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Christy Shafer stepped into the role of chief commercial officer at Marinus Pharmaceuticals at a critical time in the company’s evolution — it was prepping for the approval and eventual launch of Ztalmy for patients with CDKL5 deficiency disorder, a rare form of genetic epilepsy that predominantly affects children.

“It’s a little-known fact that 90% of drug candidates fail and they’re never brought to market,” Shafer said. “It’s a daunting number when you’re in the clinical stage of identifying where your drug is going to be placed in the marketplace. So that was step No. 1 for us to beat the odds to bring this drug to market.”

Ztalmy represents the first and only approved treatment for a disorder that affects about one in 40,000 live births, which classifies it as an ultra-rare disease.

“Not only do these children beat the odds, if you will, but bringing a drug to market for a disease that has never ever been treated before is beating the odds again,” Shafer added.

The industry veteran, who had spent time at Alexion Pharmaceuticals and Pacira Pharmaceuticals before joining the rare disease-focused Marinus in November 2020, now has her eyes on the road ahead and launching Ztalmy globally with partners in Europe, EMEA, China and Russia.

“We’re all grown up,” she said. “Now, it’s about expanding awareness ... and access.”

Shafer, who spent more than 20 years in the operating room with “biologics, pharmaceuticals, and devices,” was open to a new avenue when the opportunity presented itself to move into the rare disease space.

“I thought day in and day out that we were making a difference,” she said. “It felt wonderful to be able to change the progression of medicine with a new device or a new biologic and treat patients differently. But rare disease brought me that and then so much more. I was able to launch drugs in the rare neurology space that affected me personally more than I could have ever imagined. When I was asked to come to Marinus and build out the commercial team, I will say I was daunted at first, but I thought what an amazing opportunity. What other company could I marry not only my skills in the hospital setting with a potential launch of our IV product with my rare disease passion. It really felt like serendipity. I couldn’t have imagined bringing together those two bridges that have been so good to me over the years.”

Here, Shafer shares how her experience as a competitive swimmer guides her leadership philosophy, how she built a team and commercial blueprint to launch Ztalmy, and why being focused on patients is key to success.

Welcome to WoW, the Woman of the Week podcast by PharmaVoice powered by Industry Dive.

In this episode, Taren Grom, editor-in-chief emeritus at PharmaVoice, meets with Christy Shafer, chief commercial officer, Marinus Pharmaceuticals.

Taren: Christy, welcome to the WoW podcast program.

Christy: Thank you, Taren. It’s a pleasure to be here with you today.

Taren: Let’s dig right in and talk about how you and Marinus beat the odds, as they say, to bring the first ever treatment approved for patients with CDKL5 deficiency disorder. And it’s a rare form of genetic epilepsy that really predominantly affects children. How did you all bring this to market?

Christy: Taren, I think you really hit the nail on the head with the ‘beat the odds’ statement there, which for any drug candidate right now is super difficult. It’s a little known fact that 90% of drug candidates fail and they’re never even brought to market. It’s a daunting number when you’re in the clinical stage of identifying where your drug is going to be placed in the marketplace. So that was step number one for us is to really beat the odds to bring this to market.

But I think that from my position as running the commercial team, we’ve really beat the odds continually throughout the last year, and we’ve just been super proud of it. This is the first and only approved indication for children and their families living with CDD or CDKL5 deficiency disorder. You did mention that it’s in a rare pediatric population, I’ll give you a couple statistics here. CDKL5 is affecting in about 1 in 40,000 live births. So this is a very ultra-rare disease, not only do these children kind of beat the odds, if you will, we’ve tried to follow their path and really bringing a drug to market that has just never ever been treated before is in and of itself beating the odds. So we’ve been very, very pleased not only to be able to present our data to the FDA in a manner that was palatable, we were able to work with them to really address what was needed for these children get an approval and then effectively bring it to market with our very, very small team.

Taren: So can you talk to me through some of the steps, the practical steps, that you took to do this? Because let’s acknowledge the fact that you are a virtual company, so that has a certain number of complexities to it. So can you walk me through the process a little bit?

