Regeneron dials up in gene therapy with Decibel deal

gene therapy for congenital deafness

Regeneron has agreed a $109 million deal to acquire Decibel Therapeutics and its early-stage gene therapy for a form of congenital deafness, several years after forming a partnership to tackle hearing loss.

The main focus of the deal is Decibel's DB-OTO, an adeno-associated virus (AAV) gene therapy designed to correct mutations in the otoferlin or OTOF gene, which codes for a large protein expressed in hair cells in the cochlea (inner ear) that is involved in the process of transmitting sound into neuronal signals.

DB-OTO is already being tested in the phase 1/2 CHORD trial running in the US, UK, and Spain, which started in May and will follow patients until 2030. It is enrolling up to 22 children with biallelic OTOF mutations.

Regeneron's $4-per-share transaction also includes a contingent value right (CVR) of up to $3.50 per share if DB-OTO meets clinical development and regulatory targets within a specified time frame, which could increase the value of the deal to $213 million.

The agreement is something of a lifeline for Boston, Massachusetts-based Decibel, which went public in 2021 with a $127 million offering, but has started to run out of cash, with its latest update indicating it had sufficient reserves to last only into the first half of next year.

Adding Decibel builds Regeneron's position in gene therapy, giving it full ownership of its first clinical-stage project and adding to an early-stage alliance with ViGeneron on AAV-based therapies for disorders of the eye, signed last year.

Decibel's pipeline also includes DB-020, a drug to prevent damage to the ear caused by cisplatin chemotherapy that is in phase 1b, as well as preclinical gene therapy candidates for hearing loss associated with mutations in the GJB2 and STRC genes.

"We believe that Decibel's assets and specialised team will further strengthen our genetic medicines portfolio, enabling Regeneron to accelerate the development of innovative genetic therapies and a rich pipeline of hearing loss treatments," said George Yancopolous, Regeneron's chief scientific officer.

There are other biotechs working on otoferlin-targeting gene therapies, including France's Sensorion, which filed for approval to start trials of its OTOF-GT therapy last month, as well as Eli Lilly, which picked up a candidate called AK-OTOF as part of its $610 million takeover of Akouos last year. AK-OTOF was due to start a phase 1/2 trial in May.