1.2 Key events1

There have been a number of key events that have affected or will affect UCB financially:

1.2.1 Important agreements/initiatives

  • February 2018 – UCB and an investor syndicate led by Novo Seeds launched Syndesi Therapeutics to develop novel therapeutics for cognitive disorders. Syndesi Therapeutics has exclusively licensed a first-in-class small molecule program from UCB. A series A investment totaling € 17 million will fund the clinical development of the lead compound up to early proof-of-concept in humans.
  • Early 2018, UCB and partner Vectura decided to license out UCB4144/VR942, a dry powder inhaled biologic which successfully completed Phase 1 in 2017.
  • March 2018 – UCB acquired Element Genomics in the U.S. to strengthen UCB’s genomics and epigenomics research platform to identify novel drug targets.
  • April 2018 – UCB agreed to acquire midazolam nasal spray (USL261) from Proximagen. USL261 is a nasally administered investigational midazolam formulation intended as a rescue treatment of acute repetitive seizures in patients with epilepsy. Closing occurred in June 2018. The new drug application was accepted for filing by the FDA in August, following previous orphan drug status and fast-track designation.
  • May 2018 – UCB has entered into an agreement with Science 37, Los Angeles, CA (U.S.), a trailblazing company focused on “site-less” clinical trials. Science 37’s decentralized clinical trial approach combines technologies that can fundamentally change the way clinical trials are run. With this collaboration, UCB aims to provide a better patient experience, to innovate and accelerate clinical studies in a patient-focused way and to bring new solutions to patients faster.
  • May 2018 – The U.S. Court of Appeals for the Federal Circuit (CAFC) has affirmed the Delaware District Court and confirmed the validity of U.S. patent RE38,551 related to Vimpat® (lacosamide), UCB’s anti-epileptic drug.
  • In September, in line with its strategic focus, UCB sold its subsidiary “Innere Medizin”. “Innere Medizin” has been successfully promoting pharmaceutical products in Germany for many years, mainly in the internal medicine area for cardiovascular and respiratory diseases.

We must increase awareness, we must increase understanding, and we must hope the medical community continues to search for the treatments that can help us all.

Sander, living with lupus

1.2.2 Regulatory update and pipeline progress

Neurology

  • In January 2018, UCB filed Vimpat® (lacosamide) for pediatric patients living with partial-onset epilepsy at four years and older in Japan.
  • In February, the Phase 2b study with padsevonil started for drug resistant epilepsy patients. First results are expected in H1 2020.
  • In March, UCB0107, a humanized, immunoglobulin monoclonal antibody with a specificity for human tau, entered the clinical phase 1 program.
  • In May, Briviact® (brivaracetam) oral formulations were approved in the U.S. indicated as monotherapy and adjunctive therapy in the treatment of partial onset (focal) epileptic seizures in patients age four years and older.
  • In June, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for Briviact® to extend the therapeutic indication to include adjunctive therapy in the treatment of partial onset seizures with or without secondary generalization in patients with epilepsy from 4 years of age. The European Commission approved this in July.
  • In August, the new drug application for midazolam nasal spray was accepted for filing by the FDA, following previous orphan drug status and fast-track designation.
  • Positive phase 2 results for Briviact® (brivaracetam) in acute repetitive seizures were achieved in July.
  • UCB pioneered with the extrapolation concept in China: in March 2018 UCB filed Keppra® (levetiracetam) for monotherapy of partial onset epilepsy seizures based on extrapolation from adjunctive therapy with sound scientific rationale and was approved in August. In September, UCB submitted Vimpat® (lacosamide) IV (intravenous) and oral formulation for the adjunctive therapy of partial onset epilepsy seizures in children above 4 years and for adults, based on extrapolation.
  • In October, UCB announced positive results from a phase 2 study with a novel, subcutaneous FcRn (neonatal Fc receptor) monoclonal antibody, rozanolixizumab, in patients with myasthenia gravis (MG), achieving proof-of-concept. These results support the acceleration of rozanolixizumab development with a confirmatory study in MG starting in Q2 2019.
  • In December, Vimpat® (lacosamide) was approved in China as adjunctive therapy in the treatment of partial-onset seizures with or without secondary generalization in adult and adolescent patients 16 years of age and older with epilepsy. In January 2019, Vimpat® was approved in Japan for the treatment of partial onset seizures in children 4 years of age and older. In addition, two new formulations have been approved, IV (intravenous) and dry syrup.
  • In December, Keppra® (levetiracetam) for monotherapy of epilepsy as well as an updated pregnancy language was submitted to the U.S. authorities. The application was accepted for filing by the FDA in January 2019. The Keppra® pregnancy label has been approved in the EU in April 2018.
  • At the end of 2018, one phase 1 project in neurology, UCB3491, was terminated due to lack of patients for recruitment – driven by sufficient standard of care.

