CLRN1

Three months after hearing loss-focused company Akouos secured $105 million in a Series B financing round, the Boston-based company is eying an initial public offering valued at about $100 million. Three months after hearing loss-focused company Akouos secured $105 million in a Series B financing round, the Boston-based company is eying an initial public offering valued at about $100 million. Akouos is a genetic medicine company developing potential gene therapies to restore and improve hearing loss. The company’s lead program is AK-OTOF, a gene therapy for people with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene. Approximately 7,000 people are affected by this mutation, the company speculates. In its filing with the U.S. Securities and Exchange Commission, Akouos said advances in technology have paved a path for potential one-time treatment of hearing disorders with a gene therapy treatment. For its lead candidate, the company said it can restore otoferlin expression through targeted delivery of a proprietary AAVAnc. The company is currently designing a Phase I/II trial for AK-OTOF and Akouos said it anticipates results will enable the company to quickly determine a clinical response. “There are no pharmacologic therapies currently approved for the treatment or management of OTOF-mediated hearing loss, or any other form of sensorineural hearing loss. We believe our product candidate has the potential to restore physiologic hearing and provide long-lasting benefits to these individuals and their families. We plan to submit an investigational new drug application, or IND, for AK-OTOF for OTOF-mediated hearing loss to the FDA in 2021, and we expect to report preliminary clinical data in 2022,” the company said in its SEC filing. In addition to AK-OTOF, Akouos has a number of programs in its developmental pipeline. It said its most advanced programs address a range of inner ear cells and leverage different modalities. These programs include CLRN1 for Usher Type 3A, an autosomal recessive disorder characterized by progressive loss of both hearing and vision; GJB2 for a common form of monogenic deafness and hearing loss; and delivery of an anti-VEGF, an inhibitor of vascular endothelial growth factor, or VEGF, a protein that can cause abnormal blood vessel growth, for the treatment of vestibular schwannoma, a tumor of the auditory vestibular nerve. Akouos filed its IPO prospectus last week with the U.S. Securities and Exchange Commission. The company plans to list on the Nasdaq Stock Exchange under the ticker symbol AKUS. Akouos was formed in 2016 and to date has raised $162.7 million to support its research and development efforts. Following its March Series B financing round, Manny Simons, president and chief executive officer of Akouos, predicted that 2020 would be a year of growth for the company. hbspt.cta.load(4413123, '92ebc33d-a4a8-4c10-b41e-a52c39dc69ea', {});