“Our Journey Is Not Yet Complete”: A Q&A with EE’s First-Ever Chief Medical Officer, David Waltz, MD

For more than three decades, David Waltz, MD, has been working toward a singular goal: developing better treatments for people with cystic fibrosis (CF).

His career has taken him from the bedside at Boston Children’s Hospital to the forefront of CF drug development at Novartis and Vertex Pharmaceuticals, where he helped lead the development of the groundbreaking mutation-targeted therapies, called CFTR modulators, that transformed what was possible for 90% of people with CF.

But for Dave, the work isn’t finished.

Approximately 10% of the CF community does not benefit from those breakthrough modulator therapies. And now, as Emily’s Entourage’s first-ever Chief Medical Officer (CMO), Dave is bringing decades of clinical and drug-development experience—and the same urgency that helped move the CFTR modulators from the lab to FDA approval in just three years and seven months—to the final 10%

We sat down with Dave to talk about the people who shaped his career, what he learned developing some of the most transformative therapies in CF history in record time, and why he believes the pieces are in place to deliver a breakthrough for the final 10% of the CF community still waiting.

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Q: Welcome to Emily’s Entourage, Dave! What does it mean to you to join EE as its first-ever Chief Medical Officer?

A: Joining Emily’s Entourage as CMO feels like the continuation of a long journey of developing better treatments for people with CF. After gaining an introduction to clinical trials at an academic medical center and further learning how to design, execute, and interpret those trials from the industry side at Novartis and Vertex, I feel that what is missing from my career is developing an effective therapy for the final 10%.

EE is uniquely poised to help deliver such a therapy, and I feel honored to be able to continue my journey here.

Q: What first drew you to cystic fibrosis?

A: I first became familiar with CF as a medical student and took care of people with CF as a pediatric resident at Strong Memorial Hospital in Rochester, New York. However, my involvement with CF solidified during my time as a pediatric pulmonology fellow at Boston Children’s Hospital, which has a large and world-renowned CF center.

People with CF were my best teachers—both about the manifestations of CF and how a disease can impact a person’s life; even better than my attending physicians! I grew close with the people with CF for whom I cared and their families, and this inspired me to devote my career to finding better treatments for CF.

Q: After decades in the field, what continues to motivate you—especially when it comes to the final 10%?

A: My motivation comes from seeing how the CFTR modulator therapies have transformed CF, and knowing that the final 10% is unable to benefit from that transformation.

Leading the teams that developed the first dual-combination CFTR modulator through Phase 3 and approval, and then leading the team for the first triple-combination therapy for CF  from before it entered clinical trials all the way through approval, were some of the best experiences of my life.

Working with smart, talented colleagues who were willing to push boundaries with a sense of urgency for those waiting for those therapies would have been enough, but seeing the impact the modulators have had on the CF community was unbelievable.

Knowing that the final 10% are waiting for a similar transformational impact is what motivates me in my work with EE.

Q: You’ve spent your career on both sides of medicine: 18 years in academic medicine and 18 years in industry. What led you from one to the other—and ultimately to EE?

A: Although the people with CF whom I cared for seemed genuinely appreciative of my work with them, the transition from academic medicine to industry was driven by the feeling that I was doing little more than giving band-aids to a serious disease.

I felt we could do better, and the development of the modulators showed that we could.

Now we need to deliver an effective therapy for the final 10%, and that spurred my desire to join EE in this work.

 

Q: How did your experience advancing the CFTR modulators transform life for approximately 90% of the CF community change your sense of what’s possible?

A: When I first joined Vertex, the first CFTR modulator that addressed the root cause of CF had just been approved. While the modulator was transformative, it was effective in less than 10% of people with CF.

My first role at Vertex was leading the team that designed and carried out the Phase 3 trials for the first dual CFTR modulator therapy for individuals with two copies of the most common F508del genetic variant, which accounts for almost 50% of the CF population. Although this therapy was only modestly effective, it slowed the rate of decline in lung function and reduced pulmonary exacerbations—important and impactful outcomes for those with CF who benefited. It was the bridge to get to the even more effective triple combination CFTR modulators, which were approved only a few years later.

Working on the triple combination modulators that have now benefitted 90% of the CF community showed me how truly effective the right treatments could be in people with CF and inspires me to work to deliver similarly effective treatments for the final 10%.

Q: The breakthrough CFTR modulators moved from the lab to FDA approval in a record-breaking three years and seven months. How did your team move with that kind of speed?

A: While it usually takes 8–15 years to develop a drug, at Vertex we were able to take the CFTR modulators from the creation of a chemical in the lab to approval by the FDA in three years and seven months. That includes laboratory testing in animals and all three phases of clinical trial testing in humans—Phases 1, 2, and 3—and that speed is almost unheard of.

We did so by thinking both outside the box and well ahead of ourselves. We were planning the Phase 3 trials even before the animal testing required to start Phase 1 was done.

At EE, we are working with companies developing therapies for the final 10% to help them plan ahead in a similar manner.

Q: There’s often a tremendous gap between a promising discovery in the lab and a treatment reaching people who need it. What does it take to close that gap faster?

A: Sometimes when you start a journey, you never really know where you’re going to end up. And that’s okay, as it allows you to take your time to discover and explore along the way.

Moving a discovery from the lab toward a real treatment often follows such a meandering path, but the additional discovery and exploration takes extra time, and many among the final 10% who are waiting for new therapies don’t have that extra time.

Because at EE we know how important it is to the final 10% to move quickly, we’re taking a different approach to drug discovery. We bring a clear vision of what is needed along the journey of developing an effective treatment and work with industry partners to complete that journey as quickly as possible.

Q: What will you focus on as EE’s CMO, and how will you work alongside Chief Scientific Officer Chandra Ghose, PhD?

A: As CMO at EE, I will focus on helping prepare the CF community to quickly execute the clinical trials needed to bring an effective therapy to the final 10%.

I will work closely with Chandra as she identifies the most promising therapeutic approaches, and I will help identify fast and efficient ways to get those unique therapies to the final 10%.

Q: You helped bring a breakthrough to life for much of the CF community, but not 100% of it. What drives you to make sure the story doesn’t end there?

A: When I left Vertex, I felt I had helped accomplish a lot, and I was gratified at seeing the impact that highly effective CFTR modulators have had on most people with CF.

My career has been devoted to helping people with CF, and the lack of an effective therapy for the final 10% means our work—and our journey—is not yet completed.

Completing this journey is what motivates me at EE, and I hope that my experience will help us complete that journey as quickly as possible.

Q: Finally, what excites you most about this moment—and what should the Entourage know about what comes next?

A: There has been an explosion in knowledge about genetic therapies in the last five years, and approval bodies like the FDA are more and more open to working in a collaborative way, with a sense of urgency, to deliver these highly promising therapies to people in need.

The CF community is uniquely positioned to get genetic therapies to the final 10% quickly, and EE is even more uniquely positioned to help identify both the most promising therapies and the people with CF who can participate in clinical trials of those therapies.

EE will work with the CF community and the FDA to lay the foundation for efficiently testing promising therapies and getting them to the final 10% as quickly as possible.