Christy: Sure. So I joined the organization in November of 2020, and at that point in time, we did not have an approval yet. And I will say it was daunting to start what exactly what was needed for these kids was a giant question mark. So we’re anticipating a positive opinion from the FDA, and there’s a lot of pre-work that needs to be done where you go at risk to do this. And the number one thing that I really rested on was who was right to build this team; and for me, the people were the most important thing. The people who were going to be able to effectively build a campaign for these families was number one.

And I have to say, Taren, I probably went a little bit outside of the box and thought about what was going to be right. I would interview folks and I would have people tell me, ‘I’ve done this so many times, I’ve been doing this for decades. I know the exact way to get this right’; and I have to say, intuitively, I knew that that wasn’t right. We’re the first and only indicated product here so nobody’s done this before. And I didn’t want to do the same thing over and over again that other brands had done or other commercialization efforts. So what I really focused on people who were curious and who knew that the patient was the number one most important thing here and they were willing to figure out what was right versus tell me upfront what was right. And so I built a very passionate, very compassionate team that was willing to figure it out versus you fill in the blanks with what they already knew.

Taren: Perfect. So what were some of those initiatives that were let’s say again using the bunny quotes “outside the box”? What were some of the different things you did do?

Christy: I think a lot of companies will say ‘we’re going to work with the advocacy groups early and often to understand what’s important to these patients.’ Now, for us, we’d already been doing that for a very, very long time on the clinical side of our business. But commercially, what was important for a clinical trial is going to be different than what would be important commercially for these patients. So what we did is, again, really work early and often, but we didn’t want to build our plan in the opposite direction. And what I currently am saying is we didn’t want to think about how they could fit into our box, but we wanted to fit into theirs. And I often find that we’re trying to encourage the advocacy groups that our way of thinking is right and we did it oppositely. We said, “What’s the most important thing to you?” And so we really dictated our plan on that information and how we were taught by them to say what is the most important thing. Now seizures associated with CDKL5 is what we are indicated for. However, these children have a lot of other comorbidities associated with that and we want to forget that. So the highlight of what our drug has been able to do is really where we’re focused without forgetting the whole child.

Taren: Interesting. So what were some of the top things that these parents asked you to think about and to focus on? Can you share that?

Christy: Sure. Again, this is 1 in 40,000, so it’s very rare. I will say I do not have this experience, but really leading with the remembrance of the fact of what these families go through. So these children tend to be profoundly affected and they wanted us to remember that they do oftentimes feel alone, if you would. Again, they’ve never had a drug that was specifically indicated for the treatment of seizures associated with CDD. But what they really wanted to, again, remember was is that they’re rare but they’re still people; they still have feelings; they still want to have wonderful good days; they still want to live their lives; and that they are not only dictated by this disease. So, again, keeping the child central to everything that we’re doing. And I’ll say here, Taren, CKL5 deficiency disorder has been around for years and years and years. It’s only been identified in the last decade which there are several adults on our drug now that have been able to gain access. So, again, it’s really thinking about the person and not the disease.

Taren: Thank you for that. And when we talk about pediatric clinical studies and pediatric approvals, it’s a whole other level of complexity because we’re dealing with such young patients. So that had to factor in quite a bit in terms of the process as well, I would imagine.

Christy: Yeah. So one of the most impressive regulatory teams I have ever had the great pleasure of working with, our clinical development team again listened very distinctly. We knew that we were going to need a large patient population. And what’s fun in the ultra-rare disease is a large patient population still is oftentimes under a hundred patients which is what we were looking at. That’s very, very large, and we’re super excited to say that that’s the largest clinical trial that’s ever been done in this patient population because of its rare nature.

