Episode 4 of First in Human features Justin To, Chief Operating Officer of Skeletal Dysplasias and Gene Therapy at BridgeBio. First In Human is a biotech podcast that interviews industry leaders and investors to learn about their journey to in-human clinical trials. Presented by Vial, a tech-enabled CRO. Episodes launch every other Tuesday.
Simon Burns: Justin, thank you so much for joining us on the First in Human podcast.
Justin To: Yeah. Thanks so much, Simon. Really looking forward to it and thanks for the invitation.
Simon Burns: Before we jump in, a quick background on me, co-founder and CEO of Vial. Set out to reimagine clinical trials and make them far more efficient. Justin, do you wanna give us a quick introduction?
Justin To: Yeah. Happy to. Justin To, currently the Chief Operating Officer at BridgeBio’s gene therapy unit and skeletal dysplasia unit. Again, pleasure to join you and join this podcast.
Simon Burns: Awesome. You’ve had a really interesting journey and, you’re doing really impressive work. Maybe tell us a little bit more about your journey from, Flatiron Healthcare Tech to the therapeutic side.
Justin To: Yeah. It’s actually a funny story. In about 2016, as I was still at McKinsey and Company, which is the consulting company. When I was looking for my next opportunity, I actually had a chance to meet Neil Kumar, who’s the founder and CEO of BridgeBio. I actually had a invitation to essentially join BridgeBio as its second or third employee. At that time though I thought it was a little bit too early for me and actually decided to take another offer to join Nat and Zach at Flatiron Health as a product manager, because I wanted something a little bit more established at that time.
Flatiron was a really fantastic experience, where I [inaudible 00:01:28] work and learn from so many talented people there. It really turned out to be a great training ground for so many now current leaders, and then even then leaders, in health tech. Between having Nat and Zach, who are huge visionaries in the field. Vinita and Jay, who are now at A16Z [inaudible 00:01:46], Amy Abernathy and Brad Hirsch at Verily. The list just goes on and on. And it’s just so crazy how, much of a training ground it was for health tech.
While I had a really great experience at Flatiron I realized in my entire time there that my heart was always in biotech and therapeutic development. Not just thinking about services and technology of how we could accelerate therapy development, but actually developing the drugs themselves.
So in the back of my head, I always had you know, what if I joined Bridge one day? And Neil was gracious enough such that after a year or so at Flatiron you know, we reconnected and he still had that slot open for me to join Bridge. So while I wasn’t the second employee at Bridge, more like the 10th I was still able to kind join very early in the journey.
Simon Burns: It’s crazy to hear you list it out there. I don’t know if I’d fully comprehended or thought about the scale of the Flatiron mafia. It’s really quite, impressive. I guess it’s just, getting started now. But-
Justin To: Oh yeah. I’m sure I’ve only listed off the tip of the iceberg. And I can list at least like 30 other people who are doing super well right now.
Simon Burns: Totally. Excited to see it. So tell us a little bit more about Bridge-BridgeBio. Gets a lot of talk about your kind of approach to reimagine how therapeutics development happens. But just give us maybe a sense of where it started and how you think that the thesis and the strategies change.
Justin To: BridgeBio’s a really interesting company and case study. It’s actually one of those really rare founder led biotechs where it wasn’t kind of started from a venture capital company. And really, the original vision came from our founder Neil Kumar, who, when he was at Third Rock, he noticed that there was a lot of funding and money going towards the establishment of what we call platform companies. So the next big CRISPR company, the next big gene therapy company, the next big immuno-oncology company.
But individually, there’s just so many great one off programs or assets that were essentially stranded at academic labs or small biotechs where we know from human genetics that they were gonna work or had a high probability of working. But they just weren’t getting the right funding. So BridgeBio ultimately served as a, way to accumulate and sweep up all these stranded assets and provide a portfolio of therapies for rare monogenic diseases.
Since then, our focus and scope and scale have changed over the years, but at the end of the day, BridgeBio’s mission is to simply discover, create, test, and deliver transformative medicines for patients who suffer from genetic diseases and cancers with clear genetic drivers. So it’s a bit of a long answer, but in short our vision is to be the leading genetic, medicines company.
Simon Burns: I want to go back to the, earlier comments you were making about the shift from, health tech, digital health into therapeutics. There’s a lot of people thinking about doing the same now. The founder-led bio movement is bringing a lot of people into, the space. What resources or, tips would you give them in terms of learning about biotech if they’re looking at making the jump?
Justin To: That’s a great, question. And I really admire the kind of surge of kind of founder-led biotechs right now. I think we can’t always just leave it up to like the venture capital companies to kind of start the next great biotech company or the next great health tech company, right? A few tips I would have there, the first is really get to meet and, listen. I think now that they’re having a number of folks who have successfully started founder-led biotechs get to know them. Get to meet them. Reach out, sure, you might not be able to get responses from all your cold calls, cold emails, but you just got to do it, right? And learn from their stories and their mistake and their battle scars. And that’s one that’s very important.
I [00:05:00] think two is clearly articulating the value proposition that the therapeutic approach or the kind of technology that you’re building. Help potential investors understand that look, part of the reason why we’re starting off this company, or starting this program, is because there’s a very strong scientific rationale there or very strong kind of business need for that approach.
Simon Burns: One of the aspects I think a lot of people take when they think about jumping into bios, they’re very technology-driven. They think a lot about the technology. But BridgeBio has not only done well, I think, in developing the technology, making smart science bets, also smart clinical strategy bets and, regulatory bets. Maybe give us a sense of your learnings there. Regulatory strategy, obviously you’ve done a lot of work in looking and getting a fast track designation. That’s no easy feat. What are the lessons learned there, and how would you advise biotech companies on thinking about regulatory strategy?
