Behind the breakthroughs - menin inhibitors: A conversation with Troy Wilson, PhD, JD

In my Behind the Breakthroughs blog series, I’ve been talking with drug development experts and clinician scientists and patients  about what it takes to advance transformational therapies from the lab to patients who need them. The path between discovery and getting a drug approved can be fraught with challenges. Breakthroughs take years of research, persistent scientists, partnerships with academic institutions and biotech or pharmaceutical companies, plus steady investment every step of the way. 

That ongoing research, collaboration and funding is critical for innovation—especially for aggressive and difficult-to-treat blood cancers like acute myeloid leukemia (AML). Progress against blood cancers depends almost entirely on therapeutic innovation and access to treatment, and our new life-years saved analysis shows that there’s a great need for new AML therapies.

Each year, 20,000 Americans are diagnosed with AML, and many of those patients will die within a few years (and for older patients, sometimes within days or weeks) of diagnosis. We know from years of research that AML is not just one disease—there are more than 10 types and many rare mutations. 

"Breakthroughs take years of research, persistent scientists, partnerships with academic institutions and biotech or pharmaceutical companies, plus steady investment every step of the way."

E. Anders Kolb, MD, President and CEO, Blood Cancer United

Menin inhibitors for AML

Menin inhibitors are a new class of drugs that treat AML that carries certain gene mutations. Two menin inhibitors are approved for use in the United States, and more are on the way. Blood Cancer United has a long history of funding support in this area.

Blood Cancer United made its first grant for menin more than 20 years ago in 2005, when we funded scientists at Dana Farber Cancer Institute, Stanford University, University of Chicago, and Children’s Hospital of Philadelphia, who identified this protein as a key driver in the development of some forms of AML. 

Ongoing support over the next several years, through our research grants and Therapy Acceleration Program (TAP), helped researchers at the University of Michigan develop and refine drug compounds to target the menin protein inside cancer cells. 

In 2014, we helped those scientists hand off the technology to a biopharmaceutical company that could begin testing the drug in clinical trials. That drug, ziftomenib, was approved by the FDA in November 2025. This followed earlier approval of the menin inhibitor revumenib one year prior. 

Today I’m pleased to be talking with Troy Wilson, PhD, JD, president, CEO and co-founder of Kura Oncology, the biotech company that brought ziftomenib to market. Troy’s background spans organic chemistry, law, finance, large and small pharma, and entrepreneurship. He has learned drug development from every angle and dedicated his career to delivering transformative therapies to patients, focusing on some of cancer’s most challenging targets. 

Meet Troy Wilson

E. Anders Kolb, MD: Your career and background are certainly wide-ranging. How’d you get started in drug development? 

Troy Wilson, PhD, JD: My introduction to drug development was a bit accidental. I was trained initially as a bioorganic chemist at Berkeley, but I realized early on that I was not on a career path in academia, as many of my colleagues were. So, I went to law school, graduated from New York University, and much to everyone's surprise, did not go into patent law, which would have been the default. Instead, I went into corporate finance and securities. About a year into private practice, I was recruited to Novartis, where I joined their Genomics Research Institute in San Diego. It was an incredible opportunity to collaborate with biotech companies, academic institutions, and lead both business development and legal teams.

I did that for four years, and then started my first company, Ambrx, in 2000.
That began an odyssey of working with a lot of really talented people, building teams and companies to do innovative drug discovery and development. And now at Kura, I’m working to commercialize innovation, which combines both the business and the science side. 

Drug discovery vs. commercialization

EAK: I want to call out a distinction you made between drug development and drug commercialization, where you take a drug from development to FDA approval and the marketplace. I think that’s a fine distinction that a lot of people don’t think about. Can you expand on that?

