Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q4 2022 Earnings Call Transcript

Sean Nolan: Well, I’ll give you my answer to that and Suku, please opine. But when you look at FDA guidance and you look at accelerated approval, there’s generally a consideration of a controlled trial. And that controlled trial is one of the designations would be as an example, natural history. So I think there’s a distinction between a controlled trial and a double blind placebo controlled trial. And the language written in the responses that we got from the FDA are clear on those. When they were talking about MFM32, they were talking about a double blind placebo controlled trial, when they were talking about alternate trial designs that met the bar of clinically meaningful and objective end points, they talked about a controlled trial. So I think they’re speaking to the letter of the guidance, but Suku, I would ask you for further clarification on that.

Sukumar Nagendran: Yes. Thanks, Sean. So given that it’s an ultra-rare disease, doing a placebo controlled trial, I don’t think it will be practical and could take forever. So if there are alternative study design, necessary or have to be discussed, the simplest would be to dose a few more patients where you already have pre-treatment natural history for comparison. So with that caveat, our hope is that the collective dataset that we submit to the FDA will qualify for a serious review and potential appropriate approval if the FDA thinks that’s the best way path forward for the patient community. So I would leave it at that. And Sean, is there any other question you wish me to address? So hopefully, we addressed the question that was asked.

Sean Nolan: No, I think you did.

Sukumar Nagendran: Okay. Thank you.

Joon Lee: Thanks.

Operator: There are no further questions at this time. I would like to turn the floor back over to Sean Nolan for closing comments.

Sean Nolan: Thank you, everyone for their time and interest in what we’re trying to accomplish here at Taysha. I’m very pleased that the progress we’ve made in the short time period in 2023, we’ve got a long way to go, we are working hard, and we look forward to sharing future updates with you in the coming months. Thank you all and have a good night.

Operator: This concludes today’s teleconference. You may disconnect your lines at this time. Thank you for your participation.