Dr Thomas Rossi​ talks to RARE Revolution about what motivated him to join Venthera as CEO, and enter the world of rare disease. He discusses the importance of meeting the people they are serving; the patients whose lives they are working to improve, and how this inspires the team to dream big

CEO Series: meeting the beating hearts behind the RARE brands

1.

What made you want to move into the wide world of rare disease, and then specifically Venthera and what did that journey look like?

What attracted me most was a combination of beautiful science and a sense of moral purpose and mission.   

When I was contacted by BridgeBio in 2017, I had never heard of venous or lymphatic malformations; I didn’t know much about oncology drugs and the biological pathways they affect. But I did recognise a compelling discovery story in the work that gave rise to Venthera, and that excited me. The very first step in this journey was a technical challenge. As that progressed, I also had the opportunity to talk with the leading researchers in vascular anomalies and came away with a strong sense of validation of the unmet need in this disease community.

The real clincher, the thing that sunk the hook way deep for me, was when I had a chance to meet people who were living with the disease and were engaged in patient advocacy groups. I participated in the Klippel-Trenauny Support (KTS) bi-annual meeting and met a lot of folks who were willing to dive deep into their personal stories and explain how venous and lymphatic malformations affected their lives and families. They talked about their aspirations for their lives—if there only was a way to address these lesions. I went to that meeting thinking I was fighting against a disease but came away believing in my heart that I was fighting for people. That’s a big difference.

That’s the trifecta for me. A personal desire for deep purpose. Elegant and inspiring science. And amazing people who got into my heart.

2.

What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?

Many of our challenges stem from the fact that we are doing something novel, and there is no tried and true pathway for us to follow. I love that. We will face the normal challenges of finding the right people to participate in our clinical study. Beyond that, we are studying a difficult disease to characterise. The technical hurdles of doing precision medicine in this population are enormous. We will be borrowing techniques from oncology research, but we will be applying them to a unique disease which, though it shares some genetics with certain types of cancer, has a variety of phenotypes that make the use of the established techniques challenging. We must adapt and overcome.

Another type of challenge is one of imagination.  As we learn about who we can and cannot help with our initial product, we will constantly be asking ourselves: “what more can we do; how do we reach the people who may not respond to this therapy?” Our vision is to bring hope to every person living with venous and lymphatic malformations, and that will require a constant drive of innovative thinking.

3.

What is your proudest moment in your career thus far?

That’s a tough question for an industry veteran because I’ve had so many moments (not all of them proud!). I am proud of every drug I’ve touched that ultimately made it to the market and brought relief to people who needed it. When I worked in a senior R&D executive position at Johnson & Johnson, we had many successes, but the two that stand out for me are Topamax® for epilepsy (because it was an in-house discovery, and it was great to see it go from idea to product) and Regranex® for diabetic foot ulcers because of the devastating impact that ulcers have and the high number of amputations that arise from them. In the smaller companies I’ve led, I’m proud that two products have made it from inception to marketing approval—Mirvaso® for the treatment of erythematic rosacea and Twirla® a 7-day contraceptive with low hormone doses. 
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In those smaller companies, and in Venthera, too, it is a very proud moment when inventors and investors place their trust in me to take their idea and turn it into a viable pharmaceutical product. If I had to sum it up, that’s what I’m most proud of—the fact that people place their trust in me to take their most treasured ideas and run with them.

4.

What and who are your personal and professional inspirations and why?

Wow. I work with so many great and inspiring people. 

I want to give a shout-out to my graduate research advisor, Dr. Isiah Warner. He is an inspirational leader on so many levels. When my wife and I moved down to Texas to study in his research group, we were newlyweds (we just celebrated our 40th anniversary!). He created a welcoming environment for us two Yankees in our first experience down south and literally welcomed us into his family. He taught me by example about the importance of personal bonding in building a strong research team. He taught me about professional work ethic beyond anything I ever imagined. And he taught me not only how to think and do scientific research, but also the paramount importance of being able to clearly articulate my ideas both orally and in written publications, and how to debate and modify my thinking based on rigorous scientific discussion. 
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On the other end of my career, I’d also like to recognise my colleague, Dr. Agis Kydonieus.  He is a chemical engineer, who has taught me everything I know about the science of drug delivery and also everything I’m starting to believe about retirement. We are co-inventors on numerous patents, and he has taught me the knack of the industrial inventive process and development of intellectual property. He is also inspirational in his desire to keep working well into an age when most people have retired: not because he has to, but because he wants to. This is a good model for me at this point in my life.

5.

What advice would you give someone considering working in the rare disease space?

First and foremost, do everything you can to meet people and families who are living with the condition you’re studying. Get to know their stories and their journeys. I guarantee you will find their resilience and determination to live full lives inspiring and motivating. Get everyone on your team to meet them, too.
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These programs are not about chasing large markets. They are about doing everything in your power to help specific individuals within the community. It really helps to maintain commitment and urgency when you can put faces and names to the people in your community of interest. And it helps immensely in pressure testing your ideas, to see if they would be truly helpful to the people you are trying to serve. This is a level of information you can’t glean from market research reports.