TW: In biotech you don’t always get the opportunity to bring a drug to the market. In many cases, a drug winds up not working. You end up pivoting or sometimes folding the company and trying something new. Or you may have something promising that is acquired by another, larger company during development. Of all the companies I’ve co-founded or led, Kura’s ziftomenib is my first commercial launch. We quite deliberately set out to discover, develop, and commercialize that menin inhibitor. And hopefully it will lead to additional pipeline opportunities in the future. 

Why menin inhibitors? 

EAK: What makes menin inhibitors effective against a rare disease like AML? And what made you think it was the place to be for your first commercial venture? 

TW: One of the advantages I have from my background is that I have always stayed very close to the science. And the science of menin inhibitors shows they have a unique constellation of properties that make them ideal for treating acute leukemias. They have this mechanism in acute leukemia where, rather than killing cancer cells, they actually cause them to mature into becoming non-cancerous cells that live a few weeks and then die off. 

Kura’s menin inhibitor, ziftomenib, is a once daily oral medicine approved to treat adults with an advanced form of AML with a mutation in a gene called NPM1. Many of these patients are older and cannot tolerate other available treatments like intensive chemotherapy or stem cell transplants, so this gives them another option. Our ongoing studies are also showing that the drug works well for some patients in combination with other cancer-treating therapies. Think of acute leukemia as a smoldering fire. We can get it controlled, but you always worry there’s disease hidden somewhere that can flair back up. Ziftomenib is like putting a wet blanket on the leukemia. If those leukemia cells begin to proliferate again, they’re targeted by the drug. 

We haven’t seen that previously. That’s what motivates us to try to go all the way to the front line so that combination treatment becomes a standard of care. That's what we're working toward step by step. 

Challenges in developing drugs for rare diseases

EAK: The high cost and risk of drug development can make it difficult for promising therapies for smaller patient populations to move forward. What are some of the unique challenges in discovering, developing, and commercializing targeted therapies in a rare disease like AML? 

TW: As a drug development company, we always put patients first, and it underlies everything we do. But we also exist in an ecosystem where investors need to make a return on investment. Pharma companies typically focus on medicines they believe can generate billions of dollars in annual revenue. That can be challenging when you’re developing targeted therapies for a disease like AML that’s rare and may have a smaller market. 

It isn’t always easy to convince investors to give you the financial support you need to get to the goal line in a rare disease. It takes a lot of determination and smart people, along with a fair amount of good fortune.

Something we’ve heard consistently from investors from the very early days of the program was “Will this be a blockbuster for AML?”  We have to be able to show that not only are we doing good for patients and for society, but we’re going to make investors a compelling return. And I think you can do that in the case of menin inhibitors, as with some other targeted therapies.

I’m quite optimistic that we have the potential to transform the standard of care for AML by bringing these combination therapies to the frontline, while creating a compelling investment opportunity. And my hope is that this stimulates even more investment in other therapies for rare diseases. Because patients absolutely need it. 

EAK: It is possible to make a responsible investment that leads to huge impact for patients and the standard of care—especially in areas where treatment options are limited. 

Scientific progress takes a team approach

EAK: You talked about compiling a team of experts. How have you collaborated with researchers and academia and with Blood Cancer United? 

TW: We would not have zifotmenib without researchers Jolanta Grembecka, Ph.D. and Tomasz (Tomek) Cierpicki, Ph.D. at the University of Michigan and Blood Cancer United’s funding of their work to develop small molecule agents against menin in KMT2A-rearranged AML. They did absolutely pioneering research. 

Once they had something approximating a treatment that could work in patients, we at Kura brought our skills, expertise, financial resources, and ideas on how to develop and operationalize it, helping to ensure the drug could survive the journey from clinical development to market. And that was a very close partnership among all of us. 

I’ve always been guided by something Tom Daniel, the chief scientific officer at my first company, Ambrx, told me: “It’s our job to generate these drug candidates and then put them in the hands of really talented scientists and clinicians who will teach us how to use them.”  

