BridgeBio Pharma, Inc. Common Stock Bernstein Insights: Healthcare Leaders and Disruptors – 3rd Annual Healthcare Forum

Great. Thank you so much for having us, Jeffrey, and thanks to the whole Bernstein team. Thomas Trimarchi, President and CFO of BridgeBio, joined by Julie Miller Everett, our Chief Operating Officer of our skeletal dysplasias business. Many of you will know Julie from her critical role in the Attruby launch, and we have just moved her over to help with the infigratinib launch, where she brings more than a decade of commercial experience, particularly in the ultra-rare and skeletal dysplasia space. So really privileged to have her with me here today. Maybe I will start just by saying a few words and setting some context for hopefully a great dialogue here with you today, Jeffrey.

BridgeBio is a company that at the highest level, we are here in this world to help as many patients living with genetic disease as we possibly can in the shortest amount of time that we can get that done. We do that through creating first or best-in-class medicines, and putting them out into the marketplace.

Today, the business late-stage portfolio is incredibly unique, I would say. If you have studied the industry, you are certainly looking at it today as I know you are. First, we have Attruby, which is our best-in-class, near complete stabilizer for ATTR cardiomyopathy. We are about six quarters into that launch. The importance of that launch to our business cannot be overstated. So at once, I think that has proven us as a capable commercial organization, and, I think that is widely regarded as one of the better biotech launches we have seen in recent years. Annualizing north of $1 billion today globally, on track to be on a full year basis, blockbuster status globally by the end of this year. Really, that is owed to a few things. First, this is a large and expanding marketplace. We are seeing something like 15,000, 16,000 patients come to the bottom of the funnel every year.

Really attractive from a patient needs standpoint. I think we have clearly a differentiated and best-in-class stabilizer, which are post some of the recent data sets, I would say solidified as the backbone of- first-line therapy.

Really exciting molecule there. Actually, this morning, we had some incredibly important news come out. We published the first of its kind real-world data set comparing Attruby directly to Tafamidis- That is great.

where we have seen about a third improvement in the composite of including all-cause mortality and CV hospitalization. Really important data set that has not really yet been disseminated- Yeah into the marketplace and impacted prescribing behaviors.

That will take place over the coming year. So really proves us out as a competent commercial organization and also I think gives us the confidence to launch Yeah the next three products, not only in the U.S. but globally.

So, I think the truly unique thing about BridgeBio today is that still early days of one product launch and another three coming in the next year. So we've got Limb-Girdle Muscular Dystrophy Type 2I/R9, which is first in class molecule for a high unmet need form of muscular dystrophy. There we've got a product that can actually remarkably improve function on several fronts. One, patients are able to breathe better than when they started therapy. They're able to walk better, and they're able to actually, through measure of general symptom scores like the North Star Ambulatory Assessment, restore overall function and in a statistically significant way against placebo. So really exciting molecule there that's going to do a lot of good for patients. This is a clear billion-dollar opportunity.

ADH1 in encaleret is another small molecule that's read out phase III in the last year or so. Again, basically functionally cured about 76% of the patients in our phase III. So, really important data set that's going to change a lot of lives in this marketplace. And there again, we've got a billion-dollar opportunity, no competition, looking forward to building that market. Finally, achondroplasia, which I hope we have plenty of time to speak about given we've got Julie with us. This is today a billion-dollar marketplace where we're going to come in with a clearly distinct, differentiated, and unique molecule.

First and only oral in the space and a pivotal data set that really is giving the market exactly what it's been asking for many years, which is really a profound effect on height, but more importantly, a compelling effect on the health of these children, whether it's proportionality, or some of the things we've been able to demonstrate recently, including an effect on sleep apnea and ear infection. So overall, big picture, acoramidis we see is on track to deliver $4 billion-plus in peak year sales, and then you've got another $4 billion, I would say, de-risked and about to see uptake into the marketplace next year, with expansion available Yeah to us beyond that.

There are some interesting opportunities, including hypochondroplasia and other skeletal dysplasias, as well as post-surgical hypoparathyroidism. That is the opportunity in the next 18 months, I would say.

Beyond that, our hope is that we can create many new meaningful medicines for patients beyond the four that I talked about and have a durable business that can grow for many decades. Beyond the four products that hopefully we will spend most of the time on today, we have had a very prolific, productive discovery engine that has Yeah continued to put molecules in the clinic.

I think when the time is right, we can access some of that pipeline growth. It is housed today in our sister company called GondolaBio, which is private. With that long-winded, perhaps, context setting, maybe we can get right into the Q&A.