6.

Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?

Let’s start with what the government is doing well. I think it’s great that the orphan drug act, rare paediatric disease, and break through designations exist. They provide strong incentives to lower the cost and increase the rewards associated with the economics of developing drugs for rare populations. Without the economic incentives, it just can’t happen.  And you can see that these regulatory innovations have had an impact in increasing the rare-disease interest level within the industry. So, kudos for that.

Can they do more? Yes. I think taking a holistic view of the ecosystem for developing drugs for rare diseases requires that one also consider the question of barriers to access for patients. Let’s face it, even the best-intentioned companies must operate at a profit in order to exist, and that often translates into high (sometimes outlandish) pricing for rare-disease therapies. I would love to see an environment where everyone who needs access can get it, full stop. How do we get there? I wish I knew. I am not one to advocate for government price controls.

Here are two ideas, though.

For a drug truly targeting a rare disease population and without the potential for use in a broader population, perhaps the period of exclusivity for the innovating company should not be limited by patent expiration. Perhaps some companies might be willing to contractually agree to longer periods of exclusivity if they also abide by restrictions on pricing and a limit on distribution for off-label use.
For drugs that do have the potential for use in broader populations, perhaps the regulatory requirements for orphan drug designation or fast track approvals might be relaxed to allow a first entry in a rare condition followed by expansion into a bigger market. More buyers should translate to lower prices per dose. 
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I’m sure a lot of people who are more well versed in access and pricing issues have better ideas than mine. I just know that all the elegant science in the world is of no use unless we can ensure that the people who need these products can get them.

7.

​What would you say are some of the biggest motivators for your employees?

We are colleagues more than employer/employees. Many of us have worked together on other projects, and there is a lot of personal relationship capital that we’ve all invested in each other. That keeps us tuned in to working as a team and helping each other through the inevitable setbacks one encounters in these journeys. I also look for opportunities to have the team meet at least once a year with a guest who has venous or lymphatic malformations. I want everyone on the team to feel personally invested in helping someone specific—to put a face to our goals, and never give up until we make their lives better. 

8.

What are the toughest parts of being a CEO, and conversely what are the most rewarding?

I will flat out start off by saying most days I love being a CEO of a small company. Most days. 

It’s kind of a funny job with a broad range of responsibilities and many sleepless nights come with the territory. The most challenging responsibility can be to ensure that the company is adequately capitalised at each stage of development. In some companies, working with a private investor syndicate, managing the Board can almost become a full-time job, especially when funds are getting low and the syndicate is a little shaky in its commitment.  That is not as much fun as leading the actual development of a product; it can be quite stressful. At Venthera, that’s a fairly easy part of the job because we are supported entirely by BridgeBio. Part of their model is to take the pressure of capital raising off the backs of their CEOs and free us to focus on developing excellent pharmaceuticals for the communities we are serving. I like that a lot.

The most rewarding aspect on a day-to-day basis is working with highly talented colleagues, who are performing at their peak to achieve goals and overcome obstacles. That scratches an intellectual itch for all of us, and there is a zen to it: especially when you know the team is performing at a level, with limited resources, that big pharma bureaucracies just can’t achieve. Long term, the most rewarding thing is to look back and say “We beat the odds.  Despite everything, we got this drug across the goal line.”  When you can do that, you know you’ve done something good for the people you’re really trying to help and also for the team that made it happen.

9.

What would be your one wish for Venthera for the year ahead?

I hope that we have a dynamite first-in-human study result and that we are able to demonstrate the safety, tolerability, and a hint of efficacy that will be necessary to justify continued investment of time and money into Venthera. That is our next big step in bringing hope to people in the venous malformation and lymphatic malformation community.

10.

If you weren’t CEO of Venthera, what was Plan B?  What did your 10-year-old self want to do as a job?

My 10-year-old self was more interested in riding bicycles and fishing than planning a career! But it was at that age that my parents bought me a home chemistry set, which I found fascinating. Although I’m in the first generation of my family to have gone to college, my father had an intellectually curious mind. I grew up reading Popular Science and Popular Mechanics (still waiting for those flying cars we can build for ourselves!) and National Geographic. Dinner conversations often centred around the topics from these magazines and observations of the world around us. So, I guess the idea of being a scientist was implanted in my thinking at an early age without me even knowing it.

If I wasn’t working for Venthera, I suppose I’d be doing what I was before Venthera came along: a little consulting here and there, looking for inventors I might be able to help, but mostly spending my time riding my bicycle for charity and racing cars for fun. Lately I’ve become interested in a post-Venthera idea to try my hand at formulating some skin care products based on natural ingredients sourced from the oceans and the New England forests. I think it would be fun to do something self-funded where I can head into work, put on a lab coat, and see what I can invent.