UCB and Biogen Announce Positive Topline Results From Phase 3 Study of Dapirolizumab Pegol in Systemic Lupus Erythematosus and are Initiating Second Phase 3 Study in 2024

• Phase 3 PHOENYCS GO study met the primary endpoint demonstrating clinical improvement in moderate-to-severe systemic lupus erythematosus; Clinical improvements were observed among key secondary endpoints measuring disease activity and flares.
• UCB and Biogen are advancing dapirolizumab pegol with the objective to address the substantial unmet medical need for people living with SLE, where there are limited treatment options.
• SLE is a chronic, debilitating autoimmune disease that affects multiple organ systems and disproportionately affects women.

BRUSSELS and CAMBRIDGE, Mass., Sept. 24, 2024 (GLOBE NEWSWIRE) -- UCB (Euronext Brussels: UCB) and Biogen Inc. (NASDAQ: BIIB) today announced positive topline results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol, a novel Fc-free anti-CD40L drug candidate, in people living with moderate-to-severe systemic lupus erythematosus (SLE). Dapirolizumab pegol, in addition to standard-of-care (SOC) treatment, met the primary endpoint to demonstrate greater improvement of moderate-to-severe disease activity as assessed by achievement of British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) after 48 weeks versus placebo in addition to SOC. Clinical improvements were observed among key secondary endpoints measuring disease activity and flares.

The safety profile of dapirolizumab pegol was generally consistent with previous studies and with that expected in study participants with systemic lupus erythematosus receiving an immunomodulator.

"These positive results with dapirolizumab pegol represent encouraging progress in the development of medicines that can improve the lives of those living with lupus, an area that remains one of high unmet medical need and where women are disproportionately affected,” said Fiona du Monceau, Head of Patient Evidence at UCB. "We have confidence in the unique mode of action of dapirolizumab pegol which targets multiple inflammatory pathways involved in the pathogenesis of SLE. As we pursue the next steps in the clinical development of this potentially differentiated treatment, we extend our appreciation to the patients, study investigators and the clinical community for their ongoing support and participation in this important research.”

Based on the successful outcome of the PHOENYCS GO study, UCB and Biogen are initiating a second Phase 3 trial of dapirolizumab pegol in 2024, PHOENYCS FLY. Participants from the PHOENYCS GO study will continue to be followed in a long-term open-label study.

"Our hypothesis is that impacting the CD40L pathway, a central mechanism in immune response, would translate to significant impact on SLE disease burden. These results demonstrate that dapirolizumab pegol has the promise to provide meaningful benefit in this serious, chronic, and often devastating disease,” said Diana Gallagher, MD, Head of AD, MS and Immunology Development Units at Biogen. "We are committed to delivering new treatment options for this autoimmune disease and believe the overall efficacy and safety seen in PHOENYCS GO support further development of dapirolizumab pegol in SLE.”

PHOENYCS GO (n= 321) is a multicenter, randomized, double-blind, placebo-controlled, parallel-group study of dapirolizumab pegol as an add on therapy to standard of care compared to placebo with standard of care. The primary outcome measure was improvement of moderate-to-severe disease activity at Week 48 using BICLA, an established, composite primary efficacy endpoint for measurement of clinical disease activity based on patient medical history, clinical examination and laboratory tests.

Detailed results from the PHOENYCS GO study will be presented at an upcoming medical congress.

About Systemic Lupus Erythematosus (SLE)
SLE, the systemic form of lupus, is a chronic, multifactorial autoimmune disease that can affect multiple organ systems with periods of illness or flares alternating with periods of inactivity.¹ SLE can present itself in several ways including rash, arthritis, anemia, thrombocytopenia, serositis, nephritis, seizures or psychosis.² SLE is associated with a greater risk of death from causes such as infection and cardiovascular disease.