Justin To: Yeah. I thought of a few different questions within that, right? How do we kind of be smart around regulatory strategy? How do we become smart around clinical trial strategy? And what is fast track designation, and why does it help? So three separate things there that I’ll answer back to back.
The first is in terms of thinking about regulatory strategy I think every company has a little bit of a different approach to this, but you know, we at BridgeBio try to be relatively aggressive in thinking through regulatory strategy. Not just thinking through what has been done and what has been done successfully, but how can we push the boundaries of it, right? How can we create the right arguments with the FDA and other regulatory agencies. The second piece of just thinking through clinical trial strategy is you know, I think it’s really key, especially in rare diseases, to know the patient communities and get to work with them very closely. No one knows the disease better than patients and families. Especially in rare disease. And so we make a point early on in our program development, to meet with patient advocacy groups, to learn from them, to invite them to speak, and to solicit their input on how we design our clinical trial. The way that we design… So like endpoints, the way that we think about how often they need to visit. We want to get all their input on that, because these patients already facing enough burden as is from their disease. They don’t need additional burden from the clinical trial. And I think we try to incorporate as much of that as possible when designing a trial even very early on.
Simon Burns: Trial design is incredibly important. I’m curious what the lessons learned are in clinical trial design and clinical trial operation, not only from the rare disease component, but also in managing gene therapy that adds some additional complications too. But just in general, what have you learned running trials in the last few years?
Justin To: So you know, in addition to my advice a-around kind of getting the patient communities involved and soliciting their input and being thoughtful about that Especially for any type of genetic disease trial or rare disease trial is to go broader than, you would have expect originally. Don’t hesitate to explore multiple sites, multiple geographies, right? Because oftentimes, these patients may be s-scattered and… Or not all seen in one single point of care or single kind of key institute.
And while it may seem obvious, I think you know, investing and having a pretty broad but focused footprint will eventually pay the dividends in how you think about enrollment rates and how about… And time to completion of a trial. So you know, obviously be smart, be diligent about right sizing, how many sites you have based on your trial size, but I think investment early in a broad footprint is important, because there’s just so much heterogeneity between sites in terms of how slow one site can open up versus another. And the logistics of actually enrolling patients. So if… It helps you hedge your bets a bit.
Simon Burns: One thing I’ve heard recently is that it’s never too early to start talking to patient advocacy groups, especially rare disease. That critical advice and just getting input on how to best reach the communities is key in doing that. Earlier is better. Thoughts there? Do you a-agree with that, that general sentiment and advice?
Justin To: absolutely. I think that’s one that I feel like BridgeBio’s done a really good job of, where again, even in the earliest phases of development we started to meet with advocacy groups, educate them on our approach and why we think it, it serves a unique, unmet need and between having patient ad boards where we learn about what endpoints matter the most to them, to even having protocol advisory committees where they actually review our clinical trial protocols and think through how else we can optimize the patient experience there. That has been tremendously helpful in making sure that we can find patients who are interested in enrolling in our trial. And not only that, but that we’re measuring endpoints that patients actually care about. So I, think it has… It’s really been a key part of our success and… In rare disease drug development.
Simon Burns: Obviously at Vial we think a lot about the future of clinical trials, where technology can be applied to drive faster, more efficient trials. Having seen it up, close, I’m curious what you think the areas of opportunity are for technology, and what’s the long term here? What do we get in terms of five or 10 years out? What does the trial of the future look like?
Justin To: Yeah! I think that’s, a great question. From an operational perspective, I think there’s still significant room for improving clinical trial conduct. And that’s the most obvious place where technology at least for me, within drug development can be first applied. I kind of break it up into two or three different buckets.
The first is just purely site selection. Even right now, it’s kind of wild to me that oftentimes when we’re selecting sites or doing feasibility, it’s still very opaque just like how many patients that they see who like have a certain disease. How many patients would fit a certain criteria. What are their characteristics? There’s often between what we learn from a site when we first talk to them and what the reality is, there’s often such a gap. And I think technology there would [00:10:00] be so much more helpful in kind narrowing that gap. So site selection is a major one. The second is just purely site start up. Right now, there’s just way too much heterogeneity right now, manual processes involved in starting up a site. It’s kind of ridiculous to me that one site could take one month to start up, another site could take 12 months to start, up. We’ve seen that delta way too often, and it just… Part of it is because there are just so many antiquated processes right now that just are on pen and paper or, something like that that just becomes the [inaudible 00:13:00] limiting step for a site, right? Everything grinds to a halt on the slowest step, and I think that is a place where technology can really help sites start up a lot faster.
Simon Burns: I totally agree. Site startup is, one of the most kind of tractable, obvious problems where it’s input, output. Get the information, review it, look to approve, green light the site for enrollment, and yet the… Despite the fairly kind of linear path that you would think site startup should take, it doesn’t at all follow a linear path. Lots of track changes, Excels, lots of PDF redlines, lots of the type of things that product managers and engineers get excited to go solve. With that, Justin, thank you so much for jumping on and, chatting with us. Any closing words of wisdom for biotech founders, founder-led biotech enthusiasts looking to jump in?
Justin To: Yeah. So I mean, j-just technology. Right now, I think it is definitely a, tough time for biotech and health tech in general right now with the general macro environment, but you know, I really believe that if there is a strong mission, a strong therapeutic thesis, and a strong science background, the best companies will eventually manage to weather the storm and be able to survive this kind of tough environment. And on the other side of it there’s been a wave of great new therapeutics coming, so really looking forward to that. I’d just advise people to weather the storm as is, and I think it’s going to pay dividends.
Simon Burns: Justin, thanks so much for the time. Appreciate it.
Justin To: Thanks so much, Simon.