Tenaya Therapeutics, Inc. Common StockTNYA
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Tenaya Therapeutics, Inc. Common Stock Canaccord Genuity's 46th Annual Growth Conference

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Whitney IjemBiotech Analyst

Whitney Ijem. I am one of the biotech analysts here at Canaccord, and it is my pleasure to be joined by Tenaya. I think I said good afternoon. Tenaya, this afternoon, pleased to be joined by Eric Hyllengren, CFO. Thank you for making the trip. Thank you for coming. We have a lot to cover, so diving right in. For anybody who is not familiar with the story, can you provide just a high-level overview of what attracted you to the company, because you are relatively new, and what is the team trying to build over the next 5-10 years?

Eric HyllengrenCFO

Yeah, great one. Thanks for having me, Whitney. I have been in the chair almost a month, so still new guy. When I thought about Tenaya and joining the company, really it is about the mission. We are looking for precision medicines for heart disease. We are modality agnostic, so we have gene therapy, we have small molecule, but really we are trying to address the underlying cause of the disease, and so the mission of the company really resonated with me. Also, the science, when I looked at the data presented to date, strong data, very encouraging. The team. The team that I met with during the interview process, very excited and energized to succeed in this space. We have a big year here, Q4, and we will get into this, but regulatory updates coming, our plans for advancing our small molecule TN-301.

Eric HyllengrenCFO

Also we will talk about those in Q4, so a lot of near-term events for the company as well.

Whitney IjemBiotech Analyst

Okay. Excellent. Maybe starting with TN-201, gene therapy. Can you just briefly review what that is? What is MYBPC3-associated HCM, and the kind of key unmet need in this patient population?

Eric HyllengrenCFO

Right. As you said, with MYBPC3 in HCM, we are talking about about 120,000 patients in the U.S. here. That is about 20% of all HCM. Really what we see with these patients is a thickening of the left ventricle, so the heart is not able to pump blood where it needs to go in the body effectively. There is really a lack of effective treatments out there, especially with folks with more severe disease. We feel like targeting this area really our gene therapy can improve outcomes and results for these folks.

Whitney IjemBiotech Analyst

Okay. Which endpoints are most important to you guys as you guys are monitoring the data and as you are talking to the physicians as well?

Eric HyllengrenCFO

Sure. As I mentioned, these left ventricles thicken or they get large, so reducing the thickness of the left ventricle. LVMI is very important to us. We feel like that then would contribute to better outcomes. We are having discussions with regulators on appropriate endpoints, but the data we have shown to date has been very strong in that area, so we would think that would be appropriate.

Whitney IjemBiotech Analyst

Okay. Got it. The expansion cohort in the study is ongoing. Can you remind us which dose is being used, what the target enrollment is, and I guess, yeah, if there has been any changes there as the study has evolved over time?

Eric HyllengrenCFO

Yeah. So in this cohort 2, it is the higher dose, so 6E13. Cohort 1 was half of that, and really well tolerated from a safety perspective. And again, the LVMI reductions we saw were very meaningful in the majority of patients. I think also what is important is from a feel and function kind of symptoms, those improved as well for most patients. So it is not just LVMI, but it is how these folks are feeling.

Whitney IjemBiotech Analyst

Okay, perfect. You just touched on it as well with safety, all important in general, but particularly against the bad things that have been happening in the broader cardiac gene therapy space. Can you talk about what you have seen there Yeah with what you have had?

Eric HyllengrenCFO

Right. Very well tolerated thus far. Favorable safety profile at both doses. We really have not seen, I think maybe what you are hinting at is some other folks in the space have seen some issues. We really have not seen that, so we are very confident that what we are moving forward is safe.

Whitney IjemBiotech Analyst

Mm-hmm. Okay. The data so far in the ongoing expansion cohort in adults.

Whitney IjemBiotech Analyst

Correct. You are also running a natural history study in pediatric patients.

Whitney IjemBiotech Analyst

Right. What was the rationale for that study and what are you learning so far?

Eric HyllengrenCFO

Yeah. When we started, there really was not a whole lot of data out there in pediatrics, and so we thought that there was a need there to look at that population. Really the younger the age of the symptom onset, the higher the complications later on. So it is really can you catch these patients early? So thought that that was a good thing to look into, and we are. Yes, we think that there could be a possibility that ultimately we study this in pediatric down the road.

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