UCB Presents Final Results from Phase II Study of Rozanolixizumab in Primary Immune Thrombocytopenia (ITP) at 2019 ASH Annual Meeting

Brussels (ots/PRNewswire) - - Phase II data demonstrate that rozanolixizumab was
well tolerated by patients with primary ITP across all dose groups

- Clinically relevant improvements in platelet count and decrease in
immunoglobin G (IgG) levels were observed in all dose groups

- Safety, tolerability and efficacy data support Phase III development of
rozanolixizumab for primary ITP

- Rozanolixizumab's subcutaneous route of administration could provide a new
treatment option for patients with primary ITP

UCB, a global biopharmaceutical company, today announced positive results from a
Phase II study (TP0001; NCT02718716) of its novel, first-in-class subcutaneous
(SC, under the skin) monoclonal antibody, rozanolixizumab, in patients with
primary immune thrombocytopenia (ITP). The data were presented during an oral
presentation today at the 61st American Society of Hematology (ASH) Annual
Meeting & Exposition in Orlando, Florida.

The Phase II TP0001 study to assess the safety, tolerability and efficacy of
rozanolixizumab was designed to explore a multiple dose regimen in order to
inform the dosing strategy for further development in ITP. Sixty-six patients
received either a single dose (1 x 15 mg/kg or 1 x 20 mg/kg) or multiple doses
(5 x 4 mg/kg, 3 x 7 mg/kg, 2 x 10 mg/kg weekly) of SC rozanolixizumab. The total
weekly dose was similar in all treatment groups, ranging from 15 to 21 mg/kg.

Clinically relevant improvements (i.e., reaching >=50x109/L) in platelet count
and decreases in immunoglobin G (IgG) levels were observed across all dose
groups, with higher response rate (55-67% in 1 x 15 mg/kg and 1 x 20 mg/kg dose
groups vs 36-45% in 5 x 4 mg/kg, 3 x 7 mg/kg and 2 x 10 mg/kg dose groups) and
shorter time to response achieved by the 1 x 15 mg/kg and 1 x 20 mg/kg
rozanolixizumab dose groups.i

Results confirm that rozanolixizumab was well tolerated across all dose
groups,[i] consistent with previous rozanolixizumab studies.[ii],[iii] The most
commonly reported adverse event was headache, with mild-to-moderate headaches
seen at higher doses; other reported adverse events included diarrhea and
vomiting. These events were usually of short duration and the majority of events
resolved without treatment. No patient discontinued the study due to side
effects.i

"ITP is a severe, often chronic disease that can have a significant, long-term
impact on people's health and quality of life. Despite approved therapies, there
is still an urgent need for new treatment options that are well tolerated and
provide a sustained increase in platelet count," said Professor Tadeusz Robak,
Professor of Hematology at the Medical University of Lodz, Poland. "These Phase
II results for rozanolixizumab suggest the treatment could reduce IgG
autoantibody levels and improve platelet count for people living with primary
ITP."

Individuals living with ITP experience unpredictable and debilitating symptoms
including spontaneous bruising, bleeding and fatigue that can greatly impact
their activities of daily life.[iv] Additionally, the limited current treatment
options for people with ITP can be time-consuming and invasive. There is a need
to discover new solutions that have the potential to improve patients' health
outcomes and quality of life. Rozanolixizumab is an advanced SC anti-neonatal Fc
receptor (FcRn) therapy currently in clinical development and has the potential
to provide a targeted, convenient option to optimize individualized patient
care.

"Our research has enabled us to better understand rare, IgG
autoantibody-mediated diseases such as ITP, including where gaps exist within
the treatment paradigm and the overall patient experience," said Dr. Iris
Loew-Friedrich, Chief Medical Officer, Executive Vice-President, UCB. "These
results reaffirm our belief that targeting the neonatal Fc receptor pathway
could have the potential to transform the treatment experience for people with
ITP. We look forward to expanding this knowledge in Phase III trials in ITP and
other patient populations."

About the rozanolixizumab clinical studyi

TP0001 (NCT02718716) is a Phase II, multi-center, open-label, multiple-dose
study of rozanolixizumab in patients with primary ITP. Sixty-six patients were
assigned to one of five groups with different dosing regimens (5 x 4 mg/kg, 3 x
7 mg/kg, 2 x 10 mg/kg weekly, 1 x 15 mg/kg or 1 x 20 mg/kg), receiving
rozanolixizumab by SC infusion (multiple doses were administered at weekly
intervals). All patients were monitored for an 8-week observation period after
completion of treatment. The primary objective of the study assessed safety and
tolerability of rozanolixizumab administered by SC infusion, and the secondary
objective considered the clinical efficacy (platelet count) and pharmacodynamic
(total IgG) effects. The study was designed to explore a range of therapeutic
doses in order to develop an appropriate dosing regimen for patients with ITP.

Rozanolixizumab was well tolerated across all dose groups (4-20 mg/kg) with
mild-to-moderate headaches seen at higher doses; no patient discontinued the
study due to side effects.

