Kate Haviland knows how to push through clinical setbacks and turn breakthrough science into life-changing therapies. As former CEO of Blueprint Medicines, Kate’s leadership reflects the biggest lessons in biotech: holding conviction despite ambiguity, earning trust from teams, and building the resilience needed to turn bold science into real impact for patients.
Kate joined me to talk about navigating clinical setbacks, capital allocation under uncertainty, and balancing conviction with data. — Sajith Wickramasekara
* Editor’s note: The conversation has been edited for length and clarity.
Turning a phase 3 failure into a blockbuster drug
Sajith Wickramasekara: Let's start with Ayvakit. It’s a first-in-class therapy for systemic mastocytosis. Under your leadership, it's grown to half a billion dollars of revenue, with label expansions across multiple indications.
Like the journey of most medicines, I'm guessing it wasn't a linear path. What’s the history of the molecule and the setbacks you faced?
Kate Haviland: The founders of Blueprint Medicines were the folks who developed Gleevec, putting targeted therapies on the map. They had worked for years to engineer Gleevec into a very selective and potent inhibitor for that specific mutation — and they couldn’t do it.
So that was the founding premise of Blueprint Medicines: to develop a library of high-quality molecular chemicals across the kinome.
Ayvakit was designed to inhibit a specific mutation on a kinase, and had the potential to be impactful in both GI stromal tumors (GIST) and systemic mastocytosis (SM), which is a spectrum of disease that ranges from a very severe leukemia (where patients have about six months to live) to a disease where patients live near-normal lifespans, but have a huge disease burden.
We began developing Ayvakit across multiple indications, but put it first in GIST patients, where we saw responses right away.
Sajith: So was that it? The clinic, and then you got a blockbuster drug?
Kate: Not really — that phase 3 study failed. It was a pretty expensive failure.
We had tested the medicine in late-line patients with the same mutational profile, and saw nice overall response rates. But when we moved up the line, the tumors were more heterogeneic and the study failed.
But that didn’t so much change our view of Ayvakit, as it did the opportunity.
“We knew that the medicine works in these specific, small populations, but those opportunities wouldn’t create enough return for investors or enable us to continue investing in research.”
Sajith: So what did you do?
Kate: Luckily, Ayvakit was so potent, we saw activity at very low doses. That opened up an opportunity to go into the less severe portion of systemic mastocytosis. There's about 60,000 people with SM in the United States: about 2,500 have the more aggressive leukemic form, while the vast majority have indolent systemic mastocytosis.
We decided to take a chance and moved it forward at a much lower dose. In the advanced setting, our labeled dose is 200 mg per day; the labeled dose for the less severe patients is 25 mg per day.
We were rolling the dice that we could have an impact on a patient's disease at the dosage, while making it a tolerable medicine that someone could take chronically, for years.
And that’s what happened. It’s really the indolent form of systemic mastocytosis that’s enabled Ayvakit to do over $500 million.
Sajith: Was that a controversial decision at the time, to run it at a tenth of the dose?
Kate: Yes, particularly when you’re an oncology drug developer. In oncology, you go as high as you can, because the disease is so aggressive and you're trying to save people's lives.
This was a different value proposition. Our oncology team had to think about a very different type of disease, one more akin to allergic inflammation. It was very much a stretch for us.
Sajith: You built a team for one type of outcome, but you needed a team for a different outcome.
Kate: What’s amazing is that the team that ran the phase 3 study for GI stromal tumors also ran the phase 3 study in indolent systemic mastocytosis. That really reflects our philosophy at Blueprint: smart people can do many things, if you support them in doing it.
Sajith: How did you manage employee and investor confidence while executing a pivot like this?
Kate: As CEO, your job is to paint the picture of where the science can go, while also being grounded in what the science is telling you. Is there a signal that’s worth continuing to invest in? Or are you squinting too hard to see something that's really not there?
“It's a balance between what the data’s telling you and also understanding the limitations of any given dataset.”
If you have a great team, conviction, and take a data-driven approach, you’ll find your way through. As a leader, you need to show confidence that we’ll pivot with what the science is telling us. But you also have to explain why.
It’s the same with investors. Like why did we believe that Ayvakit was going to be a really important medicine for indolent systemic mastocytosis (ISM)?
We had to spend a lot of time talking about ISM, because it wasn't a well-characterized disease. No medicines have ever been approved for it before, and so we had to help people understand how important the medical need was.
With leukemia, where someone may die in six months, that’s obvious how severe it is. With something like indolent systemic mastocytosis, you have to bring that to life. How bad is it for people who live with this disease? We had to educate ourselves, our employees, and our investor community about why this was meaningful — why it was worth our time and resources.
Landing a multi-billion dollar acquisition
Sajith: How acquisitions actually happen is probably a total mystery to most people.
