Expanding Research Into Rare Diseases

Chris Clark, global head of asset and commercial strategy, rare diseases, UCB, discusses UCB’s expanded efforts to treat patient populations with rare diseases, including UCB’s work on two investigational treatments for myasthenia gravis.

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Welcome back to PopHealth Perspectives, a conversation with the Population Health Learning Network where we combine expert commentary and exclusive insight into key issues in population health management and more.

Today, we are joined by Chris Clark, global head of asset and commercial strategy at UCB. He discusses UCB's expanded efforts to treat patient populations with rare diseases, including UCB's work on two investigational treatments for myasthenia gravis. Chris?

I'm Chris Clark, and I'm the head of asset and commercial strategy for rare diseases at UCB. I've been with UCB for 21 years now.

I've worked in several different areas and commercial environments including some areas which resonate with the policy analyst area. I've worked on long-range innovation projects, such as predictive analytics, artificial intelligence, software as a medical device, and programs which can help patients and health care professionals make informed decisions about their medicines.

This experience has provided some great foundations as we build a new Rare Disease Organization at UCB and progress the development program for our portfolio of medicines in the rare disease space—including an anti-FcRn candidate as well as a complement C5 candidate.

Can you talk about ways UCB is building on its scientific knowledge and heritage of understanding the perspective of patients to expand into new areas?

UCB has decades of experience supporting the epilepsy community. Through that, we've learned the value of bringing innovative, meaningful treatments and solutions focusing on the elements which bring real value to the lives of patients.

What started a decade and a half ago as words on the page has now become our core compass that guides our decision-making. Everything we do is structured around how we might best produce value for patients.

The natural evolution of that, over time, has brought us to a finer granularity in terms of understanding the populations that we serve, the subpopulations inside of larger indications, and ultimately, has brought us to this point where we're now entering the fields of rare diseases.

Specifically, first, we're looking at myasthenia gravis with two different assets. We know that in rare disease populations you, in order to be successful on behalf of patients, have to bring a robust offering that is a combination of clear scientific advancement that matters to patients but also robust educational and support programs, and a true engagement in the community that supersedes what might be typically expected in broader pharma marketplaces. Here, we have to engage and make sure that everything we do is being balanced by the needs of the patient community that we serve.

How will this expansion help patients with rare neurological diseases and what about for gene therapies?

There are many different available treatments out there. Despite this, there still are a significant number of people with myasthenia gravis that experience unpredictable burden in their disease, and this impacts their ability to perform many of the activities of daily living.

Our goal is, by this expansion into rare diseases, to go beyond just minimizing clinical manifestations, but to really get involved in, hopefully, improving the entire treatment paradigm altogether. A goal here that we have is to genuinely elevate the standard of care via our portfolio.

We begin efforts such as this in the way that we do everything at UCB, and that is by starting with the patient. I'm really proud of our teams, and that, recently, at the American Academy of Neurology meeting, our team, in collaboration with myasthenia gravis patients, led the way in sharing a groundbreaking study on the patient impact of myasthenia gravis.

Some of the key learnings from this about the constantly adapting activities and the fluctuation of the disease itself make living with MG very unpredictable. This leads to sacrifices and tradeoffs in many aspects of life that really are devastating for the myasthenia gravis patients.

More importantly, we also learned that this is commonly misunderstood. These patients with a significant unmet need find a disparity in understanding between what's really going on with them and what their physician perceives.

It's a real opportunity to improve that, for us, really illuminates a pathway to elevate the standard of care. UCB invests approximately 25% or more of our revenue in R&D to support new compounds and to address the needs of patients living with many different diseases, including many rare, autoimmune neuromuscular diseases, such as MG.

Even though this only affects a small patient population, it's a patient population with a really legitimate unmet need that we think that we can help improve. For that, we're working with two different investigational compounds.

One of our development medicines, zilucoplan, is a once-daily, subcutaneous, self-administered peptide inhibitor of complement C5. We believe that in the future this could offer the potential to deliver value for patients living with myasthenia gravis, and with this in mind we're currently conducting a study to try and validate this hypothesis.

