Regeneron Touts Encouraging Early Data From Investigational Gene Therapy For Rare Form Of Hearing Loss

On Monday, Regeneron Pharmaceuticals Inc. REGN announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene.

Regeneron snapped up DB-OTO through its $109 million acquisition of Decibel Therapeutics in August 2023.

Congenital deafness (hearing loss present at birth) affects approximately 1.7 out of every 1,000 children born in the U.S. Approximately half of these cases have genetic causes.

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However, otoferlin-related hearing loss is ultra-rare. It is caused by variants in the OTOF gene, which lead to a lack of a functional otoferlin protein critical for communication between the inner ear’s sensory cells and the auditory nerve.

These include 72-week results showing speech and development progress in the first child dosed at 10 months of age and initial results in 11 children (aged 10 months to 16 years old) – three of whom received DB-OTO bilaterally (in both ears).

Among the 11 participants with at least one post-treatment assessment, 10 demonstrated a notable response, with improved hearing at various decibel hearing levels. Additionally, among five participants with 24-week assessments, three experienced improvements in average hearing thresholds to “nearly normal” or normal hearing levels.

One participant did not experience a change from their baseline hearing at 24 weeks post-dosing.

Across all 12 participants, the surgical procedure and DB-OTO were well tolerated and no adverse or serious adverse events were considered related to DB-OTO.

Five of the 12 participants experienced transient post-surgical vestibular adverse events (e.g., nystagmus, nausea, dizziness, vomiting), which resolved within 6 days of dosing.

Price Action: REGN stock closed at $713.25 on Monday.

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