We started with ziftomenib thinking it would work on KMT2A rearranged AML. Today, nearly 10 years later, I’m very optimistic that ziftomenib will work in probably 60 to 70% of genetically defined acute leukemias. We have some data coming out at the American Society of Hematology annual scientific meeting that will help us understand that. And now we’re also evaluating menin inhibitors in solid tumor cancers, as well as in diabetes. 

This progress would not be possible without  a lot of effort outside our company, with scientists and clinicians teaching us how to use these menin inhibitors. It’s an essential part of maximizing a drug.

And that process is needed for every successful drug. You start with a plan and an idea. And once you’re showing sufficient safety and tolerability, you must then be open to where else that idea can go. I find that whole process and collaboration magical. 

EAK: It’s such a testament to the fact that blood cancers are often the proving ground for treating other cancers, and why partnership and funding early and throughout a drug’s development and expansion is so important in getting innovative therapies to more patients. 

"This progress would not be possible without  a lot of effort outside our company, with scientists and clinicians teaching us how to use these menin inhibitors. It’s an essential part of maximizing a drug."

Troy Wilson, PhD, JD, President and CEO, Kura Oncology

Speeding pediatric therapies

EAK: Let’s talk about pediatric drug development. I’m a pediatric oncologist, and prior to joining Blood Cancer United my career was focused on research, drug development, and caring for kids with acute leukemias. 

Drug development in rare pediatric diseases like AML has always lagged. Therapies typically aren’t tested in kids till proven effective and safe in adults. As you mentioned, there’s little incentive for companies and investors when the market is small. But I believe it’s our moral imperative to get promising drugs to all kids who need them, no matter how rare the disease. 

Blood Cancer United’s Pediatric Acute Leukemia Master Clinical Trial (PedAL) was designed to speed this process. 

What was it about menin inhibitors that made you take a chance to evaluate them in kids early? To me, it's self-evident, but it's not the standard.

TW: When we say we put patients first, we mean it. And that's not always the easy decision. That's not always what investors want to hear. But when you prioritize patients, the decisions get a lot easier. 

As you know, Andy, in infants and children who have AML with KMT2A rearrangements, that disease is so aggressive. First, we needed to ensure that we understood ziftomenib well enough that physicians and care teams could use it safely in children.

Once we established that, then it was clear: we have an obligation to make it available. If you change the life of a child and that child's family, you've done something important for the world. I'm glad to see that not only the US regulators, but the European regulators are increasingly mandating that you have pediatric development as part of your drug development plan. I think that's the right thing to do. 

We are privileged to be able to develop new medicines for patients. And I think with that comes a responsibility that we see that all patients get access. You have to do it in the right way, and we're very grateful to be collaborating with PedAL and with Blood Cancer United. We're all working toward the common good.

Looking to the future

EAK: I'd love to know what excites you most, just not just about menin inhibitors, but
what they’ve taught us about diseases we once thought were untreatable. 

TW:  Well, it seems I’ve spent my career on drugging the "undruggable” conditions. At Kura we’re also working on developing farnesyl transferase inhibitors (FTIs). We’re now learning that they can work very well with other targeted therapies and could be transformative in kidney and pancreatic cancers. 

Biotech is this unique place where you can do something magical. I think with menin inhibitors and FTIs, we at Kura can drive a lot of benefit for patients over the next 10 to 20 years and beyond. 

• Read more about treating Acute Myeloid Leukemia (AML)   
• Learn about our Therapy Acceleration Program (TAP)
• Learn about our Pediatric Acute Leukemia Master Clinical Trial (PedAL)
• Read my other Behind the Breakthroughs blogs:
  • A conversation with Brian Druker MD and survivor Mel Mann
  • A conversation with Carol Gallagher, PharmD 

Blood Cancer United has relationships with many companies and research partners working in blood cancer, including Kura and other companies developing therapies in related areas. This conversation is for general education and discussion of research, innovation, and patients’ needs. Blood Cancer United does not endorse any specific company, product, treatment, or clinical trial, and treatment decisions should be made between patients and their care teams.