Rami Levin, president and CEO of rare disease biopharma Saniona, has been on both sides of the proverbial pharmaceutical fence: large indications and rare diseases. After he served as president at global rare diseases company, Sobi, he knew rare disease was the side of the fence he wanted to be on for the rest of his career.

“My experience at Sobi as president of the North America organization was my first real dive into rare diseases and it taught me that working in rare disease indications is truly rewarding,” Levin says.

After six years at Sobi, Levin had the opportunity to join Saniona as CEO and build a company around rare diseases. Saniona is focused on ion channel modulators to address rare diseases and is conducting phase 2b clinical trials for its product called Tesomet, which was developed to treat hypothalamic obesity and Prader-Willi syndrome (PWS) — two serious rare disorders characterized by severe weight gain, disturbances of metabolic functions, and uncontrollable hunger.

Through its ion channel expertise, Saniona is also advancing two wholly owned ion channel modulators:  SAN711, which is in phase 1 and may be applicable in the treatment of rare neuropathic disorders, and SAN903, which is in preclinical development for rare inflammatory, fibrotic and hematological disorders.

Here, Levin, a PharmaVoice 100 honoree, shares his thoughts on what it takes to be successful in rare disease research.

PharmVoice: Having led teams in several large pharmaceutical companies, including EMD Serono and Merck Group, how does “rare” differ when it comes to managing teams and interacting with key stakeholders?

Rami Levin: Rare disease organizations are typically smaller, so an obvious difference is you don't need as many people, but I think the main difference is that in rare diseases, there's a common understanding that the interaction with patients as we develop our drugs is critical. Every organization says it cares about patients, but there’s a huge difference between saying you care and truly caring. And I don't think you can be a successful rare disease company if you don't truly care about, interact with, and listen to the patients. They are the experts in their disease, in their struggles and in understanding their needs. We have to listen to them as we develop drugs for those indications. So, a key difference is the alignment of the team around the importance of listening to patients and carrying that through to everything we do.

From a stakeholder perspective, historically the patients, the investors, the healthcare providers were thought of separately. There were the patient meetings, the investor meetings, the key opinion leader meetings with physicians — those were all separate meetings. In recent years, those have blended, so now you have patients listening to investor calls and physicians listening to patient meetings and patient calls. That means we need to be very mindful of where some of the sensitivities are and consider the different informational needs of the various stakeholders.

How is Saniona making a difference for rare disease patients?

We’re in mid- to late-stage clinical development with our lead asset, Tesomet, for hypothalamic obesity and PWS. In the case of hypothalamic obesity we’re the first company to have received an orphan drug designation, which would enable us to bring the drug to the market faster for patients who really need it. There is also currently no treatment for hyperphagia, the uncontrollable hunger that is a debilitating feature of PWS.

In addition, we have a whole drug discovery engine behind us, with a every intent to continue to develop additional rare disease treatments. We have a team of researchers who have worked together on ion channels for 20 years.

How does your approach differ in terms of how you engage with patients?

One of our first hires was the head of patient advocacy, which is not very common for a clinical stage company. But we understand the criticality of having someone leading patient advocacy so that we constantly interact with the patient organizations, the patients themselves and the families.

We invite families to present at our town hall meetings in the company, because I believe that it's important for everyone in the company to understand why we do what we do. When you listen to a parent talk about their life and the challenges and their struggles, it just motivates everyone in the company to just work just that much harder to bring our products to market as quickly as possible.

What have you seen to be some of the biggest struggles for the rare disease community? And how can rare disease pharma companies like Saniona work to alleviate those?

One of the biggest challenges rare disease patients face is just the lack of information. With a disease like Alzheimer’s or Parkinson’s, not only are there many companies working on drugs, there’s also a ton of information available. But the vast majority of rare diseases have no product available and also very little information.

You can imagine a parent going to a doctor and being told their child has a rare disease where there is no treatment. That parent has probably never heard of that disease, there’s no readily available information and they have no idea where to find information. So, information would be No. 1.

By the same token, what families typically seek are other families who are struggling with the same rare diseases. In many cases, there is no family in your area, so finding someone to share your struggle with is another priority. If we consider the struggles faced by parents of children with Prader-Willi syndrome, where patients want to eat all the time and parents have to lock food cupboards and refrigerators, the burden is enormous.

What engagement and collaboration tips can you share with others in the rare disease space?

The first is to start early. Make sure you interact with patients and patient advocacy groups as soon as possible. Establish principles for engagement because those groups will want to know how to interact with you. What we found helpful was to set expectations within the company of how to interact with patients and patient organizations, but also clarify for them how they can interact with us.

Then establish a mindset of advocacy within the organization. As we develop clinical trials, we need to speak to these patient groups, in part to help us recruit into our clinical trials, but also to make sure that their concerns are addressed as we develop the clinical trials. As we prepare for the launch of the product, we need to make sure that not only do we have these functions in place, such as head of advocacy, but we have to prioritize and listen to patient expectations and needs.