Mereo BioPharma : Harnessing Agility and Customer Intimacy to Drive Success in the Rare Disease Space

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Left to right:

Akihiro Ko, CEO of Elixirgen Therapeutics, Denise Scots-Knight, CEO of Mereo BioPharma, Jason Tardio, COO of Ovid Therapeutics and moderator Kevin Baruzzi, Partner at Simon-Kucher.

Harnessing Agility and Customer Intimacy to Drive Success in the Rare Disease Space

During the recent World Orphan Drug Congress USA 2023 in Washington DC, our CEO Denise Scots-Knight joined a thought-provoking panel discussion, 'Bigger isn't necessarily better - examining the creativity and flexibility smaller companies have in the rare disease space', alongside Akihiro Ko, Chief Executive Officer of Elixirgen Therapeutics, Jason Tardio, Chief Operating Officer of Ovid Therapeutics and moderator Kevin Baruzzi, Partner at Simon-Kucher.
The session challenged the conventional belief that bigger is better in (bio)pharma, especially when it comes to the development and commercialization of orphan drugs and treatments for rare diseases. The panellists shed light on how smaller companies possess inherent advantages to be successful in this specialized sector.
Unparalleled customer intimacy
One of the resounding themes that emerged was the unmatched customer intimacy smaller companies can achieve. In these organizations, absolutely everyone has direct contact with customers. "At Mereo, we further reinforce this opportunity to hear directly from our customers by inviting people living with the conditions we're developing treatments for to talk with the entire company, so we all - no matter what our role - feel connected to our mission", explained Denise Scots-Knight. This close proximity to the communities and people directly affected by a given condition enables a deep understanding of the needs, challenges and aspirations of patients, caregivers and healthcare professionals. As a result of actively soliciting this first-hand experience, smaller companies can tailor their development and commercialization strategies to address specific aspects of rare diseases effectively.
Other participants also highlighted the value of being able to put the patient needs as the company's 'North Star'. Akihiro Ko commented: "It's critical for the full team to truly believe in the company's mission because it's that genuine love for the work that allows us to overcome the obstacles that small biotechs involved in rare disease face to get these novel therapies to patients in need." Whereas larger organisations might often be forced to balance a multi-faceted, complex portfolio on the basis of synergies or other business drivers, a smaller company can ask itself: 'where's the unmet medical need?' and 'how can we explore opportunities to address this?'. The ability to consistently challenge the organisation about what is in the best interests of the patients and the families, in a way that might not be possible for a larger entity, is an area where smaller organisations can harness a different way of advancing their business.
Flexible, real-time decision making
With a small footprint of highly specialized and engaged individuals, smaller companies can adapt swiftly to changing circumstances, altering their course when necessary. Being small grants a distinct advantage because bringing key decision-makers together in real-time to brainstorm innovative solutions is daily life and culture. This agility ensures that small biotech remain focused on their mission and can rapidly respond to emerging opportunities or challenges. "At Mereo, we identify assets and programs focused on rare disease that have been de-prioritized by larger companies due to changes in their strategic direction. In less than five years, we now have two rare disease programs addressing significant unmet need about to enter Phase 3 trials, thanks to our resilience and agility", pointed out Denise Scots-Knight.
Other participants highlighted the potential of the consistent information flow and a real-time, cross-functional approach to rapidly accelerate drug development. The need to bring the team together to get things done, no matter what their function, means that R&D is already talking with Commercial - there is no need to bridge between functions later on. In larger companies, instead, huge vertical organisations need to exist to address the size, scale and complexity of the portfolios. "When you need to harness all your internal resources to move things forward, you are forced to sit down together and share learnings. So the 'all hands on deck' approach unleashes creativity and the cross-functional collaboration unlocks potentially disruptive thinking. A challenge becomes a virtue", noted Denise Scots-Knight.
Pioneering & co-creation to successfully address unmet needs
Rare disease development often requires pioneering and path-creating - doing things for the first time. Indeed, being able to do so is often the key to success, particularly when developing a treatment where there has been none before. The panellists emphasized that a handful of individuals who possess deep understanding of rare disease sector, the curiosity to dig deep into a therapy area with unmet needs and the ability to foster key relationships in these small and highly networked expert communities can, together, make a substantial personal contribution to advancing the field.
The smaller organizational structure fosters synergy and efficient decision-making, and this can expedite the pace of drug development. However, small biotechs are not only able to harness collaboration within their four walls but can also promote co-creation by working in tandem with external stakeholders such as payors, regulatory bodies, and patient organizations to address a significant unmet need. When there is no established pathway to follow, you need to ask questions, and not be afraid to do so. "Not only do we listen to the patient communities, but we are also involving regulatory bodies, value assessors like HTAs, and the payors at an early stage of development to make sure clinical trial programs meet their requirements. We believe this will support more productive value assessments, informed decision-making and productive reimbursement discussions in the future, expediting access to innovative therapies, which is the end goal for all of us," noted Denise Scots-Knight.
The challenge of attracting the right talent
Being a small company comes with its challenges too, and this is not only down to the obvious lack of resources, when compared to big pharma. Being small on its own is not enough to guarantee success. A certain mindset is required to work in a small biotech - wearing multiple hats, being ready to 'figure things out' on your own, as well as being unafraid to draw on your ingenuity, experience and network when you can't - and that isn't for everyone.

Many organisations encourage creativity; the small biotech environment demands creativity - it is a critical success factor. You might not have everything you need inside your team, but you need to know where to find it, and fast. You need to be able to prioritise quickly - deciding what to do, as well as what not to do. And have a growth mindset: draw on your well of experience, while not being afraid to learn as you go.
"It requires a special kind of person to be able to deliver in a constantly evolving, entrepreneurial 'build it yourself' environment. I always ensure that our new potential team members have a solid and real understanding of what this means," added Denise Scots-Knight. "Very often the question is not 'how was it done before?', because there is no before to follow, but 'why don't we do it like this?'. Sometimes the way it has never been done before is the right way forward. And it needs a certain type of person to think this way, but when a team like this comes together, it's incredible".

While challenges exist, by embracing their ability to deliver with creativity and flexibility, smaller companies are playing a vital role in transforming the rare disease landscape, ultimately benefiting patient communities worldwide.

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