On Sunday, Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) unveiled updated data from its investigational gene therapy DB-OTO for profound genetic hearing loss caused by variants of the otoferlin (OTOF) gene.
The New England Journal of Medicine published the report. The annual American Academy of Otolaryngology-Head and Neck Surgery meeting presented the data.
These latest results from the pivotal CHORD trial show 11 out of 12 participants have experienced clinically meaningful hearing improvements. This includesthree who achieved normal hearing levels.
Additionally, eight participants with longer follow-up showed stability or continued improvement in their hearing, and among the three who completed speech assessments, all showed significant improvement.
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Nearly all participants (11 of 12; 14 of 15 treated ears) demonstrated improved hearing, responding within weeks of treatment. As published and presented, the trial met the primary endpoint with 9 participants experiencing hearing improvements at a threshold of less than 70 dBHL as assessed by behavioral pure tone audiometry (PTA) at week 24.
Six could hear soft speech without assistive devices, and three were further able to detect whispers (achieving normal hearing sensitivity).
One participant who did not meet the primary PTA endpoint at week 24 further improved to achieve “nearly normal” hearing sensitivity at week 48.
Nine participants also demonstrated an auditory brainstem response (ABR) at ≤90 decibels (dB), achieving the trial’s key secondary endpoint.
Hearing improvements remained stable or continued to improve in eight participants who had follow-up visits of more than 36 weeks (up to 72 weeks).
Speech development was also assessed in the three participants who were followed for at least 48 weeks. All showed significant improvements.
One of these participants demonstrated the ability to identify one- and two-syllable words with no visual cues and could respond to distant sounds and speech in noisy environments.
Across all 12 participants, both the surgical procedure and DB-OTO were well tolerated, and no DB-OTO-related adverse findings were reported. Two participants experienced serious adverse events. One was attributed to a cochlear implant surgical complication, and the other to a recent vaccination.
The U.S. regulatory application for DB-OTO is planned for later this year.
Price Action: Regeneron stock is down 1.7% at $554.95 at the last check on Monday.
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