fibrocytes of the spiral ligament in both rodents and non-human primates.
- Connexin26 expressed by OTO-825 forms functional gap junctions, which are required for proper functioning of cochlear hair cells.
- Two conditional knockout models developed in mice mimic the human setting for GJB2 deficiency by displaying hearing loss and damage to the integrity of cochlear tissue.
- A single intracochlear administration of OTO-825 rescues expression of Connexin26 in both models.
- OTO-825 induces significant improvement in hearing across multiple frequencies and normalizes cochlear morphology in both models.
Mutations in the GJB2 gene account for approximately 30% of congenital hearing loss cases. Patients with GJB2 mutation can have severe-to-profound deafness in both ears that is identified in screening tests routinely performed in newborns.
Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs with a focus on hearing loss and tinnitus. For additional information please visit www.otonomy.com.