Arcturus Therapeutics Holdings Inc. Common Stock CG 46th Annual Growth Conference
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Thank you so much for joining. I am Whitney, one of the biotech analysts here at Canaccord, and it is my pleasure to be joined by Arcturus Therapeutics and President and CEO, Joseph Payne. Joe, thank you for being here. I am going to dive right in. We have a lot to cover.
Yeah not a lot of time.
Let us do it. Just starting with a high-level overview of the company for anybody who is not familiar, who is Arcturus?
Where are you today, and what are you trying to do?
Yeah. Arcturus is an mRNA medicines company. We are based in San Diego, and we are a next-generation mRNA company with technologies that are differentiated from the other players in the mRNA field. We are very well known for our delivery technology, especially for inhaled messenger RNA delivery to the lung, and also injectable mRNA to the liver. We have a vaccine enterprise. We have an approved COVID vaccine in 32 countries that we have regained strategic control over, which is a new development. We utilize our vaccine enterprise to fund the therapeutics franchise, and we have a flagship lung program for cystic fibrosis. This is an inhaled messenger RNA to treat CF, potentially. We have an injectable mRNA for the liver for a rare liver disease called ornithine transcarbamylase deficiency.
We have these two flagship assets that represent the platform, and for the lung and the liver, and that is where we are creating a lot of enterprise value. The fuel for this company comes in part from our vaccine enterprise, where we have an approved product in 32 countries.
Perfect. Starting with CF, which is where we spend the most time speaking with investors. I guess you mentioned a little bit, but what is ARCT-032? Can you talk a little bit about the lipid and what it is delivering and how you are getting it there?
Yeah. Since we are in Boston, there is a very successful company here in town that treats cystic fibrosis very well. Most of the CF drugs out there are modulating a broken or dysfunctional transporter in the lungs called CFTR. That is not what we do at Arcturus. We are not modulating a broken or dysfunctional transporter. We are building a new one. Arcturus' drug, ARCT-032, is a messenger RNA molecule that expresses or makes a brand-new CFTR protein in the lung. It is delivered with our technology. The registered trademark for our technology is LUNAR. Our lipid nanoparticle, LUNAR, delivers the messenger RNA to the bronchial epithelial cells after inhalation through a nebulizer. We not just access these cells, but get into these cells and release the mRNA to express or make the brand-new CFTR for these folks.
The initial target population for this drug is Class 1 CF because Class 1 is also described as nulls, meaning they do not have any CFTR in their lungs. There is nothing to modulate. So there are no drugs for these people presently. The standard of care is very limited, and so we are pursuing an area of very high unmet medical need with very limited competition, and that is where we are.
Excellent. Okay. You hit the nail on the head here. Sometimes some of the pushback we get on ARCT-032 is, "Well, Vertex, they have got CF handled." But as you alluded to, you are targeting the patients that Vertex does not- Correct.
Yeah. does not address. So that kind of has bleed through to the efficacy story here as well.
You have talked about having enough data from ARCT-032 by the end of the year to be able to figure out how to move forward.
Yeah. How are you thinking about the efficacy bar?
What are you focused on?
Yeah Why or why not is what we've seen from Vertex there or not?
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