Disc Medicine 3rd Annual Guggenheim Healthcare Innovation Conference

It's great to be here.

Sure, that's great. Yeah, Disc, we've been public for a little over three years, and we focus on molecules that modulate fundamental metabolites in red blood cells. Heme and iron have been the focus. We generally focus on clinically validated mechanisms, and at this point, we had a phenomenal phase III readout for our lead program to address rare porphyria. Then we have a pipeline that's also read out with great phase II proof-of-concept data, all presumably progressing towards phase III trials in the area of myeloproliferative neoplasms.

It's a big deal. It's a big deal. I mean, these patients, the primary experience they have every day is inability to tolerate sunlight. The average is around 30-40 minutes before they have excruciating pain attacks. It's fundamentally life-altering, this disease. Patients end up having to live entirely inside, choose their hobbies, careers, their family life entirely dictated by inability to be exposed to sunlight. So profound disease, and this will be the first therapy. It'll be only the second therapy available, but it's the first mechanism that gets to the underlying cause of the disease, which is a buildup of a toxic chemical. We're very proud of the way this trial enrolled incredibly rapidly and then succeeded in the statistics with what are fairly tricky endpoints.

What's really gratifying to see is the way the reduction in that fundamental toxic metabolite translated into profound clinical improvement across everything that we measured.

Yeah. I think if you are looking for a floor, you could look at the historical academic research, which pegs the number pretty consistently at about one in 100,000, which would lead to an assessment of about 3,000 to 4,000 patients. There is a company that has the only approved product. They estimated around 4,000 patients. That is kind of the world we came into, and then we investigated claims data, claims databases, and what we saw in there was about 14,000 patients with a diagnosis. We think it is pretty robust because there is a unique ICD-10 code for this disease, and these 14,000 patients all have had care under that code. We have done a fair amount of effort to scrape out what might be spurious coding events. We feel pretty good about that.

What we see in there is about 6,000 patients who appear to be more engaged in their healthcare around the disease by showing multiple different claim events, whereas there is about 8,000 who we view as disengaged, which is not at all surprising given the limited care that is available to these patients. I think you have those 8,000 patients who are getting diagnosed and then confronted with very few care options and simply retreating back to a life of avoiding sunlight. The way we look at the launch is we are launching into what we believe to be about 6,000 engaged patients who we think we will be able to access and reach with a sales force of about 24 reps. Then there will be a longer-term effort to activate and reach and make available what we think is an exciting new therapy for those 8,000 disengaged patients.

Yeah, I think there are a number of rare diseases where the patient number is similar and have shown, can be kind of used as a model system, like HAE, PNH. These have all turned into very substantial rare disease markets. There is a hepatic porphyria, which is a rarer disease, but the launch curve, again, not a bad way to look at it.