Arcturus Therapeutics Holdings Guggenheim 3rd Annual Healthcare Innovation Conference

Yeah, thanks, Evan. It's good to be with you.

Sure. Arcturus is an mRNA therapeutics company, and we have two exciting assets that have data catalysts in 2027. We've now guided a data presentation for the complete fourth cohort of phase II data for the second quarter in 2027 for our CF program. This is our inhaled messenger RNA therapeutic to replace the CFTR protein. That's going to be a meaningful data set and a lot of interest in that program. Then we have a rare liver disease called ornithine transcarbamylase deficiency. That's an intravenously dosed mRNA therapeutic that's gaining a lot of momentum. We have a successful product there that we've a successful candidate that we've reformulated. We call this ARCT-2601, and that program is starting its clinical trial, or at least we anticipate it to start early next year with some top-line data in the second half of 2027.

Two different programs, they are mRNA therapeutics utilizing next-generation technology from Arcturus that is compellingly differentiated from the field. A lot there, but a lot of potential value creation in the next 12 months.

Yeah. When we refer to the LUNAR platform, LUNAR is related to our lipid nanoparticle technology, and the chemistry that we utilize for this lipid nanoparticle technology is different from everybody else in very important ways. We have already established safety and tolerability and efficacy of this delivery technology for intramuscular injections. We have an approved product, by the way, a COVID vaccine product in 30-plus countries that uses this technology. But in the liver, we have also shown it is safe and tolerated and works well with our gen 1 product for OTC deficiency. He referred to LUNAR 2.0, though. We are really excited about this technology because we have shown that it is 38 times better in primates than LUNAR 1.0, which was already safe, tolerated, and working, and it is already incorporated into products globally. So we now have something that is 38 times better.

When you have a technology like this, a lipid nanoparticle technology that is so much more improved in primates, you have options of how do you apply this to your pipeline. Do you want to make things more potent? Do you want to make it more safe or more convenient? For the OTC asset, it has already been proven to be safe and tolerated and working, so we are going to focus on convenience. Rather than someone coming into the hospital every two weeks at 0.5 mgs per kg and sitting in a chair for three hours, we are pivoting to less frequent dosing, maybe once a month, for example, and a lower dose, and sitting in a chair with a nurse coming to your home and administering the drug and sitting in the chair for less than an hour. So we are applying it to more convenience.

It's a wonderful technology that we can utilize for multiple therapeutics now in the liver, and it opens the door to new therapeutics like PKU, and we've added that to our pipeline as well.

Yeah. Cystic fibrosis is a well-understood rare disease because of very successful companies in the space. Most of the CF community is treated with modulators, and these companies that have modulators are in the business of fixing or modulating a broken transporter, and that's not what we do. Arcturus is completely different. We're in the business of building a brand new transporter using an mRNA molecule that's inhaled, that goes inside these lung cells, these bronchial epithelial cells, to express and make a brand new CFTR. This is very relevant to the Class I population. These are nulls. They do not have a broken transporter. They have no transporter. This is an ideal therapeutic for these individuals.

They represent. The people that do not respond or benefit from modulators is approximately 15%, so it's still a significant portion that can be. About half of that 15% are Class I individuals. We've also seen a high prevalence of these Class I subjects in certain countries and certain regions. So there seems to be pockets where it's much higher than 15%. We've seen some regions reach 35%, 40% of the patient population. So, it's addressing a very clear unmet need for the most serious form of CF is Class I. If you do not have any CFTR, you can imagine that being the most serious unmet medical need.

Yeah, why don't you We have our CMO. Why don't we Sure walk through the data we have collected for CF?