Lilly’s hearing loss win
This week, Eli Lilly shared big news about its investigational gene therapy: the treatment restored hearing in the first trial patient, an 11-year-old child.
The trial is being conducted by Lilly’s subsidiary, Boston-based Akouos. According to Akouos, millions of people are deaf or hard of hearing as a result of genetic mutations affecting any one of over 150 genes linked to inner ear dysfunction. The company believes that in some cases, it’s possible to recover hearing by delivering a healthy copy of the affected gene to the cells that need it — and the positive clinical results in this first patient support that assertion.
Akouos' dual adeno-associated viral vector-based gene therapy is intended for the treatment/ potential restoration of hearing in individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene. It’s administered one time, delivering copies of the normal OTOF gene directly into the inner ear, via Akouos’ sterile investigational medical device.
According to Lilly, an estimated 200,000 individuals worldwide live with OTOF-mediated hearing loss.
It’s early days for the treatment, but Lilly plans to present the trial results, including initial data from a second participant, at an upcoming Association for Research in Otolaryngology meeting. As more patients of varying ages are treated, more will be learned about the degree to which hearing can be improved and whether or not that improvement can be sustained. It also opens the door for the use of the therapy on other gene mutations that affect hearing.
The delivery method represents the first time a gene therapy has been used as a potential treatment for hereditary hearing loss in the U.S. — an incredible leap forward. —Karen Langhauser
Sandoz is open for business
Signaling its newfound independence, Sandoz made its first acquisition earlier this week by clinching a deal for the ophthalmology drug Cimerli from Coherus BioSciences.
Cimerli is a biosimilar to Roche and Novartis’ blockbuster ophthalmology therapy, Lucentis. Lucentis lost its exclusivity in 2021, and that same year the FDA approved Samsung Bioepis and Biogen’s Byoovis as an interchangeable biosimilar for the drug across three indications: wet age-related macular degeneration, macular edema following retinal vein occlusion, and myopic choroidal neovascularization. But in 2022, Cimerli became the first biosimilar product capable of substituting for Lucentis across all five approved indications, adding diabetic macular edema and diabetic retinopathy.
The deal with Coherus, which was accompanied by an upfront cash payment of $170 million, not only marks Sandoz's entry into the acquisition arena but also underscores its determination to carve out a distinct identity in the industry.
What’s exciting is that this transaction is harbinger of future moves. With a robust pipeline boasting over 15 biosimilar molecules, the message is clear — Sandoz is open for business, ready to explore new opportunities and committed to crafting a narrative of success in the competitive biosimilar industry. — Andrea Corona