Regeneron Expands Gene Therapy Portfolio with Decibel Therapeutics Acquisition

Regeneron has announced a significant move in the gene therapy sector by acquiring Decibel Therapeutics for $109 million. The deal centers around DB-OTO, an adeno-associated virus (AAV) gene therapy aimed at correcting mutations in the otoferlin (OTOF) gene, which plays a crucial role in the auditory system by transmitting sound into neuronal signals.

DB-OTO is currently under evaluation in the phase 1/2 CHORD trial across the US, UK, and Spain, which commenced in May and is set to monitor patients until 2030. The trial is enrolling up to 22 children with biallelic OTOF mutations, marking a significant step forward in addressing congenital deafness.

The acquisition includes a contingent value right (CVR) of up to $3.50 per share, potentially increasing the deal's total value to $213 million, contingent upon DB-OTO meeting specific clinical development and regulatory milestones.

This agreement represents a crucial development for Decibel Therapeutics, which has been facing financial challenges despite its 2021 public offering. The acquisition by Regeneron not only provides a lifeline for Decibel but also enhances Regeneron's gene therapy portfolio, granting it full ownership of its first clinical-stage project.

Decibel's pipeline also features DB-020, a drug in phase 1b trials aimed at preventing ear damage caused by cisplatin chemotherapy, alongside preclinical gene therapy candidates targeting hearing loss associated with mutations in the GJB2 and STRC genes.

George Yancopolous, Regeneron's chief scientific officer, emphasized the strategic importance of this acquisition, stating, "We believe that Decibel's assets and specialized 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."

The gene therapy landscape for otoferlin-targeting treatments is competitive, with other biotechs like France's Sensorion and Eli Lilly also advancing their respective therapies. Sensorion recently sought approval for its OTOF-GT therapy trials, while Eli Lilly acquired AK-OTOF through its takeover of Akouos, with a phase 1/2 trial expected to commence.