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With a strong pharma background that includes bringing non-US biotechs stateside in the rare diseases space, Rami Levin, CEO of Saniona, talks about the importance of growing his company’s business and his dedication to that mission.
Pharm Exec: How and why did you get involved in rare diseases?
Levin: I worked with rare diseases at other pharma companies prior to my current role as president and CEO of the Danish pharma Saniona. But my first immersion in rare diseases came when I joined Swedish pharmaceutical company Sobi more than eight years ago. It was there that I was introduced to the staggering statistics around rare diseases and the significant unmet need: There are more than 7,000 identified rare diseases but less than 10% have an FDA-approved treatment.
It was also at Sobi that I formed relationships early on with wonderful organizations like the National Organization for Rare Disorders (NORD) and Global Genes, as well as the patient advocacy groups that represented the ultra-orphan conditions we treated, all in an effort to build deeper connections with the patient communities we served.
These relationships showed me just how critical it is to incorporate patient insights into everything you do when you work in rare diseases. We were able to connect with patients and their families, hear their stories and experiences, and turn those interactions into insights that we could bring to our daily work, from research and discovery to clinical development to patient services. Our conversations further highlighted to me how meaningful it is to be able to bring patients and families hope by letting them know that there are teams working to address their disease by developing a treatment that could have a life-changing impact. After that, I knew I wanted to continue working on rare diseases and with the rare disease community. When offered, I could not pass up the opportunity to lead Saniona, a company focused on discovering, developing, and ultimately commercializing therapies for patients suffering from rare diseases.
Pharm Exec: You have a history of bringing non-US biotechs stateside. In your experience, what are the greatest challenges in building a footprint in the United States for a European company?
Levin: If you are in the rare disease space in particular, the US has the most established regulatory pathways, the most developed rare disease market, and the most extensive healthcare investor base in the world, which makes it a critical market for biopharma companies.
This is why Sobi hired me as president of their North American affiliate—they knew they needed to build up their US presence. At Sobi, I had the opportunity to build and lead the North American organization from infancy to a team of approximately 300 employees generating over half a billion US dollars in annual revenue.
When Saniona hired me to be its president and CEO, I saw a similar opportunity. I was the company’s first employee in the US, and over the past two years, I have built a team of more than 25 US employees with expertise in US regulatory, clinical development, technical operations, and finance to allow us to initiate two Phase IIb trials with our lead product Tesomet for hypothalamic obesity (HO) and Prader-Willi syndrome (PWS), and a Phase I trial with SAN711 for rare neuropathic disorders.
There are always challenges with building a US organization, ranging from raising capital to ensuring you engage with regulators and patients early, while also establishing a physical presence and bringing onboard the right talent to support all the above. The greatest challenge really depends on the stage and focus of the company. At Saniona, we recently initiated two global clinical trials under US Investigational New Drug Applications (INDs), so building an experienced regulatory and clinical development team has been a significant focus for us.
Pharm Exec: How did the COVID-19 pandemic affect your plans for bringing Saniona to the United States?
Levin: We knew we wanted to conduct clinical trials under an IND with the FDA, to support eventual filings for approval in the US. This made building a clinical and regulatory team with US experience critical. When I joined the team in January 2020, my first hire was our US-based chief medical officer, Rudolf Baumgartner. This was an incredibly important position, and yet because of the pandemic, I had to hire Rudy without ever having met him. In fact, we worked together for two months before ever meeting in person. Initially, we were the only two US-based employees, while the rest of our research organization operated in Denmark. COVID also made travel to Denmark impossible, so we quickly became very good at connecting virtually.
While unexpected, we did not allow the pandemic to hinder our vision of bringing Saniona to the US. Even in a virtual environment, Saniona was able to raise $65 million in funding—something I would never before have believed was possible. We also grew our US team to more than 25 people within two years—most whom we interviewed and hired without ever meeting face-to-face. I’m proud to say this team was not only built but has learned to collaborate and thrive despite most of them never having met in person. We have achieved multiple clinical milestones. We have also formed strong relationships with the PWS and HO patient advocacy communities, despite not being able to sit down face-to-face as we usually would.
Pharm Exec: Why is it important to expand your rare disease programs to the United States? Do you have further plans for global expansion?
Levin:As I mentioned, the US has the most established regulatory pathways, the most developed rare disease market, and the most extensive healthcare investor base in the world. The resources and opportunities available here make the US a critical market to consider, particularly for biopharma companies looking to advance potential treatments for rare diseases, the majority of which represent patient populations that are suffering and eagerly awaiting a treatment option.
Saniona is currently focused on our work in the US and Europe, but our vision is to build an international rare disease company, improve the lives of rare disease patients around the world and, ultimately, we want to ensure our treatments are delivered to all patients who need them.
Pharm Exec: What potential do you see for this market moving into 2022 and beyond?
Levin: New rare diseases are being discovered every day and the unmet need only continues to grow. With so few approved treatments available, I see significant opportunity for continued scientific innovation and advancement that can truly benefit patients. I am proud of Saniona’s contributions thus far—which, in addition to Tesomet, include advancing multiple additional candidates from our proprietary ion channel drug discovery engine with the potential to treat rare neuropathic disorders as well as rare inflammatory and fibrotic disorders—and I look forward to what is on the horizon as we work toward our vision to improve the lives of rare disease patients around the world.