Immunology

  • A label update for Cimzia® (certolizumab pegol) in pregnancy and breastfeeding was approved in Europe (January 2018) and in the U.S. (March 2018), making it the first anti-TNF treatment option that could be considered for women with chronic inflammatory disease throughout the pregnancy journey.
    In March 2018, the Cimzia® pre-filed syringe received approval in the U.S. for the option to store it at room temperature for a single period of up to 7 days, within the approved shelf-life, thus helping better address patient needs.
    Also in March, UCB announced the filing of Cimzia® with the State Drug Administration (SDA, former CFDA) in China for the treatment of moderate-to-severe rheumatoid arthritis. In June, the SDA has granted priority review.
    In April, the European Committee for Medicinal Products for Human Use (CHMP) recommended approval of a label extension for Cimzia®, to include a new indication in adult patients with moderate-to-severe plaque psoriasis. The European Commission endorsed this in June.
    In May, Cimzia® was approved for adults with moderate-to-severe plaque psoriasis in the U.S.
    Also in May, UCB announced positive topline results from C-AXSPAND, a Phase 3 placebo-controlled study to investigate the efficacy of Cimzia® on the signs and symptoms of active axial spondyloarthritis (axSpA) in patients without x-ray evidence of ankylosing spondylitis (AS). In September, these data were submitted to the U.S. regulatory authorities for non-radiographic axial spondyloarthritis (nr-axSpA) and were accepted for filing in October. In August, the Japanese authorities approved the Cimzia® AutoClick® device. In September, the label update for Cimzia® in pregnancy and breastfeeding was approved in Japan. Also in September and in Japan, positive phase 3 results were achieved for Cimzia® in patients with psoriasis and psoriatic arthritis. Submission to the Japanese agency took place in January 2019.
  • During the course of the first half of 2018, further studies with bimekizumab in moderate to severe psoriasis were initiated. Out of the ongoing three Phase 3 studies, two include an active comparator, namely ustekinumab, and adalimumab. Results are expected by the end of 2019. An additional Phase 3b study to compare bimekizumab directly with secukinumab was initiated in June. The comparative studies have been designed to demonstrate superiority over active comparators on robust endpoints.
  • In July, a full evaluation of early-stage clinical studies of seletalisib in Sjögren’s syndrome and activated P13K Delta Syndrome (APDS) showed positive results and no new safety signal was observed. However, in light of its other upcoming R&D investments and as part of its regular portfolio prioritization, UCB has decided to deprioritize further internal development of seletalisib.
  • In October, UCB and its partner Biogen announced top-line results from a Phase 2b study with dapirolizumab pegol (DZP) in moderately-to-severely active systemic lupus erythematosus. UCB and Biogen continue to further evaluate these data while assessing potential next steps.
  • At the end of 2018, one phase 1 project, UCB6673, was returned to the partner – due to prioritization within the UCB pipeline.

Bone

  • Early January 2019, UCB and Amgen announced the approval of Evenity™ (romosozumab) in Japan. Evenity™ is approved in Japan to reduce the risk of fractures and increase bone mineral density in men and post-menopausal women with osteoporosis at high risk of fracture. One week later, the U.S. Food and Drug Administration (FDA) Bone, Reproductive and Urologic Drugs Advisory Committee (BRUDAC) voted positively for the approval of romosozumab. While the FDA is not bound by the Advisory Committee’s recommendations, it takes the advice into consideration when making its decision. The European Medicines Agency (EMA) is currently reviewing a marketing application for romosozumab and interactions with the agency are ongoing.

All other clinical development programs are continuing as planned.

1 From 1 January 2018 up to the publication of date of this report