Talphera, Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Right. Well, just briefly on Talphera and who we are, we're an acute care company focused on nephrology, in particular in renal care in the acute care setting, as mentioned. We've got three major catalysts over the course of this next year. One is a pivotal study readout on our lead program and really highly focused program. That's nafamostat mesylate NIAD for anticoagulation with extracorporeal circuit. We plan on having that data around the end of this year, that's our goal, and filing the PMA within three months thereafter. It has breakthrough designation and a six-month review clock, so the goal is to have this product approved and on the market in the second half of next year. So it could look very different from that standpoint versus where we are today.

Yeah, I'll turn that to Dr. Aslam to answer that.

Sure. Thank you. We are obviously over 75% enrolled, so that's great. I think that the most difficult and challenging part really is behind us. Finding sites which met our criteria based on the learnings we had from our earlier sites. We came up with a target profile of the sites, which would be a site that has a nephrologist as the investigator and has access to medical ICUs as well, because that's where most of our patients coming from. The changes that we implemented, and we had all the target profile sites up and running, and we have seen tremendous improvement and progress over past few months in terms of enrollment where we are. I think everything right now is going as planned. I feel very confident that we'll wrap this thing up before the end of the year.

If you add on this, we have monthly roundtable meetings with our research teams, and now that they see the study essentially in home stretch, they even are more engaged and excited, and they kind of understand the profile of nafamostat once it's approved and how it addresses their unmet needs. There's a lot of excitement from them to actually finish up the enrollment and get this into approval process.

You got to remember, Dr. Shakil Aslam, when he came in, made some significant modifications to the protocol, all in alignment with the FDA. The relationship with the FDA has been outstanding. We got the breakthrough designation under Dr. Aslam's recommendation. They reduced the N from 166 patients all the way down to 70. They took other certain protocol changes to consideration. It's made a much smoother pathway so that the new institutions that gradually came on and those that we had inherited had an easier time to enroll patients.

Yeah. In terms of the primary endpoint, I can say with a lot of confidence there is essentially no risk to the study. That's very, I would say, daring statement to make, and I'll tell you why. It's not a clinical endpoint per se. We're looking at activated clotting time, which is a measure of how long it takes for blood to clot. All anticoagulants, they work by slowing down the process, so it takes longer for blood to clot. That's very objective. You take a patient whose clotting time is less than 150 seconds, you give them a blood thinner, which is supposed to prolong their clotting time. We get them into a therapeutic range, and we measure it six times during first 24 hours.

Our primary endpoint based out of those six readings that we get, we pick four readings and average them out to a placebo, which will have no effect on ACT. It's a very objective endpoint. On top of it's not an endpoint where you just use a fixed dose. In many clinical trials, you pick a dose or two, and you do your intervention, and then after some time, you see whether it worked or not. Sometimes, not everybody's going to respond the same way to the same dose. Here, we actually go up on the dose within 15 minutes to see if we increase the ACT where we want it. That makes it very foolproof or fail-proof endpoint.