But to your point, working with pediatrics is very difficult in clinical trials because not only do you need to identify children affected with the disorder, but imagine as a parent or a caregiver really saying, “Okay, this is the right thing to do for my child.” It’s not the same as when an adult proactively engages in a clinical trial; they can make a decision. But when you’re thinking of engaging your child in something like this, there’s a lot of emotion behind that. Again, they’re on a lot of different medications at the same time; and there’s a lot that needs to go into making that decision along with your healthcare provider.

Taren: Absolutely. Thank you for that as well. So tell me, what was the first thing you did when you heard the news that the approval was done, that you received the approval?

Christy: That’s a great question, and I don’t know if this is going to sound very sophisticated. So after we were jumping up and down and being so excited, we had made so many plans for that day. The PDUFA had big red Sharpie circles on our calendars, and it came earlier in the day than we had originally thought so it caught us off-guard just a tad. But what we wanted to do mostly, Taren, was celebrate with the people that mattered the most to us that had really helped build this program and that was the advocacy groups. The largest advocacy group in the United States is the Center for CDKL5 Research and it was led and developed by caregivers who have children that are affected with CDD and they are the ones that we knew were going to have the most emotions surrounding this outside of the research and outside of the decades long research that went into this. They were the ones that now are going to be seen. They’ve never had a drug ever that was going to be associated with this disorder and I think that it was a huge, huge milestone for them. And those were the first people that we wanted to call and tell and really give our congratulations to because it was their efforts really that shined the light on this disorder.

Taren: That’s nice. What a great way to celebrate such a big win so congratulations again. Now that part’s done, now the hard work comes in, right? Not that it hasn’t been hard up to this point, but what are your goals now for 2023?

Christy: That’s great. We’re kind of all grown-up now, if you will, Taren. So in 2022, it was all about just establishing us as a product, really introducing Marinus. Nobody had known in the community who Marinus was from a healthcare provider standpoint. We did a great job, by the way. We’ve established our product just central to the management of CDD, but now it’s about expanding that awareness. I will say that along with the advocacy groups, we’re super proud that they are now growing their base of centers of excellence so we’re doing the same thing. We also want to expand our base of physicians who not only may not be aware that they are treating or caring for a CDD patient, but also are they aware that there is an approved product; are they aware that there is an ICD-10 code. So it’s all about that expansion now.

We’ve been very, very fortunate to have really provided an access strategy for patients that has been seamless; we’re super proud of that. But now we need to be able to control that and ensure that we optimize our strength and our tools for our patients and caregivers if there are any hurdles that they need to overcome. And I will say last year we didn’t have a global footprint, but we’re very, very proud that our core values are treating all patients that are appropriate for Ztalmy. So we’re very proud to say that not only are we launching here in the United States right now actively, but we did just get a positive opinion from the CHMP for a European approval. We do have a European partner in Orion, and now we have partners in China, in EMEA, in Russia. And so, again, this is our ongoing effort to ensure that we are gaining access for patients on a global scale.

Taren: That’s very exciting. And as you said, now you’re all grown up so now you have to manage these partnerships and on a global basis, nonetheless; that requires a whole different set of skills. So are you looking to build out the teams some more?

Christy: If we go back to other focuses, so we’re always looking to expand our teams – yes, we need to be able to effectively support these launches globally. I will say though that in the partners that we have chosen, they are very well established organizations that have partnered really beautifully for us to make sure that we have a global look and feel to Ztalmy. So we’re fortunate that we don’t need to build out a full global infrastructure here at Marinus, but I will also mention that we do have two other ongoing phase 3 trials; one which would be an expanded indication in TSC and another in a different formulation; it’s an IV formulation in something called refractory status epilepticus.

So, as we’re looking to expand our team here in the United States, those three initiatives, not only globally so that we can support those launches, but now looking at the potential for us to expand not only our current label but an additional label on the IV franchise. So our teams are going to hopefully in the near term double and triple again.

Taren: That’s amazing. So it’s very exciting and you have a lot of energy around all of this. How do you keep yourself buoyed up and how do you manage all of those like different parts of the job and making sure everybody’s working and pulling in the same direction? How do you manage all of that?

