1 From Patient Value Strategy to action in R&D

  • Thanks to innovative extrapolation, getting faster access to patients
  • Bringing clinical studies directly into patient’s home via our Partnership with Science 37
  • Patients play an active role
  • Identifying sub-group patient populations where we can have the biggest impact
  • Design patient-friendly clinical trials
  • In-house development or partnership, out-licensing, spin-off

The discovery and development of new drugs is a long and complicated process.

It is lengthy:

from the first test to the approval, it takes an average 12 years1

It is costly:

each R&D project costs approximately € 2 billion1

It is risky:

out of 10 000 potential molecule
only 1 or 2 project(s) will make it to the patients1

The evolving health ecosystem, along with pressures to reduce complexity, time and cost, make the old development model unsustainable.

1 EFPIA – The Pharmaceutical Industry in Figures – Key data 2018 – Accessed 15 February 2019

To tackle these challenges, UCB initiated the New Development Paradigm (NDP) to support our Patient Value Strategy and enhance our drug development approach in order to reduce the development time, bring down the development cost, and create differentiation and value. It is UCB’s aim to create novel routes to rapid patient access and maximize patient value throughout the drug development lifecycle, in line with 3 well-defined objectives:

Innovate our drug development approach

Differentiate the medicines we develop

Accelerate and optimize access to our drugs

The New Development Paradigm truly helps us realized our patient value strategy and get our medicines to patients faster, adresse their key needs and provide them with greater value. The NDP consists of 14 patients value drivers organized into 4 pillars:

  1. Patient Insights to Patient Outcomes
    At UCB, everything starts and ends with the patient. This systematic approach helps ensure that patients’ experiences, perspectives, needs, and priorities are incorporated and meaningfully captured into the development and assessment of our solutions. We engage patients and patient advocates before, during and after the development continuum to gain insights into patient unmet needs and preference that is translated into UCB development strategy that delivers real value.
  2. Data and its Utility
    We deep dive in clinical trial databases and real world evidence to better understand patient outcomes, populations, segmentation and complex heterogeneity of each patient’s journey, to answer a specific question or enhance clinical design. We also assess the direct/indirect impact of a therapeutic intervention on healthcare outcomes and cost, which can be used to show additional value of medicines to healthcare systems.
  3. Innovative Approaches
    We look at biomarkers that can be used to assess target engagement, proof of concept, prognostic and predictive outcomes, surrogate endpoints, and safety. We apply innovative studies that include alternative and flexible study designs and use data to decide on how to modify aspects of the study without undermining the productivity of R&D. We also incorporate digital technology to support drug development.
  4. Global Populations
    We intend to focus on sub-group patient populations such as pediatrics, geriatrics, women of childbearing age, etc.. They are normally not considered in core development programs due to high-risk medical considerations. At the same time, we want to include less-defined markets (e.g. Japan, China, Brazil, and Russia) earlier in the development process, in order to expedite patient access to therapeutics in these regions.

Our R&D focus is very clear: to get the right molecule to the right patient for the right indication.

Our pipeline builds the basis of UCB’s future hence we invested 25% of revenue into R&D in 2018. We focus on breakthrough innovative approaches with the goal of developing new highly differentiated solutions that will significantly impact the lives of patients. We have developed a unique partnership with patients at every step of the clinical development process to identify needs and inform study design and operations.

We connect with them to get their insights and experiences which lead us to a deeper understanding of their needs. Moreover, building on recent advances in human biology, genetics and biomarkers and big data, we are working on new ways to scientifically identify sub-group patient populations so that we can better predict which patients will respond to our medicines.

We then leverage our internal scientific expertise, our proprietary technology platforms and external network of internationally renowned scientists and academics to identify the next generation of breakthrough candidates. We set up clear milestones that enable us to make robust data-driven decisions. We aim for a strong signal - positive or negative - so we can rapidly advance promising molecules into innovative therapies or stop unviable options and reallocate resources within our pipeline.

We will only progress molecules in-house if we can have the biggest impact in our core therapeutic areas: immunology and neurology. We might not bring all projects to the market and may decide at some point to partner with external parties/organizations to maximize project potential and reach as many patients as possible. For a company of our size, it is vital to remain focused.

Changes since February 2018:

  • Early 2018, UCB and partner Vectura decided to license out UCB4144/VR942
  • UCB0107 first in human (March 2018)
  • midazolam acquired from Proximagen (April 2018) and filed (August 2018)
  • seletalisib in Sjögren’s Syndrome and APDS deprioritized (July 2018)
  • End 2018, radiprodil (UCB3491) in infantile spasm was terminated due to lack of patients for recruitment – driven by sufficient standard of care. UCB6673 was returned to the partner – due to prioritization within the UCB pipeline.
  • Evenity™ (romosozumab) appropval in Japan (Jan. 2019)

CIDP: Chronic Inflammatory Demyelinating Polyneuropathy

Evenity™ is the trade name of romosozumab which has been provisionally approved by the U.S. Food & Drug Administration (FDA) and the European Medicines Agency (EMA). CIDP: Chronic inflammatory demyelinating polyneuropathy

Here are the major milestones we reached or plan to reach:

Grow & Prepare
(2015-2018)

Evenity™ (romosozumab)
midazolam
  • acquisition and filing (2018)
bimekizumab
dapirolizumab pegol
padsevonil
  • Phase 2a completed (2015 – 2017)
  • Phase 2b start (2018)
seletalisib
  • Phase 2a start in Sjögren’s Syndrome (2015)
  • Phase 1 start in APDS (2016)
  • deprioritization (2018)
rozanolixizumab
  • proof of concept achieved in myasthenia gravis (2017 – 2018)
  • proof of concept achieved in immune thrombocytopenia (2016-2018)
Phase 1 projects
  • radiprodil (UCB3491) Phase 1 (2016-2018)
  • UCB0107 (anti-Tau) entered Phase 1 (2018)

We stayed focus, invested in and divested some activities

  • UCB6352 out-license to Syndax

Accelerate & Expand
(2019-2021)

Evenity™ (romosozumab)
  • approval (Japan – Jan 2019)
  • regulatory decision
    (EU & U.S. – Q2 2019)
  • launch in various countries across the world
midazolam
  • regulatory decision in acute repetitive seizure (U.S. – Q2 2019)
padsevonil
  • Phase 3 start (2019)
  • Phase 2b results in drug resistant epilepsy (H1 2020)

We strengthen our R&D to deliver new innovative compounds in shorter cycle time

Breakthrough & Lead
(2022-2025)

  • We broaden patient access to Evenity™ and midazolam
  • We hope to bring bimekizumab, padsevonil and rozanolixizumab to patients
  • We deliver breakthrough solutions