Taysha touts early Rett gene therapy data, but safety challenges dampen enthusiasm

2024-06-18

基因疗法临床结果

Taysha Gene Therapies provided a peek at new data for its Rett syndrome gene therapy on Tuesday, highlighting evidence of clinical improvement in two adults and two paediatric patients. Investors, however, may have been wary of the “challenging side effects” seen in the children, as the biotech’s stock dropped more than 26% on the data.

The biotech emphasised that the adverse events (AEs) experienced by both paediatric patients were related to a separate immunosuppressant treatment that led to “significant challenges.” The two reported serious AEs were deemed unrelated to the gene therapy, TSHA-102, and were resolved.

Taysha said TSHA-102 had a well-tolerated safety profile with no dose-limiting toxicities, and that an independent data monitoring committee has given the company the go-ahead to begin testing a higher dose of the gene therapy in paediatric patients starting in the third quarter.

Overall, both the early data in the paediatric pair, as well as the long-term data in the two adults, showed improvements across several key clinical areas, including motor skills, communication, socialisation, autonomic function, and seizures.

Clinical improvements

The REVEAL Phase I/II study has thus far dosed four patients aged six, seven, 20 and 21, which each had follow-up data available for 12 weeks, eight weeks, 52 weeks, and 36 weeks after receiving the single-dose gene therapy, respectively.

Both adult patients demonstrated improved breathing patterns and circulation. The 20-year-old also experienced improved hand function and vocalisation, and was able to sit unassisted. Their seizure events were stable.

The 21-year-old had improved posture and stability, and showed an increased social interest and response to spoken words. They saw a significant reduction in seizures.

“It’s encouraging that we continue to see improvements across multiple clinical domains in the longer-term assessments with no diminution of effect,” said Elsa Rossignol, who was the principal investigator for the adult cohort.

Similarly, the paediatric patients demonstrated improved hand function and an increased social interest. The seven-year-old was able to maintain eye contact more frequently, had greater stability while walking, and experienced more seizure-free days.

The six-year-old saw an improvement in their swallowing as well as their communication, and also reduced their breath-holding.

“The initial improvements observed across multiple areas of disease in both paediatric patients are encouraging early signs of possible benefit,” commented Colleen Buhrfiend, the principal investigator for the paediatric portion of the trial.

For more on the gene therapy landscape, see Vital Signs: Gene therapy’s last stand.

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