Can you just take us inside the Sanofi acquisition and the M&A process in general?
Kate: That old adage that great companies are bought, not sold, is true. You have to work on science that’s important. Have a differentiated view and continue to demonstrate the value that you're bringing to patients, because at the end of the day, that's what we all want to be doing.
It’s the CEO’s job to make sure the company has optionality — and that includes in business development. During my tenure at Blueprint, we brought in over $1.3 billion in business development transactions.
Part of that is talking to your colleagues in big pharma and always knowing what they're strategically interested in. Over the years, I was able to build relationships with the transaction leads, business unit leaders, and some of the CEOs in big pharma. It is very much a relationship-based event, and it always starts with business development.
BLU-808 was our next drug in development coming through the clinic. The idea was that if we could calm down mast cells, which are core to many diseases of type 2 inflammation, we could have a big impact. One of our investors called it a potential oral Dupixent because of this. So Sanofi was an obvious choice to have a conversation around BLU-808.
“We knew that if it truly did have the opportunity to be impactful across numerous disease states of type 2 inflammation, we’d need a partner to bring it forward and realize its full value.”
Conversations with Sanofi started around BLU-808 and then evolved to also include other things like Ayvakit.
Sajith: What was the turning point that convinced you that an acquisition was the right path forward for the company? Because it sounds like it started as a partnership conversation.
Kate: You want to be a company that has a really strong go-forward operating plan, and we were very confident both in Ayvakit’s trajectory and our ability to commercially execute through and beyond a blockbuster medicine. We were also excited about the potential of BLU-808 plus all the other things that we were doing at the company.
You have to ask yourself: Will an acquisition by a larger company get this medicine to more patients globally faster? Will BLU-808 go into more disease states faster than we can do it on our own? Can we get a value for it that’s a good return for our investors?
The answer is not always yes. With Sanofi, given the strategic focus and interest in the Blueprint team, that was important to me. Sanofi’s plan is to be the world leaders in immunology in 2030 — and they have a proprietary pipeline of programs coming through that’s really exciting.
Their flagship product, Dupixent, is an incredible medicine, and it’s in partnership with Regeneron. So all of their commercial and medical teams are shared between the two. They were looking for a high-performing team to build out their own infrastructure and people. That was different from other conversations I was having, where Sanofi was interested in more than just the assets.
Sajith: What was the most controversial or difficult part of the decision?
It was the decision that another company could get this medicine to patients faster, and could realize the potential of the science in a way that we just couldn’t. Our team was executing exceptionally well, but we made the decision that Sanofi could help us go bigger.
Why collaboration is not a zero-sum game
Sajith: You've been called Blueprint's deal mastermind, structuring collaborations with Roche, Ipsen, CStone, Zai Lab, and Lengo. What do you wish more people understood about successful biopharma collaborations?
Kate: First of all, everyone should be humble and think about what your company does exceptionally well — and then value the people across the table for what they do really well.
With every deal you do, issues will crop up, and some of that may be cultural. In biotech, we often parallel-process, while larger companies often do things more sequentially. You have to figure out how to negotiate those types of day-to-day plans around programs that you're collaborating with, or that you’ve licensed.
Some people approach these transactions as a winner-take-all, zero-sum game. But everyone has to feel like they've won some things, and given some things.
“If one party walks away feeling that they've gotten everything on their list, that's not going to be a successful beginning for a collaboration.”
Think about your priorities, how to find that middle ground, and importantly, understand what your collaborator needs strategically. And then structure the deal in such a way that they don’t lose that value, otherwise they'll want to walk away.
Why it’s not always about the deal size
Sajith: You've said before that financials are obviously important, but they should never be the driver of the deal. Can you just give an example of a deal where another factor was much more important?
Kate: Business development should be core to achieving your corporate strategy. In the early days of Blueprint, we had a program in hepatocellular carcinoma (HCC).
We had some great early data, but we needed to find a way to increase durability. In the meantime, we had all these other programs moving forward. So we decided to partner for our liver cancer program.
At that moment, from a macro perspective, China was really opening up to innovative medicine development. We also took advantage of the fact that HCC is highly prevalent in China. Numerous biotech companies were being created in China looking for innovative medicines.
We were one of the first biotech companies to do a deal in China. We were deprioritizing our HCC program for ourselves, but wanted to see it move forward.
We didn’t choose the partner that gave us the highest upfront, we actually chose a partner that could navigate this new system in China. It's such a different place to work, including developing medicines but also commercializing them, so we prioritized strategic fit over economics in that case.
A CEO’s biggest responsibilities
Sajith: Biotech leadership often requires you to make big capital and risk allocation decisions that blend technical, scientific, clinical, and regulatory judgment. How do you make those really big calls?
