Member Spotlight: Q&A with Akcea Therapeutics

Nov 01, 2017

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. This month we spoke with Paula Soteropoulos, president and chief executive officer of Akcea Therapeutics, who has worked in global drug development for nearly 30 years, including senior roles at Genzyme and Moderna Therapeutics. She serves on the Board of Directors of uniQure B.V. and the Advisory Board of the Tufts University Chemical and Biological Engineering Department. Paula has been involved with a number of initiatives to support the development of female leadership in business, including the Healthcare Business Women’s Association and Women Unlimited.

Tell us about Akcea and your current initiatives.

Akcea is a public company dedicated to the development and global commercialization of novel therapies for lipid-related cardiometabolic disease and championing rare disease patient advocacy and collaboration. Our goal is to transform the treatment of unaddressed or under-addressed lipid-related cardiometabolic disorders with leading-edge, RNA-targeted medicines.

We are currently focused on preparing for the global commercialization of volanesorsen, our lead therapy in development for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease of enzyme dysfunction in which the clearance of chylomicrons (formed by a high level of triglycerides) is impaired. People with FCS can experience life-threatening pancreatitis and severe, recurrent abdominal pain, among other life-limiting symptoms. We have filed for regulatory approval for volanesorsen in the United States, Canada, and the European Union. We are also advancing a pipeline of three additional, novel, clinical-stage candidates for the treatment of multiple serious lipid-related cardiometabolic disorders.

How do Akcea’s activities help patients now and into the future?

Everything we do at Akcea ultimately comes back to understanding and serving our patient community better, and we know the responsibility that comes with the work we do. Currently, there is no approved therapy to treat FCS, and these patients live with tremendous unmet medical need. Beyond the clinical aspects of their disease, we are working hard to convene, listen, understand and support our community through every aspect of their medical journey – a fitting word to describe what patients with rare diseases go through, when diagnosis often initially eludes primary care physicians.

With external audiences, we work to raise awareness of these rare conditions and how they are linked to cardiometabolic disease, a huge and growing source of morbidity and mortality worldwide. Increased awareness helps with diagnosis rates, therapeutic investment, medical practice and the development of support networks.

Among our initiatives is, dedicated to patients with familial chylomicronemia syndrome, the disease targeted by our lead therapeutic candidate volanesorsen. In addition to information about the biology and symptoms of FCS, aims to help patients manage their condition from multiple angles including dietary information, recipes and aids to staying on track with dietary management. We also provide how-tos on symptom tracking, best practice for record keeping, links to relevant communities and assistance with identifying suitable healthcare providers.

What differentiates Akcea as an organization?

There is not a day that goes by in our office without a conversation about the importance of what we’re doing for patients who are waiting for a treatment; it drives us and our work. We’re working in disease areas nobody has been able to address in the past, and that presents us with the opportunity of being first, and the responsibility that comes along with that pioneering role – connecting the community, providing disease awareness education, supporting diagnostic knowledge among physicians, and enhancing understanding of the natural history and burden of disease.  For example, we recently presented results of a first-of-its-kind study called “IN-FOCUS”, in which we invited 166 patients from around the world to share the holistic experience of living with FCS. We firmly believe that in rare diseases there is a particular need to understand as much as we can about the totality of the impact of living with a rare disease, not only the direct symptomatic effects.

As a biotechnology company, what also differentiates us from others in the industry is the gender balance reflected among our leadership, Board of Directors and the wider team. We strongly believe that a diversity of opinion leads to better decisions.  As CEO of Akcea and throughout my career, I have worked to support the advancement of female leadership across multiple industries, both within my organization and through external initiatives. I think it’s incredibly important that young women see a place for themselves as they rise through the ranks. It’s my responsibility – along with other women in executive and senior positions – to encourage our next generation of biotech leaders. To this end, Akcea was recently named a “Top 100 Women Led Business” in Massachusetts for the second year in a row. I’d like to see more representation from our industry in this group.

What do you see as the biggest challenge facing the life sciences industry today?

It’s really a matter of perspective and priorities what someone terms ‘the biggest’ challenge. But I do think that our industry has to invest more in our pipeline of talented women to support and advocate for opportunities to help them grow as leaders, to take risks on them and to encourage them to put themselves forward for those next opportunities. And I think this issue deserves attention and resources from all members of the life sciences industries. Certainly the results of the recent study commissioned by MassBio suggests there is room for better understanding and improvement in these areas.

What’s next for your organization / what are you focused on in the coming year?

With our recent IPO this summer and regulatory filings for volanesorsen in the United States, Europe and Canada, we now turn to preparing for the commercialization of volanesorsen for FCS patients who currently lack effective pharmacological treatments.

We are building our networks within the patient and physician communities and other resources to support a multinational launch. We have been focused on identifying patients, connecting them to each other, and providing them with resources to ensure they feel supported and more empowered in managing their condition. That work is continuing with our patient engagement activities over the next year, but we are also now expanding our discussions with physicians – one of our key partners in delivering novel therapies to rare disease patient communities.  We believe part of our role is helping physicians understand this disease and its unique challenges in both diagnosis and treatment.  Our growing field team is interfacing with physicians towards that end, as well as dietitians and nurses to create layers of support for our patient community.  It’s about awareness and understanding of the disease and then engaging to ensure each patient can attain a better quality-of-life. 

In addition, we have a number of ongoing or planned new Phase 2 studies for the other candidates in our pipeline, which are exciting and have the potential to further benefit the patient community for underserved lipid-related cardiometabolic diseases. 

Akcea continues to grow at a rapid pace and our team is excited and driven to be bringing forward potential life-saving and life-altering therapies to patients in need. 

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