We're also working on another development medicine, rozanolixizumab, a subcutaneously administered, humanized, monoclonal antibody. This type of medicine has been designed to block interaction at the neonatal FC receptor with IgG, with the aim of inhibiting the IgG recycling, therefore inducing the removal of the pathogens, which are IgGs themselves, in the case of myasthenia gravis.

We believe with those two different approaches, there could be a natural synergy that can be created to help us elevate the treatment paradigm and, hopefully, create a unique approach for patients.

We believe that the real opportunity to lead here is to help the community understand which type of medicine could benefit specific, targeted patient populations. The nature of a lot of our investment in R&D at this point is to try to provide some of that evidence for the future.

We also go beyond just providing treatments and also try to partner across scientific, academic, government, and patient advocacy groups to improve the lives of patients living with MG across the full spectrum of their lived experience to the extent that we can.

We engage with patient communities and learn from those who live with and treat MG every day to collaborate, and hopefully work together as a partner in the health systems’ goals of alleviating disease and doing that in an efficient way to ensure that despite the fact these diseases are, they indeed are diseases that we can focus on and have a real impact and drive an improvement in patient lives.

When we talk about technical evolution, in the future we believe new approaches will drive a fundamental change in how diseases are treated. Moving from symptomatic treatment to more disease modification, and eventually, hopefully, towards a cure for several severe, chronic diseases.

We believe gene therapy has this potential, and therefore, we're significantly contributing towards that ambition. In 2020, UCB acquired Handl Therapeutics and announced a collaboration with Lacerta Therapeutics, both of which strengthen and expand our current pipeline, our capabilities, and our therapeutic platforms in gene therapy and, hopefully, put us on a pathway to bring differentiated medicines in the future that could really bring some unique outcomes and hopefully reach some of those ambitions that we spoke of.

What do payers need to know about these advances? Will they have to adjust their formulary designs?

We'll take time to educate, inform, and discuss advances with all stakeholders, whether they be HCPs, healthcare providers, or those in access and care of all across the stakeholder spectrum. We're going to do this through an extensive program that ensures that all are well-informed and well-equipped.

Our goal is to help the community by illuminating a pathway to a better treatment paradigm overall, and that we believe with our portfolio approach, and with candid authentic education for all stakeholders we can best reach that aspiration.

How will health care professionals be able to use this new knowledge that UCB has identified in every day clinical practice?

We question and challenge the status quo constantly, guided by a commitment to meeting patient needs in the best ways possible. UCB pursues innovations that transform and reinvent patient care beyond our medications to thoughtfully address the challenges patients face throughout their care journey and within the health care system.

Beyond our R&D into new treatments, UCB is developing the tools, technology, education, and support to foster a more connected and tailored experience for patients and their families, their caregivers, and their health care professionals.

That's just about it for my questions. Is there anything else that you would like to add to the conversation or anything you feel like you'd still like to say?

UCB has decades of experience supporting patient communities with severe unmet need. One shining example from our heritage is our partnership that has lasted in multiple decades with the epilepsy community, in which we've delivered innovative science and meaningful treatments, but also much broader solutions and partnership to the community.

That view of what it means to genuinely be engaged and to lead on behalf of patients with severe diseases is exactly the dedication that we're bringing forward now to help folks that have a wide range of challenging neurological and hematological conditions.

Our engagement will be manifest in scientific solutions, agile approaches where we will engage the community in partnerships and through strategic programs that are all designed to generate an elevation in the standard of care, and hopefully, alleviate suffering for many folks that are currently part of a population that is really underserved.

We're intentional in our pursuit of developing new treatments that address the needs of specific patient populations, particularly those that have limited treatment options, and for whom their unmet need is profound.

Hopefully, through that focus, and that continued granularity, a focus on smaller patient populations, we’ll get closer and closer to our objective of creating true patient value for as many as possible. We define that through outcomes that are best-in-class, experiences that drive patient preferences, and access for the right patients that's as broad as possible. That's what we define at UCB as patient value, and that's our goal.

Thanks for tuning in to another episode of PopHealth Perspectives. For similar content, or to join our mailing list, visit populationhealthnet.com.

*Editor's note: This transcript has been edited for clarity.