An estimated 90 percent of people living with lupus are women; most begin to see symptoms between the ages of 15-55.^3,4,5 Individuals from populations of African, Hispanic, Asian and Native American descent are at a greater risk of earlier onset and more aggressive disease.^6,7 Pregnancy in women with SLE is high risk, with higher maternal and fetal mortality and morbidity than the general population.^8,9

About Dapirolizumab Pegol
Dapirolizumab pegol is a novel investigational humanized Fc-free polyethylene glycol (PEG)-conjugated antigen-binding (Fab’) fragment. Dapirolizumab pegol inhibits CD40L signaling which has been shown to reduce B cell activation and autoantibody production, mitigate type 1 interferon (IFN) secretion, and attenuate T cell and antigen-presenting cell (APC) activation.¹⁰ Dapirolizumab pegol is presently in Phase 3 clinical development for the treatment of systemic lupus erythematosus (SLE) under a collaboration between UCB and Biogen.¹¹

About UCB 

UCB, Brussels, Belgium (http://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. UCB is listed on Euronext Brussels (symbol: UCB).

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient’s lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

The company routinely post information that may be important to investors on its website at www.biogen.com. Follow us on social media - Facebook, LinkedIn, X, YouTube.

Forward looking statements UCB
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References:

1. Tselios K, Gladman DD, Touma Z, et al. Disease course patterns in systemic lupus erythematosus. Lupus. 2019;28(1):114-122.
2. Fanouriakis A, Tziolos N, Bertsias G, et al. Update ?n the diagnosis and management of systemic lupus erythematosus. Ann Rheum Dis. 2021;80(1):14-25. doi:10.1136/annrheumdis-2020-218272
3. Petri M. Epidemiology of systemic lupus erythematosus. Best Pract Res Clin Rheumatol. 2002;16(5):847-58. Epub 2002/12/11. doi: 10.1053/berh.2002.0259. PubMed PMID: 12473278.
4. Rees F, Doherty M, Grainge M, Davenport G, Lanyon P, Zhang W. The incidence and prevalence of systemic lupus erythematosus in the UK, 1999-2012. Ann Rheum Dis. 2016;75(1):136-41. Epub 2014/10/01. doi: 10.1136/annrheumdis-2014-206334. PubMed PMID: 25265938; PubMed Central PMCID: PMCPMC4717400.
5. Pons-Estel GJ, Ugarte-Gil MF, Alarcón GS. Epidemiology of systemic lupus erythematosus. Expert Rev Clin Immunol. 2017;13(8):799-814.
6. Carter EE, Barr SG, Clarke AE. The global burden of SLE: prevalence, health disparities and socioeconomic impact. Nat Rev Rheumatol. 2016;12(10):605-20. Epub 2016/08/26. doi: 10.1038/nrrheum.2016.137. PubMed PMID: 27558659.
7. Kheir JM, Guthridge CJ, Johnston JR, Adams LJ, Rasmussen A, Gross TF, et al. Unique clinical characteristics, autoantibodies and medication use in Native American patients with systemic lupus erythematosus. Lupus Sci Med. 2018;5(1):e000247. Epub 2018/03/14. doi: 10.1136/lupus-2017-000247. PubMed PMID: 29531773; PubMed Central PMCID: PMCPMC5844376.
8. Mehta B, Luo Y, Xu J, Sammaritano L, Salmon J, Lockshin M, et al. Trends in Maternal and Fetal Outcomes Among Pregnant Women With Systemic Lupus Erythematosus in the United States: A Cross-sectional Analysis. Ann Intern Med. 2019;171(3):164-71. Epub 2019/07/10. doi: 10.7326/M19-0120. PubMed PMID: 31284305.
9. Bitencourt N, Bermas BL. Pharmacological Approach to Managing Childhood-Onset Systemic Lupus Erythematosus During Conception, Pregnancy and Breastfeeding. Paediatr Drugs.
10. Furie RA, Bruce IN, Dörner T, et al. Phase 2 randomized, placebo-controlled trial of dapirolizumab pegol in patients with moderate to severe active systemic lupus erythematosus (SLE). Rheumatology (Oxford).2021;60(11): 5397-407.
11. ClinGov.gov (NCT04294667). A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus (PHOENYCS GO) 2023 [cited August 2024] Available at: https://clinicaltrials.gov/ct2/show/NCT04294667. Retrieved July 25, 2024.