Founded in 2016, Boston-headquartered Akouos has integrated expertise across otology, inner ear drug delivery, and gene therapy.
Its lead candidate AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the otoferlin gene (OTOF), received FDA clearance for its Investigational New Drug (IND) application in September to initiate a Phase 1/2 pediatric trial (In April 2021, the FDA granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy).
Lilly will pay $487m in cash with the potential for the deal’s value to rise to $610m upon certain clinical and regulatory milestones.
Building a gene therapy portfolio
Aside from AK-OTOF, additional pipeline programs span across multiple inner ear conditions, and include AK-CLRN1 for Usher Type 3A, an autosomal recessive disorder characterized by progressive loss of both hearing and vision; GJB2 (which encodes connexin 26) for a common form of monogenic deafness and hearing loss; and AK-antiVEGF for the treatment of vestibular schwannoma.
The deal has been approved by the boards of directors of both companies.
Lilly established its gene therapy program via its acquisition of New York’s Prevail Therapeutics in 2021 for $880m in cash, which saw the US pharma giant gain AAV9-based gene therapies for patients with neurodegenerative diseases.
"Gene therapy offers tremendous opportunity to provide durable treatments for patients with genetically defined disease; this is our second acquisition in gene therapy, following the acquisition of Prevail Therapeutics," noted Daniel Skovronsky, M.D., Ph.D., Lilly's chief scientific and medical officer, and president of Lilly Research Laboratories. "With Akouos, we are optimistic that we can make a difference for people with hearing loss and other inner ear conditions."
"I am proud of the commitment and passion of our team, which has established Akouos as a pioneer in inner ear genetic medicine, as demonstrated by our work to advance the first investigational therapy for a genetic form of hearing loss into clinical development," said Emmanuel Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. "Joining Lilly – a company that shares our purpose to make life better for people around the world – will help us accelerate the development of a broad pipeline of inner ear genetic medicines."