Christy: Well, first of all, I really love that you just said ‘buoyed up’, that is my favorite of the day. And I say that because I’ve used the water very, very frequently as kind of a driving force. I grew up as a competitive swimmer and I grew up in a swimming family. And I’ve used the analogy of the black line at the bottom of the pool as being a grounding force for a swimmer. And really just knowing that it was there, really staring at it on a regular basis, and just knowing that it was going to lead you to the other end of the pool as another analogy of what we’re doing here. So for my teams really kind of identifying what that grounding force is has been the cornerstone to our success. So our black line is how do we effectively communicate the benefits of our drug to not only healthcare providers, but the caregivers and the patients around the globe.

And as long as that stays very central to everything that we do, we tend to make the right decisions. Now, I will also say that going back to my initial of hiring a passionate and compassionate team has helped me be able to do that. They are the ones who have really built this infrastructure that has resonated with those that matter in the community. And, again, if they’re buoyed to anything, they’re buoyed to these families. They’re buoyed to what matters to them to help them make the right decisions. So I try to give them as much autonomy. We lead with an enormous amount of trust, and I honor their intuition and their instincts on a regular basis so that we’re making the right decisions, and it’s led us down a really beautiful path.

Taren: That’s fantastic. And I love that swimming analogy. Does it tie into the name of the company as well, Marinus? I mean, because that has a little maritime kind of vibe to it.

Christy: Well, Taren, I’ll say to you it’s kind of a happy accident, but we love our Marinus name. And because we are a virtual company, I’ll share with you that we tend to like to go places where water is involved. And so when we come together as a team whether or not we’re at our home office or we’re somewhere, we’ve always been on boats together to have a sunset sale or we use a lot of verbiage around the water. But, again, I do think that that’s a happy accident considering that Marinus, the company, is a couple days old already.

Taren: Gotcha. That’s great. I’d love how it all kind of ties together there. So tell me a little bit about your background, if you don’t mind. What led you to Marinus?

Christy: We could probably talk about this for some time but I’ve spent over 20 years now and I had to do the math for you, Taren, which aged me a bit. So I’ve been 23 years in the healthcare industry, and that was another small kind of happy accident for me. I was a student. I graduated from the University of Maryland with a degree in cell biology and genetics; and as most kids, I did not know what I wanted to do and nothing to me was like ‘this is it.’ And it wasn’t until I was presented with an opportunity in healthcare that I even knew that it was an option. And I started my career selling capital equipment in the operating room. I remember the very first day, Taren, that I walked into an operating room having no idea, no clue what it is that I was about to embark upon. I stood in that OR watching a procedure for hours and hours and I was like, ‘this is the greatest place, and this is one of the cooler things that anybody could ever do, and how did I get so lucky?’

So, I spent the better part of almost 20 years in the operating room with biologics, with pharmaceuticals, with devices and I thought that it was the bee’s knees, if you will, until I went into rare disease. I made the change kind of on a hope and a prayer that it was going to be a great opportunity to stretch me in a different direction, but it offered something way, way greater. I’ve always been very confident that everything I was doing was with the patient in mind. I thought day in and day out that we were making a difference. It felt wonderful to be able to change the progression of medicine with a new device or a new biologic and treat patients differently. But rare disease brought me that and then so much more. I was able to launch drugs in the rare neurology space that affected me personally more than I could have ever imagined. And not to discount the years and years that I worked in the operating room, it was fascinating. I’ve seen open heart procedures, I’ve seen babies being born, but there’s something about the ultra-rare disease community that has really fed me personally that I can’t imagine doing anything else.

So, when I was asked to come to Marinus and build out the commercial team, I will say I was daunted at first but I thought what an amazing opportunity. What other company could I marry not only my skills in the hospital setting with a potential launch with our IV product with my rare disease passion. And it really felt a little bit like serendipity, Taren. I couldn’t have imagined building those two bridges that have been so good to me over the years that I could now kind of bring together.

Taren: I love that. Thank you for sharing that story with us. When I speak to folks in that rare disease space, it’s a visceral passion. Nobody gets into it and says, “Eh, you know, it’s okay.” 