Kate: As CEO, one of the most important things you need to do is capital allocation, particularly as you have a growing organization. We had to think very hard as we moved from discovery to development, and then development to commercial stage. You have to constantly build the next set of expertise and infrastructure, but you still have to fund the mature parts of the organization.
It's definitely an art, not a science. And often, you find out if you got it right in the rearview mirror, years later.
The executive team has to be aligned on what the three most important value-drivers are. If everything is a priority, nothing's a priority. Make sure you’re fully funding those, but also find creative ways to continue working on other things that may be of value, but aren't the priority at the company. Like for instance, our HCC program that we deprioritized in our portfolio, but moved forward by out-licensing.
“You have to constantly evaluate priorities and decide how you’re going to shift or allocate capital differently. We certainly stopped as many programs as we brought forward.”
Building culture of empowerment and empathy
Sajith: Half of Blueprint's executive team are women, which is above industry norm. How do you build a company and culture that recruits and develops the best people, no matter their demographic background?
Kate: Building culture intentionally is one of the most important things that you need to do as a leader — and it is critical from the earliest stages.
We work in a very dynamic environment where there's ambiguity, no perfect data, facts that change with new datasets — and you have to be able to pivot. To do that well, you need a diversity of people and opinions.
“If you're going to make good decisions, you need that robust debate and diversity of perspectives around the table. There should still be one person whose job it is to make the decision, but it's incumbent upon them to listen and integrate all these viewpoints.”
There are very few decisions where the door shuts behind you. Most of the time, you move down the line, get more information — and if you need to pivot, you pivot. You just constantly have to keep moving forward.
From a culture perspective, I believe in guardrails, not checklists. Because if you give any great team of people a checklist, they'll go and dutifully fill out every box.
“Templates and checklists may be more efficient in the moment, but actually undercuts productivity, resilience, and the ability to manage ambiguity.”
But if you just give guardrails and ask for a plan, the team is going to own it and come back with something really thoughtful.
Sajith: You should keep decision-making closest to the programs and people who know the most about them as possible.
Kate: Leadership is not hierarchy. If people are executing because their boss told them to, you're never going to get the most out of your people; you won’t be able to leverage their talent, creativity, and expertise.
“You have to believe that being a leader is about setting this context for great people to do their best work, and setting parameters around which someone can really contribute to their fullest ability. Only then are you going to succeed in being more than the sum of your parts.”
Sajith: Your fingerprints are all over Blueprint, from commercial to international and manufacturing. How has that breadth of internal experience shaped your leadership style as CEO?
Kate: I managed every function for at least some amount of time.
You gain a lot more empathy and understanding. In biotech, we’re all working on the same program and pulling the same direction. But we’re doing things that are very different on a day-to-day basis from each other.
Working with all these teams, you learn a tremendous amount about all these different aspects of the business. And it goes directly to your ability to allocate capital and decide where to take a risk versus where not to.
Risky bets that ultimately paid off
Sajith: Looking back, what's the riskiest bet you've made in your career?
Kate: Professionally, probably one of the biggest risks I took was to redirect Blueprint Medicines, whose heritage was very much in oncology. We were formed and defined by that, myself included.
We were a pretty young company, to then really pivot around what the science was telling us. But we were all excited about the opportunity around mast cell biology. The knowledge we were gaining through our experience with Ayvakit, and then BLU-808, felt so powerful.
I don't believe that Sanofi bought us for Ayvakit. I think they bought us because we are some of the world leaders in understanding and modulating mast cell biology to impact disease. Moving Blueprint in that direction and stopping some of our EGFR lung cancer programs (including the only program we’d acquired), was a bold decision.
People thought we were crazy for not pursuing it. But we didn't think we were going to have the impact we needed for frontline patients. So we decided to allocate our capital more towards BLU-808 and move in the direction of modulating mast cell biology. That was a pretty big decision.
Advice for the next generation of scientists
Sajith: What advice do you have for the next generation of scientists, as some of them move on to the business side of biotech?
Kate: One of things I loved about Blueprint is that both our Chief Commercial Officer and our Chief Business Officer both had PhDs. They were scientists first.
That curiosity, a willingness to raise your hand and do new things, an ability to integrate information quickly, and to be curious about what your colleagues in different departments are doing — those are all the qualities that I look for in people.
I'm also really proud that at Blueprint, we had people completely change functions and move across the company in different roles.
Earlier, I described the team who ran the phase 3 study for GI stromal tumors that failed; they ended up executing the first-of-its-kind phase 3 study for indolent systemic mastocytosis, with an endpoint that they had to develop. They did a tremendous job and got us to the new class of medicines for these patients, blazing a new regulatory and clinical development path.
I truly believe that people can come up learning curves quickly if they have the right kind of drive, support, and confidence from their managers. Put people in the right setting, and your career can take off in so many directions.