CAMP4 Therapeutics Corporation Common Stock Chardan's 10th Annual Healthcare Conference

Well, first, thank you for having me. Second, I am going to take you everywhere, every time I go speak with investors because you did a great job talking about ADAR. Just for the folks that do not know what ADAR editing is, I will spend one minute on it. Our drug product, when people think about the word editing, people think about gene therapy and CRISPR. We are not that, right? Our drug product in this case is an oligonucleotide that is similar to an ASO from Ionis or an siRNA from Alnylam. That is the drug product. Easily manufacturable and very scalable. We use that to think about this as a landing pad for an endogenous protein, in this case, called ADAR to come and make a single alphabet change.

The good news is that at any given point in time, about 2%-3% of cells in our body convert this A to I to G change by itself, we do not have to do anything. All we do is provide the drug as a landing pad and a targeting moiety to come and make that change.

As Kei mentioned, there are many uses for it. One of it is the repair of a pathogenic variant. I am an optimist on technology, so I think that the DNA editing guys will eventually get there outside of certain areas where we think we can be differentiated. But the real benefit of the technology is going after post-translational modifications, making a change in an amino acid, and giving a biological pathway and activating it.

Nobody can do that necessarily in large complex diseases where you want to activate some pathways in a very specific manner. That is really our sandbox. There are other things that we can do, but that is our focus at the current moment. That brings us to our lead program, which is KRRO-121. For KRRO-121, we do exactly what I said. We make a single amino acid codon change on a protein called glutamine synthetase. Let us just call it GS. It is a protein that is present in the liver, and it is utilized to, for lack of a better word, detoxify ammonia. It converts ammonia into glutamine, and in patients that have a genetic mutation that fall under a category of diseases called urea cycle disorders, where they cannot clear ammonia, this is the pathway that takes over.

We are able to stabilize this protein momentarily, transiently, not permanently, and we are able to clear and blunt an ammonia response in these patients that go through pretty severe scenarios. I am sure we will touch on that in a second. But 121 is, we are now in the clinic. We have started a SAD/MAD study. We have finished dosing our first cohort. We just announced that a few weeks ago. We also guided to getting into patients in the first half of next year, the data in the second half of next year.

We have additional things in the pipeline I am sure we will talk about, but maybe stop there.

Yeah. I think the way that we think about targets in general is we need to have a high level of confidence that it's going to work in humans. The background that we look at is what sort of genetic evidence really exists for some of the targets that we go after. So glutamine synthetase is very unique. There are case studies where much like there were case studies for PCSK9 when they were first developed, there's both a loss of function and a gain of function at the specific site or the amino acid that we're changing. We know that if you have a gain of function and you were to hyperactivate the site, you end up with creating high ammonia in patients.

In cases where you were to stabilize the site and prevent this degradation, you end up with low ammonia and a very improved systemic performance. So between those two gain of function and loss of function, we knew that biologically this is going to work. So the only question that we're answering in the clinic is can we get enough drug there, and can the change that we make be sustainable? So from a preclinical standpoint, we've demonstrated making this change makes a lot of sense in stabilizing the protein. We have both OTC mouse subsets of urea cycle disorders when they're preclinical models we've looked at, if you were to challenge with an ammonium chloride, we can prevent and blunt the ammonia response, and we've shown it across multiple models. Then we've also demonstrated in monkeys that we can direct the cells to actually make the change.

That's the most unique thing about this product. Most drugs, when they're developed for urea cycle disorders, are very mutation specific. We have competitors that are looking at a subset of urea cycle disorders like OTC. Whereas this is the first time outside of nitrogen scavengers, which is a standard of care, that you have a drug that can go after across all mutations. So we call that pan-UCD, which is very unique. So we've been able to demonstrate now across multiple mutational models, both survival as well as blunting of the ammonia response in patients. Our hope is that in patients, because we can blunt this response, they can eat more protein. They don't necessarily need to worry about what we call as intercurrent events. So if you have an infection, the ammonia goes up. It's like hypoglycemia. When you have type 1 diabetes, you go to bed, you're catabolic, you forgot to eat dinner, and the next thing you know is that your ammonia levels shoot up, and you end up in the hospital.

A drug like ours can provide that peace of mind for these patients. I think the pre-clinical data that we've generated builds that case for it.