Christy: You’re a hundred percent right. I couldn’t imagine anybody saying that. And they’re in the wrong spot, obviously, if that was their feeling.

Taren: Absolutely. You said you have a personal connection; do you have somebody in your sphere that has a rare disease? Do you mind my…

Christy: That’s a great question and I don’t. I really think that there’s just the connection that I have to the patient here, right? I don’t meet these patients or very rarely. We don’t have that as an opportunity. But there is an undeniable connection to rare disease patients when you’re working on these projects that is so palatable and so emotionally connecting for me that I love. I cannot say anything else other than I have a very, very strong love for it and I think it’s because of that connection. I love people, I love working with people. I have been given the opportunity to lead many, many, many different kinds of teams and that connection to people has always been very important to me. And now knowing that we have in effect changed patients’ lives for the better is something that is very, very rewarding.

Taren: That’s wonderful. Thank you so much again. You’re sitting in a C-suite spot, you’re influencing the careers of others, you’re influencing the lives of so many. Do you consider yourself to be a role model?

Christy: I think that’s a difficult question for me to answer, but I’m going to try. Self-identifying in that way is difficult. I would rather be seen as a role model from the vantage point of others rather than me defining myself in that way. However, I have been told that I am, and for me what that means is that I need to be a leader who listens and I need to be able to lead with my authentic self so that that gives the opportunity for folks to feel like they can also lead with their own authentic selves. But I will also say the most important thing for me is to lead with humility. I 100% know that I don’t have all the answers and I’m okay with it and I don’t need anyone to think that I do have all of the answers. It goes similarly to the team that I built. None of them too believe that they have all of the answers, but as long as we continue to empower each other, that’s when we can find the answers.

Taren: Very good. I love that humility piece. So is that one of your leadership strengths and what are some of the others?

Christy: So I will say, as I just mentioned, leading with that humility is number one. But I always have wanted to listen and learn from others, and I think those are probably my largest leadership strengths. I think my team would say that again not only leading with that humility, but one of the most important things that they have felt here or been driven by is the empowerment that they feel. I trust in their abilities and I have routinely underscored that their intuition is one of the most important powers that they should and can lean into. And I think that that empowerment to say ‘I know what we should be doing next’ because of any given information that we have, and then being given the opportunity to lean into that is really beautiful. And that’s the feedback that I’ve been given is that that empowerment is almost everything to them.

Taren: Fantastic. What a great lesson. What are some of the biggest leadership lessons you’ve learned along the way that you can share with others who might want to consider taking a commercial path or another path for that matter? What are some of the things that have helped you along your journey?

Christy: You know, Taren, everything; and I mean everything is about people and change. I was having a great conversation with one of my leaders this week where we were identifying a significant amount of change and that we were okay with it. The only constant in our world is change and how we adapt to it is really how we should measure ourselves. I heard somebody say the other day “This too shall pass,” and this too shall pass no matter if you’re in a spot that you’re perceiving as good or bad, right? And so knowing that this too shall pass at any given point in time and being okay with what comes down the pike, as long as you’ve made the appropriate transition plans or whatever it is, is just to be okay but know that people and change will be your constant. And it’s okay to be challenged.

Taren: Absolutely. You should be challenged, otherwise you’ve already hit your level at that job, so it’s good to want to be challenged, right?

Christy: Yeah. I mean it’s the only thing that helps us grow. And growth can hurt sometimes, but in the end we’re always a little taller, right?

Taren: I love that.

Christy: Maybe we have a different vantage point at that different height.

Taren: That’s a great sound bite. Yes, growing makes us taller and gives us a different vantage point; that’s fantastic. With your people focus, I am going to assume that you also are involved or interested in mentoring. Is that a correct assumption?

