From lab to clinic to NASDAQ: how one female healthcare entrepreneur is doing it all
Dr. Aimee Payne, MD/PhD, Co-Founder of Cabaletta Bio, on all that she has learned building a highly successful cell therapy venture. Dr. Payne now serves as Co-Chair of the Scientific Advisory Board, alongside continuing her work in research lab and seeing patients in her dermatology practice.
How has a physician-scientist translated her clinical and research experience into a healthcare company with an IPO of $74.8M and current market cap of $260M? In this episode of Spotlight on Women in Health Ventures, Dr. Aimee Payne, MD/PhD, co-founder of Cabaletta Bio, shares her journey to becoming an entrepreneur. Cabaletta Bio is using cell therapy technology to target the autoimmune skin condition pemphigus vulgaris (PV), with their research steadily expanding to other indications. Her secret? Always seeing opportunity for growth and improvement.
Dr. Payne co-founded Cabaletta Bio after treating patients with PV at her dermatology practice. Treatment for autoimmune diseases usually consists of global suppression of the immune system, which leaves patients with tremendous sensitivity to infection, and these treatments have only moderate effectiveness. Dr. Payne started her research lab in 2006, and she began looking for something about PV that was targetable: B-cell immune profiles, disease-causing antibodies, amino acid patterns. She was looking for a “signature smoking gun.”
In the years that followed, Dr. Payne developed the technology, and through collaboration and support from institutional and national resources, she launched Cabaletta Bio along with Dr. Michael Milone and Dr. Steven Nichtberger in 2017.
From academic physician to entrepreneur
Though seemingly disparate worlds, academia and entrepreneurship, Dr. Payne highlights, are not so distinct, as many of the skills that one needs for research and professorship carry over to the business world. “Most people who are in academics have learned how to develop a story”, as one needs to do when applying for a grant or presenting research. Dr. Payne has dedicated much of her time to bridging these two worlds through networks and mentoring.
Dr. Payne recognizes that “ultimately, the relationship between academic research and commercialization is absolutely synergistic and essential.”
After completing the proof of concept for her technology in the lab, Dr. Payne saw that traditional academic funding would not be enough to get the novel therapy for PV her lab had developed to everyone who needs it. Many researchers see the space between the lab and the market as a “Valley of Death”, which is why Dr. Payne emphasizes the importance of resources other than those traditionally used by the academic world (i.e., grants). In addition to such funding, commercialization requires interfacing with manufacturing companies, considering factors such as market size and competitors, and thinking about patients’ and investors’ needs. Working closely with the University of Pennsylvania, the team used their professional and academic experiences and relationships to develop Cabaletta, from proof of concept to clinic to market.
Seeking out the right resources
Dr. Payne found guidance in a course offered at the University of Pennsylvania, the Capstone course for the Vagelos Life Sciences and Management program (LSM). This experience is an intensive, year-long, project-based course in which senior LSM students form a business plan for a novel medical technology or therapy. With her group of 4–5 students, Dr. Payne developed the many aspects vital to building a successful company, including preclinical strategy, product development across phases, and financial planning.
While this course is specific to the LSM program, the material covered is vital for anyone looking to start a healthcare company.
She highlights that while not everyone might have access to such a resource, it is vital to “talk to people who have done what you’re trying to do before.”
There may not always be a direct example or precedent, so Dr. Payne also points out the importance of networks, such as groups like Advancing Innovation in Dermatology, and even Theia.
A duty to change the cell therapy space
One choice Dr. Payne had to make early on was whether to sell her technology to another company, or to follow through with founding Cabaletta with her team. In her experience, she found that many cellular therapies, especially CAR-T therapies, were concentrated in the cancer space. She says that not many companies had the mental and financial bandwidth to take on the new project and to give it the attention it deserved.
She recognized that with her many years of working with patients with PV, she was in a unique position to change the space. PV is a relatively rare disease, with less than 3.2 cases per 100,000 population, but it is severely debilitating to those who have it, causing painful blisters on the skin and mucous membranes and deeply disrupting patients’ quality of life. A rare autoimmune condition like PV could easily be overlooked as the primary indication for a novel therapeutic modality. To ensure that the therapy made it to the patients who need it most, Dr. Payne reflects that
“to a certain extent it was a responsibility, because no one had thought of the opportunities and the challenges as much as we had.”