In the study, clinically relevant improvements in platelet count (to >=50x109/L)
were observed in patients with primary ITP receiving rozanolixizumab across all
dose groups and decreases in serum IgG concentration were observed. By day 8,
more patients receiving a single, higher-dose infusion achieved platelet counts
of >=50x109/L (58.3% and 54.5% in the 1 x 15 mg/kg and 1 x 20 mg/kg dose groups,
respectively) compared with patients in the multiple-dose cohorts (7.1%, 14.3%
and 27.3% in the 5 x 4 mg/kg, 3 x 7 mg/kg, and 2 x 10 mg/kg groups,
respectively). A faster time to response was also observed in the 1 x 15 and 1 x
20 mg/kg dose groups (median 7 days and 5 days, respectively), compared with
patients in the multiple-dose cohorts (14 days in both the 5 x 4 mg/kg and 3 x 7
mg/kg groups and 8 days in the 2 x 10 mg/kg group) following treatment.

About primary immune thrombocytopenia

Primary ITP is an acquired autoimmune disorder characterized, in most cases, by
the presence of pathogenic IgG autoantibodies, with an estimated prevalence of
approximately 10 people per 100,000 (USA)[v]. Pathogenic IgG autoantibodies
target platelets and megakaryocytes (platelet precursors), leading to the
removal and destruction of both circulating and newly formed
platelets[vi],[vii],[viii] ultimately resulting in a propensity for bleeding in
patients with ITP. The standard of care for patients with newly diagnosed ITP
consists of corticosteroids or intravenous immunoglobulin (IVIg).[ix] Patients
intolerant to corticosteroids or with contraindications are treated with IVIg or
anti-D (where appropriate), either alone or in combination. Subsequent
treatments include immunosuppressive agents (e.g., azathioprine and
mycophenolate mofetil), rituximab, TPO receptor agonists (e.g., eltrombopag and
romiplostim) or splenectomy.[x]

About rozanolixizumab

Rozanolixizumab is a subcutaneously administered, humanized monoclonal antibody
that specifically binds, with high affinity, to human FcRn. It has been designed
to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing
the removal of pathogenic IgG autoantibodies.[ii],[xi]

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.[xii],[xiii] The
safety and efficacy of rozanolixizumab has not been established; it is not
currently approved by any regulatory authority worldwide.

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 7,500 people in approximately 40
countries, the company generated revenue of EUR4.6 billion in 2018. UCB is
listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news

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pricing and reimbursement.

References

[i] Robak, T., Kasmierczak, M., Jarque, I. et al. (2019). Rozanolixizumab, an
anti-FcRn Antibody: Final results from a phase II, multiple-dose study in
patients with primary immune thrombocytopenia. Blood. 134(1)

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

[iii] Bril, V., M. Benatar, M. Brock 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):pS43.001

[iv] Kohli, R. and S. Chaturvedi (2019) Epidemiology and Clinical Manifestations
of Immune Thrombocytopenia. Hamostaseologie(EFirst)

[v] National Organization for Rare Disorders (NORD). Immune thrombocytopenia.
Retrieved from: https://rarediseases.org/rare-diseases/immune-thrombocytopenia/
Accessed December 2019

[vi] Cortelazzo, S., G. Finazzi, M. Buelli, A. Molteni, P. Viero, and T. Barbui
(1991) High risk of severe bleeding in aged patients with chronic idiopathic
thrombocytopenic purpura. Blood. 77(1):p31-3

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

[viii] Chan, H., J.C. Moore, C.N. Finch, T.E. Warkentin, and J.G. Kelton (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

[ix] Neunert, C., W. Lim, M. Crowther et al. (2011) The American Society of
Hematology 2011 evidence-based practice guideline for immune thrombocytopenia.
Blood. 117(16):p4190-207

[x] Provan, D., R. Stasi, A.C. Newland et al. (2010) International consensus
report on the investigation and management of primary immune thrombocytopenia.
Blood. 115(2):p168-86

[xi] 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

[xii] U.S. Food and Drug Administration (FDA). (2018). Orphan drug designations
and approvals. Retrieved from: https://www.accessdata.fda.gov/scripts/opdlistin
g/oopd/detailedIndex.cfm?cfgridkey=636618 Accessed December 2019

[xiii] European Medicines Agency (EMA). (2019). Public summary of opinion on
orphan designation. Retrieved from:
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182131
Accessed December 2019

Contact:
+32-2-473-78-85-01
jim.baxter@ucb.com / Laurent Schots
Media Relations
(Global) UCB
T: +32-2-559-92-64
laurent.schots@ucb.com / Ally Funk
U.S. Communications
UCB
T: +1-770-970-8338
ally.funk@ucb.com / Investor Relations

Antje Witte
Investor Relations
UCB
T: +32-2-559-94-14
antje.witte@ucb.com / Isabelle Ghellynck
Investor Relations
UCB
T: +32-2-559-95-8
isabelle.ghellynck@ucb.com

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