Christy: It is a very correct assumption, but I will say nothing is more rewarding than when somebody asks you to either provide mentorship on a team or for their personal growth. That is one of the can I say compliment that you could ever, ever receive. Now, remember, Marinus is a tiny little company. We kind of feel large at the moment with we’re just over 150 employees, but comparatively to large pharma we’re a baby pharma. And that doesn’t always provide all of, let’s say, the bells and whistles of a large company, but what I have been so impressed by is our human resources department who says it’s okay that we’re small, but that doesn’t mean that we don’t develop our people. That doesn’t mean that we don’t take maybe what’s perceived as a luxury of development and growth and any number of things for granted, and I think that we shouldn’t see them as luxuries. And so we do offer mentors, we do offer coaching opportunities, and I have been so proud that we still know that we need to continue to grow, and it’s not just all about maybe the numbers on the screen but it is about the people too.

Taren: Fantastic. And it’s nice to hear that people are still the focus even in a small company and how important that is. That’s a valuable lesson. Is there anybody who’s had a particular influence on your career?

Christy: It’s so hard to pick. I think I’ve been really lucky, and I don’t know if lucky is the right word, Taren, but it’s really the only one that comes to mind. I have been so very fortunate to have learned from folks that I would consider the best and not just the best at you drug development or the best at launching a new therapy, but the best at being really good people and good people leaders. And that’s where I want to reside is to say I feel confident in my continued growth as a people leader. So the first one that comes to mind is the person who made me a people leader, the person who identified my capabilities as an individual contributor to say your talents are wonderful here, but I think that they’re greater than that, and there is no greater compliment than to say, “I think that you have the capability to affect others positively.” And so that’s really the person that means the most to me because that was really what set my career off into people leadership.

Taren: Thank you for sharing that. You noted before that you were a competitive swimmer. What else is that most people don’t know about you?

Christy: Typical commercial leader, Taren, is one that is personable and maybe a little gregarious, loud – whatever the adjective is that you want to put there. But I will say that as I have aged I’ve now realized there’s a term for it but the introverted extrovert. And I’ve said this in groups before and people say, “No, that’s not you. No, you’re just an extrovert. You’re just an extrovert.” And so I’ve been doing a lot of research and soul searching to say, “What the heck am I really?” and I really do think I’m an introverted extrovert, meaning that I still really enjoy people, I get a lot of energy from them, I love to be around them, I love to hear about people’s lives and their stories and what makes them tick but I also really love to watch an episode of Ted Lasso at 9 o’clock at night by myself and recharge in a way so that it affords me the opportunities to do those other really lovely things. So I think, again, I don’t know if people agree with me but that is kind of how I recharge now and how I gained that energy so that I can still be that people person during the day.

Taren: Christy, that’s too funny. I’m glad they found a name for us because I too am in that same boat and I get a pushback too when I say I’m really an introvert, and they’re like, “No way” and I’m like, “Yes, I am” and they don’t believe me either. So that’s funny. This has been a great conversation and I thank you so much for sharing so much of your leadership lessons and your passion for the ultra-rare disease space. So I’m going to ask you our WoW question – what is that WoW moment that either changed the trajectory of your career or has left a lasting impression on you?

Christy: Undoubtedly, Taren, it has got to be taking the position as the commercial leader here at Marinus Pharmaceuticals. When I was asked to take on this extraordinary role, my first answer was “No way.” I did not believe that I had the capabilities to have the entire infrastructure on my shoulders, but for me having a leader who knew me, who had worked with me before to say, “I believe in you, I know that you can do this, and I want you to do this” was one of my greatest, greatest gifts. And almost three years later, I could imagine doing nothing else. I could not imagine not effectively supporting and helping these patients and it has been my greatest gift.

Taren: Well, that’s a mic drop moment right there. Christy, thank you so much for everything. Thank you for your time today, and I love speaking with you, and I want to wish you and Marinus continued great success in helping patients who are in desperate need of help. So thank you for all the work you’re doing.

Christy: Thank you, Taren. It’s been a really lovely morning.

Thanks for listening to this episode of WoW, the Woman of the Week podcast. For more WoW episodes, visit pharmavoice.com.