Publicado 09/09/2020 7:01:24 +02:00CET

Final Phase II Results for UCB's Rozanolixizumab in Primary Immune Thrombocytopenia (ITP) Published in Blood Advances (2

Rozanolixizumab is under clinical development with the aim of improving the lives of people with pathogenic IgG-autoantibody-driven autoimmune diseases, including ITP, myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP), by driving removal of pathogenic IgG autoantibodies.

Rozanolixizumab, an investigational monoclonal antibody, was granted orphan drug designation for the treatment of ITP by the US Food and Drug Administration on 30 April 2018 and by the European Commission on 11 January 2019.(12,13) The safety and efficacy of rozanolixizumab has not been established; it is not currently approved by any regulatory authority worldwide.

About UCB in Rare Diseases

At UCB, we don't just see patients or population sizes, we see people in need. Through decades of serving the neurology and immunology communities, we have improved lives with impactful medicines and by enhancing the social and emotional well-being of patients. As a continuation of our heritage, we are now expanding our efforts to tackle rare neurological and immunologic diseases where current options offer little hope, including investigational treatments for primary immune thrombocytopenia (ITP), myasthenia gravis (MG), chronic inflammatory demyelinating polyneuropathy (CIDP) and progressive supranuclear palsy (PSP).

About UCB

UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With 7,600 people in approximately 40 countries, the company generated revenue of EUR4.9 billion in 2019. UCB is listed on Euronext Brussels . Follow us on Twitter: @UCB_news

Forward-looking statements

This press release contains forward-looking statements based on current plans, estimates and beliefs of management. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, political, regulatory or clinical results and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions which could cause actual results to differ materially from those that may be implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, product liability claims, challenges to patent protection for products or product candidates, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws and hiring and retention of its employees.

Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. UCB is providing this information as of the date of this document and expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report a change in its expectations.

There is no guarantee that new product candidates in the pipeline will progress to product approval or that new indications for existing products will be developed and approved. Products or potential products which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences between the partners. Also, UCB or others could discover safety, side effects or manufacturing problems with its products after they are marketed.

Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement.

*Dr. James Bussel is a paid consultant for various companies in the ITP space including Amgen, Argenx, Dova, Johnson and Johnson, Momenta, Principia, Rigel and UCB.

References

1. Robak T., Kazmierczak M., Jarque I., Musteata V., Trelinski J. et al. Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia. Blood advances 2020; 4(17):4136-46

2. Kohli, R, Chaturvedi, S (2019) Epidemiology and Clinical Manifestations of Immune Thrombocytopenia. Hamostaseologie 2019; 39(3):238-249

3. Kiessling, P, Lledo-Garcia, R. S, Watanabe et al. (2017) The FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A randomized phase 1 study. Sci Transl Med. 9(414)

4. Bril, V, Benatar, M. Brock, M et al. (2019) Proof-of-Concept and Safety of the Anti-FcRn Antibody Rozanolixizumab in Patients with Moderate-to-Severe Generalized Myasthenia Gravis (GMG): A Phase 2a Study. Neurology (Abstracts: AAN 71th Annual Meeting, Philadelphia) Neurology 2019; 92(15 Suppl.): abs S43.001

5. National Organization for Rare Disorders (NORD). Immune thrombocytopenia. Retrieved from: https://rarediseases.org/rare-diseases/immune-thrombocytopen... [https://rarediseases.org/rare-diseases/immune-thrombocytopen...] Accessed September 2020

6. Cortelazzo, S., Finazzi G., Buelli, M., Molteni, A.,Viero P. and Barbui, T. (1991) High risk of severe bleeding in aged patients with chronic idiopathic thrombocytopenic purpura. Blood. 77(1):p31-

7. Chang, M., Nakagawa P.A., Williams S.A. et al. (2003) Immune thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoantibodies inhibit megakaryocytopoiesis in vitro. Blood. 102(3):p887-95

8. Chan, H., Moore J.C., Finch C.N., Warkentin T.E., and Kelton J.G. (2003) The IgG subclasses of platelet-associated autoantibodies directed against platelet glycoproteins IIb/IIIa in patients with idiopathic thrombocytopenic purpura. Br J Haematol. 122(5):p818-24

9. Neunert, C., Terrell, D.R., Arnold, D.M., et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829-3866.

10. Provan, D., Arnold D.M., Bussel, J.B., et al. (2019) Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. (2019) 3 (22): 3780-3817.

11. Smith B, Kiessling A, Lledo-Garcia R, et al. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs2018;10:1111-30

12. U.S. Food and Drug Administration (FDA). (2018). Orphan drug designations and approvals. Retrieved from: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detai... [https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detai...] Accessed September 2020

13. European Medicines Agency (EMA). (2019). Public summary of opinion on orphan designation. Retrieved from: https://www.ema.europa.eu/en/medicines/human/orphan-designat... [https://www.ema.europa.eu/en/medicines/human/orphan-designat...] Accessed September 2020

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