UCB Accelerates Anti-FcRn Rozanolixizumab in Myasthenia Gravis Into Confirmatory Development Phase

Brussels (ots/PRNewswire) -

- Positive outcomes in proof-of-concept study with subcutaneous
rozanolixizumab in patients with myasthenia gravis (MG): clinically
meaningful improvement in multiple disease-related endpoints
- Strong 68% mean reduction of Serum IgG and IgG Autoantibodies
observed
- Safety profile in-line with subcutaneous dosing in phase 1 and the
safety profile observed in the proof-of-concept immune
thrombocytopenia (ITP) study
- Proof-of-concept has been achieved in myasthenia gravis
- Confirmatory development study with rozanolixizumab in patients
with myasthenia gravis to start in H2 2019

UCB today announced positive results from a phase 2 study (MG0002;
NCT03052751) with a novel, subcutaneous FcRn (neonatal Fc receptor)
monoclonal antibody, rozanolixizumab, in patients with myasthenia
gravis (MG), achieving proof-of-concept. Based on these results UCB
intends to accelerate the development of rozanolixizumab with a
confirmatory study in MG starting in the second half of 2019.

Professor Vera Bril, MD, University of Toronto, coordinating
investigator for the MG0002 study said: "I am very excited about
these positive results with subcutaneous rozanolixizumab. Today,
there is a clear need for safe, effective, non-invasive,
non-burdensome therapies for patients with generalized MG, who
continue to face serious, potentially life-threatening symptoms
associated with their disease."

Dr Dhaval Patel, Executive Vice President and Head of NewMedicines
at UCB said: "First I would like to express my sincere thanks to the
patients, investigators, care partners and all those who contributed
to this important study. The results strengthen our conviction that
reducing pathogenic autoantibodies with the most advanced
subcutaneous anti-FcRn therapy in clinical development may offer an
innovative approach to improve outcomes and treatment experience for
patients with myasthenia gravis. In addition, the results give rise
to the expectation of potential therapeutic benefit in other IgG
autoantibody-mediated conditions."

Full data from MG0002 show that subcutaneous infusions of
rozanolixizumab were safe and well tolerated and resulted in clinical
improvement over the entire duration of the study. Clinical benefits
were observed across several pre-specified disease-related endpoints,
including Quantitative Myasthenia Gravis (QMG) score, Myasthenia
Gravis Composite (MGC) responder rate and Myasthenia
Gravis-Activities of Daily living (MG-ADL) score.

Building on the potential clinical utility of rozanolixizumab in
other neurological conditions driven by pathogenic immunoglobulin G
(IgG) autoantibodies, UCB will initiate a phase 2 study in patients
with chronic inflammatory demyelinating polyneuropathy (CIDP) in the
first quarter 2019. UCB is also advancing development in immune
thrombocytopenia (ITP), with results from an on-going dose ranging
study expected at the end of 2018.

Study Design: MG0002 (NCT03052751) was a Phase 2 randomized,
placebo-controlled, proof-of-concept trial that enrolled 43 MG
patients from North America and Europe with generalized muscle
weakness and a total QMG of at least 11. MG0002 compared three
once/week subcutaneous infusions of placebo (N=22) and 7 mg/Kg
rozanolixizumab (N=21) on days 1, 8, 15 and were compared during a
four-week period (dosing period 1).

After dosing period 1, participants were re-randomized to receive
either 7 mg/Kg or 4 mg/Kg rozanolixizumab on days 29, 36, and 43
(dosing period 2) with continued observation until day 99.
Conventional therapies were allowed and included corticosteroids
and/or immunomodulatory agents and/or Cholinesterase inhibitors. The
protocol included no specific headache prophylaxis and mandated
withdrawal of patients with severe headache. Pre-specified analyses
of safety and efficacy across both dosing periods looked at the data
following six subcutaneous infusions of rozanolixizumab.

Study Results: At the end of dosing period 1: The
baseline-corrected delta in QMG between rozanolixizumab and placebo
was -0.7 (p = 0.221), the baseline-corrected delta in MGC score was
-1.8 (p = 0.089) and the baseline-corrected delta in MG-ADL score was
-1.4 (p = 0.036). In a post-hoc analysis, the absolute change from
baseline in the MG-ADL score, an established registration endpoint,
was -2.0 for rozanolixizumab compared to -0.18 for placebo (p =
0.008).

The QMG responder rate was 38.1% compared to 22.7% for placebo
(p=0.223), the MGC responder rate was 47.6% compared to 27.3% for
placebo (p=0.144), and the MG-ADL responder rate was 47.6% compared
to 13.6% for placebo (p=0.017). Response was defined as a reduction
of 3 or more points from baseline for all scores.

During dosing period 2, additional clinically meaningful
reductions of all scores were observed. Pre-specified analyses across
the two dosing periods (i.e. following six subcutaneous infusions of
rozanolixizumab) showed clinically meaningful patient benefit
consistently across several disease-specific endpoints, including
QMG, MGC and MG-ADL. Participants on active treatment showed a marked
reduction of total IgG levels and IgG autoantibody levels. Serum IgG
concentrations reduced by 56% after two weeks of rozanolixizumab
treatment. Total IgG and anti-acetylcholine receptor (anti-AChR)
antibodies decreased by 68% from baseline during dosing period 2 in
participants receiving rozanolixizumab 7 mg/Kg in both dosing
periods.

Safety profile: Safety and tolerability of rozanolixizumab were
confirmed and in-line with subcutaneous dosing in the phase 1 program
and the safety profile observed in the proof of concept ITP study.
There was an expected greater frequency of headache (57.1%) compared
to placebo (13.6%) during dosing period 1. Per protocol, three
rozanolixizumab-treated participants with headache were withdrawn
from the study. All headaches were manageable and resolved with
standard therapies. The incidence of infections between
rozanolixizumab and placebo was similar.

The full data will be presented at a medical congress in the near
future and submitted for publication in a peer-reviewed journal.

Rozanolixizumab is a novel, subcutaneous anti-FcRn monoclonal
antibody in clinical development at UCB and not approved in any
region of the world.

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. With more than 7 500 people in approximately 40
countries, the company generated revenue of EUR 4.5 billion in 2017.
UCB is listed on Euronext Brussels (symbol: UCB). Follow us on
Twitter: @UCB_news

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ots Originaltext: UCB Pharma
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Contact:
France Nivelle
Global Communications
UCB
T +32-2-559-9178
france.nivelle@ucb.com. Laurent Schots
Global Communications
UCB
T +32-2-559-92-64
laurent.schots@ucb.com. UCB Investor Relations: Antje Witte
Investor Relations
UCB
T +32-2-559-94-14
antje.witte@ucb.com. Neil Wallace
Investor Relations
UCB
T +32-2-386-2869
neil.wallace@ucb.com

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