ProKidney Guggenheim 3rd Annual Healthcare Innovation Conference

started. Thanks everyone for joining us again this year for the Guggenheim Healthcare Innovation Conference. I'm Vamil Divan, one of the biopharma analysts here at Guggenheim. Joining us next in this room, we have the ProKidney team. Next to me, we have Bruce Culleton, the CEO, and we have Anthony Conway, Head of R&D, and then we have Greg Madison on the CCO at the end of the group here. So thanks again for joining us. Maybe, Bruce, I'll turn to you first, just for people who are maybe a little less familiar with the ProKidney story, just a little bit of an overview on the company, and obviously exciting times. A lot of exciting stuff coming up in the next few months. Kind of set the stage, and then we'll go deeper from there.

Sure. Okay. Well, thanks, Vamil, for having us here again. So, I'll just talk about our purpose, our progress, and a little bit about our people too. So we've got 250 employees. Most of them are in North Carolina, or the majority of them are in North Carolina, where we have our manufacturing operations. We have a single purpose as a company, and that single purpose is to stabilize kidney function in patients with advanced chronic kidney disease and diabetes, and give them hope to stay away from dialysis for as long as possible. We have a single asset. The asset's called rilparencel. It's an autologous cell therapy. The cells are harvested through a standard kidney biopsy, expanded ex vivo, and then re-injected back into the kidneys. We've made substantial progress in our clinical development. We're a late-stage clinical development, very late stage. We've completed recruitment. We should finish enrollment in Q1 2027, and we expect an accelerated approval readout in Q2 2027.

Great. Thanks for that overview. So maybe you can just remind us, I think, very interesting, exciting data you've shared to date for rilparencel, maybe just an overview on what you've shared, and then also the stage for the ongoing work.

Sure. This will be a really quick summary of what we've shared.

Okay We could spend the rest of the time just talking about the data.

I'll focus on our REGEN-007 trial that was published in May of this year in the Clinical Journal of the American Society of Nephrology. In one group, one pre-specified group, the 24 patients who were in the group that most looks like how we treat patients in our phase III trial, so a very similar treatment profile, we saw in that a 4.5 mL per minute annualized difference, or a 78% improvement in patients' slope of kidney function decline before treatment with rilparencel versus after treatment. Again, that was a 78% improvement in decline in kidney function. That was from that study.

The other phase II trial that I'll speak of just briefly was the RMCL-002 study, and in that study, we saw the benefit associated with rilparencel treatment really highlighted in the patients who are at highest risk of kidney failure, those with a GFR less than 30. Based upon that information, we did change our phase III protocol to be more focused on patients who've got that highest risk of kidney failure.

Okay. Maybe you can talk about the ongoing trial now. Currently, you mentioned the accelerated endpoint readout.

Oops second quarter of next year.

What are you hoping to see? How is the study designed for that, and then also for the final readout?

Sure. We have a very rigorous phase III trial. It is for cell therapy. It is a randomized, sham-controlled, two-arm, blinded study. Eligible patients have a GFR between 20 and 35. They have to have diabetes, and they have to have a diagnosis of chronic kidney disease. After randomization, patients are allocated to receive rilparencel, which is, once the product is made, there is an injection into the biopsied kidney, and then three months later, an injection into the contralateral kidney. The sham group is a true sham group. In that arm of the study, patients actually do have sham procedures, and for all intents and purposes, they believe that they are actually getting a biopsy and a rilparencel treatment. The endpoints, there are two ways to think about this. It is one trial. There is an accelerated approval endpoint, which is eGFR slope.

We are looking for the difference between the treatment and the control group in eGFR slope. That is the accelerated approval readout, which is again expected in Q2 of next year. The same patients, the same trial, will then be followed until we get to a confirmatory endpoint. Assuming we get accelerated approval, that confirmatory endpoint is a time to composite of events, and that composite event includes a 40% loss in eGFR, a sustained eGFR less than 15, dialysis or transplant, or renal or cardiovascular death. Same study, different endpoints, accelerated approval, and confirmatory endpoint.

For the accelerated endpoint, you mentioned the prior trial where you showed a 74% benefit in sort of pre-treatment, post-treatment here. You will have the comparator arm. On that accelerated endpoint, what can you share about what you need to show from a statistical perspective, but also clinically will be meaningful?

Yeah difference between the two groups?