Solid Biosciences Inc. Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference
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Wow The clocks I've had like- Sorry, I don't want to- Oh, yeah.
Oh, I see it. Yeah. Hot mic. Hot mic. Hot mic. Hot mic. Let's stop talking about Kevin Tan.
We're okay to start? Okay.
Sure. All right. Well, thanks everybody.
Good afternoon. It's day one of the Cantor Fitzgerald Global Healthcare Conference, ending the day with one of my top picks, Solid Biosciences. Joining me on stage is Bo Cumbo, the President and CEO. Thanks so much for being here, Beau.
Yeah. Thank you, Kristen. Thank you, Cantor Fitzgerald.
Yeah. Of course. Maybe just to kick things off, do you mind providing us with a very high-level overview?
Solid Biosciences is a precision genetic medicine company, but we are primarily focused on gene therapies right now. Our two main products are with Duchenne muscular dystrophy and Friedreich's ataxia. We have other cardiac programs. They are going a little slower because we are putting all our money and all our emphasis on the two main programs, which is Duchenne and FA. We also have a very unique platform, where we build different delivery tools such as capsids and dual plasmids, promoters, and we leverage those capsids for our programs, but we also give them out to academic labs and building a pretty big profile there on delivery for gene therapy for next generation gene therapies.
Before we talk about some of your individual candidates, I think it is important to set the stage about the DMD landscape first. We have had a gene therapy on the market for three years now, a launch that has come with a few unfortunate setbacks. Big picture, what has this launch shown you in terms of patient interest and the ability for a gene therapy to succeed here?
We have been very fortunate. Obviously, we have dosed 53 patients in our INSPIRE trial. The trial is pretty much full. It has been full for a while. What the sort of trend was that we increased dosing in the trial pretty dramatically over the course of the last year and a half, and we were able to fill that trial very quickly. There was a lot of demand, and I cannot remember exactly how many patients were sort of hoping to get in the trial, but it was well over 100 when we basically shut it down. The demand was high. I think a lot of families want second generation gene therapies, especially ones with unique capsids that can provide better distribution. Also, maybe a little bit of a safer profile to date, and so we are very excited about it. The demand was high. I think there is a lot of demand for the drug.
There are a lot of people that want the drug. Unfortunately, the trial is relatively closed in the United States. We are going to dose about three to four more little boys at the very end of this year using the CMC commercial material.
That's the only children that we're going to dose going forward in the INSPIRE trial. The reason we're doing it that way is so we have some bridging data from our phase I/II to our phase III using the commercial material, and that'll be 3 to 4 patients worth of dosing that we'll do at the end of the year. That's a long-winded way of saying the demand was intense. We believe that it's going to be intense for our phase III trial once we get Europe up and running, as well as the sites in the U.S. And it shows you what it's going to look like if we can get this drug to the patients commercially. I think that there's going to be a very big uptake relatively rapidly.
Okay. For DMD gene therapy, we've seen situations where biomarker data nicely translates to clinical outcomes, and then situations where this hasn't happened. Do you think there are particular biomarkers at this stage that are most predictive of clinical outcomes we should be paying attention to?
Yeah. No, it's a great question. Look, let's take a step back. 3 years ago, we were a $38 million market cap company, and we were really having to do a lot of work, understand where we could put our money for our programs. So one of the things we did as a company is we didn't take a look at one biomarker or biological correlate. We looked at 10 to 12, and we feel that it was extremely important to us at the time. Now it's important for you as investors, and it's important to families and physicians to look at what we believe is the most comprehensive data set that's been put together in Duchenne, period. So we don't look at one biomarker or one biological correlate. We look at a host of them. I call it a waterfall.
Some of my other colleagues internally call it a bridge. But realistically, it starts when the needle goes in the arm. You look at the VGs per nucleus, then you go Western blot, mass spec, positive fibers, the sarcoglycan complex coming together. Then you take a look at nNOS production, as well as the next step, which is your biological, your muscle integrity, CK, ALT, AST. ALT, AST, you think it's a liver biomarker. In Duchenne, at baseline, they're somewhere between 300 and 500. It's the entire skeletal muscle breaking down in these kids. Then you take a step and after the acute phase, you look at the chronic phase, which is titin. You see titin that's broken down in urine and blood. That's when the muscle's completely degenerating. You look at troponin, the heart. Every one of these kids are having little spikes in troponin.
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