Right place, right time
Starting Cabaletta instead of selling the technology to another company was certainly a lot to take on. When Dr. Payne co-founded Cabaletta, she says that she had reached a point in her life when it felt right to do so: she had less childcare responsibilities, as her children had reached a relatively independent age, and she had just received tenure in 2015. She recognizes that founding the company when she was juggling the expectations of an untenured professor, such as publications and grants, would have been difficult. But by the time the opportunity to launch Cabaletta arose, she felt comfortable in her space, and Cabaletta was a next step, not something that she was trying to juggle.
She recalls a conversation with her husband, asking if they were ready for this:
“I am about to take on a 150% time job on top of my 150% time job. But how can you say no? It’s a once in a lifetime opportunity as a physician scientist, it’s a dream.”
The importance of building a balanced, dedicated team
Dr. Payne did not do it alone, though, and her relationship with her two co-founders exemplifies finding people who complement and augment one’s skills and knowledge. Dr. Michael Milone brought his perspective as a co-inventor of Kymriah, the first FDA-approved CAR T cell therapy for B cell cancers. Dr. Steven Nichtberger had had years of experience starting and building companies in the healthcare space. And Dr. Payne brought the preclinical and clinical strategy perspective, as she knew the space of dermatology and PV and had been researching the condition for almost ten years.
“We’re like three legs of a three-legged stool. We complement each other really well, and we fall down if one of us is missing.”
Dr. Payne also makes the point that no one person can give you all the types of support you need, emphasizing the importance of a diversified team. You need people to give you moral, financial, scientific, and other kinds of support. At a given point, someone can be a welcome supporter, but also a stressor, so Dr. Payne emphasizes that it is essential to have someone else to go to. And when times get hard, this support system will be vital.
The big idea
PV is an autoimmune skin disease in which painful blisters develop on the skin and mucous membranes. Cabaletta Bio’s treatment for PV relies on CAAR T cell therapy, which you might be unfamiliar with, so we’ll break it down.
CAAR T cell therapy
In chimeric antigen receptor T cell (CAR T) therapies, such as Kymriah, a CAR T cell has antibody fragments that recognize the CD19 antigen and therefore target the healthy B cells and (in the case of cancer cell targeting) leukemic cells that express this antigen. Drs. Payne, Milone, and Nichtberger were able to see a benefit to this strategy for targeting pemphigus vulgaris, but the model was flipped.
In CAAR T therapies, a chimeric auto-antibody receptor T cell is engineered to have the autoantigen on its surface, and pathogenic B cells will recognize this autoantigen with the autoantibodies on its surface. Once this interaction occurs, the engineered T cells can then kill the pathogenic B cells. Specifically, Cabaletta’s therapy for pemphigus vulgaris targets B cells that express anti-desmoglein 3 receptors; they have done sufficient research to understand that if one can rid the body of these cells, the disease will be disrupted.
This method of targeting specific cells “brings a precision medicine approach to autoimmune diseases”.
Final thoughts from Dr. Aimee Payne
There is academic recognition of implicit and systemic gender bias, and this is an issue that many women in healthcare leadership feel the pressure of.
Dr. Payne sees a clear pathway to improving representation of women in healthcare leadership as relatively clear at this point, especially from her experience of mentoring other female physician scientists.
From her perspective, the main points of action are:
- Identify and address practices and biases that perpetuate systemic gender bias.
- Establish a network of women and men who champion and educate (not just passively support) women in the career path.
- Create forums and a support network where aspiring women in healthcare and entrepreneurs can be educated about what they need and how to grow.
- Help women advocate for themselves at every step of the way.
The usage of existing CAR T therapy for autoimmune diseases instead of cancer, melding the worlds of academia and entrepreneurship, and the dedication to seeing past the current indications of Cabaletta Bio’s technology to future possibilities, all exemplify Dr. Payne’s penchant for forward-thinking solutions.
Her continuous search for the next indication, the next solution, has led to Cabaletta Bio being a force in the cell therapy and autoimmune space. “Don’t be so much of a self-critic that you sell yourself short on the opportunities… Be innovative, and look beyond the opportunities of your initial product.”
Theia is a 501(c)(3) non-profit dedicated to inspiring and empowering the next generation of women entrepreneurs and investors in healthcare. Visit our website to join our community and access resources that will support your entrepreneurial journey and pursuit of changing healthcare.
Story written by Priya Kumar and Luiza Perez.
